MS. G. SWATHI,DR. ASHA SUNDARAM,DR. JEYAPRABHA
DOI: https://doi.org/India's rare disease landscape affects over 70-90 million individuals and is fraught with both legal and psychological barriers. The systemic shortcomings in diagnosis, treatment access, and psychosocial support remain largely unaddressed despite the introduction of the National Policy for Rare Diseases (2021). These issues are further compounded by the absence of enforceable constitutional healthcare rights for patients with rare diseases. This study adopts a doctrinal legal research methodology, complemented by a comparative analysis of rare disease policies from France, Japan, and Australia. The study identified a significant average diagnostic delay of 7.6 years in India, with treatment costs exceeding ₹20 crore annually per patient. This has led to the emergence of a psychological construct termed treatment awareness distress, affecting approximately 60% of patients and their families. Based on these insights, the study proposes a six-pillar rights-based framework: (1) specialized compulsory licensing for orphan drugs, (2) integrated psychological care protocols, (3) collaborative pricing and funding mechanisms, (4) national digital rare disease registries, (5) legal aid and advocacy networks, and (6) public awareness and education campaigns. The proposed rights-based matrix, implemented through a phased, three-stage roadmap, aims to align healthcare delivery with constitutional mandates and international best practices, thereby bridging the current gaps in rare disease management.
