Abstract
Background:
The European Society for Medical Oncology (ESMO)-Magnitude of Clinical Benefit Scale (MCBS) has been accepted as a robust tool to evaluate the magnitude of clinical benefit reported in trials for oncological therapies. However, the ESMO-MCBS hitherto has only been validated for solid tumours. With the rapid development of novel therapies for haematological malignancies, we aimed to develop an ESMO-MCBS version that is specifically designed and validated for haematological malignancies.
Methods:
ESMO and the European Hematology Association (EHA) initiated a collaboration to develop a version for haematological malignancies (ESMO-MCBS:H). The process incorporated five landmarks: field testing of the ESMO-MCBS version 1.1 (v1.1) to identify shortcomings specific to haematological diseases, drafting of the ESMO-MCBS:H forms, peer review and revision of the draft based on re-scoring (resulting in a second draft), assessment of reasonableness of the scores generated, final review and approval by ESMO and EHA including executive boards.
Results:
Based on the field testing results of 80 haematological trials and extensive review for feasibility and reasonableness, five amendments to ESMO-MCBS were incorporated in the ESMO-MCBS:H addressing the identified shortcomings. These concerned mainly clinical trial endpoints that differ in haematology versus solid oncology and the very indolent nature of nevertheless incurable diseases such as follicular lymphoma, which hampers presentation of mature data. In addition, general changes incorporated in the draft version of the ESMO-MCBS v2 were included, and specific forms for haematological malignancies generated. Here we present the final approved forms of the ESMO-MCBS:H, including instructions.
Conclusion:
The haematology-specific version ESMO-MCBS:H allows now full applicability of the scale for evaluating the magnitude of clinical benefit derived from clinical studies in haematological malignancies.
| Original language | English |
|---|---|
| Pages (from-to) | 734-771 |
| Number of pages | 38 |
| Journal | Annals of Oncology |
| Volume | 34 |
| Issue number | 9 |
| Early online date | 19 Jun 2023 |
| DOIs | |
| Publication status | Published - Sept 2023 |
Bibliographical note
Funding Information:The authors acknowledge the support and contribution of the ESMO Executive Board, the EHA Executive Board, the ESMO Faculty, members of the ESMO Guidelines Committee and the logistics and organizational support provided by EHA Office. Supplementary Appendix S2, available athttps://doi.org/10.1016/j.annonc.2023.06.002, lists the haematology colleagues who participated in the field testing of ESMO-MCBS:H. None declared. BK declares honoraria for lectures or advisory boards from AAA, Boehringer Ingelheim, Eli Lilly, IPSEN, Janssen-Cilag, MSD, Novartis, Roche, Eli Lilly (all outside of the submitted work).UD declares institutional financial support from ESMO for biostatistical contribution; reports honorarium as member of the Tumour Agnostic Evidence Generation Working Group, Roche. EGEdV declares institutional financial support for clinical trials or contracted research from Amgen, Genentech, Roche, CytomX, G1 Therapeutics, Bayer, Synthon, Servier, Regeneron, Crescendo Biologics and AstraZeneca; institutional financial support for advisory boards and consultancy from National Surgical Adjuvant Breast and Bowel Project, Daiichi Sankyo and Crescendo Biologics. JB declares grants, personal fees and non-financial support from Ipsen, personal fees and non-financial support from Pfizer, non-financial support from AAA, personal fees and non-financial support from Novartis, non-financial support from Nanostring, non-financial support from Roche, non-financial support from Rand, grants and personal fees from Servier and personal fees from Nutricia outside the submitted work. GC has received personal fees from AstraZeneca and Daiichi Sankyo; has received grants or contracts from Merck and AstraZeneca; has received personal fees as advisory board member for AstraZeneca, BMS; Ellipsis, Merck; Lilly, Pfizer, Roche, Veracyte; has received institutional research grant from Merck; has received institutional funding for phase I studies from Astellas, AstraZeneca, BMS, Blueprint Medicine, Daiichi Sankyo, Kymab, Novartis, Philogen, Roche, Sanofi; has received consulting fees from Roche, BMS, Novartis, Lilly, Pfizer, Seagen, AstraZeneca, Ellipsis, Gilead, Merck, Celcuity, Sanofi, Exact Sciences and Daiichi Sankyo; has received payment or honoraria for presentations, lectures, speaker bureaus, manuscript writing or educational events from AstraZeneca, Novartis, Lilly, Pfizer, Seagen, Roche and Daiichi Sankyo; has received support for attending meetings or travel from AstraZeneca and Daiichi Sankyo; has participated on a data safety monitoring board or advisory board for Roche, BMS, Novartis, Lilly, Pfizer, Seagen, Ellipsis, Gilead, Merck, Celcuity, AstraZeneca and Daiichi Sankyo; has an advisory role and is Member of the Scientific Council of Europa Donna, is an Officer of the Consiglio Superiore di Sanità, Italian National Health Council as Advisor for Ministry of Health and has an advisory role in Fondazione Beretta Cancer Research Foundation. BG reports receiving consulting fees from Vivio Health. BJPH reports receiving research grants from AstraZeneca and Sysmex, funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement Nos. 116026 and 945406, consultancy fees from Janpix Ltd and being a member of the board of the European Hematology Association. UJ reports honoraria for advisory roles from Abbvie, Amgen, BMS/Celgene, Gilead, Janssen, Merck, Miltenyi Novartis, Sanofi and Roche. He has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No. 945393. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation program and EFPIA. SFO reports research grants from Novartis and Celldex Therapeutics, speakers fee from Merck and consultancy fees from BMS, Genmab and Merck (paid to the institution). GO declares research support from Celgene, honoraria for consultancy from Celgene and Pfizer and honoraria for advisory boards from Pfizer, BMS, Celgene, AGIOS, Gilead, Astellas, JAZZ Pharmaceuticals, Servier, AbbVie. MP reports being a Scientific Board Member at Oncolytics, consultancy fees from AstraZeneca, Camel-IDS, Immunomedics, Lilly, Menarini, MSD, Novartis, Odonate, Pfizer, Roche Genentech, Seattle Genetics, Immutep, Seagen. Therapeutics, Frame Therapeutics and research grants from AstraZeneca, Immunomedics, Lilly, Menarini, MSD, Novartis, Pfizer, Radius, Roche-Genentech, Servier, Synthon (paid to the institution). MR received honoraria for lectures or advisory board participation from the following for-profit companies (all outside of the submitted work): Celgene/BMS, Ipsen, Novartis, Roche, Eisai, Eli Lilly. LS declares honoraria for advisory boards from AbbVie, AstraZeneca, BeiGene, Janssen, Lilly, honoraria for lectures from Octapharma and travel grants from BeiGene, Janssen. DT reports being a member of the EMA Healthcare Professional Working Party (HCPWP), a member of the WHO SAGE IVD and a member of the WHO EML Cancer Medicines Working Group. CCZ reports licencing fees from Imugene; being a member of Board of Directors of the Central European Cooperative Oncology Group (CECOG), member of the Board of Directors Immunotherapy of Cancer (ITOC). RW reports honoraria from Sanofi and Janssen. EM reports being a member of the European Hematology Association and its president from 2021 to 2023. All other authors have declared no conflicts of interest.
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