title: Experience with outcomes research into the real-world effectiveness of novel therapies in Dutch daily practice from the context of conditional reimbursement.
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.
authors.affiliation: Institute for Medical Technology Assessment/Department of Health Policy and Management, Erasmus University, P.O. Box 1738, 3000 DR Rotterdam, The Netherlands; Mapi Group, De Molen 84, 3995 AX Houten, The Netherlands. Electronic address: jgaultney@mapigroup.com.
authors.forename: Jennifer G
authors.initials: JG
authors.lastname: Gaultney
authors.affiliation: Institute for Medical Technology Assessment/Department of Health Policy and Management, Erasmus University, P.O. Box 1738, 3000 DR Rotterdam, The Netherlands.
authors.forename: Margreet G
authors.initials: MG
authors.lastname: Franken
authors.affiliation: Institute for Medical Technology Assessment/Department of Health Policy and Management, Erasmus University, P.O. Box 1738, 3000 DR Rotterdam, The Netherlands.
authors.forename: Carin A
authors.initials: CA
authors.lastname: Uyl-de Groot
authors.affiliation: Institute for Medical Technology Assessment/Department of Health Policy and Management, Erasmus University, P.O. Box 1738, 3000 DR Rotterdam, The Netherlands.
authors.forename: William K
authors.initials: WK
authors.lastname: Redekop
authors.affiliation: Department of Haematology, VU University Medical Centre, P.O. Box 7057, 1007 MB Amsterdam, The Netherlands.
authors.forename: Peter C
authors.initials: PC
authors.lastname: Huijgens
authors.affiliation: HOVON Data Centre, Erasmus MC Cancer Institute-Clinical Trial Centre, P.O. Box 2040, 3000 CA Rotterdam, The Netherlands.
authors.forename: Bronno
authors.initials: B
authors.lastname: van der Holt
authors.affiliation: Department of Haematology, University Medical Centre Utrecht, Utrecht, The Netherlands.
authors.forename: Henk M
authors.initials: HM
authors.lastname: Lokhorst
authors.affiliation: Department of Haematology, Erasmus University Medical Centre, P.O. Box 2040, 3000 CA Rotterdam, The Netherlands.
authors.forename: Pieter
authors.initials: P
authors.lastname: Sonneveld
mesh_terms: D000970:Antineoplastic Agents / Q000009:adverse effects / Q000627:therapeutic use; D000069286:Bortezomib / Q000009:adverse effects / Q000627:therapeutic use; D004358:Drug Therapy / Q000191:economics; D064420:Drug-Related Side Effects and Adverse Reactions / Q000191:economics; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D009101:Multiple Myeloma / Q000188:drug therapy; D009426:Netherlands; D017063:Outcome Assessment, Health Care; D011050:Policy Making; D010818:Practice Patterns, Physicians'; D012051:Reimbursement Mechanisms / Q000331:legislation & jurisprudence / Q000458:organization & administration; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D000069286:Bortezomib
keywords: Conditional reimbursement; Daily practice; Effectiveness; Observational; Outcomes research