New Guidance on Incorporating Patient-Reported Outcomes in Clinical Research
Patient-reported outcome (PRO) measures are questionnaires about symptoms, side effects, or quality of life.
“PROs are commonly used in clinical trials to track how patients feel and function,” says Angela M. Stover, PhD, assistant professor of health policy and management at University of North Carolina at Chapel Hill.
PROs are a standard endpoint in clinical trials. “What is becoming more common is the use of PROs as standard of care, where everybody coming through the door at a clinic completes a PRO, and a clinician reviews it to identify concerning symptoms,” Stover says.
PROs can be used to develop healthcare policy and regulatory decisions, and also to monitor symptoms to provide timely care tailored to the patient’s needs.
“We are rapidly developing and refining approaches for including PROs in clinical trials,” reports Devin Peipert, PhD, assistant professor of medical social sciences at Northwestern University.
Researchers are learning how to implement PROs for routine use.1 “These differing uses of PROs in research and routine care settings may lead to uncertainties for patients about why PRO data are being collected and data privacy,” Stover says.
Certainly, this issue should be addressed during the informed consent process. But as researchers and clinical trial sponsors seek to incorporate PROs in study protocols, they need ethical guidance. Peipert, Stover, and colleagues developed guidance for important ethical considerations for IRBs if PROs are included in study protocols.2
According to the guidance, IRBs should consider whether a trial includes a clear rationale for the PROs selected, along with when the PROs will be administered and how the data will be used. If the individual’s care team will be notified about concerning symptoms, when and how?
How will equitable access to PRO symptoms be monitored and mitigated? If there is relevant information included in consent forms about the PRO, how will the PRO data be collected, and how will the patient be contacted to complete PROs? “Of course, the IRB should be looking to make sure all of a trial’s participants have access to complete PROs if they want to,” Peipert adds.
IRBs should be aware there is a lack of guidance on how clinical or research staff should manage situations in which PRO data reveal concerning levels of psychological distress or physical symptoms. Sometimes, it requires an immediate response. For example, study participants may report severe pain or nausea. Currently, research staff are handling such data inconsistently.
Inclusion of underserved populations is another pressing concern. This includes individuals with limited or no access to the internet; disabilities such as sight impairment, arthritis, or cognitive function; those in poor health; or people with learning disabilities and poor literacy comprehension skills.3 “Patient-reported outcome assessment must be inclusive and equitable,” Stover says.
1. Stover AM, Haverman L, van Oers HA, et al. Using an implementation science approach to implement and evaluate patient-reported outcome measures (PROM) initiatives in routine care settings. Qual Life Res 2021;30:3015-3033.
2. Cruz Rivera S, Aiyegbusi OL, Ives J, et al. Ethical considerations for the inclusion of patient-reported outcomes in clinical research: The PRO ethics guidelines. JAMA 2022;327:1910-1919.
3. Calvert MJ, Cruz Rivera S, Retzer A, et al. Patient reported outcome assessment must be inclusive and equitable. Nat Med 2022;28:1120-1124.
Patient-reported outcomes can be used to develop healthcare policy and regulatory decisions, and also to monitor symptoms to provide timely care tailored to the patient’s needs.
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