Rare diseases remain a high unmet medical need in the US. Although each rare disease affects a small number of individuals, approximately 30 million people in the US suffer from rare diseases. Children represent about half of the population affected by rare diseases, yet pediatric drug development is limited and commonly driven by drugs being developed for adults with a disease that occurs in both populations. The Rare Pediatric Disease (RPD) Designation Priority Review Voucher (PRV) program was created to further improve novel drug development for pediatrics by offering sponsors additional benefits.
In this whitepaper, our expert reviews the origin of the RPD designation program, key definitions, the application process, and the benefits and responsibilities of obtaining designation.
Written By:
Isaiah Manoogian, PharmD, Senior Regulatory Affairs Scientist
Dr. Isaiah Manoogian graduated from the University of Connecticut School of Pharmacy in May 2021. After earning his doctorate, he moved to DC to begin his career in the pharmaceutical industry as BioPharma Global’s inaugural Post-doctoral Regulatory Affairs Fellow. Following the completion of the 1-year Fellowship, Isaiah is now a Senior Regulatory Affairs Scientist with the company. In his current role, Isaiah has solely authored and supported numerous successful applications to FDA and EMA, including orphan drug designations, rare pediatric disease designations, and fast track designations.
