Speaker
Christopher Lindsell, PhD
Professor of Biostatistics and Biomedical Informatics
Director, Vanderbilt Institute for Clinical and Translational Research (VICTR) Methods Program
Codirector, Vanderbilt Health Data Science (HEADS) Center
Topic
Searching for a Unicorn: Understanding Stakeholder Perspectives When Selecting Outcomes for Outpatient Trials
Keywords
Research outcome; Outpatient clinical trials; Patient-reported outcomes; Researcher-observed outcomes; Pragmatic trials; ACTIV-6; Days of benefit; Mean time unwell
Key Points
- A research outcome is the main piece of data used to decide if the research intervention has failed or succeeded.
- If the incorrect outcome is chosen, the question of whether the intervention has succeeded can’t be answered accurately. Finding the correct outcome requires asking the question ‘What matters?’
- In studies that are designed to help you feel better faster, we may need to prioritize symptoms and signs (patient reported outcomes) over objective measures (researcher observed outcomes).
- Outcomes should be chosen based on the purpose of the study.
- Researchers need to balance sensitivity and specificity when choosing an outcome measure.
- The ACTIV-6 study uses an overall symptom outcome scale to determine if patients ‘feel better faster.’
- ACTIV-6 uses Days of Benefit and Time to Recovery/hospitalization and death as an outcome to determine if the intervention is successful.
Discussion Themes
It’s important to strive to complete the most powerful and efficient studies that we can do.
Having multiple medicines available that balance risk and benefit differently gives patients options for their treatment. Individual patients have differing levels of risk tolerance and different ideas about what is the most beneficial outcome.
Learn more about the ACTIV-6 study.
Tags
#pctGR, @Collaboratory1