PND134 HTA AND ACCESS FOR ORPHAN DRUGS: IDENTIFICATION OF 4 CRITICAL DIFFERENCES BETWEEN UK AND US PATHWAYS

Abstract

Exponentially increasing orphan drug prices has negatively impacted reimbursement decisions, limiting patient access. Availability of orphan drugs varies jurisdictionally, as do the HTA and access processes. To compare the UK and US, four mutual themes will be identified to highlight the differences in orphan drug processes and the associated effects. A systematic literature review was conducted on publications, indexed in PubMed and EMBASE, to identify, via scoring and ranking, comparative and contrasting themes between the UK and US. Articles published after 10/06/2016 were selected using inclusion and exclusion criteria and were confirmed for validity using CASP checklists. Findings were tested and confirmed through interviews, following predefined discussion guides with experts in orphan drug HTAs and market access. Four themes emerged from the literature review, which were used to compare the UK and US: HTA pricing, reimbursement and funding, time-to-market, and patient access and prescribing. Free-pricing between US healthcare providers resulted in varying prices for the same drug, whereas the UK-based NICE HTA outcomes achieved fixed-pricing for all patients. Likewise, due to the US competitive pricing environment, orphan drugs were priced considerably higher than in the UK. Both countries offered public funding opportunities, aiding treatment financing and access. However, considerably more orphan drugs received reimbursement in the US than the UK. Both countries offered early access, or fast-track approval, in cases tackling significant unmet need. Analysis using the four criteria identified that orphan drug prices in the US were considerably higher than the UK. Both countries offered accelerated or early access schemes and public funding, yet patient access was challenged due to reimbursement and funding limitations. Although market access is higher in the US, varying healthcare plan portfolios, and price variability, limits patient access. The UK reimburses fewer orphan drugs, but all UK patients have access to reimbursed treatments.