Government to launch programme to develop 12 indigenous drugs for 8 rare diseases | Mint

New Delhi: Health Minister JP Nadda on Monday said that the central government is going to launch a scheme to develop 12 indigenous medicines for eight rare diseases. The aim of this program is to provide financial relief to many patients suffering from rare diseases in the country.

Nadda said, “As part of India’s drive towards becoming a global leader in affordable healthcare, DHR is going to launch a programme to develop 12 indigenous medicines for eight rare diseases. This initiative aims to significantly reduce the cost of treatment of conditions like muscular dystrophy and Gaucher disease, making life-saving treatments accessible and affordable to the common people.”

A health ministry official later listed these diseases: familial Duchenne muscular dystrophy (DMD), familial hypercholesterolemia, spinal muscular atrophy (SMA), Gaucher disease, Pompe disease, Fabry disease, Niemann Pick disease (NPD) and alkaptonuria.

Currently, there are 63 rare diseases listed in the National Policy for Rare Diseases (NPRD). Financial assistance is provided up to a maximum of Rs 10 lakh. 50 Lakh Treatment at Centres of Excellence (COE) for rare diseases will cost Rs 1,000 per patient.

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

The health conditions classified under the policy are osteopetrosis, which causes bone diseases; Fanconi anemia, which causes bone marrow failure, tumors, and other abnormalities; Laron syndrome, which causes growth hormone insensitivity; and immune deficiency disorders, such as severe combined immunodeficiency and Wiscott Aldridge syndrome.

The central government has set up 12 centres of excellence in major government hospitals with facilities for diagnosis, prevention and treatment of patients with rare diseases.

Rare diseases

Familial DMD is a genetic disorder that leads to progressive muscle weakness and degeneration, familial hypercholesterolemia is a disorder that causes very high levels of LDL (bad) cholesterol. This 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, anemia, enlargement of organs, abdominal swelling and pain, and bruising and bleeding.

Pompe disease is a metabolic disorder that causes the accumulation of glycogen in the body’s cells, leading to cell damage and impaired function of organs and tissues, especially muscles.

Fabry disease is a rare X-linked lysosomal disorder that results in excessive accumulation of lipids in tissues.

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

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

Other government initiatives

Highlighting the initiatives taken by his ministry in the first 100 days of the government, Nadda said a centre for evidence-based guidelines is set to be inaugurated, which will help standardise medical practices across the country and ensure better standards of care.

The Department also launched the National One Health Mission (NOHM) for pandemic preparedness to manage zoonotic diseases and epidemics.

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

Inspired by India’s historic Chandrayaan-3 mission, the Department has also launched a “World First” challenge to fund 50 high-risk, high-yield innovations in biomedical research.

“These initiatives are transformational steps in healthcare innovation, pandemic preparedness and development of indigenous medical solutions, contributing to a healthier, more resilient and self-reliant India,” he said.

Establishment of a “Research to Action” vertical in DHR will ensure that cutting-edge health research can be seamlessly integrated into policy and practice.

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