Changes needed to the National Program for Rare Diseases in India

The Government of India launched the National Program for Rare Diseases (NPRD) a few years back. In its current form, 63 rare diseases (including Atypical HUS) are covered. The program will fund a one time grant of Rs. 50 lakhs to each patient towards treatment. Decisions to fund such treatment will be made by Centres of Excellence (CoE) identified by the government.

Such a policy was unthinkable till a few years back. The debate between spending large sums of money on the treatment of a few people versus spending that money on basic healthcare necessities like preventing infant mortality, basic sanitation and other aspects of primary healthcare which impact several times more people is far from settled. While I may be biased in favour of the former due to my medical history, I do acknowledge that the choice is tricky. I am grateful therefore that the Indian government chose to set up the NPRD which impacts only a few people.

I would still suggest that two changes be made to the NPRD.

One size does not fit all

Currently, irrespective of the disease and the extremely varied types and durations of treatment, the NPRD stipulates the same grant for every patient. While the Rs. 50 lakhs might be more than enough for some, it is woefully inadequate for others. Even within the same rare disease, the requirements could be different. 

Take my disease, aHUS, for instance. Depending on the age of the patient and specific genetic mutation, the dosage and the duration of treatment with Eculizumab (the only approved drug to treat the disease) could vary. Some people would need only two vials twice a month while others could need three to four vials twice a month. Again, some patients may need it lifelong while others could need it only for a few months.

Keeping this reality in mind, the NPRD needs to allocate different funds for different patients. I understand this could make the decision process extremely complex. Who decides, for instance, which patient gets how much money? A group of experts can be constituted for each disease and a framework can be established to guide such decisions. Other solutions can be explored based on the experience of other nations where these problems have been successfully resolved.

Involvement of CoEs

In the current policy, decisions pertaining to applications for assistance under the NPRD are to be taken by CoEs. One can only imagine the delays this would bring about in getting access to the drugs by patients. The process would involve the treating physician getting the history and the clinical summary ready and then getting this information to the appropriate committee in a CoE close by. The authorities who decide on such applications in the CoE would need to then meet and review the dossier submitted. They might have further questions which would need to be communicated to the treating physician and then if everything is clear, they would decide. 

In diseases such aHUS, time is critical. Quick administration of the drug can mean the difference between remission and progression to kidney failure or even death. In such circumstances, does it really make sense to allow so much delay in deciding on the application?

One possible solution could be to not require a committee but one doctor among a panel of doctors in the CoE to decide on the application. The treating physician should be able to request urgent hearing of the case and decisions should be taken in less than 48 hours. Such models have been adopted in other nations already where similar circumstances exist.

The Government of India has shown amazing foresight and consideration for rare disease patients in coming up with the NPRD. If the kinks mentioned above can be ironed out, it would do tremendous benefits to the intended beneficiaries of this policy.