The Union Health Ministry has made generic medications available to support the care and treatment of four conditions: Wilson's disease, Gaucher's disease, Tyrosinemia-Type 1, and Dravet-Lennox Gastaut syndrome. This move will relieve individuals suffering from rare illnesses across India. All drugs used for treating rare diseases were so far being imported and were expensive. This implies that these medications will be purchased for anywhere from 60 to 100 times less than what they currently fetch on the market.

The Ministry is also in the process of making available drugs for more rare diseases, including Phenylketonuria and Hyperammonemia, over the next few months. “The approvals for these drugs are awaited,” said V.K. Paul, NITI Aayog’s member with expertise on health issues. He added that this initiative would also result in patients’ costs dropping from crores annually to mere lakhs. Additionally, a sickle cell disease drug formulation will now be made available for children.

The Central Government has prioritized 13 rare diseases and sickle cell disease. The most awaited initiative was taken in July 2022 after discussions held with the academia, pharma industries, organizations, and CDSCO department of pharmaceuticals.

"After the discussion with pharma companies, scientists, drug regulators, and academia we decided to initiate manufacture to deliver the drugs. We prioritized drugs for 13 rare diseases and for sickle cell disease also," official sources from the Union Health Ministry stated.

"This is a revolutionary change with a huge cost difference. If a drug is costing 2.5 crore then in India it will cost 2.5 lakh," sources said.

The drugs that are being launched as part of the initiative, said the official, is going to be the “game-changer” and will bring about a paradigm shift in the rare disease treatment landscape in the country and other low-income nations.

A rare disease is a health condition of a particularly low prevalence that affects a small number of people — less than 1 per 1,000 according to the World Health Organisation (WHO) — but collectively afflicts about 6-8 percent of the population in any country at any given time.

Image source: Hindustan Times