2. Office of the Vice President & General Counsel 300 North Ingalls Building, Suite 3B04 (734) 764-2178 Right to Try: Evolution of State and National Policy 2016 Michigan Research Ethics Conference Tiana L. Korley, MHA, JD April 29, 2016

3. 3 Office of the Vice President & General Counsel  General Framework  Consideration of State Laws  Overall landscape  Michigan  California  Stakeholder interest  Consideration of Federal Law  Where do we go from here?  FDA actions  Congressional outlook Agenda

4. 4 Office of the Vice President & General Counsel  Patient is terminally ill  Physician recommends use of a treatment  Patient provides informed consent  Treatment has completed phase 1 testing  Liability protections  Manufacturer  Physician  No requirement for insurers to provide coverage General Framework

5. 5 Office of the Vice President & General Counsel  Supremacy Clause  Introducing drugs into interstate commerce without safe/effectiveness [Section 505 of FD&C Act]  No challenge – yet  Tort liability – preempted? Legal Framework

6. 6 Office of the Vice President & General Counsel  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  Impact on protections for vulnerable population  Institutional Review Board review/approval Ethical Framework

7. 7 Office of the Vice President & General Counsel  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 Overall Landscape

8. 8 Office of the Vice President & General Counsel  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.” Michigan

9. 9 Office of the Vice President & General Counsel  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.” California

10. 10 Office of the Vice President & General Counsel  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. Stakeholder Interest PhRMA

11. 11 Office of the Vice President & General Counsel  H.R. 3012  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 Federal Law

12. 12 Office of the Vice President & General Counsel  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) Federal Law (cont’d)

13. 13 Office of the Vice President & General Counsel  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 Food and Drug Administration

14. 14 Office of the Vice President & General Counsel  Introduction of Form FDA 3926  Responsive to concerns from patients and physicians  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.  Replacing form that required 26 types of information and seven attachments. Food and Drug Administration (cont’d)

15. 15 Office of the Vice President & General Counsel  Introduction of Form FDA 3926  Original form designed for manufacturers seeking to begin human testing, not for physicians seeking use by single patients Food and Drug Administration (cont’d)

16. 16 Office of the Vice President & General Counsel  What took so long? Food and Drug Administration (cont’d)

17. 17 Office of the Vice President & General Counsel  “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 Food and Drug Administration (cont’d)

18. 18 Office of the Vice President & General Counsel  PRA notice published March 2016  Comments were due April 11, 2016  Estimates 2,394 total annual responses  1,153 requests for individual patient access  1,241 follow up submissions  790 physicians  Average burden per response: 45 minutes  Reduction in burden relative to current process by 15,797 hours Food and Drug Administration (cont’d)

19. 19 Office of the Vice President & General Counsel  Will these state laws withstand legal challenge?  When will FDA Form 3926 be finalized?  When FDA finalizes Form 3926, will state legislatures repeal existing RtT legislation? Outstanding Questions

20. 20 Office of the Vice President & General Counsel Congressional Outlook

21. 21 Office of the Vice President & General Counsel Questions?