Entry to market of new medicines and medicines treating rare diseases: issues arising from value assessment processes in the European Union, the United Kingdom and Canada

To achieve access to new medicines within markets, manufacturers need to first receive marketing authorisation and subsequently seek funding from healthcare insurance. In countries where access to healthcare is free, the allocation of finite resources poses substantial challenges. Increasingly in these settings, health technology assessment (HTA) is used to inform funding decisions whilst seeking to promote healthcare financial sustainability and macro- and micro-economic efficiency. Variations in access to medicines can occur as countries implement HTA differently. These variations are further highlighted in medicines used to treat rare diseases, known as orphan medicines. Due to their high prices and high uncertainty about their clinical benefit, some HTA bodies have specialised assessment frameworks for orphan medicines to safeguard equity by considering additional dimensions of value beyond clinical- and/or cost-effectiveness. In this thesis, I explored how differences in HTA systems and processes may contribute to access variations of new medicines and medicines for rare diseases across settings. First, I outlined a conceptual framework that showed how HTA is operationalised; I also mapped HTA systems across 32 countries. Second, through a Delphi panel of European stakeholders, I identified features of HTA that facilitate access to new medicines. Third, I observed whether the presence of specialised assessment frameworks might translate to more favourable funding recommendations for and timely access to orphan medicines by comparing two settings where these medicines are treated differently. Finally, I evaluated whether HTA recommendations are aligned with funding decisions for orphan medicines in a decentralised healthcare setting where the HTA body has an advisory role. The main contributions of this thesis are fivefold: (i) it develops a conceptual framework that allows comparisons of HTA systems regardless of how well-developed they are; (ii) it generates evidence on the performance of HTA features, looking at HTA holistically, against different access metrics; (iii) it examines whether efforts to optimise access to orphan medicines across the market access pathway may translate into more favourable reimbursement decisions; (iv) it studies whether HTA recommendations are followed in funding decisions; and (v) it provides recommendations on what features of HTA need improvement to optimise patient access.

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