Launch sequencing of pharmaceuticals with multiple therapeutic indications: evidence from seven countries

BMC Health Services Research

Research

Aurelio Miracolo¹  Mackenzie Mills¹  Panos Kanavos¹  Daniel Michaeli² 

[1] Department of Health Policy and LSE Health - Medical Technology Research Group (MTRG), London School of Economics, Houghton St, WC2A 2AE, London, UK;Department of Health Policy and LSE Health - Medical Technology Research Group (MTRG), London School of Economics, Houghton St, WC2A 2AE, London, UK;Fifth Department of Medicine, University Hospital Mannheim, Heidelberg University, Mannheim, Germany;Department of Personalized Oncology, University Hospital Mannheim, Heidelberg University, Mannheim, Germany;

关键词: Oncology;    Clinical Development;    Regulatory Approval;    HTA;    Indication-based pricing;   

DOI  :  10.1186/s12913-023-09095-2

 received in 2022-08-11, accepted in 2023-01-23,  发布年份 2023

来源: Springer

【 摘 要 】

BackgroundNew medicines are increasingly being identified as efficacious across multiple indications. The impact of current pricing and reimbursement policies on launch decisions across these indications remains unclear.ObjectiveThis paper, first, maps marketing authorisation and HTA coverage recommendation sequences of multi-indication medicines across Germany, France, England, Scotland, Canada, Australia, and the USA, and, second, evaluates the clinical characteristics, clinical development time and coverage recommendation time of multi-indication medicines, drawing comparisons between the first and subsequent indications of an approved molecule.MethodsMedicine approvals by the Food and Drug Administration between 2009–2019 were screened to identify multi-indication products with approved oncology indications. Data on clinical trial characteristics, clinical performance and HTA outcomes were extracted from publicly available regulatory approval and HTA reports.ResultsRelative to subsequent indications, first indications were more likely to receive conditional marketing authorisation, have an orphan designation, have a single arm phase II pivotal trial and lower MCBS score. Subsequent indications had faster HTA coverage recommendation times in England and Canada. While the majority of first indications received HTA coverage recommendations across all settings, the proportion of subsequent indications with HTA coverage recommendations was lower and uptake varied considerably across settings.ConclusionsDiscordance in the value of first versus subsequent indications can pose major challenges in systems that define price based on the initial indication. Current pricing and reimbursement systems generate significant fragmentation in the approval and availability of multi-indication products across settings.

【 授权许可】

CC BY   
© The Author(s) 2023

【 图 表 】

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