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CMS panel expresses confidence in patient-reported outcomes tools
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A CMS panel today voted with intermediate to high confidence that four tools used to measure patient-reported outcomes were valid and generalizable to the Medicare cancer population.
The four tools include Patient-Reported Outcomes-Common Terminology Criteria for Adverse Events (PRO-CTCAE), MD Anderson Symptom Inventory (MDASI), the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30), and the Patient-Reported Outcome Measurement Information System (PROMIS).
The Medicare Evidence Development & Coverage Advisory Committee (MEDCAC) conducted a daylong hearing during which several clinicians and pharmaceutical representatives testified about the role of patient-reported outcomes (PROs) in clinical trials and the challenges in measuring them.
“This meeting [was] the science of two emerging fields coming together: the science of PROs and the science of cell-based therapy,” said acting committee chair Joseph Ross, MD, MHS, associate professor of medicine and public health at Yale University School of Medicine. “Both are now coming to the cusp of actual clinical practice, which is exciting for us. As a general internist and a geriatrician, [I believe] we have to figure out how can we best generate evidence and inform decisions not just in very specialized treatment centers, but also broadly.”
This MEDCAC panel is part of the CMS national coverage determination for chimeric antigen receptor (CAR) T-cell therapy, which was opened in May. However, the panelists voted on the validity of PRO assessment tools in the context of Medicare patients undergoing cancer treatment including, but not limited to, CAR T-cell therapy.
MEDCAC panelists voted with a score of 1 through 5 — where 1 represented low confidence and 5 high confidence — on whether seven PRO assessment tests were valid and generalizable to the Medicare population.
Panelists voted with intermediate to high confidence that PRO-CTACAE, MDASI, EORTC-QLQ-C30 and PROMIS fit these criteria.
Panelists expressed low to intermediate confidence in the University of Washington Quality of Life, Electronic Self-Report—Cancer and the Functional Living Index—Cancer tools, mainly due to limited use and lack of data supporting superiority to more established PRO assessments.
“In our opinion, [the PRO-CTACAE, MDASI, EORTC-QLQ-C30 and PROMIS] are the tools that are well established, well tested and perform well,” said presenter Ethan Basch, MD, director of cancer outcomes research program and professor of health policy and management and hematology/oncology at UNC Lineberger Comprehensive Cancer Center and University of North Carolina at Chapel Hill. “If I were designing a trial, with some caveats, these are tools that I would be comfortable using.”
The panelists agreed that the four tools in which they expressed at least intermediate confidence included a breadth of measures in emotional, social and physical well-being; were easily applicable to clinical study; and were transferable to community practice settings.
Panelists largely agreed that PRO assessments at fixed time-dependent intervals (ie, weekly, monthly or yearly) were more appropriate than variable event-dependent intervals (ie, upon admission and after discharge).
Due to a lack of data, the panelists did not come to a consensus about whether a meaningful durable treatment effect could be determined with a PRO tool at 6 months, 12 months or 24 months.
When asked about their confidence in using various control populations for PRO assessments, the panelist expressed intermediate to high confidence in using baseline quality-of-life outcomes from patients undergoing usual care as control. However, panelists expressed less confidence in using historical data as control, largely because these modern tools may not be comparable to historical measures.
“Only a few of the PROs — like PROMIS — are able to be cross-walked to other historical [measures], like EQ5D,” committee member Joseph S. Cheng, MD, MS, FACS, FAANS, Frank H. Mayfield professor and chair in the department of neurosurgery at the University of Cincinnati College of Medicine, said. “I voted down because some of the ones we chose would not be able to be cross-walked.”
During the open discussion portion of the MEDCAC hearing, several panelists applauded CMS’s effort to evaluate PRO assessment tools, while highlighting the need for further discussion and a continuous improvement on the tools.
“The patient perspective is really critical and it’s valuable that CMS is actually asking these questions and addressing this issue,” committee member Thomas F. Goss, PharmD, senior vice president of Boston Health Associates, said during the hearing. “The field is in motion and it’s evolving, and I think there is valuable information here that will guide decisions made by patients and decisions made by payers. We’d be well-served to remember that when we don’t have complete clinical information, PRO data can at least provide good color and give guidance.” – by Cassie Homer
Disclosures: Ross reports institutional funding from Blue Cross Blue Shield Association, Johnson & Johnson and Medtronic. Basch reports research funding from the NCI and the Patient-Centered Outcomes Research Institute, and his research group helped develop PRO-CTCAE. Cheng and Goss report no relevant financial disclosures.