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

Abstract

ObjectiveValue 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).MethodsHere 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.ResultsIn 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.ConclusionsBased 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.

Adolescents and young adults (AYA) with cancer: a position paper from the AYA Working Group of the European Society for Medical Oncology (ESMO) and the European Society for Paediatric Oncology (SIOPE)

“…It will be important over time to identify internationally valid means to capture the value using system performance indicators in AYA services. 58,59 Access to clinical trials…”

Skip pattern approach toward the early access of innovative anticancer drugs

“…To this end, the AIFA assessments of UMN could be integrated with the AB assessment using a validated ESMO-MCBS version developed for hematological malignancies, such as that recently suggested by ESMO and the European Hematology Association. 6 …”

“…20 In Italy, as in other European countries, different regulatory tools for EA to innovative drugs are available, even though a comprehensive evaluation process of the new drug which takes into consideration the added value (compared with available options) and the place in therapy in different settings is still lacking. A French experience 6 reported the absence of correlation between the price of new cancer drugs and their added therapeutic benefit, as assessed by the French High Authority of Health Scale and the ESMO-MCBS for drugs introduced in France between 2004 and 2017.…”

“…As regards the UMN, the criteria adopted by AIFA were ESMO Open found to be adequate. On the other hand, as far as the AB is concerned, the use of the ESMO-MCBS v1.1 scale 5,6 was considered not applicable in most indications, because it does not take into account the specific and particular aspects of hematological diseases. Moreover, as regards the assessment of the QE, the low number of randomized clinical trials (RCTs) strongly affects the evaluation of quality of evidence.…”

“…This scale was applied both in curative and non-curative settings of most solid tumors and drugs with higher scores were considered for inclusion in ESMO guidelines. 4 As regards the hematological area, a recent publication by Kiesewetter et al 6 describes a collaborative project between ESMO and the European Society of Hematology for the validation of ESMO-MCBS criteria in hematological malignancies. In 2017, AIFA identified the criteria for classification of innovative drugs focusing on therapeutic need, added therapeutic value and quality of evidence.…”