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

SUBMITTER: Chakraborty M 

PROVIDER: S-EPMC10021652 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature