Indirect treatment comparison and cost-minimization analysis of riociguat versus selexipag in patients with pulmonary arterial hypertension.

OBJECTIVES: The comparative efficacy between riociguat and selexipag in patients with pulmonary arterial hypertension (PAH) has never been described in literature. Our aim was to prepare indirect treatment comparison (ITC) to evaluate the cost-effectiveness of riociguat in Czechia. METHODS: A systematic literature review identified two relevant trials with comparable endpoints to inform a Bucher ITC of relative and absolute effects. Given the comparable efficacy of riociguat and selexipag, a cost-minimization analysis (CMA) was conducted. RESULTS: A Bucher ITC provided evidence for the comparable relative efficacy of riociguat defined as the odds of unimproved functional class III 0.761 (95% CI 0.372 to 1.558; p = 0.455) compared to selexipag and a comparable absolute efficacy defined as a difference in the 6-minute walking distance of 10.560 meters (95% CI -10.692 to 31.812; p = 0.330). The CMA identified riociguat as the cost-saving therapy. CONCLUSIONS: Switching to riociguat represents the cost-saving therapy for PAH patients who were inadequately compensated with the PDE5i+ERA therapy. Consequently, riociguat has been introduced to the list of reimbursed medicines in Czechia from October 2021. Based on two global trials, we prepared the first indirect treatment comparison followed with CMA of these therapies that may improve future decision-making for PAH indications.

Highly Innovative Drug Program in the Czech Republic: Description and Pharmacoeconomic Results-Cost-Effectiveness and Budget Impact Analyses.

BACKGROUND: Highly innovative drugs (HIDs) can be granted 2 to 3 years of temporary reimbursement (TR) to provide timely patient access and to collect real-world evidence through registries in the Czech Republic. A TR applicant does not need to comply with cost-effectiveness (CE) requirements and the willingness-to-pay threshold. It is only when mandatory transition to permanent reimbursement (PR) status occurs does the drug need to comply with CE and willingness-to-pay requirements. OBJECTIVES: To describe and evaluate the HID program in the Czech Republic by analyzing the pharmacoeconomic results when a drug starts with TR status and transitions to PR status. METHODS: The study was a retrospective analysis of reimbursement decisions of HIDs. All drugs approved for TR (valid from January 2008 to January 2018) were identified. A description of the HIDs and their pharmacoeconomic results were analyzed. RESULTS: Fifty TR drugs were identified. Most (68%) were oncology drugs and 44% were orphan drugs. After the expiration of their TR status, 83% were successfully transitioned to PR status. Cost-utility analysis was used to support CE results in 42% of the TR drugs. The mean incremental cost-effectiveness ratio (cost/quality-adjusted life-year) of drugs that entered TR status was €97,868. When the time came for transition to PR status, the mean incremental cost-effectiveness ratio was €34,086 (lower by 65%). Net budget impact increased by 3% and decreased by 25% in the first and fifth years, respectively, after applying for PR. CONCLUSIONS: This analysis provides better insight into the HID program for costly innovative drugs over a 10-year follow-up. A successful transition to PR status was observed for most of the HIDs (83%).