Launch Sequencing of Pharmaceuticals with Multiple Therapeutic Indications: Evidence from Seven Countries

Abstract

Abstract Background. New 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. Objective. This paper, first, maps marketing authorisation (MA) 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. Methods. Medicine approvals by the Food and Drug Administration between 2009-2019 were screened to identify 25 multi-indication medicines. Data on clinical trial characteristics, clinical performance and HTA outcomes were extracted from publicly available regulatory approval and HTA reports. Results. Relative 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 a surrogate primary endpoint. Clinical performance was similar across first and subsequent indications. Subsequent indications had faster HTA coverage recommendation times in England, Germany, and Canada. While the majority of first indications received HTA coverage recommendation across all settings, the proportion of subsequent indications with HTA coverage recommendation was low and uptake varied considerably across settings. Conclusions. Discordance 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.