JMIR Publications

ABSTRACT

Background:

ClinicalTrials.gov (CT.gov) is the most comprehensive Internet-based register of different types of clinical studies. Expanded access is the use of unapproved drugs, biologicals, or medical devices outside of clinical trials. One of the key problems in expanded access is the availability to both health care providers and patients of information about unapproved treatments.

Objective:

To evaluate CT.gov as a potential source of information about expanded access programs.

Methods:

We assessed the completeness of information in the records of 228 expanded access programs registered with CT.gov from February 2017 through May 2020. Moreover, we examined what percentage of published expanded access studies have been registered with CT.gov.

Results:

We found that some important data were missing from the records of many programs. Information that was missing most often included detailed study description, facility information, central contact person, and eligibility criteria (55.26%, 53.95%, 41.67%, and 17.54% of the programs, respectively). Furthermore, 14% of the programs were registered retrospectively. We also showed that only 33 out of 77 (42.9%) of expanded access studies performed in the USA and published from 2014 through 2019 were registered with CT.gov.

Conclusions:

Currently CT.gov is a quite fragmentary source of data on expanded access programs. This problem is important because CT.gov is the only publically-available primary source of information about specific programs. We suggest what actions should be taken by different stakeholders to fully exploit this register as a source of information about expanded access. Clinical Trial: Not applicable.