Maurie Markman, MD
It was not that long ago that decisions regarding cancer management were essentially made exclusively by the treating physician, with patients simply being informed of “the plan” for their treatment. In fact, in some settings, patients first learned of "the plan" after initial therapy was undertaken and completed. It may seem foreign today, but in the not-so-distant past, a woman being tested for breast cancer would only know she had the disease when she awoke from surgery to find that her breast—and perhaps considerably more—had been removed.
Today, such events should not occur. Decision-making in oncology is recognized as a process that includes detailed discussions with, and input from, the patient, his or her own advisers, and family. In all but the most unusual of circumstances, it is ultimately the patient who makes the final decisions regarding how his or her cancer will be managed.
Simply put, it is the individual who should have the authority to make final healthcare decisions. This applies whether we are talking about the rather silly issue of whether or not one should drink coffee or very hot beverages because the International Agency for Research on Cancer had previously considered the former and currently considers the latter to be “possibly carcinogenic,”1
or whether an individual woman must decide if having a bilateral mastectomy is appropriate in the management of her breast cancer.2
Of course, optimal decision making occurs when the individual is informed regarding the most currently available and scientifically reliable data. Further, the opinion of experts with recognized credentials and experience may be quite helpful, but surely the potential benefits and harms associated with a particular course of action are best assessed by the patient, who will be experiencing those potential benefits and assuming those potential harms.
Despite the stunning pace of this generation of medical knowledge in the oncology arena and the growing availability of clinically relevant data to the public, it is increasingly common that providing a definitive answer to a crucial clinical question that may help determine what is the optimal management for a particular individual is simply not possible.
It is not difficult to find a number of poignant instances in cancer management where considerable, and often rancorous, debate exists among knowledgeable academic experts regarding the utility of a particular approach. I would strongly argue that patients should be provided the complete available data, and they can decide how to proceed.
An excellent example of this approach is provided by a brief examination of the results of a study exploring the clinical utility of erythropoietin in the management of metastatic breast cancer.3
Both the potential benefits and risks associated with this class of supportive care medications, and the intense debate surrounding use of this product, have been well described in the oncology literature.
A trial involving more than 2000 patients, who were randomized to either receive or not receive erythropoietin, concluded, “this study did not achieve noninferiority objective in ruling out a 15% increased risk in progressive disease/death.” The authors commented that red blood cell (RBC) transfusions “should be the preferred approach for the management of anemia in this population.”3
In addition to the concern that the trial investigators seem to have largely ignored the known potential serious complications associated with RBC transfusions,4
their blanket statement regarding the “preferred approach” rather stunningly appears to negate the critical input of the patient in this principally palliative clinical setting.
Finally, it is appropriate that we consider the ongoing and seemingly never-ending debate regarding the clinical utility of screening for cancers of the breast and prostate. A major issue in this highly controversial subject is that demonstrating a reduction in cancer-specific or overall mortality, the rational endpoint for a cancer screening strategy, almost certainly requires years, or even decades, of follow-up.
However, the technology, or specific diagnostic strategies, utilized in this arena today may be associated with objectively superior specificity and sensitivity, reducing the risk of both false-positive or false-negative results, compared with what was utilized in the peer-reviewed report claiming the absence of utility or cost-effectiveness of a particular older approach.