Cancer checkpoint inhibitors have received considerable and broad interest because of their ability to generate durable responses in many hitherto intractable malignant tumors^1,2 and for improvements in overall survival in several randomized trials. These promising drugs, and their underlying preclinical science, formed the basis of the 2018 Nobel Prize in Medicine.³

Immune checkpoint inhibitor (ICI) drugs have gained popularity in oncology because of their ability to boost a person’s immune response against cancer cells.¹ We recently estimated that 43.6% of US patients with cancer are eligible for ICI therapy, and up to 12.5% of patients respond to it.² However, those were best-case estimates, and postmarketing studies for several of these drugs have failed to show improvement in overall survival or progression-free survival.³ The US Food and Drug Administration (FDA) has revised some ICI drug labels, and future changes may follow.⁴ Accordingly, we sought to reestimate the eligibility of ICIs, taking into consideration recent FDA label changes and results of postmarketing studies.

The ability to identify medical reversals and other low-value medical practices is an essential prerequisite for efforts to reduce spending on such practices. Through an analysis of more than 3000 randomized controlled trials (RCTs) published in three leading medical journals (the Journal of the American Medical Association, the Lancet, and the New England Journal of Medicine), we have identified 396 medical reversals. Most of the studies (92%) were conducted on populations in high-income countries, cardiovascular disease was the most common medical category (20%), and medication was the most common type of intervention (33%).

Health-related quality of life (QoL) and other patient-reported outcomes are vital to assessing patient perspective and experience. They reflect patient satisfaction and perceived benefits of an intervention that are not necessarily captured by other end points. These outcomes are commonly used in clinical trials, and regulatory and reimbursement agencies have begun to require these data as part of their evaluation process.¹