The hot buzz word in the health care reform sector is "comparative effectivness research," and the lay press is picking up on the partisan rumblings in Washington over this provision in the recent stimulus legislation. But what is CE research, and why should we care about the minutiae involved in the bickering of a bunch of Washington politicians?

In fact, we should care very much, as it could change the way physicians practice medicine and consumers use health care.

What is the argument? Mainly, and this should come as no surprise, the fight is over money. Specifically, the words "cost" and "clinical," tied to a $1.1 billion provision in the recovery act detailing comparative effectiveness research.

What is CE research, and what will the $1.1 billion go toward? According to the report accompanying the legislation, the money "shall be used to conduct or support research the evaluate and compare clinical outcomes, effectiveness, risk and benefits of two or more medical treatments and services that address a particular medical treatment." (The breakdown: $400 million to NIH, $400 million to be used at the discretion of HHS, and $300 to AHRQ.)

Essentially, it's a Consumer Reports for prescribers: for patient X with disease Y, what's the best course of action to treat the disease? There isn't a way to compare and contrast medications, devices, surgeries, preventive care, etc yet. And yet is the operative word - that's what CE research should be able to do.

CE research isn't new - researchers have been engaged in these types of studies for decades. Randomized clincal trials are a type of CE research, so pharma companies are already practiced in this area (albeit with a financial incentive). And government entities have been conducting research for years, so there are people in place with knowledge and expertise. For examples, check out the list of reviews on AHRQ's Effective Health Care initiative for comparisons on everything from diabetes to prostate cancer. Not only that, but Medicare already practices a form of CE research by reimbursing certain treatment courses over others.

In theory, this sounds like just the solution we need to cut health care costs and improve our own health. So what's the problem? This is where the minutiae comes in and becomes critically important.

1. Cost: Many equate comparative effectiveness with cost effectiveness. A number of studies have found that older, cheaper generic drugs work just as well or better than newer, more expensive drugs (antipsychotics and antihypertensives are two of the more well-known studies). In this particular instance, that is incorrect. The report accompanying the legislation specifically states, " The conferees do not intend for the comparative effectiveness research funding included in the conference agreement to be used to mandate coverage, reimbursement or other policies for any public or private payer."

Importantly, the word "cost" was removed from any language dictating the activities of the Federal Coordinating Council for Comparative Effectiveness, which would oversee the research and funding. Senate Finance Committee Chairman Max Baucus, D-Mont., is re-introducing his Comparative Effectiveness Research Act of 2008 this year, which notably does not give the council the ability to make coverage decisions.

The American Medical Association's stated position is that the research is for information purposes, not for coverage decisions - the goal is to help patients and prescribers make informed decisions based on evidence, not to dictate which medications a patient can receive. This leads us to the next big issue...

2. Clinical: While the stimulus legislation left out "cost," it also left out "clinical" - and that worries some who think the research may eventually be used to make cost decisions. Another worry is that the government will mandate from on high how doctors practice medicine, creating a one-size-fits-all approach to treatment.

Many may believe that doctors, not government employees or insurance companies, should be responsible for their health care. As Dr. Val Jones says in her MedPage blog post, "population-based conclusions do not always provide a clear "best choice" for individual patients."

Think about the current explosion of personalized medicine and pharmacogenomics - our genetic makeup, our environment, our concomitant medications and diseases - it is hard to apply one regimen to an entire heterogenous population.

But the CE research provision isn't about giving doctors one choice - it is about giving them the spectrum of choices, along with guidance on what may be best for a particular situation. The legislation report says that Congress "recognizes that a 'one-size-fits-all' approach to patient treatment is not the most medically appropriate solution to treating various conditions," and the legislation includes "language to ensure that subpopulations are considered when research is conducted or supported with the funds provided in the conference agreement."

In a Newsweek article, Sharon Begley quotes physician and blogger Kevin Pho, who sums it up nicely: "An unbiased source of data, not drug companies, could really help us in primary care," he says. "There have to be allowances for individual differences, but you need standards."

Can you get brain cancer if you don't have a brain?

Here's a real life example, covered in both medical and lay news over the past several years, of how CE research could help cut costs and improve our care.

In June 2004 Dr. Brenda Sirovich (VA Outcomes Group, White River Junction) and Gilbert Welch  (VA Outcomes Group and Center for the Evaluative Clinical Sciences at Dartmouth) published a startling paper in JAMA. The title should give you a clue to the content: "Cervical Cancer Screening Among Women Without a Cervix." What? That would be like screening the Scarecrow in Wizard of Oz for brain cancer. Surely doctors aren't that ... I don't know what the word is, but it isn't good.

They conducted a study to see whether the 1996 U.S. Preventive Services Task Force recommendation, that routine Pap smears are unnecessary for women who had undergone a hysterectomy (including removal of the cervix), had made a difference in Pap smear screening among hysterectomy patients. They estimated that almost 10 million women, about half of the 22 million women who have had hysterectomies, were still being screened.

What are the recommendations for Pap screening? The U.S. PSTF updates recommendations as evidence becomes available, and the list from 2003 still stands. If you have had a total hysterectomy for benign disease, or are older 65 and have had adequate recent screening with normal Pap smears and are not otherwise at high risk for cervical cancer, you don't need yearly screening. So why are they still getting the annual checkup?

The NY Times picked the story up, and some of the doctors quoted in the story cited habit and fears of litigation as reasons why they continue to screen.

The screening is presumably doing some good - SEER statistics list 12,200 new cases of cervical cancer and 4,100 estimated deaths in 2003. In 2005 the number of deaths had dropped slightly to 3,924, and by 2008 the numbers drop to 11,070 new cases and 3,870 deaths (all races, all ages). But there are costs and benefits to screening, and comparative effectiveness research could help doctors and patients decide the best course.

So what's going to happen now? Well, nobody is entirely certain. The stimulus bill passed, so there is a whole lotta money out there for research. And Baucus is going to re-introduce his bill as a long-term solution to CE research. In the meantime, ask your doctors about the choices you have for treatment, and hope they don't suggest scanning for an organ you don't have.

More reading on the cervical cancer screening debacle:
J Med Screen. 2003;10(4):189-95.
J Gen Intern Med. 2004 Mar;19(3):243-50.
JAMA. 2004 Jun 23;291(24):2990-3.
Am J Med. 2005 Feb;118(2):151-8.

More takes on comparative effectiveness:
North by Northwestern blog
NY Times "Well" blog, March 3, 2009