Estimates show that a country like India has a high illness burden in addition to a high burden of rare diseases. However, despite the significant prevalence of rare diseases in India, research has shown that very few Indian patients participate in worldwide drug development programs for people with uncommon diseases. This has to change.

The US government′s clinical trial registry states that 202 trials were conducted for the 63 orphan drugs that were approved after 2008. Only 9 of these had been undertaken in India. Practically less than 1% of the remaining 193 trials included individuals of Indian origin.

By neglecting the inclusion of Indian population in clinical studies Global pharma corporations are overlooking the health and lives of up to 80–100 million people suffering from rare diseases. Pharma producers and researchers may be interested in India because of the country′s highly diverse ethnic and racial population. 

So, it is necessary to start with the data currently available for the rare diseases already existing in the country. Global pharma companies may encounter numerous difficulties during the journey. Despite the difficulties, they ought to think about involving more Indian patients in their orphan medication clinical studies. Indian patients′ inclusion would at least give patients the extremely unusual chance to get access to treatment for at least the duration of the trial. Additionally, since 80 % of uncommon diseases have hereditary causes, children are the ones who are most affected.

This insight would improve the scientific validity and rigour of the entire drug development and discovery process in addition to meeting the demands of Indian patients.