October 2019


A Value of Information Analysis of Research on the 21-Gene Assay for Breast Cancer Management 

Kunst N, Alarid-Escudero F, Paltiel A, Wang SY.
Value in Health. 2019;22(10):1102-1110.

OBJECTIVES
The 21-gene assay Oncotype DX (21-GA) shows promise as a guide in deciding when to initiate adjuvant chemotherapy in women with hormone receptor–positive early-stage breast cancer. Nevertheless, its routine use remains controversial, owing to insufficient evidence of its clinical utility and cost-effectiveness. Accordingly, we aim to quantify the value of conducting further research to reduce decision uncertainty in the use of the 21-GA.

METHODS
Using value of information methods, we first generated probability distributions of survival and costs for decision making with and without the 21-GA alongside traditional risk prediction. These served as the input to a comparison of 3 alternative study designs: a retrospective observational study to update risk classification from the 21-GA, a prospective observational study to estimate prevalence of chemotherapy use, and a randomized controlled trial (RCT) of the 21-GA predictive value.

RESULTS
We found that current evidence strongly supports the use of the 21-GA in intermediate- and high-risk women. Further research should focus on low-risk women, among whom the cost-effectiveness findings remained equivocal. For this population, we identified a high value of reducing uncertainty in the 21-GA use for all proposed research studies. The RCT had the greatest potential to efficiently reduce the likelihood of choosing a suboptimal strategy, providing a value between $162 million and $1.1 billion at willingness-to-pay thresholds of $150 000 to $200 000/quality-adjusted life years.

CONCLUSION
Future research to inform 21-GA decision making is of high value. The RCT of the 21-GA predictive value has the greatest potential to efficiently reduce decision uncertainty around 21-GA use in women with low-risk early-stage breast cancer.

 

On the Optimization of Bayesian D-Efficient Discrete Choice Experiment Designs for the Estimation of QALY Tariffs That Are Corrected for Nonlinear Time Preferences

Doupe P, Faghmous J, Basu S.
Value in Health. 2019;22(7):808-815.

OBJECTIVE
This article explains how to optimize Bayesian D-efficient discrete choice experiment (DCE) designs for the estimation of quality-adjusted life year (QALY) tariffs that are unconfounded by respondents' time preferences.

METHODS
The calculation of Bayesian D-errors is explained for DCE designs that allow for the disentanglement of respondents' time and health-state preferences. Time preferences are modelled via an exponential, hyperbolic, or power discount function and the performance of the proposed DCE designs is compared with that of several conventional DCE designs that do not take nonlinear time preferences into account.

RESULTS
Based on the achieved D-error, asymptotic standard error, and estimated sample size to obtain statistically significant estimates of the discount rate parameters, the proposed designs outperform the conventional DCE designs.

CONCLUSION
We recommend that applied researchers use appropriately optimized DCE designs for the estimation of QALY tariffs that are corrected for time preferences. The TPC-QALY software package that accompanies this article makes the recommended designs easily accessible for health-state valuation researchers.

Implementing Patient-Reported Outcome Measures in Clinical Breast Cancer Care: A Systematic Review

van Egdom L, Oemrawsingh A, Verweij L, Lingsma H, Koppert L, Verhoef C, Klazinga N, Hazelzet J.
Value in Health. 2019;22(10):1197-1226.

BACKGROUND
Patient-reported outcome measures (PROMs) are increasingly being used to improve care delivery and are becoming part of routine clinical practice.

OBJECTIVE
This systematic review aims to give an overview of PROM administration methods and their facilitators and barriers in breast cancer clinical practice.

METHODS
A systematic literature search was conducted in Embase, MEDLINE, PsycINFO, Cochrane Central, CINAHL, and Web of Science for potentially relevant articles from study inception to November 2017. Reference lists of screened reviews were also checked. After inclusion of relevant articles, data were extracted and appraised by 2 investigators.

RESULTS
A total of 2311 articles were screened, of which 34 eligible articles were ultimately included. Method and frequency of PROM collection varied between studies. The majority of studies described a promising effect of PROM collection on patients (adherence, symptom distress, quality of life, acceptability, and satisfaction), providers (willingness to comply, clinical decision making, symptom management), and care process or system outcomes (referrals, patient-provider communication, hospital visits). A limited number of facilitators and barriers were identified, primarily of a technical and behavioral nature.

CONCLUSIONS
Although interpreting the impact of PROM collection in breast cancer care is challenging owing to considerations of synergistic (multicomponent) interventions and generalizability issues, this review found that systematic PROM collection has a promising impact on patients, providers, and care processes/ systems. Further standardization and reporting on method and frequency of PROM collection might help increase the effectiveness of PROM interventions and is warranted to enhance their overall impact.

November 2019


Cannabis and Health Research: Rapid Progress Requires Innovative Research Designs

Hutchinson K, Bidwell C, Ellingson J, Bryan A
Value in Health,  2019;22 (11), 1289 - 1294

ABSTRACT
The United States has witnessed enormous changes concerning the acceptance of medicinal and recreational cannabis use. Sixty-three percent of the US population has access to medicinal cannabis markets, which offer increasingly diverse and potent cannabis products. Considering the rapidly changing cultural, political, and legal landscape, the scientific literature does not adequately inform public policy, medical decision making, or harm reduction approaches. The goals of this paper are to (1) investigate the state of cannabis research on medical conditions commonly treated with cannabis, (2) review the barriers that have led to large gaps between cannabis use and available empirical data, and (3) suggest a path forward with new research designs to address these gaps. Thus, we aim to advance a more nuanced understanding of the barriers to cannabis research and suggest innovative research designs necessary for rapid development of a meaningful knowledge base.

 

Multicriteria Decision Analysis to Support Health Technology Assessment Agencies: Benefits, Limitations, and the Way Forward

Baltussen, Rob et al.
Value in Health,  2019;22 (11), 1283 - 1288

OBJECTIVE
Recent years have witnessed an increased interest in the use of multicriteria decision analysis (MCDA) to support health technology assessment (HTA) agencies for setting healthcare priorities. However, its implementation to date has been criticized for being “entirely mechanistic,” ignoring opportunity costs, and not following best practice guidelines. This article provides guidance on the use of MCDA in this context.

METHODS
The present study was based on a systematic review and consensus development. We developed a typology of MCDA studies and good implementation practice. We reviewed 36 studies over the period 1990 to 2018 on their compliance with good practice and developed recommendations. We reached consensus among authors over the course of several review rounds.

RESULTS
We identified 3 MCDA study types: qualitative MCDA, quantitative MCDA, and MCDA with decision rules. The types perform differently in terms of quality, consistency, and transparency of recommendations on healthcare priorities. We advise HTA agencies to always include a deliberative component. Agencies should, at a minimum, undertake qualitative MCDA. The use of quantitative MCDA has additional benefits but also poses design challenges. MCDA with decision rules, used by HTA agencies in The Netherlands and the United Kingdom and typically referred to as structured deliberation, has the potential to further improve the formulation of recommendations but has not yet been subjected to broad experimentation and evaluation.

CONCLUSIONS
MCDA holds large potential to support HTA agencies in setting healthcare priorities, but its implementation needs to be improved.

Pneumococcal Disease: A Systematic Review of Health Utilities, Resource Use, Costs, and Economic Evaluations of Interventions

Shiri T, Khan K, Keaney K, Mukherjee G, McCarthy N, Petrou S
Value in Health,  2019;22 (11), 1329 - 1344

BACKGROUND
Pneumococcal diseases cause substantial mortality, morbidity, and economic burden. Evidence on data inputs for economic evaluations of interventions targeting pneumococcal disease is critical.

OBJECTIVES
To summarize evidence on resource use, costs, health utilities, and cost-effectiveness for pneumococcal disease and associated interventions to inform future economic analyses.

METHODS
We searched MEDLINE, Embase, Web of Science, CINAHL, PsycINFO, EconLit, and Cochrane databases for peer-reviewed studies in English on pneumococcal disease that reported health utilities using direct or indirect valuation methods, resource use, costs, or cost-effectiveness of intervention programs, and summarized the evidence descriptively.

RESULTS
We included 383 studies: 9 reporting health utilities, 131 resource use, 160 economic costs of pneumococcal disease, 95 both resource use and costs, and 178 economic evaluations of pneumococcal intervention programs. Health state utility values ranged from 0 to 1 for both meningitis and otitis media and from 0.3 to 0.7 for both pneumonia and sepsis. Hospitalization was shortest for otitis media (range: 0.1-5 days) and longest for sepsis/septicemia (6-48). The main categories of costs reported were drugs, hospitalization, and household or employer costs. Resource use was reported in hospital length of stay and number of contacts with general practitioners. Costs and resource use significantly varied among population ages, disease conditions, and settings. Current vaccination programs for both adults and children, antibiotic use and outreach programs to promote vaccination, early disease detection, and educational programs are cost-effective in most countries.

CONCLUSIONS
This study has generated a comprehensive repository of health economic evidence on pneumococcal disease that can be used to inform future economic evaluations of pneumococcal disease intervention programs.

December 2019


Does the Institute for Clinical and Economic Review Revise Its Findings in Response to Industry Comments?

Cohen JT, Silver MC, Ollendorf DA, Neumann PJ
Value in Health,  2019;22 (12), 1396 - 1401

BACKGROUND
The Institute for Clinical and Economic Review (ICER) has gained prominance through its work conducting health technology assessments of pharmaceuticals in the United States.

OBJECTIVE
To understand the influence of industry comments on pharmaceutical value assessments conducted by ICER.

METHODS
We reviewed 15 ICER reports issued from 2017 through 2019. We quantified ICER’s revisions to its cost-effectiveness analysis (CEA) estimates between release of its draft and revised evidence reports and whether ratios shifted across ICER-specified categories of high, intermediate, or low value. We also reviewed industry-submitted comments recommending revision to ICER’s CEAs, noting ICER’s response as no change, text revised, assumption(s) revised, or conclusion revised. We evaluated each comment in terms of clarity, whether it offered an alternative to ICER’s approach, and whether it characterized the expected impact of revision on ICER’s analysis.

RESULTS
We identified 53 ICER-reported ratios. Of these, 45 (84.9%) changed between the draft and revised report, but 26 changes (57.8%) were small (10%). Six ratios shifted across value categories. We identified 256 industry comments recommending that ICER revise its CEA. Of these, 159 (62%) lacked clarity, 145 (57%) offered no alternative, and 243 (95%) did not characterize their impact on ICER’s estimated ratio. Ninety-one comments (35.5%) caused ICER to revise its assumptions, but only 5 (2.0%) caused ICER to revise its conclusions. Four of these 5 comments characterized their impact on ICER’s findings..

CONCLUSIONS
Changes in ICER’s estimates of cost-effectiveness between its draft and revised evidence reports are generally modest. Greater precision in industry comments could increase the influence of industry critiques, thus enhancing the dialogue around pharmaceutical value.

Excess Costs and Economic Burden of Obesity-Related Cancers in the United States

Hong YR, Huo J, Desai R, Cardel M, Deshmukh A
Value in Health,  2019;22 (12), 1378 - 1386

BACKGROUND
Obesity is a significant risk factor of several cancers that imposes a substantial economic burden on US healthcare that remains to be quantified. We estimated the excess costs and economic burden of obesity-related cancers in the United States.

METHODS
From the Medical Expenditure Panel Survey (2008-2015) data, we identified 19 405 cancer survivors and 175 498 non-cancer individuals. We estimated annual health expenditures using generalized linear regression with log link and gamma distribution by cancer types (stratified by 11 obesity-related cancers and other cancer types), controlling for sociodemographic and clinical characteristics. All cost estimates were adjusted to 2015 USD value.

RESULTS
The average annual total health expenditures were $21 503 (95% CI, $20 946-$22 061) for those with obesity-related cancer and $13 120 (95% CI, $12 920-$13 319) for those with other cancer types. There was a positive association between body mass index and health expenditures among cancer survivors: for each additional 5-unit increase in body mass index, the average predicted expenditures increase by $1503 among those with obesity-related cancer and by $722 among those with other cancers. With adjustments for sociodemographic and clinical characteristics, the mean incremental expenditures of treating obesity-related cancer were 2.1 times higher than those of other cancers ($4492 vs $2139) and more considerable among the non-elderly cancer population. Obesity-related cancers accounted for nearly 43.5% of total direct cancer care expenditures, estimated at $35.9 billion in 2015.

CONCLUSIONS
The economic burden of obesity-related cancer in the United States is substantial. Our findings suggest a need for the inclusion of comprehensive obesity prevention and treatment in cancer care.

How Should We Capture Health State Utility in Dementia? Comparisons of DEMQOL-Proxy-U and of Self- and Proxy-Completed EQ-5D-5L

Martin A, Meads D, Griffiths A, Surr C
Value in Health,  2019;22 (12), 1417 - 1426

BACKGROUND
Dementia-specific and proxy-completed preference-based measures have been proposed for use in intervention studies involving people living in residential care, in instances where generic, self-reported preference-based measures have been deemed inappropriate.

OBJECTIVE
This study was conducted to investigate the construct validity, criterion validity, and responsiveness of DEMQOL-Proxy-U and of self- and proxy-completed EQ-5D-5L.

METHODS
The analysis used a 3-wave, individual-level data set of 1004 people living with dementia in residential care that included self-completed EQ-5D-5L and formal-carer and informal-carer proxy-completed EQ-5D-5L and DEMQOL-Proxy-U utility values, in addition to other nonutility cognitive measures (Functional Assessment Staging [FAST], Clinical Dementia Rating [CDR], Cohen-Mansfield Agitation Inventory [CMAI]) and health-related quality of life (HRQOL) measures (nursing home version of the Quality of Life with Alzheimer's disease scale [QOL-AD-NH], Quality of Life in Late-Stage Dementia [QUALID] scale). Construct validity, criterion validity, and responsiveness were assessed using correlation, Bland-Altman plots, and panel data regression models.

RESULTS
Self-completed EQ-5D-5L failed to reflect clinically important differences and changes in FAST, CDR, and CMAI but did capture the resident's own view of HRQOL (QOL-AD-NH). As dementia severity increased, collection of EQ-5D-5L-proxy and DEMQOL-Proxy-U data was more feasible than collection of self-completed EQ-5D-5L. These formal-carer and informal-carer proxy measures also better reflected changes in FAST, CDR, and CMAI but did not capture the resident's own view of HRQOL (QOL-AD-NH), despite adequately capturing the proxy's own view of the resident's HRQOL (QUALID). This indicates discrepancies between a proxy's view and resident's view of the impact that tangible declines in health, cognition, or functional abilities have on HRQOL. The EQ-5D-5L-proxy and DEMQOL-Proxy-U were generally poor substitutes. Regardless of which proxy completed it, the EQ-5D-5L-proxy was typically more responsive than the DEMQOL-Proxy-U to changes in CDR, FAST, and CMAI, indicating that use of the DEMQOL-Proxy-U is not always justified.

CONCLUSIONS
Disparities in the measurement properties of different utility measures mean that choices about how to measure utility in trials could affect economic evaluation outcomes and hence how resources are allocated for dementia care.

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