Work

Opioid Use After Open Resection or Video-Assisted Thoracoscopic Surgery for Early-Stage Lung Cancer

Year: 2018

Type: article

Abstract: small cell lung cancer, both scoring 4 on the ESMO-MCBS scale.Of the remaining single-arm trials, 8 (29.6%) scored 3, 8 (29.6%) scored 2, 5 (18.5%) scored 1 and 4 trials (14.8%) scored 0.Discussion | The observation of common and durable response to targeted therapy and immunotherapy has raised questions about whether traditional randomized trials are necessary to support drug approval.6 The ability to study a drug's ORR, especially in clearly defined target populations, makes the singlearm study design appealing as a pathway to approval.The ability to identify target populations has been aided by increasing understanding of the molecular basis of cancer, which has allowed anatomically defined cancers to be subdivided into smaller, molecularly defined subtypes more likely to respond to targeted therapies.Our data confirm that ORR is a common end point in single-arm studies supporting accelerated approval of anticancer therapies.However, a small proportion of single-arm studies met the threshold for substantial clinical benefit defined by ESMO-MCBS, version 1.1.These results are unsurprising because, to meet substantial benefit thresholds using the ESMO-MCBS, a single-arm trial must show substantial efficacy as well as improvement in quality of life or have data from confirmatory postmarketing study.The low proportion of single-arm trials that meet the threshold for substantial benefit may reflect a lack of supporting data or unrealistic thresholds in the ESMO framework.In conclusion, approximately one-third of trials supporting FDA drug approval meet the threshold for substantial clinical benefit as measured by ESMO-MCBS.Compared with RCTs, approvals supported by single-arm studies are less likely to show substantial benefit using ESMO-MCBS.It is likely that this reflects the high thresholds for substantial benefit used to calibrate the ESMO-MCBS for single-arm trials.

Source: JAMA Oncology

DOI: 10.1001/jamaoncol.2018.4387