Developing Personalized Clinical Outcome Assessments

The Food and Drug Administration (FDA) has taken steps over the last several years to encourage and facilitate the collection of robust and meaningful patient and caregiver input to inform medical product development and regulatory decision-making. An important focus of this effort has been on the development and use of clinical outcome assessments (COAs). One emerging challenge in the development and interpretation of COAs is the heterogeneity of signs, symptoms, and functional impairment that patients exhibit in numerous disease areas, including many rare diseases. A standardized COA applied uniformly to all patients may overlook symptoms that are important to some patients or include symptoms that are irrelevant to others, potentially rendering the assessment insensitive to meaningful treatment effects. A possible solution to these problems include employing a “personalized COA” approach, in which the COA may vary across patients in an effort to measure the most important and relevant signs, symptoms, functions, as well as the degree of severity of these concepts in each individual. While this approach shows promise, there are a number of outstanding questions relating to the development, implementation, and evaluation of personalized COAs.

To support further progress in this area, the Duke-Margolis Center for Health Policy convened this expert workshop in order to 1) explore and discuss methodologies and best practices surrounding personalized COAs, and 2) identify specific recommendations on methodologies to explore and advance the use of personalized COAs in medical product development.

Funding for this conference was made possible in part by a cooperative agreement with the FDA’s Center for Drug Evaluation and Research. The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services nor does mention of trade names, commercial practices, or organizations imply endorsements by the U.S. Government.