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EHA Evaluation of the ESMO-Magnitude of Clinical Benefit Scale Version 1.1 (ESMO-MCBS v1.1) for Haematological Malignancies

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EHA Evaluation of the ESMO-Magnitude of Clinical Benefit Scale Version 1.1 (ESMO-MCBS v1.1) for Haematological Malignancies

Barbara Kiesewetter et al. ESMO Open.

Abstract

Objective: Value frameworks in oncology have not been validated for the assessment of treatments in haematological malignancies, but to avoid overlaps and duplications it appears reasonable to build up experience on existing value frameworks, such as the European Society for Medical Oncology-Magnitude of Clinical Benefit Scale (ESMO-MCBS).

Methods: Here we present the results of the first feasibility testing of the ESMO-MCBS v1.1 for haematological malignancies based on the grading of 80 contemporary studies for acute leukaemia, chronic leukaemia, lymphoma, myeloma and myelodysplastic syndromes. The aims were (1) to evaluate the scorability of data, (2) to evaluate the reasonableness of the generated grades for clinical benefit using the current version and (3) to identify shortcomings in the ESMO-MCBS v1.1 that require amendments to improve the efficacy and validity of the scale in grading new treatments in the management of haematological malignancies.

Results: In general, the ESMO-MCBS v1.1 was found to be widely applicable to studies in haematological malignancies, generating scores that were judged as reasonable by European Hematology Association (EHA) experts. A small number of studies could either not be graded or were not appropriately graded. The reasons, related to the differences between haematological and solid tumour malignancies, are identified and described.

Conclusions: Based on the findings of this study, ESMO and EHA are committed to develop a version of the ESMO-MCBS that is validated for haematological malignancies. This development process will incorporate all of the usual stringencies for accountability of reasonableness that have characterised the development of the ESMO-MCBS including field testing, statistical modelling, evaluation for reasonableness and openness to appeal and revision. Applying such a scale will support future public policy decision-making regarding the value of new treatments for haematological malignancies and will provide insights that could be helpful in the design of future clinical trials.

Keywords: ESMO-MCBS; clinical benefit; haematologic malignancies; value frameworks.

Conflict of interest statement

Competing interests: BK has received honoraria for lectures from Celgene and Novartis; NB has received honoraria for consulting and advisory role from Ariad, Amgen, Novartis and Pfizer; EGEdV declares advisory role for Daiichi Sankyo, Merck, NSABP, Pfizer, Sanofi and Synthon, and institutional financial support for clinical trials or contracted research for Amgen, AstraZeneca, Bayer, Chugai, CytomX Therapeutics, G1 Therapeutics, Genentech, Nordic Nanovector, Radius Health, Roche and Synthon; PG has received honoraria for lectures/advisory boards and research funding from AbbVie/Pharmacyclics, Acerta/AstraZeneca, BeiGene, Janssen, Gilead, Novartis, Sunesis and Verastem; NG has received honoraria for lectures, advisory boards and research support from Amgen, Pfizer and Incyte; VG-C has received honoraria for lectures from Janssen and for participation in advisory boards from Prothena; BH has received honoraria for lectures from Novartis, Pfizer and BMS, and for participation in advisory boards from Novartis, GlaxoSmithKline, Abbvie, Pfizer, BMS and Incyte; UJ has declared honoraria from Abbvie, BMS, Celgene, Amgen, Roche, Novartis, Gilead, Bioverativ, Janssen, MSD, Sandoz, Takeda and Pfizer; M-VM has received honoraria for lectures from Janssen, Celgene, Takeda and Amgen, and for participation in advisory boards from Janssen, Celgene, Takeda, Amgen, AbbVie, GlaxoSmithKline and PharmaMar; MR has received honoraria for lectures from Novarits, Ipsen, Celgene and Eisai; LS has received honoraria for lectures from Janssen, AbbVie, AstraZeneca, and for participation in advisory boards from Janssen; PS has received honoraria for lectures and advisory boards from Celgene, Janssen, Takeda and Amgen, and research support from Celgene, Janssen and Amgen.

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