Rare disease patients in India are rarely involved in international orphan drug trials

Abstract

We wished to determine whether rare diseases patients from India had been enrolled in international trials to develop novel orphan drugs. There are two reasons to be interested in this. (a) Different ethnic or racial groups may respond differently to a particular drug. India has huge ethnic diversity, and to exclude such participants is to severely limit the diversity of any trial; (b) Even if a suitable drug for a rare disease is available in India, it may be astronomically priced, in a country where most healthcare expenditure is out-of-pocket. We identified 63 orphan drugs, approved by the US Food and Drug Administration (FDA) after 2008, for which there were 202 trials in the US government’s clinical trial registry, ClinicalTrials. gov. Only nine of these trials had run in India. These trials pertained to six drugs. The drugs were for the conditions B-cell Lymphoma, Chronic Myeloid Leukemia, Gaucher disease Type 1, Malaria, Myeloma and Pulmonary Arterial Hypertension. Further research is required as to why patients from India are not part of foreign drug development programmes for rare diseases. We then asked how many of the remaining 193 trials had recruited people of Indian origin, residing in other countries, and found that not more than 1% of these trials had done so. Also, only 11 of the 193 trials had recruited from other lower income countries. Participation from low-income countries in trials for orphan drugs is poor.