Define "Effective": The Curious Case of Chronic Cancer
- Define "Effective"The Curious Case of Chronic Cancer
Comparative effectiveness research tends to focus on head-to-head comparisons of treatment approaches: Is A more effective than B? What does the evidence say? From these comparisons, the research aims to guide physicians' recommendations and, thereby, patients' choices.
But what if the definition of effectiveness is itself in contention? Can comparative effectiveness research still inform efforts to improve the quality of health care and particularly the physician-patient relationship? Chronic cancer offers a way to explore these questions and their ethical dimensions.
The emerging idea that cancer can be a chronic disease reflects immense changes in oncology over the past decade. More drugs, new therapeutic approaches, and better outcomes have enabled many patients to live longer with cancer, and, thus, with cancer treatment and its consequences. Some cancers, such as chronic myelogenous leukemia, have officially become chronic diseases. Oncologists may also use the term "chronic" to characterize advanced (stage III or IV) disease that is not curable but is stable, or may become stable, in response to treatment. Still other cancers may be considered chronic because they follow cycles of recurrence, treatment, and remission that can be anticipated.
Among the recognized ethical goals of chronic care is for clinicians to help patients accommodate the burdens of living with a serious disease, its treatment, and the impairments that may result, so that the patient's life is not defined solely by the illness. But oncology professionals—including medical oncologists, cancer surgeons, nurse practitioners, palliative care specialists, and other clinicians who work in cancer centers—have not yet agreed on an ethical framework for managing cancer as a chronic disease. The chronic disease paradigm established for primary care clinicians, which emphasizes the patient's self-management of a disease and the clinician's responsibility to support the patient, suggests parallel obligations for both. However, most cancers differ from other serious chronic diseases in their ability to adapt and become resistant to treatment. Therefore, patients' psychological burden of living from scan to scan with the disease they will probably die of is compounded by uncertainty over whether their current regimen is working, and how long it will be until the task of self-management will require them to learn, adapt to, and trust a different regimen. Change is their norm.
Andrew D. Seidman, an oncologist at Memorial Sloan-Kettering Cancer Center, has written that metastatic breast cancer, which is increasingly treated and experienced as a chronic disease, "often behaves biologically ('reads') like a novel with many chapters."1 The plot of this novel will reflect the natural history of the disease, the individual patient's biological factors (such as her hormone-receptor status), the history of efforts to intervene in the disease's progression, and how the patient responds to and tolerates treatment, as well as her values and preferences concerning it. Luck is also a factor.
Current debates within clinical oncology over how to manage metastatic breast cancer demonstrate how oncologists have learned to use comparative effectiveness research and suggest how they may draw on it to develop a normative framework for managing chronic cancer. Fewer than ten years ago, some breast oncologists thought that several chemotherapy drugs given simultaneously would produce better outcomes [End Page 17] than single chemotherapy agents given sequentially. However, a major study published in 2003 in the Journal of Clinical Oncology compared two agents given singly and in combination and concluded that the combination offered no lasting therapeutic benefit over the drugs in sequence.2 While the combination might elicit a greater response initially, it did not increase patients' chances of living longer and was both more toxic and more detrimental to their quality of life.
In his frequently cited editorial commenting on this study, Seidman noted that while some individual patients might derive greater benefit from combination therapy than from sequential, single-agent therapy, as yet "we lack high-level, evidence-based medicine to guide such choices."3 According to Seidman, comparative effectiveness research sent "a loud message" to oncologists to avoid thinking of combination therapy as a more-is-better "gold standard" of care.
Seidman also introduced a...
