Rare Disease Policy fails to meet dire treatment needs of patients: ORDI

No specific funding allocated for the treatment of Rare Diseases (Group 2 and Group 3 disorders); rather it suggests relying on crowdfunding which is not a practical solution

Organization for Rare Diseases India (ORDI) believes that many things are woefully missing in the National Policy for Rare Diseases, 2021 (NPRD, 2021). For one thing, the government has taken a rather cavalier approach to the issue of affordability of the care needed to sustain a life of dignity for persons living with rare diseases. To tackle the issue of affordability, the NPRD 2021 suggests that an ailing/dying patient or their family should rely on crowdfunding for treatment. 

The NPRD invokes the Rashtriya Arogya Nidhi (RAN) Scheme, stating that Rs 20 lakh would be given to diseases falling within Group 1 of the NPRD, 2021 (i.e., disorders amenable to one-time curative treatment. This includes diseases such as mucopolysaccharidosis (MPS) type I within the first two years of age. Adrenoleukodystrophy (early stages), before the onset of hard neurological signs, immune deficiency disorders like Severe Combined Immunodeficiency (SCID), Chronic Granulomatous disease, Wiskot Aldrich Syndrome, Osteopetrosis, Fanconi Anemia etc. This amount is inadequate to cover the costs of treatments for these diseases, which are needed more than once. State governments have been given the discretion to provide for the treatment needed to manage diseases listed in Group 2 (i.e., diseases that require long term care). The NPRD, 2021, does not give details on funding, allocation, responsibilities, and the government’s role in providing supportive care (i.e., care that comes from the second line of treatment). It merely leaves it to the states to figure out how they want to handle it.

All this while, despite health being a state subject, the states have done nothing significant for rare disease patients; even in cases that have treatment as low as diet management. Expecting the clueless state to manage group 2 and 3 disorders on its own without any sort of guidance is a huge flaw in the policy. The woefully inadequate funding allocated by the Government, and the offhand suggestion of relying on crowdfunding and CSR initiatives means that patients and their families are left largely in the lurch when it comes to affording any kind of care. Similarly, the NPRD 2021, mentions the need to encourage local innovation and manufacturing, as a way to reduce costs of drugs, devices and other therapies. While this is encouraging, the fact that no real commitment or allocation has been made as to how much money will go into this or should ideally go into this is a real spirit dampener. It means that more time will be lost in trying to set up systems to promote this kind of research. For example, setting up a research fund, with little to no guidance on how projects are selected, what kind of oversight should be exercised on such projects, and how the fund itself will be administered and sustained. 

There is yet another challenge to be addressed- and that is data. How has the list of rare diseases currently in the NPRD 2021 been collated without clearly defining or setting out the parameters of what would be considered a rare disease? Without data on the number of persons living with a certain rare disease, how will companies know the demand, or how to structure their clinical trials? How will any government funding, allocation, and schemes work, if we don’t have a clear metric to decide what diseases are considered rare diseases? Finally, there are 7000+ known rare diseases. A piece-meal approach, that counts only some rare diseases and not all, is a terrible one with terrible consequences. Not being “listed” amounts to not being counted, and being counted, could mean the difference between life or death. 

“The NPRD, 2021, is a mockery of the right to health each person in India enjoys because it ignores the true needs of the rare disease community in India,” says Prasanna Shirol, Founder Director, ORDI. “The lack of foresight and grounding in the rare disease community’s reality shines through the cold and thoughtless drafting of the NPRD 2021. A truly shocking thing, given the hundreds of high court cases, individual representations, and representations spanning almost two decades by patient advocacy groups. But we will continue to fight on – till every person living with a rare disease, irrespective of age, or any socio-economic factors, has the right to health.” 

ORDI is currently conducting discussions with their stakeholders to collate necessary information and submit an extensive report to the Ministry of Health and Family Welfare to review the policy.