Douglas Paul, PharmD, PhD discusses the Orphan Drug Act and the issue of whether Pharma and Biotech companies are “gaming the system,” when bringing drugs or devices before the FDA.

The FDA Office of Orphan Products Development (OOPD) aims to advance the development of drugs and devices that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. In fulfilling that task, OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products.

The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the United States.

The Rare Pediatric Disease Priority Review Voucher Program states that a sponsor who receives an approval for a drug for a “rare pediatric disease” may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.