RESUMO
Purpose: To estimate the cost-effectiveness of Nefecon in addition to the best supportive care (BSC) vs BSC in a hypothetical cohort of commercially insured adult patients with primary immunoglobulin A nephropathy (IgAN) from a United States (US) societal perspective. Methods: A lifetime horizon, semi-Markov model was developed that consisted of nine health states: chronic kidney disease (CKD) stage 1, 2, 3a, 3b, 4, end-stage renal disease (ESRD) with dialysis, ESRD without dialysis, post-kidney transplant, and death. Health state occupancy was estimated from individual patient-level data from the Phase 3 randomized controlled trial NefIgArd Part A (NCT03643965). Additional scenarios evaluated the impact of varying the time horizon, discounting, costs included, rounds of treatment, and the method used to calculate transition probabilities. Results: In the deterministic base case analysis over a lifetime horizon, Nefecon plus BSC (hereafter Nefecon) had an incremental cost of $3,810 vs BSC. Nefecon resulted in a mean survival gain of 0.247 quality-adjusted life years (QALYs), 0.195 life years (LYs), and 0.244 equal value life years (evLYs) vs BSC alone - this resulted in incremental cost-effectiveness ratios (ICERs) of $15,428 per QALY, $19,502 per LY, and $15,611 per evLY gained. Probabilistic sensitivity analyses estimated that with willingness to pay thresholds of $100,000, $150,000, and $250,000 per QALY gained, Nefecon would be cost-effective over BSC in 66.70%, 75.02%, and 86.82% of cases, respectively. In the scenario analysis, Nefecon remained cost-effective with 4 rounds of treatment. Conclusion: Nefecon was associated with LY and QALY gains vs BSC, with an incremental cost of $3,810. Based on these values, with a willingness to pay threshold of $100,000 per QALY gained, Nefecon was found to be a cost-effective treatment for US adults with primary IgAN.
RESUMO
OBJECTIVE: The analysis estimates projected population outcomes resulting from the introduction of a plant-derived influenza vaccine formulated as quadrivalent virus-like particles (QVLP) in Canada. METHODS: Using Monte Carlo simulations, the number of influenza cases, general practitioner visits, inpatient admissions, intensive care unit (ICU) admissions, and deaths due to influenza-associated illness were estimated under no vaccination, plant-derived QVLP vaccines only, or egg-derived vaccines only. The base case analysis examined the adult Canadian population in two subgroups: 18-64 years of age during the 2017/18 season and 65+ years of age during the 2018/19 season. Efficacy data were obtained from QVLP clinical trials. Vaccine effectiveness data for egg-derived vaccines were calculated from observational studies from the corresponding influenza seasons. Scenario analyses examined the impact of varying absolute vaccine effectiveness or vaccination coverage from base case inputs. RESULTS: In the base case analysis, plant-derived QVLP vaccines led to an additional reduction in the burden of influenza over egg-derived vaccines for both population subgroups. In the 18-64 subgroup, QVLP vaccines were associated with 2.63% (48,029; 95% credible interval [Crl]: 42,723-53,336) fewer influenza cases than egg-derived vaccines. In the 65+ subgroup, QVLP vaccines led to 4.82% (27,918; 95% Crl: 25,440-30,397) fewer influenza cases, and reductions in the number of inpatient admissions by 4.77% (1167; 95% CrI: 851-1483) and deaths by 4.75% (326; 95% CrI: 107-546) compared to egg-derived vaccines. Further reductions were observed in scenario analyses considering the potential increase in vaccine coverage. CONCLUSION: Use of plant-derived QVLP influenza vaccines may contribute to greater reductions in influenza cases and influenza-related outcomes, including inpatient admissions and deaths, compared to egg-derived vaccines currently available in Canada.
