Evaluating the Effect of Interventions on Patient Function in Trials of Severely Ill Patients

• Activity Number: 146 - Methods to Evaluate and Correct for Bias in Patient-Reported Outcomes in Clinical Trials: Advancing the Validity of Patient-Centric Drug Development
• Type: Invited
• Date/Time: Tuesday, August 4, 2020 : 10:00 AM to 11:50 AM
• Sponsor: Biopharmaceutical Section
• Author(s): Elizabeth Colantuoni* and Daniel O Scharfstein and Ximin Li
• Companies: Johns Hopkins Bloomberg School of Public Health and Johns Hopkins University and Johns Hopkins University
• Keywords: Randomized controlled trial; Patient functional outcomes; Truncation due to death

Abstract

Mortality is a common primary endpoint in randomized controlled trials (RCTs) of severely ill patients. However, outcomes other than mortality, e.g., quality of life or other functional outcomes, are increasingly defined as co-primary and key secondary endpoints in such RCTs. Comparing functional outcomes across treatment arms is complicated since functional outcomes do not exist for patients who experience death, known as “truncation due to death”. We conducted a review of published RCTs of seriously ill patients from five high-impact general medical journals from 2014 to 2019 to identify the prevalence of functional outcomes and the statistical analysis approaches applied to these outcomes. We will review the findings from the review and describe two statistical approaches to compare functional outcomes “truncated due to death” within the context of a completed RCT evaluating cognitive outcomes 1 year after randomization in patients requiring care in an intensive care unit.