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The Union health ministry’s new proposal to offer a one-time financial assistance for rare disease patients instead of implementing the national policy for treatment of rare diseases won’t provide the relief that thousands of sufferers in India have been waiting for. The plan mooted as an interim arrangement will be a setback for thousands of patients who bank on government support for treatment, say civil society groups.
Last week, Union health minister JP Nadda had told Parliament about the interim measure. “Ad interim, the standing finance committee has approved a proposal for adding a sub-component under the umbrella scheme of Rashtriya Arogya Nidhi for provision of one-time financial assistance to those below threshold poverty line for specified rare diseases which require one-time treatment,” the minister said in Lok Sabha.
While Nadda did not specify an amount, according to people in the know, the government is considering a one-time treatment assistance of up to Rs.5 lakh per patient.
Patient advocacy groups rap the cosmetic nature of the new initiative. “Majority of rare diseases are progressive and require life-long treatment and continuous support. We need awareness campaigns and quick implementation of national treatment policy. A one-time support is inadequate in most cases,” Archana Panda, co-founder of Cure SMA India, a non-profit group that works to make spinal muscular atrophy (SMA) treatment accessible to Indians, told Pharmabiz.
In the case of SMA, a disease that leads to loss of motor neurons and early death, Nusinersen is the only drug approved by the US Food and Drug Administration. Developed by US-biotech major Biogen and marketed as Spinraza, the drug is administered directly to the central nervous system intravenously and the therapy could cost around $750,000 in the first year. “We know it is an uphill task, but the funds could be generated through CSR initiatives and non-profit groups if we have a clear-cut treatment policy in place. We have approached the government several times to make Spinraza available in India to no avail,” Panda added.
The World Health Organisation defines rare disease as an often debilitating lifelong condition with a prevalence of one or less per 1,000 population. It is estimated that one in 20 Indians is affected by one of the 7,000 diseases listed as rare diseases, of which only around 300 could be treated. The most common rare diseases recorded in the country are haemophilia, thalassemia, sickle-cell anaemia and primary immuno deficiency in children, auto-immune diseases, Lysosomal storage disorders such as Pompe disease, Hirschsprung disease, Gaucher’s disease, Cystic Fibrosis, Hemangiomas and various forms of muscular dystrophies.
As of now, only a few states such as Delhi, Karnataka and Tamil Nadu have adopted some proactive steps. While a rare disease board has been set up by the Delhi government to review patients, Tamil Nadu has established an exclusive diagnostic facility for sufferers.
Though the health ministry has recently eased regulations to import unapproved drugs in small quantities for personal use, the measure is not helpful in the case of rare disease sufferers owing to exorbitant cost.
“Whenever we approach the government, we are told about this provision in the Drugs and Cosmetics Act to import unapproved drug for personal use. Apparently we're being asked by the government to personally import a drug that costs US$ 750,000 in the first year and US$ 375,000 annually after that. With the rollback of the national treatment policy, how many of us can afford this therapy?” laments the mother of an SMA patient.
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