1. FDA’s Patient Advocacy Programs: Patient Representative Program & Patient Network Andrea Furia-Helms, MPH James Valentine, MHS Office of Special Health Issues (OSHI)

2. 2 Patient Representative Program Help to capture the unique perspective of patients and family members directly affected by a serious life- threatening disease Serve as SGEs (Special Government Employees) at advisory committee meetings to review products and policies related to serious and life-threatening diseases Work directly with FDA review divisions in clinical trial and post trial review phases Participate in other FDA-related activities where the patient perspective is needed

3. 3 Who are Patient Representatives? Patients Family and/or caregivers Patient/community advocacy groups members

4. 4 Ways Patient Representatives Serve Advisory Committee Meetings Public Meetings, Workshops Informal discussion groups Medical product development meetings –Meetings between FDA and the medical product manufacturers –FDASIA Section 1137

5. 5 FDASIA Section 1137 – Patient Participation in Medical Product Discussions Signed into law on July 9, 2012 by President Obama Full title: The Food & Drug Administration Safety & Innovation Act (FDASIA) Language: ‘‘(a) IN GENERAL.—The Secretary shall develop and implement strategies to solicit the views of patients during the medical product development process and consider the perspectives of patients during regulatory discussions, including by— (1) fostering participation of a patient representative who may serve as a special government employee in appropriate agency meetings with medical product sponsors and investigators; and (2) exploring means to provide for identification of patient representatives who do not have any, or have minimal, financial interests in the medical products industry.

6. 6 Criteria for Becoming a Patient Representative Personal experience with the disease or condition, either as a patient or as a primary caregiver) Able to be objective while representing the concerns of patients with that disease, and willing to communicate their views Knowledgeable about treatment options for the disease and research in that area No obvious financial or ethical conflicts of interest for self or immediate family member

7. 7 Steps in Becoming a Patient Representative 1) Resume –patient/caregiver experience –advocacy experience –ability to represent other patients –knowledge and skills related to disease area –alternate disease experience –work experience –education 2) Phone Interview 3) SGE (Special Government Employee) clearance

8. 8 Training Patient Representatives Individualized FDA 101 training and Advisory Committee orientation by OSHI staff Ongoing training Monthly webinar training Annual workshop Mentoring by senior patient representatives

9. 9 Benefits of Patient Representative Input Involving patients from early stages of medical product development when their input can have significant impact Helping patients develop the knowledge and skills to make meaningful contributions to product development based on their unique experience and perspective Integrating patient input in a meaningful way throughout the medical product development life cycle

10. 10 Patient Representative Contribution to Deliberations Trial Design Entry Criteria Endpoints Drug Toxicity Issues Quality of Life Issues Study Recruitment Informed Consent Expanded Access (a.k.a Compassionate Use) Product Labeling

11. 11 Patient Representative Disease-Specific Contacts Andrea Furia-Helms – all except for cancer and HIV/AIDS 301-796-8455 Deborah Miller – cancer 301-796-8472 Helene Clayton-Jeter – device and radiological issues 301-796-8452 Richard Klein-HIV/AIDS 301-796-8454 OSHI Main Line 301-796-8460

12. FDA Patient Network

13. 13 Background Program Under Development Since Spring 2010 Goals: –Scale up current OSHI educational and advocacy activities –Institute a more proactive approach to patient engagement –Primary FDA patient educational and advocacy resource Audiences: –Patients & Caregivers –Independent Patient Advocates –Patient Advocacy Organizations 13

14. 14 Planned Network Activities Multifaceted Website, including: –Medical Product Development 101 –Library of Written & Multimedia Educational Resources –Updates on FDA actions, upcoming meetings, opportunities to comment (i.e. dockets) –Interactivity (i.e. feedback mechanisms, live chats) Biweekly Email Newsletter Annual Meeting Listening Sessions and Other Briefings 14

15. 15 Inaugural FDA Patient Network Annual Meeting – May 18, 2012 Theme: FDA Working with Patients to Explore Benefit-Risk: Opportunities & Challenges Presentations by FDA experts from CDER, CBER, and CDRH on benefit- risk Patient Perspectives Panel & Audience Discussion About 300 participants (in-person and on webcast) Recording available on FDA website

16. 16 The FDA Patient Network Website will provide information and educational resources on topics including: Ongoing FDA Initiatives How Medical Products Get Approved Clinical Trials Participation Accessing Investigational Products using Expanded Access Off-Label Use of Approved Drugs FDA Advisory Committees Much more!

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19. 19 The FDA Patient Network Website will facilitate engagement between FDA and the patient community: Opportunities to Speak at Upcoming Public Meetings Apply to Become an FDA Patient Representative Join a Live Chat Listen to Recorded Webinars with FDA Experts Comment on FDA Regulations Report Adverse Events on MedWatch Submit a Question to the FAQ Much more!

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21. 21 Timeline to Website Launch Spring 2012 – Complete Website Development Summer 2012 – Award Hosting Contract Early Fall 2012 - Soft Launch of Website 21

22. 22 Want to Stay in the Loop? Subscribe to our Patient Network News bi-weekly email newsletter for updates on: Product Approvals Product Safety Drug Shortages Opportunities for Comment/Guidances Announcements Upcoming Meetings Announcement of Patient Network Annual Meeting Notification of the Website Launch Subscribe at http://bit.ly/PNNews