Parametric Survival Extrapolation of Early Survival Data in Economic Analyses: A Comparison of Projected Versus Observed Updated Survival |
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| Affiliation: | 1. Odette Cancer Centre, Sunnybrook Health Sciences Centre, Toronto, Ontario, Canada;2. University of Toronto, Toronto, Ontario, Canada;3. Canadian Centre for Applied Research in Cancer Control, Toronto, Ontario, Canada;4. Cancer Care Ontario, Toronto, Ontario, Canada;1. Health Economic Assessment Network, Paris, France;2. French National Center for Scientific Research, Paris, France;3. Sciences Po, Center of the Sociology of Organizations, Paris, France;4. Sociology and Anthropology Department, Paris 8 University, Paris, France;5. Rhumatology Department, Cochin Hospital, Paris, France;6. French League Against Rheumatism (AFLAR), Paris, France;7. UCB Pharma, Colombes, France;8. Rhumatology Department, Grenobles Alpes University Hospital, Echirolles, France;9. Kantar Health, Gentilly, France;1. School of Rehabilitation Sciences, McMaster University, Hamilton, Ontario, Canada;2. School of Physiotherapy, Western University, London, Ontario, Canada;3. Department of Rehabilitation Medicine and Department of Plastic, Reconstructive, and Hand Surgery, Erasmus Medical Centre, Rotterdam, The Netherlands;4. Hand and Wrist Center, Xpert Clinic and Handtherapie Nederland, Rotterdam, The Netherlands;1. Centre for Health Economics, Monash University, Melbourne, Australia;2. School of Public Health and Preventive Medicine, Monash University, Melbourne, Australia;3. Department of Surgery, School of Clinical Sciences at Monash Health, Monash University, Melbourne, Australia;4. Department of Cardiothoracic Surgery, Monash Health, Monash University, Melbourne, Australia;5. Department of Haematology, Monash Health, Monash University, Melbourne, Australia;6. School of Public Health, Curtin University, Perth, Australia;1. School of Population Health, Curtin University, Perth, Australia;2. Department of Community Medicine, University of Tromsø, Tromsø, Norway;3. Division of Health Services, Norwegian Institute of Public Health, Oslo, Norway;1. Exeter Test Group, University of Exeter Medical School, St Luke’s Campus, Exeter, England, UK;2. Health Economics Group, University of Exeter Medical School, St Luke’s Campus, Exeter, England, UK;3. PenTAG, University of Exeter Medical School, St Luke’s Campus, Exeter, England, UK;4. University of Exeter, Exeter, England, UK |
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| Abstract: | ObjectivesTo establish the value of cancer drugs by cost-effectiveness analysis, lifetime parametric survival extrapolations are often fitted to early data. Recent literature suggests that the benefit of cancer agents in primary publications is often different compared with updated data. This study aimed to examine the projected survival based on parametric extrapolations compared with observed survival based on updated data.MethodsUS Food and Drug Administration oncology approvals from January 2006 to December 2015 were reviewed to identify randomized controlled trials, with updated overall survival (OS) or progression-free survival (PFS) data within 5 years. Individual patient data were reconstructed using established methods on initial and updated publications. Projected survival was calculated as the best-fit parametric restricted mean survival time (RMST) based on extrapolated initial Kaplan-Meier curves whereas observed survival was calculated as observed RMST based on updated Kaplan-Meier curves. Mean deviations, mean absolute error (MAE), mean absolute percentage error, and linear regressions were conducted to examine the relationship between projected and observed survival.ResultsIn total, 32 randomized controlled trials were included. The MAE between the projected RMST and observed RMST was 3.18 months (OS) and 2.84 months (PFS) and absolute percentage error of 100% (OS) and 23% (PFS), suggesting substantial imprecision of the projected RMST in predicting the updated RMST. The linear regression indicated MAE increased as time extrapolated and as the percentage of censored patients increased.ConclusionsThis study demonstrated substantial difference in projected survival between initial and updated publications. Health technology assessment committees need to be aware of the potential uncertainty of incremental effectiveness and resultant value-for-money assessment when making reimbursement decisions based on initial publications with immature survival data. |
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| Keywords: | survival analysis survival extrapolation health technology assessment |
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