QoL data lacking for oncologic therapies when given regulatory approval
medwireNews: A large proportion of systemic oncology therapies lack evidence of quality of life (QoL) improvements at the time of regulatory approval by the US FDA and EMA, study findings indicate.
Furthermore, among the treatments with evidence of statistically significant improvements in QoL, “few have demonstrated clinically meaningful improvements,” despite the importance of QoL benefits to patients, report Kelvin Chan (Sunnybrook Health Sciences Centre, Toronto, Ontario, Canada) and co-authors in JAMA Network Open.
Chan and team systematically reviewed supporting clinical trial data for 214 FDA and 170 EMA oncology drug indications (36% and 31% hematologic indications, respectively) that were approved between January 2006 and December 2017.
They found that, at the time of regulatory approval, 14% of FDA indications and 26% of EMA indications had published QoL data. These proportions increased to 40% and 58%, respectively, when postapproval QoL publications (to October 2019) were also considered.
The researchers measured QoL benefits using the ASCO Value Framework (ASCO-VF) version 2.0 and the ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) version 1.1 QoL bonus criteria.
At the time of approval, 6% of FDA indications and 9% of EMA indications met the ASCO-VF QoL bonus criteria, and 7% and 11%, respectively, met the ESMO-MCBS criteria, which excludes hematologic malignancies.
When all published data were analyzed, the proportions of FDA- and EMA-approved indications showing clinical benefit according to ASCO-VF standards were 13% and 21%, respectively, while a respective 18% and 25% showed benefit according to ESMO-MCBS standards.
However, the investigators found that although some studies showed QoL benefits, just 3% of FDA indications and 6% of EMA indications demonstrated clinically meaningful improvements in QoL at the time of approval, with the proportions increasing to 6% and 11% when all data were considered.
Chan et al say: “Given the high value patients place on QOL end points, the scarcity of available and favorable QOL evidence for approved systemic oncology therapies should be recognized.
“This is especially important in the context of systemic therapy with palliative intent, as the goals of therapy may evolve over the course of disease to prioritize QOL over quantity of life.”
They suggest that randomized clinical trials “should be encouraged to not only improve collection and reporting of QOL evidence, but to do so in a manner that provides informative evidence of meaningful clinical benefit, to aid in regulatory approval and clinical decision-making that best supports patient-centered care.”
The authors also believe “regulatory agencies should seek to enhance consideration of QOL evidence as a more considerable constituent of their approval processes and requirements, to reflect evolving patient priorities.”
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