Experience with outcomes research into the real-world effectiveness of novel therapies in Dutch daily practice from the context of conditional reimbursement |
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| Institution: | 1. Institute for Medical Technology Assessment/Department of Health Policy and Management, Erasmus University, P.O. Box 1738, 3000 DR Rotterdam, The Netherlands;2. Mapi Group, De Molen 84, 3995 AX Houten, The Netherlands;3. Department of Haematology, VU University Medical Centre, P.O. Box 7057, 1007 MB Amsterdam, The Netherlands;4. HOVON Data Centre, Erasmus MC Cancer Institute-Clinical Trial Centre, P.O. Box 2040, 3000 CA Rotterdam, The Netherlands;5. Department of Haematology, University Medical Centre Utrecht, Utrecht, The Netherlands;6. Department of Haematology, Erasmus University Medical Centre, P.O. Box 2040, 3000 CA Rotterdam, The Netherlands;1. Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston, MA, USA;2. Boston Scientific, Marlborough, MA, USA;1. Institute of Health Policy and Management (iBMG), Erasmus University, PO Box 1738, 3000 DR Rotterdam, The Netherlands;2. Amgen, PO Box 3345, 4800 DH Breda, The Netherlands;3. Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, PO Box 80082, 3508 TB Utrecht, The Netherlands;1. LSE Health, Department of Health Policy, London School of Economics and Political Science, London, United Kingdom;2. Scientific Advice, National Institute for Health and Care Excellence, London, United Kingdom;1. Bioethics Policy Studies, Ewha Womans University, Seoul, South Korea;2. Health Insurance Review and Assessment Service, Seoul, South Korea;3. College of Pharmacy, Ewha Womans University, Seoul, South Korea;4. College of Pharmacy, Gyeongsang University, Jinju, South Korea;1. Postdoctoral Fellow, Department of Population Medicine, Harvard Medical School and Harvard Pilgrim Health Care Institute, Boston, MA, United States;2. Professor and Director, Centre for Health Services and Policy Research, School of Population and Public Health, University of British Columbia, Vancouver, BC, United States;3. Associate Professor, Department of Population Medicine, Harvard Medical School and Harvard Pilgrim Health Care Institute, Boston, MA, United States |
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| Abstract: | Policymakers more often request outcomes research for expensive therapies to help resolve uncertainty of their health benefits and budget impact at reimbursement. Given the limitations of observational data, we assessed its usefulness in evaluating clinical outcomes for bortezomib in advanced multiple myeloma patients. Data were retrospectively collected from patients included in the pivotal Assessment of Proteasome Inhibition for Extending Remissions trial (APEX; n = 333) and two groups of daily practice patients treated with bortezomib following progression from upfront therapy (n = 201): real-world patients treated as of May 2009 (RW-1; n = 72) and June 2012 (RW-2; n = 129). Prognosis, treatment, and effectiveness were compared. Outcomes research was useful for policymakers for addressing to whom and how bortezomib was administered in daily practice. It was limited however in generating robust evidence on real-world safety and effectiveness. The quality of real-world evidence on effectiveness was low due to missing data in patient charts, existing treatment variation and the dynamics in care during the novel drug's initial market uptake period. Policymakers requesting real-world evidence on clinical outcomes for reimbursement decisions should be aware of these limitations and advised to carefully consider beforehand the type of evidence that best addresses their needs for the re-assessment phase. |
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| Keywords: | Outcomes research Effectiveness Observational Daily practice Conditional reimbursement |
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