In a typical clinical trial, the results are reported in purely medical or biological terms. Did the patients in the treatment group live longer than those in the control group? Did the drug shrink the tumor or reduce symptoms? Were clinical measures such as blood pressure or cell counts affected? These are the details that the Food & Drug Association and the physician community look for when they decide to approve or prescribe new therapies. But looking at a new treatment’s effects in a medical vacuum might miss critical details about its actual usefulness out in the real world, where patients have different priorities and health care dollars are finite.
To create a more well-rounded and practical clinical trial, medical researchers need to reach outside of their discipline for expertise. Or, they can bring those experts into the hospital fold, as was recently done with the establishment of the University of Chicago Program in the Economics of Cancer. Led by Ya-Chen Tina Shih, an economist who specializes in the economic aspects of cancer care, the program has a unique premise: to study the economics of a disease that produces estimated yearly costs of $270 billion and rising in the United States. In a field where new treatments, devices, and procedures appear with startling frequency, Shih’s group aims to weigh the costs and benefits of these new technologies so that patients receive the best, most logical care rather than just the hot, new, often-pricey thing on the market.
“I see it as a place to bring researchers together to look at economic issues in cancer,” Shih said. “The issues to be addressed can be large policy issues or a cost-effectiveness analysis comparing two different treatments. What we would like to do is provide an environment where if there are oncologists who want to study those questions, they don’t have to try to learn everything themselves. They can team up with economists or people in operation research or health services research, and can work on issues together. Similarly, people with no medical training who are interested in exploring those questions can find their clinical collaborators here.”
Calculating the cost of cancer is harder than it might seem. A diligent researcher could, with much effort, simply total up all the money spent on drugs, procedures, doctor’s appointments, and devices, and calculate a price tag for cancer or cancer treatment. But one must also take into account the indirect cost of missing work, either temporarily due to illness, side effects, or surgery, or permanently due to death. Other factors are even harder to convert into dollars, such as quality of life under different treatments, while still others are politically fraught, such as cost-effective analysis to determine whether a new treatment is a significant enough improvement over the current standard of care to justify coverage by insurance companies.
Economists can estimate these figures retrospectively, after a given treatment has been out on the market for a few years or more, but at that point the horse is long out of the barn. If a new treatment is given to patients for three years, then found to be less cost-effective than the standard of care it replaced, it could unnecessarily cost society millions or billions of dollars. Shih hopes that the Program in the Economics of Cancer will help cancer researchers design clinical trials with such economic questions in mind, so that information about costs can be gathered before the widespread diffusion of a new technology that provides a very small benefit at substantial cost.
“You don’t at the conclusion of a trial say ‘let’s add a cost-effectiveness analysis to that.’ By then, it’s way too late,” Shih said. “The idea is to get more people interested in collecting this data at early timepoints, so by the time they really want to answer a question, they have the data to answer it.”
In the shadow of “death panel” fear-mongering, cost-effectiveness remains a dirty term for many in the health care world – the 2010 Affordable Care Act emphasized comparative effectiveness over its dollars-and-cents cousin. Economists are often accused of putting the bottom line before the patient when considering the costs of a new therapy, Shih said, but that knee-jerk response ignores the financial burden put upon patients when very expensive treatments hit the market with little to no economic diligence.
“For me, a frustration is when you bring up the issue of cost, people consider that an ethical discussion,” Shih said. “I don’t think discussing cost issues is unethical. It is important to understand that such discussion isn’t about not giving patients treatment, it’s about giving them the right treatments, treatments that they can afford. In fact, it would be a very serious problem if you started giving a treatment and people don’t have the money to finish it.”
At the University of Chicago, where reducing persistent health disparities for cancer and other conditions is a popular research interest, making sure new therapies are worth the steep price is a relevant pursuit.
“I think a lot of clinicians have an interest in knowing the impact of newer technologies, how it is cost-effective, or how it is being used in the community, beyond the clinical trial population,” Shih said. “I think a lot of the physicians here, because we serve under-served populations, have an interest in knowing if there is any kind of access issue, or if cost is going to be a barrier for people getting good care. Those are the types of information produced from our research that might be helpful to them.”