OBJECTIVES: The purpose of this study was to analyze the relationship between costly early access program (EAP) treatments (i.e., products made available under articles 71b-71c) and current list prices.

METHODS: A 2020 report by Kägi et al. ¹ was used to identify the 10 most expensive EAP treatments in 2017 and 2018. Information about dosing regimen was then collected, with treatments with off-label use (i.e., articles 71a) and/or variable dosing removed from consideration. After calculating the annual doses of treatments with uniform doses, the expected annual cost based on the current list price was calculated. These figures were then compared to reported EAP annual costs.

RESULTS: 11 of the 20 patients examined had fixed-dose treatments, 4 of whom used off-label treatments. Of the remaining 7 fixed-dose patients, 3 unique treatments were identified, the most common being Orkambi (lumacaftor/ivacaftor). The average magnitude of difference between EAP and list price costs was found to be 19% (range: 2-40%). However, roughly equal numbers of list price costs were more expensive than and cheaper than EAP costs (4 treatments more expensive, 3 cheaper). Of note is the fact that there were variations in reported annual treatment costs for the 5 patients using Orkambi, despite all treatments requiring an identical fixed dose.

CONCLUSIONS: The results of this analysis were inconclusive due to the limited sample size. However, several valuable observations were still made, including the variance in costs for identical treatment with Orkambi, and the equal numbers of more expensive and cheaper list prices compared with EAP prices. EAP product pricing requires a clear strategy to ensure the EAP price does not negatively affect the future negotiated commercial price.