House bill earmarks $3 billion in funds for comparative research

A bipartisan House bill proposed last month would target $3 billion in federal and private funds over five years for studies comparing the effectiveness of prescription drugs, medical devices and surgical procedures in real-world clinical practice.

The money would go to the Dept. of Health and Human Services' Agency for Healthcare Research and Quality, although a U.S. comptroller-appointed advisory board general would determine research priorities.

The bill comes on the heels of similar proposals from America's Health Insurance Plans and the BlueCross BlueShield Assn. that call for a new, independent entity to do comparative effectiveness research. Sen. Hillary Rodham Clinton (D, N.Y.) said she will soon introduce legislation of her own on the issue and has made it part of her health care campaign platform in her bid for president. Business and consumer groups, worried about the rising cost of health care, lauded the House proposal.

Comparative effectiveness research "is intended to give good information to patients and clinicians about which treatments are right for different individuals," said AHRQ Director Carolyn M. Clancy, MD. "As we see more scientific breakthroughs being translated into practical treatments that clinicians can use today, the question that comes up more and more often is, 'Which one should I use for my patients?' "

Because most clinical trials undertaken to secure Food and Drug Administration approval compare new treatments with placebo methods, it is often unclear how well they fare against existing therapies. The recent Clinical Outcomes Utilizing Revascularization and Aggressive Drug Evaluation -- COURAGE -- trial is an example of comparative effectiveness research that experts say is much too rare.

Also, experts say, there are huge gaps in the evidence for how interventions tested in the controlled setting of a clinical trial fare in the messy reality of everyday practice.

"It's one thing to demonstrate the efficacy of a particular pharmaceutical agent or device to get it approved, but it's a very different story to see how that evidence is being integrated into routine medical practice," said W. David Helms, PhD, CEO of AcademyHealth, an organization representing health services researchers that supports the idea.

The House bill, sponsored by Reps. Tom Allen (D, Maine) and Jo Ann Emerson (R, Mo.), would establish an "all-payer" financing system in which Medicare and private health plans would each be required to contribute funding based on their proportionate share of enrollees, with Medicare's amount limited to $200 million a year.

AHRQ now has $15 million a year to fund comparative effectiveness research, though that total is already scheduled to double next year, Dr. Clancy said.

Studying cost?

"It is critical that we get the best value for our health care dollar," Allen said in introducing the bill, but added that the bill's focus is on the clinical effectiveness of competing therapies, not their cost effectiveness.

About one-third of the $2 trillion in U.S. health system spending goes to care that is unnecessary or ineffective, experts estimate. And while politicians and payers see comparative effectiveness research as a way to reduce wasteful spending, drug- and device-makers fear it could hurt their bottom lines. They worry the findings will be used to deny physicians and patients their choice of therapies, a concern some doctors share.

According to Peter J. Neumann, ScD, the hopes -- and fears -- about comparative effectiveness research may be overblown. "There's an expectation that this research will reveal a lot of waste in the system and save money. That's probably unlikely," said Dr. Neumann, director of the Center for the Evaluation of Value and Risk in Health at Tufts-New England Medical Center in Boston. "Often, comparative effectiveness research doesn't remove the hard choices. You may find that a new drug works a little better but costs a lot more."

How to use that evidence, Dr. Neumann said, is still left unresolved. Under the House bill, AHRQ would not be empowered to make coverage decisions or recommendations.

Sean R. Tunis, MD, former chief medical officer at the Centers for Medicare & Medicaid Services, said that when it comes to the relative effectiveness of costly medical interventions, ignorance is not bliss.

"In the next five, 10, or 15 years, there is going to be serious downward pressure on health care spending," Dr. Tunis said. "Either we are going to handle it with information that helps us make the most of what we spend, or we do it indiscriminately."

Dr. Tunis' major concern about the House bill is that it delegates to an advisory board to prioritize what to study and how to study it. He fears that costly, time-consuming head-to-head clinical trials will be pushed aside in favor of retrospective mining of electronic medical records and claims data. Without randomization, Dr. Tunis said, such approaches could yield less-reliable evidence.

"Whenever you have a proposal that everybody's in favor of, it means you haven't been specific enough about what you're planning to do," he said.

AMA policy supports "legislation that would greatly expand the scope and budget of the AHRQ as the central federal agency" on quality. The AMA does not have policy on funding for comparative effectiveness research.