Why Reinvent the Wheel? Use or Modification of Existing Clinical Outcome Assessment Tools in Medical Product Development [Editor's Choice]


Assessment of clinical benefit in treatment trials can be made through report by a clinician, a patient, or a nonclinician observer (eg, caregiver) or through a performance-based assessment. The US Food and Drug Administration (FDA) published a final guidance for industry for one type of clinical outcome assessment (COA)—patient-reported outcome (PRO) measures—in 2009 that described how FDA reviews PRO measures for their adequacy to support medical product–labeling claims. Many of the principles described in the PRO Guidance could be applicable to the other types of COAs, including instruments completed by clinicians (ie, clinician-reported outcome assessments) and nonclinician observers (ie, observer-reported outcome assessments). FDA guidance describing the regulatory expectations for all COA types including performance outcome assessments, which are based on the patient’s performance of a defined task or activity, is in progress to meet requirements described within the 21st Century Cures Act and PDUFA VI. This communication highlights potential ways in which existing instruments might be modified or used “as is” to conform to good measurement principles. An industry and a regulatory perspective are described.


Elektra J. Papadopoulos Elizabeth Nicole Bush Sonya Eremenco Stephen Joel Coons

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