Kickoff Meeting Precision Medicine Task Force July 17, 2015 Leslie Kelly Hall, Co-Chair Jon White, Co-Chair

Agenda Welcome, Opening Remarks & Workgroup Introduction Overview of the Precision Medicine Initiative Workgroup Charge and Workplan Public Comment Adjourn 1

Precision Medicine Task Force Membership MemberOrganization Co-Chairs Leslie Kelly HallHealthwise Jon WhiteONC / Agency for Healthcare Research and Quality (AHRQ) Members Mary BartonNational Committee for Quality Assurance (NCQA) Lisa GallagherHealthcare Information and Management Systems Society (HIMSS) David McCallie, Jr.Cerner Corporation Andrey OstrovskyCare at Hand Eric RoseIntelligent Medical Objects Andrew WiesenthalDeloitte Consulting, LLP Federal Ex Officio James BreelingVeterans Health Administration (VHA) Josh DennyNational Institutes of Health (NIH) Betsy HumphreysNational Library of Medicine (NLM) Mitra RoccaFood and Drug Administration (FDA) Linda SanchesHHS Office for Civil Rights (OCR) Invited Guests Mina HsiangUnited States Digital Service (USDS) / Office of Management and Budget (OMB) Claudia WilliamsWhite House Office of Science & Technology Policy ONC Staff Maya UppaluruONC – Federal Staff Lead Debbie BucciONC - Technical Advisor 2

The President’s Precision Medicine Initiative Kathy Hudson, DJ Patil, Stephanie Devaney 3

“And that’s why we’re here today. Because something called precision medicine … gives us one of the greatest opportunities for new medical breakthroughs that we have ever seen.” President Barack Obama January 30,

The President’s Precision Medicine Initiative (PMI) Mission Statement To enable a new era of medicine through research, technology, and policies that empower patients, researchers, and providers to work together toward development of individualized treatments. 5

Precision Medicine Concept is not new  Consider prescription eyeglasses, blood transfusions…  Prospects for broader application raised by recent advances in basic research, technology development, genomics, proteomics, metabolomics, EHRs, Big Data, mHealth, etc.  Reinforced by 2011 National Research Council report What is needed now  Development of rigorous research program to provide scientific evidence needed to turn concept into reality  Recruitment of the best and brightest from multiple disciplines to join the team  Development of standards and resources for generating and sharing data  The right policies and technology to make the vision of PMI possible 6

Precision Medicine Initiative: Timing is Everything Precision medicine is an emerging approach for disease prevention and treatment that takes into account people’s variation in genes, environment, and lifestyle. Now is the time: Americans’ Growing Desire to Be Partners in Research Advances in data science and bioinformatics Better technologies for biomedical analysis FDA-cleared technologies for genomics are now available Human-genome sequencing continues to get cheaper and faster Availability of new data – microbiome, diagnostics, and sensor data Availability of Existing Research Cohorts 7

PMI: Components Science Priorities:  Cancer clinical trials – expand on successes in cancer precision medicine  Large Research Cohort – 1 million or more Americans sharing their research & health data to advance precision medicine for all diseases  Standards and resource development Policy Priorities  Streamline regulatory review of genomic technologies  Ensure adequate privacy safeguards are in place Agency$ Million National Institutes of Health Cancer Cohort $200 $70 $130 Food and Drug Administration$10 Office of the National Coordinator for Health Information Technology $5 TOTAL$215 Proposed FY16 Budget 8

PMI: Policy and Privacy Priorities To develop a new research and care model, PMI will:  Engage Stakeholders: White House, HHS, other federal agencies will solicit input from patient groups, bioethicists, technologists, privacy experts, civil liberties advocates, etc.  Modernize Regulations: Food and Drug Administration (FDA) to review regulatory landscape, support changes to advance precision medicine and protect public health  Safeguard Privacy: The White House has developed draft core privacy principles; in addition we are looking at any gaps that need to be addressed to ensure that participants’ data is sufficiently protected. 9

Science Priorities: PMI for Oncology  Goal: Apply tenets of precision medicine to cancer  To reach this goal, PMI-Oncology will enable research to:  Use NCI clinical trials as models –NCI-MATCH: solid tumors, lymphomas (multi-drug, multi-arm) –Lung-MAP: squamous cell lung cancer (multi-drug, multi-arm, randomized)  Identify new cancer subtypes, therapeutic targets  Test combination therapies  Partner with private sector to test precision medicine  Understand and combat drug resistance

Science Priorities: National Research Cohort  ≥1 million U.S. volunteers –Numerous existing cohorts (many funded by NIH) –Outreach to underrepresented groups –New volunteers  Participants will be: –Centrally involved in design, implementation –Able to share genomic data, lifestyle information, biological samples – all linked to their electronic health records –Can choose how, when to participate in research studies  Will forge new model for scientific research that emphasizes: –Engaged participants –Open, responsible data sharing with privacy protections 11

Considerations To-Date: Assembling the Cohort  Ensure cohort is efficient and inclusive  Leverage existing cohorts  Include underserved and underrepresented groups, possibly through FQHCs  Consider including children  Enable military members and veterans to participate  Consent obtained from all as PMI cohort participants 12

Considerations To-Date: Data  Data must be highly secure and broadly accessible –Hybrid data model –Essential core elements –Utilize passive and active data collection  Strong de-identification to increase research use of data 13

Considerations To-Date: Participant Engagement  Maximize transparency about use of participant data  Return information to participants  Provide flexibility on how and when to participate  Open, responsible data sharing with privacy protections 14

Precision Medicine Task Force Charge & Workplan 15

Task Force Charge Identify opportunities for innovative collaboration around pilots and testing of standards that support health IT interoperability for precision medicine Recommend existing standards that are currently ready to support PMI Identify emerging standards and reference implementations that may require further pilot testing in order to support PMI Identify gaps in available data standards related to PMI 16

MeetingsTask  July 17, :30 am - 1:00 pm ET Kick-off Meeting Review charge Identify action steps Wednesday July 29, :30 - 3:00 pm ET Review workgroup comments / testimonial for experts Discuss action steps Wednesday August 5, :00 - 1:30 pm ET Review workgroup comments / testimonial for experts Wednesday August 19, :00 - 1:30 pm ET Develop Preliminary/Draft Task Force Recommendations Monday August 31, :00 - 1:30 pm ET Finalize Task Force Recommendations Prep for Sept. HITSC presentation. Thursday September 10, :30 - 3:00 pm ET Present Final recommendations to HITSC Precision Medicine Task Force Meeting Dates/Workplan 17