Transcript speaker materials

Right to Try: Evolution of State and
National Policy
2016 Michigan Research Ethics Conference
Tiana L. Korley, MHA, JD
April 29, 2016
Office of the Vice President
& General Counsel
300 North Ingalls Building, Suite 3B04
(734) 764-2178
Agenda

General Framework

Consideration of State Laws
 Overall landscape
 Michigan
 California
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 Stakeholder interest
Consideration of Federal Law
Where do we go from here?
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FDA actions
Congressional outlook
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General Framework

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Patient is terminally ill
Physician recommends use of a treatment
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Patient provides informed consent
Treatment has completed phase 1 testing
Liability protections
 Manufacturer

 Physician
No requirement for insurers to provide coverage
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Legal Framework

Supremacy Clause
 Introducing drugs into interstate commerce
without safe/effectiveness [Section 505 of FD&C
Act]

No challenge – yet

Tort liability – preempted?
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Ethical Framework
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Experimental nature given completion of only phase 1
testing
Potential financial commitment
Limited quantities – diversion from clinical trials

Dichotomy of terminal illness vs. non-terminal illness
 Multiple sclerosis
 Chronic pain
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Impact on protections for vulnerable population
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Institutional Review Board review/approval
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Overall Landscape

According to NCSL, as of March 31, 2015, 36 states
and DC are considering these laws; 12 signed laws

Unclear whether there is a demonstrable case for
facilitated access
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Michigan
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SB 991
Bill sponsor in Committee hearing: “[d]enying a
person the right to access drugs that might save their
own life flies in the face of our due process rights
embedded in the Constitution.”
Governor Snyder: “[a]llowing Michiganders dealing
with extremely difficult medical situations to try
alternative treatment options could extend or save
their lives.”
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California
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Veto of AB 159
“Patients with life threatening conditions should be
able to try experimental drugs, and the United States
Food and Drug Administration’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.”
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Stakeholder Interest
PhRMA
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2014 communication
Importance and relevance of clinical trial process
Expanded access programs as “commitment to
patients.”
RtT legislation as “well-intentioned” but that
bypassing FDA oversight and the clinical trials process
“…is not in the best interest of patients and public
health, and is unlikely to achieve our shared goal of
bringing innovative, safe and effective medicines to
patients as quickly as possible.”
Focus on modernizing the clinical trial, drug
development and FDA review process.
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Federal Law
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H.R. 3012
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Bars the federal government from prohibiting or
restricting the production, manufacture, distribution,
prescribing, dispensing, possession, or use of an
experimental drug, biological product, or device that
is: (1) intended to treat a patient who has been
diagnosed with a terminal illness; and (2) authorized
by, and in accordance with, state law.
 Arguably supplants the entire regulatory authority
of FDA
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Federal Law (cont’d)
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Defines an "experimental drug, biological product, or
device" as one that: (1) has successfully completed a
phase 1 clinical investigation; (2) remains under
investigation in a clinical trial approved by the Food
and Drug Administration; and (3) is not approved,
licensed, or cleared for commercial distribution under
the Federal Food, Drug, or Cosmetic Act or the Public
Health Service Act.
13 cosponsors (delegations)
 Arizona (3)

Indiana (3)
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Food and Drug Administration
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Introduction of Form FDA 3926
FDA has not taken a position on RtT legislation that is
pending.
FDA has approved more than 99 percent of
compassionate use applications received during the
2010 to 2014 fiscal years.
Critics
 Many terminally ill patients do not know of
available pathway
 Paperwork has historically been unduly
burdensome
 Substantial reduction with Form FDA 3926
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Food and Drug Administration
(cont’d)
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Introduction of Form FDA 3926
 Responsive to concerns from patients and
physicians
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Streamlined process
Designed to greatly simplify and accelerate the
process by which a physician can request that FDA
permit the use of an investigational drug while it’s
still being tested to establish its safety and
effectiveness.
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Replacing form that required 26 types of
information and seven attachments.
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Food and Drug Administration
(cont’d)
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Introduction of Form FDA 3926
 Original form designed for manufacturers seeking
to begin human testing, not for physicians seeking
use by single patients
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Food and Drug Administration
(cont’d)
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What took so long?
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Food and Drug Administration
(cont’d)
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“This draft guidance, when finalized, will represent
the FDA’s current thinking...”
 Timing – competing priorities
FDA use of draft guidance on such an important
topic
Does not apply to other types of expanded access
requests – IND only
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Food and Drug Administration
(cont’d)
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PRA notice published March 2016
 Comments were due April 11, 2016
 Estimates 2,394 total annual responses
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1,153 requests for individual patient access
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1,241 follow up submissions
790 physicians
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Average burden per response: 45 minutes
 Reduction in burden relative to current
process by 15,797 hours
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Outstanding Questions
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Will these state laws withstand legal challenge?
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When will FDA Form 3926 be finalized?
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When FDA finalizes Form 3926, will state legislatures
repeal existing RtT legislation?
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Congressional Outlook
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Questions?
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