Neoadjuvant and adjuvant end-points in health technology assessment in oncology.

Health technology assessment (HTA) of clinical and economic value of a new intervention is an integral step in providing the access of patients to innovative cancer care and treatment. Overall survival (OS) is the preferred criterion for demonstrating the therapeutic efficacy in HTA given its direct clinical and patient relevance. However, with often long life expectancy of patients with early cancer, analysis of OS becomes less practical. Partially due to this reason, pathological complete response (pCR) and time-to-event end-points like disease-free survival are frequently incorporated into the pivotal clinical trials in the neoadjuvant and adjuvant settings. However, there exists a discrepancy between different national HTA bodies regarding the acknowledgement of patient relevance of these end-points. In this article, we analysed the perspectives of patients on different aspects of end-points used in clinical trials in early cancer. Gathered evidence strongly suggests that complete tumour eradication and reduced risk of recurrence provide important psychological benefits thus signifying that pCR and time-to-event end-points are directly relevant to patients. Additionally, we reviewed opinions on patient relevance of neoadjuvant and adjuvant therapy end-points adopted by HTA bodies during the recent evaluations. We found that improvements in end-points used in the adjuvant setting were commonly considered as valuable to patients. In contrast, opinions on patient relevance of neoadjuvant therapy end-points varied between the national HTA bodies. Universal acknowledgement of patient relevance of therapeutic end-points for early cancer by HTA bodies is necessary to balance the inequality in uptake of innovative therapies into national healthcare systems.

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Déclaration de conflit d'intérêts

Conflict of interest statement The authors declare the following financial interests/personal relationships, which may be considered as potential competing interests: Nadia Harbeck received honoraria for lectures and/or consulting from Amgen, Astra Zeneca, BMS, Celgene, Daiichi-Sankyo, Lilly, MSD, Novartis, Odonate, Pierre Fabre, Pfizer, Roche, Samsung, Sandoz/Hexal and Seattle Genetics. Andreas Schneeweiss reports grants from Celgene, Roche, AbbVie and Molecular Partner; and personal fees from Roche, AstraZeneca, Celgene, Pfizer, Novartis, MSD, Tesaro and Lilly; outside the submitted work. Kurt Miller served as advisor and reports honoraria from Astellas, Bayer, BMS, Ferring, Janssen, MSD, Novartis, Pfizer and Roche. Claus Garbe reports personal fees from Amgen, MSD and Philogen; grants from Roche; and grants and personal fees from BMS, Novartis, NeraCare and Sanofi; outside the submitted work. Wilfried EE Eberhardt reports personal fees from MSD/Merck, Roche, Pfizer, Sanofi Aventis, Novartis, Takeda and Ingelheim; and grants and personal fees from Bristol Myers Squibb, AstraZeneca and Eli Lilly; outside the submitted work. Jens P Klussmann reports personal fees from Merck and BMS; and grants from MSD. Marc-Oliver Grimm reports grants and personal fees from Novartis and BMS; and personal fees from Pfizer, Bayer HealthCare, Astellas, Intuitive Surgical, Sanofi Aventis, Hexal, Apogepha, Amgen, AstraZeneca, MSD, Janssen Cilag, Ono Pharma, Ipsen Pharma, Medac and Merck; outside the submitted work. Diana Lüftner reports personal fees from MSD, Loreal, Eli Lilly, Pfizer, GSK and Teva; and grants and personal fees from Novartis; outside the submitted work. Peter Thuss-Patience, Frank Griesinger, Barbara Wollenberg and Thomas Zander have no potential conflict of interest to declare.