Benefits of Comparative Effectiveness Research

(July 26, 2011)

Clinical research is a broad term covering many aspects of investigating health and disease to establish new facts, ideas or connections. It is often used to describe experimental studies that examine the effect of interventions on patient or community populations. One type of clinical research that is receiving much attention recently due to the Patient Protection and Affordable Care Act under the Obama administration is Comparative Effectiveness Research (CER).

The Patient Protection and Affordable Care Act calls for the increased visibility of CER as a critical component of the nation’s portfolio of biomedical research. CER is the direct comparison of the benefits and harms of two or more health care interventions used to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The aim of CER is to learn which treatments work best, for whom, and under what circumstances in order to be able to assist patients, clinicians, purchasers and policy-makers in making informed health care decisions at both the individual and national levels in a timely manner.

Established in 2000, the American Lung Association Asthma Clinical Research Centers (ACRC) network has always known the value of CER and results of our effectiveness studies have had a direct impact on patient care in the field of asthma. In its first CER trial, the network examined whether a low-dose of a relatively inexpensive asthma medicine (theophylline), a drug that had fallen out of favor with doctors due to concerns over side effects, could be used safely and effectively as an alternative to the widely used, more expensive, add-on asthma therapy, montelukast.

The study titled “The Effectiveness of Low Dose Theophylline as Add-On Therapy In Treatment of Asthma” (LODO) published in the American Journal of Respiratory and Critical Care Medicine in 2007, found that while theophylline nor montelukast did not improve asthma control as an add-on asthma therapy, the former was just as effective as the latter in improving asthma control in patients not on inhaled corticosteroids, a common form of asthma treatment. This finding offered an immediate clinical interpretation for physicians treating patients unable or unwilling to use inhaled corticosteroids by providing a safe and inexpensive asthma treatment alternative to the expensive therapy option.

“The ALA-ACRC has always believed that comparative effectiveness research is the key to understanding what works in the real world by narrowing the research-to-practice gap,” Dr. Norman Edelman, chief medical officer for the American Lung Association states “We support the government’s recent focus in comparative effectiveness research”.

The ALA-ACRC followed LODO with the Leukotriene Modifier, Corticosteroid or Corticosteroid-Salmeterol Trial (LOCCS), which examined whether asthma therapy could be stepped down in patients with mild asthma that was well controlled with the use of low-dose inhaled corticosteroids twice daily. LOCCS compared twice a day inhaled corticosteroids to less intensive once-a-day treatment strategies.

The results of LOCCS published in the New England Journal of Medicine in 2007, showed that a simpler regimen of a once-a-day inhaler of inhaled fluticasone plus salmeterol was just as effective as twice-daily treatment of inhaled corticosteroids in patients with mild persistent asthma. These results offered immediate benefits for patients by showing that a simpler treatment plan with few doses to take daily and fewer prescription refills – reducing the cost for medications and perhaps leading to a decrease in side effects – was just as effective in maintaining asthma control. A more convenient treatment schedule would also be expected to result in greater treatment compliance.

“Unfortunately, when it comes to choosing the right medicine or the best health care treatment, clear and dependable information can be very hard to find,” says Dr. Robert Wise, director of the ALA-ACRC Network Data Coordinating Center. “Comparative effectiveness research provides us with a mechanism to develop, disseminate and use new knowledge in a timely manner in order to support clinical decision-making and patients’ health”.

The American Lung Association Asthma Clinical Research Centers (ACRC) is a network of 19 centers across the U.S. conducting large scale clinical trials with the mission to advance the clinical care and treatment of people with asthma. Clinical trials can be distinguished as either efficacy or effectiveness trials. Efficacy trials are primarily designed to evaluate the effects of an intervention under ideal circumstances limiting researchers to draw generalization to large populations whereas, effectiveness trials are primarily designed to evaluate the effects of a health care intervention under conditions in which it will be applied. As a multi-centered network, the ACRC has focused on conducting effectiveness trials. It is well poised to conduct effectiveness trials because the network has the ability to enroll large numbers of patients from diverse populations to ensure that the research findings are relevant to large groups of patients and can be interpreted with the highest level of scientific authority. Currently, the ACRC is recruiting participants for a number of studies relating to lifestyle factors and treatment options for asthma. For more information, go to http://www.lung.org/finding-cures/our-research/acrc/protocols-recruiting.html.