Navigating Rare Disease Management and Orphan Drug Research in India

This review assesses India's rare disease (RD) management framework and the challenges in orphan drug development. It highlights gaps in regulations, data management, and resource allocation, affecting patient access to affordable treatments. The National Policy for Treatment of Rare Diseases (NPRD) and initiatives like the National RD Registry aim to address these gaps. Recommendations include integrated RD management systems, strengthened registries, and incentives for indigenous drug development to enhance accessibility and affordability.

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