TY  - JOUR
A1  - Kiesewetter, Barbara
A1  - Cherny, Nathan I.
A1  - Boissel, Nicolas
A1  - Cerisoli, Francesco
A1  - Dafni, Urania
A1  - Vries, Elisabeth G. E. de
A1  - Ghia, Paolo
A1  - Gökbuget, Nicola
A1  - González-Calle, Verónica
A1  - Huntly, Brian
A1  - Jäger, Ulrich
A1  - Latino, Nicola Jane
A1  - Douillard, Jean-Yves
A1  - Malcovati, Luca
A1  - Mateos, Maria-Victoria
A1  - Ossenkoppele, Gert J.
A1  - Porkka, Kimmo
A1  - Raderer, Markus
A1  - Ribera, Josep-Maria
A1  - Scarfò, Lydia
A1  - Wester, Ruth
A1  - Zygoura, Panagiota
A1  - Sonneveld, Pieter
T1  - EHA evaluation of the ESMO-magnitude of clinical benefit scale version 1.1 (ESMO-MCBS v1.1) for haematological malignancies
T2  - ESMO open
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.
KW  - ESMO-MCBS
KW  - clinical benefit
KW  - haematologic malignancies
KW  - value frameworks
Y1  - 2020
UR  - http://publikationen.ub.uni-frankfurt.de/frontdoor/index/index/docId/52971
UR  - https://nbn-resolving.org/urn:nbn:de:hebis:30:3-529716
SN  - 2059-7029
N1  - Copyright information: © 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. http://creativecommons.org/licenses/by-nc/4.0/
This is an open access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited, any changes made are indicated, and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/.
VL  - 5
IS  - 1, e000611
SP  - 1
EP  - 18
PB  - BMJ
CY  - London
ER  -