Patient advocacy groups seek more funds for treatment of rare diseases

‘Focus on research will help to cut the cost for healthcare systems’

On Rare Diseases Day which fell on Sunday, stakeholders demanded more funds for treatment of patients afflicted with known rare diseases, besides more research.

According to the Indian Society for Clinical Research, there are 7,000 known rare diseases that have affected 300 million people across the globe. Of them, 70 million are in India, and most have no or very limited treatment options.

Society president Chirag Trivedi said one in 20 persons would live with a rare disease at some point in life. A large number of patients had no access to appropriate diagnosis or treatment. “This should be a wake-up call for clinical researchers,” he said. Focus on research could reduce the cost for healthcare systems and improve the quality of life of patients and their families, he said.

Patient advocacy groups and health experts have appealed to the Ministry of Health and Family Welfare to allocate money for treatment of patients diagnosed with treatable rare genetic disorders.

The International Gaucher Alliance and the Organisation of Rare Diseases India have separately moved the Health Ministry for a corpus of ₹100 crore for treatment of those diagnosed with disorders like lysosomal storage disorders (LSDs).

Karnataka, Kerala and Tamil Nadu have provided proof of funding from their own health budgets for diagnosis and treatment of treatable genetic conditions such as Pompe disease, Gaucher disease and MPS1, according to Prasanna Shirol, co-founder and executive director, Organisation of Rare Diseases India. Manjit Singh, national president, LSD Support Society, said nearly 190 patients, mostly diagnosed with LSDs, had registered themselves with the Rare Disease Cell of the Union Health Ministry in the last three years for treatment support. “Some of them have already lost their lives for lack of timely support,” he said, urging the government to work out a national policy and introduce a sustainable funding mechanism.

In March 2020, in response to a public interest litigation petition, the Madras High Court directed the Union government to pay ₹4.4 crore for treatment of rare disease patients in Tamil Nadu and instructed the State government to allocate ₹5 crore for treatment of 11 rare disease patients in the State. The Health Department of each of the three States has urged the Union government for a matching grant to sustain the programme.

An official of the Tamil Nadu government said the State had received ₹4.4 crore from the Union government, and the sum had been deposited in the account concerned to be used for treatment.