The quality of economic studies of cancer pharmacogenomics: a quantitative appraisal of the evidence.

This study evaluated the quality of health economic studies of cancer pharmacogenomics (PGx). A systematic search of the literature for economic studies of PGx was conducted in four common cancers. Evaluation of study quality was carried out using the quality of health economic studies instrument. Thirty-nine articles met our eligibility criteria and were selected and accepted for further statistical analyses. The majority of articles (85%) were studies focusing on breast cancer. The overall weighted mean quality score was 85.10, with a range from 21 to 100. Eighty-seven percent of articles were categorized as good quality, whereas some 10 and 3% were categorized as moderate and poor quality, respectively. The quality of economic studies of cancer PGx is generally good but varied widely. We identified several attributes that are predictive of quality. Our findings may be useful for oncologists, health economists and decision makers interested in evaluating studies involving PGx.

A national study of breast and colorectal cancer patients' decision-making for novel personalized medicine genomic diagnostics.

AIM: Molecular diagnostics are increasingly being used to help guide decision-making for personalized medical treatment of breast and colorectal cancer patients. The main aim of this study was to better understand and determine breast and colorectal cancer patients' decision-making strategies and the trade-offs they make in deciding about characteristics of molecular genomic diagnostics for breast and colorectal cancer. PATIENTS & METHODS: We surveyed a nationally representative sample of 300 breast and colorectal cancer patients using a previously developed web-administered instrument. Eligibility criteria included patients aged 18 years and older with either breast or colorectal cancer. We explored several attributes and attribute levels of molecular genomic diagnostics in 20 scenarios. RESULTS: Our analysis revealed that both breast and colorectal cancer patients weighted the capability of molecular genomic diagnostics to determine the probability of treatment efficacy as being of greater importance than information provided to detect adverse events. The probability of either false-positive or -negative results was ranked highly as a potential barrier by both breast and colorectal patients. However, 78.6% of breast cancer patients ranked the possibility of a 'false-negative test result leading to undertreatment' higher than the 'chance of a false positive, which may lead to overtreatment' (68%). This finding contrasted with the views of colorectal cancer patients who ranked the chance of a false positive as being of greater concern than a false negative (72.8 vs 63%). Overall, cancer patients exhibited a high willingness to accept and pay for genomic diagnostic tests, especially among breast cancer patients. Cancer patients seek a test accuracy rate of 90% or higher. Breast and colorectal cancer patients' decisions about genomic diagnostics are influenced more by the probability of being cured than by avoiding potential severe adverse events. CONCLUSION: This study provides insights into the relative weight that breast and colorectal cancer patients place on various aspects of molecular genomic diagnostics, and the trade-offs they are willing to make among attributes of such tests.

Provision of personalized genomic diagnostic technologies for breast and colorectal cancer: an analysis of patient needs, expectations and priorities.

AIM: Several novel pharmacogenomic diagnostic tests are commercially available for breast and colorectal cancer, and are increasingly being used in clinical practice for improving treatment decisions. However, there is little evidence evaluating the value of these new genomic technologies from the perspective of patients. As part of an ongoing effort to understand the continuum of the process of adoption of genomic diagnostics, our aim in this study was to examine the value of genomic diagnostics to breast and colorectal cancer patients, and their willingness to adopt and use genomic diagnostics. PATIENTS & METHODS: We conducted six focus groups of breast and colorectal cancer patients from the oncology clinics at The Methodist Hospital, Houston, TX, USA. An adapted Q-sort instrument was also administered to focus group participants. RESULTS: The majority of breast and colorectal cancer patients are interested in using novel genomic diagnostics for deciding about treatment options. Most participants in our study expressed a willingness to pay out-of-pocket for genomic testing (z = 0.736). Reliability and validity of genomic testing were of significant concern (z = 1.32) for the majority of breast and colorectal cancer patients. Participants identified several facilitators and barriers within health systems that might either facilitate or impede the widespread adoption and use of genomic diagnostics in healthcare delivery. CONCLUSION: This study demonstrates breast and colorectal cancer patients' willingness to adopt and pay for novel genomic diagnostics, as well as identifies several salient factors associated with patient preferences for genomic diagnostics.

The quality of pharmacoeconomic evaluations of age-related macular degeneration therapeutics: a systematic review and quantitative appraisal of the evidence.

AIM: To appraise the quality of published pharmacoeconomic studies of therapeutic interventions for age-related macular degeneration (AMD). METHODS: Systematic review of the literature and evaluation of study quality using the Quality of Health Economic Studies instrument. A systematic search of the English-language literature for economic studies of therapeutic interventions for AMD from 1990 to March 2008 was performed. RESULTS: A total of 3637 articles were initially identified. Only 24 met eligibility criteria and were rated using the Quality of Health Economic Studies. The mean quality overall rating was 61.6, with quality scores ranging from 18 to 92. There was a higher mean quality score in the studies designed as clinical trials versus observational type designed studies (mean=74.7(11.4), 52.6 (16.5) respectively, p=0.002) and studies in which the statistical analyses were clearly presented versus studies in which the statistical analyses were not so clear (mean=74.3 (12.3), 53.1 (16.1) respectively, p=0.004). Interestingly, government funded studies exhibited a similar mean quality score to studies that were funded by industry (mean=71.0 (15.1), 61.7 (18.5) respectively, p=0.25). A general linear model was fitted using those independent variables which were significantly associated with quality score. The variables 'study design' and 'statistics presented clearly' were found to be jointly significant and explained nearly 70% of the variation in the dependent variable (R(2)=0.68). CONCLUSIONS: Our analysis reveals that the methodological quality of the health economic analysis of AMD therapeutic interventions in the literature is suboptimal. There is considerable variation in methodological rigour between the articles, and we have identified several attributes that are predictive of study quality.

Innovation in personalized medicine: BiDil® as a case study for integrating clinical and policy developments.

BiDil® (hydralazine and isosorbide dinitrate) represents an interesting application of personalized medicine - the first pharmaceutical specifically approved by a regulatory agency, the US FDA, for an indication in a particular population based on race as a surrogate phenotypic marker, without a companion genomic diagnostic directed at measuring drug responsiveness. The focus of this paper is to use BiDil as a case study of a personalized medicine application and evaluate its clinical and policy-relevant characteristics as an illustrative example of the usefulness of the Evaluation Data for Assessing Personalized Medicine Translation (EDAPT) evidence base.