1. Patient Engagement throughout the Biopharmaceutical Lifecycle: Tips for Effective Patient Advocate/Industry Collaboration to Improve Patient Access and Outcomes Lisa Egbuonu-Davis, MD, MPH, MBA LED Enterprise, LLC September 15, 2014

2. Key Points Patients and patient advocacy organizations have a critical role to play in driving science and systems – research and policy agendas to support sustainable product innovation and health care delivery systems in order to ensure access and outcomes Level of patient participation has been increasing: –Consultation –Involvement –Partnership/Shared Leadership (ref: Carman KL. Health Affairs) Focus of this presentation will emphasize industry/patient partnerships with an emphasis on sustainable development and access to imedical product innovation 2

3. Key Principles for Good Collaboration Understand each partner’s objectives Focus on shared goals Respect each other’s strengths Understand constraints (regulatory, stakeholders) Take time to develop relationships (work at correct points of contact) Start with smaller projects and build 3

4. Biopharma Industry Needs from Patient Advocates Trusted communication source with: –Members –Media Patient perspective on research priorities and therapeutic approaches Relationships with key opinion leaders Clinical trial participation – may want input on design 4

5. Biopharma Industry Needs from Patient Advocates Policy Influence –Screening and treatment guidelines –Health care advisory panels –Regulatory Approval, Risk/Benefit –Coverage and Reimbursement BUT- NEEDS AND ACTIONS VARY WIDELY AMONG COMPANIES, AS DOES ORGANIZATIOAL STRUCTURE, KEY CONTACTS 5

6. Patient Advocates Needs from Industry Information/Education on Disease Treatment Options/Research Fundraising for Research Support for Advocacy and Awareness Patient Support for Medical Care, Social Support, Funding CT access and support/ input on design Board and Committee Members VARIES WITH ORGANIZATION AND MISSION 6

7. Patient Engagement for Value and Access (Faster Cures:Value and Coverage) Understand regulator and payer value definitions and evidence required Collaborate with industry and payers early in process and continue throughout Precompetitive consortia and collaborations for data platforms and standards Share data platforms for interoperability Support open consent platforms for patient data 7

8. Patient Engagement for Value and Access (Faster Cures:Value and Coverage) Collect patient relevant outcomes and develop valid measures for payers/regulators/guidelines Collect universal symptom and quality of life data Patient registries, on line communities for measuring quality of life and patient preferences Understand risk/benefit in target disease 8

9. Patient Engagement for Value and Access (Faster Cures:Value and Coverage) Understand what treatments work for whom, when and at what cost- including CER Advocate for standardized care guidelines to equalize access to best practice care Educate members about quality and financial realities of innovators and payers for open communication Support transparency re health care cost and quality per physician, setting, service Expand access to effective treatments 9

10. Patient Engagement Throughout the Lifecycle All phases –Patient and public awareness –Advocacy – health care system and financing –Knowledge exchange and peer support –Registries –Clinical practice guidelines –Regulatory and payer awareness 10

11. JDRF- Patient Advocacy for Artificial Pancreas a Comprehensive Lifecycle Approach Basic and clinical research Regulatory pathway creation Extensive reimbursement/insurance coverage advocacy Evidence generation for potential cost- saving 11

12. Patient Engagement Throughout the Lifecycle Discovery through Early Development (proof of concept) –Research- Drug Discovery –Research- Priority Setting –Bio-repositories –Disease specific outcomes measures (PROs) –Clinical trial enrollment/design –Early engagement with regulators and payers 12

13. Patient Advocate/Industry Collaboration- Discovery through Early Development JDRF – Pfizer Centers for Therapeutic Innovation Collaboration -Translational Research -Partnership on Research Design 13

14. Patient Engagement Throughout the Lifecycle Full Development –Research- Priority Setting –Bio-repositories –Disease specific outcomes measures (PROs) –Clinical trial enrollment/design –Regulatory and Reimbursement Processes 14

15. Patient Advocacy/Industry Collaboration – Full Development to Approval Cystic Fibrosis Foundation and Vertex Collaboration MMRF- Multiple Myeloma Research Consortium 15

16. Patient Engagement Throughout the Lifecycle Marketed Product/Routine Clinical Use –Real World Studies –Managed Access –Risk/Benefit –Patient Reported Outcomes –Continued Value Demonstration 16

17. Patient Advocate/Industry Collaboration – Marketed Product/Routine Use MMRF- CoMMPass- Onyx Patients Like Me – UCB Medicare Part D – Protected Therapeutic Classes – MHA/NAMI/Industry/Providers 17

18. Patient Advocate/Industry Collaboration - Challenges Differences in priorities/emphasis Unclear organizational structures, roles, points of contact Limited resources: staff, time, funding Concerns re conflicts of interest Concerns re trust/transparency 18

19. Patient Advocate/Industry Collaboration – Suggested Approaches and Resources Clarify your objectives Look for companies with good overlap, disease areas, stated interests in research/patient support/policy Look for company functions directly related to common interest – research, medical affairs, policy, market access 19

20. Patient Advocate/Industry Collaboration – Suggested Approaches and Resources Research and policy meetings –Leverage links with key researchers to access industry Leverage consortia for voice, impact, access Resources for advocacy organizations –Faster Cures tools papers on Value and Access and toolkits –MMRF consultative advice to patient advocacy organizations –Some consultants focus on advocates 20

21. Questions 21