CER used effectively overseas for many years
Many studies involve cancer
Comparative effectiveness research (CER) is gaining momentum in the United States, but it's been used for years in other countries.
"Other countries have far more experience than we do in this area," says Patricia Pittman, PhD, executive vice president of AcademyHealth of Washington, DC. AcademyHealth is a professional society for health services research and policy.
Two new reports from AcademyHealth examine CER, with one looking at domestic CER and the other looking at comparative effectiveness research around the globe.1,2
Researchers found 689 U.S. comparative effectiveness studies through Clinicaltrials.gov and HSRProj, and they identified another 617 comparative effectiveness studies through interviews with research funders.1
"For those two databases, we wanted to make sure there was no overlap," Pittman says. "We looked at different study designs, major variables, and we looked at clinical studies, observational studies, registry studies, research synthesis, meta-analysis, and other synthesis designs."
The number of comparative effectiveness studies listed on Clinicaltrials.gov is only 5% of the new studies added to these sources each year.
Also, most of the CER involves cancer treatment, the study finds.1
The study's findings suggest that CER is used to compare a variety of treatments, including behavioral therapy, surgical strategies, and minimally-invasive therapies, Pittman says.
The more than $1 billion investment in CER from the federal stimulus package will undoubtedly inflate the number of comparative effectiveness studies underway, Pittman says.
"And there's potential to do this kind of work in a much more efficient way than what could be done 15 years ago because of new technology and the availability of data," Pittman says. "There will be a major shift in how this is done over the next five to 10 years."
However, there are issues that need to be addressed, and this is where studies examining CER can be helpful.
"There are two completely distinct areas in policy," Pittman says. "One is how CER is funded and whether the emphasis should be on drugs alone or drugs and devices."
How will the findings be used?
The other policy domain involves how the findings are used, she adds.
"Should it be used to inform health plans, public or private, or formulary decisions?" she says. "Is it for clinical decision-making or at the level of the patient and family?"
The way the scope is defined in the first domain depends on what kinds of decisions are relevant, she notes.
"The controversy in this country is whether the findings would be implemented in a way that would become a requirement, that would be mandatory," she says. "There's some misinformation about what's happening in Europe."
For the most part comparative effectiveness is used as recommendations, not a requirement in Europe, Pittman says.
"In the case of the United Kingdom, comparative effectiveness research is strongly tied to the decision-making process," she adds. "But in Germany, they are just recommendations."
The U.K. established the National Institute for Clinical Excellence (NICE) as part of a strategy to inform practice and policy decisions, including spreading best practices.2
Germany created the German Institute for Quality and Efficiency in Healthcare in 2004 as part of a reform strategy. Then in 2005, the institute introduced cost-effectiveness and clinical effectiveness as conditions of evaluating coverage and reimbursement. But the institute's main role has been to identify national quality standards.2
Australia's Pharmaceutical Benefits Advisory Commission (PBAC) began to consider drug costs directly in its decision making in the late 1980s. And in 1993, the PBAC began considering comparative value in its decision making, meaning a higher price was acceptable only when the drug offers greater efficacy or reduced toxicity or both when compared with current alternatives.2
The idea of CER being used by a government-run health care entity in gatekeeper decisions is what is very controversial in the United States, Pittman notes.
The United States already went through a period in the early 1990s in which HMOs restricted access to care, Pittman says.
"And there's a backlash from some in the industry and a fear among consumers about restricted access to care," she explains. "The wounds of that period in our history are still somewhat raw, so there's a lot of sensitivity around rationing, even though we ration every day."
At present there are clinical research findings that could impact coverage decisions, and yet no one is afraid of these, Pittman says.
"So to deny producing more science-based knowledge doesn't make any sense," she adds. "The controversy is over whether that knowledge would be used in a mechanical way to force a decision."
No mandatory applications
Everyone could agree that there should be more knowledge generated around comparative effectiveness treatments and interventions, while also acknowledging that there shouldn't be mandatory applications of findings, Pittman says.
AcademyHealth's recent study on CER around the globe includes six to 10 take-away messages about what can be learned from other countries, Pittman says.
"Most of the lessons are around governance issues, how entities were set up to fund research and to what extent research is linked," she explains. "It has lessons about the degree of political support."
Inevitably, there are winners and losers in a system that uses CER, she notes.
"Those companies whose products are not highly ranked will not be happy with findings," Pittman says.
Companies already conduct some CER for marketing purposes, but they only publish their findings when it shows their drug to be the more effective one, she adds.
A look at CER internationally highlights a series of challenges, including technical issues.
"There is a challenge of people not being willing to have their data be available for studies," Pittman explains. "Also there are enormous political challenges, and we've already seen these with efforts to oppose funding."
Or if people can't oppose the funding then they oppose the use of the research, she adds.
References
- A first look at the volume and cost of comparative effectiveness research in the United States. AcademyHealth. June, 2009:1-20.
- Chalkidou K, Anderson G. Comparative effectiveness research - international experiences and implications for the United States. AcademyHealth. July, 2009:1-19.
You have reached your article limit for the month. Subscribe now to access this article plus other member-only content.
- Award-winning Medical Content
- Latest Advances & Development in Medicine
- Unbiased Content