In recent years, rapid developments in haematology research resulted in a considerable expansion of treatment options. The development of instruments to measure clinical benefit is essential in the current scenario where increasing numbers of treatments for haematological malignancies (HMs) are becoming available, often targeting a small and defined subpopulation of patients. For this, several value frameworks have been published by different organisations and institutions taking into account or emphasising different aspects contributing to such an evaluation. These frameworks vary in terms of their definition of value, target audience and methodology, and each of them has specific limitations, which should be taken into consideration when interpreting their outputs. Until now, value frameworks developed in oncology have not been validated in the setting of HMs.

 

The European Society for Medical Oncology (ESMO) has developed such a value framework called the ESMO—Magnitude of Clinical Benefit Scale (ESMO-MCBS). Initially published in 2015, the scale is a validated and reproducible tool in solid tumour oncology with a particular focus on the clinical benefit. The ESMO-MCBS was developed to generate clear, valid and unbiased grading of the magnitude of clinical benefit demonstrated in therapeutic studies that could be used for a number of purposes including public health policy and health technology assessment (HTA), clinical decision-making, medical publication and journalism. The ESMO-MCBS grading highlights those treatments which substantially improve the duration of survival and/or the quality of life (QOL) of patients with cancer and aims to distinguish them from trials demonstrating more limited and sometimes even marginal benefits. The ESMO-MCBS was revised (version 1.1) in 2017, based on feedback and queries from clinicians, patients, researchers and representatives of the pharmaceutical industry, and a dynamic process of internal peer review. Version 1.1 incorporates 10 revisions and most importantly allows also for scoring of single-arm studies. The ESMO-MCBS assigns categorical benefit scores to European Medicines Agency (EMA) approved drugs, based on results from ‘positive’ randomised clinical trials: (1) superiority trials that have demonstrated a statistically significant result for the primary endpoint of the study, or secondary in case of overall survival (OS) and (2) non-inferiority trials, reaching a conclusion of non-inferiority. Primary or secondary endpoints included in the scoring system are OS, progression-free survival (PFS), QOL, treatment toxicity or response rates. In developing the ESMO-MCBS scale, ESMO aspired to meet standards for ‘accountability for reasonableness’, incorporating extensive field testing, statistical modelling and peer review of the ‘reasonableness’ of the generated results into the development process. The ESMO-MCBS is currently incorporated in ESMO’s clinical practice guidelines and is being used as part of HTA processes.