A “validity argument” approach takes into account that the context of each trial is different and attempts to overcome barriers historically associated with measuring and reporting patient-reported outcomes in clinical trials.
Patient-reported outcome measures hold great promise for clinical trials, but more work remains to reduce challenges historically associated with integrating these measures, write the authors of a paper recently published in Health and Quality of Life Outcomes.
The paper shares takeaways from a July 2018 DCRI Think Tank, which convened representatives from academia, industry, regulatory agencies, and the NIH to discuss the challenges linked to incorporating patient-reported outcomes in clinical trials. The DCRI Think Tank was chaired by DCRI’s Adrian Hernandez, MD, MHS and Kevin Weinfurt, PhD.
Patient-reported outcome measures are assessed via questionnaires and include information about patient symptoms, treatment side effects, and functioning and quality of life. By soliciting this information directly from the patients, investigators are able to learn more about the variability of patients’ experiences.
However, challenges to collecting and including this kind of data in a clinical trial remain, including:
- They are sometimes viewed as more subjective or less reliable than “hard” outcomes such as hospitalization or mortality.
- There are time and budget constraints associated with patient-reported outcome measures.
- Reporting often does not include guidelines for interpreting patient-reported outcome measures, which perpetuates a sense of mystery around them.
- Documentation for development of these measures may be lacking for those measures developed prior to FDA guidance.
- Stakeholders are uncertain about what and how much evidence is needed to support inclusion of patient-reported outcome measures in a trial.
It was this last challenge that became the primary focus of the DCRI Think Tank, and discussion at the meeting led to the conclusion that context is necessary when deciding how much evidence is needed to support inclusion of a patient-reported outcome measure in a trial. Attendees advocated for the uptake of “validity arguments” that would provide a compelling rationale explaining the interpretation and use of a specific patient-reported outcome measure. The inclusion of patient-reported outcome measures in clinical trials will continue to improve as discussion around what makes a compelling rationale continues, and as the use of validity arguments contributes to a repository of examples.
The authors of the paper, which was led by Duke Department of Population Health Sciences faculty member Theresa Coles, PhD, note that unanswered questions remain concerning the validity argument approach. While a repository of validity argument examples would be helpful, such a resource would have to be managed and maintained; furthermore, more work would need to be done to integrate these explanations into regulatory approval processes.
Read the entire paper in Health and Quality of Life Outcomes.