People with rare diseases remain a medically underserved population even in a developed country. We can then well imagine the plight of such patients in India. The ‘Rare Disease Day’ is intended to bring together the patients and families with rare diseases to discuss the need for greater awareness, more research, and better access to diagnosis and treatment. I am not sure how various authorities, including our Government, are deliberating on this healthcare issue.
People suffering from ‘orphan diseases’ often face huge challenges compared to more common diseases. These include delay in getting an accurate diagnosis, few treatment options and difficulty finding medical experts. Many such rare diseases have no approved treatment. Moreover, treatments for ‘orphan diseases’ tend to be in most cases more expensive than treatments for more common diseases.
This year, the “Rare Disease Day” will be observed in India also, on February 28, though these are not very much talked about in our country, nor is there any proper definition in place for such diseases, as yet.
The drugs meant for treating ‘orphan diseases’ have been very appropriately termed as ‘orphan drugs’, mainly due to commercial reasons, as such drugs will be used on much fewer patients with commensurate return on investments towards R&D. Thus spending expensive R&D resources toward such drugs may not make sound commercial sense.
To address this need, in 1983, the Orphan Drug Act was passed by the Congress in the USA to extend financial incentives for companies to develop treatments for rare diseases. Since then, nearly 330 ‘orphan drugs’ and biologics have been approved by the U.S. FDA, which estimates that from 11 to 14 million Americans would benefit from these ‘orphan drugs’. However, despite such commendable measures taken by the US FDA, around 15 million Americans still leave with such ‘orphan diseases’ for which there is no approved treatment.
It is interesting to note that some of these ‘orphan diseases’ are now being diagnosed in India, as well. As India takes rapid strides in medical science, more of such ‘orphan diseases’ are likely to be known in our country.
Thus the moot question is how does India address this issue with pro-active measures? In the USA, even by giving adequate financial incentives, this problem could not be effectively addressed for commercial reasons.
In my view, one of the ways to properly address this issue is to follow the model of our very own the Council of Scientific and Industrial Research (CSIR) for an ‘Open Source Drug Discovery’ (OSDD) program with global partnerships, wherever required. This initiative has been pioneered by the well known scientist and Director General of CSIR Dr. Samir Brahmachari. Andrew Witty, the CEO of GlaxoSmithKline also had mooted a similar idea in another context in not too distant past.
Therefore, to address the issue of ‘orphan diseases’, in my opinion, the OSDD model with partnerships between private, public and academia will not only prove to be a viable and more practical model to discover ‘orphan drugs’, but will also help India to effectively contribute to this important global issue – not just by observing the ‘Rare Diseases Day’ on February 28 or 29, each year.
By Tapan Ray
Disclaimer: The views/opinions expressed in this article are entirely my own, written in my individual and personal capacity. I do not represent any other person or organization for this opinion.