BILL ANALYSIS Ó
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|SENATE RULES COMMITTEE | SB 149|
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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. 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:
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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:
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. 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.
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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 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.
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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 disease 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 his or her behalf; and,
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e) 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:
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
Law.
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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:
a) The number of requests made for an investigational
drug, biological product, or device.
b) The number of requests that were approved.
c) The duration of treatments.
d) The success or failure of the investigational drug,
biological product, or device in treating the terminal
disease with which the eligible patient was diagnosed.
e) Any adverse event for each investigational drug,
biological product, or device.
f) Costs paid by each eligible patient for each
investigational drug.
g) 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.
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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
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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
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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
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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.:YesLocal: 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: (Verified5/28/15)
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City of Murrieta
Union of American Physicians & Dentists
OPPOSITION: (Verified5/28/15)
Association of Northern California Oncologists
California Medical Association
California Nurses Association
Prepared by:Melanie Moreno / HEALTH /
5/31/15 12:53:01
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