Research participants should be informed when studies are being conducted to determine which treatments provide the best bang for the buck, argue the authors of a Viewpoint in today’s issue of JAMA.
Health care reform and the urgent need to cut rising health care costs have led to a growing number of comparative-effectiveness studies that may pit treatments against each other to assess the relative risks, benefits, and costs of the treatments. Franklin G. Miller, PhD, National Institutes of Health Department of Bioethics, and colleagues argue that when treatment costs are a motivating factor behind a study, participants have a right to know.
Miller and colleagues highlighted the Comparison of the Age-Related Macular Degeneration Treatment Trials (CATT) as an example of study motivated in part by differential drug costs. The CATT compared the relative costs, safety, and effectiveness of 2 drugs used to treat macular degeneration. Ranibizumab has been approved by the US Food and Drug Administration (FDA) as a macular degeneration treatment, but many clinicians use a similar drug bevacizumab, which was originally approved as a cancer treatment, in part because it is 40 times cheaper. A report from the US Department of Health and Human Services’ Office of Inspector General (OIG) found, for example, that between 2008 and 2009, Medicare Part B “paid physicians $40 million for 936 382 Avastin (bevacizumab) treatments and $1.1 billion for 696 927 Lucentis (Ranibizumab) treatmetnts.”*
The CATT study mentioned the cost differential but did not spell out that costs were one of the motivating factors behind the study. The study by Miller and colleagues suggests simple language that could be used to let study participants know that cost is a motivating factor. Miller discussed his and his colleagues’ views with news@JAMA.
news@JAMA: Why did you decide to write this Viewpoint?
Dr Miller: There are more comparative effectiveness studies being conducted and one of the background motivations for the studies is the relative costs of treatments. A lot of drugs are expensive, and there is some real value in seeing how these stack up. They rarely are done solely for cost. There are usually other clinical considerations. It was my perception that cost motivation is not routinely described to patients as a reason why a study is done.
news@JAMA: What makes the CATT study such a good case study?
Dr Miller: It’s an excellent study. It stands out in that you have 2 treatments that are biologically almost essentially the same—one is FDA-approved, and one is [prescribed] off label. The FDA-approved drug is 40 times more expensive. With the newly released data about Medicare payments to physicians, it turns out that ophthalmologists are among the highest paid. A fair amount of that has to do with Lucentis [ranibizumab]. I think most ophthalmologists prescribe the cheaper option.
We weren’t trying to criticize the study, it’s a very valuable study. The cost wasn’t fully described as a motivation, but that is really the norm. I was on data safety monitoring committee for the study, and we approved the consent form.
news@JAMA: What did the CATT study ultimately find?
Dr Miller: There was no difference in the safety and effectiveness of the two. This is not surprising because of the biological similarity.
news@JAMA: How might informing participants about cost motivations affect their behavior?
Dr Miller: That’s a question we don’t have an answer to. For patients with chronic conditions it might be better for them to get an equivalent, but cheaper treatment. They might be more motivated to participate, and some are altruistically motivated. There might be some who could be turned off.
news@JAMA: What is the main take-home message you’d like to pass along to researchers and potential research participants?
Dr Miller: When a part of the motivation to do a randomized study has to do with the relative costs of treatment it should be clearly laid out. We suggested some simple language.
*This blog has been updated with information from the OIG report.