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Competing Interests: Disclosure AV reports personal fees for speaker, consultancy and advisory roles for AstraZeneca, Amgen, Basilea, BeiGene, Bayer, Boehringer Mannheim, Bristol-Myers Squibb (BMS), BTG, Daiichi Sankyo, Eisai, GlaxoSmithKline (GSK), Imaging Equipment Ltd (AAA), Incyte, Ipsen, Jiangsu Hengrui Medicines, Merck Sharp & Dohme (MSD), Pierre Fabre, Roche, Sanofi, Servier, Sirtex, Tahio and Terumo; research funding from Incyte and Servier; participation in educational activities for Onclive and Oncowissen.de. JB reports personal fees for advisory board membership from Basilea, BMS, Incyte and Taiho and institutional funding from Incyte. JE reports personal fees as an invited speaker from AstraZeneca, Bayer, Boston Scientific, Eisai, Ipsen, MSD, Roche and Servier; personal fees for advisory board membership from AstraZeneca, Basilea, Bayer, BeiGene, Eisai, Incyte, Ipsen, Merck Serono, MSD, Roche and Servier; personal fees for steering committee membership from MSD; institutional funding as a local principal investigator (PI) from Agios, Bayer, BeiGene, BMS, MSD, Novartis, Roche, Servier and Taiho, and as a coordinating PI from BeiGene, BMS and a non-remunerated role as a PI for Unicancer. RKK reports personal fees for advisory board membership from Exact Sciences, Gilead and Kinnate Biopharma; personal fees for Independent Data Monitoring Committee (IDMC) membership (2018-2020) from Genentech/Roche; institutional funding from Partner Therapeutics; institutional funding as a local PI from Agios, AstraZeneca, BMS, Eli Lilly, EMD Serono, Genentech/Roche, Loxo Oncology, Merck, Novartis, QED, Relay Therapeutics, Surface Oncology and Taiho and as a coordinating PI from AstraZeneca, Bayer, Exelixis and Merck; institutional funding as a steering committee member from Agios, AstraZeneca and Merck and non-remunerated roles as a PI for AstraZeneca, Bayer, Exelixis, and Merck; a non-remunerated advisory role as chair and member of the IDMC for Genentech/Roche and Merck. HJK reports fees paid to his institution as an invited speaker from CCO and Medtalk; fees paid to his institution for advisory board membership from AstraZeneca and Janssen; institutional funding as a local PI from Exelixis, Incyte, ITM Solucin GmbH, MSD, Roche and Taiho; is the EURACAN subdomain leader G5.1; member of the national guideline committee for BTC and HCC in the Netherlands. DM reports personal fees as an invited speaker from Amgen, AstraZeneca, Bayer, BMS, Foundation Medicine, HalioDX, Incyte, Leo Pharma, Merck Serono, MSD, Pierre Fabre, Roche, Sanofi, Servier, Veracyte and Viatris; personal fees for advisory board membership from Agios, Amgen, AstraZeneca, Bayer, BMS, HalioDX, Incyte, Merck Serono, MSD, Pierre Fabre, Roche, Servier and Taiho; personal fees for a writing engagement from Medscape; personal fees for expert testimony from AbbVie; travel expenses for medical congresses from Amgen, Bayer, BMS, Merck Serono, Pierre Fabre, Roche, Sanofi, Servier and Viatris. JNP reports personal fees for advisory board membership from AstraZeneca. LR reports personal fees as an invited speaker from AbbVie, Amgen, Bayer, Eisai, Gilead, Incyte, Ipsen, Lilly, Merck Serono, Roche, Sanofi and Servier; personal fees for advisory board membership from Amgen, ArQule, AstraZeneca, Basilea, Bayer, BMS, Celgene, Eisai, Exelixis, Jiangsu Hengrui Medicine, Genenta, Incyte, Ipsen, IQVIA, Lilly, MSD, Nerviano Medical Sciences, Roche, Sanofi, Servier, Taiho Oncology and Zymeworks; institutional funding as a local PI from Agios, ARMO BioSciences, AstraZeneca, BeiGene, Eisai, Exelixis, FibroGen, Incyte, Ipsen, Lilly, MSD, Nerviano Medical Sciences, Roche and Zymeworks; travel expenses from AstraZeneca and Ipsen. AS reports personal fees as an invited speaker from Incyte and Roche; personal fees for advisory board membership from Aignostics, Amgen, AstraZeneca, Bayer, Eli Lilly, Illumina, Janssen, MSD, Pfizer, Seattle Genetics and Thermo Fisher; fees paid to his institution for advisory board membership from BMS, Novartis and Takeda; institutional funding from Bayer, BMS, Chugai and Incyte. JWV reports personal fees as an invited speaker from Incyte, Ipsen, Mylan, Servier and Delcath; personal fees for advisory board membership from Agios, AstraZeneca, Autem, Baxter, Cantargia, Debiopharm, Genoscience Pharma, HUTCHMED, Imaging Equipment Ltd (AAA), Incyte, Keocyt, Medivir, Mundipharma EDO, NuCana BioMed, QED, Servier, Sirtex, Taiho and Zymeworks; institutional funding from AstraZeneca and Redx. MD reports personal fees as an invited speaker from Amgen, Bayer, Lilly, Merck KGaA, Pfizer, Pierre Fabre and Roche; personal fees for advisory board membership from Basilea, Bayer, Boehringer Ingelheim, Daiichi Sankyo, GSK, HalioDX, Ipsen, Lilly, Pierre Fabre, Rafael, Roche, Servier, SOTIO Biotech and Zymeworks; institutional funding from Bayer, Keocyt and Roche; institutional funding as a local PI from Amgen and Rafael.

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., (© The Author 2017. Published by Oxford University Press on behalf of the European Society for Medical Oncology. All rights reserved. For Permissions, please email: journals.permissions@oup.com.)