[What is a cost-benefit analysis?].

Purpose It is uncertain whether modest benefits from adjuvant chemotherapy in stage II colorectal cancer justify the toxicity, cost, and inconvenience. We investigated the usefulness of defective mismatch repair (dMMR), BRAF, and KRAS mutations in predicting tumor recurrence and sensitivity to chemotherapy. Patients and Methods Immunohistochemistry for dMMR and pyrosequencing for KRAS/BRAF were performed for 1,913 patients randomly assigned between fluorouracil and folinic acid chemotherapy and no chemotherapy in the Quick and Simple and Reliable (QUASAR) trial. Results Twenty-six percent of 695 right-sided colon, 3% of 685 left-sided colon, and 1% of 407 rectal tumors were dMMR. Similarly, 17% of right colon, 2% of left colon, and 2% of rectal tumors were BRAF mutant. KRAS mutant tumors were more evenly distributed: 40% right colon, 28% left colon, and 36% rectal tumors. Recurrence rate for dMMR tumors was half that for MMR-proficient tumors (11% [25 of 218] v 26% [438 of 1,695] recurred; risk ratio [RR]...

https://ascopubs.org/doi/pdf/10.1200/jco.2010.30.1366

Value of Mismatch Repair, KRAS, and BRAF Mutations in Predicting Recurrence and Benefits From Chemotherapy in Colorectal Cancer

Microsatellite instability as a marker of prognosis and response to therapy: a meta-analysis of colorectal cancer survival data.

Summary Background The diagnosis of hypertension has traditionally been based on blood-pressure measurements in the clinic, but home and ambulatory measurements better correlate with cardiovascular outcome, and ambulatory monitoring is more accurate than both clinic and home monitoring in diagnosing hypertension. We aimed to compare the cost-effectiveness of different diagnostic strategies for hypertension. Methods We did a Markov model-based probabilistic cost-effectiveness analysis. We used a hypothetical primary-care population aged 40 years or older with a screening blood-pressure measurement greater than 140/90 mm Hg and risk-factor prevalence equivalent to the general population. We compared three diagnostic strategies—further blood pressure measurement in the clinic, at home, and with an ambulatory monitor—in terms of lifetime costs, quality-adjusted life years, and cost-effectiveness. Findings Ambulatory monitoring was the most cost-effective strategy for the diagnosis of hypertension for men and women of all ages. It was cost-saving for all groups (from −£56 [95% CI −105 to −10] in men aged 75 years to −£323 [−389 to −222] in women aged 40 years) and resulted in more quality-adjusted life years for men and women older than 50 years (from 0·006 [0·000 to 0·015] for women aged 60 years to 0·022 [0·012 to 0·035] for men aged 70 years). This finding was robust when assessed with a wide range of deterministic sensitivity analyses around the base case, but was sensitive if home monitoring was judged to have equal test performance to ambulatory monitoring or if treatment was judged effective irrespective of whether an individual was hypertensive. Interpretation Ambulatory monitoring as a diagnostic strategy for hypertension after an initial raised reading in the clinic would reduce misdiagnosis and save costs. Additional costs from ambulatory monitoring are counterbalanced by cost savings from better targeted treatment. Ambulatory monitoring is recommended for most patients before the start of antihypertensive drugs. Funding National Institute for Health Research and the National Institute for Health and Clinical Excellence.

https://www.thelancet.com/pdfs/journals/lancet/PIIS0140-6736(11)61184-7.pdf

Cost-effectiveness of options for the diagnosis of high blood pressure in primary care: a modelling study.

'This report gives the latest information on how Aboriginal and Torres Strait Islander people in the Northern Territory are faring according to a range of measures of health status and outcomes, determinants of health and health system performance. Indicators are based on the Aboriginal and Torres Strait Islander Health Performance Framework. The report highlights the main areas of improvement and continuing concern.' - Back cover

http://www.territorystories.nt.gov.au/jspui/bitstream/10070/270343/1/Health%20performance%20frame%20work%20-%20Northern%20Territory.pdf

Aboriginal and Torres Strait Islander health performance framework

Evidence of clinical utility is a key issue in translating pharmacogenomics into clinical practice. Appropriately designed randomized controlled trials generally provide the most robust evidence of the clinical utility, but often only data from a pharmacogenomic association study are available. This paper details a method for reframing the results of pharmacogenomic association studies in terms of the comparative treatment effect for a pharmacogenomic subgroup to provide greater insight into the likely clinical utility of a pharmacogenomic marker, its’ likely cost effectiveness, and the value of undertaking the further (often expensive) research required for translation into clinical practice. The method is based on the law of total probability, which relates marginal and conditional probability. It takes as inputs: the prevalence of the pharmacogenomic marker in the patient group of interest, prognostic effect of the pharmacogenomic marker based on observational association studies, and the unstratified comparative treatment effect based on one or more conventional randomized controlled trials. The critical assumption is that of exchangeability across the included studies. The method is demonstrated using a case study of cytochrome P450 (CYP) 2C19 genotype and the anti-platelet agent clopidogrel. Indirect subgroup analysis provided insight into relationship between the clinical utility of genotyping CYP2C19 and the risk ratio of cardiovascular outcomes between CYP2C19 genotypes for individuals using clopidogrel. In this case study the indirect and direct estimates of the treatment effect for the cytochrome P450 2C19 subgroups were similar. In general, however, indirect estimates are likely to have substantially greater risk of bias than an equivalent direct estimate.

/pdf/indirect-estimation-of-the-comparative-treatment-effect-in-426810wl69.pdf

Indirect estimation of the comparative treatment effect in pharmacogenomic subgroups.

For a number of years pharmacogenomics has held substantial promise with regard to improving use of drug therapy, but meaningful translation to the clinic has generally been disappointing. One of a number of important criteria that influences this successful translation is the cost–effectiveness of routinely assessing pharmacogenomic markers prior to initiating drug therapy [1,2]. Here we will highlight and briefly explore four key issues relating to the cost–effectiveness of pharmacogenomics:

Cost-effectiveness of genotyping to guide treatment.

This report presents the results of a qualitative study of the perspectives and experiences of country Aboriginal patients (and some of their immediate family and carers) who received health care in Adelaide public hospitals. This study is part of a larger project that aims to add to existing knowledge of what works well and what needs improvement in the system of care for Aboriginal patients from rural and remote areas of South Australia.

Managing Two Worlds Together: Study 3—The Experiences of Patients and Their Carers

Reducing the gap between the need for and access to effective and best practice care at the end-of-life is a goal of the Australian palliative care sector. Largely absent from the suite of plans, s...

The role of ambulance services in reducing the gaps in access to end-of-life care: From conversations to policy

This report presents four case studies that map the patient journeys of country Aboriginal patients and carers who travelled from rural or remote South Australia to Adelaide for hospital care. It analyses the common elements in these journeys and their implications for health care providers. This study is part of the Managing Two Worlds Together project, and its goal is to illustrate the complexities involved in these real patient journeys, and how city and country health services prepare for and respond to these complexities

Brita Pekarsky

Papers

Indirect estimation of the comparative treatment effect in pharmacogenomic subgroups.

Cost-effectiveness of genotyping to guide treatment.

Managing Two Worlds Together: Study 3—The Experiences of Patients and Their Carers

The role of ambulance services in reducing the gaps in access to end-of-life care: From conversations to policy

Managing two worlds together : Study 4 - Complex country Aboriginal patient journeys