Background: A patient-reported outcome (PRO) is a subjective report that comes from a patient without interpretation by a clinician. Because of the increasingly significant role of PROs in the development and evaluation of new medicines, the US Food and Drug Administration (FDA) issued a formal guidance to describe how PRO instruments will be reviewed and evaluated with respect to claims in approved medical product labeling. Meanwhile, PROs continue to appear in oncology clinical trials more frequently; however, it is unclear how payers and policymakers can use PRO data in the context of decision-making for cancer treatments.
Objective: The objective of this article is to discuss the challenges and opportunities of incorporating oncology-related PRO data into payer decision-making.
Discussion: Payer concerns with PRO instruments are often related to issues regarding measurement, relevance, quality, and interpretability of PROs. Payers may dismiss PROs that do not independently predict improved outcomes. The FDA guidance released in 2009 demonstrates, as evidenced by the case of ruxolitinib, how PRO questionnaires can be generated in a relevant, trustworthy, and meaningful way, which provides an opportunity for payers and policy decision makers to focus on how to use PRO data in their decision-making. This is particularly relevant in oncology, where a recent and sizable number of clinical trials include PRO measures.
Conclusion: As an increasing number of oncology medications enter the market with product labeling claims that contain PRO data, payers will need to better familiarize themselves with the opportunities associated with PRO questionnaires when making coverage decisions. PRO measures will continue to provide valuable information regarding the risk-benefit profile of novel agents. As such, PRO measures may provide evidence that should be considered in payers' decisions and discussions; however, the formal role of PROs and the pertinence of PROs in decision-making has yet to be understood.