Clinical Research on Orphan Drugs

Clinical research is the backbone of developing orphan drugs, which are essential for treating rare diseases. These research efforts face unique challenges, such as small patient populations and ethical considerations in trial design. Adaptive clinical trials, patient registries, and international collaboration are essential to gather sufficient data for safety and efficacy assessments. Regulatory incentives, including fast-track designations and extended market exclusivity, encourage pharmaceutical companies to invest in.

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