Patient-Reported Outcomes in Oncology Drug Labeling in the United States: A Framework for Navigating Early Challenges.

Background: Despite an increased use of patient-reported outcomes (PROs) in oncology clinical trials, integrating the patient perspective into drug approval decisions and documentation has been challenging.

Objectives: To review important regulatory and measurement terminology, and to provide oncology outcomes researchers and those involved with building oncology programs with tools to plan PRO data collection, particularly in relation to drug efficacy claims for drug labeling in the United States.

Discussion: When contemplating a PRO measurement strategy for oncology clinical trials, outcomes researchers are challenged in several ways. First, given multiple stakeholders, researchers must communicate with their scientific, commercial, and regulatory colleagues using often misunderstood terms, such as "label," "claim," "end point," "outcome," and "concept." Second, because stakeholders do not always have access to data from early-stage clinical trials and do not contribute to the target drug's profile in early development, researchers are often unable to address the most important question in building a measurement strategy: What do we want to say about our drug? To overcome these challenges, researchers can systematically develop an end point model to facilitate communication among drug development stakeholders using a common language and to link the building blocks of a PRO measurement strategy, including claims, concepts, questionnaires, and end points. We developed a model that characterizes a disease by its proximal signs and/or symptoms and increasingly distal health outcomes to provide researchers potential measurement concepts that can be instrumental in selecting PRO questionnaires for use in studies.

Conclusion: PRO data collected in clinical trials should be used in drug development to evaluate the drug's efficacy; it is encouraging that US regulators are willing to work with drug sponsors to overcome the challenges associated with the development, implementation, and interpretation of PROs. The tools discussed in this article can facilitate the planning process for oncology researchers, as well as assist in communicating with US regulators.