April 2022: Missing study records on Clinicaltrials.gov for orphan drugs approved by FDA– A TransperiMED Report

An article “No records found for 220 clinical trials of orphan drugs approved by the FDA” has been published on April 27, 2022, on TranspariMED.

According to the article, around 63 orphan drugs were approved by the FDA between 2009 – 2019, which accounted for 422 clinical trials.  A total of only 202 clinical trials could be located on ClinicalTrials.gov while 220 study records could not be traced. The study further focused on comparing data elements such as enrolment and study phase. It was observed that the enrolment data did not match for 30% of cases and the study phase data matched only for 75% cases.

The study team also stated that there could be various reasons why a trial could not be found on ClinicalTrials.gov, including that some studies may have run before ClinicalTrials.gov was established, some were Phase 1 trials that were not required to be registered until Final rule, some trials may remain unregistered in the US and some matches of studies on ClinicalTrials.gov may not have been identified, although such a match did exist.

The original article can be accessed here.