Review of Methods of Incorporating Mortality in Cost Effectiveness Analyses of Lipid-Lowering Therapies Explores Limitations of Randomized Controlled Trial Data

BEVERLY HILLS, Calif., June 1, 2017 /PRNewswire/ -- As healthcare expenditures in the US rise, there is increasing scrutiny of the value of new therapies. One method of estimating value is cost effectiveness analysis, a well-established framework for assessing the costs and clinical outcomes of an innovation. One area of potential confusion in analyses of cardiovascular disease interventions is how mortality is incorporated in these cost effectiveness analyses.

Specifically, cost effectiveness studies may either limit themselves to studying only things for which a randomized controlled trial has found a statistically significant benefit, or they may use other ways to estimate the effect of a given intervention on mortality.

Partnership for Health Analytic Research (PHAR, LLC), a leading health services research consultancy, published a systematic review titled "A Systematic Literature Review of Methods of Incorporating Mortality in Cost Effectiveness Analyses of Lipid-Lowering Therapies" in the peer-reviewed Journal of Medical Economics that helps clarify how these studies are typically done.

The new study carefully examined 100 published cost effectiveness analyses related to cardiovascular disease. The authors found that 99/100 of studies assumed the intervention they studied improved mortality, even though 88/100 studies did not have direct, statistically significant randomized controlled trial evidence to support this benefit.

Similarly, among the 41 studies that included multiple lipid-lowering therapies, as opposed to those comparing a single agent versus placebo, 40 assumed a mortality benefit, but none were supported by direct randomized controlled trial evidence of a mortality benefit.

The explanation for these findings is the lack of available data. A prior review of randomized controlled trials found that only 3/27 trials showed a statistically significant mortality effect. These 3 trials were unique in that they compared an active therapy to placebo, were conducted between 1997 and 2002, and had a longer median study duration of 5-6 years. As many recent trials are not designed to assess mortality, researchers must be prepared to incorporate available data into analyses.

As Dr. Michael S. Broder, MD, MSHS, President of PHAR, notes: "One might expect that treatment efficacy would always be based on randomized controlled trials with clear outcome differences. But FDA requirements for trials often mean the trials are too small, and observation periods too short, to see a clear mortality benefit."

As a result of the study, Dr. Broder believes that given the limitations of randomized controlled trial data, using the best available evidence, including intermediate or composite outcomes, is the right approach methodologically.

About:
Founded in 2004, Partnership for Health Analytic Research (PHAR, LLC) is a clinically-focused health services research consultancy. PHAR combines rigorous methodology with extensive clinical expertise to conduct high quality health economics and outcomes research for life sciences companies, specialty societies, and not-for-profit health advocacy groups. For more information about PHAR, LLC, visit the company's website at www.pharllc.com.

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SOURCE PHAR, LLC