FDA Views: Orphan Drug Designation, Patient Representative Voice, Clinical Trial Diversity – Drug and Device Digest

FDA BRIEF: Week of July 29, 2016


The Rise in Orphan Drug Designations: Meeting the Growing Demand 

by: Gayatri Rao, M.D., J.D., Director for The Office of Orphan Products Development

Dr. Gayatri Rao

Rare Disease drug development became reality with Orphan Drug Act in 1983

  •  Orphan Drug Designation Program
  • Financial incentives to encourage companies
  • Major tax credits to defray the cost of clinical trials
  • Seven years of market exclusivity
  • No user fee

Continued growth of  FDA’s Office of Orphan Products Development – 30% increase in 2016 vs. 2015

  • Strive to review 75%  designation requests within 90 days
  • Designated drugs need to satisfy criteria for designation and the financial incentives

Sponsors play a critical role in ensuring high quality and complete designation requests


The Unique Voices of Our Patient Representatives

by: Robert M. Califf, M.D., Commissioner &  Heidi C. Marchand, Pharm.D., Assistant Commissioner, Office of Health and Constituent Affairs

Robert Califf

Heidi Marchand

Patient Representative Workshop

  • Met with 21 inspirational patients and patient caregivers who are FDA patient representatives
  • Patients add context and content to the science – important in regulatory decision-making
  • Include perspectives and voices in entire medical product continuum : development to review and evaluation to post-market surveillance
  • Training patient representatives to serve on FDA advisory committees, meetings and workshops

Unique skill set and experiences

  • Elite world class athlete – benefit/risk experience with anticoagulants
  • Caregivers for rare disease  – advocates to find a cure
  • Parent of child with two craniotomies – importance of opioids for pain

2016 FDA Patient Representative Group photo

FDA looking for new and better ways to integrate patient voice







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