Guide and resources to help researchers plan their orphan drug development.
Clinical Trials Toolkit
This Rare Diseases Clinical Trials Toolbox has been developed as a practical aid for developers of clinical trials on medicinal products for human use regardless of therapeutic area.
Advisory Committee for Therapeutics Toolkit
Provides procedural advice and guidance to replicate the successful TREAT-NMD Advisory Committee for Therapeutics (TACT) model in other rare disease communities.
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