Govt to launch a programme to develop 12 indigenous drugs for 8 rare diseases

New Delhi: The Central government is set to launch a scheme to develop 12 indigenous drugs for eight rare diseases, health minister J.P. Nadda said on Monday. The programme aims to provide financial relief to many rare disease patients in the country.

“As part of India’s drive towards becoming a global leader in affordable healthcare, DHR is set to launch a programme developing 12 indigenous drugs for eight rare diseases. This initiative will aim to drastically reduce the cost of treatment for conditions like muscular dystrophy and Gaucher’s disease, making life saving therapies accessible and affordable for the masses,” said Nadda.

A health ministry official later listed the diseases as: Familial Duchenne Muscular Dystrophy (DMD), Familial Hypercholesterolemia, Spinal Muscular Atrophy (SMA), Gaucher Disease, Pompe Disease, Fabry Disease, Niemann Pick Disease (NPD) and Alkaptonuria.

Currently, the National Policy for Rare Diseases (NPRD) lists 63 rare diseases. Financial support is provided of up to ₹50 lakh per patient during the course of the treatment at centres of excellence (CoEs) for rare diseases.

Since the launch of the policy in 2021, approx. 1,118 patients have benefited under NPRD.

Health conditions classified under the policy are osteopetrosis that causes bone diseases; Fanconi Anemia that causes bone marrow failures, tumours and other abnormalities; Laron Syndrome that causes growth hormone insensitivity; and immune deficiency disorders such as severe combined immunodeficiency and Wiskot Aldrich Syndrome.

The Union government has established 12 centres of excellence situated at premier government hospitals with facilities for diagnosis, prevention and treatment of patients with rare diseases. 

The rare diseases

Familial DMD is a genetic disorder that leads to progressive muscle weakness and degeneration, Familial Hypercholesterolemia is a disorder that causes LDL (bad) cholesterol level to be very high. The condition begins at birth and can cause heart attacks at an early age.

SMA is a genetic disorder that causes progressive muscle weakness and atrophy. 

Gaucher disease causes bone pain, anaemia, enlarged organs, a swollen, painful belly, and bruising and bleeding.

Pompe Disease is a metabolic disorder that causes a buildup of glycogen in the body’s cells, damaging cells and impairing the function of organs and tissues, especially muscles.

Fabry Disease is a rare X-linked lysosomal disorder that results in excessive deposition of lipids in the tissues.

NPD is a group of inherited disorders that cause lipids to build up in the cells of the brain, liver, and spleen.

Alkaptonuria, or black urine disease, is a very rare inherited disorder that prevents the body fully breaking down two protein building blocks (amino acids) called tyrosine and phenylalanine.

Other government initiatives

Nadda, while highlighting the initiatives taken by his ministry in the first 100 days of the government, said a Centre for Evidence-Based Guidelines is ready for inauguration to help standardize medical practices nationwide, ensuring better standards of care.

The department also launched the National One Health Mission (NOHM) for Pandemic Preparedness to manage zoonotic diseases and pandemics.

The government also launched the Med-Tech Mitra online platform to help meditech innovators, startups and industry partners fulfill regulatory compliance.

Inspired by India’s landmark Chandrayaan-3 mission, the department has also undertaken “First in the World” challenge to fund 50 high-risk, high-reward innovations in biomedical research. 

“These initiatives represent transformative steps in healthcare innovation, pandemic preparedness, and the development of indigenous medical solutions, contributing to a healthier, more resilient and Atmanirbhar Bharat,” he said.

The establishment of the “Research to Action” vertical in DHR will ensure that cutting-edge health research is seamlessly integrated into policy and practice.

The government said that Viral Research and Diagnostic Laboratories (VRDLs) across the country have been strengthened through funding support. “Six of these VRDLs are being converted into Integrated Research and Diagnostic Laboratories (IRDLs) covering larger domain of infectious diseases. Construction of Zonal laboratories of National Institute of Virology (NIV) has also been initiated,” it said.