FDA BRIEF: Week of Nov. 14, 2016

FDA Voice

Orphan Disease R & D Has a Home at FDA

John J. Whyte, MD, MPH, Director, Professional Affairs and Stakeholder Engagement, FDA

Gayatri Rao, MD, JD, Director, Office of Orphan Products Development, FDA


FDA Research on Zika Virus Vaccines and Therapeutics

Neonatal mouse model provides a new platform for Zika virus research

  • New mouse model developed by FDA for exploring activity of Zika vaccines/therapeutics
  • C57BL/6 mouse strain susceptible to Zika, develops neurological symptoms and eventually recover from disease

PLoS Publication


  • Acknowledges ‘ fragmentation’: FDA approval/clearance vs. approval for coverage and payment
  • States shared sources of evidence for both Agencies while still applying the most appropriate criteria to their decision making
  • Clarified differing FDA and CMS standards:
    • FDA: Product approval or clearance, based on  “substantial evidence”
    • CMS: Coverage approval based on  “reasonable and necessary”
  • Recommends early involvement of health systems and payers to help to understand and address the kinds of evidence needed to incorporate the new product into practice
  • Highlights several FDA-CMS initiatives to facilitate gathering adequate evidence for both Agencies
  • Provides perspectives on ‘personalized medicine’



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