The study demonstrated that cyclosporine provides a 4.2% improvement in quality of life over conventional lubricant therapy, or vehicle; therapy with the vehicle alone provides a 2.9% improvement.
"The vehicle itself creates value, improving quality of life. However, adding the drug agent further enhances the quality of life," said Melissa M. Brown, MD, MN, MBA, president, and chief executive officer of the Center for Value-Based Medicine, Flourtown, PA. She was the principal author of a study published in Archives of Ophthalmology (2009;127:146-152). She also works in the Department of Ophthalmology at Scheie Eye Institute, University of Pennsylvania School of Medicine, Philadelphia.
To put the figures for total quality-of-life gains for cyclosporine in perspective, Dr. Brown compared them with other medical therapies. She pointed out that statins, widely used to treat high cholesterol, are associated with a 4% gain, and that this value is based on improvements in both quality of life and length of life, while cyclosporine and vehicle together produce a 7.1% gain.
The combined value gain also is higher than that of interventions such as alpha-adrenergic blockers—for prostate hyperplasia—or laser photocoagulation or intravitreal pegaptanib sodium (Macugen, Eyetech)—both for subfoveal choroidal neovascularization.
Dr. Brown and colleagues carried out the study at the Center for Value-Based Medicine, where medical interventions are evaluated by converting clinical trial data into a value-based measurement using utility analysis. This enables researchers to assess comparative effectiveness or conferred patient value, average cost-effectiveness (compared with no treatment), and incremental cost-effectiveness (compared with sham or vehicle treatment). With this information, they can determine whether the treatment increases quality or length of life.
In ophthalmology, value is typically based on improvement in quality of life rather than increased longevity.
"The important thing is that we look at value first," Dr. Brown said. "Does it improve the quality of life or does it improve the length of life? If it doesn't do one of those things, we probably ought to rethink the use of the intervention. Then we look at cost, when interventions confer similar value for similar health states."
In this way, she added, drugs are assessed by quality and effectiveness rather than primarily on being less expensive than alternatives.
For the study of cyclosporine, the researchers used data from two 6-month, large, multicenter, randomized trials for moderate to severe dry eye disease, which had been part of the new drug application submitted by Allergan to the FDA.
Other studies also were used, and adverse events associated with cyclosporine topical therapy were factored into the model. The end result is a numerical value stated in quality-adjusted life year (QALY) gain and percentage of improvement in quality of life, as well as a value for the cost-utility ratio using dollars per QALY.
Utility analysis is a useful tool for ophthalmologists because it can put numbers on treatments to confirm that they improve quality of life, due to the high value people place on vision, Dr. Brown said.
"A lot of our treatments, even expensive treatments, are very cost-effective because they so improve the quality of life," she said.
Having validation of the comparative effectiveness and cost effectiveness of cyclosporine enhances clinicians' confidence in including this treatment option when managing the growing public health problem of dry eye syndrome in an aging population, she added.
Dr. Brown reports being a shareholder in the Center for Value-Based Medicine. Allergan Inc. is a client of the Center for Value-Based Medicine and supported the current study with a grant.