Patient Reported Outcomes Today and Tomorrow


Applied Clinical Trials

Patient Reported Outcomes (PRO) is a topic that has lived behind the scenes in drug development and clinical trials for quite awhile. But with pushes from industry and the release of the final FDA PRO Guidance for Industry, and not a little help from the way healthcare reform and data collection have evolved, PRO is a bona fide regulatory topic that gets a lot of attention these days.

On Monday, June 14 from 10:30 to 12 pm, DIA attendees can learn more about the FDA’s process for reviewing PRO measures to support labeling claims, hear an example of a PRO instrument submitted to the Agency for qualification review, and learn about how sponsors can work together through consortia to develop new trial outcome measures, including the Critical Path Institute’s PRO Consortium approach to public-private partnerships. The session is titled “Qualification of Patient-Reported Outcome (PRO) Tools to Support Labeling Claims: Development, Evaluation, and a Consortium Approach” and will be chaired by Laurie Burke, MPH, RPh, Associate Director, Study Endpoints and Label Development, CDER, FDA.

Applied Clinical Trials spoke with Nancy Kline Leidy, PhD, Senior Vice President, Scientific Affairs, United BioSource Corporation, about recent developments in the PRO field and about her presentation “Development and Documentation of PRO Tools for Regulatory Review.” “The EXACT PRO-Initiative was the first consortium to develop a PRO instrument for use in clinical trials and we’ve learned a lot since its inception,” said Leidy.

The EXACT PRO-Initiative addressed the need for a single, standardized PRO instrument for evaluating frequency, severity, and duration of exacerbations of COPD. Over 10 different pharmaceutical companies provided unrestricted support for the initiative, which involved clinical, research, and regulatory experts. Explained Leidy, “We all worked together toward a common goal of improving outcome measurement in this important treatment area, and we are pleased the instrument is now being used in clinical studies.”

Leidy will present on the Initiative’s instrument development process, from the critical step of concept clarification and determination of target claims, through the organization of supporting evidence presented to the FDA in the EXACT-PRO evidence dossier. She will also discuss current activities of the Initiative as they relate to the FDA’s instrument qualification review process.

Leidy discussed current sponsor challenges in the PRO space. “When the intent is to use a PRO as a primary or secondary efficacy endpoint, sponsors need to make certain that there is adequate and convincing evidence to support the selection and use of a given instrument for the intended purpose,” she explained. Do all instruments need to go through the Critical Path Institute to be qualified for a specific purpose? “There are many different paths sponsors can follow as they develop or select and validate PRO instruments for use in their development program,” Leidy explained, “including independent or collaborative efforts or through the Institute.”

Recently, Leidy attended a medical specialty conference where discussion of PROs was widespread. Physicians, nurses, academicians, clinical investigators, and methodologists were exchanging ideas, empirical results, and new insight into the effect of disease and treatment on PROs. “Everyone is talking about PROs now,” observed Leidy. “It’s part of the language. It’s part of the culture.” From a topic that began as part of a discussion between pharmaceutical companies (and only certain departments at that) and the FDA, PRO endpoints are emerging as an equal among clinical efficacy endpoints.

The best part about the PRO field as it continues to advance? According to Leidy, “It is definitely hearing the patient’s voice become louder and clearer.”

“PRO instruments quantify how patients feel or function, with the data coming directly from the patients themselves. They give us metrics with which we can better understand the process and expression of various health conditions and fully evaluate the effects of treatment. Ultimately, PRO information will help us more effectively assess the benefits and risks of health care interventions,” explained Leidy.

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