Clinical Outcome Assessments: Establishing and Interpreting Meaningful Within-Patient Change

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 of the key challenges in this process relates to the interpretation of meaningful-within patient change in the measure over time – specifically, how to establish the threshold of change in the measure that can be interpreted as being clinically meaningful, rather than just statistically significant.

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 meaningful within-patient change, and 2) identify specific recommendations on methodologies used to derive and interpret meaningful within-patient change with use of COA endpoints 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.