OBJECTIVES

Cystic fibrosis conductance regulator (CFTR) gene modulators, ivacaftor, lumacaftor/ivacaftor and tezacaftor/ivacaftor, have substantially improved patients’ lives yet significantly burden healthcare budgets. This analysis aims to compare pricing and reimbursement of aforementioned cystic fibrosis medicines, developed by Vertex Pharmaceuticals, Inc., across European countries.

METHODS

Clinical trial registries, national databases, health technology assessment reports and grey literature of Belgium, the Netherlands, Ireland, the United Kingdom, France, Germany, Denmark, Sweden, Switzerland, Austria, Spain and Poland were consulted. Publicly available efficacy results, prices, reimbursement statuses, economic evaluations, budget impacts and managed entry agreements of CFTR modulators were reported.

RESULTS

All interventions improved lung clearance and expiratory volume. Lowest prices were captured in Switzerland at €426 per DDD, €307 per DDD and €186 per ADD for Kalydeco®, Orkambi® and Symkevi®, respectively. Spain had the highest prices for Kalydeco® and Symkevi® at €851 per DDD and €337 per ADD, whereas Orkambi® was most expensive in Poland at €983 per DDD. In all countries, treatments were deemed not to be cost-effective. The annual budget impact of these CFTR gene modulators varied between countries and depended on factors such as local product prices, size of target population, scope of costs and discounting. However, all modulators were fully reimbursed in majority of countries except for France, Germany, Sweden and Poland that decided on partial or no reimbursement. Managed entry agreements were confidential but commonly adopted to address financial uncertainties.

CONCLUSIONS

Discrepancies in reimbursement decisions and prices for Kalydeco®, Orkambi® and Symkevi® across European countries were detected. More transparency and increased country collaboration might stimulate uniform patient access to these cystic fibrosis medicines.