Evaluation of the National Policy for Rare Diseases(NPRD) 2021 and Ayushman Bharat in Addressing Financial Barriers, Infrastructure Gaps, and Healthcare Accessibility Challenges

Background: The governance of rare disease management in India presents a complex policy challenge due to exorbitant treatment costs, inadequate healthcare infrastructure, and systemic barriers to equitable access. Disorders such as Gaucher’s disease and spinal muscular atrophy demand prolonged, resource-intensive interventions that remain financially inaccessible to most patients. Objectives: This study aims to critically evaluate the effectiveness of India’s key policy frameworks—namely the National Policy for Rare Diseases (NPRD) 2021 and Ayushman Bharat—in addressing core challenges related to financial support, healthcare infrastructure, and equitable policy implementation for rare disease management. Methods: A qualitative analysis was conducted focusing on the financial mechanisms under the Rashtriya Arogya Nidhi scheme, operational performance of designated Centers of Excellence, and the viability of expanding Ayushman Bharat to include a broader spectrum of rare disease therapies. The study also draws comparisons with international models, including the United States’ Orphan Drug Act. Results: While NPRD 2021 provides for financial subsidies and the creation of specialized care centers, and Ayushman Bharat offers broader health insurance coverage, limitations in fiscal allocation, infrastructure, and policy execution continue to undermine these initiatives. Lessons from global frameworks highlight the potential of integrating sustainable financing, regulatory incentives, and stakeholder collaboration. Conclusion: India’s rare disease policy landscape requires a multi-pronged approach encompassing systemic reforms, expanded insurance coverage, and enhanced public–private partnerships. International best practices can serve as strategic models to reinforce India’s policy architecture and ensure timely, affordable, and accessible care for rare disease patients.