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Comparative Effectiveness Research Helps Define Value in Cancer Care

September 2014, Vol 5, No 7

Los Angeles, CA—Comparative effectiveness research (CER) can be beneficial to determining value in cancer therapies, but challenges remain in applying this approach in oncology.

Daniel C. Malone, RPh, PhD, Professor of Pharmacy, University of Arizona College of Pharmacy, Tucson, shared his insights and experience related to CER in value-based oncology therapy at the Fourth Annual Conference of the Association for Value-Based Cancer Care.

An evidence-based medicine triad—consisting of individual clinical knowledge, the values and expectations of patients, and the best available clinical evidence—is important to improving value in medicine, said Dr Malone (Figure).

What is Evidence Based Medicine?

He emphasized the need to consider patient values as well as individual provider preferences in addition to clinical evidence.

CER provides best scientific evidence in making decisions about patient care. “It is the synthesis of evidence that compares benefits and harms,” said Dr Malone. He cautioned that the positive and negative aspects of therapies must be considered, to help patients and their providers make better decisions and, ultimately, to improve health.

Incorporating CER in Oncology

“One of the problems with the federal initiatives for CER is that we can’t talk about costs. But when it comes to CER and using technologies efficiently, we have to bring cost into it,” Dr Malone insisted.

Using randomized controlled trials to test cancer drugs presents a problem for the CER approach, because it would be unethical to randomize patients with cancer to a placebo or to a knowingly ineffective comparator to assess the relative value of a drug.

Pragmatic trials, however, differ from efficacy studies, which measure an intervention’s performance under ideal, controlled circumstances.

“Pragmatic trials are really what we call effectiveness trials,” Dr Malone explained. “Does this drug work when we get it out into usual practice?” Pragmatic trials allow cancer drugs to be evaluated in a comparative manner.

The strict inclusion and exclusion criteria used in efficacy studies also prevent the treatment from being used in a wide range of patient populations. Sometimes the patient population that can gain the most benefit from a treatment is not able to receive it until much later than other populations. “We tend to see these products that are used more in advanced stages of cancer eventually moving into the earlier stages of cancer,” Dr Malone said.

Observational studies and systematic reviews are also used in CER to evaluate treatment options.

Defining Value in Cancer Care

Dr Malone asked, “How do we define value in cancer care? Obviously, it’s unmet medical needs. Another component to the value of therapy is its advantages over existing options.” These include better efficacy and fewer side effects.

He acknowledged that evaluating unmet medical needs from a comparative effectiveness standpoint is challenging. “There is a whole host of intangible factors, such as patient quality of life. There are other factors that we may not be able to grasp via some quantitative approach that we need to take into account,” Dr Malone said.

He cautions against simply using a measure of central tendency, such as median survival or median progression-free survival, to represent outcomes for a constellation of patients. “Means and medians ignore outliers. They ignore distribution,” Dr Malone warned.

He advocates the use of hazard ratios and confidence intervals to better understand the variation of an oncology treatment.

“We really haven’t come to grasp what defines cost-effectiveness,” said Dr Malone. “It’s very complicated. Every drug can be cost-effective in the right patient at the right time.”

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