Value of New Drugs for Hematologic Cancers—Improving Quality, Extending Survival

Wayne Kuznar

March 2015, Vol 6, No 2 - Economics of Cancer Care


Although often criticized as being overly expensive, innovations in drug development for hematologic malignancies meet standard benchmarks for cost-effectiveness, delivering value for their cost, suggest a team of health economics researchers led by Peter J. Neumann, ScD, Director, the Center for the Evaluation of Value and Risk in Health, Tufts Medical Center, Boston (Saret CJ, et al. Blood. 2015 Feb 5. Epub ahead of print).

In their systematic review, Dr Neumann and colleagues looked at 29 cost-effectiveness studies of new drugs for hematologic malignancies developed between 1996 and 2012, showing that the mean incremental cost-effectiveness ratio (ICER) of these targeted therapies was <$50,000 per quality-adjusted life-year (QALY) in approximately 75% of the studies and <$100,000 per QALY in almost 90%.

The <$50,000 per QALY is accepted as the benchmark used to define cost-­effectiveness. The majority of the studies therefore met this <$50,000 per QALY, regardless of the source of funding for the research (ie, industry or no industry).

“Looking at the price alone is not sufficient. That price has to be looked at in the context of what we’re getting back for it,” said coinvestigator Joshua T. Cohen, PhD, MA, Deputy Director of the Center for the Evaluation of Value and Risk in Health. “So if there is a treatment that really improves quality of life or extends life substantially…that is something that is worthy of a higher price, and that’s the point.”

The effects of innovative agents in hematology on length of life, quality of life, and cost, and therefore their value, have been the topic of much heated discussion, said Cayla J. Saret, Research Assistant at the Center for the Evaluation of Value and Risk in Health. “We’ve been doing some research in the area of interventions for blood cancer, and we weren’t aware of any other reviews of the literature that looked at the cost-effectiveness of these interventions overall,” Ms Saret told Value-Based Cancer Care.

The 29 peer-reviewed studies selected focused on 9 hematologic agents—alpha interferon (Multiferon), alemtuz­umab (Campath), bendamustine (Treanda), bortezomib (Velcade), dasatinib (Sprycel), imatinib (Gleevec), lenalidomide (Revlimid), rituximab (Rituxan) alone or in combination, and thalidomide (Thalomid). The studies reported 44 ICERs.

A total of 73% of the ICERs were <$50,000 per QALY and 86% were <$100,000 per QALY. The highest median-reported ICER was for chronic myeloid leukemia, at $55,000 per QALY, and the lowest was for non-Hodg­­kin lymphoma, at $21,500 per QALY.

The median ICER reported by industry-funded studies was $26,000 per QALY, which was lower than the $33,000 per QALY median reported by studies not funded by the industry, a nonsignificant difference.

“Our review suggests many new treatments for hematologic malignancies may confer reasonable value for money,” the investigators wrote. “Despite the high costs of new drugs, the cost-­effectiveness ratio distributions are comparable to those for cancers overall and other healthcare fields.”

“As opposed to looking at the price in isolation, or the benefits in isolation, we’re hoping that decision makers also take into account cost-effectiveness, and this is one tool for them as they make decisions,” said Ms Saret. “They may be looking at affordability. They may be look­ing at side effects. They may be looking at overall budget. This isn’t to say that this is the only thing a decision should be based on, it’s just pointing out one more factor to take into consideration.”

An appropriate threshold for cost-­effectiveness has not been clearly established, said Dr Cohen. The United Kingdom uses a slightly lower threshold than the $50,000 or $100,000 per QALY used in the United States. “There are people who argue the benchmark should be even higher [than $50,000 per QALY] when you look at what is actually spent on healthcare as a proxy for what society effectively deems as acceptable,” Dr Cohen said. He and Dr Neumann and Dr Weinstein had tackled this subject in a separate perspective last year, arguing that no single threshold is appropriate in all decision contexts, but in general, the $50,000 per QALY threshold may be too low (Neumann PJ, et al. N Engl J Med. 2014;371:796-797). n