BILL ANALYSIS Ó
SB 149
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Date of Hearing: July 7, 2015
ASSEMBLY COMMITTEE ON BUSINESS AND PROFESSIONS
Susan Bonilla, Chair
SB 149(Stone) - As Amended 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:
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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:
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new drug or device application, and has not withdrawn,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approval has not been withdrawn, terminated or suspended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
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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.
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:
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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 wellbeing of subjects involved in research
conducted or supported by the U.S. Department of Health and
Human Services (HHS).
THIS BILL:
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1) Enacts the Right to Try Act (Act) and makes the following
definitions:
a) "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.
b) "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.
c) "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
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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"
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.
e) "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.
f) "State regulatory board" means the MBC or the OMB.
g) "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.
1) 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.
2) Authorizes a manufacturer to provide an IDBPD to an eligible
patient without receiving compensation; requires an eligible
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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,
but 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
Law.
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.
8) Requires a manufacturer that provides an IDBPD to an eligible
patient to report the following information to the State
Department of Public Health:
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a) The number of requests made for an IDBPD;
b) The number of requests that were approved;
c) The duration of treatments;
d) The success of failure of the IDBPD in treating the
terminal disease with which the eligible patient was
diagnosed;
e) Any adverse event for each IDBPD;
f) Costs paid by each eligible patient for each
investigation drug; and,
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 the person is
not an eligible patient.
9) 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.
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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
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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
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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
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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.
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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:
a. 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.
b. 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.
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c. 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
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proposed by this committee:
a. 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.
b. 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:
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(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)
c. 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:
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"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:
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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
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