Rare Disease - Episode 16

Episode 17 - Future Outlook on the Orphan Drug Market

As Hugh Fatodu, RPh, MBA, cites the Orphan Drug Act of 1983 and explains how the act has incentivized pharmaceutical companies to expand on research and continue to develop a variety of orphan drugs for the treatment of rare diseases, the panelists explore the evolving orphan drug market and discuss their concerns.

Mr Fatodu explains that earlier-approved orphan drug products are now being used beyond their original indications and states that he is concerned about the quality of the data that is being reviewed to support product usage. He believes that this factor may affect patient exclusivity and drug pricing.

Maria Lopes, MD, MS, and Michelle Petri, MD, MPH, further discuss rising drug costs for newly developed agents and explain how expensive pricing affects a clinician’s choice to administer an appropriate drug to a patient.

The panelists conclude their discussion by reviewing cost versus quality perceptions as the US transitions into a new delivery model of care.