OBJECTIVES: This study aims to investigate the willingness to pay for orphan drugs assessed by TLV for reimbursement 2015-2022.

METHODS: A report recently published by TLV includes a list of 61 orphan prescription drugs within the reimbursement scheme 2015-2022. As available, published decisions and decision reports, were retrieved from the TLV website. Data were extracted on the TLV base-case ICER, QALYs, assessed indication, population size, disease severity, decision outcome and whether the product had a three-party negotiated price.

RESULTS: Out of 61 prescription drugs, 50 products had reports available on the TLV website and 11 did not. This could be explained by the possibility for companies to withdraw applications. A total of 68 decisions were retrieved since some products had multiple decisions 9 decisions were assessed with a cost minimization analysis and were excluded from further analysis. 47 approvals by TLV were found with ICERs (TLV base case) in the range SEK 40,000 to 1,450,000. For the rejections the range was SEK 1 to 12.8 million. QALYs gained were reported for 15 approved drugs and 6 rejected, ranging between 0.36 to 4.16, and 1.7 to 5.49, respectively. The disease severity ranged from medium to very high, 39 of the approved cases had a high or very high severity. In the rejected group, severity was high or very high in cases 15 (4 not reported). 26 decisions had a restriction to a smaller patient population within the indication and/or a follow up request. 9 decisions included had a three-party negotiated net price in the TLV base case ICER.

CONCLUSIONS: The results from this study indicate that there are few positive reimbursement decisions for orphan drugs with a high disease severity were TLV has accepted an ICER above 1 mSEK.