This month’s infographic breaks down the global access challenges faced by patients with rare disease. Between 2000 and December 2018, the U.S. Food and Drug Administration had approved some 547 orphan therapies, and its EU counterpart, the European Medicines Agency, had authorized 164. Adding in 2019’s totals — 21 for the FDA and 17 for the EMA — that comes to 568 and 181, respectively.
Despite a pick-up in the pace of approvals the last few years, though, treatments are available for only 5% of the roughly 7,000 rare diseases. As if that weren’t enough, other challenges persist for these patients, including access issues born of high prices and licensing delays. Lest anyone think such issues get easier ex-U.S., think again; they’re common across the globe.
The result: Availability of orphan drugs is worse than that of regular pharma products. New models are needed to support providers and patients. MM&M thanks Rareiti for their assistance in compiling these data.