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Incorporating patient-reported outcomes optimally into clinical trials remains challenging
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I fully support incorporation of patient-reported outcomes (PROs) into clinical trial design. However, I will take this opportunity to discuss challenges of incorporating PROs into clinical trials.
As I see it, the question is not should we incorporate PROs into clinical trials, but how to do so effectively and efficiently to provide clinically and scientifically meaningful data. Ideally, integration of PROs should begin in the early phases of trial development using a team science approach to map each PRO assessment strategy to the most relevant unanswered questions in the condition under study. Use of a common set of core PRO measures is helpful to optimize cross-study comparisons.
However, adapting the choice and timing of assessments to the specific circumstance of each trial is most likely to provide meaningful information. For example, the same measure may perform differently in varied settings. The timing of response may influence its clinical relevance. What defines a “clinically meaningful” result may differ by setting and requires interpretation. Finally, engaging our patients and their advocates early in this process is key to understanding which questions to ask and when.
A practical challenge faced when proposing addition of PROs to clinical trials is a concern regarding patient burden. As a geriatric oncologist, studying the value of geriatric assessment, I have heard this concern. Respecting our patients’ time is paramount. Strategies to minimize burden include use of efficient measures, avoidance of redundancy, feasibility assessment and optimal timing. Learning from past measure performance in specific settings is imperative.
Importantly, we must engage our patients. I cannot recall having had a patient complain to me in my practice that I was spending too much time getting to know them. The same is likely true in clinical trials — we simply need to optimize our approach.
Finally, it is important to recognize that PROs may not adequately capture certain characteristics, such as physical function or cognition. Objective assessments provide complementary, but distinct, information from PROs. Studies have shown that objectively measured physical function provides unique information to predict health-related events and as an outcome assessment. Recognizing limitations of specific PROs related to sensitivity or bias can inform optimal measure selection and knowledge gained.
Integration of PROs into clinical trials is likely to promote value-based patient-centered care. The challenges can be overcome, and the gain is worth the effort.
For more information:
Heidi D. Klepin MD, MS, is a geriatric oncologist at Wake Forest Baptist Comprehensive Cancer Center. She can be reached at hklepin@wakehealth.edu.
Disclosure: Klepin reports scientific advisory roles with Celgene and Genentech.