BILL ANALYSIS Ó
AB 159
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Date of Hearing: April 21, 2015
ASSEMBLY COMMITTEE ON BUSINESS AND PROFESSIONS
Susan Bonilla, Chair
AB 159
Calderon - As Amended April 13, 2015
NOTE: This bill is double referred, having been previously heard
by the Assembly Committee on Health on April 14, 2015 and
approved on a 17-0 vote.
SUBJECT: Investigational drugs, biological products, and
devices.
SUMMARY: Permits a manufacturer of an investigational drug,
biological product or device to make the product available to
eligible patients, with a serious or immediately
life-threatening disease or condition, and authorizes a health
benefit plan to provide coverage; prohibits the Medical Board
of California and the Osteopathic Medical Board of California
from taking any disciplinary action against a licensee based
solely on the physician's recommendation, prescription of a
biological product or device or treatment of a patient with such
products or devices; prohibits a state agency from altering any
recommendation made to the federal Centers for Medicare and
Medicaid Services regarding a provider's certification to
participate in the programs based solely on the recommendation
that a patient have access to a drug, product or device; and,
prohibits an official, employee or agent of the state from
blocking an eligible patient's access to the drug, product or
device.
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EXISTING LAW:
The Business and Professions Code
1)Establishes the Medical Board of California (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 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new drug or device application, and has not withdrawn,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,
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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,
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.
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2)Prohibits any new drug from being introduced into interstate
commerce unless an application has been approved by the FDA.
3)Under federal 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 wellbeing of subjects involved in research
conducted or supported by the U.S. Department of Health and
Human Services (HHS).
THIS BILL:
1)Allows the manufacturer of an experimental drug or device to
provide an investigational drug or device to an eligible
patient.
2)Specifies that the manufacturer may provide for free, or may
charge the patient to pay for the costs of the drug or device.
3)Defines "eligible patient" as one who has met all the
following criteria:
a) Has a serious or immediately life-threatening disease or
condition;
b) Has considered all other treatment options currently
approved by the FDA;
c) Has been unable to participate in a clinical trial
within 100 miles of their home, or has not been accepted;
d) Has received recommendation from their physician for the
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drug or device;
e) Has given written, informed consent; and,
f) Has documentation form their physician attesting that
the patient has met these requirements.
4)Defines "health benefit plan" as any plan or program that
provides, arranges, pays for or reimburses the cost of health
benefits and includes, but is 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.
5)Defines "immediately life threatening disease or condition" to
mean a stage of disease in which there is a reasonable
likelihood that death will occur within a matter of months or
in which premature death is likely without early treatment.
6)Defines "serious disease or condition" to mean a disease or
condition associated with morbidity that has a substantial
impact on day-to-day functioning.
7)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 U.S.
FDA, but has not been approved for general use by the FDA and
remains under investigation in a clinical trial approved by
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the FDA.
8)Defines "physician" as a physician and surgeon licensed under
the Medical Practice Act or an osteopathic physician and
surgeon licensed under the Osteopathic Act.
9)Defines "state regulatory board" as the MBC and the OMB.
10)Defines "written, informed consent" as a written document
that has been approved by the physician's IRB or an accredited
independent institutional review board, is signed by an
eligible patient, or his or her legally authorized
representative where the patient lacks the capacity to
consent, and attested to by the patient's physician and a
witness that, at a minimum, does all of the following:
a) Explains the currently approved products and treatments
for the serious or immediately life-threatening disease or
condition from which the patient suffers;
b) Attests to the fact that the patient, or where the
patient lacks the capacity to consent, his or her legally
authorized representative, concurs with the patient's
physician in believing that all currently approved and
conventionally recognized treatments are unlikely to
prolong the patient's life;
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c) Clearly identifies the specific proposed investigational
drug, biological product or device that the patient is
seeking to use;
d) Describes the potentially best and worst outcomes of
using the investigational drug, biological product or
device and describes the most likely outcome;
e) States that the patient's health benefit plan and health
care provider are not obligated to pay for the
investigational drug, biological product or device or any
care or treatments consequent to use of the them;
f) States that the patient's eligibility for hospice care
may be withdrawn if the patient begins curative treatment
and that care may be reinstated if the curative treatment
ends and the patient meets hospice eligibility
requirements;
g) States that in-home health care may be denied if
treatment begins; and,
h) States that the patient understands and is liable for
all expenses consequent to the use of the investigational
drug, biological product or device.
11)Allows a health plan to provide coverage for the drug or
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device, or the costs of services related to the use of the
drug or device.
12)Prohibits a state regulatory board form revoking, failing to
renew, or taking any other disciplinary action against a
physician's license based on the recommendation, prescription,
or treatment of the drug or device, provided the
recommendation or prescription is consistent with the medical
standards of care.
13)Indicates that written, informed consent shall be consistent
with the informed consent requirements of the Protection of
Human Subjects in Medical Experimentation Act.
14)Specifics that health benefit plans are not required to
provide coverage for the cost or use of any investigational
drug, biological product or device or the costs of services.
15)States that if an eligible patient dies while being treated
by an investigational drug, biological product of device made
available pursuant to this article, the patient's heirs are
not liable for any outstanding debt related to the treatment
or lack of insurance for the treatment.
16)Prohibits a state agency from altering a recommendation made
to the federal Centers for Medicare and Medicaid Services
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regarding a health care provider's certification to
participate in the Medicare or Medicaid program based solely
on the recommendation from an individual health care provider
that a patient have access to the drug or device.
17)Prohibits an official, employee, or agent of the state from
blocking or attempting to block an eligible patient's access
to an investigational drug or device pursuant to this article.
Specifies that counseling, advice, or a recommendation
consistent with medical standards of care from a licensed
healthcare provider is not a violation of this section.
18)Specifies that this article does not create a private cause
of action against a manufacturer of an investigational drug or
device, or against any other person or entity involved in the
care of an eligible patient using the drug or device, for any
harm done to the patient resulting from the drug or device, so
long as the manufacturer or other person or entity is
complying in good faith with the terms of this article, unless
there was a failure to exercise reasonable care.
FISCAL EFFECT: Unknown. This bill is keyed fiscal by the
Legislative Counsel.
COMMENTS:
1)Purpose. This bill is sponsored by the author. According to
the author, "Terminally ill patients often do not have the
luxury of waiting for the FDA to grant compassionate use of
participating in the lengthy process of clinical trials. AB
159 gives terminally ill patients a chance to try potentially
life-saving treatments after all other options have been
exhausted. It removes barriers for patients who need to
immediately obtain investigational treatments, while also
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protecting physicians, hospitals, and manufacturers from
retribution."
2)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
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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 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.
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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 investigational drugs, biological products
or devices that they recommend to patients or help them gain
access to. The Compassionate Access Act of 2010 (H.R. 4732)
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sought to address this issue by providing immunity to the
manufacturer, distributor, administrator, sponsor or physician
from suit or liability.
If AB 159 is enacted, the MBC and OMB would be prohibited from
taking action against physician's license for prescribing or
treating a patient with an investigational drug, biological
product or device.
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.
3)Prior 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.
SB 149 (Stone) of the current legislative session, is largely
similar to this bill, with differences primarily in
definitions and liability exemptions. STATUS: SB 149 is
pending in the Senate Business, Professions and Economic
Development Committee.
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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.
ARGUMENTS IN SUPPORT:
The Los Angeles County Board of Supervisors writes in its letter
of support, "Five states, Colorado, Louisiana, Missouri,
Michigan and Arizona, have recently enacted bi-partisan
legislation or voter-approved ballot initiatives to establish
"Right to Try" laws. Terminally ill patients, who have
exhausted their options to find a cure and who have identified a
physician and pharmaceutical company willing to assist them,
deserve the right to experimental treatments that could prolong
their lives."
ARGUMENTS IN OPPOSITION:
The California Medical Association opposes the bill and writes,
"While we appreciate the good intentions behind the bill, we
have significant patient safety concerns with allowing access to
unproven drugs outside of 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?In addition, since state law can't
supersede federal law, companies may not want to provide their
treatments outside of FDA processes. The key to current
processes is that they be uniformly regulated and monitored to
ensure both proper participation and consumer protection."
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The California Nurses Association/National Nurses United opposes
the bill and writes in their letter, "While apparently well
meaning, this legislation promises vulnerable patients and their
families that there will be a state program for obtaining drugs
that is similar to the FDA expanded access program only shorter.
This short cut is purportedly a means of increasing access to
drugs and devices for terminally ill patients. This bill would
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
physician's recommendation to an eligible patient for
prescription or treatment with the investigations drug or
device. This bill also purports to provide protection by
prohibiting a state agency from altering any recommendation made
to the federal Medicare and Medicaid program for these
activities."
The Association of Northern California Oncologists (ANCO) also
opposes the bill and states, "The ANCO Board cited several
reasons to oppose AB 159, including the difficulty of
identifying "terminally-ill" patient, the danger a "right to
try" outside the context of a clinical trial would present to
adult clinical trial enrollment, the lack of an informed consent
process to protect patients seeking investigational drugs, and
the lack of appropriate physician supervision of the use of
these drugs. Despite very low adult clinical trial enrollments,
this nation's clinical trial system has resulted in the
relatively rapid evolution of cancer care. Any threat to adult
clinical trial enrollment would result in slowing the
development of cancer care in the future."
AMENDMENTS:
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1)As was noted in the Assembly Committee on Health's analysis,
the bill does not require any agency or company to track
whether patients are able to acquire the experimental drugs or
devices they are seeking. As such, the author should require
the IRB to send a report of the status or results of requests
made, the duration of treatments and costs paid by patients
for the experimental drugs or devices to the DPH, the MBC and
the OMB.
On page 6, after line 36 add the following:
(b) The physician's institutional review board or an
accredited institutional review board is required to send a
bi-annual report to the Department of Public Health, the
Medical Board of California and the Osteopathic Medical Board
with the following information:
(i) The number of requests made
(ii) The status of the requests made
(iii) The duration of treatment
(iv) The costs of the treatment paid by patients
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2)The bill specifies that a physician may prescribe a drug,
"provided that the recommendation or prescription is
consistent with the medical standards of care." However, when
a physician prescribes a medication for off-label use, they
are inherently deviating from the standards of care. Thus,
the language of the bill should specify that the standards of
care that the physician is adhering to are those that are
approved by the institutional review board. To this end, the
author should amend the bill as follows:
On page 6, line 36, strike the following, medical standards of
care and insert the following, protocol as approved by the
institutional review board .
3)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 6, line 25, add the following:
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
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safety committee of the clinical trial.
4)In order to confirm the patient's diagnosis of a terminal
illness, the author should amend the bill to require the
primary physician to retain verification from a consulting
physician of the patient's diagnosis and prognosis.
On page 3, line 22, add the following after "her":
primary physician and a consulting physician
On page 3, line 29, add the following after "her":
primary physician and a consulting physician
On page 4, line 17, add:
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"Primary "P p hysician"
On page 4, after line 19 add the following:
"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, that
the eligible patient is competent, acting voluntarily, and has
made an informed decision.
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REGISTERED SUPPORT:
Los Angeles County Board of Supervisors
REGISTERED OPPOSITION:
California Medical Association
California Nurse Association/National Nurses United
Association of Northern California Oncologists
Analysis Prepared by:Le Ondra Clark Harvey, Ph.D. / B. & P. /
(916) 319-3301
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