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Public health policy and health economics

2140 - Magnitude of Clinical Benefit of Randomized Controlled Trials Supporting US Food and Drug Administration Approval of Drugs for Solid Tumours.


09 Sep 2017


Public health policy and health economics


Cancers in Adolescents and Young Adults (AYA);  Bioethical Principles and GCP


Consolacion Molto Valiente


Annals of Oncology (2017) 28 (suppl_5): v511-v520. 10.1093/annonc/mdx385


C. Molto Valiente1, E. Amir2, A. Ocana Fernandez3, A. Templeton4, L. del Carpio Huerta1, J. Del Paggio5, A. Barnadas1, C. Booth6, A. Tibau1

Author affiliations

  • 1 Oncology Department, Hospital de la Santa Creu i Sant Pau and Universitat Autònoma de Barcelona, 8026 - Barcelona/ES
  • 2 Department Of Medical Oncology And Hematology, Princess Margaret Cancer Centre and the University of Toronto, M5G2M9 - Toronto/CA
  • 3 Medical Oncology Department And Translational Research Unit, Albacete University Hospital, Albacete/ES
  • 4 Department Of Medical Oncology, St Claraspital and Faculty of Medicine, University of Basel, Basel/CH
  • 5 Division Of Cancer Care And Epidemiology, Queen’s University Cancer Research Institute, Kingston/CA
  • 6 Departments Of Oncology And Public Health Sciences, Queen’s University Cancer Research Institute, K7L 3N6 - Kingston/CA


Abstract 2140


The European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) is a validated and reproducible tool to assess the magnitude of clinical benefit from drugs for solid tumors. Here, we evaluate characteristics and outcomes of clinical trials supporting approval by the FDA and their association with ESMO-MCBS.


We searched the Drugs@FDA website for applications of anticancer drugs from January 2006 to December 2016. Drug labels and reports of registration trials were reviewed and study characteristics, efficacy, toxicity and quality of life outcomes as well as regulatory pathways were collected. For randomized controlled trials (RCTs) ESMO-MCBS grades were applied. Meaningful clinical benefit was defined as a grade of A or B for trials of neo/adjuvant intent and 4 or 5 for those of palliative intent. Comparisons between groups were assessed using Logistic regression and the Mann Whitney U test.


We identified 137 studies; 109 (80%) of which were RCTs. These led to the approval of 63 individual drugs for 118 licensed indications. Among the 105 RCTs for which the ESMO-MCBS could be applied, 7 (6%) were in the neo/adjuvant setting and 98 (94%) in the palliative setting. Only 46 (44%) met the ESMO-MCBS clinically meaningful benefit threshold (100% of neo/adjuvant trials and 41% of palliative trials). In multivariable analysis of palliative therapy trials, meaningful ESMO-MCBS grades were associated with phase III trials (compared to phase II; OR 38.45, P = 0.004), those with overall survival as their primary endpoint (compared to intermediate endpoints; OR 8.28, P = 0.001) and trials of targeted drugs with companion diagnostics (OR 11.62, P 


In patients with advanced solid tumours, fewer than half of RCTs supporting FDA approval meet the threshold for clinically meaningful benefit using validated scales.

Clinical trial identification

Not applicable

Legal entity responsible for the study





All authors have declared no conflicts of interest.

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