ESMO 2016 Press Release: First Feasibility Study of the ESMO-MCBS Scale in Rare Tumour Entities

LUGANO-COPENHAGEN – The results of the first study analysing the application of the ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) in a real-life context for rare tumour entities, were announced today at the ESMO 2016 Congress in Copenhagen.  

The ESMO-MCBS is a new tool to quantify the clinical benefit of a certain drug for the treatment of cancer and its application in daily clinical practice. It was first evaluated on common tumour entities by one of Europe’s largest cancer centres, the Medical University of Vienna (MUV), which also conducted this new study.

Dr Barbara Kiesewetter at MUV states, “We assessed data on neuroendocrine tumors (NET), glioblastoma, sarcomas, thyroid, pancreatic, ovarian, head/neck and urothelial cancers. Data was analysed in a three-step approach: we collected data on regimens in daily use at the MUV, then analysed the data with the ESMO-MCBS as our second step and finally we evaluated and discussed the data in terms of clinical feasibility and practicability in daily practice."

“We noted a phenomenon whereby the ESMO-MCBS particularly highlights the clinical benefit to be expected of new immunomodulatory drugs. This was also observed in our field testing on common tumour entities and may help us to implement these treatments in daily practice in the near future.  We are particularly excited that new data on check point inhibitors for rare entities is due to be available soon. For example, in head and neck cancers, the CHECKMATE141 data currently scores for an ESMO-MCBS score of 3 (field testing) based on available results2 but might improve to an even stronger recommendation with more mature survival data,” she continued.

“The practicability of applying the ESMO-MCBS is somewhat limited for very rare tumours, for example sarcoma and glioblastoma, due to a lack of randomized data. However, the ESMO-MCBS was applicable in most situations where controlled trials were available such as the data on salvage treatment with pazopanib for soft tissue sarcoma achieving a score of 3 based on a significant progression free survival (PFS) gain of 3 months in comparison to 1.6 months in the placebo arm3.

“For sarcomas, practicability of ESMO-MCBS was limited due to a lack of trials in many indications. However, the ESMO-MCBS was useful whenever randomized data were available. For example, the ESMO-MCBS score of 4 clearly underlines the clinical benefit achieved by adding dacarbazine to gemcitabine in pre-treated soft tissue sarcoma (4). This is in line with our clinical experience and supports further use of the ESMO Magnitude of Clinical Benefit Scale,” Kiesewetter explained.

The ESMO-MCBS has been designed to assess the therapeutic benefit of drugs registered for the treatment of cancer. + It considers the predefined primary and secondary study endpoints:  overall survival and progression-free survival in terms of absolute gain and lower end of the 95% confidence interval of the corresponding hazard ratio and quality of life or toxicity respectively. Data of the new treatment is then analysed with respect to the duration of response or survival in the control arm, which has to be entered in corresponding forms and results in a clinical benefit ranking.

 “We found that the ESMO-MCBS is a helpful tool for clinical practice in rare tumours, as well as for common tumour entities, if randomized data is available. It supports treatment decisions based on the expected clinical benefit. It is very simple to use and we feel that it is going to prove to be a very important tool for daily clinical practice based on our study results. Clinicians can go back to the data when considering new treatments and use the ESMO-MCBS online to analyse what can be expected from a new approach,” concluded Dr Kiesewetter.

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Notes to Editors

References

Abstract 1364O_PR “The ESMO-Magnitude of Clinical Benefit Scale (MCBS) in rare tumour entities: A real life experience at the Medical University Vienna” will be presented by Barbara Kiesewetter during the Proffered Paper session, Public health and health economics on Monday 10 October 2016, 16:30 to 18:00 (CEST) in Room Oslo.

2 Ferris RL, Blumenschein GR, Fayette J, et al. Further evaluations of nivolumab (nivo) versus investigator’s choice (IC) chemotherapy for recurrent or metastatic (R/M) squamous cell carcinoma of the head and neck (SCCHN): CheckMate 141. 2016 ASCO Annual Meeting J Clin Oncol 34, 2016 (suppl; abstr 6009)

3 Van der Graaf WT, Blay JY, Chawla SP, et al. Pazopanib for metastatic soft-tissue sarcoma (PALETTE): a randomised, double-blind, placebo-controlled phase 3 Trial. Lancet. 2012;379(9829):1879-86.

4 García-Del-Muro X, López-Pousa A, Maurel J, et al. Randomized phase II study comparing gemcitabine plus dacarbazine versus dacarbazine alone in patients with previously treated soft tissue sarcoma: a Spanish Group for Research on Sarcomas study. J Clin Oncol. 2011;29(18):2528-33.

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Abstract for 1364O_PR

The ESMO-Magnitude of Clinical Benefit Scale (MCBS) in rare tumor entities: A real life experience at the Medical University Vienna

B. Kiesewetter, M. Raderer, C. Marosi, T. Brodowicz, G. Prager, M. Krainer, T. Fuereder, G.J. Locker, C.C. Zielinski
Medicine I, Clin. Div. of Oncology and Comprehensive Cancer Center, Medizinische Universitaet Wien (Medical University of Vienna), Vienna, Austria

Background: The ESMO-MCBS is a new tool to quantify the clinical benefit of a certain drug for the treatment of cancer. Recently, we have evaluated the practicability of the MCBS in daily practice of treating common tumor entities at our center. However, to date there is no experience of using the MCBS for rare tumors.

Methods: This study analyses the feasibility of the MCBS for rare tumor entities at the Clinical Division of Oncology, Medical University Vienna. We developed a 3-step approach to address this question. First, we retrospectively collected data to gain an overview on treatments in regular use. Second, we scored data with the MCBS, and third, evaluated results with the corresponding program directorships to assess the feasibility in a real-life context.

Results: We assessed data on neuroendocrine tumors (NET), glioblastoma, sarcomas, thyroid-, pancreatic-, ovarian-, head/neck- and urothelial cancer. The following results were obtained: 1.) NET and thyroid cancer: Recently published trials are comparable in design and efficacy and the MCBS scores are consistent with the clinical benefit seen in practice. However, the MCBS added little information on the question of optimal sequencing except for differentiated thyroid cancer where prior treatment (TKI or not) suggests an algorithm. 2.) Pancreatic cancer: only two major 1st-line trials are currently available and a direct comparison is difficult due to diverging populations. 3.) Sarcomas: MCBS reflects well the situation of GIST and was useful for randomized data on non-GIST sarcoma, but MCBS practicability was limited in rare subtypes due to a lack of controlled trials. 4.) Ovarian cancer: the MCBS supports the use of bevacizumab in high-risk patients. This is in line with our experience. To date, there is only limited data for glioblastoma, head/neck- and urothelial cancer. However, if randomized trials were available, the MCBS-rating supported clinical decisions particularly for novel compounds. A broad selection of analyzed data will be presented at the meeting.

Conclusions: The MCBS is a helpful tool for clinical practice in rare tumors, if randomized data are available. It supports treatment decisions based on the expected clinical benefit.

Legal entity responsible for the study: Department of Medicine I, Medical University of Vienna

Funding: Department of Medicine I, Medical University of Vienna

Disclosure:

T. Brodowicz: All outside the submitted work: Personal fees from Roche (lecture fee), Amgen (lecture fee, advisory board), Bayer (lecture fee, advisory board), Novartis (lecture fee, advisory board), PharmaMar (lecture fee), Eisai (lecture fee, advisory board).
G. Prager: Honoraria for lectures by Merck Serono, Amgen, Bayer, Servier, Lilly, Celgene, Roche, Sanofi Aventis.
C.C. Zielinski: Honoraria for advisory boards by Bristol Myers-Squibb, AstraZeneca, Imugene, Roche
All other authors have declared no conflicts of interest.

Keywords: quality control, rare tumor, clinical benefit, ESMO-MCBS