Issues That Arise During the FDA Review of Applications Containing Patient and Clinician Reported Outcomes

• Activity Number: 531
• Type: Topic Contributed
• Date/Time: Wednesday, August 12, 2015 : 10:30 AM to 12:20 PM
• Sponsor: Health Policy Statistics Section
• Author(s): Laura Lee Johnson* and Scott Komo* and Thomas Birkner* and Wen-Hung Chen* and Mahboob Sobhan*
• Companies: FDA and FDA/CDER/OTS/OB/DBIV and FDA/CDER/OTS/OB/DBI and FDA/CDER/OND/SEALD and FDA
• Keywords: Patient reported outcomes (PRO) ; Clinical outcome assessment (COA) ; Personalized medicine ; Missing data ; Data standards

Abstract

Increasingly, clinical outcomes assessments (COAs) including patient reported outcomes and clinician reported outcomes (PROs and ClinROs) are included as endpoints in product applications submitted to the Food and Drug Administration and other regulatory agencies worldwide. Such endpoints also are used in clinical care settings, as part of risk evaluation and mitigation strategies (REMS) for some medical products, and in quality improvement initiatives. Regulators and clinicians on the panel will discuss topics from an April 2015 FDA public meeting and COA guidance documents. We will share experiences and discuss common COA development, data management, and analysis problems of interest to statisticians analyzing data from COAs, data managers, and PRO developers. Other topics include evolving standards to develop and validate a PRO instrument, issues that arise in clinical trial data such as missing data for items or domains, and methods to validate minimally important differences and responder definitions. Identification, characterization and mitigation of issues are key to providing the quality of data necessary to include the patient perspective in medical product labels.