BackgroundDespite clearly improved clinical outcomes for prophylaxis compared to on-demand therapy, on average only 56% of patients diagnosed with severe hemophilia receive prophylactic factor replacement therapy in the US. Prophylaxis rates generally drop as patients transition from childhood to adulthood, partly due to patients becoming less adherent when they reach adulthood. Assessment of patient preferences is important because these are likely to translate into increased treatment satisfaction and adherence. In this study, we assessed preferences and willingness to pay (WTP) for on-demand, prophylaxis, and longer acting prophylaxis therapies in a sample of US hemophilia patients.MethodsAdult US hemophilia patients and caregivers (N=79) completed a discrete-choice survey that presented a series of trade-off questions, each including a pair of hypothetical treatment profiles. Using a mixed logit model for analysis, we compared the relative importance of five treatment characteristics: 1) out-of-pocket treatment costs (paid by patients), 2) factor dose adjustment, 3) treatment side effects, 4) availability of premixed factor, and 5) treatment effectiveness and dosing frequency. Based on these attribute estimates, we calculated patients’ WTP.ResultsOut-of-pocket treatment costs (P<0.001), side effects (P<0.001), and treatment effectiveness and dosing frequency (P<0.001) were found to be statistically significant in the model. Patients were willing to pay US $410 (95% confidence interval: $164–$656) out of pocket per month for thrice-weekly prophylaxis therapy compared to on-demand therapy and $360 (95% confidence interval: $145–$575) for a switch from thrice-weekly to once-weekly prophylaxis therapy.ConclusionImprovements in treatment effectiveness and dosing frequency, treatment side effects, and out-of-pocket costs per month were the greatest determinants of hemophilia treatment choice and WTP. The positive preferences and WTP for longer acting prophylactic therapies suggest that the uptake is likely to increase adherence, improving treatment outcomes. These preferences should also inform the Food and Drug Administration’s assessment of new longer acting hemophilia therapies.

The results of this analysis demonstrate that G5 Mobile CGM is cost-effective within the population of adults with T1DM using MDI, assuming a Canadian willingness-to-pay threshold of $50,000 CAD per QALY.

Estimates of preference-weighted health outcomes or health state utilities are needed to assess improvements in health in terms of quality-adjusted life-years. Gains in quality-adjusted life-years are used to assess the cost–effectiveness of prophylactic use of clotting factor compared with on-demand treatment among people with hemophilia, a congenital bleeding disorder. Published estimates of health utilities for people with hemophilia vary, contributing to uncertainty in the estimates of cost–effectiveness of prophylaxis. Challenges in estimating utility weights for the purpose of evaluating hemophilia treatment include selection bias in observational data, difficulty in adjusting for predictors of health-related quality of life and lack of preference-based data comparing adults with lifetime or primary prophylaxis versus no prophylaxis living within the same country and healthcare system.

To assess the economic impact of providing real time continuous glucose monitoring (CGM) for people with type 1 diabetes (T1D) and impaired awareness of hypoglycaemia (IAH) within North West (NW) London clinical commissioning groups (CCGs). Methods: The eligible population for CGM and inputs for the economic budget impact model developed were derived from published data. The model includes cost of CGM; cost savings associated with lower hypoglycaemia related hospital admissions, accidents and emergency visits; self-monitoring of blood glucose (SMBG) strip usage; and glycated haemoglobin (HbA1c) reduction-related avoided complications and insulin pump use. Results: The cost of CGM for T1D-IAH (n=3,036) in the first year is £10,770,671 and in the fourth year is £11,329,095. The combined cost off-sets related to reduced hypoglycaemia admissions, SMBG strip usage and complications are £8,116,912 and £8,741,026 in years one and four, respectively. The net budget impact within the NW London CCGs is £2,653,760; £2,588,068 in years one and four respectively. Conclusions: Introduction of CGM for T1D-IAH patients will have a minimal budget impact on NW London CCGs, driven by cost of CGM and offsets from lower hypoglycaemia-related costs, reduced SMBG strip usage, avoided HbA1c-related complications and lower insulin pump use.

Renal cell carcinoma (RCC) is the most common renal tumor and accounts for 2 to 3% of all adult malignancies. Up to 30% of patients with renal-cell carcinoma present with metastatic disease. The aim of the study was to assess the cost-effectiveness of first-line medications for patients with metastatic RCC from the payer's perspective. METHODS: A Markov model was developed using 6-week cycles for evaluating the cost-effectiveness of four mRCC first-line therapies: Interferon-␣ (3 million units (MU)/day 3 times-per-week for first week; 6MU/day 3 timesper-week for second week, 9MU/day 3 times per-week third week onward), sunitinib (50mg/day 4 weeks ON ϩ 2 weeks OFF), sorafenib (400mg/twice-a-day) and bevacizumab plus Interferon ␣ (10mg/kg/bi-weekly ϩ 9MU 3 times-per-week every 3 weeks). The model had a 10-year time horizon. The four model stages were: No progression, progression, best supportive care and death. Effectiveness data were obtained through a systematic review, resource utilization were collected from a Colombian Delphi Panel, and costs included were retrieved from Colombian tariff manual (SOAT). Effectiveness was measured by progression-free life years (PFLY) and life-years gained (LYG). Costs and outcomes were discounted with a 3% annual rate. Univariate sensitivity analysis was developed.