Comparative effectiveness research (CER) has the potential to slow health care spending growth by focusing resources on health interventions that provide the most value. In this article, we discuss issues surrounding CER and its implementation and apply these methods to a salient clinical example: treatment of prostate cancer. Physicians have several options for treating patients recently diagnosed with localized disease, including removal of the prostate (radical prostatectomy), treatment with radioactive seeds (brachytherapy), radiation therapy (IMRT), or-if none of these are pursued- active surveillance. Using a commercial health insurance claims database and after adjustment for comorbid conditions, we estimate that the additional cost of treatment with radical prostatectomy is $7,300, while other alternatives are more expensive-$19,000 for brachytherapy and $46,900 for IMRT. However, a review of the clinical literature uncovers no evidence that justifi es the use of these more expensive approaches. These results imply that if patient management strategies were shifted to those supported by CER-based criteria, an estimated $1.7 to $3.0 billion (2009 present value) could be saved each year.

Liver disease and liver cancer associated with childhood-acquired chronic hepatitis B are
leading causes of death among adults in China. Despite expanded newborn hepatitis B
vaccination programs, approximately 20% of children under age 5 years and 40% of children aged 5 to 19 years remain unprotected from hepatitis B. Although immunizing them will be beneficial, no studies have examined the cost-effectiveness of hepatitis B catch-up vaccination in an endemic country like China. We examined the cost-effectiveness of a hypothetical nationwide free hepatitis B catch-up vaccination program in China for unvaccinated children and adolescents aged 1 to 19 years. We used a Markov model for disease progression and infections. Cost variables were based on data published by the Chinese Ministry of Health, peer-reviewed Chinese and English publications, and the GAVI Alliance.

We measured costs (2008 U.S. dollars and Chinese renminbi), quality-adjusted life years,and incremental cost-effectiveness from a societal perspective. Our results show that hepatitis B catch-up vaccination for children and adolescents in China is cost-saving across a range of parameters, even for adolescents aged 15 to 19 years old. We estimate that if all 150 million susceptible children under 19 were vaccinated, more than 8 million infections and 65,000 deaths due to hepatitis B would be prevented. Conclusion: The adoption of a nationwide free catch-up hepatitis B vaccination program for unvaccinated children and adolescents in China, in addition to ongoing efforts to improve birth dose and newborn vaccination coverage, will be cost-saving and can generate significant population-wide health benefits. The success of such a program in China could serve as a model for other endemic countries.

Background— Family members of patients with established long-QT syndrome (LQTS) often lack definitive clinical findings, yet may have inherited an LQTS mutation and be at risk of sudden death. Genetic testing can identify mutations in 75% of patients with LQTS, but genetic testing of family members remains controversial.

Methods and Results— We used a Markov model to assess the cost-effectiveness of 3 strategies for treating an asymptomatic 10-year-old, first-degree relative of a patient with clinically evident LQTS. In the genetic testing strategy, relatives undergo genetic testing only for the mutation identified in the index patient, and relatives who test positive for the mutation are treated with β-blockers. This strategy was compared with (1) empirical treatment of relatives with β-blockers and (2) watchful waiting, with treatment only after development of symptoms. The genetic testing strategy resulted in better survival and quality-adjusted life years at higher cost, with a cost-effectiveness ratio of $67 400 per quality-adjusted life year gained compared with watchful waiting. The cost-effectiveness of the genetic testing strategy improved to less than $50 000 per quality-adjusted life year gained when applied selectively either to (1) relatives with higher clinical suspicion of LQTS (pretest probability 65% to 81%), or to (2) families with a higher than average risk of sudden death, or to (3) larger families (2 or more first-degree relatives tested).

Conclusions— Genetic testing of young first-degree relatives of patients with definite LQTS is moderately expensive, but can reach acceptable thresholds of cost-effectiveness when applied to selected patients.

The major expansion of federal comparative effectiveness research launched in 2009 held the potential to supply the information needed to help slow health spending growth while improving the outcomes of care. However, when Congress passed the Patient Protection and Affordable Care Act one year later, it limited the role of cost analysis in the work sponsored by the Patient-Centered Outcomes Research Institute. Despite this restriction, cost-effectiveness analysis meets important needs and is likely to play a larger role in the future. Under the terms of the Affordable Care Act, the institute can avoid commissioning cost-effectiveness analyses and still provide information bearing on the use and costs of health care interventions. This information will enable others to investigate the comparative value of these interventions. We argue that doing so is necessary to decision makers who are attempting to raise the quality of care while reining in health spending.

BACKGROUND: -Many myocardial infarctions and strokes occur in individuals with low-density lipoprotein cholesterol levels below recommended treatment thresholds. High sensitivity C-reactive protein (hs-CRP) testing has been advocated to identify low- and intermediate-risk individuals who may benefit from statin therapy. Methods and Results-A decision analytic Markov model was used to follow hypothetical cohorts of individuals with normal lipid levels but without coronary artery disease, peripheral arterial disease, or diabetes mellitus. The model compared current Adult Treatment Panel III practice guidelines, a strategy of hs-CRP screening in those without an indication for statin treatment by current practice guidelines followed by treatment only in those with elevated hs-CRP levels, and a strategy of statin therapy at specified predicted risk thresholds without hs-CRP testing. Risk-based treatment without hs-CRP testing was the most cost-effective strategy, assuming that statins were equally effective regardless of hs-CRP status. However, if normal hs-CRP levels identified a subgroup with little or no benefit from statin therapy (<20% relative risk reduction), then hs-CRP screening would be the optimal strategy. If harms from statin use were greater than generally recognized, then use of current clinical guidelines would be the optimal strategy. Conclusion-Risk-based statin treatment without hs-CRP testing is more cost-effective than hs-CRP screening, assuming that statins have good long-term safety and provide benefits among low-risk people with normal hs-CRP.

With the awareness of maternal depression as a prevalent public health issue and its important link to child physical and mental health, attention has turned to how healthcare providers can respond effectively. Intimate partner violence (IPV) and the use of alcohol, tobacco, and other drugs are strongly related to depression, particularly for low-income women. The American College of Obstetricians and Gynecologists (ACOG) recommends psychosocial screening of pregnant women at least once per trimester, yet screening is uncommonly done. Research suggests that a collaborative care approach improves identification, outcomes, and cost-effectiveness of care. This article presents The Perinatal Mental Health Model, a community-based model that developed screening and referral partnerships for use in community obstetric settings in order to specifically address the psychosocial needs of culturally diverse, low-income mothers.

BACKGROUND: Universal testing and treatment holds promise for reducing the burden of human immunodeficiency virus (HIV) in sub-Saharan Africa, but linkage from testing to treatment sites and retention in care are inadequate.

METHODS: We developed a simulation of the HIV epidemic and HIV disease progression in South Africa to compare the outcomes of the present HIV treatment campaign (status quo) with 4 HIV testing and treating strategies that increase access to antiretroviral therapy: (1) universal testing and treatment without changes in linkage to care and loss to follow-up; (2) universal testing and treatment with improved linkage to care; (3) universal testing and treatment with reduced loss to follow-up; and (4) comprehensive HIV care with universal testing and treatment, improved linkage to care, and reduced loss to follow-up. The main outcome measures were survival benefits, new HIV infections, and HIV prevalence. 

RESULTS: Compared with the status quo strategy, universal testing and treatment (1) was associated with a mean (95% uncertainty bounds) life expectancy gain of 12.0 months (11.3-12.2 months), and 35.3% (32.7%-37.5%) fewer HIV infections over a 10-year time horizon. Improved linkage to care (2), prevention of loss to follow-up (3), and comprehensive HIV care (4) provided substantial additional benefits: life expectancy gains compared with the status quo strategy were 16.1, 18.6, and 22.2 months, and new infections were 55.5%, 51.4%, and 73.2% lower, respectively. In sensitivity analysis, comprehensive HIV care reduced new infections by 69.7% to 76.7% under a broad set of assumptions.

CONCLUSIONS: Universal testing and treatment with current levels of linkage to care and loss to follow-up could substantially reduce the HIV death toll and new HIV infections. However, increasing linkage to care and preventing loss to follow-up provides nearly twice the benefits of universal testing and treatment alone.