Abstract

Objectives

Patient-reported outcome measures (PROMs) are tools intended to capture directly from patients how they feel and/or function. They can provide important evidence to support regulatory approval, product labeling, payer reimbursement, and clinical care decisions. However, patient-reported outcome (PRO) evidence remains underutilized in regulatory, payer, and overall healthcare decision making. This initiative aimed to understand stakeholder perspectives regarding current use of PRO evidence across the US healthcare continuum and opportunities to further advance its use in healthcare-related decision making.

Methods

A total of 3 roundtable meetings were convened with an expert panel representing patients/patient advocacy groups, clinicians, PRO experts, payers/purchasers, and policy makers. Meetings were audio recorded, and transcripts were reviewed to identify key discussion points and themes via thematic analysis methods. Group members reviewed, discussed, and commented on findings as they emerged.

Results

The themes that emerged on maximizing use of PRO evidence were (1) educating stakeholders on its value, (2) ensuring standardization of PROM development methods, including maximizing patient engagement, (3) addressing PROM implementation considerations in clinical trials and practice, (4) effectively communicating PRO evidence to stakeholders, and (5) encouraging PRO evidence integration into policy and shared clinical decision-making. A 5-component framework had resulted.

Conclusions

PRO evidence is fundamental to patient-focused drug development and clinical care. Collaboration among all stakeholders making decisions related to treatment approval, access, and use is critical to realizing the value of PRO evidence, alongside greater stakeholder transparency on how PRO evidence is used to inform decisions. The 5-component framework, codeveloped by a multistakeholder expert panel, can help to support and enhance these collaborations.