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Context The cost-effectiveness of 13-valent pneumococcal conjugate vaccine (PCV13) compared with 23-valent pneumococcal polysaccharide vaccine (PPSV23) among US adults is unclear. Objective To estimate the cost-effectiveness of PCV13 vaccination strategies in adults to assist vaccination policy decision-making. Design, Setting, and Population A Markov state-transition model, lifetime time horizon, societal perspective. Simulations were performed in hypothetical cohorts of US 50-year-olds. Vaccination strategies and effectiveness estimates were developed by a Delphi expert panel; indirect (herd immunity) effects resulting from childhood PCV13 vaccination were extrapolated based on observed PCV7 effects. Data sources for model parameters included CDC Active Bacterial Core surveillance, National Hospital Discharge Survey and Nationwide Inpatient Sample data, and the National Health Interview Survey. Main Outcome Measures Pneumococcal disease cases prevented and incremental costs per quality-adjusted life year (QALY) gained. Results In the base case scenario, PCV13 given as a substitute for PPSV23 in current recommendations (i.e., vaccination at 65 years and at younger ages if comorbidities are present) cost $28,900/QALY gained compared with no vaccination and was more cost-effective than the currently recommended PPSV23 strategy. Routine PCV13 at ages 50 and 65 years cost $45,100/QALY compared with PCV13 substituted in current recommendations. Adding PPSV23 at age 75 to PCV13 at ages 50 and 65 years gained 0.00002 QALYs, costing $496,000/QALY gained. Results were robust in sensitivity analyses and alternative scenarios, except when low PCV13 effectiveness against nonbacteremic pneumococcal pneumonia was assumed or when greater childhood vaccination indirect effects were modeled. In these cases, PPSV23 as currently recommended was favored. Conclusions Overall, PCV13 vaccination was favored compared to PPSV23, but the analysis is sensitive to assumptions about PCV13 effectiveness against NPP and the magnitude of potential indirect effects from childhood PCV13 on pneumococcal serotype distribution.

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The cost-effectiveness of the varicella zoster vaccine varies substantially with patient age and often exceeds $100,000 per quality-adjusted life year saved. Age should be considered in vaccine recommendations.

Introduction:Molecular testing of fine-needle aspiration (FNA) results helps diagnose thyroid cancer, although the additional cost of this adjunct has not been studied. We hypothesized that FNA molecular testing of two indeterminate categories (follicular lesion of undetermined significance and follicular/Hürthle cell neoplasm) can be cost saving.Methods:For a hypothetical group of euthyroid patients with a 1-cm or larger solitary thyroid nodule, a decision-tree model was constructed to compare the estimated costs of initial evaluation according to the current American Thyroid Association guidelines, either with molecular testing (MT) or without [standard of care (StC)]. Model endpoints were either benign FNA results or definitive histological diagnosis.Results:Molecular testing added $104 per patient to the overall cost of nodule evaluation (StC $578 vs. MT $682). In this distributed cost model, MT was associated with a decrease in the number of diagnostic lobectomies (9.7% vs. StC 11.6%), whereas initial total thyroidectomy was more frequent (18.2% vs. StC 16.1%). Although MT use added a diagnostic cost of $5031 to each additional indicated total thyroidectomy ($11,383), the cumulative cost was still less than the comparable cost of performing lobectomy ($7684) followed by completion thyroidectomy ($11,954) in the StC pathway, when indicated by histological results. In sensitivity analysis, savings were demonstrated if molecular testing cost was less than $870.Conclusions:Molecular testing of cytologically indeterminate FNA results is cost saving predominantly because of reduction in two-stage thyroidectomy. Appropriate use of emerging molecular testing techniques may thus help optimize patient care, improve resource use, and avoid unnecessary operation.

Gene therapy provides a potential phenotypic cure for hemophilia, yet the cost of this novel treatment is high, tempering enthusiasm and raising questions regarding cost vs benefit. To evaluate the cost-effectiveness of gene therapy treatment of severe hemophilia A compared with prophylaxis with factor VIII (FVIII), we developed a Markov state-transition model to estimate the costs and effectiveness of severe hemophilia A treatment strategies from a United States health care system perspective. Quality-adjusted life-years (QALYs) were the effectiveness measure. In the base case, hypothetical cohorts of 30-year-old patients received gene therapy or FVIII prophylaxis. We obtained model probabilities and utilities from the literature and costs from Medicare reimbursement data. One-way and probabilistic sensitivity analyses were performed to test the robustness of results. Over a 10-year time horizon, total per-person gene therapy strategy costs were $1.0M and resulted in 8.33 QALYs, whereas prophylaxis cost $1.7M and resulted in 6.62 QALYs. Thus, gene therapy dominated prophylaxis (costs less and was more effective). Gene therapy remained dominant unless initial costs exceeded $1.6M and were <$100 000 per 1 QALY gained compared with prophylaxis if initial costs were <$1.7M. Results were not sensitive to variation of all other parameters over clinically plausible ranges. In a probabilistic sensitivity analysis simultaneously varying all parameters 3000 times over parameter distributions, gene therapy was dominant in 92% of model iterations. Treatment of severe hemophilia A with gene therapy is likely to be cost-saving or cost-effective compared with FVIII prophylaxis.

Clostridium difficile infection (CDI) is costly. Current guidelines recommend metronidazole as first-line therapy and vancomycin as an alternative. Recurrence is common. Faecal microbiota transplantation (FMT) is an effective therapy for recurrent CDI (RCDI). This study explores the cost-effectiveness of FMT, vancomycin and metronidazole for initial CDI. We constructed a decision-analytic computer simulation using inputs from published literature to compare FMT with a 10-14-day course of oral metronidazole or vancomycin for initial CDI. Parameters included cure rates (baseline value (range)) for metronidazole (80% (65-85%)), vancomycin (90% (88-92%)) and FMT(91% (83-100%)). Direct costs of metronidazole, vancomycin and FMT, adjusted to 2011 dollars, were $57 ($43-72), $1347 ($1195-1499) and $1086 ($815-1358), respectively. Our effectiveness measure was quality-adjusted life years (QALYs). One-way and probabilistic sensitivity analyses were conducted from the third-party payer perspective. Analysis using baseline values showed that FMT($1669, 0.242 QALYs) dominated (i.e. was less costly and more effective) vancomycin ($1890, 0.241 QALYs). FMT was more costly and more effective than metronidazole ($1167, 0.238 QALYs), yielding an incremental cost-effectiveness ratio (ICER) of $124 964/QALY. One-way sensitivity analyses showed that metronidazole dominated both strategies if its probability of cure were >90%; FMT dominated if it cost <$584. In a probabilistic sensitivity analysis at a willingness-to-pay threshold of $100 000/QALY, metronidazole was favoured in 55% of model iterations; FMT was favoured in 38%. Metronidazole, as the first-line treatment for CDIs, is less costly. FMT and vancomycin are more effective. However, FMT is less likely to be economically favourable, and vancomycin is unlikely to be favourable as first-line therapy when compared with FMT.

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