Valuable recommendations on drug development for rare diseases [review feb 2026]

Medicine Development and Access for Rare Diseases: Can We Do Better?

Please see this review on Medicine Development, Pricing, and Access in the EU: Implications and Hurdles for Rare Diseases, with recommendations based on case to system approach at RARE-NL, and special attention for A Path Towards Socially Responsible Valorization.

Medicine Development and Access for Rare Diseases: Can We Do Better? - Hollak - 2026 - Journal of Inherited Metabolic Disease - Wiley Online Library

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