The Inclusion of Economic Endpoints As Outcomes in Clinical Trials Reported to ClinicalTrials.gov

Background: As medication expenditures rise, payers are increasingly demanding evidence of economic value for new medications. The 2015 Professional Society for Health Economics and Outcomes Research (ISPOR) Task Force on Cost-Effectiveness Analysis Alongside Clinical Trials noted that clinical trials are increasingly including health care utilization endpoints to address this rising interest in economic information.

Objectives: To (a) describe the prevalence of economic endpoints in clinical trials submitted to ClinicalTrials.gov and (b) examine associations between trial characteristics and the inclusion of economic endpoints.

Methods: This retrospective review of ClinicalTrials.gov data extracted the characteristics of clinical trials that were submitted to ClinicalTrials.gov from January 2004 to December 2018; studied a drug and/or biological; and had a recruitment status of not yet recruiting, recruiting, active but not recruiting, or completed. Studies were classified as containing an economic endpoint based on 2 independent evaluations of the inclusion of endpoints relevant to costs, resource utilization, cost-effectiveness, productivity, absenteeism, presenteeism, or unemployment. Descriptive statistics were used to summarize trial characteristics, and chi-square analyses were used to evaluate differences in characteristics between trials with and without economic endpoints.

Results: Of the 104,885 trials included in the study, 1,437 (1.37%) included an economic endpoint; among later phase (phase 2/3, 3, 4) trials, 939 (2.54%) included economic endpoints. Compared with studies that did not include economic endpoints, those that did were less often industry funded (48.0% vs. 52.0%, < 0.001) and were for a high-spend specialty condition (24.1% vs. 27.4%, < 0.001). The proportion of trials that included economic endpoints increased by a small but significant amount over the time period studied, from 1.2% (2004-2008) to 1.6% (2014-2018; < 0.001).

Conclusions: A small but growing number of clinical trials are including economic endpoints. This finding may reflect continued industry concerns surrounding the cost and logistical challenges of piggybacking economic data collection alongside clinical trials and/or manufacturers' preferences for modeling for value demonstration. Future research is needed to better understand barriers to the inclusion of economic endpoints as well as the degree to which incorporating health care resource utilization collected during clinical trials into early economic modeling may reduce payer concerns about model transparency and bias.

Disclosures: No outside funding supported this study. Patterson reports past employment by Indivior, unrelated to this study. Mitchell has nothing to disclose. The research included in this study was presented as a nonreviewed student pharmacist poster at AMCP Nexus 2019; October 30-November 1, 2019; National Harbor, MD.