Monthly dosing with ranibizumab (RBZ) is needed to achieve maximal visual gains in patients with neovascular ('wet') age-related macular degeneration (wAMD). In Sweden, dosing is performed as needed (RBZ PRN), resulting in suboptimal efficacy. Intravitreal aflibercept (IVT-AFL) every 2 months after three initial monthly doses was clinically equivalent to RBZ monthly dosing (RBZ q4) in wAMD clinical trials. We assessed the cost-effectiveness of IVT-AFL versus RBZ q4 and RBZ PRN in Sweden.
A Markov model compared IVT-AFL to RBZ q4 or RBZ PRN over 2 years. Health states were based on visual acuity in better-seeing eye; a proportion discontinued treatment monthly or upon visual acuity
One barrier to economic evaluation alongside neonatal randomized controlled trials is the expense of collecting detailed patient resource information. To reduce this data collection burden, we identified the key resource items that predict daily ancillary costs for extremely low birth weight infants.
Participants were 385 infants enrolled in the Trial of Indomethacin Prophylaxis for Preterms in nine tertiary level neonatal intensive care units in Canada. Information on eighty-nine nonpersonnel resource items was abstracted from the hospital chart from admission to tertiary hospital discharge. Unit costs were derived from a provincially standardized cost accounting system. Using stepwise linear regression, models correlating total daily ancillary costs with key resource items were constructed for each of five periods of admission. Models were derived in a randomly split half of the total sample of patient days and validated against the remainder.
The 385 infants contributed resource information from 23,354 admission days. The regression model for weeks one to twelve included the covariates surfactant, chest radiograph, red blood cell transfusion, cranial ultrasound, abdominal radiograph, parenteral amino acid infusion, surgery, platelet transfusion, and echocardiogram and explained 91% of the variability in daily nonpersonnel costs (P
Colorectal cancer (CRC) is the third most commonly diagnosed cancer in Canada (excluding non-melanoma skin cancers). Bevacizumab is a recombinant humanized monoclonal antibody that selectively binds to human vascular endothelial growth factor. A sub-study confirmed its effectiveness in KRAS wild-type patients. Recent evidence has shown clinical benefit from anti-epidermal growth factor treatments cetuximab and panitumumab in these patients. The cost-effectiveness, to the Canadian healthcare system, of fluoropyrimidine-based chemotherapy (FBC) in combination with bevacizumab, cetuximab, or panitumumab was assessed for first-line treatment of KRAS wild-type mCRC patients.
A Markov model was developed and calibrated to progression-free/overall survival, using separately reported trial survival and adverse event results for each comparator. Health-state resource utilization was derived from published data and oncologist input. Utilities and unit prices were obtained from published literature and standard Canadian sources.
Results per patient are over a lifetime horizon, to a maximum of 10 years, with 5% annual discounting. Comparators are ordered by total cost and the incremental cost-effectiveness ratio (ICER) of each is determined against the previous non-dominated therapy. Compared to FBC alone, bevacizumab?+?FBC has an ICER of $131,600 per QALY gained. Compared to bevacizumab?+?FBC, panitumumab?+?FBC is dominated and cetuximab?+?FBC has an ICER of $3.8 million per QALY. In probabilistic sensitivity analysis, bevacizumab?+?FBC had ~100%, ~100%, and 98.9% probabilities of being more cost-effective than both of the other combination treatments at thresholds of $50,000/QALY, $100,000/QALY, and $200,000/QALY, respectively.
For first-line treatment of KRAS-WT mCRC, bevacizumab?+?FBC is associated with substantially lower costs as compared to panitumumab?+?FBC or cetuximab?+?FBC. Key limitations were that survival curves and adverse event rates were taken from separate clinical trials and that an indirect comparison was not included. Given these findings, bevacizumab is likely to offer the best value for money for this patient population.
To determine the incremental cost-effectiveness of indomethacin prophylaxis in extremely low birth weight infants enrolled in the Trial of Indomethacin Prophylaxis in Preterms (TIPP).
Participants in this economic evaluation were 428 infants enrolled at 9 Canadian TIPP centres. The study took a third-party payer perspective. Prior to the analysis of clinical trial data, direct medical costs were derived from chart review of 89 items of resource utilization, for each day from admission to hospital discharge. Unit costs for each resource were obtained from a provincially standardized cost-accounting system. Incremental cost-effectiveness analysis was performed, with estimation of cost-effectiveness acceptability curves through non-parametric bootstrapping.
The mean (SD) cost was $68,279 (40,317) for the placebo group and $69,629 (37,989) for the indomethacin group. Indomethacin prophylaxis cost an additional $67,500 per death or impairment averted. However, the precision of this estimate was low, such that the probability that the estimate was lower than $300,000 per death or impairment averted was only 61%. The results were similar when surgical costs were assumed to be 500% of those measured in the trial.
This study does not provide an economic rationale for the use of indomethacin prophylaxis in ELBW infants.