The European Society for Medical Oncology 'Magnitude of Clinical Benefit Scale' field-tested in infrequent tumour entities: an extended analysis of its feasibility at the Medical University of Vienna

Abstract

BackgroundThe European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) is a new tool to quantify the clinical benefit that may be anticipated from a novel anticancer treatment. We present here an analysis on the feasibility of the ESMO-MCBS in less frequent tumour entities.MethodsThis study evaluates the practicability of the ESMO-MCBS for metastatic neuroendocrine tumours (NETs), soft tissue sarcomas, glioblastoma, thyroid cancer, pancreatic cancer, head/neck cancer, urothelial cancer and ovarian cancer at the Medical University Vienna. A three-step approach including data acquisition, assessment of ESMO-MCBS scores and evaluation of results with a focus on clinical feasibility was applied.ResultsIn NET and thyroid cancer, all analysed trials were very comparable in design and efficacy, and the ESMO-MCBS scores appeared to be consistent with the clinical benefit seen in practice. For pancreatic cancer, it was more difficult to compare first-line trials due to diverging populations included in the respective studies. Concerning soft tissue sarcomas, the ESMO-MCBS was applicable for gastrointestinal stromal tumours(GIST) and ‘non-GIST’ soft tissue sarcoma with respect to data deriving from randomised studies. However, due to the heterogeneity of the disease itself and a limited number of controlled trials, limitations are noted. In ovarian cancer, the ESMO-MCBS supported the use of bevacizumab in high-risk patients. To date, there are only limited data for glioblastoma, head/neck cancer and urothelial cancer but whenever randomised trials were available, the ESMO-MCBS rating supported clinical decisions. Interestingly, nivolumab for salvage treatment of head/neck cancer rated extremely high.ConclusionThe ESMO-MCBS scores supported our common treatment strategies and highlight the potential of new immunomodulatory drugs. Our results encourage further development of the ESMO-MCBS.

Review of cancer treatment with immune checkpoint inhibitors

“…New pricing models could be a future way for reimbursement in order to simplify the access to this important class of drugs [ 59 – 61 ]. The involvement of patients and patient advocacy groups must become the norm in HTA assessment which should serve the patients to get access to innovative drugs, as outlined in the magnitude of clinical benefit scale generated by the European Society for Medical Oncology [ 62 ].…”

Treatment patterns and oncological outcome of patients with advanced small intestinal neuroendocrine tumors: real-world data from the Medical University of Vienna

“… 22 , 23 Thus, quality of life and toxicity results should also be taken into consideration when applying these therapies. 24 …”

“…22,23 Thus, quality of life and toxicity results should also be taken into consideration when applying these therapies. 24 Owing to the strict eligibility criteria of randomized controlled trials (RCTs), study populations may differ considerably (e.g. with regard to performance score or comedication) from the patient population seen in day-to-day clinical practice and hence also long-term data might not be comparable.…”

How I treat cancer special issue

“…Within the 3 years of its existence, ESMO Open – Cancer Horizons has so far published a total of 330 articles dealing with all possible aspects of cancer and cancer care. These have included papers in the following areas: epidemiological studies1 2; access to cancer care3–7; the advent of biologicals and their broad use in the clinic8 9; various aspects of magnitude of clinical benefit achieved by the use of certain drugs10 11; biological considerations on the regulation of tumour growth12 13 and propagation14–16; clinical cutting-edge papers on the treatment of cancer in selected unfamiliar situations1 17–21; new treatment options either registered by the European Medicines Agency (which ESMO Open regularly publishes in its ‘EMA Corner’22) or a broader understanding of existing compounds23–28 in an abundance of various cancers29–35 and quality of life issues of cancer patients 3 36–42. These patient-oriented publications were complemented by aspects concerning the oncological profession 4 43.…”

“…The current special issue of ESMO Open – Cancer Horizons is the result of a request we have sent out to key opinion leaders in various fields of cancer in which we have asked them to give a very succinct opinion on how they would treat cancer in a very special and circumscript situation related to predefined diagnosis. Thus, we have intended to present the oncological community with an endeavour which would encompass a series of ESMO’s attempts including the generation of guidelines,51 the results of the Magnitude of Clinical Benefit10 11 36 and personal considerations of key opinion leaders 55–58. We hope that these very short and succinct summaries will help you in your everyday care of patients with cancer who give the true meaning to all of our endeavours.…”