ClinicalTrials.gov as a Source of Information About Expanded Access Programs: Cohort Study

Area of Science:

• Clinical Informatics
• Health Services Research
• Regulatory Science

Background:

• ClinicalTrials.gov (CT.gov) is a primary registry for clinical studies.
• Expanded access allows use of unapproved treatments outside trials.
• Information availability on expanded access programs is a significant challenge for patients and providers.

Purpose of the Study:

• To evaluate ClinicalTrials.gov as a comprehensive information source for expanded access programs.
• To assess the completeness of data within CT.gov for expanded access initiatives.
• To determine the registration rate of published expanded access studies on CT.gov.

Main Methods:

• Analysis of 228 expanded access program records on CT.gov (Feb 2017-May 2020).
• Examination of the proportion of published US expanded access studies registered on CT.gov (2014-2019).
• Logistic regression and mediation analyses to identify predictors of missing information and non-registration.

Main Results:

• Significant data gaps identified in CT.gov records, including study descriptions (55.3%), facility information (54.0%), central contact (41.7%), and eligibility criteria (17.5%).
• Central contact information was more likely missing in earlier registrations (2017).
• Only 42.9% of published US expanded access studies were registered on CT.gov; no significant predictors of non-registration were found.

Conclusions:

• ClinicalTrials.gov currently provides a fragmented view of expanded access programs.
• The incompleteness of CT.gov data hinders access to vital information for healthcare providers and patients.
• Stakeholder actions are recommended to improve CT.gov's utility as a comprehensive resource for expanded access information.