Average Incremental Cost-effectiveness Ratio Research Articles

Do value thresholds for oncology drugs differ from nononcology drugs?

In the past decade, many oncologic drugs have been approved that extend life and/or improve patients' quality of life. However, new cancer drugs are often associated with high price and increased medical spending. For example, in 2010, the average annual cost of care for breast cancer in the final stage of disease was reported to be $94,284, and the total estimated cost in the United States was $16.50 billion. To determine whether value threshold, as defined by the incremental cost-effectiveness ratio (ICER), differed between oncology and other therapeutic areas. The PubMed database was searched for articles published between January 2003 and December 2013 with calculated ICER for therapeutic drug entities in a specific therapeutic area. The search term used was "ICER" and "United States." From 275 results, only those articles that reported ICERs using quality-adjusted life-years (QALY) were included. In addition, only those articles that used a U.S. payer perspective were retained. Among those, nondrug therapy articles and review articles were excluded. The mean ICER and value threshold for oncologic drugs and nononcologic drugs were evaluated for the analysis. From 54 articles selected for analysis, 13 pertained to drugs in oncology therapeutics, and the remaining 41 articles addressed ICER for drugs in other therapeutic areas. The mean and median of ICERs calculated for cancer-specific drug intervention was $138,582/QALY and $55,500/QALY, respectively, compared with $49,913/QALY and $31,000/QALY, respectively, for noncancer drugs. Among the cancer drugs, 45.0% had ICERs below $50,000/QALY and 70.0% below $100,000/QALY. In comparison, 72.0% of noncancer drugs showed ICERs below $50,000/QALY, and 90.0% had ICERs below $100,000/QALY. When a specific threshold was mentioned, it was in the range of $100,000-$150,000 in cancer drugs, whereas drugs in other therapeutic areas used traditional threshold value within the range of $50,000-$100,000. The average ICER reported for cancer drugs was more than 2-fold greater than the average ICER for noncancer drugs. In general, articles that addressed the relative value of oncologic pharmaceuticals used higher value thresholds and reported higher ICERs than articles evaluating noncancer drugs.

PSY25 - Economic Evaluation Of Prophylactic Treatment Vs On Demand For Moderate Hemophilia A In Colombia

Hemophilia A is a genetic disease in which there is a deficiency in the level of activity of the clotting factor VIII, the moderate form is defined as a blood clotting factor level from 0.01 to 0.05 IU/ml. The aim of this analysis is to estimate the cost-effectiveness of prophylactic treatment of moderate hemophilia A compared to demand treatment in Colombia. A decision tree model was developed using a life expectancy horizon and a societal perspective; annual discount rate of 3% was applied to costs and effectiveness measures. Prophylactic treatment with recombinant factor VIII (25 IU/kg 3 times per week) was compared to on demand treatment (40 IU/kg BID for 3.5 days for each bleeding episode). The base case was simulated based on a patient of 8 years old and weight of 31kg without inhibitors. Effectiveness and probabilities of adverse events were taken from the literature. Costs (direct and indirect) were taken from local tariff manuals (SOAT and SISMED). Effectiveness measures were number of cases avoided of bleeding, hemarthrosis and arthropathies. All data were validated with a clinical expert. Univariate sensitivity analysis was done. Costs are presented in 2013 USD (exchange rate US$1 = $ 1927 COP). Over the time horizon, prophylactic treatment avoids 277 episodes of bleeding and 23 of joint damage. Total expected costs with prophylactic treatment were US$ 2.7M compared to on demand treatment US$ 2.4M. The incremental cost effectiveness ratios (ICER) for prophylactic treatment were US$ 1,056/avoided bleeding and US$12,512/avoided hemarthrosis/arthropathies. Sensitivity analysis showed the robustness of the results (average ICER US$ 1,153/avoided bleeding). From the social perspective, prophylactic treatment of moderate hemophilia A, with recombinant factor VIII would be a highly cost-effectiveness intervention with strong health benefits in number of cases of bleeding and joint damage avoided.

Two-Year and Lifetime Cost-Effectiveness of Intensity Modulated Radiation Therapy Versus 3-Dimensional Conformal Radiation Therapy for Head-and-Neck Cancer

To assess the cost-effectiveness of intensity modulated radiation therapy (IMRT) versus 3-dimensional conformal radiation therapy (3D-CRT) in the treatment of head-and neck-cancer (HNC). We used a Markov model to simulate radiation therapy-induced xerostomia and dysphagia in a hypothetical cohort of 65-year-old HNC patients. Model input parameters were derived from PARSPORT (CRUK/03/005) patient-level trial data and quality-of-life and Medicare cost data from published literature. We calculated average incremental cost-effectiveness ratios (ICERs) from the US health care perspective as cost per quality-adjusted life-year (QALY) gained and compared our ICERs with current cost-effectiveness standards whereby treatment comparators less than $50,000 per QALY gained are considered cost-effective. In the first 2 years after initial treatment, IMRT is not cost-effective compared with 3D-CRT, given an average ICER of $101,100 per QALY gained. However, over 15 years (remaining lifetime on the basis of average life expectancy of a 65-year-old), IMRT is more cost-effective at $34,523 per QALY gained. Although HNC patients receiving IMRT will likely experience reduced xerostomia and dysphagia symptoms, the small quality-of-life benefit associated with IMRT is not cost-effective in the short term but may be cost-effective over a patient's lifetime, assuming benefits persist over time and patients are healthy and likely to live for a sustained period. Additional data quantifying the long-term benefits of IMRT, however, are needed.

Cost-effectiveness of optimizing prevention in patients with coronary heart disease: the EUROASPIRE III health economics project

The EUROASPIRE III survey indicated that the guidelines on cardiovascular disease prevention are poorly implemented in patients with established coronary heart disease (CHD). The purpose of this health economic project was to assess the potential clinical effectiveness and cost-effectiveness of optimizing cardiovascular prevention in eight EUROASPIRE III countries (Belgium, Bulgaria, Croatia, Finland, France, Italy, Poland, and the U.K.). METHODS AND RESULTS The individual risk for subsequent cardiovascular events was estimated, based on published Framingham equations. Based on the EUROASPIRE III data, the type of suboptimal prevention, if any, was identified for each individual, and the effects of optimized tailored prevention (smoking cessation, diet and exercise, better management of elevated blood pressure and/or LDL-cholesterol) were estimated. Costs of prevention and savings of avoided events were based on country-specific data. A willingness to pay threshold of €30,000/quality-adjusted life year (QALY) was used. The robustness of the results was validated by sensitivity analyses. Overall, the cost-effectiveness analyses for the eight countries showed mainly favourable results with an average incremental cost-effectiveness ratio (ICER) of €12,484 per QALY. Only in the minority of patients at the lowest risk for recurrent events, intensifying preventive therapy seems not cost-effective. Also, the single impact of intensified cholesterol control seems less cost-effective, possibly because their initial 2-year risk was already fairly low, hence the room for improvement is rather limited. These results underscore the societal value of optimizing prevention in most patients with established CHD, but also highlight the need for setting priorities towards patients more at risk and the need for more studies comparing intensified prevention with usual care in these patients.

Comparative Survival and Economic Benefits of Deceased Donor Kidney Transplantation and Dialysis in People with Varying Ages and Co-Morbidities

BackgroundDeceased donor kidneys for transplantation are in most countries allocated preferentially to recipients who have limited co-morbidities. Little is known about the incremental health and economic gain from transplanting those with co-morbidities compared to remaining on dialysis. The aim of our study is to estimate the average and incremental survival benefits and health care costs of listing and transplantation compared to dialysis among individuals with varying co-morbidities.MethodsA probabilistic Markov model was constructed, using current outcomes for patients with defined co-morbidities treated with either dialysis or transplantation, to compare the health and economic benefits of listing and transplantation with dialysis.FindingsUsing the current waiting time for deceased donor transplantation, transplanting a potential recipient, with or without co-morbidities achieves survival gains of between 6 months and more than three life years compared to remaining on dialysis, with an average incremental cost-effectiveness ratio (ICER) of less than $50,000/LYS, even among those with advanced age. Age at listing and the waiting time for transplantation are the most influential variables within the model. If there were an unlimited supply of organs and no waiting time, transplanting the younger and healthier individuals saves the most number of life years and is cost-saving, whereas transplanting the middle-age to older patients still achieves substantial incremental gains in life expectancy compared to being on dialysis.ConclusionsOur modelled analyses suggest transplanting the younger and healthier individuals with end-stage kidney disease maximises survival gains and saves money. Listing and transplanting those with considerable co-morbidities is also cost-effective and achieves substantial survival gains compared with the dialysis alternative. Preferentially excluding the older and sicker individuals cannot be justified on utilitarian grounds.

Preoperative staging of lung cancer with PET/CT: cost-effectiveness evaluation alongside a randomized controlled trial

Positron emission tomography (PET)/CT has become a widely used technology for preoperative staging of non-small cell lung cancer (NSCLC). Two recent randomized controlled trials (RCT) have established its efficacy over conventional staging, but no studies have assessed its cost-effectiveness. The objective of this study was to assess the cost-effectiveness of PET/CT as an adjunct to conventional workup for preoperative staging of NSCLC. The study was conducted alongside an RCT in which 189 patients were allocated to conventional staging (n = 91) or conventional staging + PET/CT (n = 98) and followed for 1 year after which the numbers of futile thoracotomies in each group were monitored. A full health care sector perspective was adapted for costing resource use. The outcome parameter was defined as the number needed to treat (NNT)-here number of PET/CT scans needed-to avoid one futile thoracotomy. All monetary estimates were inflated to 2010 <euro>. The incremental cost of the PET/CT-based regimen was estimated at 3,927 <euro> [95% confidence interval (CI) -3,331; 10,586] and the NNT at 4.92 (95% CI 3.00; 13.62). These resulted in an average incremental cost-effectiveness ratio of 19,314 <euro>, which would be cost-effective at a probability of 0.90 given a willingness to pay of 50,000 <euro> per avoided futile thoracotomy. When costs of comorbidity-related hospital services were excluded, the PET/CT regimen appeared dominant. Applying a full health care sector perspective, the cost-effectiveness of PET/CT for staging NSCLC seems to depend on the willingness to pay in order to avoid a futile thoracotomy. However, given that four outliers in terms of extreme comorbidity were all randomized to the PET/CT arm, there is uncertainty about the conclusion. When hospital costs of comorbidity were excluded, the PET/CT regimen was found to be both more accurate and cost saving.

Tiotropium's cost-effectiveness for the treatment of COPD: a cost-utility analysis under real-world conditions.

BackgroundTiotropium is reimbursed since March 2004 in Belgium for the treatment of Chronic Obstructive Pulmonary Disease (COPD). Questions however remain on this product's value for money. The purpose of this study is to calculate tiotropium's cost-effectiveness under real-world conditions.MethodsStrengths of both observational and RCT data were combined in a model. A large longitudinal (2002-2006) observational dataset of regular tiotropium users (56 321 patients) was analysed to retrieve the baseline risk for exacerbations and exacerbation-related hospitalisations the year before the first delivery of tiotropium. The relative treatment effect from the UPLIFT (Understanding Potential Long-term Impacts on Function with Tiotropium) trial was then applied to this baseline risk to reflect the effect of tiotropium treatment and calculate the intervention's incremental cost-effectiveness ratio (ICER).ResultsAfter 1000 Latin Hypercube simulations, the incremental benefit expressed as quality-adjusted life years (QALY) gained is on average 0.00048 (95% confidence interval (CI) 0.00009 - 0.00092). In combination with a substantial mean incremental cost of €373 per patient (95% CI 279 - 475), this results in an unfavourable average ICER of €1 244 023 (95% CI 328 571 - 4 712 704) per QALY gained. Results were most sensitive to the treatment effect on hospitalisations. Based on our large observational database, up to 89% of the patients were not hospitalised for COPD in the year before the first tiotropium delivery.ConclusionsThe main cause for tiotropium's unfavourable cost-effectiveness ratio is a combination of a relative high price for tiotropium, a low number of hospitalisations without tiotropium treatment (on average 0.14/year) and a non-significant treatment effect (on average 0.94) with respect to avoiding exacerbation-related hospitalisations. From an economic point of view, a revision of reimbursement modalities (e.g. with a lower price) would be justified and would entail a more efficient use of resources.

A systematic review and economic evaluation of statins for the prevention of coronary events

To evaluate the clinical effectiveness and cost-effectiveness of statins for the primary and secondary prevention of cardiovascular events in adults with, or at risk of, coronary heart disease (CHD). Electronic databases were searched between November 2003 and April 2004. A review was undertaken to identify and evaluate all literature relating to the clinical and cost effectiveness of statins in the primary and secondary prevention of CHD and cardiovascular disease (CVD) in the UK. A Markov model was developed to explore the costs and health outcomes associated with a lifetime of statin treatment using a UK NHS perspective. Thirty-one randomised studies were identified that compared a statin with placebo or with another statin, and reported clinical outcomes. Meta-analysis of the available data from the placebo-controlled studies indicates that, in patients with, or at risk of, CVD, statin therapy is associated with a reduced relative risk of all cause mortality, cardiovascular mortality, CHD mortality and fatal myocardial infarction (MI), but not of fatal stroke. It is also associated with a reduced relative risk of morbidity [non-fatal stroke, non-fatal MI, transient ischaemic attack (TIA), unstable angina] and of coronary revascularisation. It is hardly possible, on the evidence available from the placebo-controlled trials, to differentiate between the clinical efficacy of atorvastatin, fluvastatin, pravastatin and simvastatin. However, there is some evidence from direct comparisons between statins to suggest that atorvastatin may be more effective than pravastatin in patients with symptomatic CHD. There is limited evidence for the effectiveness of statins in different subgroups. Statins are generally considered to be well tolerated and to have a good safety profile. This view is generally supported both by the evidence of the trials included in this review and by postmarketing surveillance data. Increases in creatine kinase and myopathy have been reported, but rhabdomyolysis and hepatotoxicity are rare. However, some patients may receive lipid-lowering therapy for as long as 50 years, and long-term safety over such a timespan remains unknown. In secondary prevention of CHD, the incremental cost-effectiveness ratios (ICERs) increase with age varying between pound 10,000 and pound 17,000 per quality adjusted life year (QALY) for ages 45 and 85 respectively. Sensitivity analyses show these results are robust. In primary prevention of CHD there is substantial variation in ICERs by age and risk. The average ICERs weighted by risk range from pound 20,000 to pound 27,500 for men and from pound 21,000 to pound 57,000 for women. The results are sensitive to the cost of statins, discount rates and the modelling time frame. In the CVD analyses, which take into account the benefits of statins on reductions in stroke and TIA events, the average ICER weighted by risk level remains below pound 20,000 at CHD risk levels down to 0.5%. Limitations of the analyses include the requirement to extrapolate well beyond the timeframe of the trial period, and to extrapolate effectiveness results from higher risk primary prevention populations to the treatment of populations at much lower risk. Consequently, the results for the lower age bands and lower risks are subject to greater uncertainty and need to be treated with caution. There is evidence to suggest that statin therapy is associated with a statistically significant reduction in the risk of primary and secondary cardiovascular events. As the confidence intervals for each outcome in each prevention category overlap, it is not possible to differentiate, in terms of relative risk, between the effectiveness of statins in primary and secondary prevention. However, the absolute risk of CHD death/non-fatal MI is higher, and the number needed to treat to avoid such an event is consequently lower, in secondary than in primary prevention. The generalisability of these results is limited by the exclusion, in some studies, of patients who were hypersensitive to, intolerant of, or known to be unresponsive to, statins, or who were not adequately compliant with study medication during a placebo run-in phase. Consequently, the treatment effect may be reduced when statins are used in an unselected population. The results of the economic modelling show that statin therapy in secondary prevention is likely to be considered cost-effective. In primary prevention, the cost-effectiveness ratios are dependent on the level of CHD risk and age, but the results for the CVD analyses offer support for the more aggressive treatment recommendation issued by recent guidelines in UK. Evidence on clinical endpoints for rosuvastatin is awaited from on-going trials. The potential targeting of statins at low-risk populations is however associated with major uncertainties, particularly the likely uptake and long-term compliance to lifelong medication by asymptomatic younger patients. The targeting, assessment and monitoring of low-risk patients in primary care would be a major resource implication for the NHS. These areas require further research.

Economic Evaluation of Palliative Management versus Peritoneal Dialysis and Hemodialysis for End-Stage Renal Disease: Evidence for Coverage Decisions in Thailand

Objectives To examine the value for money of including peritoneal dialysis (PD) or hemodialysis (HD) into the universal health insurance scheme of Thailand. Methods A probabilistic Markov model applied to end-stage renal disease (ESRD) patients aged 20 to 70 years was developed to examine the incremental cost-effectiveness ratio (ICER) of palliative care versus 1) providing PD as an initial treatment followed by HD if complications/switching occur; and 2) providing HD followed by PD if complications/switching occur. Input parameters were extracted from a national cohort, the Thailand Renal Replacement Therapy Registry, and systematic reviews, where possible. The study explored the effects of uncertainty around input parameters, presented as cost-effectiveness acceptability frontier, as well as the value of obtaining further information on chosen parameters, i.e., partial expected value of perfect information. Results Using a societal perspective, the average ICER of initial treatment with PD and the average ICER of initial treatment with HD were 672,000 and 806,000 Baht per quality-adjusted life-year (QALY) gained (52,000 and 63,000 purchasing power parity [PPP] US$/QALY) compared with palliative care. Providing treatments for younger ESRD patients resulted in a significant improvement of survival and gain of QALYs compared with the older aged group. The cost-effectiveness and cost-utility ratios of both options for the older age group were relatively similar. Conclusion The results suggest that offering PD as initial treatment was a better choice than offering HD, but it would only be considered a cost-effective strategy if the social willingness-to-pay threshold was at or higher than 700,000 Baht per QALY (54,000 PPP US$/QALY) for the age 20 group and 750,000 Baht per QALY (58,000 PPP US$/QALY) for age 70 years.