Dr. Timothy Coté, the former director of the FDA Orphan Drug's “now represent about 40% of everything the FDA approves.”
Orphan status can not only help speed the creation of new drugs for patients in need, but also deliver substantial benefits for sponsors, including:
seven years of market exclusivity
FDA assistance with protocol development and regulatory flexibility.
Furthermore, rules allow multiple orphan designations for a single drug that can progressively expand its potential market. Novartis’ Gleevec received seven different orphan designations that led to nine different marketing indication approvals between 2001 and 2013, greatly expanding its potential market and extending exclusivity for at least one indication to 2020.
FDA has granted orphan designation to more than 2800 drugs and given marketing approval to over 430 of them.
Dr. Timothy Coté, the former director of the FDA Office of Orphan Products Development, said in a recent interview that orphan drugs “now represent about 40% of everything the FDA approves.”