In a press briefing at the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition, Cynthia E. Dunbar, MD, ASH Secretary and Chief of the Translational Stem Cell Biology Branch, Intramural Research Program of the National Heart, Lung, and Blood Institute, offered her thoughts on the findings of this comparison of the efficacy in clinical trials vs the effectiveness in the real world of treatments for multiple myeloma. She noted a key aspect of the efficacy-effectiveness gap might be explained by the overall better health of clinical trial patients, but other factors are probably important.

Cynthia E. Dunbar, MD

“The clinical trial entry criteria—regarding kidney function, blood cell counts, and other things we use for cutoffs—are part of this, and they may not always apply in real-world patients,” Dr. Dunbar said. “Also, patients able to enroll in trials are more likely to show up for treatment at the right time for every dose. They also may be from higher socioeconomic classes and perhaps better able to afford drugs to prevent side effects. In clinical trials, nurses may be encouraging them to make it through the toxicity, and so they may stay on treatment longer.”

Dr. Dunbar continued: “Clinicians and patients should consider randomized clinical trial findings as the best possible outcome. And clinicians should adjust their thinking if an individual patient is older, sicker, or less able to follow the regimen exactly when deciding how to apply the research results to their own practice. The findings do show we need to try to improve access to health care, so patients in the real world are able to obtain high-quality care all the time—just like patients in clinical trials.”

DISCLOSURE: Dr. Dunbar has received honoraria from UpToDate and research funding from Disc Medicine, Be Biopharma, and Novartis.