Rare disease patients wait (and wait) for the FDA to catch up on the drug review process

Hundreds of drugs developed to treat rare diseases are in limbo as drug makers wait for approval from the Food and Drug Administration (FDA).  FDA Commissioner Scott Gottlieb has ordered his agency to clear the backlog, reports Morning Consult.

“Orphan drugs” are treatments designed for rare diseases, also called “orphan diseases.” Orphan drug review requests doubled from 2012 to 2016, contributing to a delay in orphan drug designations. How full is the FDA in-box? More than 200 orphan drug designation requests are at least 120 days old.

Gottlieb pledges to improve the FDA review processes so all new requests receive a response within three months.

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