Costs associated with anticancer therapeutics continue to rise, although it also is recognized that the overall effectiveness of therapy has improved.
Maurie Markman, MD
The financial impact of a cancer diagnosis on patients and their families is increasingly recognized as a serious and worsening societal concern.1,2 Costs associated with anticancer therapeutics continue to rise,3,4 although it also is recognized that the overall effectiveness of therapy has improved.5
The relationship between the benefits of care versus the cost of specific interventions (including the administration of pharmaceutical agents) has been a raging debate within the health care community for decades. The urgency associated with the question has only increased in recent years with the rapid acceleration in the development of novel approaches for managing multiple serious medical conditions.
No area of medicine has been more affected than oncology by the striking success of innovation such as new drugs and devices that improve the quantity and quality of life, which essentially has developed in the absence of a coherent strategy to control the rapid rise in the cost of care. This rapid rise has occurred despite the concerted efforts of governmental and private payers and employers. Over the past several years, a number of organizations and academics have advanced their opinions about this societal dilemma. These efforts include attempts to define the “value” of a specific intervention, and, notably, to declare that a particular intervention or drug may not provide sufficient value to justify its use in routine (noninvestigative) oncology care.
This commentary highlights the question of how value in cancer care will be determined in the future and specifically acknowledges the critical difference between the population versus the individual when considering these issues. Samuel Hellman, MD, a noted radiation oncologist and a 2016 Giants of Cancer Care® award winner, eloquently articulated the matter in his provocative 2014 essay published in the Journal of Clinical Oncology6:
“The utilitarian [approach] is clearly appropriate to public health considerations, but what is learned for public health may not necessarily be in the best interest of an individual patient….Too often we assume that survival or cure is a sufficient metric, with no similar quantitative measure of other factors. This often leads to the so-called best treatment being not what the patient wants. All personal care requires consideration of both the helpful and harmful consequences of treatment in the context of individual patient comorbidity, preferences, and fears.”
This commentary can only begin to scratch the surface of the critical questions posed by Dr Hellman. How much do we really know about the wishes of individuals with cancer and other serious medical conditions regarding their care and their perceptions of meaningful outcomes, as opposed to what clinical investigators, academic leaders, and health policy experts have suggested, or at times proclaimed, in peer-reviewed publications as being the most relevant? Below are several examples to consider.
This commentary can only begin to scratch the surface of the critical questions posed by Dr Hellman. How much do we really know about the wishes of individuals with cancer and other serious medical conditions regarding their care and their perceptions of meaningful outcomes, as opposed to what clinical investigators, academic leaders, and health policy experts have suggested, or at times proclaimed, in peer-reviewed publications as being the most relevant? Below are several examples to consider.7
This degree of discordance between what oncologists claim to have measured and what patients report they actually experienced raises a concern about the reliability of published clinical trial assessments for providing a meaningful assessment at the individual patient level of the risk-to-benefit ratio associated with study regimens.
In a survey of 374 women treated for breast cancer, investigators found that the patients’ perception of the quality of their care was strongly related to the “process of getting care” rather than a measure of the specific medical interventions delivered.8 Therefore, how patients with cancer feel they are being treated by their clinical team may be as important—if not more important—as the regimens they receive.
Several recently reported studies have noted the frequent use in breast cancer (early stage or advanced/metastatic disease) of tumor markers or imaging techniques where there was limited or no evidence that such testing improves clinical outcomes, particularly in individuals at low risk of progression or where there was no evidence of involvement of the organ being examined (eg, central nervous system screening in the absence of neurologic symptoms).9,10 The studies’ authors high-light the need for future investigation to understand the reasons for widespread use of these strategies. Of note, there is the reassurance “value” that testing may provide a patient when a negative test result is obtained. How does one calculate the emotional versus financial cost benefit associated with such individual reassurance?
The final example relates to the fundamental goals of therapy and the primary outcomes of anticancer drug therapy studies. The industry gold standard is a statistically significant improvement in overall survival in an appropriately powered randomized phase 3 trial. However, in a provocative detailed interview with 22 survivors of ovarian cancer, the respondents challenged this assertion, suggesting instead that “they preferred an individualized approach to care focusing on quality of life.”11
Clearly, there is much to learn through a far more expansive exploration of “value,” as defined by the patient with cancer, that hopefully will take place in the future.