BILL ANALYSIS Ó
SENATE COMMITTEE ON
BUSINESS, PROFESSIONS AND ECONOMIC DEVELOPMENT
Senator Jerry Hill, Chair
2015 - 2016 Regular
Bill No: SB 149 Hearing Date: April 27,
2015
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|Author: |Stone |
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|Version: |April 14, 2015 |
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|Urgency: |No |Fiscal: |Yes |
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|Consultant|Sarah Mason |
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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
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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:
a) A new drug or new device for which DPH has approved a
new drug or device application, and has not withdrawn,
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terminated, or suspended that approval; or,
b) 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)
1)Prohibits a person from manufacturing any drug or device in
California unless he or she has a valid license from the DPH.
(HSC § 111615)
2)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)
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.
(HSC § 24170 et seq.)
4)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
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thousand dollars ($10,000) per occurrence, or both the
imprisonment and fine. (HSC §111825)
5)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)
6)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)
7)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.)
8)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:
a) "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
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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.
b) "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.
c) "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.
d) "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.
e) "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.
f) "State regulatory board" means the MBC or the
Osteopathic Medical Board of California (OMBC).
g) "Terminal illness" means a disease that, without
life-sustaining procedures, will result in death in the
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near future or a state of permanent unconsciousness from
which recovery is unlikely.
1) 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.
2) 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.
3) 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.
4) 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.
5) 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.
6) Provides that a violation of the Act shall not be subject to
penalties for other violations of provisions of the Sherman
Act.
7) 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.
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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.
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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.
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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
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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
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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.
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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 :
a) 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
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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.
b) 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:
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Los Angeles County Board of Supervisors
Pharmaceutical Research and Manufacturers of America (PhRMA)
Opposition:
Association of Northern California Oncologists
California Medical Association (CMA)
-- END --