Savina, Marion Gourgou, Sophie Italiano, Antoine Dinart, Derek Rondeau, Virginie Penel, Nicolas Mathoulin-Pelissier, Simone and Bellera, Carine 2018. Meta-analyses evaluating surrogate endpoints for overall survival in cancer randomized trials: A critical review. Critical Reviews in Oncology/Hematology, Vol. 123, p. 21.
Saad, E D and Buyse, M 2017. Exploratory analysis of the association of depth of response and survival in patients with metastatic non-small-cell lung cancer treated with a targeted therapy or immunotherapy. Annals of Oncology, Vol. 28, Issue. 11, p. 2629.
Michiels, Stefan Saad, Everardo D. and Buyse, Marc 2017. Progression-Free Survival as a Surrogate for Overall Survival in Clinical Trials of Targeted Therapy in Advanced Solid Tumors. Drugs, Vol. 77, Issue. 7, p. 713.
Devlin, Nancy J. and Lorgelly, Paula K. 2017. QALYs as a measure of value in cancer. Journal of Cancer Policy, Vol. 11, p. 19.
Ciani, Oriana Buyse, Marc Drummond, Michael Rasi, Guido Saad, Everardo D. and Taylor, Rod S. 2017. Time to Review the Role of Surrogate End Points in Health Policy: State of the Art and the Way Forward. Value in Health, Vol. 20, Issue. 3, p. 487.
Woolacott, Nerys Corbett, Mark Jones-Diette, Julie and Hodgson, Robert 2017. Methodological challenges for the evaluation of clinical effectiveness in the context of accelerated regulatory approval: an overview. Journal of Clinical Epidemiology, Vol. 90, p. 108.
Hettle, Robert Corbett, Mark Hinde, Sebastian Hodgson, Robert Jones-Diette, Julie Woolacott, Nerys and Palmer, Stephen 2017. The assessment and appraisal of regenerative medicines and cell therapy products: an exploration of methods for review, economic evaluation and appraisal. Health Technology Assessment, Vol. 21, Issue. 7, p. 1.
Harshman, Lauren C. Xie, Wanling Moreira, Raphael B. Bossé, Dominick Ruiz Ares, Gustavo J. Sweeney, Christopher J. and Choueiri, Toni K. 2017. Evaluation of disease-free survival as an intermediate metric of overall survival in patients with localized renal cell carcinoma: A trial-level meta-analysis. Cancer,
Xie, Wanling Regan, Meredith M. Buyse, Marc Halabi, Susan Kantoff, Philip W. Sartor, Oliver Soule, Howard Clarke, Noel W. Collette, Laurence Dignam, James J. Fizazi, Karim Paruleker, Wendy R. Sandler, Howard M. Sydes, Matthew R. Tombal, Bertrand Williams, Scott G. and Sweeney, Christopher J. 2017. Metastasis-Free Survival Is a Strong Surrogate of Overall Survival in Localized Prostate Cancer. Journal of Clinical Oncology, Vol. 35, Issue. 27, p. 3097.
Laporte, S. Chapelle, C. Bertoletti, L. Ollier, E. Zufferey, P. Lega, J.-C. Merah, A. Décousus, H. Schulman, S. Meyer, G. Cucherat, M. and Mismetti, P. 2017. Assessment of clinically relevant bleeding as a surrogate outcome for major bleeding: validation by meta-analysis of randomized controlled trials. Journal of Thrombosis and Haemostasis, Vol. 15, Issue. 8, p. 1547.
Burke, Harry B. 2016. Predicting Clinical Outcomes Using Molecular Biomarkers. Biomarkers in Cancer, Vol. 8, p. BIC.S33380.
Vitry, Agnes Mintzes, Barbara and Lipworth, Wendy 2016. Access to new cancer medicines in Australia: dispelling the myths and informing a public debate. Journal of Pharmaceutical Policy and Practice, Vol. 9, Issue. 1,
Ciani, Oriana Buyse, Marc Drummond, Mike Rasi, Guido Saad, Everardo D. and Taylor, Rod S. 2016. Use of surrogate end points in healthcare policy: a proposal for adoption of a validation framework. Nature Reviews Drug Discovery, Vol. 15, Issue. 7, p. 516.
Amur, S LaVange, L Zineh, I Buckman-Garner, S and Woodcock, J 2015. Biomarker Qualification: Toward a Multiple Stakeholder Framework for Biomarker Development, Regulatory Acceptance, and Utilization. Clinical Pharmacology & Therapeutics, Vol. 98, Issue. 1, p. 34.
Chibaudel, Benoist Bonnetain, Franck Tournigand, Christophe de Larauze, Marine Hug de Gramont, Armand Laurent-Puig, Pierre Paget, Jérôme Hadengue, Alexandra Notelet, Dominique Benetkiewicz, Magdalena André, Thierry and de Gramont, Aimery 2015. STRATEGIC-1: A multiple-lines, randomized, open-label GERCOR phase III study in patients with unresectable wild-type RAS metastatic colorectal cancer. BMC Cancer, Vol. 15, Issue. 1,
Drummond, Michael 2015. When do performance-based risk-sharing arrangements make sense?. The European Journal of Health Economics, Vol. 16, Issue. 6, p. 569.
Ciani, Oriana Buyse, Marc Garside, Ruth Peters, Jaime Saad, Everardo D. Stein, Ken and Taylor, Rod S. 2015. Meta-analyses of randomized controlled trials show suboptimal validity of surrogate outcomes for overall survival in advanced colorectal cancer. Journal of Clinical Epidemiology, Vol. 68, Issue. 7, p. 833.
Objectives: Licensing of, and coverage decisions on, new therapies should rely on evidence from patient-relevant endpoints such as overall survival (OS). Nevertheless, evidence from surrogate endpoints may also be useful, as it may not only expedite the regulatory approval of new therapies but also inform coverage decisions. It is, therefore, essential that candidate surrogate endpoints be properly validated. However, there is no consensus on statistical methods for such validation and on how the evidence thus derived should be applied by policy makers.
Methods: We review current statistical approaches to surrogate-endpoint validation based on meta-analysis in various advanced-tumor settings. We assessed the suitability of two surrogates (progression-free survival [PFS] and time-to-progression [TTP]) using three current validation frameworks: Elston and Taylor's framework, the German Institute of Quality and Efficiency in Health Care's (IQWiG) framework and the Biomarker-Surrogacy Evaluation Schema (BSES3).
Results: A wide variety of statistical methods have been used to assess surrogacy. The strength of the association between the two surrogates and OS was generally low. The level of evidence (observation-level versus treatment-level) available varied considerably by cancer type, by evaluation tools and was not always consistent even within one specific cancer type.
Conclusions: Not in all solid tumors the treatment-level association between PFS or TTP and OS has been investigated. According to IQWiG's framework, only PFS achieved acceptable evidence of surrogacy in metastatic colorectal and ovarian cancer treated with cytotoxic agents. Our study emphasizes the challenges of surrogate-endpoint validation and the importance of building consensus on the development of evaluation frameworks.
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