Developing therapies for rare diseases involves complex incentives, unique requirements, and often extensive patient engagement. The authors of RAPS’ Orphan Drug Development for Rare Diseases, Sundar ...
The biopharma industry has hit a setback in its ambition to leverage President Donald Trump’s “big, beautiful bill” to amend an Inflation Reduction Act (IRA) provision currently affecting treatments ...
The European Medicines Agency (EMA) has designated allopurinol as the first orphan drug for the treatment of Marfan syndrome, a rare connective tissue disease which has no cure to date. This disease ...
Kivo reports that over half of new FDA-approved drugs are now for rare diseases, thanks to the Orphan Drug Act of 1983, which ...
Rare disease definitions and ODD frameworks vary across regulatory jurisdictions, reflecting differences in regulatory philosophy, evidentiary standards, and health system context. Although a recent ...
A provision of the summer’s One Big Beautiful Bill Act bumping a slew of orphan drugs from Medicare price negotiation is estimated to cost the program $8.8 billion over the course of a decade, ...
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