FDA BRIEF: Week of May 2, 2016
- Part of FDA’s Patient-Focused Drug Development initiative
- Obtain input from patients, caretakers,patient representatives on : (1) Effects of Parkinson’s disease that matter most to patients and (2) Patients’ perspectives on treatments for Parkinson’s disease.
Key Themes
- Progressive, devastating disease: Difficulty of living with the unexpected onset and progression of symptoms – bradykinesia, dyskinesia, tremor, dystonia, sleep disturbances, cognitive impairment, fatigue, and constipation.
- Impacts all aspects of patients’ lives: Severe limitations in performing at work, caring for self and family, and maintaining relationships and emotional toll
- Complexity of disease management: Selection of options, timing, pill burden, adjustments to daily changes in symptoms
- Using combination of drug and non-drug treatments. For optimal symptom management
- Need for medications for delaying symptom onset or slowing symptom progression
EvGen Part II:
Building Out a National System for Evidence Generation
Rachel E. Sherman, M.D., M.P.H., Associate Deputy Commissioner
Robert M. Califf, M.D., Commissioner
EvGen Part I posted previously in this blog
Need to remove barriers to data sharing
- Common approach for data presentation, reporting and analysis
- Transparent rules of engagement across all stakeholders
- Output public good and readily accessible to all stakeholders.
Vision for EvGen
- Common attributes for drug/biologic/device reviews: Characterization of individuals and populations and associated clinical outcomes
- Secondary use(s) of data in related fileds
Examples of Related Initiatives
- Sentinel Initiative
- National Patient-Centered Clinical Research Network (PCORnet)
- NIH Health Care Systems Research Collaboratory
- Reagan-Udall Foundation Innovation in Medical Evidence Development and Surveillance (IMEDS) Evaluation Program
- National Device Evaluation System (NDES)
More in future postings