CER efficacy linked to five key initiatives
CER efficacy linked to five key initiatives
Linking knowledge is very important
There are a number of ways that comparative effectiveness research (CER) might impact health care research, practice, and policy, according to a new study.
The study highlights the ways CER will have to be developed if it's to ultimately improve clinical care, says Caleb Alexander, MD, assistant professor in the department of medicine at MacLean Center for Clinical Medical Ethics, University of Chicago Hospitals in Chicago, IL.
When comparative effectiveness research is done in an uncoordinated approach that lacks rigor, objectivity, and timeliness, it misses the opportunity to provide the most useful clinical information to physicians, the study notes.1
The study suggests that these initiatives be implemented in order for CER to succeed in improving the U.S. health care system:
1. Generate data prior to widespread adoption of a drug or treatment.
"This is a tricky issue because there are a lot of regulations in place that prevent the widespread uptake of therapies once they hit the market," Alexander says. "Nor am I suggesting that there should be, in many cases."
Still, policymakers have the challenge of making certain they don't stifle innovation while safeguarding public health and risk exposure, he adds.
"At the time a drug has been approved, it's been studied in just a few thousand patients, and that's not always sufficient to identify harms or to generate knowledge of comparative effectiveness of two different treatments," Alexander says.
Since the Food and Drug Administration (FDA) doesn't require sponsors to actively compare their treatment to existing treatments and allows placebo-controlled trials in drug approval, many people might be exposed to an approved drug before data are available showing whether or not it is as effective and safe as existing treatment, he adds.
"Think about Vioxx and Cox-2 inhibitors, which are an example of how these drugs were widely diffused into general practice and were used widely beyond the populations that would benefit from them, only to have subsequent discovery of [their] considerable harm," Alexander says.
2. Link knowledge of effectiveness with strategies for improving clinical practice.
"This is one of the most important things," Alexander says.
"I think many people tend to think the substandard clinical practice is due to knowledge deficits alone," he explains. "But time and time again, we see non-evidenced-based use of treatments in settings where there is no knowledge deficit."
Knowledge is just one of the drivers of clinical practice, Alexander says.
"You need to appeal to clinicians' rational choice decision-making where knowledge is the primary driver," he says.
One of the reasons why evidence alone doesn't change clinical practice is because clinicians are flooded, overwhelmed with existing evidence, Alexander says.
Another reason is that local standards of care dictate one way of practicing, and there are other social and psychological obstacles, as well, he says.
Finally, the pharmaceutical industry's marketing and promotion of drugs is very effective and may guide physicians to use therapies that are not evidence-based, he adds.
3. Examine the effectiveness of more than just drugs and devices.
"Often times, people tend to think automatically of drugs and devices when thinking of comparative effectiveness, but we argue this research should encompass other levels of health delivery and organization," Alexander says.
For example, there could be a comparison of two procedures with each other, or a comparison of procedures with medications, he says.
CER could involve comparisons of different systems of care or different administrative structures, he adds.
"The reason this is important is because prescription drugs only account for 10% of our health care setting, and if you take a searchable procedure like surgery for lower back pain, the cost of that surgery dwarfs the cost of prescription drugs used to manage the condition," Alexander says.
"So we're trying to highlight how CER should be used to compare different health care strategies, at many different levels," he explains.
4. Rethink the FDA approval process.
"The historical focus of the FDA is on common harms and on evaluating drugs' efficacy against placebo," Alexander says.
This policy has led to drugs being studied in small and very select populations that have less comorbidity than the general populations seen by physicians.1
Also, the focus on placebo-controlled trials means that drugs that have no advantage over existing drugs can be approved for marketing without having to demonstrate why they're needed.1
"So many policymakers have called for the FDA to abandon placebo-controlled trials and move to active comparators," he says.
Any drug or device approved under this model would have been compared with alternatives already on the market, Alexander explains.
"Me-too drugs serve a valuable purpose because they force competition between members of a drug class, and in some therapeutic categories, heterogeneity exists, so they aren't entirely bad," he says. "But, nevertheless, we argue that FDA approval increasingly should use active comparators rather than placebos, if we're serious about generating [useful] data."
5. Consider not just effectiveness, but the cost of alternative treatments.
Cost-effectiveness research is controversial in the U.S. because of concerns that it will lead to people being denied care they desire or need.1
But treatments cannot be compared without a discussion of cost.
"You can't talk about value without talking about costs," Alexander says. "It's naïve to think we can only talk about effectiveness and not talk about the cost of therapy."
It won't work well to ignore the cost and expect better knowledge to improve clinical practice, he adds.
"We have to consider the cost of treatment too, and those who object to this based on their claims that it will lead to rationing are often alarmists," Alexander says. "Or they are overlooking the fact that rationing is taking place every day in our health care system."
Reference
- Alexander GC, Stafford RS. Does comparative effectiveness have a comparative edge? JAMA. 2009;301(23):2488-2490.
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