Rare Diseases and Orphan Drugs
Rare diseases affect a small percentage of the population but collectively impact millions worldwide. Orphan drugs, developed specifically for these conditions, face unique challenges due to the limited market size. However, incentives such as the Orphan Drug Act and global collaborations have significantly boosted research and development. Despite high development costs, orphan drugs represent a lifeline for patients with otherwise untreatable diseases. The pathway from discovery to market is complex, involving rigorous regulatory scrutiny, innovative trial designs, and patient advocacy support to ensure that these drugs are both safe and accessible.
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