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Patient Advocates: Flies In The Ointment Of Evidence-Based Care

July 18th, 2011

The women recounted how their lives had been saved as they pleaded for the Food and Drug Administration not to withdraw approval for Avastin as a treatment for advanced breast cancer. They did so even without evidence that it provides benefit and with evidence that it confers risks. Their efforts were ultimately not successful: the FDA panel voted unanimously to withdraw approval. (Some will quibble with this blunt representation of the question debated at the hearing. To read more about this, look here, here and here, for example, for more nuanced discussion.)

Despite careful consideration of the evidence by the panel, however, the indelible picture presented by the media was of heartless government bureaucrats depriving women of a drug that could save their lives, and by extension depriving children of their moms, parents of their daughters and spouses of their partners in the name of one-size-fits-all, evidence-based medicine.

The 30-second reports by broadcast media focused largely on the emotional appeals of the women. As Kevin Outterson noted in his media scorecard in the Incidental Economist blog, print readers sometimes had to get to reach paragraph six or seven to learn about the scientific concerns: Since the drug was provisionally approved in December, additional research failed to confirm that Avastin provided reliable benefit in women with metastatic breast cancer.

This emotionally fraught picture undoubtedly contributes to mounting public opposition to evidence-based care imposed by government-initiated policies to improve the quality and outcomes of care and, in the process, reduce unnecessary expenditures.

Advocacy by individuals for research and treatment of specific diseases is not new. The report Back to Basics: HIV/AIDS Advocacy as a Model of Catalyzing Change released by FasterCures and HCM Strategists a few weeks ago details the remarkable national movement that changed the face of a disease and the life prospects of those who have it.  Similarly, advocates for a cure for breast cancer in the United States have been extraordinarily successful at ensuring that resources are allocated and research agendas funded and at raising awareness about the threat of the disease in every corner of the country.

In past decades, the aims of advocates have frequently been at cross purposes with those of government officials charged with balancing a national research agenda. (See Harold Varmus’ account of his clashes with patient advocacy organizations during his tenure as director at NIH.) But those clashes took place in a time of relative plenty – the NIH budget was in the process of doubling and the nation was enthusiastic about wars against various diseases. Rarely were treatment cost implications drawn from research findings.

The urgent need to reduce the cost of health care has raised the stakes of collisions when the unique needs of individuals come into conflict with taxpayer investments. And nowhere are these collisions more evident than in across-the-board efforts to improve health outcomes and reduce costs by encouraging clinicians to provide care based on research about what works for most patients with the same diseases.

Sheila Rothman, writing about Advocacy Organizations and Evidence-Based Medicine in JAMA, recently illustrated this conflict by citing the responses of disease-related advocacy organizations to an Institute of Medicine survey. Many of the groups, when asked about an essential benefits package “contended that evidence-based medicine should serve merely as an aid in decision-making, not as the basis for it.” Some groups “insist on unfettered access to branded drugs, despite the availability of generics, and access to still unproven interventions.” Others urge “unlimited access to screening technologies, regardless of evidence or cost.”

You can imagine the individual voices behind each example she cites: the woman who is here today because her breast cancer was found by a mammogram when she was 41 (objecting to the evidence-based suggestion of raising the age when screening starts) or the relieved parents whose autistic son is doing well in second grade after four years of applied behavior analysis (despite a lack of evidence for the effectiveness of this expensive intervention).

When we, the public, hear these voices echoing our own concern for our loved ones and ourselves, do we identify with them, with their implication that each of us is unique and that understanding and treating this uniqueness constitutes the art of medicine? Or do we take a broader view, one that insists that for the good of all — slowing the rise in health care costs for the nation, better outcomes for the population — our unique needs may be overlooked and our choices may be limited? That unless we have the resources ($88,000 per year for Avastin, for example), we may forgo a chance to try high-risk treatments that may save the life of our baby, our sister or our dad?

The Intractable Conflict Between Individuals And Evidence-Based Medicine

The conflict between evidence-based medicine and individuals is at the core of the struggle to reduce the cost of care.  I fear it is intractable and will remain so.


  • Because when faced with deciding between the possible survival of their own child, for example, versus “the greater good,” most people will choose the former.
  • Because many clinicians resist the perceived and real constraints on their judgment imposed by institutional incentives to adhere to evidence-based guidelines and they communicate this rejection to their patients.
  • Because the gut-wrenching love that fuels patients and family advocacy leads to policies that perfectly coincide with those of the pharmaceutical and device industries, providing populist cover for well-funded efforts to prohibit the use of evidence about effectiveness and cost when making decisions about insurance coverage.

On the other side there is overwhelming evidence of waste and poor quality — including harm to patients — associated with variations in health care and with costly and unnecessary care.  When resources are finite, every course of treatment with an expensive marginally effective cancer drug displaces resources for more certain remedies.

Conflicts will arise more fre­quently as we learn that some treatment options are not available to us because the evi­dence is not strong enough to support their coverage or there is evidence indicating that potential harms outweigh potential benefits.

Resolving The Conflict: The Need For An “Ongoing Messy Discussion

I have no idea how this conflict will be resolved, but I have a hunch that the exigencies of cost will prevail, and that “shared decision making” will soon include an examination of my credit rating in anticipation of huge payments for treatment, in addition to discussion of the risks and benefits of the treatment itself. But all of us –- whether we favor evidence or my-insurance-should-cover-whatever-might-work as our guiding principle — have to face the tremendous uncertainty inherent in both approaches.  There are limits to what can be predicted and controlled. Cure and the relief of pain are possible sometimes, as is the risk that intervention will exacerbate suffering.

As a person who is alive (I think) because of some long-shot chemother­apies and non-guideline-based surgeries, and who is hopeful about potential for targeted therapies, I am deeply sympathetic to the position of the patients who testified at the FDA Avastin hearing. And I am simultaneously certain that the only way to improve the effectiveness of health care and get some handle on its cost requires that evi­dence play a far more muscular role in shaping the practice of medicine and the delivery of care.

This is not an academic debate for me: Its resolution may at some point constrain my choices and limit my future.  It’s not an academic debate for you either. We need to talk about the tensions and uncertainty, with respect for each other and with open minds. I’m not sure what solutions are possible but without an ongoing, messy discussion, we won’t find out.

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3 Responses to “Patient Advocates: Flies In The Ointment Of Evidence-Based Care”

  1. jim jaffe Says:

    this interesting and important piece raises a key question implicit in health reform legislation about whether anything that can provide any benefit to anybody should be available to all irrespective of cost. my answer is no. the FDA serves a different role, though, in determining whether something is safe enough to use and provides some benefit. the fact that a drug passes this test doesn’t suggest that an individual patient should use it — or that insurance should pay for it.

  2. Linda Bergthold Says:

    There was one patient advocate at the Avastin hearing who had the courage to address the potential harms and risks at issue. She was treated rather poorly by the other women in the room, who apparently booed her. Talk about a messy debate! Of course there are problems with RCTs, but until those, like Mr. Walker, who oppose EBM, come up with a better way to calculate benefits and harms, we must rely on the best science we possibly can. And RCTs, when well designed, are much better than poorly controlled observational studies or anecdotal evidence such as that presented by some of the patients at the hearing. It is not particularly helpful to make a blanket attack on EBM. What would be helpful is a discussion about how individual patient needs can best be served within the context of well designed research.

  3. swalker581 Says:

    An excellent discussion of the tension between “evidence-based medicine” (which actually means 50-year-old, statistics driven, population-based medicine) and the perspectives of patients and many physicians. I would propose however, that advancing knowledge of the biology of disease offers a third choice. What we are learning about the molecular drivers of disease, and the personalized nature of many diseases, is that the “greater good” is not well served by our current concept of “evidenced-based medicine.” “Evidence-based medicine” relies almost exclusively on the rules of statistics (specifically relative-frequentist statistics) and produces little more than rigid, non-representive comparisons of the median outcomes between two groups of patients enrolled in randomized controlled trials. It is now fact, not speculation, that many cancers – like for example breast cancer – are not a single disease. At the genetic level, breast cancer is perhaps a dozen, or 20, or even more different cancers. Cancer subtypes can vary between tumors in the same person, and over time in the same tumors. This knowledge (which I reiterate is fact) is rendering the population-based approaches on which “evidence-based medicine” is built, scientifically wrong. Put simply, evidence-based medcine approaches have never worked very well, and now we know why. The path forward for the development of new medicines must be targeting treatments to the diseases individuals actually have, instead of toward a “disease” that the median patient in a trial population had, and then assuming everyone else had the same disease. They didn’t – which is why half the patients in both the treatment arm and the control arm of every randomized trial does worse, and the other half does better, when compared with the median patient. Consider that those two median patients (one in the control arm and the other in the treatment arm) directly to produce the result of the randomized trial probably don’t even have the same disease sub-type, and the hopelessness of the “evidenced-based medcine” approach becomes clear. The key word here when it comes to getting costs under control is not simple cost, but rather cost-effectiveness. Evidence-based medicine as currently applied is obsolete. Personalized, targeted medicine, in some cases already here, is slowly expanding but not at the rate now possible. Those clinging to evidence-based medicine approaches (esepcially at the FDA) are dramatically slowing and even preventing the arrival of better methods. The first step in achieving cost effectiveness is to make sure we are using the best science, and to make sure that science is advancing in an efficient manner without the drag of obsolete policy and regulation. As our system advances, the opportunities to increase cost-effectiveness will also become apparent. Unfortunately, the obsolete concepts embodied in “evidence-based medicine” are the basis for the proposed Cost Effectiveness Research that will ultimately (let’s not kid ourselves about this) be used to make coverage decisions. It really would be an enormous mis-step to institutionalize an obsolete approach into new agencies and regulatory processes. Doing so would stall medical advancement even more than it is currently stalled by FDA’s rigid reliance on obsolete “evidence-based” approaches. The conversation will indeed be messy, and it is already underway. I was at the Avastin hearing as an advocate for those women, and their attendance is a strong signal to policy makers that patients intend to have a seat at the table. If they are not given one willingly by policy makers, they will build their own seats, and if necessary, their own table. The message, well-supported by new scientific knowledge, is that the greater good and the individual good are not separable. In fact, they are the same thing. One won’t come without the other.

    Steven Walker, Co-Founder, Abigail Alliance for Better Access to Developmental Drugs

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