FDA News: precisionFDA, Patient Voice, Drug Safety Priorities, Priority Review Vouchers, Open Positions-Consumer & Patient Representatives – Drug and Device Digest

FDA BRIEF: Week of October 3, 2016


precisionFDA’s Next Challenge? Conduct an App-a-Thon!


Zevana Tezak

Zivana Tezak, Ph.D.,  Associate Director, Science and Technology, CDRH

Elaine Johanson

Elaine Johanson,  precisionFDA Project Manager, Deputy Director Office of Health informatics


  • Online research portal
  • FDA scientists + Silicon Valley minds
  • Part of President Obama’s Precision Medicine Initiative (PMI)


  • Advance the use of next generation sequencing (NGS)
  • Map entire human genome; data, analytics, and sequencing tools
  • Open source cloud-based space for data, ideas, and methodologies sharing
  • > 1,600 participants


 “App-a-Thon”: Closes Oct 28, 2016

  • Add NGS software apps for simulations, benchmarking, data integration, mapping portions of the genome, or identifying genetic variants
  • Add to the precisionFDA app library
  • FDA provides framework and materials, storage, and compute capacity
  • Results to be highlighted by FDA Commissioner at World Precision Medicine Congress on Nov. 14, 2016 in Washington D.C

App-a-Thon in a Box toolkit.


Our 20th Patient-Focused Drug Development meeting: Enhancing the patient’s voice in FDA’s approach to drug review and development


Theresa Mullin

Theresa M. Mullin, Ph.D., Director, Office of Strategic Programs, CDER

Patient Focused Drug Development (PFDD)  program launched as part of PDUFA V

  • PFDD meetings helps FDA strengthen  understanding of targeted disease areas and hear directly from patients, families, and caregivers about symptoms that matter most to them
  • 20th PFDD public meeting  with organ transplant patients occurred Sept 27th
  • All meetings chronicled in Voice of the Patient reports
  • Plan to hold 4 more PFDD meetings in 2017

Externally-led Patient-Focused Drug Development Meetings

  • To expand PFDD benefits, FDA welcomes patient-focused meetings organized by the patient groups themselves
  • Interested patient groups can submit a letter of intent

Externally-led Patient-Focused Drug Development Meetings


2016-2017 CDER Drug Safety Priorities: Initiatives and Innovation


CDER’s programs to ensure safety of drug products and evolving drug safety enterprise

Miletones and broad picture of FDA’s multidisciplinary collaborations and partnership

Drug Safety Oversight Across the Product Lifecycle

Advancing Drug Safety Science

  • JumpStart – Enhancing Pre-Market Drug Review with Digital Tools
  • Postmarketing Safety Surveillance and Oversight
  • Improving Drug Safety through Research
  • Advancing Drug Safety Science through Public-Private Partnerships

Improving Operations and Management in Support of Drug Safety

  • The 2015 GAO Report: A Call To Improve Data on Safety Issues
  • Opioid Addiction and Abuse: Addressing a National Crisis
  • Safe Use Initiative
  • Working to Ensure Drug Product Quality
  • Compounded Drugs and Drug Supply Chain Security

 Communicating Drug Safety: A Global Public Interface

  • Expert Responses to Public Queries
  • Social Media and Online Tools
  • Drug Safety Communications
  • Safety Labeling Changes
  •  Risk Communications Research


FY 2017 User Fees for Priority Disease Review Vouchers

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Rare Pediatric Disease Priority Review Voucher 

Tropical Disease Priority Review Voucher 

 OPEN- Consumer Representative Positions on FDA Advisory Committee

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  • Represent consumer perspective on issues and actions before Advisory Committee
  • Serve as liaison between Committee and interested consumers, associations, coalitions, and consumer organizations
  • Facilitate dialogue with Committees on scientific issues that affect consumers.. FDA is currently recruiting to fill

Consumer Representative positions.

OPEN- Nominations for Voting Members for CDRH Patient Engagement Advisory Committee


  • Improve communication of benefits, risks, clinical outcomes
  • Increase integration of patient perspective in regulatory process
  • Identify new approaches, unforeseen risks, unintended consequences resulting from FDA policy

CDRH Patient Engagement member



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