Guest Blog: Advocacy groups threaten evidence-based guidelines

Josh Freeman, MD is Chair of the Department of Family Medicine at the University of Kansas School of Medicine. The following is an excerpt from a post first published on his blog, Medicine and Social Justice.

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Two recent “Commentaries” in the same issue of JAMA address different challenges to the implementation of evidence-based practice guidelines. One, “Direct-to-consumer cardiac screening and suspect risk evaluation,” addresses the challenges posed by the potential for profit that occurs when companies market screening tests directly to the consumer (DTC) that may not be indicated, may not be indicated for the people who it is marketing to, or may even be harmful to the recipients of such screening. This harm can be physical, as in untoward events, or in risks inherent in the further procedures for those who “screen positive” but turn out to have been “false positives.” The harm is also financial, for there is a cost to doing these tests – to the individual (sometimes) or to their insurer; in the latter case, whether that insurance is public (e.g., Medicare, Medicaid) or private, the cost is to all of us. And, of course, that cost is the reason for such marketing, as it is what translates into profit for the company selling the test.

In the case of cardiac screening, Lovett and Liang, using the recommendations of the US Preventive Services Task Force (USPSTF), the American Heart Association/American College of Cardiology Foundation (ACCF/AHA), describe heavily-marketed tests that can cause more harm than benefit to the many people who are not in the narrowly-targeted group for whom the tests are indicated; other tests which have valid indications although, again, in the appropriate populations; and finally tests for which there is little or no evidence. The marketing of these tests can incur anxiety or fear in people who have no reason to be worried about these conditions and may occur outside the physician-patient relationship, thereby not allowing people to get the physician’s analysis of the results even when the test is indicated, and cost a great deal of money. This is particularly true when a possibly-positive test needs to be followed up with a number of other tests to rule it out, this time covered by insurance. They make several suggestions for how DTC companies should be regulated. They conclude that, “DTC cardiac testing may pose more harm than benefit to many consumers. Oversight efforts are needed to protect the public from inappropriate testing and to ensure that the health care dollar is spent on care promoting health in an evidence-based fashion.” I would say that is a mild suggestion!

The issue raised by the other article is probably more insidious; because it lacks the obvious profit motive present in DTC marketing, we may be less wary. In "Health advocacy organizations and evidence-based medicine," Sheila Rothman examines the tendency for groups created around a single disease (e.g., breast cancer, epilepsy, autism, etc.) to push for screening tests, diagnostic tests, and treatments for “their” disease whether or not there is evidence to support their use. In response to an Institute of Medicine (IOM) survey that sought to identify what tests and treatments should be included in a basic benefits package, “They contended that EBM [evidence-based medicine] should serve merely as an aid in medical decision making, not as the basis for it. Outcome data, they insist, should not limit patient choice or restrict available services.”

This is truly remarkable. These groups are basically saying “our disease is so important we should screen everyone, everyone who screens positive should have diagnostic tests, and everyone who has it should be able to get any treatment that they want, even if there is weak or no evidence for its efficacy. And, of course, someone else should pay for it.” This would probably be unreasonable if there was only one such disease, but there are hundreds and each is the “most important” for its advocacy organization. The reason that this is more insidious is that these groups are not seeking to make a profit on these tests and treatments (usually); they are (usually) functioning as advocates for people who are truly suffering. However, the fact that people are suffering does not mean that society should screen large numbers of low-risk people for all of these conditions.

Sometimes, advocacy groups may have a financial interest in a test or treatment, but even when they do not, they may push for policies that allow physicians to prescribe expensive brand-name drugs even when generic drugs have not been tried. Or they may demand the availability of expensive but unproven treatments: “’Autism Speaks’ criticized insurance companies that refused to pay $300,000 for 4 years of applied behavior analysis therapy for toddlers, despite a lack of evidence of effectiveness.” That is a lot of money even if we know a treatment is effective, but it is an awful lot when we do not. The issue is that this organization is focused only on people with “its” disease, and has no concern for what the money could otherwise be used for to treat other people with proven therapies. Maybe this is their role, but there is no reason that the rest of society must buy into this logic.

Cancer, of course, has the strongest set of advocacy organizations. In this context, the Food and Drug Administration recently approved several new drugs for metastatic prostate cancer. The cost can be over $90,000 for each treatment cycle and might exceed $500,000. In the June 28, 2011 NY Times article, “New prostate cancer drugs extend lives but raise costs”, Andrew Pollack notes that Medicare is going to look into whether to pay for these drugs, but that “…some patient advocates and politicians portrayed the review as a step toward rationing.” Excuse me? Don’t we already have rationing based on whether or not one has money or insurance? It is not okay for “patient advocates” to advocate for some people to get any kind of treatment paid for by someone else (e.g., Medicare) when everyone who has that medical need is not able to get care. And since there will be more things to spend money on than there is money, there needs to be a more rational system than “Me first!” This system is the use of evidence-based guidelines.

Rothman concludes her commentary: “However valuable independent advocacy organizations are for a democratic society and however important their services provided to targeted populations, their advocacy positions and the related underlying assumptions must be scrutinized with the same diligence as those of other stakeholders. There should be no automatic assumption that all health advocacy organizations deserve special standing or represent the common good.”

And for good reason - because they probably do not.

How politically unpopular research helps us make better medical decisions

When a new drug goes on the market for, say, diabetes, doctors are typically bombarded by advertising messages that promote it. Patients may see television commercials touting the new drug’s advantages over older ones and advising them to "talk to your doctor" about obtaining a prescription. But since the U.S. Food and Drug Administration only requires drug companies to prove that new drugs work better than placebos (sugar pills), there’s often little or no reliable information about whether a new drug is actually an improvement over existing therapies.

How, then, can a doctor like me make good choices about which treatment to give each patient? To provide answers, last year's health reform law devoted billions of dollars over the next decade to "comparative effectiveness" research, or research that directly compares different tests and treatments to determine which are better at improving health in particular groups of patients.

Funding for comparative effectiveness studies was recently targeted for budget cuts by politicians who argue that such research may limit patients' treatment choices and lead to the withdrawal of insurance coverage for drugs that are found to be costly or ineffective. In reality, however, that’s not necessarily the case. For example, when the FDA recently concluded that the cancer drug Avastin does not help patients with breast cancer (and often causes serious side effects such as bleeding, heart attacks, and heart failure), officials were quick to assure the public that the Medicare program will continue to pay for it anyway.

What has been lost in the political debate is the value of comparative effectiveness studies in helping doctors and patients make better treatment decisions. For example, the Effective Health Care Program at the Agency for Healthcare Research and Quality (AHRQ), which sponsors comparative effectiveness research for many conditions that I commonly treat among my patients, recently published a patient-friendly guide to medicines for type 2 (adult-onset) diabetes that clearly spells out the advantages and disadvantages of the many drugs available to lower blood sugar. Patients can read this guide online or print out a copy to take with them to their doctor. (Full disclosure: I am a former employee of AHRQ, but was not involved with this program.)

Also, in a 2009 study published in the Archives of Internal Medicine, Mayo Clinic researchers concluded that a "decision aid" for patients with diabetes based on a previous version of the AHRQ medication guide improved patients' knowledge and involvement in their care. This decision aid consisted of six flash cards containing basic information about the effects of five types of diabetes drugs on weight change, blood sugar levels, side effects, and ease of use (when to take it and how often blood sugar testing is required).

In a highly charged political environment, it’s tempting to score points (and scare patients) by falsely equating comparative effectiveness research with “rationing.” But I believe that the valuable information obtained from this research, rather than restricting patients' treatment options, will instead empower them and their doctors to make better decisions about their health care.

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The above post was first published on my Healthcare Headaches blog at USNews.com.

Doctors, don't "dispense as written"

I've always favored prescribing generic drugs over handing out brand-name samples, since the latter, while initially "free" for patients, can actually be less effective and cost them more money in the long run. In fact, the only patient for whom I can remember routinely writing "Dispense as Written" (forcing the pharmacy to dispense the brand-name drug rather than the generic) on prescriptions was a special case: she insisted that I do so, because she believed that the brand-name worked better for her condition than the generic did. (And she may very well have been right, although she would have been a rare exception to the rule that generics are therapeutically equivalent to the brand-name drugs they replace.)

In an excellent story published in yesterday's Washington Post, however, health columnist and fellow Georgetown faculty physician Ranit Mishori reports that up to half of U.S. doctors prescribe brand-name drugs because they erroneously believe that generics are less effective, costing the U.S. health care system up to $8 billion per year. And that's only the cost savings associated with the differential in drug prices, which, in the case of the cholesterol drug Zocor (generic name: simvastatin), can be nearly $400 per month. Mishori goes on to write:

But the cost benefits may run deeper, because several studies also show that patients taking generics are more likely to take them properly. According to [Harvard Medical School professor William] Shrank, patients paying more for pharmaceuticals are “less likely to take their medication” as prescribed. Financially burdened by the cost of their drugs, they are more likely to skip doses so that their drug lasts longer, to cut the pill in half or, when they see the price rung up at the checkout counter, to leave the pharmacy with no medication at all. “And that,” he says, “is bad for clinical outcomes.” And expensive, too, when you consider the added burden to the system of people unwell because they are priced out of the medications they require.