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
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
- Go to www.fda.gov/orphan for guidance
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
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
FDA looking for new and better ways to integrate patient voice
