Christine Mitchell, "Expanded Access and Right to Try Laws"
1 like1,497 views
The document discusses the complexities and ethical concerns surrounding expanded access and 'right to try' laws for investigational medical products not yet approved by the FDA. It highlights the case of Josh Hardy, which triggered significant debate about compassionate use and patient access to experimental treatments. The text concludes with calls for refining the system to better balance patient needs with clinical trial integrity while addressing legal and ethical challenges.
![17
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”
17](https://image.slidesharecdn.com/mitchellcompassionateusepreview2016-160129215447/85/Christine-Mitchell-Expanded-Access-and-Right-to-Try-Laws-17-320.jpg)
Christine Mitchell, "Expanded Access and Right to Try Laws"
- 1. 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
- 2. 2 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
- 3. 3 Paradigm Case: Josh Hardy 3
- 4. 4 Josh developed an adenovirus infection when he was recovering from a BMT for myelodysplastic disorder. 4
- 5. 5 His physicians asked Chimerix for compassionate access to brincidofovir, a drug in phase 3 trails 5
- 6. 6 NO 6 …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….
- 7. 7 Going viral 7 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.
- 8. 8 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. 8
- 9. 9 9
- 10. 10 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
- 11. 11 #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 11
- 12. 12 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 12
- 13. 13 Two responses in 2015 Compassionate Use Advisory Council “Right to try” laws
- 14. 14 “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 14
- 15. 15 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 15
- 16. 16 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 16
- 17. 17 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” 17
- 18. 18 Meanwhile… 18
- 19. 19 FDA 19 • 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)
- 20. 20 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 20
- 21. 21 Compassionate Use Advisory Committee 21 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
- 22. 22 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 22
- 23. 23 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 23
- 24. 24 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 24
- 26. 26 26