On Wednesday, March 16th, San Diego Regulatory Affairs Network (SDRAN) organized a talk focused on “Regulatory Affairs in Orphan Drug Development”. As an attendee of the talk and advocate for rare disease awareness, we are sharing the information through this blog.
What are rare diseases and orphan drugs? How are we affected and why an awareness is required? Awareness is paramount! Like many other diseases, rare diseases can affect anyone and at any time. But unlike most of the common diseases, majority of rare diseases do not have adequate diagnostic tools and treatments. A disease is designated as a “rare disease” (also called “orphan disease”) if it affects fewer than 200,000 people in USA at a given time. Although the definition and number vary from country to country, the basic concept is same, that is, a rare disease affects only a few people in a population. However, if we see the big picture, there are more than 6,800 rare diseases that affect about 30 million Americans (data source: NIH).
Awareness is paramount! Like many other diseases, rare diseases can affect anyone and at anytime. But unlike most of the common diseases, majority of rare diseases do not have adequate diagnostic tools and treatments.
The talk at SDRAN provided the speaker’s prospective on regulatory affairs in orphan drug development. The well planned regulatory strategies and efficient interactions between the drug developing companies and government agencies (such as, FDA and EMA) are critical for the timely approval of drug applications for clinical trails or market launch. The regulatory strategies become even more vital in orphan drug development because of scarcity of the rare disease patients’ data, variable disease course, and limited natural history. The lack of sufficient disease-related data impedes the overall drug discovery and development. Thus, it is pivotal that patients, families, physicians, patient advocacy groups, and pharma work together to gather and share the data. The data sharing helps patients to connect, physicians to identify diseases, pharma to develop new drugs, and regulatory agencies to evaluate the drug applications.
Rare-is-Special thanks SDRAN and the Speaker for their presentation on the regulatory strategies in orphan drug development!
Author: Dr. Akhilesh Kumar, Community Development, Rare Is Special
Akhilesh has research experience in Neuroscience and Bone Biology, from NIPS (Japan) and NASA Ames (USA). He has studied biosciences businesses from KGI (USA). He is enthusiastic about scientific publishing and information exchange. He is founder of Rare Is Special.
Copyright © Rare Is Special, USA
