OBJECTIVES: Standard health technology assessments (HTA) processes for reimbursement of new drugs can be prohibitively cumbersome and costly for small to mid-size pharmaceutical and biotechnology companies, and generate onerous workloads for assessment of low budget impact products. However, in some markets, reimbursement is also possible through alternative/abbreviated-access-pathways (AAP). This study assesses 13 European markets for AAPs and summarizes their key differences and similarities.

METHODS: Publicly available reimbursement policy and regulatory documents and grey literature were reviewed for the following markets: Belgium, Denmark, Finland, Germany, Ireland, Italy, the Netherlands, Norway, Portugal, Scotland, Spain, Sweden, and the United Kingdom (UK). Pathways where manufacturers provide products free of charge were not assessed.

Identified guidance were assessed based on marketing authorization status, regional/national access, pricing rules, applicant (key opinion leader [KOL] versus manufacturer), evidence requirements, submission costs, target population size and unmet need.

RESULTS: Alternative/abbreviated-access-pathways (AAPs) were identified in 8 of the 13 markets. The majority of these were offered in small and mid-size markets. In most of these markets, AAPs were predominantly: only permissible post-marketing authorization, reimbursement applications were manufacturer-initiated versus KOL-led and decisions were made at a national level. Additionally, the costs of submissions (application and dossier development costs) and evidence burden for AAPs were usually lower compared to equivalent standard HTA processes and the timelines for the review process were often shorter. Economic evidence requirements were mostly limited to budget impact analyses versus often required cost-utility analyses which utilize and customarily require time-intensive and costly decision analytic models.

CONCLUSIONS: This analysis indicates that AAPs offer viable options for small and mid-size companies or treatments with low budget impact and may help to overcome challenges in regular HTA processes like increasing data requirements and longer time to access. However, such pathways are not available in all countries.