Patient-Reported Outcomes: Design with the End in Mind

“What outcomes matter to patients?”

This question is asked at nearly every meeting in biomedical R&D and health care. As FasterCures has documented over the years, there is a big change afoot to place patients in the center of medical product discovery, development, and delivery, and we have many tools available to facilitate inclusion of their perspectives. Now is the time to get more specific about how these tools are used to ensure patients’ perspectives are kept front and center during decision-making.

Patient-reported outcomes (PROs), and their measurement instruments (PROMs), are one tool that ideally is developed in partnership with patients to capture the outcomes that matter most to them (e.g., fatigue or specific activities of daily living). Patient responses to PROMs can be translated into numerical scores and used in several ways, including to understand treatment effects or compare treatment options. Across R&D and care delivery, many partners (such as patient organizations, clinicians, payers, and more) have a stake in patient-centered PRO development and utilization, each from a different vantage point. Despite differences in viewpoint, all stakeholders agree that PROs have not reached their full potential of delivering benefits to patients. That is why FasterCures brought diverse stakeholders together to tackle the issue.

On June 14, 2017, FasterCures held a workshop, “Patient-Reported Outcomes: Design with the End in Mind,” to convene representatives from patient organizations, regulatory agencies, payers, medical product developers, clinical researchers, and others. We asked this multi-stakeholder group to discuss the PRO-related challenges and opportunities from a broad view across R&D through care delivery, starting with the perspective of patients and patient organizations. From there we identified next steps along the path to making PROs a more potent tool for incorporating patients’ perspectives into R&D and care decision-making.