The Evolving Role of Patients in the R&D Ecosystem

1. The Evolving Role of Patients in the R&D Ecosystem February 27, 2014 Margaret Anderson Executive Director, FasterCures

2. About FasterCures We are an action tank that works to improve and speed up the time it takes to get important new medicines from discovery to patients. Our name. Our mission.

3. Bringing a new discovery from lab to market is a long, expensive and risky road

4. What is venture philanthropy?

5. Find Big Ideas in Small Spaces

6. Loose affinity network of 56 nonprofit disease research foundations • Created to tackle the challenges that cut across diseases through innovative partnerships • Connected through TRAIN Central Station, an open-source web platform: www. fastercures.org/train

7. TRAIN Inventory • Thought leaders – including those in the venture capital community and media – have turned to this inventory as a resource to provide them with an in-depth understanding of current trends and practices in the venture philanthropy space.

8. TRAIN groups’ business models catalyzing progress; draw attention, recognition …Frustrated with the sluggishness, or nonexistence, of medical research…a small band of wealthy parents whose children have serious illnesses are spending millions of dollars to fund drug development...The principles they apply in their jobs -- managing complicated tasks, making investments and expecting positive results -- translate to their new endeavors. 09/07/11 “…patient groups with an entrepreneurial bent have become the drug industry’snew power brokers.” - Forbes

9. “At a time when so many companies are starving for cash to advance their best drug R&D ideas, many nonprofit foundations know they can make an impact by cleverly applying their own blend of money, networks of patients, and sincere advocacy.” When Nonprofits Drive For-Profits

10. Increasingly powerful force in early-stage research “They required us to lay out the vision for the pre-clinical and early clinical development… Most importantly, they were able to view the program with a critical eye and provide meaningful feedback.” Although the funding models may differ from organization to organization, virtually everyone agrees that the ties between biotech industry and nonprofit patient-centered organizations will continue to strengthen.

11. WSJ 9-chapter multimedia special captures the spirit of patient-driven R&D

12. Venture Philanthropy in Medical Research • Fund novel, high-risk research • Have strong scientific expertise • Bridge disciplines, institutions, & ideas • Trusted by and have access to patients

13. Patient Foundations are TRUSTEDby and have ACCESS to patients • Fund novel, high-risk research • Strong scientific expertise • Strategic players in R&D in their disease areas • Creating assets and capabilities that can be used to develop evidence for regulatory approval and to demonstrate value to payers. • clinical care and research networks • patient registries and tissue repositories • social networks • in some cases, pharmacy and care management services • Form a unified advocacy front on key public policy issues affecting the pace of research. • Bridge discipline, institutions & ideas • Trusted by and have access to patients

14. Foundations as “de-riskers” • Developing pre-clinical tools • Targeting research to support translation • Funding mechanisms that bring in industry • Managing academic science • Access to patient community • New indications for existing drugs • High-throughput screening of promising compounds • Access to scientific expertise • Advocating with FDA • Early-stage investors and/or “talent scouts”

15. “Not your grandmother’s charity” • Make both investigator-initiated and directed grants • Manage grants more actively to maximize efficiency and chances of success • Have internal scientific and/or industry expertise • Provide access to patient resources via registries, tissue banks, clinical care/research networks • Mine academic grant portfolios for promising discoveries ready for translation • Provide resources and relationships to aid in translation to clinical research and hand-off to industry • Consistency and focus to push research projects along

16. Venture philanthropy impact • Funded $400 million in research since 2000, 90% for translational research • Funded testing of over 100 therapeutic targets for Parkinson’s Disease • Creating and sharing research tools • Leading the development of a PD biomarker • Partnership with Elan Corp. gave them first look at promising projects • Approval of 6 new multiple myeloma drugs • World’s first and largest myeloma tissue bank • Full sequencing of the myeloma genome • Opened 45 clinical trials, accelerating 6 treatments to Phase III • Personalized medicine initiative includes landmark trial that will closely monitor 1,000 patients over 5 years- • >$62M to 415 drug research programs in academia and biotechnology in 18 countries • >$8.5M to 40 companies with an average investment of $250K, including 4 successful start-ups through tech transfer • Funded programs attracted hundreds of millions of dollars in follow-on funding from government grants to IPOs • Funded more than $1 billion in research since 2005 • Actively supports more than 22 new treatments in development – 10 FDA-approved • Partners include Vertex, Pfizer, PTC Therapeutics, Genzyme • 40-year-old patient registry • 74 accredited care centers conduct trials

17. Honest Brokers for Cures • In-house scientific expertise • Smart business strategies • Innovative financing models • Leadership & accountability …Frustrated with the sluggishness, or nonexistence, of medical research…a small band of wealthy parents whose children have serious illnesses are spending millions of dollars to fund drug development...The principles they apply in their jobs -- managing complicated tasks, making investments and expecting positive results -- translate to their new endeavors. 09/07/11

18. Patient Advocacy’s Impact

19. At the corner of…

21. Impatient Patients:POWERFUL PARTNERS

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