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Indigenous Drugs for Rare Diseases
Context:
The Central government is set to launch an ambitious scheme aimed at developing 12 indigenous drugs targeting eight rare diseases, as announced by the Health Minister.
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This initiative is part of India’s commitment to enhancing affordable healthcare and aims to significantly reduce treatment costs for conditions such as muscular dystrophy and Gaucher’s disease, making life-saving therapies more accessible and affordable.
Key Highlights:
- The National Policy for Rare Diseases (NPRD) provides financial assistance of up to ₹50 lakh per patient for treatment at designated Centres of Excellence (CoEs). Since the NPRD’s inception in 2021, approximately 1,118 patients have benefited from this support.
- Laboratory Enhancements: Viral Research and Diagnostic Laboratories (VRDLs) are being strengthened, with some converting into Integrated Research and Diagnostic Laboratories (IRDLs) for broader infectious disease coverage.
The specific diseases included in this initiative are:
- Familial Duchenne Muscular Dystrophy (DMD): A genetic disorder leading to progressive muscle weakness and degeneration.
- Familial Hypercholesterolemia: A condition that results in extremely high levels of LDL cholesterol from birth, increasing the risk of early heart attacks.
- Spinal Muscular Atrophy (SMA): A genetic disorder that causes muscle weakness and atrophy.
- Gaucher Disease: Characterised by bone pain, anaemia, and organ enlargement due to lipid accumulation.
- Pompe Disease: A metabolic disorder leading to glycogen buildup in cells, affecting muscle and organ function.
- Fabry Disease: An X-linked disorder causing lipid deposits in tissues.
- Niemann Pick Disease (NPD): A group of inherited disorders that lead to lipid accumulation in the brain, liver, and spleen.
- Alkaptonuria: A rare disorder preventing the complete breakdown of certain amino acids, leading to dark urine and other complications.
A Step Towards Atmanirbhar Bharat:
- This initiative aligns with the vision of Atmanirbhar Bharat (self-reliant India), aiming to reduce dependency on imported drugs and foster innovation within the country.
- By developing indigenous drugs, the programme not only addresses the immediate healthcare needs but also strengthens India’s position in the global pharmaceutical landscape.
National Policy for Rare Diseases, 2021
- The Government launched the NPRD in March 2021 to support treatment for rare disease patients.
- The policy categorises rare diseases into three groups:
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- Group 1: One-time curative treatments.
- Group 2: Long-term treatments with lower costs and documented benefits.
- Group 3: Diseases with definitive treatments but high costs and lifelong therapy.
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- The policy promotes research and development for the diagnosis and treatment of rare diseases and encourages local drug development and manufacturing.
- The Department of Pharmaceuticals has introduced the Production Linked Incentive Scheme, which provides financial incentives to manufacturers for the domestic production of various products, including orphan drugs used in treating rare diseases.