4th Annual Health Law Year in P/Review

Expanded Access and Right to Try Laws

PETRIE FLOM CENTER FOR HEALTH LAW POLICY,

BIOTECHNOLOGY AND BIOETHICS AT

HARVARD LAW SCHOOL

29 JANUARY 2016

Christine Mitchell, RN, MS, MTS

Executive Director

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Compassionate Use: use outside of a clinical trial of an

investigational medical product not yet approved by FDA

Expanded Access: typically refers to intermediate or large

size CU programs conducted by a sponsor prior to approval

of a drug

Pre-Approval Access: obtaining a medical drug, device or

biologic outside of a clinical research trial, prior to FDA

approval

Right to try: state laws that allow terminally ill patients to

request experimental drugs that have not been approved by

the FDA

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Paradigm Case: Josh Hardy

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Josh developed an adenovirus infection when he was recovering from a BMT for myelodysplastic disorder.

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His physicians asked Chimerix for compassionate access to brincidofovir, a drug in phase 3 trails

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NO

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…we made the difficult

decision two years ago to cease

our Compassionate Use

program and focus on earning

FDA approval. This is the only

path to making brincidofovir

widely available to those who

need it in the fastest manner

allowed….

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Going viral

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Our doctor told us…and he knows it will work. However the drug company has refused …basically they are not going to save a child’s life for the money.

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My goal was to get Josh Hardy the drug. If in fact the boy, Josh, died, I’d have done everything in my power to destroy the company and Mr. Moch.

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Number of pre-approval access campaigns on change.org

Source: change.org

2009

3

2010

4

2011

9

2012

12

2013

21

Average number of signatures per campaign

0

500

15k

105k

270k

395k

2014

2013

2012

2011

2010

2009

Slide from Joanne Waldstreicher CMO J&J

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#Crowdsourcing medical decisions: Ethicists worry Josh

Hardy case may set bad precedent After a 7-year-old boy

receives an experimental treatment, ethicists wonder:

What about the rest? washingtonpost.com

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Josh Hardy epilogue…

Chimerix worked with FDA to create open-label

study and enrolled Josh as the only patient

Ken Moch was replaced as CEO of Chimerix

Josh improved and was discharged home

And ethicists and lawyers have been discussing this

case and cases like it since 2014

In 2015 several steps were taken to improve

change the process for pre-approval access to

experimental drugs and devices

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Two responses in 2015

Compassionate Use

Advisory Council

“Right to try” laws

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“Right to Try” laws

• First passed in Colorado

• Currently 24 states

• Leading Proponent—

Goldwater Institute

• Do not compel companies to

grant access

• As of today, no patient has

received compassionate

access via right to try laws

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Requirements of RTT

Although there is variability among the state RTT

laws, all permit patients to

obtain investigational drugs that have completed

phase 1 safety testing

when there is a physician willing to petition for and

oversee compassionate use, and

a company willing to supply the drug (or device)

State licensing boards are prohibited from taking

disciplinary action against physicians prescribing

investigational drugs

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Ethical Problems with RTT

• Inequitable access: dx terminal illness

• Potential exploitation: lack of oversight

• Promote therapeutic misconception

• False hope: Implies an entitlement but creates no duty

to provide

• Hollow promise: Really, just “a right to beg”

• Potentially harmful: May forfeit right to hospice, home

health care, and insurance

• Favors individual over common good

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Legal Problems with RTT

• No constitutional right of access [US v Rutherford 1979; Abigail Alliance v vonEschenbach 2008]

• State laws cannot prevent the Drug Enforcement

Administration from rescinding registration of

physicians who prescribe experimental drugs

independent of the FDA

• Under the Supreme Court’s pre-emption doctrine,

state laws that conflict with federal law are without

effect

• RTT “unlikely to withstand constitutionality

challenge”

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Meanwhile…

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FDA

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• For seriously or terminally ill patients with no alternative

treatments

• Requires evidence for believing the drug is safe and

sufficiently promising for patients to try it

• Requires IRB approval of expanded access

• Only permitted if no threat to clinical trial enrollment

• FDA has an access system to

apply for CU

• Feb 2015: announced

streamlined system (45 minutes)

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Translational bioethics

• Can we create a fair process for evaluating

CU petitions

• That balances the need of individual dying

patients with the needs of future patients

• Without undermining scientific clinical

research

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Compassionate Use Advisory Committee

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NYU Working Group

To develop fair process for

evaluating requests for CU

Art Caplan, Chair; Alison

Bateman-House, Deputy Chair

Pilot: Darzalex (daratumumab) for

refractory multiple myeloma

Requests come first to Janssen

Then CompAC

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Conversation with Art Caplan

• Steep learning curve

• Fairness most important

• Scarcity of supply is real

• Rationing is real

• Provide evaluation and recommendation to J&J

• High level of interest

• JAMA publication in a few weeks

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In 2016+

We need to work on refining a

fairer,

faster,

safer system

for pre-approval use of experimental medical

products

that doesn’t undermine the clinical trial process

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Selected References

Bateman-House et al. Right to try laws: hope, hype and unintended

consequences Annals of Internal Medicine 2015 http://annals.org/article.aspx?articleid=2443961

Darrow et al. Practical, legal and ethical issues in expanded access to

investigational drugs NEJM 2015 http://www.nejm.org/doi/full/10.1056/NEJMhle1409465

Dresser. Right to try laws: the gap between experts and advocates

Hastings Center Report 2015 http://www.thehastingscenter.org/Publications/HCR/Detail.aspx?id=7406

US Food and Drug Administration. Expanded Access (Compassionate

Use) http://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm

http://blogs.fda.gov/fdavoice/index.php/2015/02/a-big-step-to-help-the-patients-most-in-need

NYU Langone Medical Center Working Group on Compassionate Use and

Pre-Approval Access http://www.med.nyu.edu/pophealth/divisions/medical-ethics/compassionate-use

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