Wave of CT future: Comparative effectiveness research gets $1.1 billion in fed stimulus bucks
[Editor's note: Clinical trial sites and research institutions soon might benefit from an increase in funding for studies involving comparative effectiveness research (CER). President Barack Obama and the U.S. Congress have agreed to spend $1.1 billion on CER, which is expected to include some clinical trials, as well as observational studies, and synthesis research. Clinical Trials Administrator asked experts in CER to discuss how this new funding might change clinical trials research. In this issue, there are articles about what CER is and how it might change the research enterprise, how to best use CER, and what types of research grants might be funded. In the October issue there will be additional stories about how CER is conducted overseas, controversies, and why CER will benefit consumers, as well as clinicians.]
Wave of CT future: Comparative effectiveness research gets $1.1 billion in fed stimulus bucks
Experts predict it will change clinical research
Comparative effectiveness research (CER) isn't new, but it's been given a huge boost this year with the stimulus package's approval of $1.1 billion for CER grants, distributed by several government agencies.
There are about 600 CER studies underway at present, and existing federal funding of $15 million for CER is a sum that provided a period of warming up for this big investment, says Patricia Pittman, PhD, executive vice president of AcademyHealth in Washington, DC. AcademyHealth is a professional society for health services research and policy and has published annual reports on funding levels of health services research, including CER studies. Pittman spoke about CER at a June listening session of the new Federal Coordinating Council for Comparative Effectiveness Research. (See snapshot of CER council.)
"There's no question this additional investment will increase the volume of clinical effectiveness research going on," Pittman says.
The Veterans Administration has been doing CER for about three decades, says Joel Kupersmith, MD, chief research and development officer for the VA. He directs a research enterprise of $1.6 billion. Kupersmith is one of the members of the Federal Coordinating Council for Comparative Effectiveness Research.
CER looks at existing and new treatments and interventions and compares them with each other, Kupersmith says.
"A typical study may have a comparison between conventional cardiology versus drug therapy," he says.
CER also will help the research industry move from randomized clinical trials to other methodology, Kupersmith says.
"The randomized clinical trials is a safe harbor for investigators, but it has certain drawbacks," Kupersmith says. "For instance, the entry criteria tend to be narrow because you're only serving certain kinds of people and are looking at efficacy instead of effectiveness."
Other methodologies offer some advantages, and these include observational studies and prospective examinations using electronic health records, he says.
For example, one instrument that might be used will compare datasets by timeline. So if investigators have a significant amount of data from the VA about patient outcomes among patients with certain conditions who started treatment at a particular point in their illness, they could compare this information to slightly different data from the same source. The comparison could be made of patients who have similar diseases and presentations, but who did not receive treatment or at least did not receive it at the same time point in their illness, he explains.
"That's an example of a technical approach that could be used and has been used by researchers," Kupersmith says. "Economists have used that approach for some time."
The point is that CER's main purpose is to stand next to the clinician and do something that will help the clinician in his or her daily practice, Kupersmith notes.
"There's been a lot of enthusiasm for comparative effectiveness research, and many stakeholders hope this research will hold promise or can offer substantive improvement in the quality of health care in the United States," says Caleb Alexander, MD, assistant professor in the department of medicine at MacLean Center for Clinical Medical Ethics, University of Chicago Hospitals in Chicago.
Alexander is the co-author of a new study on CER.1
"We were interested in thinking about the way that researchers and policymakers are approaching comparative effectiveness research and identifying the ways we thought the ultimate impact of this research could be improved," Alexander says.
Most of the new CER funding will be distributed in grants, sending research institutions more research opportunities after several years of a dry spell in federal research grants.
Also, the federal government's push for more CER likely will influence the pharmaceutical industry, resulting in their giving more private funding of comparative effectiveness studies, Pittman says.
"It could impact the way they do the drug development process," Pittman says. "We might see fewer me-too drugs, and we might prioritize more new molecules that they're confident would provide a comparative effectiveness advantage."
Some experts say CER might result in a transformation in how the Food and Drug Administration (FDA) approves drugs and that it could have a very positive impact on the drug development pipeline.
"Clearly as part of the stimulus bill, there's been a huge shot in the arm for comparative effectiveness research," Alexander says. "One could imagine that some comparative effectiveness data might be required for a subset of drugs going under review."
Currently, most CER knowledge comes from post-marketing approval studies, and this arrives late for physicians faced with treatment decisions.
"I don't personally feel I have enough expertise to say that we need to change the way the FDA approves drugs, but what I do know is that we don't have enough information to make the decisions we want to make," 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 in Pittsburgh, PA. 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 also spoke about CER at a June listening session of the Federal Coordinating Council for Comparative Effectiveness.
Doctors need more and better data to make medical decisions appropriate to their patients, Roberts says.
Here's why: "Say I have a whole bunch of randomized clinical trials that are all done well and are extremely competently executed and well written up," he says. "I'm comparing A, B, and C to a placebo."
But the physician needs to know which of the three drugs he should use, and the way many CTs are executed make it difficult to answer that question, Roberts says.
"The way trials are done now is to get FDA approval for a treatment, and this is not the same as doing a trial to obtain the information you need for a clinical setting," he adds.
Another problem is that clinical trials too often exclude the kind of patients that a physician is most likely to see in his or her practice, Roberts adds.
"When the patient of mine is further and further away from what the patient in the trial looked like, how do I know how to make that transition?" he says. "If everyone in a trial had cholesterol of a certain level and blood pressure of a certain level and weight of a certain level, but the patient in my office is off the scale on all three, how do I know the same results will apply?"
What's needed are more clinical trials and CER that show under which conditions treatment A is better than treatment B, Roberts suggests.
"We may need longer-term follow-ups, more data, more biochemical information and biomarkers to correlate the outcomes we see in these trials with the characteristics we see in patients," he adds.
"It's surprising how rarely the patient sitting across from you fits the criteria for making a decision about a drug's efficacy," Roberts says.
Comparative effectiveness research is more likely to include patients who are frailer, who have more comorbidities, and who look like the typical patients physicians see, Roberts and other experts say.
"Patients with comorbidities, especially among the elderly, generally are not in randomized clinical trials, and we have to broaden that scope," Kupersmith says.
Also, clinicians should have a more important role in medical research, he adds.
"A clinician who has an idea can put it in and have it evaluated according to what type of impact it would have on the health care system," he explains.
"Say there are two drugs in common use. Which is better? Are they equal?" he adds. "That's a question a clinician would bring to research."
Reference
- Alexander GC, Stafford RS. Does comparative effectiveness have a comparative edge? JAMA. 2009;301(23):2488-2490.
Subscribe Now for Access
You have reached your article limit for the month. We hope you found our articles both enjoyable and insightful. For information on new subscriptions, product trials, alternative billing arrangements or group and site discounts please call 800-688-2421. We look forward to having you as a long-term member of the Relias Media community.