The institute is trying to advance the cause of CER without creating a political flashpoint.
By BRETT NORMAN | 12/29/11 9:32 AM EST
So far, the new institute that’s supposed to run comparative effectiveness research has avoided the harsh political rhetoric of health care rationing and “death panels” — but its supporters worry that its luck could be about to change.
The Patient-Centered Outcomes Research Institute, set up by the health care reform law, is walking a fine line, trying to advance the cause of comparative effectiveness research without creating a political flashpoint. And that means getting as much buy-in as possible from stakeholders throughout the health care sector.
In January, PCORI will lay down two markers — its draft research agenda and its national principles — outlining the framework of a national program for comparative effectiveness research. And a lot of people are watching closely.
“They are really operating under the shadow of the death panel crazies,” said Jerry Avorn, professor at Harvard Medical School and author of "Powerful Medicines." “There’s still a lot of fear that even the most reasonable and necessary research — say, the best approach to lowering blood pressure or cholesterol, for instance — will be demagogued and demonized by those on the right.”
But tempers have cooled at least somewhat since the arguments flared in 2009 after the stimulus bill included $1.1 billion for CER. The calmer environment is leading supporters to be guardedly optimistic that PCORI will be able to proceed — albeit cautiously — without excessive political distraction.
“My belief is that much of the storm has subsided — not to say it can’t come back,” said Robert Dubois, chief science officer of the National Pharmaceutical Council. Dubois wrote a paper this month in Health Affairs with advice for how PCORI should prioritize treatments for study — transparently and based on explicit evidence, with input from all stakeholders.
“The combination of the way the legislation was written, the board of governors and the tone that they have set has satisfied most people,” he said.
The point of comparative effectiveness research is to compare two or more different ways of treating the same condition to see which one works best. The idea is that if definitive best practices can be established, they will be widely adopted by providers and may be preferentially reimbursed by payers.
Cheaper treatments that are effective would be favored.
It may sound harmless — like common sense, even, to the uninitiated — but it’s a menacing prospect to some pharmaceutical companies and medical device-makers who are concerned that their products may wind up on the wrong side of the ledger.
For this reason, Michael Cannon, director of health care studies at the Cato Institute, says good comparative effectiveness research is almost suicidal.
“The whole point of [comparative effectiveness research] is to find out what doesn’t work,” Cannon said in an email. “Every time the government has tried to do CER, the guys who provide the stuff found not to work successfully lobby to have the offending agency defunded. I see no reason to think this time will be any different. The moment it produces useful CER, PCORI is toast.”
And that’s just one source of opposition. The other includes general foes of the Affordable Care Act, who have seized on elements like the Independent Payment Advisory Board, and end of life counseling, suggesting that health reform will lead to government “pulling the plug on grandma.”
Betsy McCaughey, former lieutenant governor of New York and author of “Obama Health Law: What It Says and How to Overturn It,” fueled some of the most heated claims in 2009 about threats of government health care rationing.
McCaughey said she won’t prejudge PCORI’s agenda, but that generally “there’s an intense politicization of medical research to support an agenda that wants to save money by denying care to the elderly and sick. … The term ‘comparative effectiveness’ is code for giving care based on age and life expectancy of the patient.”
Supporters say that objection is off base, and that the research is designed to find the most effective treatment for every population.
But even the institute’s name bears the imprint of the red-hot controversy the subject fuels. During the health care reform debate, the very term “comparative effectiveness research” was abandoned in favor of “patient-centered outcomes research.” The latter has an explicit emphasis on the wishes of the patient, rather than hinting that the true goal is cheaper care.
And the ACA specifically states that PCORI will have no authority to mandate coverage or reimbursement, although it doesn’t forbid public or private payers from using the research to inform their coverage decisions, John McDonough, who worked as an aide to Sen. Ted Kennedy, reports in his book "Inside National Health Reform."
PCORI is led by Joe Selby, a widely respected former executive at Kaiser Permanente, who is well aware of the delicate political balance he has to strike.
“Everyone at PCORI, our board and our staff, recognizes the great need for research that compares different prevention, diagnosis or treatment options to see which ones work best for different people with a particular health problem,” Selby said in an emailed response to questions. “That’s at the heart of PCORI’s mission, to provide patients and those who care for them with the information they need to support health care choices.”
After the release of its draft agenda in January, PCORI will award 40 grants totaling $26 million that will fund methodological and agenda-setting research. Selby said PCORI will award another $100 million in primary research funds by the end of the year. The majority of funds will go to comparative effectiveness research studies, he said.
The ACA established a trust fund for PCORI, so it isn’t subject to the annual appropriations process, which provides a measure of security. It’s funded by the government but is independent of it, and so far, in the various debt reduction negotiations this year, it has not emerged as a target.
But laws can change, and even PCORI foes suspect the agency will tread lightly next year.
Robert Goldberg, vice president of The Center for Medicine in the Public Interest, believes that money being spent on PCORI would be better spent elsewhere. “I don’t think there’s any hard evidence that comparative effectiveness actually improves health at a reduced cost,” he said.
He co-authored a paper with John Vernon, an economist at the University of North Carolina, arguing that if a comparative effectiveness standard were built into the FDA’s premarket approval process, the impact on innovation would be severe. “To the extent that it’s just another link in the chain from the time a product is developed 'til the time it’s approved, it’s going to delay access and discourage innovation,” Goldberg says.
Harvard’s Avorn calls this “crying wolf,” because comparative effectiveness has never been a part of FDA review and including it is not in the cards.
And Goldberg acknowledges it is a purely hypothetical scenario — one that can’t be allowed to become reality.
Despite his suspicions, though, he thinks of PCORI as mostly harmless.
“I don’t see PCORI as a real threat. I think they’re more interested in shoving the money out in a neutral way not to upset anybody and see if they can build support for it after 2012,” Goldberg said. “Depending how the elections go, they could be an early target, so they’d like to build some consensus.”
Supporters agree on the last part — the need for consensus — and generally applaud the efforts to bring as many people to the table as possible. Consensus is needed, they believe, not just to keep political foes at bay, but also to advance the cause of comparative effectiveness research beyond the institute.
Large-scale comparative effectiveness studies are extremely expensive. Despite PCORI’s substantial funding, it will never be able to tackle all of the clinical issues that need to be studied.
The goal is to set up a framework that will allow others — insurance companies, research institutes and others — to build on the experience and carry out studies of their own, Dubois said.
Rita Redberg, a comparative effectiveness expert and professor at the University of California San Francisco, said she believes PCORI is doing great work under very trying circumstances.
“I think they’re going to move the needle in terms of what we need to know,” Redberg said. “They’re really doing great with the restrictions they have, but there are a lot of masters to please.”