#orphan drugs

| | Radar

EU orphan drug regulations haven’t “failed” but reforms needed, says European rare disease advocacy group

Two parents hold their child, who has spinal muscular atrophy 1. Source: EURORDIS - Rare Diseases Europe.

Last week, a report published in The BMJ slammed European drug regulations for allowing biopharmaceuticals to reap "billions" in profits on new drugs for rare diseases, but failing to incentivize sufficient R&D into new cures and treatments. A leading patient advocacy group, however, says that while the regulations may be in need of reform, they have helped advance treatments for many debilitating and deadly rare diseases.