Kickoff Meeting Precision Medicine Task Force July 17, 2017 Leslie Kelly Hall, Co-Chair Jon White, Co-Chair.

Kickoff Meeting

Precision Medicine Task Force

July 17, 2017Leslie Kelly Hall, Co-ChairJon White, Co-Chair

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Agenda

• Welcome, Opening Remarks & Workgroup Introduction

• Overview of the Precision Medicine Initiative

• Workgroup Charge and Workplan• Public Comment• Adjourn

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Precision Medicine Task Force Membership

Member OrganizationCo-ChairsLeslie Kelly Hall HealthwiseJon White ONC / Agency for Healthcare Research and Quality (AHRQ)MembersMary Barton National Committee for Quality Assurance (NCQA)Lisa Gallagher Healthcare Information and Management Systems Society (HIMSS)David McCallie, Jr. Cerner CorporationAndrey Ostrovsky Care at HandEric Rose Intelligent Medical ObjectsAndrew Wiesenthal Deloitte Consulting, LLPFederal Ex OfficioJames Breeling Veterans Health Administration (VHA)Josh Denny National Institutes of Health (NIH)Betsy Humphreys National Library of Medicine (NLM)Mitra Rocca Food and Drug Administration (FDA)Linda Sanches HHS Office for Civil Rights (OCR)Invited GuestsMina Hsiang United States Digital Service (USDS) / Office of Management and Budget (OMB)Claudia Williams White House Office of Science & Technology Policy ONC StaffMaya Uppaluru ONC – Federal Staff LeadDebbie Bucci ONC - Technical Advisor

The President’s Precision Medicine Initiative

Kathy Hudson, DJ Patil, Stephanie Devaney

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“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 ObamaJanuary 30, 2015

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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.

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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

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Precision Medicine Initiative:Timing is EverythingPrecision 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 Cohorts8

PMI: ComponentsScience 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

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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.

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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 protections12

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

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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

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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

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Data/Tech Track of PMI: Focus Areas

1. Enable patients to access their electronic health data easily and contribute it for research.

• Work with patient-data advocacy groups to align goals, understand policy issues, and provide recommendations to ONC and CMS to support PMI.

• Work with healthcare providers and vendors to resolve policy, technology, and workflow challenges.

• Host workshops with technologists, research scientists, and patient groups to identify what data is required and policies.

• Support NIH’s scientific FACA process with technical and policy guidance.

• Identify key drivers to support an ecosystem of applications create demand for data and innovation for patients.

2. Accelerate the widespread use of APIs to allow for seamless exchange of EHR data to support PMI research.

• Identify the potential range of technical options for APIs and supporting orgs.

• Secure commitments from top research organizations, healthcare organizations, and EHR vendors to decrease time to deployment of prototypes.

• Advise ONC and CMS on their healthcare interoperability roadmap and provide technical input on places where acceleration can be provided.

• Provide guidance to agencies piloting APIs and security/trust standards for easier access to health data, such as CMS, VA, DOD.

•Enforce data access rights and data misuse penalties, to ensure data flows where and how it should.

1. Enable 2. Accelerate 3. Execute3. Execute the best approaches for data management, storage, security, privacy, and usage for PMI.

• Provide advice to VA and DOD on their genomic programs (MVP & Army Stars) to allow their programs to be the seed of PMI.

• Convene experts in the security space to develop guidelines for security and privacy.

• Provide technical advice for DOD & VA health care interoperability with USDS and DOD’s procurement of a new EHR solution to enable PMI goals

Data and Technology Mission: To enable the 21st century research platform that responsibly empowers participants, researchers, and providers with the highest quality of data to power the next generation of precision medicine and health care innovations.

Hypotheses - Which curve are we on?Which curve should we be on?

Time

Revolutionary

Sop

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driv

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Poor

2010 2020

Meaningful Use 2

Meaningful Use 1

Meaningful Use 3

Minimum required for PMI

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Precision Medicine Task Force Charge & Workplan

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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

Meetings Task

July 17, 2015 11:30 am - 1:00 pm ETKick-off Meeting

• Review charge• Identify action steps

Wednesday July 29, 2015 1:30 - 3:00 pm ET

• Review workgroup comments / testimonial for experts• Discuss action steps

Wednesday August 5, 2015 12:00 - 1:30 pm ET

• Review workgroup comments / testimonial for experts

Wednesday August 19, 2015 12:00 - 1:30 pm ET

• Develop Preliminary/Draft Task Force Recommendations

Monday August 31, 2015 12:00 - 1:30 pm ET

• Finalize Task Force Recommendations• Prep for Sept. HITSC presentation.

Thursday September 10, 2015 1:30 - 3:00 pm ET

• Present Final recommendations to HITSC

Precision Medicine Task ForceMeeting Dates/Workplan

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