Is It Time to Address the Fiscal Reality of the Cancer Care Model and Consider Alternatives?

Maurie Markman, MD

This commentary begins with the poignant words of the great American philosopher Yogi Berra, who is reported to have declared: “You can observe a lot by watching.” So, we note 2 recent items in the medical world that, if interpreted correctly, should send chills down the spines of those considering the possible—and perhaps even likely— financial future of oncology care in the United States.

The first point to be highlighted is the recent regulatory approval of 2 new gene therapeutic agents for the management of sickle cell disease.¹ As a physician who cared for individuals with this extremely difficult chronic disease process during my internal medicine training, I can attest to the potentially devastating impact of sickle cell disease. The realist potential to cure or, at a minimum, substantially favorably impact the serious sequela of the long-understood genetic defect of this illness can legitimately be described as a remarkable scientific achievement.

The therapy is not without substantial risk due to the need for a myeloablative conditioning regimen, and it must be noted that efficacy beyond the existing several years of follow-up for patients who have completed the entire drug delivery process is currently unknown.¹ However, trial data are impressive, with all research subjects achieving successful engraftment with no graph failures or rejections being reported. And most relevant, acknowledging the clinical trials were relatively limited in size, greater than 90% of individuals who received 1 of the 2 sickle cell gene therapies experienced no evidence of severe vaso-occlusive events (the primary study end point) based on the specific trial-defined outcome parameters.

Although it remains unknown whether 1 or both of the newly approved agents will ultimately prevent the recognized serious long-term effects of sickle cell disease (trials evaluating this critical issue are ongoing), the relatively early results are remarkably encouraging, leading to drug regulatory approval in the United States.

So, what is the problem? The answer to this question is simple: It is the approximately $2- or $3-million price tag (depending on the agent) for 1 dose of the gene therapy, which would not include the additional substantial costs of the process, including the required myeloablative regimen.

Further, it is estimated that there are as many as 50,000 individuals with sickle cell disease who may be candidates for this novel therapy, bringing the total societal costs of providing this intervention to more than $100 billion.² There is no intent in this discussion to challenge the clinical use or societal impact associated with this novel sickle cell disease management strategy,² and the financial implications associated with gene therapy are now (or soon will be) relevant for both rare genetic diseases and more common clinically severe clinical entities (eg, hemophilia).^3-6

Further, the major impact of a new class of pharmaceuticals for a serious clinical condition is certainly not restricted to molecular manipulations. For example, consider the costs associated with remarkably effective therapy for hepatitis C infection.^7,8 Despite the well-confirmed clinical utility data, use of this therapy has been revealed to be woefully inadequate, with cost being a major contributing factor.⁹

The first part of this commentary has focused on the staggering costs of novel and increasingly effective (even curative) therapies based on our rapidly increasing understanding of the natural history, pathophysiology, and molecular biology of serious human disease.

And while one may legitimately argue that for most individuals the lifetime costs associated with management of chronic illness, such as sickle cell disease, hemophilia, and hepatitis will likely exceed those of the highlighted pharmaceutical agents, it is important to acknowledge that our health system rarely considers the long-term financial benefit (measured over several decades) when evaluating the cost-benefit relationship of a specific therapeutic intervention. In fact, because of the objectively stunning complexity of health care financing, cost vs benefit achieved in most settings will be measured by impact in a single budgetary year or, at most, a horizon of several years.

Now we briefly turn to the second item for discussion in this commentary: the most recent yearly report from the American Cancer Society regarding the current state of cancer diagnosis and cancer-related mortality in the United States.¹⁰ The available data continue to reveal the striking population-based reductions in deaths from malignant disease. This major public health success story is the result of decades of efforts to reduce smoking, the routine use of known beneficial cancer screening strategies, and the influence of increasingly effective therapeutics.

However, the report also noted that for the first time, it is anticipated there will be more than 2 million new diagnoses of cancer in the country in the coming year. Due to the aging of the population, this number will surely continue to climb each year despite all known strategies to prevent malignant disease. Although it is highly reasonable to suggest the trend of earlier diagnosis will continue to reduce both cancer-related morbidity and mortality, the larger number of new diagnoses will also result in more individuals requiring the increasingly expensive (and increasingly effective¹¹) novel therapeutic interventions.¹²

Finally, when one adds to the conversation the recognized existing financial burden assumed by patients with cancer and their families,¹³ the question must be asked: How long can this situation continue, and when will society finally be ready to deal with our country’s simply unsustainable health care fiscal model?

Maurie Markman, MD, is president of Medicine & Science at City of Hope Atlanta, Chicago, and Phoenix.

References

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