Heijnsdijk EAM, Denham D, De Koning HJ.
Value in Health. 2016;19(2):153-157.
Diabetes is one of the most prevalent and costly chronic diseases in the United States.
To analyze the risk of developing diabetes and the annual cost of diabetes for a US general population.
Data from the Medical Expenditure Panel Survey, 2008 to 2012, were used to analyze 1) probabilities of developing diabetes and 2) annual total health care expenditures for diabetics. The age-, sex-, race-, and body mass index (BMI)-specific risks of developing diabetes were estimated by fitting an exponential survival function to age at first diabetes diagnosis. Annual health care expenditures were estimated using a generalized linear model with log-link and gamma variance function. Complex sampling designs in the Medical Expenditure Panel Survey were adjusted for. All dollar values are presented in 2012 US dollars.
We observed a more than 6 times increase in diabetes risks for class III obese (BMI ≥ 40 kg/m2) individuals compared with normal-weight individuals. Using age 50 years as an example, we found a more than 3 times increase in annual health care expenditures for those with diabetes ($13,581) compared with those without diabetes ($3,954). Compared with normal-weight (18.5 ≤ BMI < 25 kg/m2) individuals, class II obese (35 ≤ BMI < 40 kg/m2) and class III obese (BMI ≥ 40 kg/m2) individuals incurred an annual marginal cost of $628 and $756, respectively. The annual health care expenditure differentials between those with and without diabetes of age 50 years were the highest for individuals with class II ($12,907) and class III ($9,703) obesity.
This article highlights the importance of obesity on diabetes burden. Our results suggested that obesity, in particular, class II and class III (i.e., BMI ≥ 35 kg/m2) obesity, is associated with a substantial increase in the risk of developing diabetes and imposes a large economic burden.
Li Y, Arellano AR, Bare LA, Bender RA, Strom CM, Devlin JJ.
Value in Health. 2017;20(4):547-555.
The National Comprehensive Cancer Network recommends that women who carry gene variants that confer substantial risk for breast cancer consider risk-reduction strategies, that is, enhanced surveillance (breast magnetic resonance imaging and mammography) or prophylactic surgery. Pathogenic variants can be detected in women with a family history of breast or ovarian cancer syndromes by multigene panel testing.
To investigate whether using a seven-gene test to identify women who should consider risk-reduction strategies could cost-effectively increase life expectancy.
We estimated effectiveness and lifetime costs from a payer perspective for two strategies in two hypothetical cohorts of women (40-year-old and 50-year-old cohorts) who meet the National Comprehensive Cancer Network–defined family history criteria for multigene testing. The two strategies were the usual test strategy for variants in BRCA1 and BRCA2 and the seven-gene test strategy for variants in BRCA1, BRCA2, TP53, PTEN, CDH1, STK11, and PALB2. Women found to have a pathogenic variant were assumed to undergo either prophylactic surgery or enhanced surveillance.
The incremental cost-effectiveness ratio for the seven-gene test strategy compared with the BRCA1/2 test strategy was $42,067 per life-year gained or $69,920 per quality-adjusted life-year gained for the 50-year-old cohort and $23,734 per life-year gained or $48,328 per quality-adjusted life-year gained for the 40-year-old cohort. In probabilistic sensitivity analysis, the seven-gene test strategy cost less than $100,000 per life-year gained in 95.7% of the trials for the 50-year-old cohort.
Testing seven breast cancer–associated genes, followed by risk-reduction management, could cost-effectively improve life expectancy for women at risk of hereditary breast cancer.
Garfield S, Polisena J, Spinner DS, Postulka A, Lu CY, Tiwana SK, Faulkner E, Poulios N, Zah V, Longacre M.
Value in Health. 2016;19(5):577-587.
Health technology assessments (HTAs) are increasingly used to inform coverage, access, and utilization of medical technologies including molecular diagnostics (MDx). Although MDx are used to screen patients and inform disease management and treatment decisions, there is no uniform approach to their evaluation by HTA organizations.
The International Society for Pharmacoeconomics and Outcomes Research Devices and Diagnostics Special Interest Group reviewed diagnostic-specific HTA programs and identified elements representing common and best practices.
MDx-specific HTA programs in Europe, Australia, and North America were characterized by methodology, evaluation framework, and impact. Published MDx HTAs were reviewed, and five representative case studies of test evaluations were developed: United Kingdom (National Institute for Health and Care Excellence's Diagnostics Assessment Programme, epidermal growth factor receptor tyrosine kinase mutation), United States (Palmetto's Molecular Diagnostic Services Program, OncotypeDx prostate cancer test), Germany (Institute for Quality and Efficiency in Healthcare, human papillomavirus testing), Australia (Medical Services Advisory Committee, anaplastic lymphoma kinase testing for non–small cell lung cancer), and Canada (Canadian Agency for Drugs and Technologies in Health, Rapid Response: Non-invasive Prenatal Testing).
Overall, the few HTA programs that have MDx-specific methods do not provide clear parameters of acceptability related to clinical and analytic performance, clinical utility, and economic impact. The case studies highlight similarities and differences in evaluation approaches across HTAs in the performance metrics used (analytic and clinical validity, clinical utility), evidence requirements, and how value is measured. Not all HTAs are directly linked to reimbursement outcomes.
To improve MDx HTAs, organizations should provide greater transparency, better communication and collaboration between industry and HTA stakeholders, clearer links between HTA and funding decisions, explicit recognition of and rationale for differential approaches to laboratory-developed versus regulatory-approved test, and clear evidence requirements.
Marshall DA, Gonzalez JM, MacDonald KV, Johnson FR.
Value in Health. 2017;20(1):32-39.
We examine key study design challenges of using stated-preference methods to estimate the value of whole-genome sequencing (WGS) as a specific example of genomic testing. Assessing the value of WGS is complex because WGS provides multiple findings, some of which can be incidental in nature and unrelated to the specific health concerns that motivated the test. In addition, WGS results can include actionable findings (variants considered to be clinically useful and can be acted on), findings for which evidence for best clinical action is not available (variants considered clinically valid but do not meet as high of a standard for clinical usefulness), and findings of unknown significance. We consider three key challenges encountered in designing our national study on the value of WGS—layers of uncertainty, potential downstream consequences with endogenous aspects, and both positive and negative utility associated with testing information—and potential solutions as strategies to address these challenges. We conceptualized the decision to acquire WGS information as a series of sequential choices that are resolved separately. To determine the value of WGS information at the initial decision to undergo WGS, we used contingent valuation questions, and to elicit respondent preferences for reducing risks of health problems and the consequences of taking the steps to reduce these risks, we used a discrete-choice experiment. We conclude by considering the implications for evaluating the value of other complex health technologies that involve multiple forms of uncertainty.
Mühlbacher A, Johnson FR, Yang J-C, Happich M, Belger M.
Value in Health. 2016;19(1):66-74.
The diagnosis of Alzheimer's disease (AD) remains difficult. Lack of diagnostic certainty or possible distress related to a positive result from diagnostic testing could limit the application of new testing technologies. The objective of this paper is to quantify respondents' preferences for obtaining AD diagnostic tests and to estimate the perceived value of AD test information.
Discrete-choice experiment and contingent-valuation questions were administered to respondents in Germany and the United Kingdom. Choice data were analyzed by using random-parameters logit. A probit model characterized respondents who were not willing to take a test.
Most respondents indicated a positive value for AD diagnostic test information. Respondents who indicated an interest in testing preferred brain imaging without the use of radioactive markers. German respondents had relatively lower money-equivalent values for test features compared with respondents in the United Kingdom.
Respondents preferred less invasive diagnostic procedures and tests with higher accuracy and expressed a willingness to pay up to €700 to receive a less invasive test with the highest accuracy.
Watts RD, Li IW, Geelhoed EA, Sanfilippo FM, St. John A.
Value in Health. 2017;20(8):1210-1215.
Concerns about pathology testing such as the value provided by new tests and the potential for inappropriate utilization have led to a greater need to assess costs and benefits. Economic evaluations are a formal method of analyzing costs and benefits, yet for pathology tests, questions remain about the scope and quality of the economic evidence.
To describe the extent and quality of published evidence provided by economic evaluations of pathology tests from 2010 to 2015.
Economic evaluations relating to pathology tests from 2010 to 2015 were reviewed. Eight databases were searched for published studies, and details recorded for the country, clinical focus, type of testing, and consideration of sensitivity, specificity, and false test results. The reporting quality of studies was assessed using the Consolidated Health Economic Evaluation Reporting Standards checklist and cost-effectiveness ratios were analyzed for publication bias.
We found 356 economic evaluations of pathology tests, most of which regarded developed countries. The most common economic evaluations were cost-utility analyses and the most common clinical focus was infectious diseases. More than half of the studies considered sensitivity and specificity, but few studies considered the impact of false test results. The average Consolidated Health Economic Evaluation Reporting Standards checklist score was 17 out of 24. Cost-utility ratios were commonly less than $10,000/quality-adjusted life-year or more than $200,000/quality-adjusted life-year.
The number of economic evaluations of pathology tests has increased in recent years, but the rate of increase has plateaued. Furthermore, the quality of studies in the past 5 years was highly variable, and there is some question of publication bias in reporting cost-effectiveness ratios.