Patient-reported outcomes essential to comparative effectiveness research

Patient-reported outcomes should be a standard part of evaluating the comparative effectiveness of cancer treatments, according to recommendations put forward by a multi-institution research group.

In an early release article published this week online in the Journal of Clinical Oncology, a research group led by Ethan Basch, MD, Director of the Cancer Outcomes Research Program at UNC Lineberger Comprehensive Cancer Center, recommends that patient-reported symptoms and health-related quality of life measures should be assessed in comparative effectiveness studies designed to generate new evidence about cancer treatment. The article was written on behalf of the Center for Medical Technology Policy in Baltimore, MD.

Comparative effectiveness research is designed to inform health-care decisions by providing evidence on the effectiveness, benefits, and harms of different treatment options. The evidence is generated from research studies that compare drugs, medical devices, tests, surgeries, or ways to deliver health care.

"Comparative effectiveness research looks at how treatment options perform in a real-world setting, and is particularly important in cancer treatment, where patients are not only fighting their disease but also enduring treatments that may have a significant impact on their ability to function and their quality of life," said Basch.

"There is no way to adequately assess a treatment's impact on the patient without including their point of view, gleaned directly from asking about their experience in a consistent and scientifically-validated manner. Without this information, stakeholders – including patients, physicians and nurses, payers, researchers and regulators – have incomplete information for decision making about a given treatment."

The group recommends evaluation of a set of twelve core symptoms in studies in advanced or metastatic cancers, use of standard, validated measurements for patient-reported outcomes, to identify specific symptoms that may be relevant in studies of treatment for particular forms of cancer, and methods to gather the relevant data efficiently and effectively.