Rare’ or ‘Orphan’ diseases have received scant attention in the public domain; even more so in the current scenario owing to the pandemic. Treatment for these diseases, where available, tend to be expensive and recurring, making it unaffordable for many. Budget for these diseases is provided by the government through Rashtriya Arogya Nidhi (RAN).

In the recent monsoon session of Parliament, a question on fund allocation and utilisation of the funds under RAN was raised. Budgets allocated to this component for 2018-19 and 2019-20 were ₹7.5 crore and ₹25 crore respectively. However, no funds were released in 2018-19 and ₹1.5 crore was released in 2019-20.

This raises two concerns: The funds allocated are low. A second more puzzling concern is that utilisation is quite low as well, despite high demand for funds. According to some numbers, around 200 children in India suffer from a set of rare diseases classified as Lysosomal Disorders (LSD), and have applied to the Health Ministry for support. Industry sources estimate that the average cost of treatment for a child weighing around 10 kg could range from ₹18-45 lakh. Several rare diseases, including LSDs, tend to be chronic in nature with high and recurring treatment costs.

However, RAN only provides for a one-time grant of ₹15 lakh as treatment support at government hospitals for specified rare diseases. In the above example, the support provided by RAN is insufficient to cover the cost of the least possible estimate of one-time treatment itself, let alone recurring expenses. Is the low allocation of funds that does not even meet minimal treatment costs a deterrent to utilisation?

Another reason for such low utilisation could be lack of knowledge of rare diseases and availability of such funds.

It is clear that the patients suffering from rare diseases are not getting adequate support. Given that health is a State subject, the onus of alleviating this falls significantly on State governments, as some recent High Court judgments prove.

Indeed, States such as Kerala and Karnataka have established a State level corpus to mobilise funds for treating rare diseases, in addition to Central funding. Another solution could be allowing rare diseases to be a part of companies’ CSR spend. Pharmaceutical companies that manufacture medicines for rare diseases can be asked to share the burden too. But for any such policy measure to take concrete shape, it is imperative that the government’s contribution becomes clearer.

Creating awareness

The second problem of awareness about rare diseases is also stark. Available evidence suggests that patients in the US and UK take an average of 7.6 and 5.6 years respectively to receive an accurate diagnosis, typically involving eight physicians. In India, it may take even longer. The policy assigns the responsibility of creating awareness about rare diseases to the Health Ministry. However, the Ministry’s response to the Parliamentary question, specifically on improving awareness, appears cursory at best.

To close this awareness gap, it is important to train existing doctors on not only rare diseases but also on funds available for patients. One sustainable model to achieve this could be a PPP (Public-Private partnership), where the private sector provides the knowledge based on their years of research, and the onus of planning and execution of programs is provided by the government.

This might be another great opportunity to rope in pharmaceutical companies that are involved in rare diseases discussion into partly solving this issue. Finally, incorporating rare diseases as part of medical curriculum is equally important to train future doctors.

Several countries, including developing economies, are looking at ways to support patients suffering from rare diseases, with government support being a key feature. It is clear that along with augmenting funds, a structured approach on how limited resources are utilised, is the need of the hour.

Pingali is faculty member in the Economics area at IIM Ahmedabad; Chandrasekaran is a PGPX 2020-21 student at IIM Ahmedabad.

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