1. How Patient Collaborations Can Drive Innovation 02 Mar 13 Bray Patrick-Lake, BS, MFS Director of Stakeholder Engagement Clinical Trials Transformation Initiative American Society for Experimental NeuroTherapeutics | 15 th Annual Meeting

2. Disclosure Duke University PFO Research Foundation Alliance for Headache Disorders Advocacy American Society for Experimental NeuroTherapeutics | 15th Annual Meeting Employee President and CEO Board member

3. Background Patients have long been frustrated by the lengthy times and high costs of developing and commercializing new therapies with low yields IOM recently identified better cooperation and communication among stakeholders as a priority to transforming the clinical research enterprise New models of patient collaboration are evolving to drive innovation and transformation American Society for Experimental NeuroTherapeutics | 15th Annual Meeting

4. Traditional Patient Collaborations Disease-specific advocacy and education-based patient organizations Perform fundraising for research Form partnerships with investigators or create centers of excellence for research and treatment Become involved with industry to promote clinical trials and investigate opportunities for development of new therapies American Society for Experimental NeuroTherapeutics | 15th Annual Meeting

5. Evolution of Patient Collaborations Driving Innovation Powered by internet, social media and mobile communications Patients are getting connected and organizing globally Impatience, limited financial resources and poor return on investment have fueled cross-sector relationships and out of the box thinking American Society for Experimental NeuroTherapeutics | 15th Annual Meeting

6. CTTI is a private public partnership co-founded by FDA & Duke University in 2007 Organization: 60-member diverse stakeholder collaboration of academia, industry, government, patients, CROs and IRBs Mission: To identify and promote practices that will increase the quality and efficiency of clinical trials Patient Leadership Council: 16-member council of patient thought leaders forming a collective voice around clinical trial issues to drive systemic improvement and transformation in the clinical trial enterprise www.ctti-clinicaltrials.org American Society for Experimental NeuroTherapeutics | 15th Annual Meeting Model: Systemic Improvement and Transformation

7. Patients Accessing Assets from External Sources: Crowdsourcing Prize4Life (www.prize4life.org) is a PAO whose mission is to accelerate the discovery of treatments and a cure for ALS by using powerful incentives to attract new people and drive innovation P4L awarded monetary prizes to encourage promising biomarker concepts and predict progression Awarded treatment prizes to fill the drug development pipeline with promising therapeutics by encouraging researchers to extend the lives of ALS mouse models by 25%. American Society for Experimental NeuroTherapeutics | 15th Annual Meeting

8. Model: PAO’s forming Partnerships to Innovate Prize4Life partnered with the Northeast ALS Consortium (NEALS) and the ALS Therapy Alliance (ATA) to develop the Pooled Resource Open-Access ALS Clinical Trials (PRO- ACT) database largest database of clinical data from ALS patients ever created P4L Partnered with InnoCentive for crowdsourcing, open innovation and prize competition www.InnoCentive.com Working groups and multi-sector collaborations on drug development, e.g. FasterCures and Health Research Alliance American Society for Experimental NeuroTherapeutics | 15th Annual Meeting

9. Creating Research Tools for the Public Domain Michael J. Fox Foundation for Parkinson’s Disease (www.michaeljfox.org) Clinical Trials Recruitment Best Practices Manual To provide trial investigators with a comprehensive look at lessons learned and best practices that have resulted in successful recruitment for PD trials across many sites Creating tools for bench scientists and putting them in the public domain Animal and cellular models, proteins, genetic resources, high-quality antibodies against PD proteins American Society for Experimental NeuroTherapeutics | 15th Annual Meeting

10. Organizing Patients Online PatientsLikeMe (www.patientslikeme.com) Creation of longitudinal registries for common and rare diseases Data-sharing platform harvesting patient-reported information from online patient communities PLM offers its services to the R&D organizations of their pharma and device clients Providing specialized information that might inform a target product profile or give insight into the real-world use of their candidate drug/device Partnerships with Merck (psoriasis) and US Dept of Veteran’s Affairs’ Epilepsy Centers of Excellence and UCB SA American Society for Experimental NeuroTherapeutics | 15th Annual Meeting

11. Patient-Funded Drug Development Companies DART Therapeutics, LLC (www.dartrx.com)www.dartrx.com Innovative, new-model biotechnology firm focused on developing therapies for Duchenne Muscular Dystrophy (DMD) Co-founded in 2010 by two patient foundations in DMD, Charley's Fund and the Nash Avery Foundation New model for research and drug development centered on the needs of families affected by DMD and potentially transportable to other pediatric rare diseases Accomplishments in first two years: Acquired a promising clinical stage drug candidate and spun out a special-purpose entity focused on its development. Conducted research on a biomarker as potential new tool to track muscle health that could enable lower costs and faster drug development timeframes Structured partnerships between patient foundations and investor capital to expedite promising programs in DMD

12. Paving the Legislative and Regulatory Pathways for New Therapies Friends of Cancer Research (www.focr.org) Cancer research think tank and advocacy organization Develops partnerships and advocates for policies that will get treatments and therapies to patients in the safest and quickest way possible Patients work with federal health agencies, congressional leadership, academic research centers and private sector industry In follow-up to the 2011 FOCR event, The Advancing Breakthrough Therapies for Patients Act was introduced and included as a component of the 2012 re-authorization of the Prescription Drug User Fee Act (FDASIA) to expedite development of new, potential “breakthrough” therapies. American Society for Experimental NeuroTherapeutics | 15th Annual Meeting

13. Conclusions Patients can drive innovation when viewed as partners in rather than subjects of research A common theme of patient-driven stakeholder collaboration has emerged in innovation embracing clinical trials networks, open access and information sharing models, and legislative overhaul New and promising models of patient collaborations should be considered in order to drive innovation such as crowdsourcing, online organizing, and patient-funded drug development companies