FDA BRIEF: Week of September 11, 2017
FDA is Advancing the Goals of the Orphan Drug Act
By: Scott Gottlieb, M.D., FDA Commissioner
Orphan Drug Modernization Plan
- eliminate a backlog of about 200 orphan drug designation requests t
- pursue policies better advance the goals of the Orphan Drug Act (ODA).
Process improvements for review efficiency
- Reorganization to leverage expertise across FDA’s medical product centers
- New workflow to eliminate redundancies and delays
New policy steps for incentives
- Hold public meeting on complex scientific and regulatory issues and appropriate orphan incentives
- Examine aspects of granting designations to meet goals intended by Congress
- Issue guidance documents to address loopholes for avoiding pediatric studies
“Continuous Manufacturing” – Common Guiding Principles Can Help Ensure Progress
Priority Therapeutic Areas for Development
Standardizing study data specific to therapeutic areas (TAs) facilitate evaluation of medical products
- Prioritization of TAs based on (1) areas of particular medical need, (2) areas with existing data standardization projects underway, and (3) areas with greater drug development pipeline activity
- New roadmap for TAs in priority groupings
- Collaboration of FDA, CDISC, Critical Path Institute, HL7’s Clinical Interoperability Council to define related clinical concepts
Image credits: FDA