Measuring the value of treatment to patients: patient-reported outcomes in drug development.

Patient-reported outcomes (PROs) can be important measures of the impact and value of new drug treatments to patients. Recently, both multisector stakeholder groups and the U.S. Food and Drug Administration have carefully considered and issued guidance on best practices for the use of PROs in measuring treatment impact. When best practices are followed and PRO data are appropriately included in drug development strategy and clinical trials, these data can be part of the evidence submitted for drug approval and included in drug labeling. One study showed that PRO data were included in 30% of a sample of new drug labels and were more concentrated in certain therapeutic areas, such as anti-inflammatory agents, vaccines, gastrointestinal agents, and respiratory and urologic agents. PRO data included in labeling, or generated in a similar scientific manner, may often then be used in other communication vehicles, such as formulary submission dossiers, journal or direct-to-consumer advertisements, publications, or continuing medical education. Meaningful and reliable PRO results regarding the effects of new treatments on how patients feel and function provide useful information to those who must make decisions about the availability and utilization of such treatments.