I had written about abandonment of care among children in the developing world as a grave concern for oncologists everywhere because of its implications for our failure to deliver the best care to the majority of the world’s cancer patients. Implicit is the need to focus on delivery of what we know. I quoted a wonderful Shattuck Lecture by Claude Lenfant in which he lamented our failure to deliver the great advances emerging from biomedical research to our patients—advances that were thus “lost in translation.”

Regrettably, Dr. Lenfant’s assessment of these failures to deliver the best care referred to our own country, not to the resource-poor countries of the developing world. While his lecture was an indictment of the slow uptake of medical knowledge among practicing physicians, a few recent vignettes from the world of oncology highlight the additional obstacles to delivery imposed by our deplorable system of medical care.

New, highly specific molecularly targeted agents are a triumph of translation of laboratory findings to the clinic. Among the most remarkable is the incorporation of all trans retinoic acid (ATRA, Tretinoin) in the treatment of acute promyelocytic leukemia (APL)—a development that has transformed the therapy for this rare, highly lethal malignancy. This unusual variant of acute myeloid leukemia is characterized by severe bleeding manifestations complicating induction therapy, and poor overall outcome—both features that have been overcome by the incorporation of ATRA, and more recently arsenic into therapeutic regimens. Disruption of the retinoic receptor on malignant cells by the molecular translocation which characterizes APL, and overcoming the differentiation block with pharmacologic doses of ATRA represents a remarkable return on investment into understanding the biology of acute leukemia. It might be argued in this case that the effect of ATRA was known empirically and that the laboratory justification for the effectiveness of this targeted therapy came later. Nevertheless, the impact on therapy and outcome of patients with APL is now indisputable.

So, what more could we ask for than an oral agent that not only results in prolonged remissions and cure of APL, but also reduces inpatient stays, transfusion requirements, and perhaps avoids stem cell transplantation for patients with this leukemia? The story of Judy Ariba highlighted in USA Today last month is sobering. This woman in her sixties with APL had the additional misfortune of having health insurance that utilized a “tiered” copayment scheme designed to control health care costs. In this scheme, patients do not pay a flat dollar amount as copayment for prescriptions; rather, patients are asked to pay a percentage of the cost of their prescriptions (varying from 25-33% or more) for expensive “specialty drugs.”

As a result, Ms. Ariba was forced to pay $1700 per month (25% of the $6800 monthly cost) as the price of maintaining her remission. And she had health insurance! We can assume that the situation is worse for more than 50 million Americans who have no health insurance; the inferior outcome for Americans with cancer who have no health insurance has been documented, and the outcome for those who depend on Medicaid for coverage is not much better.^1-4

We would probably all agree with Ms. Ariba’s pronouncement that “no one has $1700 a month” for anticancer drugs, and we might reasonably ask: What, then, is the point of designing such targeted therapies if they remain unaffordable for our patients? And wouldn’t you think that insurers would be delighted to spend the additional $1700 per month instead of covering the costs of prolonged hospitalization, marrow transplantation, etc.? Ironically, insurers will probably have to pay such costs should Ms. Ariba and patients like her develop relapse of leukemia—a circumstance that might be preventable if patients were able to comply with prescribed therapy.

Ms. Ariba’s story, of course, is not the only example of a miraculous advance “lost in translation” [or more ironically, of a miraculous advance of translational science lost in translation]. An interesting abstract from the 2011 ASCO Annual Meeting reported the results of a study of a nationally representative pharmacy claims database including more than 10,000 patients initiating oral oncolytic therapy between 2007 and 2009. Abandonment of newly prescribed oral oncolytic therapy occurred in 10% of cases, and the likelihood of abandonment increased for patients enrolled in plans with pharmacy benefit designs that required high cost sharing; claims with cost-sharing over $500 were four times more likely to be abandoned than claims with cost-sharing of $100 or less. The miraculous targeted therapies that are being added to our armamentarium against cancer are only worthwhile if patients take them, and that is only likely if our patients can afford them.

So, as we congratulate ourselves on the spectacular advances emerging from understanding the complexity of cancer on the molecular level and their translation into targeted therapies for our patients, it behooves us to take stock of our capacity to deliver these advances to our patients and how much of what we learn gets lost in translation.

Fortunately, the Affordable Care Act (ACA) includes provisions outlining a framework for essential health insurance benefits and eliminating lifetime and annual coverage limits. The ACA also offers assistance for individuals and families below 400 percent of the federal poverty level, in addition to requiring limits on out-of-pocket expenses in all insurance plans, except those that are grandfathered. The law also protects patients with chronic conditions by ensuring that health insurance is available to all individuals regardless of prior history. However, it is crucial that these provisions of the ACA are fully and effectively implemented to ensure that cancer patients do not experience the financial and health hardships associated with lack of adequate insurance.

¹Ayanian JZ, Kohler BA, Abe T, et al: The relation between health insurance coverage and clinical outcomes among women with breast cancer. N Engl J Med 329:326-331, 1993

²Kelz RR, Gimotty PA, Polsky D, et al: Morbidity and mortality of colorectal carcinoma surgery differs by insurance status. Cancer 101:2187-2194, 2004

³Roetzheim RG, Gonzalez EC, Ferrante JM, et al: Effects of health insurance and race on breast carcinoma treatments and outcomes. Cancer 89:2202-2213, 2000

⁴Roetzheim RG, Pal N, Gonzalez EC, et al: Effects of health insurance and race on colorectal cancer treatments and outcomes. Am J Public Health 90:1746-1754, 2000