The development of novel targeted therapies that capitalize on our growing understanding of the molecular underpinnings and vulnerabilities of specific malignancies has to rank among the most important advances we have seen in the 50 years since the American Society of Clinical Oncology was founded. We have, of course, had truly targeted therapies—dating to the performance of bilateral oophorectomies to deprive estrogen receptor–positive breast cancers of their ligand—since the end of the 19th century, but now we are witnessing and driving an unprecedented expansion in such agents.1
The new agents that have been most successful have truly changed the expected outcomes and treatment course for historically poor-prognosis diseases. Before the development of imatinib (Gleevec), the treatment of chronic myelogenous leukemia (CML) consisted of chemotherapy, bone marrow transplant, and supportive care.2,3 After the development of this agent, a large number of patients have been able to enjoy prolonged periods of disease control with minimal symptoms and without the earlier generation of cytotoxic agents. Moreover, this success has begotten further success with the development of related agents for resistant disease and for other malignancies with related pathophysiology.
Reasons for Noncompliance
One of the seeming advantages of many newly developed agents is their oral bioavailability. Avoiding inpatient or outpatient parenteral treatment is, or should be, less expensive and generally appeals to patients and families. There is, however, an ever-present concern about compliance.
We have studied this for years in the postoperative, adjuvant setting for breast cancer, where compliance with oral antiestrogen treatments is documented to decline over time.4 The reasons for noncompliance include toxicity, cost, convenience, and other factors, all of which we try to confront, given the evidence that prolonged therapy (now up to 10 years in many settings) is associated with further improvements in outcomes.
One would think that CML would not be subject to the same lack of compliance as has been seen in early-stage breast cancer. CML is potentially lethal in the short-term, and the treatment that imatinib replaces was generally far more debilitating and toxic, as well as less
effective.
Health-Care Challenge
The recent study by Dusetzina and colleagues,5 reported in the Journal of Clinical Oncology and reviewed in this issue of The ASCO Post, highlights a challenge for our current health-care system. Simply put, the investigators correlated the out-of-pocket expenses for imatinib with compliance. They showed that as the personal cost in dollars increases, compliance decreases. This is not a surprise, since the finding is consistent with the rest of conventional economic thinking, modeling, and behavior. The question we have to ask is whether this is acceptable.
The health-care market—if you can really call it a market—is unusual in myriad ways. For one thing, the user, in this case a patient with CML, is not the sole purchaser. A third party has prenegotiated the price as well as set the patient’s share. If the patient can’t pay, there is no immediate market response (in the form of lower pricing).
This is, to be simplistic, different from the market for used cars. There, the buyer has options (buy another seller’s car) and the seller has options (lower the price to make a deal). Because health care is so important, we establish third-party insurance (whether government run or private), and we share the financial burden of illness broadly. This, perhaps, limits some of the potential for price gouging at the individual level. But it is acutely unresponsive to the downward pressure on price that lack of demand should exert. And it is similarly inflexible in the face of one individual’s inability
to pay.
Of course, this description oversimplifies a complex and highly evolved system, but the bottom line remains that we (and our fellow citizens) have a shared interest in assuring the optimal health of everyone. Ultimately, we share the costs of everyone’s illnesses directly or indirectly, and it costs all of us when patients with a treatable serious illness are inadequately treated because of avoidable challenges. The question is this: What are we going to do
about it? ■
Disclosure: Dr. Hudis reported no potential conflicts of interest.
Dr. Hudis is President, ASCO; Chief, Breast Cancer Medicine Service, Memorial Sloan Kettering Cancer Center; and Professor of Medicine, Weill Cornell Medical College, New York.
References
1. Beatson CT: On treatment of inoperable cases of carcinoma of the mamma: Suggestions for a new method of treatment with illustrative cases. Lancet 148 (3803):162-165, 1896.
2. Sawyers CL: Chronic myeloid leukemia. N Engl J Med 340:1330-1340, 1999.
3. Goldman JM: Chronic myeloid leukemia: A historical perspective. Semin Hematol 47:302-311, 2010.
4. Partridge AH, Wang PS, Winer EP, et al: Nonadherence to adjuvant tamoxifen therapy in women with primary breast cancer. J Clin Oncol 21:602-606, 2003.
5. Dusetzina SB, Winn AN, Abel GA, et al: Cost sharing and adherence to tyrosine kinase inhibitors for patients with chronic myeloid leukemia. J Clin Oncol 32:306-311, 2013.
