TY - JOUR
T1 - EHA evaluation of the ESMO-Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS v1.1) for haematological malignancies
AU - Kiesewetter, Barbara
AU - Cherny, Nathan I.
AU - Boissel, Nicolas
AU - Cerisoli, Francesco
AU - Dafni, Urania
AU - de Vries, Elisabeth G. E.
AU - Ghia, Paolo
AU - Gökbuget, Nicola
AU - González-Calle, Verónica
AU - Huntly, Brian
AU - Jäger, Ulrich
AU - Latino, Nicola Jane
AU - Douillard, Jean-Yves
AU - Malcovati, Luca
AU - Mateos, María-Victoria
AU - Ossenkoppele, Gert J.
AU - Porkka, Kimmo
AU - Raderer, Markus
AU - Ribera, Josep-Maria
AU - Scarfò, Lydia
AU - Wester, Ruth
AU - Zygoura, Panagiota
AU - Sonneveld, Pieter
N1 - Publisher Copyright: © Author (s) (or their employer(s)) 2020. Re-use permitted under CC BY-NC. No commercial re-use. Published by BMJ on behalf of the European Society for Medical Oncology. Copyright: Copyright 2020 Elsevier B.V., All rights reserved.
PY - 2020/1/20
Y1 - 2020/1/20
N2 - 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.
AB - 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.
KW - ESMO-MCBS
KW - clinical benefit
KW - haematologic malignancies
KW - value frameworks
UR - http://www.scopus.com/inward/record.url?scp=85078326618&partnerID=8YFLogxK
U2 - https://doi.org/10.1136/esmoopen-2019-000611
DO - https://doi.org/10.1136/esmoopen-2019-000611
M3 - Article
C2 - 31958292
SN - 2059-7029
VL - 5
JO - ESMO open
JF - ESMO open
IS - 1
M1 - e000611
ER -