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EHA evaluation of the ESMO—Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS v1.1) for haematological malignancies

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—Magnitu...

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Autores principales: Kiesewetter, Barbara, Cherny, Nathan I, Boissel, Nicolas, Cerisoli, Francesco, Dafni, Urania, de Vries, Elisabeth G E, Ghia, Paolo, Gökbuget, Nicola, González-Calle, Verónica, Huntly, Brian, Jäger, Ulrich, Latino, Nicola Jane, Douillard, Jean-Yves, Malcovati, Luca, Mateos, María-Victoria, Ossenkoppele, Gert J, Porkka, Kimmo, Raderer, Markus, Ribera, Josep-Maria, Scarfò, Lydia, Wester, Ruth, Zygoura, Panagiota, Sonneveld, Pieter
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BMJ Publishing Group 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7003483/
https://www.ncbi.nlm.nih.gov/pubmed/31958292
http://dx.doi.org/10.1136/esmoopen-2019-000611
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author Kiesewetter, Barbara
Cherny, Nathan I
Boissel, Nicolas
Cerisoli, Francesco
Dafni, Urania
de Vries, Elisabeth G E
Ghia, Paolo
Gökbuget, Nicola
González-Calle, Verónica
Huntly, Brian
Jäger, Ulrich
Latino, Nicola Jane
Douillard, Jean-Yves
Malcovati, Luca
Mateos, María-Victoria
Ossenkoppele, Gert J
Porkka, Kimmo
Raderer, Markus
Ribera, Josep-Maria
Scarfò, Lydia
Wester, Ruth
Zygoura, Panagiota
Sonneveld, Pieter
author_facet Kiesewetter, Barbara
Cherny, Nathan I
Boissel, Nicolas
Cerisoli, Francesco
Dafni, Urania
de Vries, Elisabeth G E
Ghia, Paolo
Gökbuget, Nicola
González-Calle, Verónica
Huntly, Brian
Jäger, Ulrich
Latino, Nicola Jane
Douillard, Jean-Yves
Malcovati, Luca
Mateos, María-Victoria
Ossenkoppele, Gert J
Porkka, Kimmo
Raderer, Markus
Ribera, Josep-Maria
Scarfò, Lydia
Wester, Ruth
Zygoura, Panagiota
Sonneveld, Pieter
author_sort Kiesewetter, Barbara
collection PubMed
description 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.
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spelling pubmed-70034832020-02-25 EHA evaluation of the ESMO—Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS v1.1) for haematological malignancies Kiesewetter, Barbara Cherny, Nathan I Boissel, Nicolas Cerisoli, Francesco Dafni, Urania de Vries, Elisabeth G E Ghia, Paolo Gökbuget, Nicola González-Calle, Verónica Huntly, Brian Jäger, Ulrich Latino, Nicola Jane Douillard, Jean-Yves Malcovati, Luca Mateos, María-Victoria Ossenkoppele, Gert J Porkka, Kimmo Raderer, Markus Ribera, Josep-Maria Scarfò, Lydia Wester, Ruth Zygoura, Panagiota Sonneveld, Pieter ESMO Open Original Research 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. BMJ Publishing Group 2020-01-20 /pmc/articles/PMC7003483/ /pubmed/31958292 http://dx.doi.org/10.1136/esmoopen-2019-000611 Text en © 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/.
spellingShingle Original Research
Kiesewetter, Barbara
Cherny, Nathan I
Boissel, Nicolas
Cerisoli, Francesco
Dafni, Urania
de Vries, Elisabeth G E
Ghia, Paolo
Gökbuget, Nicola
González-Calle, Verónica
Huntly, Brian
Jäger, Ulrich
Latino, Nicola Jane
Douillard, Jean-Yves
Malcovati, Luca
Mateos, María-Victoria
Ossenkoppele, Gert J
Porkka, Kimmo
Raderer, Markus
Ribera, Josep-Maria
Scarfò, Lydia
Wester, Ruth
Zygoura, Panagiota
Sonneveld, Pieter
EHA evaluation of the ESMO—Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS v1.1) for haematological malignancies
title EHA evaluation of the ESMO—Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS v1.1) for haematological malignancies
title_full EHA evaluation of the ESMO—Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS v1.1) for haematological malignancies
title_fullStr EHA evaluation of the ESMO—Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS v1.1) for haematological malignancies
title_full_unstemmed EHA evaluation of the ESMO—Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS v1.1) for haematological malignancies
title_short EHA evaluation of the ESMO—Magnitude of Clinical Benefit Scale version 1.1 (ESMO-MCBS v1.1) for haematological malignancies
title_sort eha evaluation of the esmo—magnitude of clinical benefit scale version 1.1 (esmo-mcbs v1.1) for haematological malignancies
topic Original Research
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7003483/
https://www.ncbi.nlm.nih.gov/pubmed/31958292
http://dx.doi.org/10.1136/esmoopen-2019-000611
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