More than 10 children who were awaiting treatment have already lost their lives
More than 400 identified patients of rare diseases — mostly children — are yet to receive the Rs 50 lakh financial assistance for treatment guaranteed by the Union government under the National Policy for Rare Diseases, 2021. Lok Sabha member Varun Gandhi raised the issue in a letter to Union health minister Mansukh Mandviya dated January 7, 2023.
More than 10 children who were awaiting treatment have already lost their lives, he claimed. A rare disease is defined as often debilitating lifelong disease or disorder with a prevalence of one or less per 1,000 population, according to the World Health Organization (WHO).
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Most of these children suffer from Lysosomal Storage Disorders (LSD) such as Gaucher, Pompe, MPS 1, MPS II and Fabry disease, the letter read.
According to the Ministry of Health and Family Welfare crowdfunding platform, around 208 LSD patients can immediately be put on therapy, since the Drugs Controller General of India (DCGI) approved treatments for most of these diseases have been available in India for many years now.
The Union health ministry had formulated and launched the National Policy for Rare Diseases March 30, 2021. Under this, financial assistance of Rs 20 lakh was guaranteed for rare diseases amenable to one-time treatment identified under Group 1.
These included LSDs (metabolism errors), immune deficiency disorders and Osteopetrosis (when bones grow abnormally dense).
Financial assistance was not available for those suffering from rare diseases under Group 2 — diseases which require lifelong treatment with a relatively lower cost — and Group 3 — diseases for which definitive treatment is available but is costly, requiring optimal patient selection.
However, in a welcome move, the policy was changed in May 2022 when the financial assistance of Rs 20 lakh was hiked up to Rs 50 lakh to include rare diseases of all categories.
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“Till the finalisation of guidelines and in order to provide uninterrupted and enhanced financial assistance i.e up to Rs. 50 lakhs to the patients of rare diseases irrespective of the category of disease, funds may continue to be granted from the current budget head of Umbrella Scheme of Rashtriya Arogya Nidhi (RAN),” the order stated.
The government has identified 10 treating institutions across the country. These include All India Institute of Medical Sciences in New Delhi and Jodhpur, Maulana Azad Medical College in New Delhi, Lucknow’s Sanjay Gandhi Post Graduate Institute of Medical Sciences, Post Graduate Institute of Medical Education and Research Chandigarh, Mumbai’s King Edward Memorial Hospital, Institute of Post- Graduate Medical Education and Research in Kolkata and Center For Human Genetics (CHG) with Indira Gandhi Hospital, Bengaluru.
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Some 432 patients have been registered on the Digital Portal for Crowdfunding and Voluntary Donations for Patients of Rare Diseases, of which 20 are in urgent need of donation.
Implementation challenges have plagued the policy, first introduced in 2017 as the National Policy for Treatment of Rare Diseases.
By the union government’s own admission, some of the obstacles included “cost effectiveness of interventions for rare disease vis a-vis other health priorities, the sharing of expenditure between central and state governments, flexibility to State Governments to accept the policy or change it according to their situation.”
In 2018, an expert committee was set up for review the policy. Suggestions arising from their analyses gave way to the National Policy for Rare Diseases, 2021.
Research and development on treatment for rare diseases remain in a nascent stage. Only 5 per cent of identified rare diseases have treatment, according to WHO. The majority of which are out of reach for many due to fiscal reasons.
Most recently, in December 2022, the Indian Council of Medical Research invited proposals on therapeutics for inherited rare diseases.
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