Making Sure the Patient Voice Is Heard
Patient-centered research and practice have been emphasized over the past decade. In addition to being the right thing to do, a growing evidence base supports the benefits of listening to the patient voice in clinical trials and in clinical practice. Patient-reported outcome measures (PROMs) are validated questionnaires that systematically capture patients’ perspectives on how they feel, function, and live their lives.
In clinical trials, PROM data assess the impact of different treatment options from the patient perspective. These PROM results inform clinical decision-making, practice guidelines, regulatory approvals, health technology assessments, and other policy decisions. In clinical practice, an individual patient’s PROM data is used to screen for conditions and monitor progress. Individual patient PROM data can improve patient-clinician communication, inform monitoring and management, improve symptom control and health-related quality of life, enhance efficiency, and in some cases, increase survival.
The PROTEUS (Patient-Reported Outcomes Tools: Engaging Users & Stakeholders) Consortium, which includes AcademyHealth as a member, was formed to help navigate the use of PROMs in clinical trials and clinical practice. The Consortium includes more than 50 patient, clinician, research, health system, industry, policy, and regulatory groups from the US and around the world. PROTEUS disseminates and promotes implementation of tools and resources to optimize the use of PROMs in research studies and in clinical care.
Understanding Intervention Impacts from the Patient Point of View
The patient perspective has much to contribute to clinical trials and other research studies that aim to assess the efficacy and effectiveness of different interventions. From evaluations of new medications to assessments of lifestyle interventions, PROMs provide valuable information regarding how interventions impact patient functioning and well-being. “Fundamentally, only patients can describe their experiences, so PROMs provide future patients with vital information on how they may feel and function on different treatments, and provide clinicians with evidence to support clinical practice and shared decision making,” says Michael Brundage, MD, MSc, PROTEUS Co-Principal Investigator and Professor of Oncology and Public Health Sciences at Queen’s University.
To obtain these patient insights using PROMs requires a SMART approach: Specify the methods clearly, Measure the outcomes appropriately, Analyze the data properly, Report the results clearly, and Translate the findings into practice. For each step in this process, PROTEUS offers a curated collection of core methodologic tools plus original checklists, videos, and reporting templates. “The PROTEUS tools provide a unique and important resource to both academic and industry trialists to provide training on PRO methods,” says Linda Nelsen, MHS, who recently retired from the pharmaceutical industry.
Guiding Care Based on the Patient Perspective
It may seem obvious that the patient’s perspective should contribute as much as laboratory values and imaging studies to informing care. But a range of considerations must be addressed to design, implement, and manage PROM systems for use in routine care. PROMs provide a rigorous approach to enable systematic incorporation of the patient’s point of view to monitor progress and inform management. The PROTEUS Guide to Implementing Patient-Reported Outcomes in Clinical Practice synthesizes existing resources to provide updated, unified, comprehensive guidance on implementing and optimizing PROM systems.
"Patients state that our use of PROMs creates a collaborative care environment,” says Kenneth Meehan, MD, Director of the Transplant and Cellular Therapy Program at Dartmouth Hitchcock Medical Center and the Dartmouth Cancer Center. “Our research demonstrates that patients feel more involved in their care and future treatment decisions."
Judy Baumhauer, MD, MPH, Professor of Orthopaedic Surgery, University of Rochester Medical Center agrees. “Patients come to orthopaedics to improve physical function and decrease pain. Knowing the impact of a surgical treatment, like a knee replacement, on these symptoms using PROMs allows us to identify patients who will benefit from surgery rather than guess. This improves patient centeredness and value.”
However, if the patient’s perspective is not captured and communicated effectively, the benefits described above may not be attained. “If PROMs are burdensome or lack utility, patients may become frustrated and less likely to complete them accurately,” says Elissa Thorner, a patient advocate who serves on the PROTEUS Steering Committee. “Conversely, when patients see that PROMs are valued and used in their care, they are more likely to participate, leading to a more collaborative care experience and better health outcomes.”
We Can Hear You Now
PROMs provide a powerful tool to promote patient-centeredness. Resources from the PROTEUS Consortium help amplify the patient voice so that it can be heard in research and care. These data provide a valuable resource for health services and comparative effectiveness research. Interested researchers can also learn more about the implementation of PROMs in clinical practice at the upcoming AcademyHealth Conference on the Science of Dissemination and Implementation in Health on December 8-11 in Arlington, VA.