ESMO-Magnitude of Clinical Benefit Scale for haematological malignancies (ESMO-MCBS:H) version 1.0

Kiesewetter, Barbara; Dafni, Urania; Vries, Elisabeth G.E. de; Barriuso, Jorge; Curigliano, Giuseppe; González‐Calle, Verónica; Galotti, M.; Gyawali, Bishal; Huntly, Brian J. P.; Jäger, Ulrich; Latino, Nicola Jane; Malcovati, Luca; Oosting, Sjoukje F.; Ossenkoppele, Gert J.; Piccart, Martine; Raderer, Markus; Scarfò, Lydia; Trapani, Dario; Zielinski, Christoph; Wester, Ruth; Zygoura, Panagiota; Macintyre, Elizabeth; Tabernero, Josep; Amaral, Teresa; Calles, Antonio; Gomez‐Roca, Carlos; Roitberg, Felipe; Llavero, N. Tarazona; Passaro, Antonio; Zarkavelis, George

doi:10.1016/j.annonc.2023.06.002

Cited by 10 publications

(6 citation statements)

References 41 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Furthermore, future research might repeat this study with the inclusion of haematological malignancies given the recently released ESMO-MCBS for this population. 22 Our results are aligned with previous Canadian research. Lexchin found that 75% of clinicians who disagreed with a negative funding recommendation declared a conflict with the manufacturer under review.…”

Section: Discussionsupporting

confidence: 91%

Characteristics of clinician input in Canadian funding decisions for cancer drugs: a cross-sectional study based on CADTH reimbursement recommendations

Jenei,

Meyers

2023

BMJ Open

View full text Add to dashboard Cite

ObjectiveTo examine characteristics of clinician input to the pan-Canadian Oncology Drug Review (pCODR) for cancer drug funding recommendations from 2016 to 2020.Design, setting and participantsDescriptive, cross-sectional study including 62 reimbursement decisions from pCODR from 2016 to 2020.InterventionspCODR recommendations were analysed for the number of clinicians consulted on each submission, affiliation, number of submissions per clinician, declared financial conflicts of interest (FCOIs), randomisation, type of blinding, primary endpoint, study phase, and whether the study demonstrated improvement in overall survival (OS) and progression-free survival (PFS).Main outcome measuresThe main outcome was clinician support for the initial funding recommendation. Secondary outcome measures were the association between clinician FCOIs and clinical benefit in positive recommendations.ResultsThe study consisted of 62 submissions, in which 48 included clinician input. A total of 129 unique clinicians provided 342 consultations. The majority (59%) provided input on less than 5 submissions; however, a small proportion (4%) consulted on over 10. Nearly all clinicians were physicians (125; 96%). From the 342 consultations, 228 declared financial conflicts (67%). The most common conflicts were payments for advisory roles (51%) and honorariums (23%). Of the 48 cancer drugs under review, clinicians recommended funding 46 (96%). Only 12 (25%) demonstrated substantial benefit, according to the European Society for Medical Oncology Magnitude of Clinical Benefit Scale score. Drugs recommended for funding were more likely to have improved PFS and OS data. However, most cancer drugs supported by clinicians demonstrated no change in health-related quality of life (HRQoL), including one that demonstrated worsened HRQoL. There was no statistically significant difference between FCOI status and recommending drugs with health gains.ConclusionClinicians offer crucial information on funding decisions. However, we found clinicians strongly supported funding nearly all cancer drugs under review, despite most not offering substantial benefit to patients nor gains in quality of life. While these drugs might be helpful options in clinical practice, funding numerous cancer drugs may be unsustainable for public health systems.

show abstract

Section: Discussionsupporting

confidence: 91%

Characteristics of clinician input in Canadian funding decisions for cancer drugs: a cross-sectional study based on CADTH reimbursement recommendations

Jenei,

Meyers

2023

BMJ Open

View full text Add to dashboard Cite

show abstract

“…The paradigm shift towards targeted medicine leads to specific challenges, which, for example, also affect many oncological and advanced therapeutic medicinal products (ATMPs). These will be the initial focus of the EU assessments from 2025, and adequate evidence assessments (e.g., as represented in ESMO’s Magnitude of Clinical Benefit Scale) are needed [ 26 , 27 , 28 , 29 ]. It has been argued that [ 30 ] medical societies’ representatives are the key connecting elements across the three pillars of market access processes that rely on clinical guidelines reflecting evidence-based medicine, necessary for benefit/risk (i.e., regulatory) and HTA assessment.…”

Section: Discussionmentioning

confidence: 99%

An Inclusive Civil Society Dialogue for Successful Implementation of the EU HTA Regulation: Call to Action to Ensure Appropriate Involvement of Stakeholders and Collaborators

Desmet,

Julian,

Van Dyck

et al. 2024

JMAHP

View full text Add to dashboard Cite

Objectives: Stakeholder involvement has long been considered a success factor for a joint European health technology assessment (HTA) process, and its relevance is now anchored in the EU HTA Regulation’s (EU HTAR) legislative wording. Therefore, we aimed to explore the roles, challenges, and most important activities to increase the level of involvement per stakeholder group. Methods: At the 2022 Fall Convention of the European Access Academy (EAA), working groups addressed the involvement of patients, clinicians, regulators, health technology developers (HTD), and national HTA bodies and payers within the EU HTA process. Each working group revisited the pre-convention survey results, determined key role characteristics for each stakeholder, and agreed on the most important activities to fulfill the role profile. Finally, the activities suggested per group were prioritized by plenary group. Results: The prioritized actions for patients included training and capacity building, the establishment of a patient involvement committee, and the establishment of a patient unit at the EC secretariat. For clinicians, it included alignment on evidence assessment from a clinical vs. HTA point of view, capacity building, and standardization of processes. The most important actions for regulators are to develop joint regulatory-HTA guidance documents, align processes and interfaces under the regulation, and share discussions on post-licensing evidence generation. HTDs prioritized scientific advice capacity and the review of the scoping process, and further development of the scope of the assessment report fact checks. The top three actions for national HTA bodies and payers included clarification on the early HTD dialogue process, political support and commitment, and clarification on financial support. Conclusions: Addressing the activities identified as the most important for stakeholders/collaborators in the EU HTA process (e.g., in the implementation of the EU HTA Stakeholder Network and of the guidance documents developed by the EUnetHTA 21 consortium) will be key to starting an “inclusive civil society dialogue”, as suggested by the European Commission’s Pharmaceutical Strategy.

show abstract

“…We will further access the clinical benefits of ADCs for haematological malignancies according to the currently published ESMO-MCBS:H V.1.0. 45 Another main limitation is publication selection bias. However, we implemented a rigorous search strategy to mitigate it.…”

Section: Discussionmentioning

confidence: 99%

Application of the ESMO Magnitude of Clinical Benefit Scale to assess the clinical benefit of antibody drug conjugates in solid cancer: a systematic descriptive analysis of phase III and pivotal phase II trials

Ding,

Yuan,

Wang

et al. 2024

BMJ Open

View full text Add to dashboard Cite

ObjectiveThe aim of this study was to assess the clinical benefit value of approved antibody drug conjugates (ADCs) for solid tumours using the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) V.1.1.DesignSystematic descriptive analysis.Data sourcesPubMed was searched for publications from 1 January 2000 to 18 October 2023.Eligibility criteriaWe included the phase III randomised controlled trials or phase II pivotal trials leading to approval of ADCs in solid tumours.Data extraction and synthesisTwo independent reviewers extracted data and discrepancies were resolved by consensus in the presence of a third investigator.ResultsESMO-MCBS Scores were calculated for 16 positive clinical trials of eight ADCs, which were first approved by the US Food and Drug Administration (FDA), the European Medicines Agency (EMA), the China National Medical Products Administration and the Japanese Pharmaceuticals and Medical Devices Agency for solid cancers. Among 16 trials, 4 (25%) met the ESMO-MCBS benefit threshold grade, while 12 (75%) of the regimens did not meet the ESMO-MCBS benefit threshold grade. 5 (31%) of the 16 trials had no published scorecard on the ESMO website due to the approval by other jurisdictions but not by the FDA or EMA. Discrepancies between our results and the ESMO scorecard were observed in 4 (36%) of 11 trials, mostly owing to integration of more recent data.ConclusionsESMO-MCBS is an important tool for assessing the clinical benefit of cancer drugs, but not all drugs met the meaningful benefit threshold.

show abstract

ESMO-Magnitude of Clinical Benefit Scale for haematological malignancies (ESMO-MCBS:H) version 1.0

Cited by 10 publications

References 41 publications

Characteristics of clinician input in Canadian funding decisions for cancer drugs: a cross-sectional study based on CADTH reimbursement recommendations

Characteristics of clinician input in Canadian funding decisions for cancer drugs: a cross-sectional study based on CADTH reimbursement recommendations

An Inclusive Civil Society Dialogue for Successful Implementation of the EU HTA Regulation: Call to Action to Ensure Appropriate Involvement of Stakeholders and Collaborators

Application of the ESMO Magnitude of Clinical Benefit Scale to assess the clinical benefit of antibody drug conjugates in solid cancer: a systematic descriptive analysis of phase III and pivotal phase II trials

Contact Info

Product

Resources

About