Using patient-generated information to drive research and improve care. To drive research and improve care, we NEED patient-generated information

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1 Using patient-generated information to drive research and improve care To drive research and improve care, we NEED patient-generated information

2 Transformation is happening NETWORKED WORLD PERSONALIZED MEDICINE ADAPTIVE CLINICAL TRIALS ROBUST SOCIAL NETWORKS EMPOWERED PATIENT AFFORDABLE CARE ACT SCIENTIFIC ADVANCES HEALTH IT

3 Finding Big Ideas in Small Spaces

4 TRAIN groups business models catalyzing progress; draw attention, recognition patient groups with an entrepreneurial bent have become the drug industry s new power brokers. - Forbes 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.

5 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

6 Patient Foundations are TRUSTED by 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 Bridge disciplines, institutions & ideas Trusted by and have access to patients Form a unified advocacy front on key public policy issues affecting the pace of research.

7 The Kalydeco Story Developed by Vertex Pharmaceuticals with a $75 million investment from the Cystic Fibrosis Foundation First medicine to directly affect the genetic defect that causes the disease The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development. - FDA Commissioner Margaret Hamburg

8 Cystic Fibrosis Foundation ASSETS INCLUDE: 40-year-old patient registry A nationwide network of nearly 80 CF clinical research centers of excellence that specialize in conducting trials to evaluate the safety/efficacy of new CF therapies. Actively recruiting patients into trials on behalf of their industry partners Actively supports more than 30 new treatments in development 4 FDA approved Partners include Vertex, Copernicus, Lantbio, Pfizer, Gilead, Alnara

9 T1D Exchange The T1D Exchange model was conceptualized as a solution to mitigate barriers and inefficiencies for clinical and translational research. Products & Services include: MyGlu & Exploration Portal o Patient engagement platform o Access to T1DX data Specialty services o Patient recruitment & study/trial support o Custom study modules Dynamic biorepository o Living biobank strategy o Ability to collect targeted samples

10 Alpha-1 Foundation Invested almost $47 million to support Alpha-1 Antitrypsin Deficiency research and programs in 94 institutions globally. Created an Alpha 1 registry and a DNA and tissue bank. Provides care management services for payers which provides revenue to support its research operation. Invested $250,000 in creating guidelines for providers, apps to facilitate their adoption, and provides Continuing Medical Education via Medscape.

11 Michael J. Fox Foundation At this year s Partnering for Cures, MJFF presented a challenge it sponsored to create opportunities for patients to contribute data about the real experience of Parkinson's disease (PD) via their mobile phones, which are some of the most pervasive forms of monitoring devices. Over 16 weeks, MIT researchers collected information through mobile phones from 9 PD patients and 7 healthy controls. Through MJFF's PD Data Challenge, researchers sought to develop the best way to benefit patients and clinicians through analysis of these objective, passively collected data. The winning entry was chosen following more than 630 downloads of the dataset from teams in 21 countries. LIONsolver provided proof of concept for a 'machine learning approach' that could unveil clues to PD onset and progression embedded in data collected on smartphones.

12 Patients Like Me An online platform where more than 200,000 patients across hundreds of diseases track and share their experiences and connect with others. Brings a level of research rigor to the real-world observations of the patients on the platform. This year PLM received a $1.9 million grant from the Robert Wood Johnson Foundation to create an Open Research Exchange online platform for validating patient-reported outcomes measures.

13 Multiple Myeloma Research Foundation Launched MMRF CoMMpass Study CoMMpass = Relating Clinical Outcomes in MM to Personal Assessment of Genetic Profile 10 year study w/1,000 patients Molecular analysis of every patient-sequential tissue sampling to identify how molecular profile may affect clinical progression Better treatment targeted to each patient s biological makeup

14 Honest Brokers for Cures In-house scientific expertise Smart business strategies Innovative financing models Leadership & accountability

15 Turn to patients as partners, true partners Patients/patient groups have the data. But data are worthless unless we ask the right questions. What data are needed to build evidence necessary to assure access? A recent PwC report noted that in its survey of insurers, the largest proportion of respondents rated employers and patient of disease advocacy groups as not influential as sources of evidence.

16 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

17 Inventory of Medical Research Foundation s Partnership Practices

18 The 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.

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