Incremental Cost-effectiveness Threshold Research Articles

Cost-Effectiveness Rivaroxaban versus Enoxaparin for Prevention of Venous Thromboembolism after Knee Replacement Surgery in Iran.

The highest risk of developing venous thromboembolism (VTE) is seen in patients who have undergone orthopedic surgery. One of the most common methods to reduce the risk of thromboembolism in these patients is anticoagulant prophylaxis. Rivaroxaban is one of the anticoagulants that has a lower cost than other anticoagulants and has a significant effect on people's quality of life as it is edible. The study aimed to determine the cost-effectiveness of rivaroxaban as compared with enoxaparin for venous thromboembolism prophylaxis in knee replacement patients in Iran. It was a quantitative and economic evaluation study with a cost-effectiveness approach and an applied study because its results could be used directly for policy-making and decision-making in the health system. The study was conducted in 2019 and 2020. This study considered the health system perspective. The study population included all knee replacement patients. The sample included 203 patients referred to Shafa Yahyaeian Hospital and 300 patients referred to Rasoul Hospital in Tehran. The study was conducted in two steps. A systematic review of studies was conducted in the first step. The CHEERS checklist was used to evaluate the quality of the studies in the systematic review. The EQ-5D questionnaire was used in the second step to calculate the QALY, and the cost collection form was used to calculate the direct medical cost. The data were analyzed through a decision tree, and Stata and Tree age pro softwares were the analysis tools. Also, according to the per capita GDP index for Iran in 2018, the incremental cost-effectiveness threshold was considered to be $10,000. The results of this study showed that during the prophylaxis period, rivaroxaban was one and a half times less costly than enoxaparin. Quality of life in uncomplicated conditions were 0.85 QALY for rivaroxaban and 0.69 QALY for enoxaparin. Based on the results of this study, the cost of rivaroxaban during the prophylaxis was $ 160.97 and the quality of life was 0.85 QALY and the cost of enoxaparin was $ 276.07 and the quality of life was 0.69 QALY. The cost difference between the two interventions was $ 115.09 and the outcome difference was 0.16 QALY. The incremental cost-effectiveness ratio was $ 189.40 for rivaroxaban and $ 416.28 for enoxaparin. According to the results of this study, rivaroxaban reduced the duration of hospitalization by an average of 2 days in asymptomatic patients (prophylaxis period) compared to enoxaparin. Rivaroxaban, an oral medication, reduced costs and increased the quality of life in people undergoing knee replacement surgery compared with an enoxaparin injection vial. This drug was less costly for the patient and health systems and its use was cost-effective as a thromboprophylaxis drug following knee replacement surgery.

Estimating the range of incremental cost-effectiveness thresholds for healthcare based on willingness to pay and GDP per capita: A systematic review.

BackgroundDecision-making in healthcare policy involves assessing both costs and benefits. In determining the cost-effectiveness (CE) threshold, willingness to pay (WTP) per quality-adjusted life year (QALY), GDP per capita, and other factors are important. However, the relationship between WTP/QALY or GDP per capita and the CE threshold is unclear. It is important to clarify the relationship between WTP/QALY and GDP to provide a clear basis for setting the CE threshold.ObjectiveThe purpose of this study was to compare WTP/QALY and GDP per capita, and to develop a new CE threshold range based on WTP using GDP per capita. The relationship between WTP/QALY and healthy life expectancy (HALE) was also investigated.MethodsWe searched MEDLINE, EMBASE and Web of Science from 1980/01/01 to 2020/12/31 using the following selection criteria (latest search: Dec 2021):1, studies that estimated WTP/QALY; 2, the general population was surveyed; 3, the article was in English. From the collected articles, we obtained average values of WTP/QALY for various countries and compared WTP/QALY with GDP per capita. The correlation between WTP/QALY and HALE was also examined.ResultsWe identified 20 papers from 17 countries. Comparison of mean WTP/QALY values with GDP per capita showed that most WTP/QALY values were in the range of 0.5–1.5 times GDP per capita, though the median values were less than 0.5 times. Comparison of WTP/QALY with HALE showed a statistically significant positive correlation when Taiwan was excluded as an outlier.ConclusionsOur results suggest a CE threshold range of 0.5–1.5 times GDP per capita is appropriate but lower than the WHO-recommended range of 1–3 times. The correlation between WTP/QALY and HALE suggests that investment in healthcare is reflected in an increased healthy life expectancy. Since WTP is based on consumer preferences, this range could be used to set a generally acceptable criterion.

Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries.

Background: Nusinersen is an orphan drug intended for the treatment of spinal muscular atrophy (SMA), a severe genetic neuromuscular disorder. Considering the very high costs of orphan drugs and the expected market entry of cell and gene therapies, there is increased interest in the use of health technology assessment (HTA) for orphan drugs. This study explores the role of the economic evaluation and budget impact analysis on the reimbursement of nusinersen. Methods: Appraisal reports for nusinersen were retrieved from reimbursement and HTA agencies in Belgium, Canada, France, England and Wales, Germany, Italy, Ireland, Scotland, Sweden, the Netherlands, and the United States. Detailed information was extracted on the economic evaluation, the budget impact, the overall reimbursement decision, and the managed entry agreement (MEA). Costs were adjusted for inflation and currency. Results: Overall, the reports included limited data on budget impact, excluding information on the sources of data for cost and patient estimates. Only three jurisdictions reported on total budget impact, estimated between 30 and 40 million euros per year. For early-onset SMA, the incremental cost-effectiveness threshold (ICER) ranged from €464,891 to €6,399,097 per quality-adjusted life year (QALY) gained for nusinersen versus standard of care. For later-onset SMA, the ICER varied from €493,756 to €10,611,936 per QALY. Although none of the jurisdictions found nusinersen to be cost-effective, reimbursement was granted in each jurisdiction. Remarkably, only four reports included arguments in favor of reimbursement. However, the majority of the jurisdictions set up an MEA, which may have promoted a positive reimbursement decision. Conclusion: There is a need for more transparency on the appraisal process and conditions included in the MEA. Additionally, by considering all relevant criteria explicitly during the appraisal process, decision-makers are in a better position to justify their allocation of funds among the rising number of orphan drugs that are coming to the market in the near future.

Establishing a reasonable price for an orphan drug

BackgroundThis paper addresses the question of what a reasonable price for an orphan drug is. The research proposes a way to adjust an established payer/HTA body incremental cost-effectiveness threshold (CET) to take account of differences in patient populations and costs of research and development in order to sustain prices that generate rates of return from investments in developing orphan drugs that are no greater than the industry average.MethodsWe investigated the cost of conducting research for orphan drugs as compared to non-orphan drugs, as well as patient population sizes targeted by orphans and non-orphans. We provided an empirical illustration based on novel drug approvals of orphan and non-orphan drugs of the FDA between 2011 and 2015 (N = 182).ResultsUsing, for illustration, the NICE incremental CET (£20 K per QALY) as an anchor and adjusting by R&D costs and expected market revenue, we estimated the adjusted reasonable CET for orphan drugs to be £39.1 K per QALY at the orphan population cut-off and £78.3 K per QALY at the orphan population mid-point. For ultra-orphan drugs the adjusted CET was £937.1 K.ConclusionsWe propose one general method for establishing a reasonable price for an orphan drug, based on the proposition that rates of return for investments in developing orphan drugs should not be greater than the industry average. More research is required on data and assumptions, but with the data and assumptions we use, we find that in order to secure such a reasonable price for an orphan drug, the CET for orphans would need to be higher. This could be one approach for establishing the maximum allowable price society should be willing to pay, although decision-makers may still wish to negotiate a lower price, or refuse to pay such a premium over the value-based price in order to treat these groups of patients.

PMU74 HEALTH TECHNOLOGY ASSESSMENT IN BULGARIA – 3 YEAR REPORT CARD

A designated Health Technology Assessment (HTA) body was introduced in Bulgaria in December 2015, as part of the National Centre for Public Health and Analyses (NCPHA). The assessment process involves a weighted point-based scoring system covering 11 key criteria, including efficacy, safety, QoL, cost-effectiveness, and budget impact. A therapy scoring 75% of the maximum 965 points can be recommended for addition to the Positive Drug List (PDL), while a score between 50% and 75% allows PDL access with specific restrictions (e.g. specific populations only). This research evaluates the impact of the Bulgarian HTA committee. All publicly-available HTA assessments issued by the Bulgarian HTA committee (01/01/2015-31/12/2018) were screened and key information extracted. 75 HTA reports were published on the NCPHA website. 68/75 (91%) of the assessments received a positive recommendation for PDL addition, including 47/75 (63%) receiving unconditional positive recommendations (scoring ≥75% in the scoring criteria). Only 7/75 (9%) received a negative opinion. 24/75 (32%) of the assessments were in oncology, 21/24 (88%) of which received a positive recommendation (46% unconditionally). Many reports state that an incremental cost-effectiveness threshold of 37,000BGN ($21,700USD) per QALY applies in the assessment process. The Bulgarian healthcare system is taking major strides in the direction of maximising the opportunity to offer cost-effective healthcare in a systematic approach. Further, from 2018, only cost-effective medicines with a positive recommendation and offered at a confidential discount to Bulgaria’s National Health Insurance Fund (NZOK) will be reimbursed, eliminating the prior costly practice of covering a basic threshold for a multitude of drugs, with patient co-pay. With almost two thirds of assessed therapies receiving unconditional recommendations to date, this HTA process holds promise to both relieve pharmaceutical expenditure, which has put immense pressure on NZOK in recent years, whilst maintaining access to innovative therapies.

Cost-effectiveness analysis of the use of letermovir for the prophylaxis of cytomegalovirus in adult cytomegalovirus seropositive recipients undergoing allogenic hematopoietic stem cell transplantation in Italy.

Background: The aim of the analysis is to assess the efficiency of the allocation of economic resources related to the use of letermovir cytomegalovirus (CMV) prophylaxis in adult seropositive recipients (R+) patients receiving an allogenic hematopoietic stem cell transplantation (HSCT), compared with a no-prophylaxis strategy, assuming preemptive antiviral administration in both groups from the perspective of the Italian National Health Service (NHS), through a cost-effectiveness analysis.Methods: The model used is based on a decision tree which simulates on a lifetime horizon the progression of CMV infection, considering two alternatives: the use of letermovir CMV prophylaxis, followed by preemptive therapy in case of clinically significant CMV infection, or the avoided use of letermovir CMV prophylaxis, considering direct medical costs (referred to 2018) and quality-adjusted life years (QALYs), both discounted considering a 3% annual rate. Two scenarios were considered, representing the differences related to regional contexts and clinical practice of different typologies of hospitals (public or private accredited with Regional Health Services).Results: The use of letermovir prophylaxis compared with no prophylaxis strategy would lead to an increase of QALYs and direct medical costs in the two scenarios considered, with a mean increase of 0.45 QALYs, and an increase of direct medical costs of 10,222.4 € and of 10,809.9 € in the two scenarios. The incremental cost-effectiveness ratios are equal to 22,564 €/QALY and 23,861 €/QALY. The probabilistic sensitivity analysis conducted showed a percentage of results below the threshold of 40,000 €/QALY of 67.4% and 71.3%; and below a threshold of 25,000 €/QALY equal to 50.4% and to 53.0%.Conclusions: The use of letermovir CMV prophylaxis in adult R+ patients receiving allogenic HSCT, compared with a no-prophylaxis strategy, would be cost-effective for the Italian NHS considering the incremental cost-effectiveness thresholds of 40,000 €/QALY and of 25,000 €/QALY.

Predicting Therapy Success and Costs for Personalized Treatment Recommendations Using Baseline Characteristics: Data-Driven Analysis.

BackgroundDifferent treatment alternatives exist for psychological disorders. Both clinical and cost effectiveness of treatment are crucial aspects for policy makers, therapists, and patients and thus play major roles for healthcare decision-making. At the start of an intervention, it is often not clear which specific individuals benefit most from a particular intervention alternative or how costs will be distributed on an individual patient level.ObjectiveThis study aimed at predicting the individual outcome and costs for patients before the start of an internet-based intervention. Based on these predictions, individualized treatment recommendations can be provided. Thus, we expand the discussion of personalized treatment recommendation.MethodsOutcomes and costs were predicted based on baseline data of 350 patients from a two-arm randomized controlled trial that compared treatment as usual and blended therapy for depressive disorders. For this purpose, we evaluated various machine learning techniques, compared the predictive accuracy of these techniques, and revealed features that contributed most to the prediction performance. We then combined these predictions and utilized an incremental cost-effectiveness ratio in order to derive individual treatment recommendations before the start of treatment.ResultsPredicting clinical outcomes and costs is a challenging task that comes with high uncertainty when only utilizing baseline information. However, we were able to generate predictions that were more accurate than a predefined reference measure in the shape of mean outcome and cost values. Questionnaires that include anxiety or depression items and questions regarding the mobility of individuals and their energy levels contributed to the prediction performance. We then described how patients can be individually allocated to the most appropriate treatment type. For an incremental cost-effectiveness threshold of 25,000 €/quality-adjusted life year, we demonstrated that our recommendations would have led to slightly worse outcomes (1.98%), but with decreased cost (5.42%).ConclusionsOur results indicate that it was feasible to provide personalized treatment recommendations at baseline and thus allocate patients to the most beneficial treatment type. This could potentially lead to improved decision-making, better outcomes for individuals, and reduced health care costs.

The cost-effectiveness of introducing a vaccine for the prevention of herpes zoster and post-herpetic neuralgia in elderly Danes

Herpes Zoster is an unpleasant and painful skin condition associated with a risk of developing post-herpetic neuralgia if the pain persists. Herpes Zoster most often occurs in elderly people. This study examines the cost-effectiveness of introducing a vaccine against Herpes Zoster in the Danish health care sector. The cost-effectiveness is examined in a Markov lifetime model framework and populated with Danish data and results from the international literature. From the base case analysis, with an ICER of DKK 261,372 per QALY gained, it seems that an implementation of a vaccine against HZ in the Danish population aged 65 years or more might be cost-effective within generally accepted incremental cost-effectiveness thresholds. Furthermore, an age-specific analysis showed that although the incidence of herpes zoster generally increases with age, targeting the vaccine at persons aged 50-69 years rather than the persons aged 65 years or more, seems to be a more cost-effective strategy from the societal perspective. However, these results are very sensitive to changes in vaccine price, utility values, discount rates, and especially, to the vaccine duration of protection.Published: Online December 2016. In print December 2017.

Cost-effectiveness of PCSK9 Inhibitor Therapy in Patients With Heterozygous Familial Hypercholesterolemia or Atherosclerotic Cardiovascular Disease.

Proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors were recently approved for lowering low-density lipoprotein cholesterol in heterozygous familial hypercholesterolemia (FH) or atherosclerotic cardiovascular disease (ASCVD) and have potential for broad ASCVD prevention. Their long-term cost-effectiveness and effect on total health care spending are uncertain. To estimate the cost-effectiveness of PCSK9 inhibitors and their potential effect on US health care spending. The Cardiovascular Disease Policy Model, a simulation model of US adults aged 35 to 94 years, was used to evaluate cost-effectiveness of PCSK9 inhibitors or ezetimibe in heterozygous FH or ASCVD. The model incorporated 2015 annual PCSK9 inhibitor costs of $14,350 (based on mean wholesale acquisition costs of evolocumab and alirocumab); adopted a health-system perspective, lifetime horizon; and included probabilistic sensitivity analyses to explore uncertainty. Statin therapy compared with addition of ezetimibe or PCSK9 inhibitors. Lifetime major adverse cardiovascular events (MACE: cardiovascular death, nonfatal myocardial infarction, or stroke), incremental cost per quality-adjusted life-year (QALY), and total effect on US health care spending over 5 years. Adding PCSK9 inhibitors to statins in heterozygous FH was estimated to prevent 316,300 MACE at a cost of $503,000 per QALY gained compared with adding ezetimibe to statins (80% uncertainty interval [UI], $493,000-$1,737,000). In ASCVD, adding PCSK9 inhibitors to statins was estimated to prevent 4.3 million MACE compared with adding ezetimibe at $414,000 per QALY (80% UI, $277,000-$1,539,000). Reducing annual drug costs to $4536 per patient or less would be needed for PCSK9 inhibitors to be cost-effective at less than $100,000 per QALY. At 2015 prices, PCSK9 inhibitor use in all eligible patients was estimated to reduce cardiovascular care costs by $29 billion over 5 years, but drug costs increased by an estimated $592 billion (a 38% increase over 2015 prescription drug expenditures). In contrast, initiating statins in these high-risk populations in all statin-tolerant individuals who are not currently using statins was estimated to save $12 billion. Assuming 2015 prices, PCSK9 inhibitor use in patients with heterozygous FH or ASCVD did not meet generally acceptable incremental cost-effectiveness thresholds and was estimated to increase US health care costs substantially. Reducing annual drug prices from more than $14,000 to $4536 would be necessary to meet a $100,000 per QALY threshold.

Using CART to Identify Thresholds and Hierarchies in the Determinants of Funding Decisions.

There is much interest in understanding decision-making processes that determine funding outcomes for health interventions. We use classification and regression trees (CART) to identify cost-effectiveness thresholds and hierarchies in the determinants of funding decisions. The hierarchical structure of CART is suited to analyzing complex conditional and nonlinear relationships. Our analysis uncovered hierarchies where interventions were grouped according to their type and objective. Cost-effectiveness thresholds varied markedly depending on which group the intervention belonged to: lifestyle-type interventions with a prevention objective had an incremental cost-effectiveness threshold of $2356, suggesting that such interventions need to be close to cost saving or dominant to be funded. For lifestyle-type interventions with a treatment objective, the threshold was much higher at $37,024. Lower down the tree, intervention attributes such as the level of patient contribution and the eligibility for government reimbursement influenced the likelihood of funding within groups of similar interventions. Comparison between our CART models and previously published results demonstrated concurrence with standard regression techniques while providing additional insights regarding the role of the funding environment and the structure of decision-maker preferences.

PHP198 - DEFINING END-OF-LIFE: IS THE SHORT LIFE EXPECTANCY CRITERION BIASED AGAINST YOUNG PEOPLE AND CHILDREN?

Health technology assessment (HTA) agencies use end-of-life (EoL) criteria to justify higher incremental cost-effectiveness thresholds to improve access to treatments that may not otherwise demonstrate adequate value for money. We argue that, as currently defined, EoL criteria are biased against young people and children and consider some possible alternative approaches. A key component of HTA EoL criteria is short life expectancy. For instance, the National Institute for Health and Care Excellence and the Scottish Medicines Consortium EoL criteria include a life expectancy requirement of less than 24 and 36 months, respectively. The short life expectancy criterion reflects the notion that the patient has a terminal illness, and death from that disease is imminent. However, a recent HTA assessment involving a pediatric cancer drug raises questions about the adequacy of the short life expectancy criterion. In this case, life expectancy of the indicated population was greater than 3 years, with median overall survival of approximately 4 years. However, in a pediatric population, this significantly reduced life expectancy satisfies our intuitive sense of being at the EoL, but it does not meet the HTA short life expectancy criterion. On this basis we argue that current HTA EoL criteria are biased against young people and children. We consider potential alternatives to the short life expectancy criterion, including: eliminating the criterion; utilizing a separate threshold for non-elderly patients; and redefining the criterion in terms of the proportion of a life cut short. We argue for use of the latter concept and that equity considerations help to justify the use of a higher cost-effectiveness threshold in non-elderly patient populations. As currently defined the short life expectancy criterion is biased against non-elderly patients with a limited life expectancy. Redefining EoL criteria using the concept of a life cut short may help to address this problem.

Evaluation of Health Economics in Radiation Oncology: A Systematic Review

Despite the rising costs in radiation oncology, the impact of health economics research on radiation therapy practice analysis patterns is unclear. We performed a systematic review of cost-effectiveness analyses (CEAs) and cost-utility analyses (CUAs) to identify trends in reporting quality in the radiation oncology literature over time. A systematic review of radiation oncology economic evaluations up to 2014 was performed, using MEDLINE and EMBASE databases. The Consolidated Health Economic Evaluation Reporting Standards guideline informed data abstraction variables including study demographics, economic parameters, and methodological details. Tufts Medical Center CEA registry quality scores provided a basis for qualitative assessment of included studies. Studies were stratified by 3 time periods (1995-2004, 2005-2009, and 2010-2014). The Cochran-Armitage trend test and linear trend test were used to identify trends over time. In total, 102 articles were selected for final review. Most studies were in the context of a model (61%) or clinical trial (28%). Many studies lacked a conflict of interest (COI) statement (67%), a sponsorship statement (48%), a reported study time horizon (35%), and the use of discounting (29%). There was a significant increase over time in the reporting of a COI statement (P<.001), health care payer perspective (P=.019), sensitivity analyses using multivariate (P=.043) or probabilistic methods (P=.011), incremental cost-effectiveness threshold (P<.001), secondary source utility weights (P=.010), and cost effectiveness acceptability curves (P=.049). There was a trend toward improvement in Tuft scores over time (P=.065). Recent reports demonstrate improved reporting rates in economic evaluations; however, there remains significant room for improvement as reporting rates are still suboptimal. As fiscal pressures rise, we will rely on economic assessments to guide our practice decisions and policies. We recommend improved adherence to published guidelines and further research to determine the clinical implications of our findings.

Costs and Cost-Effectiveness of Carotid Stenting Versus Endarterectomy for Patients at Standard Surgical Risk

The Carotid Revascularization Endarterectomy versus Stenting Trial (CREST) demonstrated similar rates of the primary composite end point between carotid artery stenting (CAS) and carotid endarterectomy (CEA), although the risk of stroke was higher with CAS, and the risk of myocardial infarction was higher with CEA. Given the large number of patients who are candidates for these procedures, an understanding of their relative cost and cost-effectiveness may have important implications for health care policy and treatment guidelines. We performed a formal economic evaluation alongside the CREST trial. Costs were estimated from all trial participants over the first year of follow-up using a combination of resource use data and hospital billing data. Patient-level health use scores were obtained using data from the SF-36. We then used a Markov disease-simulation model calibrated to the CREST results to project 10-year costs and quality-adjusted life expectancy for the 2 treatment groups. Although initial procedural costs were $1025/patient higher with CAS, postprocedure costs and physician costs were lower such that total costs for the index hospitalization were similar for the CAS and CEA groups ($15 055 versus $14 816; mean difference, $239/patient; 95% CI for difference, -$297 to $775). Neither follow-up costs after discharge nor total 1-year costs differed significantly. For the CREST population, model-based projections over a 10-year time horizon demonstrated that CAS would result in a mean incremental cost of $524/patient and a reduction in quality-adjusted life expectancy of 0.008 years compared with CEA. Probabilistic sensitivity analysis demonstrated that CEA was economically attractive at an incremental cost-effectiveness threshold of $50 000/quality-adjusted life-year gained in 54% of samples, whereas CAS was economically attractive in 46%. Despite slightly lower in-trial costs and lower rates of stroke with CEA compared with CAS, projected 10-year outcomes from this controlled clinical trial demonstrate only trivial differences in overall healthcare costs and quality-adjusted life expectancy between the 2 strategies. If the CREST results can be replicated in clinical practice, these findings suggest that factors other than cost-effectiveness should be considered when deciding between treatment options for carotid artery stenosis in patients at standard risk for surgical complications. Clinical Trial Registration- URL: http://clinicaltrials.gov. Unique Identifier: NCT00004732.

Common Drug Review Recommendations

The Common Drug Review (CDR) was created to provide a single process to review the comparative clinical efficacy and cost effectiveness of new drugs, and then to make formulary listing recommendations to Canadian publicly funded drug benefit plans. The objective was to conduct an in-depth analysis of Canadian Expert Drug Advisory Committee (CEDAC) recommendations to date, to explore predictors and possible explanatory factors associated with negative recommendations. Final recommendations were identified from inception (September 2003) to 31 December 2009. Using only publicly available information, recommendations were analysed under the following categories: submission specifics, drug characteristics, clinical factors and economic factors. Descriptive analyses were conducted, followed by statistical analyses, to determine which factors independently predicted a 'do not list' (DNL) recommendation. The database consisted of 138 unique final recommendations. The overall DNL rate was 48%. Significant differences in DNL rates were observed between therapeutic areas, ranging from 0% for HIV antivirals up to 88% for analgesic drugs. In the univariate analysis, several factors were significantly associated with a DNL recommendation, including first-in-class drugs and use of clinical scales as an outcome. In the multivariate regression, four factors were significantly predictive of a DNL recommendation: clinical uncertainty (odds ratio [OR] 14), price higher than comparators (OR 9), request for reconsideration (OR 10) and price as the only economic evidence used (OR 18). Incremental cost-effectiveness thresholds were not predictive of recommendations. The hypothesis that economic factors did not impact recommendations when clinical factors were included first was supported by the analysis. This analysis documented an evidence-driven process that simultaneously weighted multiple factors. Clinical uncertainty and price considerations, but not economic results, had a strong impact on the recommendations. Insufficiency of clinical evidence may have resulted from the gap in evidence available at the time of product launch and the absence of demonstrated benefits to support innovative drugs.

Cost-Utility Analysis: Current Methodological Issues and Future Perspectives

The use of cost–effectiveness as final criterion in the reimbursement process for listing of new pharmaceuticals can be questioned from a scientific and policy point of view. There is a lack of consensus on main methodological issues and consequently we may question the appropriateness of the use of cost–effectiveness data in health care decision-making. Another concern is the appropriateness of the selection and use of an incremental cost–effectiveness threshold (Cost/QALY). In this review, we focus mainly on only some key methodological concerns relating to discounting, the utility concept, cost assessment, and modeling methodologies. Finally we will consider the relevance of some other important decision criteria, like social values and equity.

Cost-effectiveness targets for multi-detector row CT angiography in the work-up of patients with intermittent claudication.

To determine the costs, sensitivity for detection of significant stenoses, and proportion of equivocal multi-detector row computed tomographic (CT) angiography results in the work-up of patients with intermittent claudication that would make this imaging examination cost-effective compared with gadolinium-enhanced magnetic resonance (MR) angiography. A decision model was used to compare the societal cost-effectiveness of a new imaging modality with that of gadolinium-enhanced MR angiography. Main outcome measures were quality-adjusted life years (QALYs) and lifetime costs. By using threshold analysis of a given willingness to pay per QALY, target values for costs, sensitivity for detection of significant stenoses, and proportion of cases requiring additional work-up with intraarterial digital subtraction angiography owing to equivocal results of the new modality were determined. The base case evaluated was that of 60-year-old men with severe intermittent claudication and assumed an incremental cost-effectiveness threshold of 100,000 US dollars per QALY. If treatment were limited to angioplasty, a new imaging modality would be cost-effective if the costs were 300 US dollars and the sensitivity was 85%, even if up to 35% of patients needed additional work-up. When both angioplasty and bypass surgery were considered as treatment options, a new imaging modality was cost-effective if the costs were 300 US dollars, the sensitivity was higher than 94%, and 20% of patients required additional work-up. Multi-detector row CT angiography, as compared with currently used imaging modalities such as MR angiography, has the potential to be cost-effective in the evaluation of patients with intermittent claudication.