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[Editor's note: President Barack Obama and the U.S. Congress have agreed to spend more than $1 billion on comparative effectiveness research (CER), which some critics say will lead to an unethical strategy of the government justifying rationing health care. Others say it will provide needed information to clinicians about which medical treatment works best. But from an IRB perspective, will CER trials pose any new or more serious risks to human subjects? IRB Advisor asks CER experts nationwide to help us answer this question and others about CER in two stories in this month's issue.]
Is comparative effectiveness research the 'Big Bad Wolf' critics charge?
CER will improve health care, many say
Human subjects research is conducted chiefly for the ideal of improving medical practice, and comparative effectiveness research (CER) is a big step toward this goal, several CER experts say.
"The first important point about comparative effectiveness research and why many of us feel it is so crucial is there is so much information that is not in the right format for making medical decisions," says Mark S. Roberts, MD, MPP, president of the Society for Medical Decision-Making and a professor of medicine, health policy, and management and industrial engineering at the University of Pittsburgh School of Medicine. Roberts also is chief of the section of decision sciences and clinical systems modeling in the department of medicine at the University of Pittsburgh.
Roberts spoke about CER at a June 2009 listening session of the new Federal Coordinating Council for Comparative Effectiveness.
"Most of our medical information comes from randomized, controlled trials," Roberts explains. "The reason for that is they tend to have narrowly focused populations where the investigator is making sure they can prove a particular medication or device being tested works."
In these trials, the comparison typically is between the device or drug and placebo.
"So, the clinical trial (CT) gives you different parts of answers to the problem, but it never gives you the specific information to answer a physician's question," Roberts adds.
As CER increases, IRBs will become involved in the change.
"IRBs play a very important role in overseeing the conduct of clinical trials research and, increasingly, the establishment of comparative effectiveness will play a larger role in clinical trials, says Caleb Alexander, MD, assistant professor in the department of medicine at the MacLean Center for Clinical Medical Ethics of the University of Chicago Hospitals. Alexander recently published a paper on CER.1
With a $1.1 billion jolt of federal stimulus package funds entering the research funding pipeline for CER, IRBs soon will receive proposals involving CER. But they need not worry about this being an entirely new research concept for ethical review, one expert says.
"We've been doing comparative effectiveness research for 30 years," says Joel Kupersmith, MD, chief research and development officer for the Department of Veterans Affairs (VA) in Washington, DC. Kupersmith is a member of the Federal Coordinating Council for Comparative Effectiveness.
The VA has a cooperative studies program and a large health services research section that do comparative effectiveness research, he says.
"We also, as part of a health care system, have ways of translating research into practice through guidelines," Kupersmith says. "At times, these are the result of our comparative effectiveness research."
IRBs will need to review CER using the same ethical standards and considering all patient protection aspects, Kupersmith says.
"They also should make sure the research has scientific quality so we don't put patients through something that is not scientifically rigorous," he adds.
As part of CER, CT sites might use novel and different methodologies than are commonly used, Kupersmith says.
Some studies employing new methodologies might be exempted from IRB review; others might force the IRB to look at human subjects research in a slightly different light. Either way, IRBs will adapt to the change, Kupersmith says.
When these have been introduced in protocols at the VA, the VA IRBs have adjusted, he notes.
"We have the world's best trials methodologists, and they're highly respected," Kupersmith says. "Sure there are sometimes intense discussions within an IRB, but I don't think it's a problem at all."
CER critics say the federal stimulus bill that includes CER funding was passed too quickly and without public debate about its various components, including CER funding.
"It's not clear what comparative effectiveness research will affect and in what way," says Grace-Marie Turner, president and founder of Galen Institute in Alexandria, VA. Turner also spoke at the June listening session of the new Federal Coordinating Council for Comparative Effectiveness.
A worst-case scenario would be if CER were used to inform economic decisions about patients' treatment and if these decisions were made by the federal government, Turner says.
"It gets down to how it will affect any one patient," Turner says. "Even though the research is based on an average person, will it apply to every single patient?"
In the United Kingdom, patients' proposed treatments routinely are turned down because of health care economic policies affected by CER, she says.
"That's what they do," Turner adds. "They ration and decide what they'll pay for and what they won't."
Others argue that CER poses no ethical threats in itself.
"I'm not aware of any serious new ethical challenges posed by CER," Alexander says.
"I think the most contentious area of these efforts is the question of assessing the costs of treatment and not just the clinical effectiveness," he says. "These are people who say once you start assessing costs you're on a slippery slope."
They raise concerns that with greater focus on costs, treatment will be denied and quality will suffer, Alexander says.
"And I think they're wrong," he says. "There is an Alan Garber quote that talks about how assessing value without costs is like trying to look at the restaurant menu without looking at prices."
Alan Garber, MD, PhD is a professor of medicine, biochemistry and molecular biology, and molecular and cellular biology in the division of diabetes, endocrinology and metabolism at Baylor College of Medicine in Houston.
"It's simply foolish to think we're not already rationing care," Alexander says. "We ration care through a number of means, so arguments that we should not look at costs because it will lead to denials of necessary care are misguided."
Skeptics of CER say it takes control of medical decisions out of the hands of physicians and patients and places them in the hands of a governmental bean counter.
"Absolutely, there will need to be decisions made about balancing resources," Turner says. "But do we want the government to make those decisions, or do we want doctors and patients making those decisions?"
It comes down to how comparative effectiveness research would be used, Turner says.
"I think it's a great idea to compare one treatment to another," she says. I just don't want the government to decide in an abstract way that this treatment is right and this treatment is wrong."
Since it's the Obama administration pushing for more comparative effectiveness research at the same time national health care reform is being debated and considered by Congress, the topic is bound to create controversy, some say.
"Since the push for this partly is from Washington, and since people differ in their ideas of the role cost should play in the research, naturally there is room for people to disagree and for debates to become contentious," Alexander says.
What is not being disputed is that the establishment of CER will play a larger role in clinical trials.
"I think that increasingly IRBs will review research that includes a goal of establishing comparative effectiveness," Alexander says. "But it will take place in the role of clinical trials."
Alexander recommends that IRBs begin discussing comparative effectiveness research now with the general idea that enthusiasm is building for this type of research and how it might improve clinical practice.
"I think it's important that IRB members are aware of the current directions of health policy at the local, as well as at the national level, and comparative effectiveness research is a part of the current picture," he says.
"IRBs already are reviewing and supervising very complex clinical trials, often of new and unproven therapies that have a variety of toxicities and risks," he adds. "So, I don't think comparative effectiveness research is likely to introduce any new and substantively different risks or administrative complexities."