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Balancing early access with uncertainties in evidence for drugs authorized by prospective case series - systematic review of reimbursement decisions

Journal article
Authors Susanna Maria Wallerstedt
M. Henriksson
Published in British Journal of Clinical Pharmacology
Volume 84
Issue 6
Pages 1146-1155
ISSN 0306-5251
Publication year 2018
Published at Institute of Neuroscience and Physiology, Department of Pharmacology
Pages 1146-1155
Language en
Links https://doi.org/10.1111/bcp.13531
Keywords cost-effectiveness, decision making, uncertainty, evidence-based health care, prospective case series, chronic myeloid-leukemia, cell lung-cancer, end-points, brentuximab, vedotin, phase-ii, survival, safety, trial, lymphoma, postapproval, Pharmacology & Pharmacy
Subject categories Pharmacology

Abstract

AimsTo review clinical and cost-effectiveness evidence underlying reimbursement decisions relating to drugs whose authorization mainly is based on evidence from prospective case series. MethodsA systematic review of all new drugs evaluated in 2011-2016 within a health care profession-driven resource prioritization process, with a market approval based on prospective case series, and a reimbursement decision by the Swedish Dental and Pharmaceutical Benefits Agency (TLV). Public assessment reports from the European Medicines Agency, published pivotal studies, and TLV, Scottish Medicines Consortium and National Institute of Health and Care Excellence decisions and guidance documents were reviewed. ResultsSix drug cases were assessed (brentuximab vedotin, bosutinib, ponatinib, idelalisib, vismodegib, ceritinib). The validity of the pivotal studies was hampered by the use of surrogate primary outcomes and the absence of recruitment information. To quantify drug treatment effect sizes, the reimbursement agencies primarily used data from another source in indirect comparisons. TLV granted reimbursement in five cases, compared with five in five cases for Scottish Medicines Consortium and four in five cases for National Institute of Health and Care Excellence. Decision modifiers, contributing to granted reimbursement despite hugely uncertain cost-effectiveness ratios, were, for example, small population size, occasionally linked to budget impact, severity of disease, end of life and improved life expectancy. ConclusionFor drugs whose authorization is based on prospective case series, most applications for reimbursement within public health care are granted. The underlying evidence has limitations over and above the design per se, and decision modifiers are frequently referred to in the value-based pricing decision making.

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