Life Year Saved Research Articles

Projecting the Health and Economic Impact of Road Safety Initiatives: A Case Study of a Multi-country Project

Objective: The Road Safety in 10 Countries (RS-10) project will implement 12 different road safety interventions at specific sites within 10 low- and middle-income countries (LMICs). This evaluation reports the number of lives that RS-10 is projected to save in those locations, the economic value of the risk reduction, and the maximum level of investment that a public health intervention of this magnitude would be able to incur before its costs outweigh its health benefits. Methods: We assumed a 5-year time implementation horizon corresponding to the duration of RS-10. Based on a preliminary literature review, we estimated the effectiveness for each of the RS-10 interventions. Applying these effectiveness estimates to the size of the population at risk at RS-10 sites, we calculated the number of lives and life years saved (LYS) by RS-10. We projected the value of a statistical life (VSL) in each RS-10 country based on gross national income (GNI) and estimated the value of the lives saved using each country's VSL. Sensitivity analysis addressed robustness to assumptions about elasticity, discount rates, and intervention effectiveness. Results: From the evidence base reviewed, only 13 studies met our selection criteria. Such a limited base presents uncertainties about the potential impact of the modeled interventions. We tried to account for these uncertainties by allowing effectiveness to vary ±20 percent for each intervention. Despite this variability, RS-10 remains likely to be worth the investment. RS-10 is expected to save 10,310 lives over 5 years (discounted at 3%). VSL and $/LYS methods provide concordant results. Based on our estimates of each country's VSL, the respective countries would be willing to pay $2.45 billion to lower these fatality risks (varying intervention effectiveness by ±20 percent, the corresponding range is $2.0–$2.9 billion). Analysis based on $/LYS shows that the RS-10 project will be cost-effective as long as its costs do not exceed $5.14 billion (under ±20% intervention effectiveness, the range = $4.1–$6.2 billion). Even at low efficacy, these estimates are still several orders of magnitude above the $125 million projected investment. Conclusion: RS-10 is likely to yield high returns for invested resources. The study's chief limitation was the reliance on the world's limited evidence base on how effective the road safety interventions will be. Planned evaluation of RS-10 will enhance planners’ ability to conduct economic assessments of road safety in developing countries.

Cost-effectiveness Analysis of a Randomized Study Comparing Radiosurgery With Radiosurgery and Whole Brain Radiation Therapy in Patients With 1 to 3 Brain Metastases

In this study, we compare 2 treatment options and determine cost-effectiveness and cost-utility. We carried out a decision analysis populated with data from patients with brain metastasis in a concurrent trial randomized to either stereotactic radiosurgery (SRS) and observation or SRS and whole brain radiation therapy. Outcomes included actual life years saved (LYS), quality-adjusted life years (QALYs), and incremental cost-effectiveness ratio (ICER). Costs used were from the healthcare perspective and utilities were captured through a time-trade-off method, using 10-year, 5-year, and 1-year time horizons. One-way sensitivity analyses were carried out to determine robustness of the decision analysis model. Compared with SRS and whole brain radiation therapy, SRS and observation not only had a higher average cost ($74,000 vs $119,000, respectively) but also a higher average effectiveness (0.60 LYS vs 1.64 LYS, respectively) with an ICER of $44,231/LYS or $41,783/QALY (with utilities captured using a 10-year horizon). Slightly higher ICER estimates were achieved with utilities captured using the other time horizons ($43,280/QALY and $44,064/QALY, respectively). Sensitivity analysis showed that the following variables had the highest impact on the ICER: probability of no recurrence in recursive-partitioning analysis class 2 after SRS and observation; probability of being alive after SRS and observation in recursive-partitioning analysis class 2 and being treated for recurrence. Compared with other interventions in the $50,000 to $100,000/QALY cost-effectiveness range, the application of SRS and observation, with subsequent neurosurgical management of recurrences, is shown to be a reasonable treatment modality for brain metastases.

The cost efficiency of HPV vaccines is significantly underestimated due to omission of conisation-associated prematurity with neonatal mortality and morbidity

Introduction: Cervical intraepithelial neoplasia (CIN) represents the precursor of invasive cervical cancer and is associated with human papillomavirus infection (HPV) against which two vaccines have been approved in the last years. Standard treatments of high-grade CIN are conisation procedures, which are associated with an increased risk of subsequent pregnancy complications like premature delivery and possible subsequent life-long disability. HPV vaccination has therefore the potential to decrease neonatal morbidity and mortality. This has not been taken into account in published cost-effectiveness models. Material and Methods: We calculated the possible reduction rate of conisations for different vaccination strategies for Germany. Using this rate, we computed the reduction of conisation-associated preterm deliveries, life-long disability and neonatal death due to prematurity. The number of life-years saved (LYS) and gain in quality-adjusted life-years (QALYs) was estimated. The incremental costs per LYS / additional QALY were calculated. Results: The reduction of conisation procedures was highest in scenario I (vaccination coverage 90% prior to HPV exposition) with about 50%. The costs per LYS or additional QALY were lowest in scenario I, II and III with 45,101 € or 43,505–47,855 € and rose up to 60,544 € or 58,401–64,240 € in scenario V (50% vaccinated prior to sexual activity + additional 20% catch-up at a mean age of 20 y). Conclusion: Regarding the HPV 16 / 18 vaccines as “vaccines against conisation-related neonatal morbidity and mortality” alone, they already have the potential to be cost-effective. This effect adds up to reduction of cervical cancer cases and decreased costs of screening for CIN. Further studies on cost-effectiveness of HPV vaccination should take the significant amount of neonatal morbidity and mortality into account.

PD06-04: Uptake and Economic Impact of First-Cycle Colony Stimulating Factor Use during the Adjuvant Treatment of Breast Cancer.

Abstract Background: In 2002, pegfilgrastim was approved by the FDA and the benefits of dose-dense breast cancer(BC) chemotherapy were reported. A meta-analysis revealed these benefits were limited to hormone receptor negative tumors (20% risk reduction), where hormone receptor positive cancers had a minimal 2% risk reduction. We examined first-cycle growth factor use(FC-CSF) before and after 2002 and estimated US expenditures for dose-dense chemotherapy by hormone receptor status. Methods: We identified subjects in SEER-Medicare>65 years oldwith stage I-III BC between 1998 and 2005who had≥1 chemotherapy claim within one year of diagnosis. We classified those with an average cycle length <21 days as having had dose-dense therapy. The associations of patient, tumor, and physician-related factors with receipt of any CSF and FC-CSF use were analyzed using GEE. Costs and event-free life-years saved were estimated for patients by HR status. Results: Among the 11,143 patients identified, 5,356(48.1%) hada CSF claim during therapy and 2095(18.8%) had FC-CSF. CSF use increased from 25.4% to 70.9%, FC-CSF increased from 6.4% to 46.9% and pegfilgrastim increased from 5% to 85%. During this time receipt of the combination of an anthracycline and a taxane increased from 12% to 47%. In a multivariable analysis, any CSF use was associated with age, treatment by an oncologist, and chemotherapy type; and negatively associated with black/Hispanic race, rural residence and shorter chemotherapy duration. FC-CSF use was associated also with higher SES. For hormone receptor negative patients, the cost per event-free life year saved was $5,512, with annual costs of $19.5 million. For hormone receptor positive patients, however, there was no savings in life years (and therefore no relevant cost per event-free life year saved) with annual costs of $38.8 million for those over 65. The annual costs were estimated at $86.1 million for the hormone receptor positive patient population incorporating all ages. Conclusions: Our study demonstrated a widespread increase in the use of CSF's and specifically first cycle pegfilgrastim in elderly women receiving adjuvant chemotherapy for breast cancer. This 4-fold increase came shortly after published reports of the benefits of dose-dense therapy. While this approach is very cost-effective in women with hormone receptor negative tumors, it has come at great financial cost in women with hormone receptor positive tumors who are less likely to have a meaningful benefit. In order to reduce the total costs of cancer-care, efforts should be made to ensure that these treatments are reserved for only those who benefit substantially from their use. Citation Information: Cancer Res 2011;71(24 Suppl):Abstract nr PD06-04.

Cost-Effectiveness of Lanthanum Carbonate versus Sevelamer Hydrochloride for the Treatment of Hyperphosphatemia in Patients with End-Stage Renal Disease: A US Payer Perspective

Objective To assess the cost-effectiveness of lanthanum carbonate (LC) versus sevelamer hydrochloride (SH) as a treatment for hyperphosphatemia in end-stage renal disease (ESRD) patients. Methods A Markov model was developed to estimate health outcomes; quality-adjusted life years (QALYs) and life-years saved (LYS), as well as associated costs. The model incorporated patient-level data from a randomized head-to-head crossover study that compared the reduction of serum phosphorus using LC and SH for 4 weeks each. The model included patients previously treated with calcium-based binders. Both the intent-to-treat (ITT) population and the cohort of patients who completed treatment in both periods of the study (i.e., completer population) were assessed. The baseline risks of cardiovascular disease (CVD), all-cause mortalities for CVD, and non-CVD patients were derived from a large US renal database. Patient outcomes were modeled for 10 years, and incremental cost-effectiveness ratios (ICERs) were calculated for LC relative to SH. Deterministic and probabilistic sensitivity analyses (PSA) were performed to test the robustness of the base-case model. Results For the ITT population, the ICERs of LC versus SH were $24,724/QALY and $15,053/LYS, respectively (in US dollars). When the completer population was considered, the ICERs of LC versus SH were $15,285/QALY and $9,337/LYS ( Table 2), respectively. The PSA indicated 61.9% and 85.8% probabilities for ITT and completer populations of LC being cost-effective at the $50,000/QALY willingness-to-pay threshold, respectively. Conclusion LC is a cost-effective strategy compared with SH in the treatment of ESRD patients with hyperphosphatemia who were previously treated with calcium-based binders. Sensitivity analyses demonstrated the robustness of the pharmacoeconomic model.

Evaluation of primary HPV-DNA testing in relation to visual inspection methods for cervical cancer screening in rural China: an epidemiologic and cost-effectiveness modelling study

BackgroundA new lower-cost rapid-throughput human papillomavirus (HPV) test (careHPV, Qiagen, Gaithersburg, USA) has been shown to have high sensitivity for the detection of high grade cervical intraepithelial neoplasia.MethodsWe assessed the outcomes and cost-effectiveness of careHPV screening in rural China, compared to visual inspection with acetic acid, when used alone (VIA) or in combination with Lugol's iodine (VIA/VILI). Using data on sexual behaviour, test accuracy, diagnostic practices and costs from studies performed in rural China, we estimated the cost-effectiveness ratio (CER) and associated lifetime outcomes for once-lifetime and twice-lifetime screening strategies, and for routine screening at 5-yearly, 10-yearly and IARC-recommended intervals. The optimal age range for once-lifetime screening was also assessed.ResultsFor all strategies, the relative ordering of test technologies in reducing cervical cancer incidence and mortality was VIA (least effective); VIA/VILI; careHPV@1.0 pg/ml and careHPV@0.5 pg/ml (most effective). For once-lifetime strategies, maximum effectiveness was achieved if screening occurred between 35-50 years. Assuming a participation rate of ~70%, once-lifetime screening at age 35 years would reduce cancer mortality by 8% (for VIA) to 12% (for careHPV@0.5) over the long term, with a CER of US$557 (for VIA) to $959 (for careHPV@1.0) per life year saved (LYS) compared to no intervention; referenced to a 2008 GDP per capita in Shanxi Province of $2,975. Correspondingly, regular screening with an age-standardised participation rate of 62% (which has been shown to be achievable in this setting) would reduce cervical cancer mortality by 19-28% (for 10-yearly screening) to 43-54% (using IARC-recommended intervals), with corresponding CERs ranging from $665 (for 10-yearly VIA) to $2,269 (for IARC-recommended intervals using careHPV@1.0) per LYS.ConclusionsThis modelled analysis suggests that primary careHPV screening compares favourably to visual inspection screening methodologies in rural China, particularly if used as part of a regular screening program.

Cost‐effectiveness of treatment of childhood acute lymphoblastic leukemia with chemotherapy only: The influence of new medication and diagnostic technology

Survival for childhood acute lymphoblastic leukemia (ALL) has reached 80-90%. Future improvement in treatment success will involve new technologies and medication, adding to the pressure on limited financial resources. Therefore a retrospective cost-effectiveness analysis of ALL treatment with chemotherapy only according to the two most recent Dutch Childhood Oncology Group treatment protocols was performed. The most recent protocol ALL10 included more expensive medication (pegasparaginase) and implemented a new diagnostic technique (minimal residual disease levels) compared to the previous ALL9 protocol. Fifty children from a single center cohort were included. All direct medical costs made during treatment, including those in satellite hospitals, were determined. Costs per life year saved (LYS) were calculated. The cost-effectiveness ratio of the most recent treatment protocol was determined. LYS were calculated based on national 5-year event-free survival. Mean total costs were between $115,858 (ALL9) and $163,350 (ALL10) per patient. Hospital admissions (57%) and medication (11-17%) were important drivers of overall costs, and were higher in the most recent protocol ALL10. Costs per LYS were $1,962 (ALL9) and $2,655 (ALL10) and the cost-effectiveness ratio was $8,215. Treatment of childhood ALL with chemotherapy only is well within accepted ranges of cost-effectiveness. The use of new technology and more expensive medication in the most recent protocol ALL10 lead to higher costs but more LYS. In future (ALL) treatment protocols, costs in relation to effects should be taken into account in order to establish more cost-effective disease management without jeopardizing survival and quality of life.

COST-EFFECTIVENESS OF THE 5-YEARS CARDIOVASCULAR PREVENTION PROGRAM IN RUSSIAN POPULATION: 6C.02

Purpose: To evaluate the economic efficiency of 5-years cardiovascular prevention program in primary care of Russian Federation. Methods: In 1977–1990 in Moscow a large prevention project was realized. The project was realized in two primary care areas of Moscow and included men with baseline age 40–59. One primary care area was intervention area where cardiovascular prevention program was realized during the 5 years. Other area was control, where no additional interventions were conducted. Participants of intervention (n = 3488) and control primary care areas (n = 3168) were similar. The cardiovascular prevention program included physician's consultations for patients with hypertension and ischemic heart disease (8.0 in year), individual preventive counseling for participants with hypercholesterolemia overweight and obesity and sedentary lifestyle and group preventive counseling for the smoking patients. In this study we calculated the gain of life years saved (LYS) and quality adjusted life years (QALY) in intervention group compared with control group (during the 5 years on the intervention and during 10 years of total follow up period. Also we calculate the total cost of the program including the first screening, intervention costs and follow-up costs in the prices of 2008 year. The cost utility analysis was conducted with calculation of cost per gained QALY during 5 years and during 10 year. Results: The number of gained LYS in the intervention group was 45.7 on 1000 participants in 5 years and 139.4 – in 10 years. The number of QALY was 46.2 and 132.7 on 1000 participants in 5 and 10 years accordingly. The total costs were 174 124$ on 1000 participant in 5 years and 237 928$ in 10 years. Cost per QALY gained was 3769$ during the 5 years and 1793$ during the 5 years. The gross domestic product on capita in 2008 was 11 806$. Conclusion: Cardiovascular prevention program for men aged 40–59 is highly cost-effective.

Increasing fruit and vegetable consumption: a cost-effectiveness analysis of public policies

In many countries, consumption of fruits and vegetables (F&V) is below recommended levels. We quantify the economic and health effects of alternative policy (P) scenarios aiming to increase F&V consumption: (P1) 3.4% reduction in VAT, (P2) €100/year/person F&V stamp policy designed for low-income consumers (LIC) and (P3) €10 M information campaign. An economic model of the F&V market provides F&V consumption variations to a health impact model, leading to the number of deaths avoided (DA) and life-years saved (LYS). We compare the cost per statistical DA and LYS, taking into account the public costs of alternative policies. This analysis is applied to France in 2006. Relative risks of death for one additional F&V portion are disease dependent (range: 0.84-0.99). The highest variations in F&V consumption levels (less than +10 g/day/person on average) and health effects (<+600 DA, <+10 000 LYS) are modest. The costs/LYS are smaller for information campaign (€3 k), followed by VAT reduction (€99 k) and food stamp policy (€403 k). However, the information campaign leads to less LYS than VAT reduction. The food stamp policy reduces health inequalities between LIC and others, whereas the other ones can increase them. Our results suggest that (i) LYS are larger with VAT reduction than F&V stamps policies, (ii) information campaigns are the most cost-effective and (iii) market forces can limit the impacts of public health policies designed to favour F&V consumption increase.

Cost Effectiveness of Intensive Lipid-Lowering Treatment for Patients with Congestive Heart Failure and Coronary Heart Disease in the US

A recent study found fewer hospitalizations for congestive heart failure (CHF) patients receiving high-dose versus low-dose statin therapy. To examine the cost effectiveness of high-dose versus low-dose statin therapy in CHF patients. Two scenarios (literature-based [base-case scenario] vs trial-based post-event mortality [alternative scenario]) assessed the cost effectiveness of atorvastatin 80 mg/day (A80) versus atorvastatin 10 mg/day (A10) in patients with both CHF and coronary heart disease (CHD) [CHF/CHD], using a lifetime Markov model. The model predicts treatment-specific probabilities of major and minor cardiovascular events and death, based on clinical trial data. The quality of life and costs were literature based. Measures included costs per life-year saved (LYS) and QALY gained. Health consequences and costs were discounted at 3.0% annually. Analyses were conducted from the payer perspective and valued in $US, year 2006-7 values. Literature-based mortality estimates (base case) increased life-years and QALYs for A80 compared with A10 (incremental cost-effectiveness ratios [ICERs]: $US9600 per LYS; $US13 600 per QALY). At a willingness to pay of $US100 000 per QALY, A80 was cost effective in 80% of simulations. A10 dominated A80 when using trial-based mortality estimates (alternative scenario). At a willingness to pay of $US100 000 per QALY, A80 was cost effective in 48% of simulations. Intensive A80 treatment may be cost effective versus A10 in cardiovascular prevention in CHF/CHD patients in the US, due to projected gains in life expectancy and health-related quality of life. However, the results are highly sensitive to assumptions about the mortality rate in the model. When using the mortality rate observed in the trial, A10 dominates A80.

Willingness to Pay for Cancer Prevention

Cancer inflicts great pain, burden and cost upon American society, and preventing cancer is important but not costless. The aim of this review was to explore the upper limits that American society is paying and appears willing to pay to prevent cancer, by enforced environmental regulations and implemented clinical practice guidelines. Cost-effectiveness studies of clinical and environmental cancer-prevention policies and programmes were identified through a comprehensive literature review and confirmed to be officially sanctioned and implemented, enforced or funded. Data were collected in 2005-6 and analysed in 2007. The incremental cost-effectiveness ratios (ICERs) for clinical prevention policies ranged from under $US2000 to over $US6 000 000 per life-year saved (LYS), exceeding $US100 000 per LYS for only 11 of 101 guidelines. Median ICERs for tobacco-related ($US3978/LYS), colorectal ($US22 694/LYS) and breast ($US25 687/LYS) cancer prevention were within generally accepted ranges and tended not to vary greatly, whereas those for prostate ($US73 603/LYS) and cervical ($US125 157/LYS) cancer-prevention policies were considerably higher and varied substantially more. In contrast, both the median and range of the environmental policies were enormous, with 90% exceeding $US100 000 per LYS, and ICERs ranging from $US61 004 to over $US24 billion per LYS. Notwithstanding a relatively large and accessible literature evaluating the cost effectiveness of clinical and environmental cancer-prevention policies as well as the availability of ICERs for the policies identified in this study, the apparent willingness to pay to prevent cancer in the US still varies greatly and can be extremely high, particularly for many of the environmental cancer-prevention policies.

Cost-effectiveness analysis of a randomized study with stereotactic radiosurgery (SRS) alone versus SRS plus whole brain radiation therapy (WBRT) for patients with brain metastasis

6624 Background: This paper illustrates the incremental cost-effectiveness ratio (ICER) and cost-utility comparison of SRS alone versus SRS plus WBRT for brain metastasis. Methods: A decision analysis model, including subsequent treatment for recurrences, was developed and populated with data from patients randomized to either SRS alone (SA) versus SRS plus WBRT (SW), using costs from the healthcare institution perspective and outcomes based on actual life years saved (LYS) and quality-adjusted-life-years (QALYs). Utilities were captured through a time-trade -off methodology, utilizing three time horizons: 10 years, 5 years, and 1 year. Tornado diagrams and one-way sensitivity analyses were conducted to determine robustness of the model. Results: Fifty-eight patients were included in the final analysis, with 31 patients in the SA arm and 27 patients in the SW arm. The average cost of the SA arm is $119,000 versus $74,000 for the SW arm. The average effectiveness for the SA arm was 1.64 LYS versus 0.60 LYS for the SW arm. The ICER for total costs per LYS and QALYs were: $43,986 per LYS; $42,136 per QALY (10year); $43, 719 per QALY (5year); $44,686 (1year) for the SA arm. The surgical cost per LYS was $7,131 for the SA arm, and the radiation cost per LYS analysis was dominated. Tornado diagrams revealed that the following variables had the highest impact on the ICER: probability of being alive with SA in recursive partitioning analysis (RPA) class 2 and no recurrence; probability of no recurrence in RPA class 2 with SA; probability of being alive with SA in RPA class 2 and being treated for recurrence; probability of no recurrence in RPA class 1 with SA; and probability of being alive with no recurrence in RPA class 1 with SA. One-way sensitivity analysis exhibited a wide range variation for the first three of the above mentioned probabilities, with the final two having less variation. Conclusions: Compared to other interventions in the $50,000 to $100,000 per QALY cost-effectiveness range from an economic perspective, the application of SA, with subsequent surgical management of recurrences, is shown to be a reasonable treatment modality for brain metastasis. No significant financial relationships to disclose.

Modeling the cost-effectiveness of different oral antiviral therapies in patients with chronic hepatitis B

Chronic hepatitis B (CHB) is a common disease associated with high morbidity, mortality and impact on healthcare costs. Several oral antiviral therapies can lead to complete virologic response, which is associated with prevention of disease progression. The aim of this study was to estimate the cost-effectiveness of the oral antiviral treatments lamivudine, adefovir, telbivudine, entecavir and tenofovir, in patients with CHB. A Markov model was used to project the lifetime complications and costs in cohorts of both HBeAg-positive and HBeAg-negative CHB patients treated with one of the above drugs or no treatment. Rescue therapy with two different combination therapies (adefovir plus lamivudine or tenofovir plus entecavir) with their corresponding costs and efficacy rates was also considered. The probabilities of disease progression were based on serum HBV DNA levels. Disease and complication costs were assessed using the perspective of the Spanish National Health System. The highest rate of virologic response was obtained with tenofovir, and this translated to its higher life years saved (LYS) and quality adjusted life years (QALY) compared with the rest of the alternatives in HBeAg-positive and HBeAg-negative patients. Tenofovir is associated with lower costs and higher efficacy over entecavir, telbivudine and adefovir in HBeAg-positive patients, and telbivudine and entecavir in HBeAg-negative patients. The incremental cost-effectiveness ratios with respect to the rest of the alternatives are below the common reference efficiency threshold of 30,000 euro per LYS/QALY. In chronic HBV infected patients, tenofovir is a cost-effective or even cost-saving strategy compared with other available treatment options for CHB.

The Health and Economic Impact of Cervical Cancer Screening and Human Papillomavirus Vaccination in Kidney Transplant Recipients

The risk of cervical cancer in women who are kidney transplant recipients is increased, but little is known about the effectiveness of screening and human papillomavirus (HPV) vaccination in this group of women. We sought to determine the cost effectiveness of annual screening for cervical cancers using conventional cytology, liquid-based cytology (LBC), and pretransplant HPV vaccination in kidney transplant recipients. Three deterministic Markov models were developed to compare the costs and health outcomes in a cohort of women (n=1000) with kidney transplants aged 18 to 69 who underwent annual screening using conventional cytology, LBC, and HPV vaccination in HPV naïve women. After a screening period of 50 years, the incremental benefits of screening using conventional cytology compared with no screening were 0.05 life years saved (LYS) (18.2 days of lives saved), the incremental costs were $608, giving an incremental cost-effectiveness ratio of $12,160 per LYS. Compared with conventional cytology alone, the incremental cost-effectiveness ratios of annual screening using LBC and HPV vaccination before transplantation (assuming nonwaning efficacy) were $127,000 and $152,333 per LYS, respectively. The recommended policy of annual screening using conventional cytology is cost effective. The replacement of conventional cytology with LBC is likely to provide minimal survival benefits but considerable costs. Assuming the reported trial-based vaccine efficacy in HPV naïve women, a program of HPV vaccination before kidney transplantation is unlikely to be cost effective. Additional data about the long-term efficacy and safety of HPV vaccination is required before it should be included as standard care of renal transplant recipients.

Medullary thyroid cancer: early detection and novel treatments

Medullary thyroid cancer (MTC) is derived from the parafollicular cells of the thyroid. Understanding the molecular biology behind specific mutations of the RET gene and their prognostic implications have led to the establishment of tailored treatment modalities for certain patients. We review the most recent studies on the molecular biology, calcitonin screening, diagnosis, imaging, and treatment of MTC. Newly identified rearranged during transfection point mutations have helped with MTC prognosis and have resulted in the establishment of new treatment guidelines. Screening for MTC in the United States with basal serum calcitonin for patients with thyroid nodules would cost $11,793 per life-year saved (LYS), compared with colonoscopy and mammography screening. For metastatic or recurrent disease, neck ultrasound, chest computed tomography scan, liver MRI, bone scintigraphy, and axial skeleton MRI have been proven superior to 18F-FDG PET/computed tomography. For patients with nonoperable metastatic disease, novel chemotherapeutic agents, such as vandetanib, targeting rearranged during transfection, vascular endothelial growth factor receptor and epidermal growth factor receptor, are showing promise. Such agents are currently in phase II trials. There have been several recent advances in the diagnosis, molecular biology, imaging, and treatment options of MTC. By potentially downstaging of disease, and treating metastatic disease more effectively, overall survival and outcomes of patients may improve.

Cost, Effectiveness, and Cost-Effectiveness

Pluck the goose so as to obtain the most feathers with the least hissing . — —Jean-Baptiste Colbert, Minister of Finance to King Louis XIV of France Incremental or marginal cost-effectiveness ratios are founded on a number of assumptions that weaken their suitability as a way to balance competing economic and clinical priorities. We therefore propose a more relevant and responsible way to gauge the impact of clinical management strategies based on “consumer protection” principles—explicit disclosure of (1) the total magnitude of expected benefit in the target population (in life-years or quality-adjusted life-years), (2) the total monetary cost (in per capita inflation-adjusted dollars), and (3) a formal plan by which the added costs would be paid. Health policy decisions should therefore be based on the inherent trade-offs in the component measures of cost and effectiveness and not on a simple ratio of the two. Cost-effectiveness ratios are thereby rendered superfluous. Incremental or marginal cost-effectiveness ratios are widely viewed as rational ways to balance competing clinical and economic priorities that arise as a consequence of the inevitable disconnect between an individual’s wants and the society’s willingness to pay for those wants.1–5 The purpose of this essay is to question the practical relevance of these ratios with respect to strategic planning in health care, and offer a suitable alternative. In doing so, we will focus on the more pragmatic issues underlying cost-effectiveness analysis, and gloss over a variety of technical details such as the difference between a privately financed free market and the publicly financed healthcare market, between costs and charges, and between unadjusted and quality-adjusted outcomes. Our pragmatic perspective is not likely to be welcomed by orthodox experts in cost-effectiveness analysis. We believe, however, that any such criticisms can be blunted by recognizing the important distinctions between regulatory and clinical …

Cost-effectiveness of Proton Pump Inhibitor Cotherapy in Patients Taking Long-term, Low-Dose Aspirin for Secondary Cardiovascular Prevention

Patients with coronary heart disease (CHD) require long-term therapy with low-dose aspirin (ASA). Although these patients are at increased risk for upper gastrointestinal bleeding (UGIB) and proton pump inhibitor (PPI) cotherapy may reduce such risk, it is not known whether lifelong PPI cotherapy is cost-effective. A Markov model was developed to compare lifelong therapy with ASA alone vs therapy with ASA plus PPI in patients with CHD who are at least 50 years old. Base-case assumptions were (1) starting age, 65 years (range, 50-80 years); (2) UGIB risk category, average risk (range, average to 8-fold increased risk); (3) PPI effectiveness (66% (range, 25%-75%); and (4) annual PPI cost, $250 (range, $250-$1400). In the base-case analysis, ASA plus PPI resulted in fewer lifetime UGIB events (3.1% vs 9.5%) and UGIB-related deaths (0.4% vs 1.4%). At over-the-counter (OTC) PPI cost, ASA plus PPI was cost-effective, with an incremental cost-effectiveness ratio (ICER) of $40,090 per life-year saved (LYS). Varying PPI effectiveness from 75% to 25% resulted in ICERs of $35,315 to $94,578 per LYS. Varying the starting age of the cohort from 80 to 50 years resulted in ICERs of $16,887 to $79,955 per LYS. At prescription PPI cost, the ICER for average-risk patients was over $100,000 per LYS across all modeled age groups and assumptions of PPI effectiveness, but the ICER for high-risk patients was $10,433 to $51,505 per LYS. At OTC cost, PPI cotherapy is cost-effective in average-risk patients taking low-dose ASA for secondary prevention. At prescription cost, cotherapy is cost-effective for high-risk patients only.

Cost-Effectiveness of Intensive Lipid-Lowering Treatment for Patients with Congestive Heart Failure in the US

Introduction: Regular use of pharmacologic therapies such as lipid-lowering statins can decrease the risk of cardiovascular events. Furthermore, studies show that reducing LDL-C below recommended levels of 100 mg/dL with intensive (high-dose) statin therapy provides a greater clinical benefit. A recent study found fewer hospitalizations occurred for congestive heart failure (CHF) patients on high-dose than low-dose statin therapy. Hypothesis: Because high-dose statin therapy results in fewer hospitalizations and cardiovascular events versus more moderate therapy in CHF patients, it should prove to be cost effective. Methods: We constructed a lifetime Markov model designed to assess the cost-effectiveness of atorvastatin 80 mg (A80) versus atorvastatin 10 mg (A10) in CHF patients. Based on the atorvastatin therapy received, the model predicts the likelihood of experiencing major cardiovascular events (myocardial infarction, stroke, CHF hospitalization, revascularization, resuscitated cardiac arrest), minor cardiovascular events (peripheral artery disease, transient ischemic attack, angina), and death. Treatment-specific event probabilities were estimated from clinical trial data. Post-event survival, health-related quality of life, and medical-care costs were estimated using published sources. Study measures included incremental costs per life-year saved (LYS) and per quality-adjusted life-year (QALY) gained. All benefits and costs were discounted 3.0 % annually. Sensitivity analyses were performed to evaluate uncertainty regarding the effects of high-dose treatment on cardiovascular event rates and the long-term consequences of those events. Results: Treatment with A80 is estimated to increase per-patient QALYs, life years, and costs of care when compared with A10, with an incremental cost-effectiveness ratio (ICER) of $12,300 per LYS and $17,200 per QALY. In a probabilistic sensitivity analysis, the likelihood that A80 is cost-effective versus A10 (<$50,000) is 79.2% per LYS and 79.1% per QALY. Conclusions: Intensive A80 treatment is likely to be cost-effective compared to A10 in cardiovascular prevention in CHF patients in the US. The favorable cost-effectiveness is due in part to cost offsets (e.g., reduced costs of hospitalizations for CHF) and gains in life expectancy and health-related quality of life.

Cost-effectiveness analysis of routine rotavirus vaccination in Brazil

The objective of this study was to conduct a cost-effectiveness analysis of a universal rotavirus vaccination program among children < or = 5 years of age in Brazil. Considering a hypothetical annual cohort of approximately 3,300,000 newborns followed over 5 years, a decision-tree model was constructed to examine the possible clinical and economic effects of rotavirus infection with and without routine vaccination of children. Probabilities and unit costs were derived from published research and national administrative data. The impact of different estimates for key parameters was studied using sensitivity analysis. The analysis was conducted from both healthcare system and societal perspectives. The vaccination program was estimated to prevent approximately 1,735,351 (54%) of the 3 210 361 cases of rotavirus gastroenteritis and 703 (75%) of 933 rotavirus-associated deaths during the 5-year period. At a vaccine price of 18.6 Brazilian reais (R$) per dose, this program would cost R$121,673,966 and would save R$38,536,514 in direct costs to the public healthcare system and R$71,778,377 in direct and indirect costs to society. The program was estimated to cost R$1,028 and R$1,713 per life-years saved (LYS) from the societal and healthcare system perspectives, respectively. Universal rotavirus vaccination was a cost-effective strategy for both perspectives. However, these findings are highly sensitive to diarrhea incidence rate, proportion of severe cases, vaccine coverage, and vaccine price.

Calcitonin Measurement in the Evaluation of Thyroid Nodules in the United States: A Cost-Effectiveness and Decision Analysis

European studies have shown that the use of routine calcitonin screening for detection of medullary thyroid cancer (MTC) in patients with thyroid nodules increases the detection of occult MTC and may improve patient outcomes. Calcitonin screening for MTC has not been recommended in recent U.S. practice guidelines. Our objective was to determine the cost-effectiveness (C/E) of routine calcitonin screening in adult patients with thyroid nodules in the United States. A decision model was developed for a hypothetical group of adult patients presenting for evaluation of thyroid nodules in the United States. Patients were screened using current American Thyroid Association guidelines only, or American Thyroid Association guidelines with routine serum calcitonin screening. Input data were obtained from the literature, the Surveillance Epidemiology and End Results and Healthcare Cost and Utilization Project's Nationwide Inpatient Sample databases, and the Medicare Reimbursement Schedule. Sensitivity analyses were performed for a number of input variables. C/E, measured in dollars per life years saved (LYS), was calculated. Addition of calcitonin screening to current American Thyroid Association guidelines for the evaluation of thyroid nodules would cost $11,793 per LYS ($10,941-$12,646). When extrapolated to the national level, calcitonin screening for MTC in the United States would yield an additional 113,000 life years at a cost increase of 5.3%. Calcitonin screening C/E is sensitive to patient age and gender, and to changes in disease prevalence, specificity of fine needle aspiration and calcitonin testing, calcitonin screening level, costs of testing, and length of follow-up. Routine serum calcitonin screening in patients undergoing evaluation for thyroid nodules appears to be cost effective in the United States, with C/E comparable to the measurement of thyroid stimulating hormone, colonoscopy, and mammography screening.