WS01.05Time to reimbursement for elexacaftor/tezacaftor/ivacaftor across countries in the ECFS patient registry

Abstract

Objectives

Due to its high price, elexacaftor/tezacaftor/ivacaftor (ETI) is inaccessible to patients unless reimbursed by national health bodies. Such reimbursement deals are typically negotiated with the manufacturer on an individual country basis, resulting in delays and disparities in treatment access. Accordingly, we analysed timing disparities in ETI reimbursement across countries participating in the European Cystic Fibrosis Society Patient Registry (ECFSPR), examining relationships with health outcomes and national economic indicators.

Methods

Time to reimbursement (TTR) from initial regulatory approval was determined for countries within the ECFSPR using manufacturer and patient organisation press releases, national drug registries and stakeholder correspondence. Initial authorisation dates were obtained from medicines regulators. Associations between TTR, CF health outcomes and gross national income (GNI) were appraised using Spearman's Rank correlation coefficient.

Results

Of 40 countries in the ECFSPR, 31 had agreed reimbursement for ETI. Median TTR was 343 days (IQR=183.5-572.5, range 0-1,502 days) and displayed a highly positively skewed distribution (skewness=1.167, mean=434 days). Significant negative associations were found between TTR and the number of registered patients (r_s=-0.419, p<0.05) and GNI (r_s=-0.515, p<0.05). No significant associations were found between TTR and ppFEV₁, Pseudomonas aeruginosa colonisation or %F508del homozygosity/heterozygosity.

Conclusion

Time to ETI reimbursement varied significantly across the ECFSPR, demonstrating a grouping of early reimbursement deals followed by prolonged negotiations in predominantly poorer countries. Lower GNI and smaller CF populations were both associated with a longer TTR. Following the recent US authorisation of vanzacaftor/tezacaftor/deutivacaftor, these findings highlight important considerations for equitable access to existing and emerging CFTR modulator therapies internationally.