BILL ANALYSIS Ó
AB 1668
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Date of Hearing: March 29, 2016
ASSEMBLY COMMITTEE ON BUSINESS AND PROFESSIONS
Rudy Salas, Chair
AB 1668
(Calderon) - As Amended March 7, 2016
NOTE: This bill is double referred, having been previously heard
by the Assembly Committee on Health on March 15, 2016 and
approved on an 18-0 vote.
SUBJECT: Investigational drugs, biological products, and
devices.
SUMMARY: Permits a manufacturer of an investigational drug,
biological product or medical 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 Osteopathic Medical Board (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). Health and Safety Code (HSC) § 109875 et seq.
2)Prohibits the sale, delivery or giving away of a new drug or
device unless it is either: (HSC § 111550)
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),
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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. (HSC § 24170 et seq.)
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.
(HSC § 1370.4)
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. (HSC
§ 1370.6)
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. (Title 21 United States Code (USC) §
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321 et seq.)
2)Prohibits any new drug from being introduced into interstate
commerce unless an application has been approved by the FDA.
(21 USC § 355)
3)Under federal regulation: (Title 45 Code of Federal
Regulations (CFR) § 46.101 et seq.)
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). (45 CFR § §46.103)
THIS BILL:
1)Permits a manufacturer of an investigational drug, biological
product, or device may make available the manufacturer's
investigational drug, biological product, or device to an
eligible patient.
2)Specifies that a manufacturer may do both of the following:
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.
3)Defines "consulting physician" as a physician and surgeon
licensed under the Medical Practice Act or an osteopathic
physician and surgeon licensed under the Osteopathic Act who
performs the following:
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a) Examines the qualified individual and his or her
relevant medical records.
b) Confirms, in writing, the primary physician's diagnosis
and prognosis.
c) Verifies, in the opinion of the consulting physician,
that the eligible patient is competent, acting voluntarily,
and has made an informed decision.
4)Defines "eligible patient" as a person who meets all of the
following conditions:
a) Has an immediately life-threatening disease or
condition.
b) Has considered all other treatment options currently
approved by the United States Food and Drug Administration.
c) Has not been accepted to participate in the nearest
clinical trial to his or her home for the immediately
life-threatening disease or condition identified in
paragraph (1) within one week of completion of the clinical
trial application process, or, in the treating physician's
medical judgment, it is unreasonable for the patient to
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participate in that clinical trial due to the patient's
current condition and stage of disease.
d) Has received a recommendation from his or her primary
physician and a consulting physician for an investigational
drug, biological product, or device.
e) Has given written informed consent for the use of the
investigational drug, biological product, or device, or, if
he or she lacks the capacity to consent, his or her legally
authorized representative has given written informed
consent on his or her behalf.
f) Has documentation from his or her primary physician and
a consulting physician attesting that the patient has met
the requirements of this subdivision.
5)Defines "health benefit plan" as a plan or program that
provides, arranges, pays for, or reimburses the cost of health
benefits. "Health benefit plan" includes, but is not limited
to, a health care service plan contract issued by a health
care service plan, as defined in HSC § 1345, and a policy of
health insurance, as defined in § 106 of the Insurance Code,
issued by a health insurer.
6)Defines "immediately life-threatening disease or condition" as
a stage of disease in which there is a reasonable likelihood
that death will occur within a matter of months.
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7)Defines "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.
8)Defines "primary 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 Medical Board of
California or the Osteopathic Medical Board of California.
10)Defines "written, informed consent" means a written document
that has been approved by the primary physician's
institutional review board or an accredited independent
institutional review board, is signed by an eligible patient,
or his or her legally authorized representative when the
patient lacks the capacity to consent, and attested to by the
patient's primary physician and a witness that, at a minimum,
does all of the following:
a) Explains the currently approved products and treatments
for the immediately life-threatening disease or condition
from which the patient suffers.
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b) Attests to the fact that the patient, or when the
patient lacks the capacity to consent his or her legally
authorized representative, concurs with the patient's
primary physician in believing that all currently approved
and conventionally recognized treatments are unlikely to
prolong the patient's life.
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. This
description shall include the possibility that new,
unanticipated, different, or worse symptoms might result
and that death could be hastened by the proposed treatment.
The description shall be based on the primary physician's
knowledge of the proposed treatment in conjunction with an
awareness of the patient's condition.
e) Clearly states that the patient's health benefit plan,
if any, 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
investigational drug, biological product, or device.
f) Clearly 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
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eligibility requirements.
g) Clearly states that in-home health care may be denied if
treatment begins.
h) States that the patient understands that he or she is
liable for all expenses consequent to the use of the
investigational drug, biological product, or device, and
that this liability extends to the patient's estate, except
as otherwise provided in the patient's health benefit plan
or a contract between the patient and the manufacturer of
the drug, biological product, or device.
11)Requires that written, informed consent shall be consistent
with the informed consent requirements of the Protection of
Human Subjects in Medical Experimentation Act (HSC § 24170 et
seq.).
12)Specifies that a health benefit plan is not required to
provide coverage for the cost of any investigational drug,
biological product, or device, or the costs of services
related to the use of an investigational drug, biological
product, or device.
13)Indicates if the clinical trial for an investigational drug,
biological product, or device is closed due to the lack of
efficacy or for toxicity, the investigational drug, biological
product, or device shall not be offered. If notice of closure
of a clinical trial is given for an investigational drug,
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biological product, or device taken by a patient outside of a
clinical trial, the manufacturer and the patient's primary
physician shall notify the patient of the information from the
safety committee of the clinical trial.
14)Indicates if an eligible patient dies while being treated by
an investigational drug, biological product, or 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.
15)Prevents 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 if the recommendation or
prescription is consistent with protocol approved by the
physician's institutional review board or an accredited
independent institutional review board.
16)Requires the physician's institutional review board or an
accredited institutional review board to biannually report the
following information to the State Department of Public
Health, the Medical Board of California, and the Osteopathic
Medical Board of California:
a) The number of requests made for an investigational drug,
biological product, or device.
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b) The status of the requests made.
c) The duration of the treatment.
d) The costs of the treatment paid by eligible patients.
e) The success or failure of the investigational drug,
biological product, or device in treating the immediately
life-threatening disease or condition from which the
patient suffers.
f) Any adverse event for each investigational drug,
biological product, or device.
17)States that a state agency shall not alter any recommendation
made to the federal Centers for Medicare and Medicaid Services
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 an investigational drug,
biological product, or device.
18)Specifies that this bill does not create a private cause of
action, and that actions taken pursuant to this bill do not
serve as a basis for a civil, criminal, or disciplinary claim
or cause of action, as specified.
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FISCAL EFFECT: Unknown. This bill is keyed fiscal by the
Legislative Counsel.
COMMENTS:
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. [This
bill] 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
protecting physicians, hospitals, and manufacturers from
retribution."
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
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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 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
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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.
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.
This process includes a 60-day public comment period. The FDA
reviews the comments and makes revisions based on the comments.
The FDA then seeks approval from the Office of Management and
Budget, which includes an additional 30-day public comment
period, which commenced on March 10, 2016.
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
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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 the 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) sought to address
this issue by providing immunity to the manufacturer,
distributor, administrator, sponsor or physician from suit or
liability.
If AB 1668 is enacted, the MBC and the 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. Since 2014, several states have passed similar
legislation including: 1) Alabama, 2) Arkansas, 3) Colorado, 4)
Florida, 5) Indiana, 6) Louisiana, 7) Michigan, 8) Minnesota, 9)
Mississippi, 10) Missouri, 11) Montana, 12) Nevada, 13) North
Dakota, 14) Oklahoma, 15) South Dakota, 16) Tennessee, 17)
Texas, 18) Utah, 19) Virginia, and 20) Wyoming. Additionally,
Arizona passed a "Right to Try" ballot measure.
Current Related Legislation. SB 128 (Wolk) of the current
Legislative Session, permitted a competent, qualified individual
who is a terminally ill adult to receive a prescription for aid
in dying medication if certain conditions were met. STATUS:
This bill is pending in the Assembly Health Committee.
Prior Related Legislation. AB 159 (Calderon) of 2016 was
identical to this bill. NOTE: This bill was vetoed by Governor
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Brown. In his veto message he stated: patients with life
threatening conditions should be able to try experimental drugs,
and the FDA's compassionate use program allows this to happen.
The proposed changes to this program will streamline access to
these drugs. Before authorizing an alternative state pathway,
we should give this federal expedited process a chance to work.
SB 715 (Anderson) of 2015, is largely similar to this bill.
NOTE: SB 715 died in the Senate Health Committee.
SB 149 (Stone) of 2015, was largely similar to this bill, with
differences primarily in definitions and liability exemptions.
NOTE: SB 149 was held on the Senate Appropriations suspense
file.
ARGUMENTS IN SUPPORT:
The Amyotrophic Lateral Sclerosis (ALS) Association Golden West
Chapter supports the bill and writes in their letter, "The ALS
Association has a long history of supporting and advocating for
expanded access to experimental treatments for people living
with ALS. We are also champions of the research process, as
robust enrollment and participation in clinical trials is
essential to ultimately finding treatments and a cure for ALS."
The County of Los Angeles Board of Supervisors also supports the
bill and writes, "Patients with a life-threatening disease or
condition, who have exhausted their options to find a cure and
who have identified physicians and a pharmaceutical company
willing to assist them, deserve the right to experimental
treatments that could improve their health conditions or prolong
their lives."
The EveryLife Foundation for Rare Diseases strongly supports,
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"rational early access to safe and effective treatments for
patients battling life-threatening illnesses. Patients with
rare diseases and terminally ill patients have an urgent need
and deserve an opportunity for treatment, especially when the
risk of the disease far outweighs the risks of potential
treatments."
ARGUMENTS IN OPPOSITION:
The California Nurses Association/ National Nurses United
opposes the bill and writes, "This bill does not address the
real barriers to 'compassionate use' revealed in a recent
article in the esteemed New England Journal of Medicine.
Nothing in this legislation or any other legislation impacts the
availability of drugs to terminally ill patients unless the
manufacturers of the drugs allow it to be used in advance of FDA
approval. This legislation? is unlikely to withstand a
constitutional challenge based on conflict with the FDA's
enabling legislation and existing expanded-access regulation
under the Supreme Court's long-standing preemption doctrine."
The Medical Oncology Association of Southern California, Inc.
writes in their opposition letter, "Concerns with AB 1668
include the difficulty of identifying an 'immediately
life-threatening condition,' the lack of a sufficient informed
consent process to protect patients seeking investigational
drugs, and the lack of appropriate physician supervision of the
use of these drugs?While we appreciate the interest in
supporting patients, there are substantive dangers in a 'right
to try' outside the context of a clinical trial, including the
damage to adults clinical trial enrollment, which hold the
promise of the future of medicine. 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
solving the development of cancer care in the future."
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REGISTERED SUPPORT:
Amyotrophic Lateral Sclerosis (ALS) Association Golden West
Chapter
County of Los Angeles Board of Supervisors
EveryLife Foundation for Rare Diseases
REGISTERED OPPOSITION:
California Nurses Association/National Nurses United
Medical Oncology Association of Southern California, Inc.
Analysis Prepared by:Le Ondra Clark Harvey, Ph.D. / B. & P. /
(916) 319-3301
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