How Not to Run a Comparative Effectiveness Agency

A version of this article first appeared on the Guardian (UK) website.

We live in an age where the answer to many questions is available with the click of a mouse. But when it comes to health care, asking what works best gets a sick person text messages from the Tower of Babel.

If you’re in your 60s and have a slow-growing prostate tumor, should you cut it out, take a drug, stick in a radiating seed, or just watch and wait? Ask a surgeon, a urologist, a radiologist and general practitioner that question, and you might get four very different answers.

As a result, patient care – and cost – varies wildly across the U.S., usually with little difference in outcome and often driven by what local hospitals offer and what physician specialties predominate in an area. Professional medical societies, patient advocacy groups and government agencies publish competing and often conflicting policies to guide clinical practice, while money from companies with a stake in their decisions permeates the entire system, including, all too often, the guidelines offered by the U.S. government.

To cut through the cacophony of competing advice, health care reformers in the U.S. are pushing for a comparative effectiveness agency to provide physicians, patients and insurers with authoritative analyses of what works best in medicine. Much of the oomph behind the push for comparative effectiveness (CE) research is coming from the companies that pay the bills.

And with estimates of waste in the health care system approaching 30 percent (that’s $600 billion annually), who can blame them? It seems like a relatively painless way to convince doctors that they ought to abandon less effective therapeutic approaches that needlessly drive up costs. “Look, here’s what you’ve been doing and its outcomes are not as good as this other (drug, device, procedure, test) that they’re using over here; why don’t you switch to what works best?” an authoritative agency could say. “And if what works best costs less, everyone will be better off.”

Well, not exactly everyone. If your income depends on peddling pricey products and procedures – and that includes many specialist physicians and most drug, biotech, and medical device manufacturers – discovering that an “innovative” new product is no better and maybe even worse than something that is already out there is not in your financial self interest.

Opposition from those groups has usually been enough to kill serious consideration of a CE Agency in the U.S., especially one comparable to the National Institute for Clinical Excellence, which conducts comparative analyses (including cost-benefit analyses) for Great Britain. While NICE has no real power to enforce its findings, it has become the de facto gatekeeper of care standards for the cash-strapped British National Health Service, which must live within government constrained budgets.

But the era where special interests succeed in thwarting the creation of CE agency here appears to be ending. Both Democratic Party candidates have made it a centerpiece of their health care reform strategies. Here's why. Covering all or most of the uninsured without cost controls will add anywhere from $50 billion to $100 billion a year in new health care spending. Under their plans, employers who don’t provide coverage would be subjected to new payroll taxes to cover the uninsured. Unless other costs are controlled, those new taxes would be prohibitively high.

Incorporating CE analyses into coverage decisions would the best way to offset some of those costs, with the savings distributed to everyone who buys insurance. A Commonwealth Fund study released in December estimated the health care system could save $358 billion over ten years by using CE analyses – anywhere from one-third to two-thirds the cost of covering the uninsured.

The movement gained a powerful ally in January when the slow-to-move but prestigious Institute of Medicine issued its own report, “Knowing What Works in Health Care: A Roadmap for the Nation.” It called for a “single entity” to produce “credible, unbiased information about what is known and not known about clinical effectiveness.”

A CE program, the report said, should develop a common standard for evaluating evidence and writing guidelines, and set priorities for conducting its own systematic reviews based on improving health and eliminating “undesirable variation.” It should also consider economic factors, including the cost of treatment.

Unfortunately, when it came to insulating the proposed agency from commercial pressure, the IOM report came up short. The priorities of the CE program should be developed by a committee “chosen to minimize committee bias due to conflicts of interest.” In a separate section on developing clinical practice guidelines, the report called on panels to “include a balance of competing interests and diverse stakeholders, publish conflict of interest disclosures, and prohibit voting by members with material conflicts.”

This is a prescription for paralysis for which disclosure is no cure. If commercial interests have any voice in the design of the studies, the committees' deliberations, or their decisions, it will doom the agency to either issuing innocuous guidelines or risk losing its credibility when affected parties charge bias influenced stronger recommendations.

Powerful forces are already mobilizing to make certain any comparative effective agency established by Congress remains a toothless tiger. When Sen. Max Baucus (D-MT) introduced legislation in March, Pharmaceutical Research and Manufacturers of America vice president Ken Johnson
said the agency should be “structured to promote better patient health and timely patient access to needed therapies, and avoid denying or delaying patients’ access to beneficial care, as what often occurs in Europe and Australia.” The Advanced Medical Technology Association, which represents device makers, insists that “governance of any public-private entity should include representation of all stakeholders.”

This theme was echoed in a recent article in the Journal of the American Medical Association, which likened a potential CE Agency to a Federal Reserve Board for medicine. Its authors – two prominent health care economists and National Institutes of Health bioethicist Ezekiel Emanuel, who also happens to be the brother of a powerful Congressman – also called for putting “stakeholders” on the board and having input into its studies.

This builds what economists call agency capture (when a regulated industry has undue influence over the agency set up to regulate it) into the very structure of the organization. No banks sit on the Fed’s board. And its studies are conducted by researchers who are scrupulously clean of financial ties to the banks they regulate.

It’s one thing to give stakeholders a chance to advice the process – just as they have input through comment and testimony into any regulatory proceeding. But to allow industry representatives to sit on the board, and ask clinicians with conflicts of interest to conduct its studies, would undermine the new agency’s credibility at the start – and doom it to being just another babbling voice in the health care wilderness.