OBJECTIVES: Reimbursement of orphan drugs continues to pose significant challenges for decision-makers given frequent high costs and limited evidence. Historically, the willingness to pay (WTP) for new treatments appraised by the Swedish Dental and Pharmaceutical Benefits Agency (TLV) has been influenced by disease severity and uncertainty in the documentation but not by the rarity of the condition. However, in 2016, TLV for the first time accepted a higher cost per quality-adjusted life-year gained due to the rarity of the targeted condition. This study aims to explore whether the rarity of the conditions has impacted incremental cost-effectiveness ratios (ICERs) accepted by TLV from 2017 through 2022.
METHODS: Data were extracted from all TLV decisions on reimbursement applications of medicinal products from 2017 through 2022 that had orphan drug status according to European Union regulations at the time of TLV evaluation and included a TLV ICER as a basis for decision. Extracted variables included TLV ICERs, disease severity, uncertainty in the health economic analysis, and reimbursement outcome. Information on the number of patients with the disease conditions were extracted from the European Medicines Agency’s orphan drug designation decisions.
RESULTS: 22 positive TLV decisions met the inclusion criteria. The estimated mean number of patients with each condition in Sweden was 1,842 (range, 105-4,209). The mean TLV ICER was 753,628 SEK (range, 96,784-1,450,696 SEK), and only two decisions had ICERs above the previous unofficial WTP threshold for very high disease severity of 1,000,000 SEK, not accounting for the rarity of the condition.
CONCLUSIONS: Although the decision made by TLV in 2016 opened rarity for consideration in determining acceptable ICERs, the current review suggests that there has not been any clear shift in accepted ICERs as a function of rarity.
