BILL ANALYSIS Ó
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|SENATE RULES COMMITTEE | AB 159|
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THIRD READING
Bill No: AB 159
Author: Calderon (D), et al.
Amended: 7/6/15 in Senate
Vote: 21
SENATE HEALTH COMMITTEE: 8-0, 6/10/15
AYES: Hernandez, Nguyen, Hall, Mitchell, Monning, Pan, Roth,
Wolk
NO VOTE RECORDED: Nielsen
SENATE BUS, PROF. & ECON. DEV. COMMITTEE: 8-0, 6/29/15
AYES: Hill, Bates, Block, Galgiani, Hernandez, Jackson,
Mendoza, Wieckowski
NO VOTE RECORDED: Berryhill
SENATE APPROPRIATIONS COMMITTEE: 7-0, 8/27/15
AYES: Lara, Bates, Beall, Hill, Leyva, Mendoza, Nielsen
ASSEMBLY FLOOR: 74-2, 5/18/15 - See last page for vote
SUBJECT: Investigational drugs, biological products, and
devices
SOURCE: Author
DIGEST: This bill 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 that a manufacturer make available an investigational
drug, biological product, or device to an eligible patient.
ANALYSIS:
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Existing 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). 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new drug or device application, and has not withdrawn,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and that approval has not been withdrawn, terminated, or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.
2) 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.
This bill:
1) 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 that 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 by the
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United States Department of Agriculture and remains under
investigation in a clinical trial approved by the FDA.
3) Defines "eligible patient" as a person who:
a) Has an immediately life-threatening disease or
condition. 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;
b) Has considered all other treatment options currently
approved by the FDA;
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 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 participate in that
clinical trial due to the patient's current condition and
stage of disease; and,
d) Has received a recommendation from his or her primary
physician and a consulting physician, as defined, for an
investigational drug, biological product, or device.
Defines "consulting physician" as a physician and surgeon
or an osteopathic physician and surgeon who:
i) Examines the qualified individual and his or
her relevant medical records;
ii) Confirms, in writing, the primary physician's
diagnosis and prognosis; and,
iii) Verifies, in the opinion of the consulting
physician, that the eligible patient is competent,
acting voluntarily, and has made an informed decision.
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
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legally authorized representative has given written
informed consent on his or her behalf. Defines "written
informed consent" as a written document that has been
approved by the primary physician's IRB or an accredited
independent IRB, 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:
i) Explains the currently approved products and
treatments for the disease or condition from which the
patient suffers;
ii) Attests to the fact that the patient, or his
or her legally authorized representative, concurs with
the primary physician in believing that all currently
approved and conventionally recognized treatments are
unlikely to prolong the patient's life;
iii) Clearly identifies the specific proposed
investigational drug, biological product, or device
that the patient is seeking to use;
iv) Describes the potentially best and worst
outcomes of using the investigational drug, biological
product, or device and describes the most likely
outcome, as specified;
v) Clearly states that the patient's health plan
or insurer, 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;
vi) 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 eligibility requirements;
vii) Clearly states that in-home health care may be
denied if treatment begins; and,
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viii) 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.
4) Permits a manufacturer to provide an investigational drug,
biological product, or device to an eligible patient without
receiving compensation and to require an eligible patient to
pay the costs of or associated with the manufacture of the
investigational drug, biological product, or device.
5) Prohibits this bill from expanding coverage provided by
health plans or insurers, the Medi-Cal program, or county
organized health systems.
6) Specifies that this bill does not require a health plan or
insurer 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 under this bill, but
permits one to provide such coverage.
7) Prohibits an investigational drug, biological product, or
device from being offered if a clinical trial for that
investigational drug, biological product, or device is closed
due to the lack of efficacy or for toxicity. Requires the
manufacturer and the patient's primary physician, if notice
of closure of a clinical trial is given for an
investigational drug, biological product, or device taken by
a patient outside of a clinical trial, to notify the patient
of the information from the safety committee of the clinical
trial.
8) Prohibits the heirs of an eligible patient, if he/she dies
while being treated by an investigational drug, biological
product, or device made available pursuant to this bill, from
being liable for any outstanding debt related to the
treatment or lack of insurance for the treatment.
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9) 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 if the recommendation or
prescription is consistent with protocol approved by the
physician's IRB or an accredited independent institutional
review board.
10)Requires the physician's IRB or an accredited institutional
review board to biannually report to DPH, 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;
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 disease or
condition, as specified; and,
f) Any adverse event for each investigational drug,
biological product, or device.
11)Prohibits a state agency from altering 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.
12)Makes a violation of this bill not subject to the Sherman
Law.
13)Specifies that this bill does not create a private cause of
action, and that actions taken pursuant to this bill do not
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serve as a basis for a civil, criminal, or disciplinary claim
or cause of action, including, but not limited to, product
liability, medical negligence, or wrongful death, 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 for harm done to the eligible
patient or his or her heirs resulting from the
investigational drug, biological product, or device, or the
use or nonuse thereof, if the manufacturer or other person or
entity has complied with the terms of this article in
relation to the eligible patient, unless there was a failure
to exercise reasonable care.
Comments
1)Author's statement. According to the author, terminally ill
patients often do not have the luxury of waiting for the FDA
to grant compassionate use or 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 protecting physicians,
hospitals, and manufacturers from retribution.
2)Background. According to an April 9, 2015, Health Policy
Brief on the issue of right-to-try laws in Health Affairs,
under current federal regulations, patients with serious or
life-threatening illness have two primary options to access
experimental therapies that may treat their condition but that
have not yet been approved by the FDA: participate as a human
subject in a clinical trial or, for patients who cannot be
enrolled in that trial (because of their medical status or
geographic location, for example), apply to the FDA for access
to the experimental drug under the expanded access (also known
as compassionate use) program.
3)Clinical trials. According to the 2015 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
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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).
Typically, a manufacturer submits an application to the FDA
for marketing approval once a drug has successfully completed
Phase III trials. The drug may be only administered to
patients who are formally enrolled in that clinical trial. The
study population for that trial may be limited based on any
number of factors, including specific diagnosis, age, stage of
illness, or comorbidities.
4)Expanded access program. According to the 2015 Health Affairs
brief, the expanded access program is an attempt at
flexibility in the regulatory process and to allow patients
with no other treatment options a chance to try therapies they
may not otherwise have access to. The program allows patients
who meet certain eligibility requirements to receive an
experimental therapy outside of the formal clinical research
process. 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 two of the 2,472 individual, non-emergency protocols,
on one 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
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discourages doctors and manufacturers from applying. An IRB
review adds an additional layer of paperwork and potential
delay. These requirements were put in place in response to
highly publicized incidents of harm caused by unsafe drugs. In
the past decade, there have been several attempts made at the
federal and judicial levels to further relax restrictions on
the administration of experimental therapies to terminally ill
patients. One, led by the Abigail Alliance for Better Access
to Developmental Drugs, submitted a Citizen Petition to the
FDA in 2003 requesting that it make experimental therapies
available to terminally ill patients, provided that the drug
had passed Phase I testing. Following several years of
litigation, the D.C. Court of Appeals ruled against the
Abigail Alliance, stating that terminally ill patients have no
constitutional right to access experimental therapies.
5)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.
The FDA is now in the process of reviewing the public
comments and will not likely finalize the guidance before
early fall.
NOTE: Please refer to the Senate Health Committee analysis for
detailed background on this bill.
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).
One-time 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
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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 California Public Employees'
Retirement System health care coverage (General Fund and
special funds).
SUPPORT: (Verified8/28/15)
ALS Association Golden West Chapter
Los Angeles County Board of Supervisors
OPPOSITION: (Verified8/28/15)
Association of Northern California Oncologists
California Nurses Association
California Right to Life Committee, Inc.
ASSEMBLY FLOOR: 74-2, 5/18/15
AYES: Achadjian, Alejo, Travis Allen, Baker, Bigelow, Bloom,
Bonilla, Bonta, Brough, Brown, Burke, Calderon, Campos, Chang,
Chau, Chávez, Chiu, Chu, Cooley, Cooper, Dababneh, Dahle,
Daly, Dodd, Eggman, Frazier, Beth Gaines, Gallagher, Cristina
Garcia, Eduardo Garcia, Gatto, Gipson, Gomez, Gonzalez, Gray,
Grove, Hadley, Harper, Roger Hernández, Holden, Irwin, Jones,
Jones-Sawyer, Lackey, Linder, Lopez, Low, Maienschein, Mayes,
McCarty, Medina, Mullin, Nazarian, Obernolte, O'Donnell,
Olsen, Patterson, Perea, Rendon, Ridley-Thomas, Rodriguez,
Salas, Santiago, Steinorth, Mark Stone, Thurmond, Ting,
Wagner, Waldron, Weber, Wilk, Williams, Wood, Atkins
NOES: Levine, Quirk
NO VOTE RECORDED: Gordon, Kim, Mathis, Melendez
Prepared by:Melanie Moreno / HEALTH /
8/31/15 15:12:22
**** END ****
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