Monday, October 26, 2009

Who Should Sponsor Comparative Effectiveness Research?

We have tried to argue why comparative effectiveness research is a good idea. To cut and paste what I wrote in a previous post,

Physicians spend a lot of time trying to figure out the best treatments for particular patients' problems. Doing so is often hard. In many situations, there are many plausible treatments, but the trick is picking the one most likely to do the most good and least harm for a particular patient. Ideally, this is where evidence based medicine comes in. But the biggest problem with using the EBM approach is that often the best available evidence does not help much. In particular, for many clinical problems, and for many sorts of patients, no one has ever done a good quality study that compares the plausible treatments for those problems and those patients. When the only studies done compared individual treatments to placebos, and when even those were restricted to narrow patient populations unlike those patient usually seen in daily practice, physicians are left juggling oranges, tomatoes, and carburetors.
Comparative effectiveness studies are simply studies that compare plausible treatments that could be used for patients with particular problems, and which are designed to be generalizable to the sorts of patients usually seen in practice. As a physician, I welcome such studies, because they may provide very useful information that could help me select the optimal treatments for individual patients.

Because I believe that comparative effectiveness studies could be very useful to improve patient care, it upsets me to see this particular kind of clinical study get caught in political, ideological, and economic battles.

In particular, we have discussed a number of high profile attacks on comparative effectiveness research, which often have featured arguments based on logical fallacies. While some of the people making the attacks have assumed a conservative or libertarian ideological mantle, one wonders whether the attacks were more driven by personal financial interests. For example, see our blog posts here, here, here, and here. On the other hand, we discussed a clear-headed defense of comparative effectiveness research by a well-known economist most would regard as libertarian here.

Comparative effectiveness research has been discussed as an element of health care reform in the US. It turns out that the current version of the health care reform bill in the US Senate has a provision to create a Patient Centered Outcome Research Institute, which presumably would become the major organization which could sponsor comparative effectiveness research.

This institute, however, would not be a government agency (despite the name that makes it sound like it would be part of the National Institutes of Health). Moreover, here is a description of the Board of Governors who would run the institute from the current version of the bill :

BOARD OF GOVERNORS.—
(1) IN GENERAL.—The Institute shall have a Board of Governors, which shall consist of 15 members appointed by the Comptroller General of the United States not later than 6 months after the date of enactment of this section, as follows:
(A) 3 members representing patients and health care consumers.
(B) 3 members representing practicing physicians, including surgeons.
(C) 3 members representing private payers, of whom at least 1 member shall represent health insurance issuers and at least 1 member shall represent employers who self-insure employee benefits.
(D) 3 members representing pharmaceutical, device, and diagnostic manufacturers or developers.
(E) 1 member representing nonprofit organizations involved in health services research.
(F) 1 member representing organizations that focus on quality measurement and improvement or decision support.
(G) 1 member representing independent health services researchers.


Thus, only 3/15 members of the governing board would represent the patients who ultimately reap the benefits or suffer the harms produced by medical diagnosis and treatment. Further, 6/15 members represent for-profit corporations which stand to make more or less money depending on how particular comparative effectiveness studies come out. Also, 3/15 members would be physicians, some of who may get paid more to deliver particular treatments (e.g., procedures) than others (e.g., providing advice about diet and exercise).

We often discuss how clinical research sponsored by organizations with vested interest in the research turning out to favor their products or services may be manipulated to favor these interests, and sometimes suppressed if it does not. In the US, there are few unconflicted sources of sparse funds to support comparative effectiveness research. (The most significant current source is the Agency for Healthcare Research and Quality, AHRQ. For full disclosure, I have been an ad hoc reviewer of grants for that agency.)

The current draft of legislation would create the largest potential sponsor for comparative effectiveness research, but would make that organization report to representatives of for-profit companies whose profits may be affected by the results of such research. In my humble opinion, this is not much of an advance. Comparative effectiveness research controlled by corporations that stand to profit or lose depending on its results will forever be suspect.

If the government is going to support comparative effectiveness research, it ought to make sure such research is not run by people with vested interests in the outcomes coming out a certain way. I may be biased myself, but why not let the research be sponsored by AHRQ, an agency with relevant experience and no axe to grind vis a vis any particular product or service?

7 comments:

Adam J. Fein, Ph.D. said...

Dr. Poses:

I'm curious to know your perspective on comparative effectiveness research conducted by a PBM. As I discuss on my blog last week, Medco Health Solutions announced its intention to launch a comparative effectiveness study of Bristol-Myers Squibb’s Plavix versus Eli Lilly’s Effient. See Medco Takes on Eli Lilly.

Thanks,
Adam

MedInformaticsMD said...

Just my own views (MedInformaticsMD) on a PBM conducting comparative effectiveness studies. Leaves some basic questions not covered in the press release (http://medco.mediaroom.com/index.php?s=43&item=403) and not made clear at clintrials.gov (http://www.clinicaltrials.gov/ct2/show/NCT00995514?term=NCT00995514&rank=1).

Who's actually doing the legwork and what experience do they have conducting such trials; will it be contracted out? What will be their relationship to either the PBM and/or the drug companies? Will the data be made available to all stakeholders, and who will perform the analyses?

If these questions have reasonable answers, such research could be trustworthy although your suggestions as to motive are not unreasonable.

Disclosure: I was at Merck when Medco was a Merck subsidiary.


-- SS

Anonymous said...

Ultimately if CER or EBM is to work you need reliable studies with hard information. This is not the case with a number of currently used drug that have been tested to a surrogate end point as pointed out by Matthew Perrone's AP story Unproven drugs stay on market.

Using the "accelerated approval" we find "And the agency (FDA) has never pulled a drug off the market because of a lack of required follow-up about its actual benefits-"

"Of the 144 studies the FDA has required under the program since 1992, more than one-third are still pending, according to the GAO."

A case in point is:

"In the case of Shire Laboratories' low blood pressure treatment ProAmatine, the required study has gone incomplete for more than 13 years. The GAO found ProAmatine has generated more than $257 million in sales, even though "the clinical benefit of the drug has never been established."

In other cases, the FDA has failed to act even when company studies show drugs did not improve patient outcome."

It appears that a great deal of information already exist to challenge the use and effectiveness of any number of medications. The only reason I can find for not moving on these drugs are profit and the willingness of some in government to look the other way while valuable resources are squandered on pointless treatments.

Steve Lucas

james gaulte said...

My major concern about CER is that the process will be subject to a version of "regulatory capture" in which vested interests would play a key (perhaps determinative) role in making the decisions.The currently proposed organization you describe seems to be one certain vested interests would relish.

Roy M. Poses MD said...

Dr Gaulte -

Exactly!

What is striking is that in this instance the regulatory capture seems to have been built into the governance structure of this organization (at least, as proposed in this version of the bill).

It seems like some Senators see CEOs of big health care corporations as a constituency for a comparative effectiveness institute.

IMHO, this institute should first see the population as a whole as its constituency. The goal of the agency shoul be to figure out which tests and treatments are best (maximize benefits/ minimize harsm) for particular patients and people. The goal should not be to maximize the compensation of corporate CEOs. But the way the governing board is currently proposed to be constituted, that might be the effect.

MedInformaticsMD said...

The concept of using EMR data further complicates the issue of CER.

See the essay I wrote here (PDF).

Scot

Anonymous said...

One issue that needs to be addressed is the investment communities belief that medicine is a growth industry. Time and time again I see information that touts an aging population as the demographic that will drive various companies profits.

Along with this there has to be the realization that companies are now run for the benefit of the executives, not their customers (patients) and not their shareholders. Recent articles have highlighted CEO's salaries and poor year over year returns. These are immediately justified as costing just pennies per share and the cost of hiring the "right' people.

Troubling to me is the salary levels of all of those in these various companies and nonprofit's. A case in point are drug reps. They are paid a lucrative salary not based on ability or academic standing, but to create instant credibility. This is a hallmark of a marketing company, and can be seen in many companies financial statements as making up the lion's share of cost.

My limited knowledge of the current health care reform bill is that there is nothing to "bend the cost curve." What we see are concepts such as this that put in charge those that have the most to gain. Add to this an unshakable belief in technology, see any of MedinfomaticsMD's post, and we have cost skyrocketing, with no concept if what is being prescribed is in reality, helpful.

In a marketing based industry, we spin the information, while structuring the various controlling entities to maximize our profits.

Disclosure: I have no direct investment in a drug or device company, although fund's that I hold, but have no investment control over, may own shares in these companies.

Steve Lucas