OBJECTIVES: Rare disease treatments are among the most frequently emerging therapies seeking market access. The Nordic countries Denmark, Finland, Norway, and Sweden have established access processes for pharmaceuticals. Rare disease treatments are characterized by a scarcity of robust clinical data and high costs. This presents a challenge for health technology assessment. To identify challenges and opportunities in access processes, policy, and funding for rare disease treatments as compared to non-rare disease treatments we analyzed country-specific pharmaceutical access procedures.

METHODS: Analyses were based on 1) a literature review of scientific publications and publicly available reports and 2) semi-structured interviews of 1–2 experts per country working with rare disease treatment access in the industry or regulatory bodies. The interview questionnaire assessed themes within health technology assessment, pricing and reimbursement, access initiatives, and future developments in rare disease treatment access.

RESULTS: Results indicated that the Nordic countries have similarities in access procedures and requirements for assessing clinical and health-economic outcomes of pharmaceuticals. None of the countries formally give rare disease treatments special consideration in health technology assessment, but case-by-case consideration may be applied. Depending on the country, funding decisions are made at a national or regional level. The industry collaborates with stakeholders in access initiatives for rare disease treatments at the European, Nordic, and national levels. However, future advancements in access to rare disease treatments remain unclear.

CONCLUSIONS: The Nordic countries are an interesting market to explore for launching rare disease treatments. This is highlighted by similar access requirements for pharmaceuticals, a strengthening of cross-country collaboration, and a willingness to develop new access solutions to rare disease treatments. For instance, innovative pricing schemes have been developed to tackle high treatment costs and lack of robust clinical data.