IMPLICATIONS OF A NEWLY INTRODUCED RARITY-BASED PRICING FRAMEWORK IN SWEDEN: A REVIEW OF ORPHAN MEDICINE REIMBURSEMENT DECISIONS IN SCANDINAVIA
Author(s)
Viktória Molnár, MSc, Martina Yngvesson, MSc, Charlotte Linnea Möller-Madsen, MSc, Kajsa Stina Olsson, BA, MSc.
Nordic Market Access NMA AB, Stockholm, Sweden.
Nordic Market Access NMA AB, Stockholm, Sweden.
OBJECTIVES: Healthcare systems in the Scandinavian countries follow ethical principles of equal access to care and prioritisation based on patients' needs. However, Sweden and Norway are below, and Denmark is on par with the EU27 average orphan medicine rate of availability according to the 2026 WAIT report. In 2023, the Swedish Dental and Pharmaceutical Benefits Agency (TLV) proposed a new ‘staircase model’, suggesting a higher acceptable incremental cost-effectiveness ratio (ICER) for medicines treating very rare, severe diseases. From January 2025, a rarity index was gradually introduced, combining incidence and prevalence to guide ICER thresholds, improving access to orphan medicines. Our objective was to explore the use of the rarity index in Sweden and compare the reimbursement status of these products in Norway and Denmark.
METHODS: A search was conducted in the NMAi database, collating HTA documents from Sweden, Norway, and Denmark. We identified orphan medicine HTA decisions between 2024-01-01-2026-06-15 using the search terms rarity index, increased willingness-to-pay (WTP), rare OR orphan, limiting to Swedish documents. The reimbursement status, ICER levels, and population size were extracted. To compare the reimbursement status in the other Scandinavian countries, decisions for the same medicines from Norway and Denmark were collected.
RESULTS: The TLV search identified 14 unique medicines (24 assessments, including different indications). One was excluded as its orphan designation was removed. 11/13 medicines received positive recommendations (13/24 decisions). Time from EMA approval to decision averaged 5 years (range 1-12.2 years). Published ICERs after tripartite consultations ranged between 2-3.5 million SEK. In Norway 12/13 medicines were assessed, 5 recommended. In Denmark, 8/13 have been assessed, and 5 recommended.
CONCLUSIONS: The rarity index offers a structured approach to align societal WTP with severe unmet needs. The analysis suggests higher WTP in Sweden compared to previous research. Denmark had the fewest of the identified medicines recommended.
METHODS: A search was conducted in the NMAi database, collating HTA documents from Sweden, Norway, and Denmark. We identified orphan medicine HTA decisions between 2024-01-01-2026-06-15 using the search terms rarity index, increased willingness-to-pay (WTP), rare OR orphan, limiting to Swedish documents. The reimbursement status, ICER levels, and population size were extracted. To compare the reimbursement status in the other Scandinavian countries, decisions for the same medicines from Norway and Denmark were collected.
RESULTS: The TLV search identified 14 unique medicines (24 assessments, including different indications). One was excluded as its orphan designation was removed. 11/13 medicines received positive recommendations (13/24 decisions). Time from EMA approval to decision averaged 5 years (range 1-12.2 years). Published ICERs after tripartite consultations ranged between 2-3.5 million SEK. In Norway 12/13 medicines were assessed, 5 recommended. In Denmark, 8/13 have been assessed, and 5 recommended.
CONCLUSIONS: The rarity index offers a structured approach to align societal WTP with severe unmet needs. The analysis suggests higher WTP in Sweden compared to previous research. Denmark had the fewest of the identified medicines recommended.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA200
Topic
Economic Evaluation, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases