06 · GS-II: Public Health, Pharmaceuticals & Rare Diseases
National Policy for Rare Diseases: Financial Assistance Caps, Orphan Drug Manufacturing and Compulsory Licensing
NEWS PEG
- Patient advocacy groups and medical oncologists petitioned the Union Health Ministry to expand the financial assistance limit under the National Policy for Rare Diseases (NPRD) from ₹50 lakh per patient to cover lifelong maintenance therapies.
- Data indicated that over 8 crore Indians suffer from rare genetic disorders (Spinal Muscular Atrophy, Gaucher's Disease, Lysosomal Storage Disorders), where annual imported drug costs can exceed ₹2–4 crore per patient.
- The Department of Pharmaceuticals initiated schemes to promote domestic clinical synthesis of orphan drugs and explored invoking Section 84/92 of the Patents Act (Compulsory Licensing) for essential patented therapeutics.
STATIC FOUNDATION
- Rare Disease Definition (WHO): Often defined as a disease affecting 1 or fewer individuals per 1,000 population; over 7,000 distinct rare diseases identified globally, 80% having genetic origins.
- National Policy for Rare Diseases (NPRD), 2021: Categorizes rare diseases into Group 1 (one-time curative treatment), Group 2 (long-term/lifelong treatment with lower cost), and Group 3 (long-term treatment with very high cost); provides up to ₹50 lakh financial support at designated Centers of Excellence (CoEs).
- Compulsory Licensing (Section 84 & 92 of Patents Act, 1970): Allows the Controller to grant a licence to a domestic manufacturer to produce a patented drug without the patentee's consent in cases of national emergency, extreme urgency, or public non-commercial use.
PRELIMS TRAP & PYQ BRIDGE
MAINS ENHANCEMENT
- Right to Health as Part of Life (Art 21): Universalizing Rare Disease Coverage — Creating a dedicated national rare disease fund with public-private crowdfunding integration.
- Indigenous Biopharma Manufacturing: Incentivizing Orphan Drug Clinical Trials — Granting fast-track regulatory clearances and patent waivers for domestic biotechnology firms.
- Preventive Genetics: Pre-Marital & Pre-Natal Screening in Endogamous Clusters — Scaling community-level genetic counseling under the National Health Mission.