Robert Califf, MD, Commissioner of the U.S. Food and Drug Administration (FDA), discusses some of the challenges that the agency faces in regards to rare diseases.



Dr. Califf notes how important it is that the FDA ensures the efficacy and safety of new treatments in development for rare diseases. Due to unmet needs of the rare disease community, novel treatment options are essential in providing adequate care and hope to patients and their families. 

Thanks to recent technology advancements, the research and development of rare disease treatments is happening more often. In relation, the FDA is seeing an influx of indications that they must ensure meet the federal regulatory guidelines. This is where designations such as Orphan Drug Designation and Fast Track Designation can be crucial to pharmaceutical companies trying to gain FDA approval.

Dr. Califf also describes challenges of determining appropriate statistical measures for rare disease trials, a difficult task since patient populations are small.

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