Developing valid and reliable instruments for measuring patient-reported outcomes (PROs) is the most direct approach to gaining insights from patients about their symptoms, functional status, treatment preferences, and health-related quality of life. While there is growing interest among drug developers, clinicians, payers, regulators, and patients in the development and widespread use of PRO instruments, progress has been slow. In particular, there are ongoing challenges in using PROs to support product label claims. The U.S. Food and Drug Administration (FDA) released final guidance to industry on the use of PRO instruments to support labeling claims in 2009, and in 2014 released guidance on qualifying instruments that may be used over time in multiple drug development programs. However, the interpretation and implementation of those guidances have presented significant challenges, which stem from a variety of methodological, logistical, and communication issues.
On July 16, 2014, the Engelberg Center for Health Care Reform, in cooperation with the FDA, held an expert workshop that focused on the major issues related to the development and use of PRO instruments to support labeling claims. This workshop was the first in a series of meetings, and included representatives from across industry, academia, patient advocacy groups, and government agencies. This discussion will help to inform a public workshop that the FDA will hold in spring 2015, which will focus on the development, use, and qualification of drug development tools more broadly and the state of the science around psychometric measurement theory.