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1.  Assessing Progress toward Becoming a Patient-Centered Medical Home: An Assessment Tool for Practice Transformation 
Health Services Research  2013;48(6 Pt 1):1879-1897.
Objective. To describe the properties of the Patient-Centered Medical Home Assessment (PCMH-A) as a tool to stimulate and monitor progress among primary care practices interested in transforming to patient-centered medical homes (PCMHs).
Study Setting. Sixty-five safety net practices from five states participating in a national demonstration program for PCMH transformation.
Study Design. Longitudinal analyses of PCMH-A scores were performed. Scores were reviewed for agreement and sites were categorized over time into one of five categories by external facilitators. Comparisons to key activity completion rates and NCQA PCMH recognition status were completed.
Data Collection/Extraction Methods. Multidisciplinary teams at each practice completed the 33-item self-assessment tool every 6 months between March 2010 and September 2012.
Principal Findings. Mean overall PCMH-A scores increased (7.2, March 2010, to 9.1, September 2012; [p < .01]). Increases were statistically significant for each of the change concepts (p < .05). Facilitators agreed with scores 82% of the time. NCQA-recognized sites had higher PCMH-A scores than sites that were not yet recognized. Sites that completed more transformation activities and progressed over defined tiers reported higher PCMH-A scores. Scores improved most in areas where technical assistance was provided.
Conclusions. The PCMH-A was sensitive to change over time and provided an accurate reflection of practice transformation.
doi:10.1111/1475-6773.12111
PMCID: PMC3876398  PMID: 24138593
Process assessment; patient-centered care; primary health care
2.  Private Companies Providing Health Care Price Data: Who Are They and What Information do They Provide? 
Summary
There is interest in making health care price information more transparent given the increase in enrollment in high-deductible and consumer-directed health plans, and as policy efforts intensify to engage consumers to obtain high value care. We examine the role of private companies that market price transparency tools, primarily to self-insured employers – an important yet understudied topic. What companies exist? How did they emerge? What information do they provide? Where do they get that information? How does the price and quality information provided compare across companies?
PMCID: PMC4323083
3.  How do physician assessments of patient preferences for colorectal cancer screening tests differ from actual preferences? A comparison in Canada and the United States using a stated-choice survey 
Health economics  2009;18(12):1420-1439.
Background
Patient preferences can affect colorectal cancer screening test use. We compared utility-based preferences for alternative CRC screening tests from a stated-preference discrete-choice survey of the general population and physicians in Canada and the United States.
Methods
General population respondents (Canada, n=501; US, n=1087) participated in a survey with twelve choice scenarios and nine CRC screening test attributes. Physicians (n=100, both Canada and US) reported expected patient preferences. We estimated relative importance of attributes using bivariate probit regression analysis and calculated willingness-to-pay for various CRC screening tests.
Results
In 28% and 31% of scenarios, Canadian and US respondents, respectively, chose no screening over a hypothetical test. Canadian (45%) and US (46%) physicians expected patients to choose no screening more often.
For all groups the most important attribute was sensitivity, but physicians’ perception of patients’ preferences are significantly different from actual preferences. Other key attributes are those related to test performance or the testing process. Fecal DNA, colonoscopy, and virtual colonoscopy were the most preferred tests by all groups, but respondents were willing-to-pay more than physicians predicted.
Conclusion
Physicians’ perception of patients’ preferences are quite different from those of the general population. However, among general population and physicians, Canadian and US preferences were similar.
doi:10.1002/hec.1437
PMCID: PMC3964796  PMID: 19191268
colorectal cancer; screening; willingness-to-pay; discrete-choice; stated-preference
4.  The economic value of personalized medicine tests: what we know and what we need to know 
Purpose
There is uncertainty about when personalized medicine tests provide economic value. We assessed evidence on the economic value of personalized medicine tests and gaps in the evidence base.
Methods
We created a unique evidence base by linking data on published cost–utility analyses from the Tufts Cost-Effectiveness Analysis Registry with data measuring test characteristics and reflecting where value analyses may be most needed: (i) tests currently available or in advanced development, (ii) tests for drugs with Food and Drug Administration labels with genetic information, (iii) tests with demonstrated or likely clinical utility, (iv) tests for conditions with high mortality, and (v) tests for conditions with high expenditures.
Results
We identified 59 cost–utility analyses studies that examined personalized medicine tests (1998–2011). A majority (72%) of the cost/quality-adjusted life year ratios indicate that testing provides better health although at higher cost, with almost half of the ratios falling below $50,000 per quality-adjusted life year gained. One-fifth of the results indicate that tests may save money.
Conclusion
Many personalized medicine tests have been found to be relatively cost-effective, although fewer have been found to be cost saving, and many available or emerging medicine tests have not been evaluated. More evidence on value will be needed to inform decision making and assessment of genomic priorities.
doi:10.1038/gim.2013.122
PMCID: PMC3949119  PMID: 24232413
cost-effectiveness; economic value; ethical/legal/social implications (ELSI); genetic tests; personalized medicine
5.  HOW DOES COST MATTER IN HEALTH-CARE DISCRETE-CHOICE EXPERIMENTS? 
Health economics  2011;20(3):323-330.
SUMMARY
Willingness-to-pay (WTP) estimates derived from discrete-choice experiments (DCEs) generally assume that the marginal utility of income is constant. This assumption is consistent with theoretical expectations when costs are a small fraction of total income. We analyze the results of five DCEs that allow direct tests of this assumption. Tests indicate that marginal utility often violates theoretical expectations. We suggest that this result is an artifact of a cognitive heuristic that recodes cost levels from a numerical scale to qualitative categories. Instead of evaluating nominal costs in the context of a budget constraint, subjects may recode costs into categories such as ‘low’, ‘medium’, and ‘high’ and choose as if the differences between categories were equal. This simplifies the choice task, but undermines the validity of WTP estimates as welfare measures. Recoding may be a common heuristic in healthcare applications when insurance coverage distorts subjects’ perception of the nominal costs presented in the DCE instrument. Recoding may also distort estimates of marginal rates of substitution for other attributes with numeric levels. Incorporating ‘cheap talk’ or graphic representation of attribute levels may encourage subjects to be more attentive to absolute attribute levels.
doi:10.1002/hec.1591
PMCID: PMC3918954  PMID: 20217834
willingness to pay; discrete-choice experiments; decision heuristics; treatment cost
6.  Strategies to Identify the Lynch Syndrome Among Patients With Colorectal Cancer 
Annals of internal medicine  2011;155(2):69-79.
Background
Testing has been advocated for all persons with newly diagnosed colorectal cancer to identify families with the Lynch syndrome, an autosomal dominant cancer-predisposition syndrome that is a paradigm for personalized medicine.
Objective
To estimate the effectiveness and cost-effectiveness of strategies to identify the Lynch syndrome, with attention to sex, age at screening, and differential effects for probands and relatives.
Design
Markov model that incorporated risk for colorectal, endometrial, and ovarian cancers.
Data Sources
Published literature.
Target Population
All persons with newly diagnosed colorectal cancer and their relatives.
Time Horizon
Lifetime.
Perspective
Third-party payer.
Intervention
Strategies based on clinical criteria, prediction algorithms, tumor testing, or up-front germline mutation testing, followed by tailored screening and risk-reducing surgery.
Outcome Measures
Life-years, cancer cases and deaths, costs, and incremental cost-effectiveness ratios.
Results of Base-Case Analysis
The benefit of all strategies accrued primarily to relatives with a mutation associated with the Lynch syndrome, particularly women, whose life expectancy could increase by approximately 4 years with hysterectomy and salpingo-oophorectomy and adherence to colorectal cancer screening recommendations. At current rates of germline testing, screening, and prophylactic surgery, the strategies reduced deaths from colorectal cancer by 7% to 42% and deaths from endometrial and ovarian cancer by 1% to 6%. Among tumor-testing strategies, immunohistochemistry followed by BRAF mutation testing was preferred, with an incremental cost-effectiveness ratio of $36 200 per life-year gained.
Results of Sensitivity Analysis
The number of relatives tested per proband was a critical determinant of both effectiveness and cost-effectiveness, with testing of 3 to 4 relatives required for most strategies to meet a threshold of $50 000 per life-year gained. Immunohistochemistry followed by BRAF mutation testing was preferred in 59% of iterations in probabilistic sensitivity analysis at a threshold of $100 000 per life-year gained. Screening for the Lynch syndrome with immunohistochemistry followed by BRAF mutation testing only up to age 70 years cost $44 000 per incremental life-year gained compared with screening only up to age 60 years, and screening without an upper age limit cost $88 700 per incremental life-year gained compared with screening only up to age 70 years.
Limitation
Other types of cancer, uncertain family pedigrees, and genetic variants of unknown significance were not considered.
Conclusion
Widespread colorectal tumor testing to identify families with the Lynch syndrome could yield substantial benefits at acceptable costs, particularly for women with a mutation associated with the Lynch syndrome who begin regular screening and have risk-reducing surgery. The cost-effectiveness of such testing depends on the participation rate among relatives at risk for the Lynch syndrome.
Primary Funding Source
National Institutes of Health.
doi:10.7326/0003-4819-155-2-201107190-00002
PMCID: PMC3793257  PMID: 21768580
7.  Economic Evaluation of Targeted Cancer Interventions: Critical Review and Recommendations 
Scientific advances have improved our ability to target cancer interventions to individuals who will benefit most, and spare the risks and costs to those who will derive little benefit or even be harmed. Several approaches are currently used for targeting interventions for cancer risk reduction, screening and treatment, including risk prediction algorithms for identifying high-risk subgroups and diagnostic tests for tumor markers and germline genetic mutations. Economic evaluation can inform decisions about the use of targeted interventions, which may be more costly than traditional strategies. However, assessing the impact of a targeted intervention on costs and health outcomes requires explicit consideration of the method of targeting. Here we describe the importance of this principle by reviewing published cost-effectiveness analyses (CEAs) of targeted interventions in breast cancer. Few studies we identified explicitly evaluated the relationship between the method of targeting, the accuracy of the targeting test and outcomes of the targeted intervention. Those that did found that characteristics of targeting tests had a substantial impact on outcomes. We posit that the method of targeting and the outcomes of a targeted intervention are inextricably linked and recommend that CEAs of targeted interventions explicitly consider costs and outcomes of the method of targeting.
doi:10.1097/GIM.0b013e31821f3e64
PMCID: PMC3774033  PMID: 21637102
breast cancer; economic analysis; cost-effectiveness analysis; targeted therapy; personalized medicine; BRCA; trastuzumab; gene expression profiling
8.  Personalized Medicine and Oncology Practice Guidelines: A Case Study of Contemporary Biomarkers in Colorectal Cancer 
Predictive and prognostic biomarkers offer a potential means to personalize cancer medicine, although many reach the marketplace before they have been validated, and their adoption is often hindered by variable clinical evidence. Because of this variability in supporting evidence, clinical practice guidelines formulated by panels of subspecialty experts may be particularly important in guiding stakeholders’ acceptance and use of new personalized medicine biomarker tests and other nascent technologies. This article provides a structured review of the clinical evidence supporting 4 contemporary biomarker tests in colorectal cancer: K-ras and B-raf mutation analyses, mismatch repair protein testing, and the Oncotype DX Colon Cancer Assay. All 4 tests have been evaluated for guideline inclusion by the NCCN Guidelines Panel for Colon Cancer. This case study shows significant variability in the level of clinical evidence associated with these tests. In the cases of B-raf and mismatch repair protein testing, the available evidence is also inconsistent as it pertains to the specific NCCN guideline recommendation. Based on this uncertainty in the evidence base, the authors conclude that expert clinical judgment, experience, and consensus may be more heavily weighted than published clinical trial data in the evaluation of new personalized medicine biomarker tests. Potential implications of this conclusion and future directions for research are discussed.
PMCID: PMC3695822  PMID: 21233242
Biomarker; personalized medicine; guidelines; KRAS; BRAF; microsatellite instability; Oncotype DX Colon Cancer Assay
9.  Gene Expression Profile Testing for Breast Cancer and the Use of Chemotherapy, Serious Adverse Effects, and Costs of Care 
Background
As gene expression profile (GEP) testing for breast cancer may provide additional prognostic information to guide the use of adjuvant chemotherapy, we examined the association between GEP testing and use of chemotherapy, serious chemotherapy-related adverse effects, and total charges during the 12 months following diagnosis.
Methods
Medical record review was conducted for women age 30 to 64 years, with incident, non-metastatic, invasive breast cancer diagnosed 2006–2008 in a large, national health plan.
Results
Of 534 patients, 25.8% received GEP testing, 68.2% received chemotherapy, and 10.5% experienced a serious chemotherapy-related adverse effect. GEP testing was most commonly used in women at moderate clinical risk of recurrence (52.0% vs. 25.0% of low-risk women and 5.5% of high-risk). Controlling for the propensity to receive GEP testing, women who had GEP were less likely to receive chemotherapy (propensity adjusted odds ratio, 95% confidence interval 0.62, 0.39 – 0.99). Use of GEP was associated with more chemotherapy use among women at low risk based on clinical characteristics (OR = 42.19; CI 2.50 – 711.82), but less use among women with a high risk based on clinical characteristics (OR = 0.12 CI 0.03 – 0.47). Use of GEP was not associated with chemotherapy for the moderate risk group. There was no significant relationship between GEP use and either serious chemotherapy-associated adverse effects or total charges.
Conclusions
While GEP testing was associated with an overall decrease in adjuvant chemotherapy, we did not find differences in serious chemotherapy-associated adverse events or charges during the 12 months following diagnosis.
doi:10.1007/s10549-011-1628-6
PMCID: PMC3590013  PMID: 21681446
breast cancer; utilization; genomics
10.  Effectiveness of public health programs for decreasing alcohol consumption 
Patient intelligence  2011;2011(3):29-38.
Excessive alcohol consumption and the associated negative consequences are a major public health concern in the United States and throughout the world. Historically, there have been numerous attempts to develop policies and prevention programs aimed at decreasing high-risk alcohol use. Policy initiatives have demonstrated considerable effectiveness and include changes in the minimum legal drinking age, reductions in acceptable legal limits for blood alcohol concentration while operating a motor vehicle, as well as decreasing availability and access to alcohol for underage individuals. Primary prevention programs that have used exclusively educational approaches have received mixed results. Increasing effectiveness has been associated with prevention programs that have utilized a multi-component approach and have included educational initiatives with environmental changes.
doi:10.2147/PI.S12431
PMCID: PMC3505028  PMID: 23180975
alcohol abuse; underage; public health; programs; binge drinking
11.  Influence of Patient Preferences on the Cost-Effectiveness of Screening for Lynch Syndrome 
Journal of Oncology Practice  2012;8(3 Suppl):e24s-e30s.
This cost-utility analysis reports on the effect of quality of life on the value of screening all new patients with colorectal cancer for Lynch syndrome.
Purpose:
Patients and relatives have varying preferences for genetic testing and interventions related to hereditary cancer syndromes. We examined how the impact of these services on quality of life (QoL) affects the cost-effectiveness of screening for Lynch syndrome among probands newly diagnosed with colorectal cancer and their relatives.
Methods:
We constructed a state-transition model comparing screening strategies (clinical criteria, prediction algorithms, tumor testing, and upfront germline testing) with no screening to identify Lynch syndrome. The model incorporated individuals' health state utilities after screening, germline testing, and risk-reducing surgeries, with utilities persisting for 12 months in the base case. Outcomes consisted of quality-adjusted life-years (QALYs), costs, and cost per QALY gained. Sensitivity analyses assessed how the duration and magnitude of changes in QoL influenced results.
Results:
Multiple screening strategies yielded gains in QALYs at acceptable costs compared with no screening. The preferred strategy—immunohistochemistry of tumors followed by BRAF mutation testing (IHC/BRAF)—cost $59,700 per QALY gained in the base case. The duration and magnitude of decreases in QoL after decisions related to germline testing and surgeries were key determinants of the cost-effectiveness of screening. IHC/BRAF cost > $100,000 per QALY gained when decrements to QoL persisted for 21 months.
Conclusion:
Screening for Lynch syndrome in the population is likely to yield long-term gains in life expectancy that outweigh any short-term decreases in QoL, at acceptable costs. Counseling for individuals should aim to mitigate potential negative impact of genetic testing and risk-reducing interventions on QoL.
doi:10.1200/JOP.2011.000535
PMCID: PMC3348599  PMID: 22942831
12.  Tradeoffs of Using Administrative Claims and Medical Records to Identify the Use of Personalized Medicine for Patients with Breast Cancer 
Medical Care  2011;49(6):e1-e8.
Background
Administrative claims and medical records are important data sources to examine healthcare utilization and outcomes. Little is known about identifying personalized medicine technologies in these sources.
Objectives
To describe agreement, sensitivity, and specificity of administrative claims compared to medical records for two pairs of targeted tests and treatments for breast cancer.
Research Design
Retrospective analysis of medical records linked to administrative claims from a large health plan. We examined whether agreement varied by factors that facilitate tracking in claims (coding and cost) and that enhance medical record completeness (records from multiple providers).
Subjects
Women (35 – 65 years) with incident breast cancer diagnosed in 2006–2007 (n=775).
Measures
Use of human epidermal growth factor receptor 2 (HER2) and gene expression profiling (GEP) testing, trastuzumab and adjuvant chemotherapy in claims and medical records.
Results
Agreement between claims and records was substantial for GEP, trastuzumab, and chemotherapy, and lowest for HER2 tests. GEP, an expensive test with unique billing codes, had higher agreement (91.6% vs. 75.2%), sensitivity (94.9% vs. 76.7%), and specificity (90.1% vs. 29.2%) than HER2, a test without unique billing codes. Trastuzumab, a treatment with unique billing codes, had slightly higher agreement (95.1% vs. 90%) and sensitivity (98.1% vs. 87.9%) than adjuvant chemotherapy.
Conclusions
Higher agreement and specificity were associated with services that had unique billing codes and high cost. Administrative claims may be sufficient for examining services with unique billing codes. Medical records provide better data for identifying tests lacking specific codes and for research requiring detailed clinical information.
doi:10.1097/MLR.0b013e318207e87e
PMCID: PMC3383782  PMID: 21422962
medical record; claims data; breast neoplasm; personalized medicine
13.  Genomic Testing and Therapies for Breast Cancer in Clinical Practice 
Journal of Oncology Practice  2011;7(3 Suppl):e1s-e7s.
Despite almost universal testing for HER2, many women with HER2-positive cancer may not receive trastuzumab. Fewer women received newer gene expression profile testing.
Purpose:
Given the likely proliferation of targeted testing and treatment strategies for cancer, a better understanding of the utilization patterns of human epidermal growth factor receptor 2 (HER2) testing and trastuzumab and newer gene expression profiling (GEP) for risk stratification and chemotherapy decision making are important.
Study Design:
Cross-sectional.
Methods:
We performed a medical record review of women age 35 to 65 years diagnosed between 2006 and 2007 with invasive localized breast cancer, identified using claims from a large national health plan (N = 775).
Results:
Almost all women received HER2 testing (96.9%), and 24.9% of women with an accepted indication received GEP. Unexplained socioeconomic differences in GEP use were apparent after adjusting for age and clinical characteristics; specifically, GEP use increased with income. For example, those in the lowest income category (< $40,000) were less likely than those with an income of $125,000 or more to receive GEP (odds ratio, 0.34; 95% CI, 0.16 to 0.73). A majority of women (57.7%) with HER2-positive disease received trastuzumab; among these women, differences in age and clinical characteristics were not apparent, although surprisingly, those in the lowest income category were more likely than those in the high-income category to receive trastuzumab (P = .02). Among women who did not have a positive HER2 test, 3.9% still received trastuzumab. Receipt of adjuvant chemotherapy increased as GEP score indicated greater risk of recurrence.
Conclusion:
Identifying and eliminating unnecessary variation in the use of these expensive tests and treatments should be part of quality improvement and efficiency programs.
doi:10.1200/JOP.2011.000299
PMCID: PMC3092459  PMID: 21886507
14.  Health Technology Assessment and Private Payers' Coverage of Personalized Medicine 
Journal of Oncology Practice  2011;7(3 Suppl):18s-24s.
A study of major US private payers showed an important role and considerable shortcomings of external health technology assessment in coverage decisions on personalized medicine.
Purpose:
Health technology assessment (HTA) plays an increasing role in translating emerging technologies into clinical practice and policy. Private payers are important users of HTA whose decisions impact adoption and use of new technologies. We examine the current use of HTA by private payers in coverage decisions for personalized medicine, a field that is increasingly impacting oncology practice.
Study Design:
Literature review and semistructured interviews.
Methods:
We reviewed seven HTA organizations used by private payers in decision making and explored how HTA is used by major US private payers (n = 11) for coverage of personalized medicine.
Results:
All payers used HTA in coverage decisions, but the number of HTA organizations used by an individual payer ranged from one (n = 1) to all seven (n = 1), with the majority of payers (n = 8) using three or more. Payers relied more extensively on HTAs for reviews of personalized medicine (64%) than for other technologies. Most payers (82%) equally valued expertise of reviewers and rigor of evaluation as HTA strengths, whereas genomic-specific methodology was less important. Key reported shortcomings were limited availability of reviews (73%) and limited inclusion of nonclinical factors (91%), such as cost-effectiveness or adoption of technology in clinical practice.
Conclusion:
Payers use a range of HTAs in their coverage decisions related to personalized medicine, but the current state of HTA to comprehensively guide those decisions is limited. HTA organizations should address current gaps to improve their relevance to payers and clinicians. Current HTA shortcomings may also inform the national HTA agenda.
doi:10.1200/JOP.2011.000300
PMCID: PMC3092460  PMID: 21886515
15.  Capacity building for assessing new technologies: approaches to examining personalized medicine in practice 
Personalized medicine  2010;7(4):427-439.
This article focuses on the overarching question: how can we use existing data to develop the capacity to improve the evidence base on personalized medicine technologies and particularly regarding their utilization and clinical utility? We focus on data from health payers who are key stakeholders in capacity building, as they need data to guide decisions and they develop data as part of operations. Broadly defined, health payers include insurance carriers, third party payers, health-plan sponsors and organized delivery systems. Data from health payers have not yet been widely used to assess personalized medicine. Now, with an increasing number of personalized technologies covered and reimbursed by health payers, and an increasing number of emerging technologies that will require policy decisions, there is a great opportunity to develop the evidence base using payer data and by engaging with these stakeholders. Here, we describe data that are available from, and are being developed by, health payers and assess how these data can be further developed to increase the capacity for future research, using three examples. The examples suggest that payer data can be used to examine clinical utility and approaches can be developed that simultaneously address the characteristics of personalized medicine, real world data and organizations. These examples can now help us to elucidate how to best examine clinical utility in actual practice and build evaluation approaches that can be applied to future technologies.
doi:10.2217/pme.10.36
PMCID: PMC3157083  PMID: 21857867
cancer; data development; evidence development; health payer; organized delivery system; personalized medicine; pharmacy benefits manager
16.  Effects of Simplifying Choice Tasks on Estimates of Taste Heterogeneity in Stated-Choice Surveys 
Social science & medicine (1982)  2009;70(2):183-190.
Researchers usually employ orthogonal arrays or D-optimal designs with little or no attribute overlap in stated-choice surveys. The challenge is to balance statistical efficiency and respondent burden to minimize the overall error in the survey responses. This study examined whether simplifying the choice task, by using a design with more overlap, provides advantages over standard minimum-overlap methods. We administered two designs for eliciting HIV test preferences to split samples. Surveys were undertaken at four HIV testing locations in San Francisco, California. Personal characteristics had different effects on willingness to pay for the two treatments, and gains in statistical efficiency in the minimal-overlap version more than compensated for possible imprecision from increased measurement error.
doi:10.1016/j.socscimed.2009.10.021
PMCID: PMC3152257  PMID: 19880234
stated-choice approach; experimental design; overlap; taste heterogeneity; USA; HIV testing
17.  Coverage Policy Development for Personalized Medicine: Private Payer Perspectives on Developing Policy for the 21-Gene Assay 
Journal of Oncology Practice  2010;6(5):238-242.
A challenge of personalized medicine is the limited clinical evidence for many personalized medicine technologies. Here, the strategies private payers use to develop coverage policy for personalized medicine are described using the example of the 21-gene assay in breast cancer.
Purpose:
Personalized medicine is changing oncology practice and challenging decision making. A key challenge is the limited clinical evidence for many personalized medicine technologies. We describe the strategies private payers employed to develop coverage policy for personalized medicine using the example of the 21-gene assay in breast cancer.
Methods:
We examined the coverage policies of six private payers for the 21-gene assay. We then interviewed senior executives (n = 7) from these payers to elucidate factors informing coverage decisions. We additionally focused on the timing of payer decisions compared with the timing of evidence development, measured by publication of primary studies and relevant clinical guidelines.
Results:
The 21-gene assay became commercially available in 2004. The interviewed payers granted coverage between 2005 and 2008. Their policies varied in structure (eg, whether prior authorization was required). All payers reported clinical evidence as the most important factor in decision making, but all used some health care system factors (eg, physician adoption or medical society endorsement) to inform decision making as well. Payers had different perceptions about the strength of clinical evidence at the time of the coverage decision.
Conclusion:
Coverage of the 21-gene assay is currently widespread, but policies differ in timing and structure. A key approach private payers use to develop coverage policies for novel technologies is considering both clinical evidence and health care system factors. Policy variation may emerge from the range of factors used and perception of the evidence. Future research should examine the role of health care system factors in policy development and related policy variations.
doi:10.1200/JOP.000075
PMCID: PMC2936466  PMID: 21197187
18.  Clinical Practice Patterns and Cost-Effectiveness of HER2 Testing Strategies in Breast Cancer Patients 
Cancer  2009;115(22):5166-5174.
Background
Testing technologies are increasingly used to target cancer therapies. Human epidermal growth factor receptor 2 (HER2) testing to target trastuzumab for patients with breast cancer provides insights into the evidence needed for emerging testing technologies.
Methods
We reviewed literature on HER2 test utilization and cost-effectiveness of HER2 testing for patients with breast cancer. We examined available evidence on: percentage of eligible patients tested for HER2; test methods used; concordance of test results between community and central/reference laboratories; use of trastuzumab by HER2 test result; and cost-effectiveness of testing strategies.
Results
Little evidence is available to determine whether all eligible patients are tested; how many are retested to confirm results; and how many with negative HER2 test results still receive trastuzumab. Studies suggest that up to 66% of eligible patients had no documentation of testing in claims records; up to 20% of patients receiving trastuzumab were not tested or had no documentation of a positive test; and 20% of HER2 results may be incorrect. Few cost-effectiveness analyses of trastuzumab explicitly considered the economic implications of various testing strategies.
Conclusions
There is little information about the actual use of HER2 testing in clinical practice, but evidence suggests important variations in testing practices and key gaps in knowledge exist. Given the increasing use of targeted therapies, it is critical to build an evidence base that supports informed decision-making on emerging testing technologies in cancer care.
doi:10.1002/cncr.24574
PMCID: PMC2783254  PMID: 19753618
personalized medicine; targeted therapies; genomics; HER2; trastuzumab; breast cancer; utilization; cost-effectiveness; clinical practice patterns
19.  Challenges To The Translation Of Genomic Information Into Clinical Practice And Health Policy: Utilization, Preferences, And Economic Value 
It is important to understand how knowledge of genomics can be translated from basic research into clinical practice and health policies. The objective of this paper is to review existing evidence on three key factors in the adoption of personalized medicine – utilization, preferences, and economic value - using two cancer examples: HER2/neu testing and trastuzumab (Herceptin®) and genetic testing for Lynch syndrome. Our findings suggest where further research is needed to build an evidence base addressing utilization of, preferences for, and the potential costs and benefits of personalized medicine. Major challenges include a lack of linked data, the need for relevant research frameworks and methodologies, and the clinical complexities of genomic-based diagnostics and treatment.
PMCID: PMC2910510  PMID: 18535933
Personalized medicine; health policy; health services research; economics; utilization; preferences
21.  Addressing The Challenges Of The Clinical Application Of Pharmacogenetic Testing 
Pharmacogenomics aims to use molecular genetic markers to predict treatment outcome. Indeed within the past decade, there has been a rapid emergence of pharmacogenetic tests to aid clinicians to predict efficacy or toxicity for some drugs. Despite this major advance in therapeutic drug management there remain challenges to the appropriate use of pharmacogenetic tests. We discuss UGT1A1 pharmacogenetic testing to illustrate the knowledge gaps impeding widespread use of pharmacogenetic tests in the clinical setting.
doi:10.1038/clpt.2009.30
PMCID: PMC2910521  PMID: 19536122
22.  Looking back at 10 years of trastuzumab therapy: what is the role of HER2 testing? A systematic review of health economic analyses 
Personalized medicine  2009;6(2):193-215.
Trastuzumab is a targeted therapy for human EGF receptor-2 (HER2)-positive breast cancer. The effectiveness and cost–effectiveness of trastuzumab hinges not only on its clinical efficacy in responding patients, but on the ability to accurately identify appropriate therapeutic candidates. We sought to systematically review the cost–effectiveness of trastuzumab with a focus on the impact of the test(s) used for HER2 diagnosis. Our review included 17 economic evaluations or health technology assessments of trastuzumab therapy or HER2 testing. Trastuzumab was considered cost-effective in all early-stage disease studies, while one author concluded that trastuzumab was not cost-effective for metastatic disease. Only two papers considered the joint effects of test accuracy and sequencing with trastuzumab therapy. These demonstrated that trastuzumab cost–effectiveness is sensitive to HER2-test properties.
doi:10.2217/17410541.6.2.193
PMCID: PMC2910630  PMID: 20668661
cost–effectiveness; cost-utility; HER2; herceptin; sensitivity; specificity; targeted therapy; trastuzumab
23.  A Health Services Research Agenda for Cellular, Molecular and Genomic Technologies in Cancer Care 
Public Health Genomics  2009;12(4):233-244.
Background
In recent decades, extensive resources have been invested to develop cellular, molecular and genomic technologies with clinical applications that span the continuum of cancer care.
Methods
In December 2006, the National Cancer Institute sponsored the first workshop to uniquely examine the state of health services research on cancer-related cellular, molecular and genomic technologies and identify challenges and priorities for expanding the evidence base on their effectiveness in routine care.
Results
This article summarizes the workshop outcomes, which included development of a comprehensive research agenda that incorporates health and safety endpoints, utilization patterns, patient and provider preferences, quality of care and access, disparities, economics and decision modeling, trends in cancer outcomes, and health-related quality of life among target populations.
Conclusions
Ultimately, the successful adoption of useful technologies will depend on understanding and influencing the patient, provider, health care system and societal factors that contribute to their uptake and effectiveness in ‘real-world’ settings.
doi:10.1159/000203779
PMCID: PMC2844634  PMID: 19367091
Genomics; Health services research; Emerging technologies; Translational research
24.  MEDICARE FORMULARY COVERAGE FOR TOP-SELLING BIOLOGICS 
Nature biotechnology  2009;27(12):1082-1084.
doi:10.1038/nbt1209-1082
PMCID: PMC2845541  PMID: 20010576
25.  Angiotensin Receptor Blockers on the Formularies of Medicare Drug Plans 
Journal of General Internal Medicine  2007;22(8):1172-1175.
Background
The presence of angiotensin receptor blockers (ARBs) on the formularies of Medicare Part D prescription drug plans (PDPs) is vitally important to the health of seniors who cannot tolerate angiotensin-converting enzyme (ACE) inhibitors.
Objective
To determine whether ARBs are present on the formularies of PDPs and how the prescription cost-sharing for ARBs under Part D compares to cost-sharing before Part D.
Design/Participants
Cross-sectional analyses of March 2006 Medicare Part D formularies (n = 1,446) and of ARB utilization and cost-sharing for adults over the age of 64 included in the nationally representative Medical Expenditure Panel Survey.
Main Outcome Measures
(1) Presence of ARBs on Part D formularies. (2) Average out-of-pocket costs for 30-day supply of ARBs before and after Part D (both in 2006 dollars).
Results
All PDP formularies included at least 1 ARB. Most plans covered 2 ARBs (41%) and 35% covered all 7. The average monthly copay for the most commonly used ARB, valsartan, is $28 under part D, $14 before Part D for individuals with prescription coverage, and $53 before Part D for individuals without coverage.
Conclusions
Whereas ARBs are present on all Part D formularies, many seniors will pay more for these drugs under Part D. Any savings in copayments under Part D may be erased by the monthly premium and by more expensive cost-sharing when seniors reach the ‘donut hole’.
doi:10.1007/s11606-007-0235-z
PMCID: PMC2305745  PMID: 17503103
Medicare; health insurance; formularies; angiotensin receptor blockers

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