We need to be very thoughtful about which drugs or technologies we provide and approve for use, meaning new agents and devices need to be a lot better than what is already available.
—Lowell E. Schnipper, MD
Should cost be a consideration when deciding on treatment for patients with cancer, and if so, what kind of ethical dilemma does that pose for oncologists? With U.S. spending on oncology drugs expected to climb more than 20% annually over the next decade—reaching $173 billion by 2020, according to the pharmacy benefit management organization Express Scripts1—more and more oncologists are grappling with these questions. The issue of soaring health-care costs and how to pay for them is coming under ever-closer scrutiny as targeted therapies carrying hefty price tags and, in many cases, delivering only a modest benefit continue to come onto the market.
Case in Point
One recent example of this phenomenon is pertuzumab (Perjeta), the newly FDA-approved drug in the treatment of metastasized HER2-positive breast cancer. The wholesale cost of pertuzumab is $5,900 a month, and the drug is used in combination with another HER2-targeted antibody, trastuzumab (Herceptin), which costs $4,500 a month, plus docetaxel chemotherapy. A full 18-month course of of this combination regimen costs a staggering $188,000. In a phase III clinical trial,2 patients who received the three-drug combination gained a median of 6 months’ progression-free survival compared with patients treated with trastuzumab and docetaxel alone.
In 2011, the FDA approved the immunotherapy ipilimumab (Yervoy), the first medication shown to prolong survival in metastatic melanoma. In a randomized clinical trial,3 patients treated with ipilimumab lived an average of 3.6 months longer than patients in the control group. The cost for a complete course of treatment with ipilimumab, which consists of four infusions over 3 months, is $120,000.
Health-care expenditures in the United States reached a budget-busting $2.6 trillion in 2010—more than 10 times the $256 billion spent in 1980,4 and growing faster than the national income. This trend combined with reduced reimbursement for costly treatments and procedures by insurance companies, shifting more of those costs to consumers, is leading to the inevitable question: When evaluating cost vs benefit, how much is a little more time worth?
“Generally, people are willing to pay more or place a higher value on life-prolonging treatments, so there is some value to that. But ultimately the question becomes, how much can we afford?” said David H. Howard, PhD, Associate Professor in the Department of Health Policy and Management at Emory University. “Chemotherapeutics very starkly raise the issue of trading off health and money in a way that you don’t find in other areas of medicine.”
Other industrialized countries already consider a treatment’s cost when deciding whether to pay for it. For the most part, however, that discussion is not yet happening in the United States, where such a notion is equated with health-care rationing.
“There is a lot of huffing and puffing about rationing in the U.S. today that is all very politicized and misguided,” said Howard Brody, MD, PhD, Director of the Institute for the Medical Humanities and Professor of Family Medicine at The University of Texas Medical Branch at Galveston. “The fact is we already ration care. We ration care based on an individual’s ability to pay and what kind of insurance someone has, which determines ability to pay. The real questions are what type of health-care system do you have in place, and how fairly is care rationed within that system? Those are the ethical questions.”
According to Dr. Brody, the ethical debate of rationing cancer care falls into two categories. The first is the situation in which a lot of health-care dollars are spent on services proven to provide little or no benefit to the average patient. “In theory at least, you have not denied these patients anything that’s beneficial, and that puts a different ethical slant on it,” he said.
The second category poses a greater ethical dilemma for physicians—and society. “In cancer care, you often can’t say there is no benefit derived from a specific treatment. You can say that the benefit may be very small compared to a very high cost. But that low benefit is the average, meaning that some people will have no measurable benefit, their life expectancy will be the same regardless of the treatment; a few people will die sooner and in misery due to treatment toxicities; and some people will benefit greatly, gaining a significantly extended life span. Ethically speaking, rationing this kind of care is a very different matter from rationing care where, to the best of our knowledge, the benefit is truly zero,” said Dr. Brody.
According to the Dartmouth Institute for Health Policy and Clinical Practice, 30% of health-care spending, or $800 billion a year, is wasted on ineffective measures, including treatments and diagnostic tests.5 One way to combat health-care waste, said Dr. Brody, is for physicians and patients to have an honest conversation about the advantages and disadvantages of a prescribed therapy in a process he calls “shared decision-making.”
“The ethical ideal is tailoring a specific approach to a specific patient’s wishes once he understands the options and consequences of treatment. If a patient is desperate for even an extra week of life and he doesn’t care how much he suffers—he just wants to live a little bit longer—that’s one thing. On the other hand, if he does not want to suffer and prefers to have quality of life, even if it means dying sooner, that’s a different story,” said Dr. Brody.
However, shared decision-making does not solve the ethical dilemma of how to equitably allocate limited health-care resources so everyone benefits, not just those with the ability to pay for costly therapy. “Right now we have a very unfair health-care system in which some people after considering their options are able to say, ‘On balance, I’d rather have that chemotherapy even though it costs $200,000,’ because they are able to afford it, but many other people can’t. We have to address the social injustice of that,” said Dr. Brody.
What should the tradeoffs be when weighing the cost of medical treatments and their benefits, and how should health-care resources be equitably distributed? These issues need to be decided by society, not by the physician, maintained Lowell E. Schnipper, MD, Chair of ASCO’s Cost of Cancer Care Task Force, Theodore W. and Evelyn Berenson Professor in the Department of Medicine at Harvard Medical School, and Clinical Director and Chief of Hematology/Oncology at Beth Israel Deaconess Medical School, Boston.
“In the exam room, the doctor is morally obligated to be the intermediary between the patient and his disease, with the goal being either to cure him or to help him live as well as possible with that disease. Informing patients of their options becomes key to helping oncologists learn what they want. Oncologists have to be their patients’ guide by telling them what the available evidence tells us about how they are likely to benefit from treatment. But the exam room is not an opportunity to be an agent of societal change, because our moral obligation is to our patients,” said Dr. Schnipper.
“If the patient wants something the doctor feels is unethical or immoral, the doctor has the opportunity to excuse himself from the case,” he added. Nobody really does that with any frequency, but the doctor shouldn’t be forced to do something he feels is not appropriate.”
Reining in Costs
Some national initiatives recently put in place should help stem exploding health-care costs, at least for a while. The Patient-Centered Outcomes Research Institute, which was created by the Patient Protection and Affordable Care Act, is funding comparative effectiveness research to provide physicians, patients, and caregivers with evidence-based data to help them make informed decisions on treatments and health outcomes.
And last spring, as part of the American Board of Internal Medicine’s Choosing Wisely® initiative, ASCO issued its list of the Top Five common costly tests, procedures, and treatments that are not supported by evidence to have meaningful clinical benefit. The list includes unnecessary use of chemotherapy for patients with advanced cancers who are unlikely to benefit, use of costly imaging technologies for staging of early breast and prostate cancers and for detection of breast cancer recurrence, and overuse of drugs to stimulate white blood cell production in patients receiving chemotherapy.
According to Dr. Schnipper, the effort is already having positive results. “ASCO’s initiative is penetrating the exam room and influencing the conversation between the doctor and patient, specifically in the circumstance in which a patient has advanced cancer and poor performance status. I’ve had colleagues say that having the Top Five list is adding a sense of legitimacy and credibility when they have to explain to patients that they are at a point where active cancer treatment won’t have much or any likelihood of benefit,” said Dr. Schnipper. ASCO’s Cost of Cancer Care Task Force is now considering other high-cost procedures and treatments that have limited clinical benefit to add to the list.
Another initiative under the Affordable Care Act is a national pilot program on payment bundling that will be launched in January, in an effort to maximize health-care savings over the long-term. This program will test shifting to a health-care system in which all providers involved in treating a single illness, such as breast cancer, are paid under one negotiated fee rather than according to the current fee-for-service payment. The existing system often results in duplicative or unnecessary tests and the use of newer expensive treatments that may not provide greater benefit than older, cheaper chemotherapies. If these and other cost-saving initiatives prove successful, they may avert the need for severe mandatory restrictions on health care in the future.
“We need to think differently about how we ‘cost out’ technologies,” said Dr. Schnipper. “We need to be very thoughtful about which drugs or technologies we provide and approve for use, meaning new agents and devices need to be a lot better than what is already available. We might even need a model in which the drugs we use are paid for only if they provide the desired effect.” ■
Disclosure:Drs. Howard, Brody, and Schnipper reported no potential conflicts of interest.
1. Express Scripts Research & New Solutions Lab: 2011 Drug Trend Report, published April 2012. Available at www.express-scripts.com. Accessed November 12, 2012.
2. Baselga J, Cortes J, Kim SB, et al: Pertuzumab plus trastuzumab plus docetaxel for metastatic breast cancer. N Engl J Med 366:109-119, 2012.
3. Hodi FS, O’Day SJ, McDermott DF, et al: Improved survival with ipilimumab in patients with metastatic melanoma. N Engl J Med 2010; 363:711-723, 2010.
4. Centers for Medicare and Medicaid Services, Office of the Actuary, National Health Statistics Group: National Health Care Expenditures Data, January 2012. Available at www.cms.gov. Accessed November 12, 2012.
5. Dartmouth Institute for Health Policy & Clinical Practice: Reflections on Geographic Variations in U.S. Health Care, updated May 12, 2010. Available at www.dartmouthatlas.org. Accessed November 12, 2012.