Recommendations for Incorporating Patient-Reported Outcomes into Clinical Comparative Effectiveness Research in Adult Oncology (May 2012)

This EGD provides recommendations to set a minimum standard that will ensure that data collection allows for examination of patients’ directly reported experiences in diverse studies of treatment effectiveness and outcomes in oncology.

Since the 1990s, the assessment of patient-reported outcomes has become increasingly recognized as important for those treatment- and/or disease-related consequences that are directly experienced by patients such as symptoms and health-related quality of life (HRQOL).  The importance of incorporating these outcomes into cancer research and policy formation has been emphasized by major policy-making, standard setting, and regulatory entities including the National Cancer Institute, the American Cancer Society, the US Food and Drug Administration, the U.S. Centers for Medicare & Medicaid Services, and the National Institutes of Health.  Systematic collection of patient-reported outcomes in oncology reflects underlying health status more accurately than clinician reporting. Using patient-reported outcomes increases patient satisfaction with care, is valued by clinicians, and improves symptom management and patients’ overall health status.  Moreover, patient self-reporting is the regulatory standard for assessing clinical endpoints in drug approval and labeling, as described in the FDA PRO Guidance document titled, “Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims.”

CMTP obtained input from a stakeholders meeting in December 2010.  The EGD recommendations were put in their current form in April, 2011, and on May 24, 2011, the project team presented their recommendations at the ISPOR16th Annual International Meeting.