Background Assessment of clinical benefit of systemic treatments of rare diseases including gastroenteropancreatic neuroendocrine tumours (GEP-NET) is challenging. Recently several tools have been developed to grade the clinical benefit of cancer drugs. The European Society for Medical Oncology (ESMO) has developed the ESMO Magnitude of Clinical Benefit Scale (ESMO-MCBS). The American Society of Clinical Oncology (ASCO) has developed and revised the ASCO framework consisting of the Net Health Benefit (NHB) score juxtaposed against the costs of the treatment. In this review, we graded systemic treatments for GEP-NET patients with both frameworks. Methods The electronic databases (PubMed and EMBASE) were searched for papers reporting comparative trials, conducted in adult GEP-NET patients in the English language. Papers were assessed according to the ESMO-MCBS and the NHB part of the ASCO revised Framework (NHB-ASCO-F) by four independent assessors, and discrepancies were discussed. Results The search yielded 32 trials of which 6 were eligible for grading with the ESMO-MCBS resulting in scores of 2 or 3. Eight trials were eligible for grading with the NHB-ASCO-F, resulting in scores between 37.6 and 57.4. Trials that were not primary assessable by the tools were analysed separately. Consensus between assessors was reached in 68% of trials with the ESMO-MCBS and in 23% of trials with the NHB-ASCO-F. Conclusion The currently used systemic treatments for GEP-NET patients had low scores according to the NHB-ASCO-F and none could be graded as meaningful clinical beneficial according to the ESMO-MCBS. Despite the low incidence, the heterogeneous patient population and relatively long natural course of NET, future studies on new treatment modalities should aim for high clinical benefit outcomes.

中文翻译：

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根据ESMO-MCBS和ASCO框架，对晚期胰腺和胃肠道神经内分泌肿瘤患者进行全身治疗的临床益处。

背景对包括胃肠道胰腺神经内分泌肿瘤（GEP-NET）在内的罕见疾病进行全身治疗的临床益处的评估具有挑战性。最近，已经开发了几种工具来对癌症药物的临床益处进行分级。欧洲医学肿瘤学会（ESMO）已制定了ESMO临床获益量表（ESMO-MCBS）。美国临床肿瘤学会（ASCO）已开发和修订了ASCO框架，该框架由与治疗费用并列的净健康收益（NHB）评分组成。在这篇综述中，我们对两种框架的GEP-NET患者的全身治疗进行了分级。方法在电子数据库（PubMed和EMBASE）中搜索报告比较试验的论文，这些论文以英语对成年GEP-NET患者进行。根据ESMO-MCBS和ASCO修订框架的NHB部分（NHB-ASCO-F），由四名独立评估者对论文进行了评估，并讨论了差异。结果搜索产生了32个试验，其中6个符合ESMO-MCBS的评分标准，得分为2或3。8个试验符合NHB-ASCO-F的评分标准，得分为37.6至57.4。不能通过工具进行主要评估的试验将单独进行分析。使用ESMO-MCBS的试验中有68％和使用NHB-ASCO-F的试验中有23％达到了评估者之间的共识。结论根据NHB-ASCO-F，目前对GEP-NET患者使用的全身治疗评分较低，根据ESMO-MCBS，没有一项可被归类为有意义的临床获益。尽管发病率很低，