ESMO-Magnitude of Clinical Benefit Scale version 1.1

N I Cherny; U Dafni; J Bogaerts; N J Latino; G Pentheroudakis; J-Y Douillard; J Tabernero; C Zielinski; M J Piccart; E G E de Vries

doi:10.1093/annonc/mdx310

ESMO-Magnitude of Clinical Benefit Scale version 1.1

Ann Oncol. 2017 Oct 1;28(10):2340-2366. doi: 10.1093/annonc/mdx310.

Authors

N I Cherny¹, U Dafni², J Bogaerts³, N J Latino⁴, G Pentheroudakis⁵, J-Y Douillard⁴, J Tabernero⁶, C Zielinski⁷, M J Piccart⁸, E G E de Vries⁹

Affiliations

¹ Cancer Pain and Palliative Medicine Service, Department of Medical Oncology, Shaare Zedek Medical Center, Jerusalem, Israel;. Electronic address: mcbs@esmo.org.
² Laboratory of Biostatistics, School of Health Sciences, National and Kapodistrian University of Athens & Frontier Science Foundation-Hellas, Athens, Greece.
³ Methodology Direction, EORTC Headquarters, Brussels, Belgium.
⁴ ESMO Head Office, Lugano, Switzerland.
⁵ Medical Oncology Department, Ioannina University Hospital, Ioannina, Greece.
⁶ Medical Oncology Department, Vall d'Hebron University Hospital, Barcelona, Spain.
⁷ Division of Oncology, Medical University Vienna, Vienna, Austria.
⁸ Jules Bordet Institute, Universite Libre de Bruxelles, Brussels, Belgium.
⁹ Department of Medical Oncology, University Medical Center Groningen, University of Groningen, Groningen, The Netherlands.

PMID: 28945867
DOI: 10.1093/annonc/mdx310

Abstract

Background: The ESMO Magnitude of Clinical Benefit Scale (ESMO-MCBS) version 1.0 (v1.0) was published in May 2015 and was the first version of a validated and reproducible tool to assess the magnitude of clinical benefit from new cancer therapies. The ESMO-MCBS was designed to be a dynamic tool with planned revisions and updates based upon recognition of expanding needs and shortcomings identified since the last review.

Methods: The revision process for the ESMO-MCBS incorporates a nine-step process: Careful review of critiques and suggestions, and identification of problems in the application of v1.0; Identification of shortcomings for revision in the upcoming version; Proposal and evaluation of solutions to address identified shortcomings; Field testing of solutions; Preparation of a near-final revised version for peer review for reasonableness by members of the ESMO Faculty and Guidelines Committee; Amendments based on peer review for reasonableness; Near-final review by members of the ESMO-MCBS Working Group and the ESMO Executive Board; Final amendments; Final review and approval by members of the ESMO-MCBS Working Group and the ESMO Executive Board.

Results: Twelve issues for revision or amendment were proposed for consideration; proposed amendments were formulated for eight identified shortcomings. The proposed amendments are classified as either structural, technical, immunotherapy triggered or nuanced. All amendments were field tested in a wide range of studies comparing scores generated with ESMO-MCBS v1.0 and version 1.1 (v1.1).

Conclusions: ESMO-MCBS v1.1 incorporates 10 revisions and will allow for scoring of single-arm studies. Scoring remains very stable; revisions in v1.1 alter the scores of only 12 out of 118 comparative studies and facilitate scoring for single-arm studies.

Keywords: European Society for Medical Oncology; biostatistics; magnitude of clinical benefit scale; single arm studies.

MeSH terms

Biostatistics
Clinical Trials as Topic / methods*
Clinical Trials as Topic / standards
Humans
Medical Oncology / methods
Medical Oncology / standards
Neoplasms / therapy*
Outcome Assessment, Health Care / methods
Outcome Assessment, Health Care / standards
Randomized Controlled Trials as Topic / standards