'Made in India' drugs to cut treatment costs of rare diseases by nearly 100 times

Addressing the financial barriers in treatment of rare diseases, Indian pharmaceutical firms have initiated the production of medicines for conditions such as Tyrosinemia Type 1, Gaucher’s Disease, Wilson’s Disease, and Dravet-Lennox Gastaut Syndrome, resulting in cost reductions of up to 100 fold.

Indian drug companies have successfully manufactured medicines for Tyrosinemia Type 1, Gaucher’s Disease, Wilson’s Disease, and Dravet-Lennox Gastaut Syndrome, significantly lowering the treatment costs.

An additional four medicines, expected to be available by early 2024, promise further affordability compared to their imported counterparts.

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In tandem with addressing rare diseases, the Indian government has urged the pharmaceutical industry to produce an affordable oral solution for hydroxyurea, essential for treating children up to 5 years old with sickle cell disease.

Cost reduction examples

• Eliglustat Capsules (Gaucher’s Disease): Costs reduced from Rs 1.8 crore (around 0.2 million USD) -Rs 3.6 crore (around 0.4 million USD)annuallyto Rs 3 lakh (3,600 USD)-Rs 6 lakh (7,201 USD), benefitting adults with this condition causing fatty deposits on organs.

• Trientine Capsules (Wilson’s Disease): Price drop from Rs 2.2 crore (around 0.26 million USD) for a 10 kg child to Rs 2.2 lakh (around 2,600 USD), addressing excess copper deposition leading to psychiatric symptoms.

• Nitisinone Capsules (Tyrosinemia Type 1): Cost decreased from Rs 2.2 crore(around 0.26 million USD) annually for a 10 kg child to Rs 2.5 lakh(around 3,000USD), addressing a metabolic condition causing liver diseases.

• Cannabidiol Oral Solution (Dravet-Lennox Gastaut Syndrome): Reduced from Rs 7 lakh (around 8,400 USD) - Rs 34 lakh (40,809 USD) per year to Rs 1 lakh (around 1,200 USD - Rs 5 lakh (6,000 USD) for a 10 kg child, offering relief for genetic epilepsy syndromes.

Government collaboration with the pharmaceutical industry has been crucial in identifying priority conditions and facilitating non-profit production of these vital therapies. The companies have agreed to manufacture these medicines without a profit motive, recognizing the limited demand due to the rarity of these conditions.

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With 6-8 per centof the population estimated to have rare diseases, affecting 8.4 crore to 10 crore Indians, the government is actively working to make cheaper, locally manufactured therapies available for at least 13 priority conditions.

Considering the dispersed population of rare disease patients, the government is exploring ways to make these medicines available beyond centers of excellence for rare diseases.

(With inputs from agencies)