Existing law, the Federal Food, Drug, and Cosmetic Act, prohibits a person from introducing into interstate commerce any new drug unless the drug has been approved by the United States Food and Drug Administration (FDA). Existing law requires the sponsor of a new drug to submit to the FDA an investigational new drug application and to then conduct a series of clinical trials to establish the safety and efficacy of the drug in human populations and submit the results to the FDA in a new drug application.

Existing law, the Sherman Food, Drug, and Cosmetic Law, regulates the packaging, labeling, and advertising of drugs and devices and is administered by the State Department of Public Health. A violation of that law is a crime. The Sherman Food, Drug, and Cosmetic Law prohibits, among other things, the sale, delivery, or giving away of a new drug or new device unless either the department has approved a new drug or device application for that new drug or new device and that approval has not been withdrawn, terminated, or suspended or the drug or device has been approved pursuant to specified provisions of federal law, including the Federal Food, Drug, and Cosmetic Act.

The Medical Practice Act provides for the licensure and regulation of physicians and surgeons by the Medical Board of California and requires the board to take action against a licensee who is charged with unprofessional conduct. The Osteopathic Act provides for the licensure and regulation of osteopathic physicians and surgeons by the Osteopathic Medical Board of California and requires the board to enforce the Medical Practice Act with respect to its licensees.

This bill, among other things, would permit a manufacturer of an investigational drug, biological product, or device to make the product available to eligible patients with a terminal disease, an immediately life-threatening disease or condition, as specified. The bill would require a manufacturer that provides an investigational drug, biological product, or device to an eligible patient to report specified data to the State Department of Public Health. The bill would provide that the act does not require a health benefit plan, as defined, or governmental agency to provide coverage for the cost of any investigational drug, biological product, or device made available pursuant to these provisions, but would authorize a health benefit plan to provide coverage for an investigational drug, biological product, or device. The bill would also prohibit the Medical Board of California and the Osteopathic Medical Board of California from taking any disciplinary action against the license of a physician based solely on the physicians recommendation to an eligible patient regarding, or prescription for, or treatment with, an investigational drug, biological product, or device. device if the recommendation or prescription is consistent with protocol approved by the physicians institutional review board or an accredited institutional review board, and would require the institutional review board to biannually report specified information to the State Department of Public Health, among others.

Existing law, the federal Federal Food, Drug, and Cosmetic Act, prohibits a person from introducing into interstate commerce any new drug unless the drug has been approved by the United States Food and Drug Administration (FDA). Existing law requires the sponsor of a new drug to submit to the FDA an investigational new drug application and to then conduct a series of clinical trials to establish the safety and efficacy of the drug in human populations and submit the results to the FDA in a new drug application.

Existing law, the Sherman Food, Drug, and Cosmetic Law, regulates the packaging, labeling, and advertising of drugs and devices and is administered by the State Department of Public Health. A violation of that law is a crime. The Sherman Food, Drug, and Cosmetic Law prohibits, among other things, the sale, delivery, or giving away of a new drug or new device unless either the department has approved a new drug or device application for that new drug or new device and that approval has not been withdrawn, terminated, or suspended or the drug or device has been approved pursuant to specified provisions of federal law, including the federal Federal Food, Drug, and Cosmetic Act.

The Medical Practice Act provides for the licensure and regulation of physicians and surgeons by the Medical Board of California and requires the board to take action against a licensee who is charged with unprofessional conduct. The Osteopathic Act provides for the licensure and regulation of osteopathic physicians and surgeons by the Osteopathic Medical Board of California and requires the board to enforce the Medical Practice Act with respect to its licensees.

This bill, among other things, would permit a manufacturer of an investigational drug, biological product, or device to make the product available to eligible patients with a terminal illnesses, disease, as specified. The bill would require a manufacturer that provides an investigational drug, biological product, or device to an eligible patient to report specified data to the State Department of Public Health. The bill would provide that the act does not require a health benefit plan, as defined, or governmental agency to provide coverage for the cost of any investigational drug, biological product, or device made available pursuant to these provisions. The bill provisions, but would authorize a health benefit plan to provide coverage for an investigational drug, biological product, or device. The bill would also prohibit the Medical Board of California and the Osteopathic Medical Board of California from taking any disciplinary action against the license of a physician based solely on the physicians recommendation to an eligible patient regarding, or prescription for, or treatment with, an investigational drug, biological product, or device.

Existing law, the federal Food, Drug, and Cosmetic Act, prohibits a person from introducing into interstate commerce any new drug unless the drug has been approved by the federal United States Food and Drug Administration (FDA). Existing law requires the sponsor of a new drug to submit to the FDA an investigational new drug application and to then conduct a series of clinical trials to establish the safety and efficacy of the drug in human populations and submit the results to the FDA in a new drug application.

Existing law, the Sherman Food, Drug, and Cosmetic Law, regulates the packaging, labeling, and advertising of drugs and devices and is administered by the State Department of Public Health. A violation of that law is a crime. The Sherman Food, Drug, and Cosmetic Law prohibits, among other things, the sale, delivery, or giving away of a new drug or new device unless either the department has approved a new drug or device application for that new drug or new device and that approval has not been withdrawn, terminated, or suspended or the drug or device has been approved pursuant to specified provisions of federal law, including the federal Food, Drug, and Cosmetic Act.

The Medical Practice Act provides for the licensure and regulation of physicians and surgeons by the Medical Board of California and requires the board to take action against a licensee who is charged with unprofessional conduct. The Osteopathic Act provides for the licensure and regulation of osteopathic physicians and surgeons by the Osteopathic Medical Board of California and requires the board to enforce the Medical Practice Act with respect to its licensees.

This bill, among other things, would permit a manufacturer of an investigational drug, biological product, or device to make the product available to eligible patients with terminal illnesses, as specified. The bill would provide that the act does not require a health benefit plan, as defined, or governmental agency to provide coverage for the cost of any investigational drug, biological product, or device made available pursuant to these provisions. The bill would authorize a health benefit plan to provide coverage for an investigational drug, biological product, or device. The bill would also prohibit the Medical Board of California and the Osteopathic Medical Board of California from taking any disciplinary action against the license of a physician based solely on the physicians recommendation to an eligible patient regarding, or prescription for, or treatment with, an investigational drug, biological product, or device.

Existing law, the federal Food, Drug, and Cosmetic Act, prohibits a person from introducing into interstate commerce any new drug unless the drug has been approved by the federal Food and Drug Administration (FDA). Existing law requires the sponsor of a new drug to submit to the FDA an investigational new drug application and to then conduct a series of clinical trials to establish the safety and efficacy of the drug in human populations and submit the results to the FDA in a new drug application.

Existing law, the Sherman Food, Drug, and Cosmetic Law, regulates the packaging, labeling, and advertising of drugs and devices and is administered by the State Department of Public Health. A violation of that law is a crime. The Sherman Food, Drug, and Cosmetic Law prohibits, among other things, the sale, delivery, or giving away of a new drug or new device unless either the department has approved a new drug or device application for that new drug or new device and that approval has not been withdrawn, terminated, or suspended or the drug or device has been approved pursuant to specified provisions of federal law, including the federal Food, Drug, and Cosmetic Act.

The Medical Practice Act provides for the licensure and regulation of physicians and surgeons by the Medical Board of California and requires the board to take action against a licensee who is charged with unprofessional conduct. The Osteopathic Act provides for the licensure and regulation of osteopathic physicians and surgeons by the Osteopathic Medical Board of California and requires the board to enforce the Medical Practice Act with respect to its licensees.

This bill, among other things, would permit a manufacturer of an investigational drug, biological product, or device to make the product available to eligible patients with terminal illnesses, as specified. The bill would provide that the act does not require a health benefit plan, as defined, or governmental agency to provide coverage for the cost of any investigational drug, biological product, or device made available pursuant to these provisions. The bill would authorize a health benefit plan to provide coverage for an investigational drug, biological product, or device. The bill would also prohibit the Medical Board of California and the Osteopathic Medical Board of California from taking any disciplinary action against the license of a physician based solely on the physicians recommendation to an eligible patient regarding, or prescription for, or treatment with, an investigational drug, biological product, or device.

Date of Hearing: August 19, 2015

ASSEMBLY COMMITTEE ON APPROPRIATIONS

Jimmy Gomez, Chair

SBPCA Bill Id:SB 149

Author:(Stone) – As Amended Ver:July 13, 2015

Urgency: No State Mandated Local Program: No Reimbursable: No

SUMMARY:

This bill sets up a legal structure whereby individuals with serious or immediately life-threatening conditions can gain access to investigational drugs, biological products, or medical devices (investigational products). Specifically, this bill:

1. Defines investigational drug, biological product, or device as a drug, biological product, or device that has successfully completed phase one of a clinical trial approved by the United States Food and Drug Administration (FDA), but has not been approved for general use and remains under investigation in an approved clinical trial.

2. Allows, but does not require, the manufacturer of an investigational product to make the product available to an eligible patient, as defined. Specifies that the manufacturer may require a patient to pay for the costs of the investigational product.
   
   
   

3. Defines an eligible patient as one who has met a number of specified criteria, including having a serious or life-threatening condition, as defined, and having considered all FDA-approved treatment options.
   
   
   

4. Allows, but does not require, a health plan or insurer to provide coverage for the investigational product.
   
   
   

5. Prohibits a state regulatory board from taking disciplinary action against a physician's license, and prohibits a state agency from taking action against a facility's license, based on the recommendation, prescription, or treatment of the investigational product, provided the recommendation or prescription is consistent with protocol approved by an institutional review board.
   
   
   

6. Specifies it does not create a private cause of action against a manufacturer of an investigational product, or against any other person or entity involved in the care of an eligible patient using the investigational product, for any harm done to the patient resulting from it, so long as the manufacturer or other person or entity is complying with the law in good faith and exercising reasonable care.

7. Requires institutional review boards to report information about requests for investigational products to the California Department of Public Health (CDPH), the Medical Board of California (MBC), and the Osteopathic Medical Board of California (OBMC).

FISCAL EFFECT:

1. Minor and absorbable costs to CDPH, MBC, and OMBC to receive and review biannually reports from institutional review boards on the requests for treatment with an investigational product.

2. $380,000 in the first year of implementation, and $210,000 ongoing to Department of Managed Health Care (Managed Care Fund). First year costs are higher because of additional legal analysis and processing Public Records Act (PRA) requests for experimental procedures previously authorized or rejected by health plans. Ongoing costs are related to patient and provider inquiries to the DMHC’s Help Center. Many of the investigational and/or experimental Independent Medical Review (IMR) requests require thorough analysis to determine if literature and studies on the treatments are available, and to review clinical trials, if completed or available.

3. CDI costs are minor and absorbable, projected at under $10,000 annually (Insurance Fund). This committee's fiscal analysis of a very similar bill, SB 159 (Calderon), did not identify this to insurance regulators due to an assumption that the narrow eligibility criteria of the bill would apply to very few people, if any. However, it has since come to this committee's attention that although few individuals may qualify to receive treatment under this bill, responding to patient inquiries may result in increased costs to regulators.

COMMENTS:

1. Purpose. According to the author, this bill removes barriers to accessing potentially life-saving drugs for terminally-ill patients and doctors who believe an investigational drug or device could be their last hope for survival. These patients may seek compassionate use exemptions from the FDA, but the process is cumbersome and approval comes too late for many. This bill removes barriers for patients who need to immediately obtain investigational treatments, while also protecting physicians, hospitals, and manufacturers from retribution.

2. Background. Drugs and devices go through clinical trials, phase one of which establishes safety and assesses toxicity. Once a drug or devices passes phase one, there are two more phases before being approved for sale. This bill would allow manufacturers to make available investigational products, that have passed phase one trials, to patients meeting specified criteria, and would protect from liability doctors who recommend or prescribe the investigational products, as well as manufacturers who make them available.

Experimental treatments are often available by participating in a clinical trial. If a patient is unable to enroll in a clinical trial, the FDA expanded access exemption, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs or devices for serious diseases or conditions. A licensed physician is able to apply for expanded access on behalf of the patient. The physician must be willing to commit to oversee the treatment, work with the manufacturing company and FDA, obtain the drugs, monitor the patient during the course of treatment, and file necessary paperwork. The FDA states that they receive approximately 1,000 expanded use applications per year, and has approved more than 99% of those applications.

3. Support. The ALS Association – Golden West Chapter, City of Murrieta, and Union of American Physicians and Dentists support this bill.

4. Opposition. The California Medical Association, California Nurse Association/National Nurses United, Association of Northern California Oncologists oppose this bill, citing patient safety and other concerns. The Association of Northern California Oncologists believe this bill threatens the viability of adult clinical trial enrollment and would result in slowing the development of cancer care in the future.

5. Related Legislation.

1. SB 159 (Calderon), pending in Senate Appropriations Committee, is substantially similar to this bill, with differences primarily in definitions and liability exemptions.

2. SB 715 (Anderson) is largely the same as this bill with some differences in definitions. SB 715 was referred to Senate Health Committee and not heard.

3. SB 128 (Wolk), pending in the Assembly Health Committee, permits a competent, qualified individual who is a terminally ill adult to receive a prescription for aid in dying medication if certain conditions are met.

Analysis Prepared by: Lisa Murawski / APPR. / (916) 319-2081

Date of Hearing: July 7, 2015

ASSEMBLY COMMITTEE ON BUSINESS AND PROFESSIONS

Susan Bonilla, Chair

SBPCA Bill Id:SB 149

Author:(Stone) – As Amended Ver:May 5, 2015

SENATE VOTE: 39-0

NOTE: This bill was this bill is double referred, having been previously heard by the Assembly Committee on Health on June 30, 2015 and approved on a 17-0 vote.

SUBJECT: Investigational drugs, biological products, or devices: right to try.

SUMMARY: Permits a manufacturer of an investigational drug, biological product or device (IDBPD) to make the product available to an eligible patient with a terminal disease; requires a manufacturer that provides a IDBPD to a patient to report specific data to the Department of Public Health (DPH); authorizes a health benefit plan to provide coverage for an IDBPD, and prohibits the Medical Board of California (MBC) and the Osteopathic Medical Board of California (OMB) from taking disciplinary action against the license of a physician based on the physician’s recommendation to a patient to use an IDBPD.

EXISTING LAW:

The Business and Professions Code

1. Establishes the MBC, within the Department of Consumer Affairs (DCA), which provides for the licensure and regulation of physicians and surgeons, and requires the MBC to take action against a licensee who is charged with unprofessional conduct. (Business and Professions Code (BPC) § 2000 et seq.)

2. Provides for the licensure and regulation of osteopathic physicians and surgeons by the MBC within the DCA and requires the OMB to enforce the Medical Practice Act. (BPC § 2000 et seq.)

The Health and Safety Code

1. Establishes the Sherman Food, Drug, and Cosmetic Law, which regulates the packaging, labeling, and advertising of drugs and devices, administered by the DPH.
   
   

2. Prohibits, in the Sherman Law, the sale, delivery or giving away of a new drug or device unless it is either:

1. A new drug or new device for which DPH has approved a new drug or device application, and has not withdrawn, terminated or suspended that approval; or,

2. A new drug, and a new drug application has been approved for it by the U.S. Food and Drug Administration (FDA), pursuant to federal law or it is a new device for which a premarket approval application has been approved, and that approval has not been withdrawn, terminated or suspended under the FDA.

1. Establishes the Protection of Human Subjects in Medical Experimentation Act which prescribes various protections for subjects of medical experimentation relating to a bill of rights; informed consent procedures and documentation; and, the provision of specified disclosures, including the right for a subject to give or withdraw consent freely and without duress. Imposes penalties for violations of these protections.
   
   

2. Requires health plans and insurers to provide an external, independent review process to examine plan’s coverage denials of experimental or investigational therapies for individual enrollees who have a life-threatening or seriously debilitating condition and who meet other specified criteria.
   
   

3. Requires health plans and insurers to provide coverage for all routine patient care costs relative to the treatment of an enrollee or insured diagnosed with cancer and accepted in an FDA-approved cancer clinical trial, Phase I-IV, if the enrollee's treating physician, recommends participation in the clinical trial after determining such participation has a meaningful potential to benefit the enrollee or insured.

Federal Law

1. Establishes the federal Food, Drug, and Cosmetic Act, which grants authority to the FDA to oversee the safety of food, drugs, and cosmetics.
   
   
   

2. Prohibits any new drug from being introduced into interstate commerce unless an application has been approved by the FDA.
   
   
   

3. Under federal regulation:

1. Requires clinical trial sponsors to submit an Investigational New Drug (IND) application to the FDA for clinical investigation of a new drug or new indication of an approved drug, with certain exceptions;
   
   
   

2. Requires review and approval from an Institutional Review Board (IRB) before a clinical study can be initiated under an IND;
   
   
   

3. Defines an IRB as an appropriately constituted group that has been designated to review and monitor biomedical research involving human subjects, to ensure that a clinical trial is ethical and that the rights of study participants are protected; and,
   
   
   

4. Authorizes an IRB to approve, require modifications in, or disapprove research, or to suspend or terminate approval of research that is not being conducted in accordance with the IRB's requirements or that has been associated with unexpected serious harm to subjects.
   
   
   

1. Establishes the Office for Human Research Protections (OHRP), which provides leadership in the protection of the rights, welfare and wellbeing of subjects involved in research conducted or supported by the U.S. Department of Health and Human Services (HHS).

THIS BILL:

1. Enacts the Right to Try Act (Act) and makes the following definitions:

1. “Eligible patient” means a person who has a terminal disease as determined by that person’s physician and a consulting physician; his or her physician has determined that the person has no comparable or satisfactory FDA approved treatment options available to diagnose, monitor, or treat the disease or condition involved, and that the probable risk to the person from the IDBPD is not greater than the probable risk from the disease or condition; he or she has received a prescription or recommendation from his or her physician for an IDBPD; he or she has given written informed consent for the use of the IDBPD, or if he or she is a minor or lacks the capacity to provide informed consent, his or her parent, legal guardian, or legally authorized representative has given written informed consent on his or her behalf; he or she has documentation from his or her physician that the patient has met these requirements.

2. “Health benefit plan” means any plan or program that provides, arranges, pays for, or reimburses the cost of health benefits, including but not limited to, a health care service plan contract issued by a health care service plan, as specified, and a policy of health insurance issued by a health insurer.

3. “Health facility” means a facility, place, or building that is organized, maintained, and operated for the diagnosis, care, prevention, and treatment of human illness, physical or mental, including convalescence and rehabilitation and including care during and after pregnancy, or for any one or more of these purposes, for one or more persons, to which the persons are admitted for a 24-hour stay or longer, as specified.

4. “Investigational drug, biological product, or device” means a drug, biological product, or device that has successfully completed phase one of a clinical trial approved by the FDA but has not been approved for general use by the FDA and remains under investigation in a clinical trial approved by the FDA.

5. “Physician” means a physician and surgeon licensed under the Medical Practice Act or an osteopathic physician and surgeon licensed under the Osteopathic Act who is providing medical care or treatment to the eligible patient for the terminal illness. Specifies that physician does not include a primary care physician.

6. “State regulatory board” means the MBC or the OMB.

7. “Terminal disease” means an incurable an irreversible disease that has been medically confirmed and will, according to reasonable medical judgment, result in death within six months of diagnosis.

2. Authorizes a manufacturer of an IDBPD to make the IDBPD available to an eligible patient pursuant to the Act, but clarifies that a manufacturer is not required to make the IDBPD available to an eligible patient.

3. Authorizes a manufacturer to provide an IDBPD to an eligible patient without receiving compensation; requires an eligible patient to pay the costs of or associated with the manufacture of the IDBPD, and; requires an eligible patient to participate in data collection relating to the use of the IDBPD.

4. Authorizes a health benefit plan to provide coverage for an IDBPD, or device but, except as otherwise required by law, but does not require a health benefit plan or any state agency to provide coverage for the cost of any IDBPD.

5. Prohibits a state regulatory board, notwithstanding any other law, from revoking, failing to renew, or taking any other disciplinary action against a physician’s license based solely on the physician’s recommendation to an eligible patient regarding, or prescription for, or treatment with, an IDBPD.

6. Prohibits a state agency, notwithstanding any other law, from taking any action against a health facility’s license based solely on the facility’s participation in the treatment by or use of an IDBPD.

7. Provides that a violation of the Act shall not be subject to penalties for other violations of provisions of the Sherman Law.

8. Clarifies that the Act does not create a private cause of action against a manufacturer of an IDBPD, or against any other person or entity involved in the care of an eligible patient using the IDBPD, for any harm to the eligible patient resulting from the IDBPD so long as the manufacturer or other person or entity complies in good faith with the terms of this article and exercises reasonable care.

9. Requires a manufacturer that provides an IDBPD to an eligible patient to report the following information to the State Department of Public Health:

1. The number of requests made for an IDBPD;

2. The number of requests that were approved;

3. The duration of treatments;

4. The success of failure of the IDBPD in treating the terminal disease with which the eligible patient was diagnosed;

5. Any adverse event for each IDBPD;

6. Costs paid by each eligible patient for each investigation drug; and,

7. The consulting physician’s diagnosis and prognosis, and verification that the eligible patient is competent, acting voluntarily, and has made an informed decision or that the consulting physician has determined the person is not an eligible patient.

10. Requires the information collected to be confidential and collected in a manner that protects the privacy of the patient, the patient’s family, and any medical provider or pharmacist involved with the patient.

FISCAL EFFECT: According to the Senate Appropriations Committee analysis dated May 18, 2015, this bill will result in one-time costs of $20,000 and ongoing costs of $10,000 per year to review plan filings by the Department of Insurance. This bill will also result in increased costs of $380,000 in 2015 to 2016 to respond to requests for information about prior independent medical review decisions and ongoing costs of $210,000 per year to respond to consumer complaints and requests for independent medical review for access to investigational drugs by the Department of Managed Health Care. It is uncertain what the impact on state-funded health CalPERS health care coverage will be.

COMMENTS:

Purpose. This bill is sponsored by the Goldwater Institute. According to the author, “For terminal patients who have exhausted their conventional treatment options, obtaining access to potentially life-saving investigational drugs in often extremely difficult. The patient can attempt to enroll in a clinical trial, but many of the sickest individuals do not qualify to join such a trail. For these patients, their only hope for obtaining these potentially life-saving drugs is to request that the FDA grant them expanded access. SB 149…would allow a terminally ill patient a last-ditch effort to save their life. This bill would allow a patient to try an investigational drug only when certain conditions have been met, while providing important legal protections for all parties involved.”

Background. The FDA has jurisdiction over all drugs that are sold across state lines. State law provides DPH with the authority to approve for market a drug or device that is sold in California. If a product has received the FDA approval, then the DPH automatically recognizes the product's approval. In rare instances when a drug or device will be sold only in the state of California, and will not be distributed through interstate commerce, then California would be the approving body. According to the DPH, this has resulted in a few instances where California has approved a new drug or device application.

Independent from drug or device applications, manufacturers of drugs and devices must be licensed to manufacture in the state of California. The FDA does not approve manufacturers, but they do conduct inspections to ensure manufacturers are in compliance with good manufacturing practices. Under California Law, drug manufacturers must be licensed with the DPH even if not registered with FDA.

Clinical Trials. A clinical trial is a study that is carefully designed to test the benefits and risks of a specific medical treatment or intervention, such as a new drug. The FDA requires a multi-phase clinical trials process to be completed before deciding if an investigational medicine is safe and effective for a broader patient population.

According to the author’s office, the FDA has been having great difficulty in getting patients to participate in clinical trials of investigational drugs, because patients do not want to take the chance that they will be in the control group and not receive the experimental drug.

Expanded Access. If a patient is unable to enroll in a clinical trial, the FDA's expanded access exemption, also commonly referred to as “compassionate use,” provides a pathway for patients to gain access to investigational drugs or devices for serious diseases or conditions. A licensed physician is able to apply for expanded access under a single patient IND Application on behalf of the patient. The supervising physician must be willing to commit to oversee the treatment, work with the manufacturing company and the FDA, obtain the drugs, monitor the patient during the course of treatment, and file necessary paperwork. The FDA states that they receive approximately 1,000 expanded use applications per year, and has approved more than 99% of those applications. In the last four years, the FDA has denied only 33 of nearly 6,000 expanded access requests.

The FDA allows physicians to request expanded access for patients in an emergency situation over the phone or by "other rapid means of communication." The FDA states that authorization of the emergency use may be given by an FDA official over the telephone, provided the physician explains how the expanded access use will meet federal requirements for expanded access use and agrees to submit an expanded access submission within 15 working days of FDA’s initial authorization of the expanded access use.

Critics of the FDA process have raised concerns that the expanded access application is too cumbersome for physicians and patients to complete. On February 4, 2015, the FDA announced that it would be streamlining the expanded use application process, and stated that the proposed application will allow physicians to apply for experimental drugs in just 45 minutes. The FDA draft guidance on the new application process is currently in the 60-day public comment period.

Institutional Review Board (IRB). Any FDA expanded access requests for any investigational drug must be reviewed by an IRB in order to understand the risks of an experimental treatment and ensure that the patient understands them as well.

A physician submits the proposed experimental protocol, to an IRB, which reviews them to make sure patients are fully aware of potential risks and are willing to accept the level of potential risk associated with the drug. IRBs are usually associated with a hospital or research institution, but there are independent IRBs available for physicians not associated with such an institution. Physicians are governed primarily by the IRB committee policies set up by their specific institution. The FDA maintains the right to overrule the physician and IRB.

Physician Liability and Enforcement Action Against a Physician. Physicians may be concerned about facing a liability risk for IDBPDs that they recommend to patients or help them gain access to. The Compassionate Access Act of 2010 (H.R. 4732) sought to address this issue by providing immunity to the manufacturer, distributor, administrator, sponsor or physician from suit or liability.

If this bill is enacted, the MBC and OMB would be prohibited from taking action against a physician’s license for prescribing or treating a patient with an IDBPD.

Other States. Similar legislation has been signed into law in the following states: 1) Colorado, 2) Louisiana, 3) Michigan and 4) Missouri. Arizona adopted a resolution to place the issue on the November 2014 ballot, where it was approved by voters. Presently, at least 20 other states are considering similar legislation.

Current Related Legislation. SB 128 (Wolk) of the current legislative session, permits a competent, qualified individual who is a terminally ill adult to receive a prescription for aid in dying medication if certain conditions are met. STATUS: This bill is currently pending in the Senate Appropriations Committee.

AB 159 (Calderon) of the current legislative session, is largely similar to this bill, with differences primarily in definitions and liability exemptions. STATUS: AB 159 is pending in the Senate Business, Professions and Economic Development Committee.

SB 715 (Anderson) of the current legislative session, is largely the same as this bill with some differences in definitions. STATUS: SB 715 is pending in the Senate Health Committee.

AMENDMENTS:

1. As agreed to during the June 28, 2015 Assembly Health Committee hearing, the author will accept the following recommended amendments for this bill as follows:

1. As drafted, this bill requires that a patient has given written, informed consent, but does not specify what information must be included in that consent. Additionally, informed consent requirements for experimental treatments are already codified in the “Protection of Human Subjects in Medical Experimentation Act” (HSC 24170-24179). To ensure that patients are fully aware of all risks and unique requirements of this state, the author should amend the bill to explicitly state the requirements of the informed consent, and also to cross-reference the informed consent requirements already codified in current law.

2. Even when treating a single, terminally ill patient, the use of an unapproved drug is still considered human experimentation and should be bound by the highest scientific ethical standards. Lack of IRB oversight in this bill weakens patient protections. Most institutions have IRB policies in place that physicians must follow. Because this bill does not specify IRB review of treatment protocol and informed consent, physicians not bound and their institutional policies might not undergo this ethical review. This bill should be amended to require that the informed consent document and treatment protocol be approved by the physician's institutional review board or by an accredited independent institutional review board.

3. The definition of terminal disease used in this bill is inconsistent with current law, as well as other measures under consideration by this Committee this year. To be consistent with AB 159, and remove conflict with SB 128, the definition of eligible patient should be amended as follows:

On page 3, line 3:

(a) “Eligible patient” means a person to whom all of the following conditions apply:

(1) He or she has a terminal disease immediately life-threatening disease or condition as determined by that person’s physician and a consulting physician.

On page 4, delete lines 12-15 and insert:

“Immediately life-threatening disease or condition” means a stage of disease in which there is a reasonable likelihood that death will occur within a matter of months.

2. The author should also adopt the following amendments as proposed by this committee:

1. Though this bill requires the manufacturer to report specific information to the Department of Public Health, there is no requirement that the manufacturer send this information to other regulatory entities. As such, the author should require the manufacturer to send the same information to the MBC and the OMB.

On page 5, line 22, after “To the State Department of Public Health,” insert:

the Medical Board of California and the Osteopathic Medical Board.

2. As has been done in other states that have proposed similar legislation, the author should address the premature ending of a clinical trial.

On page 4, after line 31, insert the following:

(c) If the clinical trial is closed due to the lack of efficacy or toxicity, the drug must not be offered. If notice is given on a drug, product or device taken by a patient outside of a clinical trial, the pharmaceutical company and the patient’s physician must notify the patient of the information from the safety committee of the clinical trial.

On page 4, line 32 strike: (c) and insert: (d)

3. This bill requires a manufacturer to report specific information to the DPH including the patient’s diagnosis and prognosis, verification that the patient is competent, acting voluntarily and has made an informed decision, or is not an eligible patient. The DPH is to receive this information from the patient’s “consulting physician.” However, the term “consulting physician” is not specifically defined in the bill. As such, the author should amend the bill to define “consulting physician” and specify that the information required to be sent to the DPH also includes information from the patient’s physician.

On page 4, after line 5 insert:

“Consulting physician” means a physician and surgeon licensed under the Medical Practice Act or an osteopathic physician and surgeon licensed under the Osteopathic Act who shall perform all of the following:

(a) Examine the qualified individual and his or her relevant medical records.

(b) Confirm in writing the attending physician’s diagnosis and prognosis.

(c) Verify, in the opinion of the consulting physician, if the patient is an eligible patient.

(d) If the patient is eligible, verify that the eligible patient is competent, acting voluntarily, and has made an informed decision.

On page 5, line 34, after “The” insert: physician and, after “and” insert: consulting physician’s

REGISTERED SUPPORT:

The ALS Association Golden West Chapter

City of Murrieta

County of Los Angeles

Union of American Physicians and Dentists

REGISTERED OPPOSITION:

Association of Northern California Oncologists

California Medical Association

California Nurses Association

Analysis Prepared by: Le Ondra Clark Harvey, Ph.D. / B. & P. / (916) 319-3301

Date of Hearing: June 30, 2015

ASSEMBLY COMMITTEE ON HEALTH

Rob Bonta, Chair

SBPCA Bill Id:SB 149

Author:(Stone) – As Amended Ver:May 5, 2015

SENATE VOTE: 39-0

SUBJECT: Investigational drugs, biological products, or devices: right to try.

SUMMARY: Enacts the Right to Try Act, and permits a manufacturer of an investigational drug, biological product, or device to make available an investigational drug, biological product, or device to an eligible patient. Specifically, this bill:

1. Prohibits the provisions of this bill from being interpreted as requiring a manufacturer make available an investigational drug, biological product, or device to an eligible patient, as defined.

2. Defines “investigational drug, biological product, or device” as a drug, biological product, or device that has successfully completed phase I of a clinical trial approved by the Food and Drug Administration (FDA), but has not been approved for general use and remains under investigation in an approved clinical trial.

3. Defines “eligible patient” as a person who:

1. Has a terminal disease as determined by that person's physician and a consulting physician;

2. Has no comparable or satisfactory FDA-approved treatment options, as determined by his or her physician, available to diagnose, monitor, or treat the disease or condition involved, and that the probable risk to the person from the investigational drug, biological product, or device is not greater than the probable risk from the disease or condition;

3. Has received a prescription or recommendation from his or her physician for an investigational drug, biological product, or device;

4. Has given written informed consent for the use of the investigational drug, biological product, or device. If he or she is a minor or lacks the capacity to provide informed consent, his or her parent, legal guardian, or legally authorized representative must give written informed consent on his or her behalf; and,

5. Has documentation from his or her physician that the patient has met the requirements of this bill.

4. Defines “terminal disease” means an incurable and irreversible disease that has been medically confirmed and will, according to reasonable medical judgment, result in death within six months of diagnosis.

5. Permits manufacturers to:

1. Provide an investigational drug, biological product, or device to an eligible patient without receiving compensation;

2. Require an eligible patient to pay the costs of or associated with the manufacture of the investigational drug, biological product, or device; and,

3. Require an eligible patient to participate in data collection relating to the use of the investigational drug, biological product, or device.

6. Specifies that this bill does not require a health plan or any state agency to provide coverage for the cost of any investigational drug, biological product, or device, but permits health plans to provide such coverage.

7. Prohibits a state regulatory board from revoking, failing to renew, or taking any other disciplinary action against a physician's license based solely on the physician's recommendation to an eligible patient regarding, or prescription for, or treatment with, an investigational drug, biological product, or device pursuant to this bill.

8. Prohibits a state agency from taking any action against a health facility's license based solely on the facility's participation in the treatment by or use of an investigational drug, biological product, or device pursuant to this bill.

9. Specifies that this bill does not create a private cause of action against a manufacturer of an investigational drug, biological product, or device, or against any other person or entity involved in the care of an eligible patient using the investigational drug, biological product, or device, for any harm to the eligible patient resulting from the investigational drug, biological product, or device so long as the manufacturer or other person or entity complies in good faith with the terms of this bill and exercises reasonable care.

10. Requires a manufacturer that provides an investigational drug, biological product, or device to an eligible patient to report all of the following information to the California Department of Public Health (DPH):

1. The number of requests made for an investigational drug, biological product, or device;

2. The number of requests that were approved;

3. The duration of treatments;

4. The success or failure of the investigational drug, biological product, or device in treating the terminal disease with which the eligible patient was diagnosed;

5. Any adverse event for each investigational drug, biological product, or device;

6. Costs paid by each eligible patient for each investigational drug; and,

7. The consulting physician's diagnosis and prognosis, and verification that the eligible patient is competent, acting voluntarily, and has made an informed decision, or that the consulting physician has determined that the person is not an eligible patient.

11. Requires the information collected to be confidential and collected in a manner that protects the privacy of the patient, the patient's family, and any medical provider or pharmacist involved with the patient.

EXISTING STATE LAW:

1. Establishes the Sherman Food, Drug, and Cosmetic Law, which regulates the packaging, labeling, and advertising of drugs and devices, administered by DPH.

2. Prohibits, in the Sherman Law, the sale, delivery, or giving away of a new drug or device unless it is either:

1. A new drug or new device for which DPH has approved a new drug or device application, and has not withdrawn, terminated, or suspended that approval; or,

2. A new drug, and a new drug application has been approved for it by the FDA, pursuant to federal law, or it is a new device for which a premarket approval application has been approved, and that approval has not been withdrawn, terminated, or suspended under the FDA.

3. Establishes the Protection of Human Subjects in Medical Experimentation Act which prescribes various protections for subjects of medical experimentation relating to a bill of rights; informed consent procedures and documentation; and, the provision of specified disclosures, including the right for a subject to give or withdraw consent freely and without duress. Imposes penalties for violations of these protections.

4. Requires health plans and insurers to provide an external, independent review process to examine plan's coverage denials of experimental or investigational therapies for individual enrollees who have a life-threatening or seriously debilitating condition and who meet other specified criteria.

5. Requires health plans and insurers to provide coverage for all routine patient care costs relative to the treatment of an enrollee or insured diagnosed with cancer and accepted in an FDA-approved cancer clinical trial, Phase I-IV, if the enrollee's treating physician, recommends participation in the clinical trial after determining such participation has a meaningful potential to benefit the enrollee or insured.

EXISTING FEDERAL LAW:

1. Establishes the federal Food, Drug, and Cosmetic Act, which grants authority to the FDA to oversee the safety of food, drugs, and cosmetics.

2. Prohibits any new drug from being introduced into interstate commerce unless an application has been approved by the FDA.

3. Under federal regulation:

1. Requires clinical trial sponsors to submit an Investigational New Drug (IND) application to the FDA for clinical investigation of a new drug or new indication of an approved drug, with certain exceptions;

2. Requires review and approval from an Institutional Review Board (IRB) before a clinical study can be initiated under an IND;

3. Defines an IRB as an appropriately constituted group that has been designated to review and monitor biomedical research involving human subjects, to ensure that a clinical trial is ethical and that the rights of study participants are protected; and,

4. Authorizes an IRB to approve, require modifications in, or disapprove research, or to suspend or terminate approval of research that is not being conducted in accordance with the IRB's requirements or that has been associated with unexpected serious harm to subjects.

4. Establishes the Office for Human Research Protections (OHRP), which provides leadership in the protection of the rights, welfare, and wellbeing of subjects involved in research conducted or supported by the U.S. Department of Health and Human Services (HHS). OHRP helps ensure this by providing clarification and guidance, developing educational programs and materials, maintaining regulatory oversight, and providing advice on ethical and regulatory issues in biomedical and social-behavioral research.

FISCAL EFFECT: According to the Senate Appropriations Committee, one-time costs of $20,000 and ongoing costs of $10,000 per year to review plan filings by the Department of Insurance (Insurance Fund). Increased costs of $380,000 in 2015-16 to respond to requests for information about prior independent medical review decisions and ongoing costs of $210,000 per year to respond to consumer complaints and requests for independent medical review for access to investigational drugs by the Department of Managed Health Care (Managed Care Fund). Uncertain impact on state-funded health CalPERS health care coverage (General Fund and special funds).

COMMENTS:

1. PURPOSE OF THIS BILL. According to the author, terminal patients who have exhausted their conventional treatment options have difficulty obtaining access to potentially life-saving investigational drugs. The patient can attempt to enroll in a clinical trial, but many of the sickest individuals do not qualify to join such a trial. This bill would give a patient, who has exhausted all other treatment options, a last-chance effort to save their life with an option to try an experimental drug that has passed Phase 1 of the FDA safety trials. The author states that this bill ensures that terminal patients have the right to make their own medical decisions, while providing important legal protections for patients, doctors, insurance and pharmaceutical companies.

2. BACKGROUND. Patient requests for access to drugs and biologics prior to their approval have long created a dilemma for regulators, who must balance the needs of patients and their families who believe that an experimental product could save their life, with ensuring the safety of the greater population through tightly controlled clinical trials and drug approval. This issue has become increasingly visible and difficult in recent times, as views about the inherent right of patients in certain situations to unapproved products have been expressed by patients and their advocates, state legislators, members of Congress, and some court cases. In 2014, four states adopted legislation similar to this bill (Colorado, Louisiana, Michigan, and Missouri) and one state (Arizona) adopted a resolution to place the issue on the November 2014 ballot, where it was approved by voters. As of March 2015 there are at least 36 states and Washington D.C. that have examined or will examine legislation on the issue, and 12 have already signed laws.

3. FDA AND STATE APPROVAL OF DRUGS. FDA has jurisdiction over all drugs that are sold across state lines. State law gives DPH the authority to approve for market a drug or device that is sold in California. If a product has received FDA approval, then DPH automatically recognizes the product's approval. In rare instances when a drug or device will be sold only in the state of California, and will not be distributed through interstate commerce, then California would be the approving body. According to DPH, this has resulted in a few instances where California has approved a new drug or device application.

4. THE CLINICAL TRIAL PROCESS. A clinical trial is a study that is carefully designed to test the benefits and risks of a specific medical treatment or intervention, such as a new drug. The FDA requires a multi-phase clinical trials process to be completed before deciding if an investigational medicine is safe and effective for a broader patient population.
   
   

   1. Phase I is a clinical trial using a small group of healthy individuals (generally 20 to 80 volunteers). This stage is designed to assess the toxicity and dosing of a drug and whether or not there are harmful side effects associated with the drug. Phase I trial does not establish either the safety or efficacy of a drug. According to the FDA, the goal in this phase is to determine what the drug's most frequent side effects are in healthy volunteers, how the drug is metabolized and excreted, and whether there is an unacceptable level of toxicity associated with taking the drug.

2. Phase II clinical trials involve control groups and experimental groups (up to 300 individuals) to determine whether the drug is effective for the intended purpose on a particular disease or condition. This phase aims to obtain preliminary data on whether the drug works in people who have a certain disease or condition.

3. Phase III begins if the drug proves to be effective in Phase II. These studies gather further information on the drug's safety and effectiveness on different populations (several hundred to about 3,000 participants) and tests varying levels of doses in combination or in comparison with different drugs.

4. Phase IV studies are post-marketing studies the sponsor has agreed to do after the FDA approves a drug. These studies gather additional information on a product's safety, efficacy, and optimal use.
   
   

1. Expanded Access to investigational products through the FDA. If a patient is unable to enroll in a clinical trial, the FDA expanded access exemption, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs or devices for serious diseases or conditions. A licensed physician is able to apply for expanded access under a single patient IND Application on behalf of the patient. The supervising physician must be willing to commit to oversee the treatment, work with the manufacturing company and FDA, obtain the drugs, monitor the patient during the course of treatment, and file necessary paperwork. The FDA states that they receive approximately 1,000 expanded use applications per year, and has approved more than 99% of those applications. In the last four years, the FDA has denied only 33 of nearly 6,000 expanded access requests.

FDA directives allow physicians to request expanded access for patients in an emergency situation over the phone or by other rapid means of communication. The FDA states that authorization of the emergency use may be given by an FDA official over the telephone, provided the physician explains how the expanded access use will meet federal requirements for expanded access use and agrees to submit an expanded access submission within 15 working days of FDA's initial authorization of the expanded access use.

6. FDA ACTION. Critics of the FDA process have raised concerns that the expanded use application is too cumbersome for physicians and patients to complete. Critics claim that it currently requires an application that takes doctors 100 hours to fill out. The response time from the FDA ranges from a few days to a few months. On February 4, 2015, the FDA announced that it would be streamlining the expanded use application process, and stated that the proposed application will allow physicians to apply for experimental drugs in just 45 minutes. The FDA issued draft guidance and included a copy of the new form, asking for comment until April 13, 2015. The FDA is now in the process of reviewing the public comments and will not likely finalize the guidance before late summer/early fall.

7. COURT HISTORY. In 2005, the Abigail Alliance for Better Access to Developmental Drugs, with the mission of facilitating access to experimental drugs for patients, sued the FDA, claiming a constitutional due process right for terminally ill patients to access unapproved drugs. A US District court disagreed and the case was heard by the US Court of Appeals for the District of Columbia Circuit. In 2006, a divided three-judge panel of the D.C. Circuit agreed with the Alliance, finding that where there are no other FDA-approved treatment options, a terminally ill patient has a constitutionally-protected fundamental right to access investigational drugs. In 2007, the full D.C. Circuit reheard the case en banc and reversed the panel decision. The Alliance then filed a petition asking the U.S. Supreme Court to hear the case but the Supreme Court denied the petition, so for now the Court of Appeals decision stands.

8. INSTITUTIONAL REVIEW BOARDS ARE NECESSARY PATIENT SAFEGUARDS. Federal law requires that any FDA expanded use requests for any investigational drug be reviewed by an IRB, or ethics committee; a group of medical experts that evaluates the risks of an experimental treatment and ensures the patient understands them as well. The IRB process protects the people receiving the drug and ensures that the risks are reasonable given the potential benefits. A physician submits the proposed experimental protocol, including duration of treatment and the informed consent documents to an IRB, which reviews them to make sure patients are fully aware of potential risks and are willing to accept the level of potential risk associated with the drug. IRBs are usually associated with a hospital or research institution, but there are independent IRBs available for physicians not associated with such an institution. The OHRP requires and regulates the use of IRBs for any research supported or conducted by HHS (which includes National Institutes on Health funded research). While OHRP oversees IRB processes in general, each institution has their own review board, timelines, and specific policies. Physicians are governed primarily by the IRB committee policies set up by their specific institution.

9. CONTROVERSY OVER RESEARCH WITHOUT IRB APPROVAL. In 2012, two neurosurgeons from the University of California, Davis (UC Davis) were accused of performing an unapproved experimental procedure on three dying brain cancer patients. In these cases, the neurosurgeons obtained informed consent from the patients but did not communicate to the patients that the procedure had not been approved by the FDA as an investigational new treatment for testing in human subjects. The patients were also not informed that the research was not approved by UC Davis' IRB. Two of the patients developed sepsis and died within weeks of their surgeries. Both surgeons involved were banned from performing surgery and later resigned from the University. In response to these incidences, UC Davis has since tightened its innovated use policy to expressly prohibit any use of a non-FDA approved drug, biologic, or device without formal IRB and, as required, FDA approval, regardless of the purpose of the use.

10. Who will pay? If a patient is enrolled in an FDA-approved clinical trial for cancer, California law requires that the insurer pay for routine patient care costs. Health plans do not have to pay for experimental drug, but in most cases these drugs are given to clinical trial participants at no cost by the government agency, university medical center, or pharmaceutical company that sponsors the trial.

This bill authorizes, but does not require any health plan, insurer, or manufacturer to pay for the cost of the medication. Typically, health plans limit coverage to only FDA approved drugs and devices. In addition, when a plan reviews potential drugs for their formulary, they review data on clinical effectiveness, cost of the drug, potential side effects or harm, and the costs associated with treating that. Under current law, a patient denied coverage for an experimental drug or device may appeal the decision and request evaluation by Independent Medical Review, although this can be a time-consuming process. Taken together, it is unlikely that health plans would cover the cost of experimental drugs obtained through this legislation.

In the expanded access use protocols the FDA allows, after their review and approval, the manufacturer to charge a nominal fee for the cost of providing the experimental drug or device. This bill authorizes a manufacturer to charge for the experimental drug or device, without any review or approval.

11. SUPPORT. Supporters argue that patients with no other treatment options should be able to take on more risk than healthy patients would otherwise choose. The ALS Association Golden West Chapter states that they support and advocate for expanded access to experimental treatments for people living with ALS.

12. OPPOSITION. The Association of Northern California Oncologists opposes this bill citing several reasons, including the difficulty identifying "terminally-ill" patients, the lack of an informed consent process to protect patients seeking these drugs, and the lack of appropriate physician supervision of the use of these drugs. The California Medical Association states opposition due to significant patient safety concerns with allowing access to unproven drugs outside the FDA's clinical trials and compassionate use programs. The FDA ensures efficacy and safety of using such drugs, and once they are removed from these processes there is no longer an ability to protect patients and provide proper oversight. Additionally, this bill may be both premature and unnecessary, and could also potentially detract from the FDA rulemaking process that is currently ongoing. The California Nurses Association (CNA) states that this bill erroneously suggests that consumers do not have access to these drugs because physicians fear their liability to practice medicine would be compromised and their liability increased if they were to recommend such treatment. CNA points out that "nothing in this legislation impacts the availability of drugs to terminally ill patients unless the manufacturers of the drugs allow it to be used in advance of FDA approval".

13. CONCERNS. The Pharmaceutical Research and Manufacturers of America (PhRMA) has concerns with this bill, writing that while “the bill is well-intentioned, it could have serious unintended consequences that must be taken into consideration” and adds that this bill may be premature given recent developments at the federal level. PhRMA states that right-to-try legislation is unlikely to achieve the goal of bringing innovative, safe and effective medicines to patients as quickly as possible and cites the current robust nature of the current clinical trial process, the FDA's current expanded use protocols, and efforts underway to streamline the expanded use procress as reasons for concerns.

The Los Angeles County Board of Supervisors notes that that this does not provide patients with comprehensive written, informed consent. The Board "would support this bill if it was amended to include such informed consent to help ensure that patients are fully aware of the potential risks involved with receiving experimental treatment".

14. RELATED LEGISLATION.
    
    

    1. SB 715 (Anderson) is substantively similar to this bill. The bill is currently pending in the Senate Committee on Health.

2. SB 128 (Wolk) permits a competent, qualified individual who is a terminally ill adult to receive a prescription for aid in dying medication if certain conditions are met. SB 128 is currently pending in this Committee.
   
   

3. AB 159 (Calderon) is substantially similar to this bill, and also creates a Right to Try Act, permitting a manufacturer of an investigational drug, biological product, or device to make available an investigational drug, biological product, or device to an eligible patient. AB 159 was approved by this Committee on April 7, 2015, by a vote of 17-0 and is now pending in Senate Business, Professions, and Economic Development Committee.

1. Double referral. This bill is double referred. Should it pass out of this Committee, it will be referred to the Assembly Committee on Business and Professions.

2. POLICY CONCERNS.

   1. Is this bill premature? In response to requests from advocates, and the passage of several right-to-try bills in states, the FDA is promulgating new regulations to dramatically shorten the length of time it takes to apply for expanded use access. Public comments on the proposed regulations were accepted through April, 2015 and are currently under review by the FDA. Given that the FDA may arrive at the intended purpose of this goal in the very near future, the committee may wish to consider whether this bill is premature.

2. Will this bill achieve its intended goal? Shipping an investigational drug across state lines is illegal without an approved IND from the FDA. If a manufacturer outside of California agreed to provide their investigational drug to a patient under this bill, they would be in violation of federal law. Drug manufacturers are not exempted from FDA rules under this law, and therefore companies interested in gaining full regulatory approval for their products are unlikely to ignore federal regulation.

Similar bills in other states have been enacted too recently to assess their impact. Anecdotal news reports suggest that when approached by patients directly, manufacturers are requesting that they go through the FDA process instead.

3. FDA is not the limiting factor. The FDA approves 99% of expanded access requests. The ultimate decision to provide the drug to the patient is currently made by the pharmaceutical manufacturer; nothing in this bill would address or change that.

4. Conflict with other bills this session. AB 159 already approved by this Committee, is substantively the same as this bill.

1. SUGGESTED AMENDMENTS.

   1. As drafted, this bill requires that a patient has given written, informed consent, but does not specify what information must be included in that consent. Additionally, informed consent requirements for experimental treatments are already codified in the “Protection of Human Subjects in Medical Experimentation Act” (HSC 24170-24179). To ensure that patients are fully aware of all risks and unique requirements of this state, the author should amend the bill to explicitly state the requirements of the informed consent, and also to cross-reference the informed consent requirements already codified in current law.

2. Even when treating a single, terminally ill patient, the use of an unapproved drug is still considered human experimentation and should be bound by the highest scientific ethical standards. Lack of IRB oversight in this bill weakens patient protections. Most institutions have IRB policies in place that physicians must follow. Because this bill does not specify IRB review of treatment protocol and informed consent, physicians not bound and their institutional policies might not undergo this ethical review. This bill should be amended to require that the informed consent document and treatment protocol be approved by the physician's institutional review board or by an accredited independent institutional review board.
   
   

3. The definition of terminal disease used in this bill is inconsistent with current law, as well as other measures under consideration by this Committee this year. To be consistent with AB 159, and remove conflict with SB 128, the definition of eligible patient should be amended as follows:
   
   

On page 3, line 3:

(a) “Eligible patient” means a person to whom all of the following conditions apply:

(1) He or she has a terminal disease immediately life-threatening disease or condition as determined by that person’s physician and a consulting physician.

On page 4, delete lines 12-15 and insert:

“Immediately life-threatening disease or condition” means a stage of disease in which there is a reasonable likelihood that death will occur within a matter of months.

REGISTERED SUPPORT / OPPOSITION:

Support

ALS Association – Golden West Chapter

City of Murrieta

Union of American Physicians and Dentists/American Federation of State, County and

Municipal Employees, AFL-CIO

Opposition

California Medical Association (unless amended)

California Nurses Association/National Nurses United

Association of Northern California Oncologists

Analysis Prepared by: Dharia McGrew / HEALTH / (916) 319-2097

 THIRD READING  

Bill No: SB 149

Author: Stone (R), et al.

Amended: 5/5/15

Vote: 21

SENATE HEALTH COMMITTEE: 7-0, 4/22/15

AYES: Hernandez, Nguyen, Mitchell, Nielsen, Pan, Roth, Wolk

NO VOTE RECORDED: Hall, Monning

SENATE BUS, PROF. & ECON. DEV. COMMITTEE: 9-0, 4/27/15

AYES: Hill, Bates, Berryhill, Block, Galgiani, Hernandez, Jackson, Mendoza, Wieckowski

SENATE APPROPRIATIONS COMMITTEE: 7-0, 5/28/15

AYES: Lara, Bates, Beall, Hill, Leyva, Mendoza, Nielsen

SUBJECT: Investigational drugs, biological products, or devices: right to try

SOURCE: Author

DIGEST: This bill enacts the Right to Try Act, and permits a manufacturer of an investigational drug, biological product, or device to make available an investigational drug, biological product, or device to an eligible patient, as defined.

ANALYSIS:

Existing federal law:

1. Establishes the federal Food, Drug, and Cosmetic Act, which grants authority to the U.S. Food and Drug Administration (FDA), to oversee the safety of food, drugs, and cosmetics.

2. Prohibits any new drug from being introduced into interstate commerce unless an application has been approved by the FDA.

3. Provides for the following under regulation:

1. Requires clinical trial sponsors to submit an Investigational New Drug (IND) application to the FDA for clinical investigation of a new drug or new indication of an approved drug, with certain exceptions;

2. Requires review and approval from an Institutional Review Board (IRB) before a clinical study can be initiated under an IND;

3. Defines an IRB as an appropriately constituted group that has been designated to review and monitor biomedical research involving human subjects, to ensure that a clinical trial is ethical and that the rights of study participants are protected; and,

4. Authorizes an IRB to approve, require modifications in, or disapprove research, or to suspend or terminate approval of research that is not being conducted in accordance with the IRB's requirements or that has been associated with unexpected serious harm to subjects.

4. Establishes the Office for Human Research Protections (OHRP), which provides leadership in the protection of the rights, welfare, and well-being of subjects involved in research conducted or supported by the U.S. Department of Health and Human Services. OHRP helps ensure this by providing clarification and guidance, developing educational programs and materials, maintaining regulatory oversight, and providing advice on ethical and regulatory issues in biomedical and social-behavioral research.

Existing state law:

1. Establishes the Sherman Food, Drug, and Cosmetic Law (Sherman Law), which regulates the packaging, labeling, and advertising of drugs and devices, administered by the Department of Public Health (DPH).

2. Prohibits, in the Sherman Law, the sale, delivery, or giving away of a new drug or device unless it is either:

1. A new drug or new device for which DPH has approved a new drug or device application, and has not withdrawn, terminated, or suspended that approval; or,

2. A new drug, and a new drug application has been approved for it by the FDA, pursuant to federal law, or it is a new device for which a premarket approval application has been approved, and that approval has not been withdrawn, terminated, or suspended under the FDA.

3. Establishes the Protection of Human Subjects in Medical Experimentation Act which prescribes various protections for subjects of medical experimentation relating to a bill of rights; informed consent procedures and documentation; and, the provision of specified disclosures, including the right for a subject to give or withdraw consent freely and without duress. Imposes penalties for violations of these protections.

4. Requires health plans and insurers to provide an external, independent review process to examine plan’s coverage denials of experimental or investigational therapies for individual enrollees who have a life-threatening or seriously debilitating condition and who meet other specified criteria.

5. Requires health plans and insurers to provide coverage for all routine patient care costs relative to the treatment of an enrollee or insured diagnosed with cancer and accepted in an FDA-approved cancer clinical trial, Phase I-IV, if the enrollee's treating physician, recommends participation in the clinical trial after determining such participation has a meaningful potential to benefit the enrollee or insured.

This bill:

1. Enacts the Right to Try Act, and permits a manufacturer of an investigational drug, biological product, or device to make available an investigational drug, biological product, or device to an eligible patient, as defined. Prohibits this bill from requiring a manufacturer make available an investigational drug, biological product, or device to an eligible patient.

2. Defines “investigational drug, biological product, or device” as a drug, biological product, or device that has successfully completed phase one of a clinical trial approved by the FDA, but has not been approved for general use and remains under investigation in an approved clinical trial.

3. Defines “eligible patient” as a person who:

1. Has a terminal disease as determined by that person’s physician and a consulting physician;

2. Has no comparable or satisfactory FDA-approved treatment options, as determined by his or her physician, available to diagnose, monitor, or treat the disease or condition involved, and that the probable risk to the person from the investigational drug, biological product, or device is not greater than the probable risk from the disease or condition;

3. Has received a prescription or recommendation from his or her physician for an investigational drug, biological product, or device;

4. Has given written informed consent for the use of the investigational drug, biological product, or device, or if he or she is a minor or lacks the capacity to provide informed consent, his or her parent, legal guardian, or legally authorized representative has given written informed consent on his or her behalf; and,

5. Has documentation from his or her physician that the patient has met the requirements of this bill.

4. Defines “terminal disease” means an incurable and irreversible disease that has been medically confirmed and will, according to reasonable medical judgment, result in death within six months of diagnosis.

5. Permits manufacturers to:

1. Provide an investigational drug, biological product, or device to an eligible patient without receiving compensation;

2. Require an eligible patient to pay the costs of or associated with the manufacture of the investigational drug, biological product, or device; and,

3. Require an eligible patient to participate in data collection relating to the use of the investigational drug, biological product, or device.

6. Specifies that this bill does not require a health plan or any state agency to provide coverage for the cost of any investigational drug, biological product, or device, but permits health plans to provide such coverage.

7. Prohibits a state regulatory board from revoking, failing to renew, or taking any other disciplinary action against a physician’s license based solely on the physician’s recommendation to an eligible patient regarding, or prescription for, or treatment with, an investigational drug, biological product, or device pursuant to this bill.

8. Prohibits a state agency from taking any action against a health facility’s license based solely on the facility’s participation in the treatment by or use of an investigational drug, biological product, or device pursuant to this bill.

9. Makes a violation of this bill not subject to the Sherman Law.

10. Specifies that this bill does not create a private cause of action against a manufacturer of an investigational drug, biological product, or device, or against any other person or entity involved in the care of an eligible patient using the investigational drug, biological product, or device, for any harm to the eligible patient resulting from the investigational drug, biological product, or device so long as the manufacturer or other person or entity complies in good faith with the terms of this bill and exercises reasonable care.

11. Requires a manufacturer that provides an investigational drug, biological product, or device to an eligible patient to report all of the following information to DPH:

1. The number of requests made for an investigational drug, biological product, or device.

2. The number of requests that were approved.

3. The duration of treatments.

4. The success or failure of the investigational drug, biological product, or device in treating the terminal disease with which the eligible patient was diagnosed.

5. Any adverse event for each investigational drug, biological product, or device.

6. Costs paid by each eligible patient for each investigational drug.

7. The consulting physician’s diagnosis and prognosis, and verification that the eligible patient is competent, acting voluntarily, and has made an informed decision, or that the consulting physician has determined that the person is not an eligible patient.

12. Requires the information collected to be confidential and collected in a manner that protects the privacy of the patient, the patient’s family, and any medical provider or pharmacist involved with the patient.

Background

According to an April 9, 2015 Health Policy Brief on the issue of right-to-try laws in Health Affairs, under current federal regulations, patients with serious or life-threatening illness have two primary options to access experimental therapies that may treat their condition but that have not yet been approved by the FDA: participate as a human subject in a clinical trial or, for patients who cannot be enrolled in that trial (because of their medical status or geographic location, for example), apply to the FDA for access to the experimental drug under the expanded access (also known as compassionate use) program.

1. Clinical trials. According to the Health Affairs brief, clinical testing of an experimental drug is typically a three-phase process. Phase I trials are small (20 to 80 patients) and are used primarily to evaluate safety and dosing ranges, usually in healthy volunteers. Phase II trials are larger (typically 100 to 300 patients) and are designed to show early evidence of efficacy in the patients that the drug is intended to treat. Phase III trials may include hundreds or thousands of patients and are used to demonstrate that the drug is effective compared to a control (such as a placebo or a comparator drug). Typically, a manufacturer submits an application to the FDA for marketing approval once a drug has successfully completed Phase III trials. Any drug company wishing to conduct a clinical trial must first submit an IND application to the FDA, which allows the company to manufacture the drug and ship it across state lines for use in the trial. The drug may be only administered to patients who are formally enrolled in that clinical trial. The study population for that trial may be limited based on any number of factors, including specific diagnosis, age, stage of illness, or comorbidities.

2. Expanded access program. According to the Health Affairs brief, the expanded access program is an attempt at flexibility in the regulatory process and to allow patients with no other treatment options a chance to try therapies they may not otherwise have access to. The program allows patients who meet certain eligibility requirements to receive an experimental therapy outside of the formal clinical research process. An application for expanded access can be submitted by either the drug manufacturer or a licensed physician. There are three categories of expanded access: treatment, single patient, and intermediate, which are further split into two subcategories. One is “expanded access INDs,” through which the manufacturer submits a separate IND for a patient or group of patients, and the other is “expanded access protocols,” whereby the manufacturer amends the protocol under an existing IND to include the patient (or patients) seeking access. Under treatment INDs, a relatively large group of patients (hundreds or thousands) are permitted to access an experimental drug, provided that the sponsor is actively pursuing FDA approval and is in later stages of testing (or has already submitted trial results to the FDA for review). According to an article published in the January 15, 2015 New England Journal of Medicine (NEJM), the FDA has permitted almost all expanded access requests regardless of category. The FDA estimated that by 2006, approximately 100,000 patients had obtained expanded access to experimental drugs. Between 2010 and 2013, the FDA imposed clinical holds on only 2 of the 2,472 individual, non-emergency protocols, on 1 of 66 intermediate-size requests, and on none of the 41 widespread expanded-access protocols it received.

The Health Affairs brief states that critics of the expanded access program have argued that the application process is unnecessarily burdensome and lengthy (it is estimated that the IND application requires about 100 hours to complete), which discourages doctors and manufacturers from applying. An IRB review adds an additional layer of paperwork and potential delay. These requirements were put in place in response to highly publicized incidents of harm caused by unsafe drugs. In the past decade, there have been several attempts made at the federal and judicial levels to further relax restrictions on the administration of experimental therapies to terminally ill patients. One, led by the Abigail Alliance for Better Access to Developmental Drugs, submitted a Citizen Petition to the FDA in 2003 requesting that it make experimental therapies available to terminally ill patients, provided that the drug had passed Phase I testing. Following several years of litigation, the DC Court of Appeals ruled against the Abigail Alliance, stating that terminally ill patients have no constitutional right to access experimental therapies. The US Supreme Court subsequently declined to review the case. Several bills have been introduced in the US Congress that aim to relax FDA restrictions on access to experimental therapies. The most recent of these (HR 4475, the Compassionate Freedom of Choice Act of 2014) failed to make it out of committee.

3. Other states. Over the past year, the issue of access to experimental therapies has been debated at the state level, in part due to efforts of the Goldwater Institute, a libertarian think tank that appears to be the source of this bill. In February 2014, the Institute released a policy paper that outlines the major critiques of the FDA’s expanded access program and proposes model legislation for states to adopt. According to the National Conference of State Legislatures, legislation related to right-to-try have been introduced in 30 states. Arizona, Colorado, Louisiana, Michigan, and Missouri have laws on the subject.

4. FDA action. According to an article in the Wall Street Journal published on February 9, 2015, the FDA announced it would “simplify and accelerate” the application process for “unapproved investigational drugs” that have passed Phase I safety trials. It is estimated that the new application form will take 45 minutes to complete, asks attending physicians to fill in eight pieces of patient information, and requires only one attachment. The FDA issued draft guidance and included a copy of the new form, asking for comment until April 13, 2015.

5. NEJM article. The authors of the January 2015 NEJM article mentioned above call right-to-try laws misguided, and call for a more pragmatic approach. They suggest that states work collaboratively with the FDA to make expanded access more practical when it is appropriate. For example, since the FDA has acknowledged that gaining approval from an IRB can pose a barrier, states could partner with the FDA to fund multicenter IRBs that focus specifically on expanded-access requests. Such multicenter panels would conduct full reviews, but their subject-matter expertise and limited dockets would translate into faster review times. Practical obstacles to enhancing expanded access programs, including administrative burdens and industry costs, would also be best tackled by the states in partnership with the FDA. For example, a manufacturer’s reluctance to provide product because of financial concerns could be addressed by permitting companies to charge amounts closer to the likely post approval cost of drugs.

Comments

Author’s statement. According to the author, for terminal patients who have exhausted their conventional treatment options, obtaining access to potentially life-saving investigational drugs is often extremely difficult. The patient can attempt to enroll in a clinical trial, but many of the sickest individuals do not qualify to join such a trial. For these patients, their only hope for obtaining these potentially life-saving drugs is to request that the FDA grant them expanded access. SB 149, otherwise known as “Right-to-Try”, allows a terminally ill patient a last-ditch effort to save their life. This bill allows a patient to try an investigational drug only when certain conditions have been met, while providing important legal protections for all parties involved.

FISCAL EFFECT: Appropriation: No Fiscal Com.: Yes Local: No

According to the Senate Appropriations Committee:

• One-time costs of $20,000 and ongoing costs of $10,000 per year to review plan filings by the Department of Insurance (Insurance Fund).

• Increased costs of $380,000 in 2015-16 to respond to requests for information about prior independent medical review decisions and ongoing costs of $210,000 per year to respond to consumer complaints and requests for independent medical review for access to investigational drugs by the Department of Managed Health Care (Managed Care Fund).

• Uncertain impact on state-funded health CalPERS health care coverage (General Fund and special funds).

SUPPORT: (Verified 5/28/15)

City of Murrieta

Union of American Physicians & Dentists

OPPOSITION: (Verified 5/28/15)

Association of Northern California Oncologists

California Medical Association

California Nurses Association

Prepared by: Melanie Moreno / HEALTH /

5/31/15 12:53:01

**** END ****

SENATE COMMITTEE ON APPROPRIATIONS

Senator Ricardo Lara, Chair

2015 - 2016 Regular Session

SB 149 (Stone) - Investigational drugs, biological products, or devices: right to try

This bill meets the criteria for referral to the Suspense File.

Bill Summary:

SB 149 would authorize the manufacturer of an investigational drug to make an investigational drug available to a patient, under certain circumstances.

Fiscal Impact:

• One-time costs of $20,000 and ongoing costs of $10,000 per year to review plan filings by the Department of Insurance (Insurance Fund).

• Increased costs of $380,000 in 2015-16 to respond to requests for information about prior independent medical review decisions and ongoing costs of $210,000 per year to respond to consumer complaints and requests for independent medical review for access to investigational drugs by the Department of Managed Health Care (Managed Care Fund).

• Uncertain impact on state-funded health CalPERS health care coverage (General Fund and special funds). (See below.)

Background:

Under federal law and regulation, drug manufacturers must apply to the federal Food and Drug Administration for permission to market drugs. The application process includes a series of clinical trials to demonstrate the safety and effectiveness of new drugs. The clinical trial process includes a variety of requirements regarding informed consent for participants and reporting requirements on manufacturers. Current federal law also includes a process whereby seriously ill patients can access investigational drugs before they have been approved for general use and without the patient needing to be a participant in a clinical trial. In recent years, concerns have been raised that the process for approving such requests is slow and cumbersome for physicians, discouraging them from seeking such access for their seriously ill patients.

Current state law generally prohibits the sale or giving away of new drugs unless the new drug has been approved by the state Department of Public Health or the drug has been approved for use by the federal Food and Drug Administration (including premarket approval). State law authorizes the experimental use of drugs and requires certain patient protections, such as oversight by a review committee and informed consent for the patient.

Current state law authorizes patients to seek an independent medical review when a health plan or health insurer denies coverage for an experimental or investigational drug when the patient has a life-threatening or seriously debilitating condition, the patient’s physician indicates that standard therapies have not been effective, and the patient’s physician has recommended an experimental or investigational drug. If the independent medical review finds that the required conditions are met, the health plan or health insurer is required to provide coverage for the experimental or investigational drug.

Statute governing the state’s Medi-Cal program generally limits the coverage for experimental drugs and prohibits coverage of experimental drugs if the experimental drug costs more than conventional therapies.

Proposed Law:

SB 149 would authorize the manufacturer of an investigational drug to make an investigational drug available to a patient, under certain circumstances.

Specific provisions of the bill would:

• Authorize a manufacturer of an investigational drug, biological product, or device to make such product available to an eligible patient;

• Authorize a manufacturer to make an investigational product available to a patient without compensation, to require the patient to pay the costs of manufacturing the product, and/or require an eligible patient to participate in data collection;

• Specify that the bill does not require a health plan or state agency to provide coverage for the costs of an investigational product;

• Authorize a health plan to provide coverage for an investigational product;

• Prohibit a state regulatory board from taking disciplinary action against a physician based on the physician’s recommendation that a patient use an investigational product;

• Prohibit a state agency from taking regulatory action against a health facility based on a facility’s participation in treatment using an investigational product;

• Require participating manufacturers to report specified data to the Department of Public Health.

Related Legislation:

• SB 715 (Anderson) is substantially similar to this bill. That bill is pending in the Senate Health Committee.

• AB 159 (Calderon) is substantially similar to this bill. That bill is on the Assembly Floor.

Staff Comments:

The bill would authorize a manufacturer to make investigational drugs available to patients under limited circumstances. The bill itself does not require a patient’s health plan to provide coverage for the cost of the investigational drug. However, state law requires health plans to provide coverage for investigational drugs under certain circumstances. If a health plan denies coverage for an investigational drug, an enrollee can request an independent medical review of that decision. Based on the independent medical review, a health plan may be required to provide coverage for an investigational drug. It is possible that patients could use the independent medical review process to access coverage for investigational drugs provided by manufacturers under this bill. To the extent that patients enrolled in coverage provided by CalPERS do so, there could be additional costs to the state and/or local governments.

-- END --

SENATE COMMITTEE ON

BUSINESS, PROFESSIONS AND ECONOMIC DEVELOPMENT

Senator Jerry Hill, Chair

2015 - 2016 Regular

Bill No: SB 149 Hearing Date: April 27, 2015

Subject: Investigational drugs, biological products, or devices: right to try.

SUMMARY: Enacts the Right to Try Act, and permits a manufacturer of an investigational drug, biological product, or device to make an investigational drug, biological product, or device available to an eligible patient as defined.

NOTE: This bill was referred to the Senate Committee on Health first, and was passed out of that Committee on April 22, 2015 by a vote of 7-0.

Existing federal law:

1. Establishes the United States Food and Drug Administration (FDA) to protect the public health by assuring the safety, effectiveness, quality, and security of human and veterinary drugs, vaccines and other biological products, and medical devices through the Food, Drug, and Cosmetic Act (FDCA). (21 United States Code (USC) § 301 et seq.)

2. Prohibits any new drug from being introduced into interstate commerce unless an application has been approved by the FDA. (21 USC § 505 (a))

3. Under regulation: a) Requires clinical trial sponsors to submit an Investigational New Drug (IND) application to the FDA for clinical investigation of a new drug or new indication of an approved drug, with certain exceptions; b) Requires review and approval from an Institutional Review Board (IRB) before a clinical study can be initiated under an IND; c) Defines an IRB as an appropriately constituted group that has been designated to review and monitor biomedical research involving human subjects, to ensure that a clinical trial is ethical and that the rights of study participants are protected; and, d) Authorizes an IRB to approve, require modifications in, or disapprove research, or to suspend or terminate approval of research that is not being conducted in accordance with the IRB's requirements or that has been associated with unexpected serious harm to subjects. (21 Code of Federal Regulations (CFR) Part 312)

4. Establishes the Office for Human Research Protections (OHRP), which provides leadership in the protection of the rights, welfare, and well-being of subjects involved in research conducted or supported by the U.S. Department of Health and Human Services (HHS). OHRP helps ensure this by providing clarification and guidance, developing educational programs and materials, maintaining regulatory oversight, and providing advice on ethical and regulatory issues in biomedical and social-behavioral research. (45 CFR Part 46)

Existing law:

1. Establishes the Sherman Food, Drug, and Cosmetic Law (Sherman Law), administered by the State Department of Public Health (DPH), which regulates the packaging, labeling, and advertising of drugs and devices. (Health and Safety Code (HSC) § 109875 et seq.)

2. Establishes parameters for the experimental use of drugs, requiring review by a committee for the protection of human subjects, consent by the patient or patient’s parent or guardian, and prohibits a person having an ownership interest in a skilled nursing facility or intermediate care facility from prescribing an experimental drug for a patient in the facility. (HSC §§ 11115 – 111545)

3. Prohibits, in the Sherman Law, the sale, delivery, or giving away of a new drug or device unless it is either:

1. A new drug or new device for which DPH has approved a new drug or device application, and has not withdrawn, terminated, or suspended that approval; or,

2. A new drug, and a new drug application has been approved for it by the FDA, pursuant to federal law, or it is a new device for which a premarket approval application has been approved, and that approval has not been withdrawn, terminated, or suspended under the FDA. (HSC § 111550)

4. Prohibits a person from manufacturing any drug or device in California unless he or she has a valid license from the DPH. (HSC § 111615)

5. Authorizes the DPH to require any manufacturer, wholesaler, or importer of any prescription ophthalmic device in California to obtain a license for each place of manufacture. (HSC §§ 111615 and 111620)

6. Establishes the Protection of Human Subjects in Medical Experimentation Act which prescribes various protections for subjects of medical experimentation relating to a bill of rights; informed consent procedures and documentation; and, the provision of specified disclosures, including the right for a subject to give or withdraw consent freely and without duress. Imposes penalties for violations of these protections. (HSC § 24170 et seq.)

7. States that any person who violates any provision of the Sherman Law, if convicted, is subject to imprisonment for not more than one year in the county jail or a fine of not more than $1,000, or both the imprisonment and fine. States that any person who violates the law by removing, selling, or disposing of an embargoed food, drug, device, or cosmetic without the permission of an authorized agent of the department or court shall, if convicted, be subject to imprisonment for not more than one year in a county jail or a fine of not more than ten thousand dollars ($10,000), or both the fine and imprisonment. States that any person who purchases or sells a foreign dangerous drug or medical device, an illegitimate product, or suspect product, that is not approved or otherwise authorized by the FDA or that is obtained outside of the licensed supply chain regulated by the FDA, California State Board of Pharmacy, or DPH, is guilty of a misdemeanor and subject to imprisonment for not more than one year in a county jail, a fine of not more than ten thousand dollars ($10,000) per occurrence, or both the imprisonment and fine. (HSC §111825)

8. Requires health plans and insurers to provide an external, independent review process to examine plan's coverage denials of experimental or investigational therapies for individual enrollees who have a life-threatening or seriously debilitating condition and who meet other specified criteria. (HSC § 1370.4)

9. Requires health plans and insurers to provide coverage for all routine patient care costs relative to the treatment of an enrollee or insured diagnosed with cancer and accepted in an FDA-approved cancer clinical trial, Phase I-IV, if the enrollee's treating physician, recommends participation in the clinical trial after determining such participation has a meaningful potential to benefit the enrollee or insured.
   (HSC § 1370.6)

10. Licenses and regulates physicians and surgeons under the Medical Practice Act (Act) by the Medical Board of California (MBC) within the Department of Consumer Affairs (DCA). (BPC § 2000 et seq.)

11. Provides that the MBC shall take action against a physician who is charged with unprofessional conduct, as specified. (BPC § 2234)

This bill:

1. Enacts the Right to Try Act (Act).

2. Makes the following definitions:

1. “Eligible patient” as a person who has a terminal illness as determined by that person’s physician and a consulting physician; his or her physician has determined that the person has no comparable or satisfactory FDA approved treatment options available to diagnose, monitor, or treat the disease or condition involved, and that the probable risk to the person from the investigational drug, biological product, or device (IDBPD) is not greater than the probable risk from the disease or condition; he or she has received a prescription or recommendation from his or her physician for an IDBPD; he or she has given written informed consent for the use of the IDBPD, or if he or she is a minor or lacks the capacity to provide informed consent, his or her parent, legal guardian, or legally authorized representative has given written informed consent on his or her behalf; he or she has documentation from his or her physician that the patient has met these requirements.

2. “Health benefit plan” as any plan or program that provides, arranges, pays for, or reimburses the cost of health benefits, including but not limited to, a health care service plan contract issued by a health care service plan, and a policy of health insurance issued by a health insurer.

3. “Health facility” as a facility, place, or building that is organized, maintained, and operated for the diagnosis, care, prevention, and treatment of human illness, physical or mental, including convalescence and rehabilitation and including care during and after pregnancy, or for any one or more of these purposes, for one or more persons, to which the persons are admitted for a 24-hour stay or longer, as specified.

4. “Investigational drug, biological product, or device” as a drug, biological product, or device that has successfully completed phase one of a clinical trial approved by the FDA but has not been approved for general use by the FDA and remains under investigation in a clinical trial approved by the FDA.

5. “Physician” means a physician and surgeon licensed under the Act or an osteopathic physician and surgeon licensed under the Osteopathic Act who is providing medical care or treatment to the eligible patient for the terminal illness. Specifies that physician does not include a primary care physician.

6. “State regulatory board” means the MBC or the Osteopathic Medical Board of California (OMBC).

7. “Terminal illness” means a disease that, without life-sustaining procedures, will result in death in the near future or a state of permanent unconsciousness from which recovery is unlikely.

3. Authorizes a manufacturer of an IDBPD to make the IDBPD available to an eligible patient pursuant to the Act, but clarifies that a manufacturers is not required to make the IDBPD available to an eligible patient.

4. Authorizes a manufacturer to provide an IDBPD to an eligible patient without receiving compensation; requires an eligible patient to pay the costs of or associated with the manufacture of the IDBPD, and; requires an eligible patient to participate in data collection relating to the use of the IDBPD.

5. Authorizes a health benefit plan to provide coverage for an IDBPD, or device but, except as otherwise required by law, does not require a health benefit plan or any state agency to provide coverage for the cost of any IDBPD.

6. Prohibits a state regulatory board, notwithstanding any other law, from revoking, failing to renew, or taking any other disciplinary action against a physician’s license based solely on the physician’s recommendation to an eligible patient regarding, or prescription for, or treatment with, an IDBPD.

7. Prohibits a state agency, notwithstanding any other law, from taking any action against a health facility’s license based solely on the facility’s participation in the treatment by or use of an IDBPD.

8. Provides that a violation of the Act shall not be subject to penalties for other violations of provisions of the Sherman Act.

9. Clarifies that the Act does not create a private cause of action against a manufacturer of an IDBPD, or against any other person or entity involved in the care of an eligible patient using the IDBPD, for any harm to the eligible patient resulting from the IDBPD so long as the manufacturer or other person or entity complies in good faith with the terms of this article and exercises reasonable care.

FISCAL EFFECT: Unknown. This bill is keyed “fiscal” by Legislative Counsel.

COMMENTS:

1. Purpose. The Goldwater Institute is the Sponsor of this bill. According to the Author, this bill provides terminal patients a path to access potentially life-saving drugs, free of needless delays and a potential bureaucratic veto. The Author believes that the bill would give a patient, who has exhausted all other treatment options, a last-chance effort to save their life with an option to try an experimental drug that has passed Phase 1 of the FDA safety trials. According to the Author, the current process is fraught with deterrence, delays, and potential denials. The patient can attempt to enroll in a clinical trial, but many of the sickest individuals do not qualify to join such a trial. For these patients, their only hope for obtaining these potentially life-saving drugs is to request that the FDA grant them expanded access.

The Author states that only a few hundred patients are lucky enough to be granted individual expanded access each year because of the complicated, time-consuming, and expensive process currently required by the FDA. According to the Author, first, the patient must locate a doctor who is willing to complete the required paperwork which the FDA notes will take a minimum of 100 hours to complete. Few doctors have the skill or time necessary to complete such an undertaking, especially when their time is not reimbursed by insurance. Assuming the doctor is willing and able to complete the paperwork and the patient can cover the cost of doing so, the FDA then has a month to review the submission and either grant of deny the request.

The Author states that medical decisions, especially those of terminal patients, should be made by doctors and patients, not bureaucrats, and this bill ensures that terminal patients have the right to make their own medical decisions, while providing important legal protections for patients, doctors, insurance and pharmaceutical companies.

2. Background. According to an April 9, 2015 Health Policy Brief on the issue of right-to-try laws in Health Affairs, under current federal regulations, patients with serious or life-threatening illness have two primary options to access experimental therapies that may treat their condition but that have not yet been approved by the FDA: participate as a human subject in a clinical trial or, for patients who cannot be enrolled in that trial (because of their medical status or geographic location, for example), apply to the FDA for access to the experimental drug under the Expanded Access Program (also known as compassionate use) (EAP).

According to the brief, clinical testing of an experimental drug is typically a three-phase process. Phase I trials are small (20 to 80 patients) and are used primarily to evaluate safety and dosing ranges, usually in healthy volunteers. Phase II trials are larger (typically 100 to 300 patients) and are designed to show early evidence of efficacy in the patients that the drug is intended to treat. Phase III trials may include hundreds or thousands of patients and are used to demonstrate that the drug is effective compared to a control (such as a placebo or a comparator drug). Typically, a manufacturer submits an application to the FDA for marketing approval once a drug has successfully completed Phase III trials. Any drug company wishing to conduct a clinical trial must first submit an IND application to the FDA, which allows the company to manufacture the drug and ship it across state lines for use in the trial. The drug may be only administered to patients who are formally enrolled in that clinical trial. The study population for that trial may be limited based on any number of factors, including specific diagnosis, age, stage of illness, or comorbidities.

The EAP is an attempt at flexibility in the regulatory process and to allow patients with no other treatment options a chance to try therapies they may not otherwise have access to. The program allows patients who meet certain eligibility requirements to receive an experimental therapy outside of the formal clinical research process. An application for expanded access can be submitted by either the drug manufacturer or a licensed physician. There are three categories of expanded access: treatment, single patient, and intermediate, which are further split into two subcategories. One is “expanded access INDs,” through which the manufacturer submits a separate IND for a patient or group of patients, and the other is “expanded access protocols,” whereby the manufacturer amends the protocol under an existing IND to include the patient (or patients) seeking access. Under treatment INDs, a relatively large group of patients (hundreds or thousands) are permitted to access an experimental drug, provided that the sponsor is actively pursuing FDA approval and is in later stages of testing (or has already submitted trial results to the FDA for review).

According to an article published in the January 15, 2015 New England Journal of Medicine, the FDA has permitted almost all expanded access requests regardless of category. The FDA estimated that by 2006, approximately 100,000 patients had obtained expanded access to experimental drugs. Between 2010 and 2013, the FDA imposed clinical holds on only 2 of the 2,472 individual, non-emergency protocols, on 1 of 66 intermediate-size requests, and on none of the 41 widespread expanded-access protocols it received.

The Health Affairs brief states that critics of the EAP have argued that the application process is unnecessarily burdensome and lengthy (it is estimated that the IND application requires about 100 hours to complete), which discourages doctors and manufacturers from applying. An IRB review adds an additional layer of paperwork and potential delay. These requirements were put in place in response to highly publicized incidents of harm caused by unsafe drugs. In the past decade, there have been several attempts made at the federal and judicial levels to further relax restrictions on the administration of experimental therapies to terminally ill patients. One, led by the Abigail Alliance for Better Access to Developmental Drugs, submitted a Citizen Petition to the FDA in 2003 requesting that it make experimental therapies available to terminally ill patients, provided that the drug had passed Phase I testing. Following several years of litigation, the DC Court of Appeals ruled against the Abigail Alliance, stating that terminally ill patients have no constitutional right to access experimental therapies. The US Supreme Court subsequently declined to review the case. Several bills have been introduced in Congress that aim to relax FDA restrictions on access to experimental therapies. The most recent of these (HR 4475, the Compassionate Freedom of Choice Act of 2014) failed to make it out of committee.

In response to criticisms, the FDA recently sought to provide a streamlined alternative for submitting an Investigational New Drug Application (IND) for use in cases of individual patient expanded access and announced it would simplify and accelerate the application process for unapproved investigational drugs that have passed Phase I safety trials. It is estimated that the new application form will take 45 minutes to complete, asks attending physicians to fill in eight pieces of patient information, and requires only one attachment. The FDA issued draft guidance and included a copy of the new form, asking for comment until April 13, 2015. https://www.federalregister.gov/articles/2015/02/10/2015-02561/individual-patient-expanded-access-applications-form-fda-3926-draft-guidance-for-industry

3. Efforts in Other States. According to information provided by the Author, right to try laws are already in place in Arizona, Arkansas, Colorado, Indiana, Louisiana, Michigan, Mississippi, Missouri, Montana, North Dakota, Oklahoma, South Dakota, Utah, Virginia, and Wyoming. Lawmakers in Tennessee have sent a similar bill to their governor for approval. Twenty additional states are considering the law this year. Laws in many states generally allow terminally ill patients to have access to experimental drugs while others also allow health insurers to choose to provide coverage for the cost of these treatments and remove liability for physicians for recommending treatments.

4. Related Legislation This Year. SB 715 (Anderson) is substantively similar to this bill. (Status: The measure is currently pending in the Senate Committee on Health.)

SB 128 (Wolk) permits a competent, qualified individual who is a terminally ill adult to receive a prescription for aid in dying medication if certain conditions are met. (Status: This bill has been referred to the Senate Appropriations Committee.)

AB 159 (Calderon) permits a manufacturer of a drug, biological product, or device that has not yet received federal or state approval to market to make the product available to eligible patients with terminal illnesses, as specified. (Status: This measure has been referred to the Assembly Appropriations Committee.)

5. Arguments in Support. The Union of American Physicians and Dentists/American Federation of State, County and Municipal Employees (UAPD/AFSCME) supports this bill, stating that to a patient who is terminally ill, the current process to obtain potential life-saving drugs is stressful with delays and probable denials by insurance companies.

6. Concerns and Arguments in Opposition. The Los Angeles County Board of Supervisors (BOS) writes that it supports this bill if it is amended to provide comprehensive written, informed patient consent. According to the BOS, this bill does not provide patients with comprehensive, informed consent, which their County Counsel’s office deems necessary to help ensure that patients are fully aware of the potential risks involved with receiving experimental treatment.

The Pharmaceutical Research and Manufacturers of America (PhRMA) have concerns with this bill, writing that while “the bill is well-intentioned, it could have serious unintended consequences that must be taken into consideration” and adds that the bill may be premature given recent developments at the federal level. PhRMA states that right to try legislation is unlikely to achieve the goal of bringing innovative, safe and effective medicines to patients as quickly as possible and cites the current robust nature of the current clinical trial process, the FDA’s current EAP, and efforts underway to streamline the EAP as reasons for concerns.

The Association of Northern California Oncologists (ANCO) is opposed to this bill, citing the difficulty of identifying terminally ill patients, the danger of right to try outside of the context of a clinical trial, the lack of an informed consent process to protect patients and the lack of appropriate physician supervision of the use of these drugs. ANCO is concerned that any threat to adult clinical trial enrollment would result in slowing the development of cancer care in the future.

According to the California Medical Association (CMA) which is also opposed to this bill, offering unapproved therapies without credible scientific rationale or controlled monitoring could lead to not only endangering terminally ill patient lives further but potentially exploiting their hopes and circumstances, as the impeded person and their families may pay and try anything to save their lives. CMA notes that companies may not want to provide their treatments outside of FDA processes and states that it shares the Author’s goal of streamlining access to investigational drugs but only when the proper patient safety protections are in place.

7. Author’s Amendments Following the Discussion in the Senate Committee on Health. The Author advises that he plans to take the following amendments in response to concerns raised by members of the Senate Committee on Health. Specifically, the amendments will :

1. Establish a mechanism for the manufacturer to report data to the DPH on the following: the number of requests made for experimental drugs under the Act established by this bill, whether those requests were approved or denied, the use of such drugs, the duration of treatments, the success or failure of the drugs, any adverse side effects, or costs paid by patients for the drugs.

On page 5, after line 11, insert:

111546.4 (a) A manufacturer that provides an investigational drug to an eligible patient under 111546.2 shall report all of the following to the department:

(1) All requests for right-to-try medication.

(2) All approved requests for right-to-try medication.

(3) The duration of treatments

(4) The success or failure of the drug in treating the terminal illness for which the eligible patient was diagnosed.

(5) Any adverse event for each investigational drug.

(6) Costs paid by eligible patient for each investigational drug.

(7) The consulting physician’s diagnosis and prognosis, and verification that the eligible patient is competent, acting voluntarily, and has made an informed decision, or that the consulting physician has determined that the individual is not an eligible patient.

(b) The information collected shall be confidential and shall be collected in a manner that protects the privacy of the patient, the patient’s family, and any medical provider or pharmacist involved with the patient under the provisions of this part.

2. Conform the definition of terminal illness contained in SB 128 (Wolk) related to terminally ill patients receiving a prescription for aid in dying medication.

On page 4, line 3, after “Terminal”, strike “illness means a disease that, without”

On page 4, strike lines 4 through 6.

On page 4, after “Terminal”, insert “disease” means an incurable and irreversible disease that has been medically confirmed and will, within reasonable medical judgment, result in death within six months.

(g) “Terminal illness” means a disease that, without

life-sustaining procedures, will result in death in the near future

or a state of permanent unconsciousness from which recovery is

unlikely. disease” means an incurable and irreversible disease that has been medically confirmed and will, within reasonable medical judgment, result in death within six months.

The Author notes that he is still working to address concerns regarding informed consent and will take amendments on that subject at a future date.

SUPPORT AND OPPOSITION:

Support:

Union of American Physicians and Dentists/American Federation of State, County and

Municipal Employees (UAPD/AFSCME)

Concerns:

Los Angeles County Board of Supervisors

Pharmaceutical Research and Manufacturers of America (PhRMA)

Opposition:

Association of Northern California Oncologists

California Medical Association (CMA)

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SENATE COMMITTEE ON HEALTH

Senator Ed Hernandez, O.D., Chair

BILL NO: SB 149

SUBJECT: Investigational drugs, biological products, or devices: right to try

SUMMARY: Enacts the Right to Try Act, and permits a manufacturer of an investigational drug, biological product, or device to make available an investigational drug, biological product, or device to an eligible patient, as defined.

Existing state law:

1. Establishes the Sherman Food, Drug, and Cosmetic Law (Sherman Law), which regulates the packaging, labeling, and advertising of drugs and devices, administered by the Department of Public Health (DPH).

2. Prohibits, in the Sherman Law, the sale, delivery, or giving away of a new drug or device unless it is either:

1. A new drug or new device for which DPH has approved a new drug or device application, and has not withdrawn, terminated, or suspended that approval; or,

2. A new drug, and a new drug application has been approved for it by the U.S. Food and Drug Administration (FDA), pursuant to federal law, or it is a new device for which a premarket approval application has been approved, and that approval has not been withdrawn, terminated, or suspended under the FDA.

3. Establishes the Protection of Human Subjects in Medical Experimentation Act which prescribes various protections for subjects of medical experimentation relating to a bill of rights; informed consent procedures and documentation; and, the provision of specified disclosures, including the right for a subject to give or withdraw consent freely and without duress. Imposes penalties for violations of these protections.

4. Requires health plans and insurers to provide an external, independent review process to examine plan’s coverage denials of experimental or investigational therapies for individual enrollees who have a life-threatening or seriously debilitating condition and who meet other specified criteria.

5. Requires health plans and insurers to provide coverage for all routine patient care costs relative to the treatment of an enrollee or insured diagnosed with cancer and accepted in an FDA-approved cancer clinical trial, Phase I-IV, if the enrollee's treating physician, recommends participation in the clinical trial after determining such participation has a meaningful potential to benefit the enrollee or insured.

Existing federal law:

1. Establishes the federal Food, Drug, and Cosmetic Act, which grants authority to the FDA to oversee the safety of food, drugs, and cosmetics.

2. Prohibits any new drug from being introduced into interstate commerce unless an application has been approved by the FDA.

3. Under regulation: a) Requires clinical trial sponsors to submit an Investigational New Drug (IND) application to the FDA for clinical investigation of a new drug or new indication of an approved drug, with certain exceptions; b) Requires review and approval from an Institutional Review Board (IRB) before a clinical study can be initiated under an IND; c) Defines an IRB as an appropriately constituted group that has been designated to review and monitor biomedical research involving human subjects, to ensure that a clinical trial is ethical and that the rights of study participants are protected; and, d) Authorizes an IRB to approve, require modifications in, or disapprove research, or to suspend or terminate approval of research that is not being conducted in accordance with the IRB's requirements or that has been associated with unexpected serious harm to subjects.

4. Establishes the Office for Human Research Protections (OHRP), which provides leadership in the protection of the rights, welfare, and well-being of subjects involved in research conducted or supported by the U.S. Department of Health and Human Services (HHS). OHRP helps ensure this by providing clarification and guidance, developing educational programs and materials, maintaining regulatory oversight, and providing advice on ethical and regulatory issues in biomedical and social-behavioral research.

This bill:

1. Enacts the Right to Try Act, and permits a manufacturer of an investigational drug, biological product, or device to make available an investigational drug, biological product, or device to an eligible patient, as defined. Prohibits this bill from requiring a manufacturer make available an investigational drug, biological product, or device to an eligible patient.

2. Defines “investigational drug, biological product, or device” as a drug, biological product, or device that has successfully completed phase one of a clinical trial approved by the FDA, but has not been approved for general use and remains under investigation in an approved clinical trial.

3. Defines “eligible patient” as a person who:

1. Has a terminal illness as determined by that person’s physician and a consulting physician;

2. Has no comparable or satisfactory FDA-approved treatment options, as determined by his or her physician, available to diagnose, monitor, or treat the disease or condition involved, and that the probable risk to the person from the investigational drug, biological product, or device is not greater than the probable risk from the disease or condition;

3. Has received a prescription or recommendation from his or her physician for an investigational drug, biological product, or device;

4. Has given written informed consent for the use of the investigational drug, biological product, or device, or if he or she is a minor or lacks the capacity to provide informed consent, his or her parent, legal guardian, or legally authorized representative has given written informed consent on his or her behalf; and,

5. Has documentation from his or her physician that the patient has met the requirements of this bill.

4. Defines “terminal illness” as a disease that, without life-sustaining procedures, will result in death in the near future or a state of permanent unconsciousness from which recovery is unlikely.

5. Permits manufacturers to:

1. Provide an investigational drug, biological product, or device to an eligible patient without receiving compensation;

2. Require an eligible patient to pay the costs of or associated with the manufacture of the investigational drug, biological product, or device; and,

3. Require an eligible patient to participate in data collection relating to the use of the investigational drug, biological product, or device.

6. Specifies that this bill does not require a health plan or any state agency to provide coverage for the cost of any investigational drug, biological product, or device, but permits health plans to provide such coverage.

7. Prohibits a state regulatory board from revoking, failing to renew, or taking any other disciplinary action against a physician’s license based solely on the physician’s recommendation to an eligible patient regarding, or prescription for, or treatment with, an investigational drug, biological product, or device pursuant to this bill.

8. Prohibits a state agency from taking any action against a health facility’s license based solely on the facility’s participation in the treatment by or use of an investigational drug, biological product, or device pursuant to this bill.

9. Makes a violation of this bill not subject to the Sherman Food, Drug, and Cosmetic Law.

10. Specifies that this bill does not create a private cause of action against a manufacturer of an investigational drug, biological product, or device, or against any other person or entity involved in the care of an eligible patient using the investigational drug, biological product, or device, for any harm to the eligible patient resulting from the investigational drug, biological product, or device so long as the manufacturer or other person or entity complies in good faith with the terms of this bill and exercises reasonable care.

FISCAL EFFECT:

This bill has not been analyzed by a fiscal committee.

COMMENTS:

1. Author’s statement. According to the author, for terminal patients who have exhausted their conventional treatment options, obtaining access to potentially life-saving investigational drugs is often extremely difficult. The patient can attempt to enroll in a clinical trial, but many of the sickest individuals do not qualify to join such a trial. For these patients, their only hope for obtaining these potentially life-saving drugs is to request that the FDA grant them expanded access. SB 149, otherwise known as “Right-to-Try”, would allow a terminally ill patient a last-ditch effort to save their life. This bill would allow a patient to try an investigational drug only when certain conditions have been met, while providing important legal protections for all parties involved.

2. Background. According to an April 9, 2015 Health Policy Brief on the issue of right-to-try laws in Health Affairs, under current federal regulations, patients with serious or life-threatening illness have two primary options to access experimental therapies that may treat their condition but that have not yet been approved by the FDA: participate as a human subject in a clinical trial or, for patients who cannot be enrolled in that trial (because of their medical status or geographic location, for example), apply to the FDA for access to the experimental drug under the expanded access (also known as compassionate use) program.

3. Clinical trials. According to the Health Affairs brief, clinical testing of an experimental drug is typically a three-phase process. Phase I trials are small (20 to 80 patients) and are used primarily to evaluate safety and dosing ranges, usually in healthy volunteers. Phase II trials are larger (typically 100 to 300 patients) and are designed to show early evidence of efficacy in the patients that the drug is intended to treat. Phase III trials may include hundreds or thousands of patients and are used to demonstrate that the drug is effective compared to a control (such as a placebo or a comparator drug). Typically, a manufacturer submits an application to the FDA for marketing approval once a drug has successfully completed Phase III trials. Any drug company wishing to conduct a clinical trial must first submit an IND application to the FDA, which allows the company to manufacture the drug and ship it across state lines for use in the trial. The drug may be only administered to patients who are formally enrolled in that clinical trial. The study population for that trial may be limited based on any number of factors, including specific diagnosis, age, stage of illness, or comorbidities.

4. Expanded access program. According to the Health Affairs brief, the expanded access program is an attempt at flexibility in the regulatory process and to allow patients with no other treatment options a chance to try therapies they may not otherwise have access to. The program allows patients who meet certain eligibility requirements to receive an experimental therapy outside of the formal clinical research process. An application for expanded access can be submitted by either the drug manufacturer or a licensed physician. There are three categories of expanded access: treatment, single patient, and intermediate, which are further split into two subcategories. One is “expanded access INDs,” through which the manufacturer submits a separate IND for a patient or group of patients, and the other is “expanded access protocols,” whereby the manufacturer amends the protocol under an existing IND to include the patient (or patients) seeking access. Under treatment INDs, a relatively large group of patients (hundreds or thousands) are permitted to access an experimental drug, provided that the sponsor is actively pursuing FDA approval and is in later stages of testing (or has already submitted trial results to the FDA for review). According to an article published in the January 15, 2015 New England Journal of Medicine (NEJM), the FDA has permitted almost all expanded access requests regardless of category. The FDA estimated that by 2006, approximately 100,000 patients had obtained expanded access to experimental drugs. Between 2010 and 2013, the FDA imposed clinical holds on only 2 of the 2,472 individual, non-emergency protocols, on 1 of 66 intermediate-size requests, and on none of the 41 widespread expanded-access protocols it received.

The Health Affairs brief states that critics of the expanded access program have argued that the application process is unnecessarily burdensome and lengthy (it is estimated that the IND application requires about 100 hours to complete), which discourages doctors and manufacturers from applying. An IRB review adds an additional layer of paperwork and potential delay. These requirements were put in place in response to highly publicized incidents of harm caused by unsafe drugs. In the past decade, there have been several attempts made at the federal and judicial levels to further relax restrictions on the administration of experimental therapies to terminally ill patients. One, led by the Abigail Alliance for Better Access to Developmental Drugs, submitted a Citizen Petition to the FDA in 2003 requesting that it make experimental therapies available to terminally ill patients, provided that the drug had passed Phase I testing. Following several years of litigation, the DC Court of Appeals ruled against the Abigail Alliance, stating that terminally ill patients have no constitutional right to access experimental therapies. The US Supreme Court subsequently declined to review the case. Several bills have been introduced in the US Congress that aim to relax FDA restrictions on access to experimental therapies. The most recent of these (HR 4475, the Compassionate Freedom of Choice Act of 2014) failed to make it out of committee.

5. Other states. Over the past year, the issue of access to experimental therapies has been debated at the state level, in part due to efforts of the Goldwater Institute, a libertarian think tank that appears to be the source of this bill. In February 2014, the institute released a policy paper that outlines the major critiques of the FDA’s expanded access program and proposes model legislation for states to adopt. According to the National Conference of State Legislatures (NCSL), legislation related to right-to-try have been introduced in 30 states. Arizona, Colorado, Louisiana, Michigan, and Missouri have laws on the subject.

6. FDA action. According to an article in the Wall Street Journal published on February 9, 2015, the FDA announced it would “simplify and accelerate” the application process for “unapproved investigational drugs” that have passed Phase I safety trials. It is estimated that the new application form will take 45 minutes to complete, asks attending physicians to fill in eight pieces of patient information, and requires only one attachment. The FDA issued draft guidance and included a copy of the new form, asking for comment until April 13, 2015.

7. NEJM article. The authors of the January 2015 NEJM article mentioned above call right-to-try laws misguided, and call for a more pragmatic approach. They suggest that states work collaboratively with the FDA to make expanded access more practical when it is appropriate. For example, since the FDA has acknowledged that gaining approval from an IRB can pose a barrier, states could partner with the FDA to fund multicenter IRBs that focus specifically on expanded-access requests. Such multicenter panels would conduct full reviews, but their subject-matter expertise and limited dockets would translate into faster review times. Practical obstacles to enhancing expanded access programs, including administrative burdens and industry costs, would also be best tackled by the states in partnership with the FDA. For example, a manufacturer’s reluctance to provide product because of financial concerns could be addressed by permitting companies to charge amounts closer to the likely post approval cost of drugs.

8. Double referral. This bill is double referred. Should it pass out of this committee, it will be referred to the Senate Committee on Business and Professions and Economic Development.

9. Related legislation. SB 715 (Anderson), permits a manufacturer of a drug, biological product, or device that has not yet received federal or state approval to market to make the product available to eligible patients with terminal illnesses, as specified. SB 715 is pending in the Senate Health Committee

SB 128 (Wolk and Monning), permits a competent, qualified individual who is a terminally ill adult to receive a prescription for aid in dying medication if certain conditions are met, such as two oral requests, a minimum of 15 days apart, and a written request signed by two witnesses, is provided to his or her attending physician, the attending physician refers the patient to a consulting physician to confirm diagnosis and competency of the patient, and the attending physician refers the patient for counseling, if appropriate. SB 128 passed by a vote of 6-2 when it was heard in the Senate Health Committee and by a vote of 5-2 when it was heard in the Senate Judiciary Committee

AB 159 (Calderon), is substantially similar to this bill. AB 159 passed by a vote of 17-0 when it was heard in the Assembly Health Committee.

10. Support if amended. The Los Angeles County Board of Supervisors writes that that this bill does not provide patients with comprehensive written, informed consent, which their County Counsel’s office deems necessary to help ensure that patients are fully aware of the potential risks involved with receiving experimental treatment and will support this bill if it is amended to provide such consent.

11. Opposition. The California Medical Association states that they have significant patient safety concerns with allowing access to unproven drugs outside of the FDA’s clinical trials and compassionate use programs. They further argue that offering unapproved therapies without credible scientific rationale or controlled monitoring could lead to not only endangering terminally ill patients further but potentially exploiting their hopes and circumstances. The Association of Northern California Oncologists cite the difficulty of identifying a terminally-ill patient, the danger a “right to try” policy outside the context of a clinical trial would present to adult clinical trial enrollment and the lack of an informed consent process to protect patients seeking investigational drugs as reasons for establishing their oppose position on the bill. The California Nurses Association/National Nurses United (CNA) maintain that this bill does nothing to address the real barriers to “compassionate use” revealed in a recent article in the NEJM entitled Practical, Legal, and Ethical Issues in Expanded Access to Investigational Drugs, specifically stating that nothing in this legislation impacts the availability of drugs to terminally ill patients unless the manufacturer of the drugs allow it to be used in advance of FDA approval.  The CNA goes on to state instead of taking on the cost of drugs and challenging drug manufacturers that charge excessive prices for all drugs sold in California, this bill reinforces the status quo for investigational drug costs.

12. Policy comment. This bill does not contain any mechanism to report data on the number of requests made for experimental drugs under this bill, whether those requests were approved or denied, the use of such drugs, the duration of treatments, the success or failure of the drugs, any adverse side effects, or costs paid by patients for the drugs. While the bill does permit a drug company to require a patient to participate in data collection, this is not mandatory, and even if it were, there is no requirement for a drug company to share this data with policy makers. Should the state move forward with allowing the use of experimental drugs outside of the clinical trial process, the Committee may wish to consider whether there should be some regulatory oversight, or at a minimum, some data collection and reporting, and perhaps a sunset date.

SUPPORT AND OPPOSITION

Support: None received.

Oppose: Association of Northern California Oncologists

California Medical Association

California Nurses Association

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