BILL ANALYSIS Ó
SENATE COMMITTEE ON HEALTH
Senator Ed Hernandez, O.D., Chair
BILL NO: SB 149
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|AUTHOR: |Stone |
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|VERSION: |April 14, 2015 |
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|HEARING DATE: |April 22, 2015 | | |
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|CONSULTANT: |Melanie Moreno |
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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 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:
a. 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withdrawn, terminated, or suspended that approval; or,
b. 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not been withdrawn, terminated, or suspended under the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,
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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.
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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:
a. Has a terminal illness as determined by that
person's physician and a consulting physician;
b. 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;
c. Has received a prescription or recommendation
from his or her physician for an investigational drug,
biological product, or device;
d. 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
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his or her behalf; and,
e. 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:
a. Provide an investigational drug, biological
product, or device to an eligible patient without
receiving compensation;
b. Require an eligible patient to pay the costs
of or associated with the manufacture of the
investigational drug, biological product, or device;
and,
c. 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,
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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
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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
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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
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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
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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
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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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