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.