Value of Innovation in Haematology

Innovative treatments for haematologic malignancies may provide reasonable value for money

Amid the growing debate about the high price of powerful new drugs in cancer care, a recent analysis suggests that breakthrough therapies for haematological malignancies may, in many cases and with some important caveats, provide reasonable value for money spent. The analysis covers four haematological malignancies: chronic myeloid leukaemia, chronic lymphocytic leukaemia, non-Hodgkin's lymphoma, and multiple myeloma; and nine treatment agents: α interferon, alemtuzumab, bendamustine, bortezomib, dasatinib, imatinib, lenalidomide, rituximab alone or in combination, and thalidomide.

Researchers present this viewpoint, based upon a comprehensive analysis of published cost-effectiveness ratios, in Blood journal, published by the American Society of Hematology. The manuscript is being published as a Blood Forum article, a feature of the journal designed to present well-documented opinions on controversial topics and provide a sounding board for issues of importance to the science and practice of haematology.

The advent of powerful, targeted therapies over the last 15 years has revolutionised care in haematology, allowing many patients to manage once-fatal diseases as chronic conditions by taking a pill each day. While these drugs have contributed to improved patient outcomes, their high cost, sometimes 100,000 USD or more per year, has been called prohibitive and harmful by many of the foremost experts in the field. Those on the other side of the drug cost debate assert that the value of these medications, translated into years and quality of life gained, may justify a drug's high price.

Systematic review of published cost-effectiveness analyses

To better understand the value these breakthrough blood therapies provide for their cost, a team of researchers at Tufts Medical Center (Tufts MC) in Boston, USA conducted a systematic review of peer-reviewed cost-effectiveness analyses published between 1996 and 2012. The team, based at Tufts Medical Center's Center for the Evaluation of Value and Risk in Health, specifically sought out published studies in the medical and economic literature examining the cost utility of innovative treatment agents for haematological malignancies. In these types of analyses, the cost utility of a drug is depicted as a ratio of a drug's total cost per patient quality-adjusted life year (QALY) gained. This unit of measurement incorporates a treatment's impact on patients' length of and quality of life into its benefit. A lower cost/QALY ratio means a more favourable result as it represents a more efficient way to achieve health gains.

Using the Tufts Cost-Effectiveness Analysis Registry, a database of cost-effectiveness articles, the team found 29 studies and grouped them by nine treatment agents and four haematological disease types. The team evaluated factors such as the quality of each study's methodology and its value to decision makers and compared the cost per QALY ratios reported. Of the published studies analysed, 22 were funded by industry.

After comparing the cost-effectiveness ratios of drugs by disease indication and treatment type, the research team observed that most ratios were lower or more favourable than thresholds commonly used in the United States as benchmarks for cost-effectiveness (50,000 USD/QALY or 100,000 USD/QALY).

The median ratios reported by industry-funded studies (26,000 USD/QALY) were more favourable than the median reported by non-industry-funded studies (33,000 USD/QALY), although the difference was not statistically significant. The median reported cost-effectiveness ratio was highest for chronic myeloid leukaemia (55,000 USD/QALY) and lowest for non-Hodgkin lymphoma (21,500 USD/QALY).

"Given the increased discussion about the high cost of these treatments, we were somewhat surprised to discover that their cost-effectiveness ratios were lower than expected," said senior study author Peter Neumann, ScD, Director of the Center for Evaluation of Value and Risk in Health at Tufts MC. "Our analysis had a small sample size and included both industry- and non-industry-funded studies. In addition, cost-effectiveness ratios may have changed over time as associated costs or benefits have changed. However, the study underscores that debates in health care should consider the value of breakthrough drugs and not just costs."

The study was funded by internal resources at the Center for the Evaluation of Value and Risk in Health; no dedicated external funding was provided. The Center receives funding from US federal, private foundation, and pharmaceutical industry sources.


Saret CJ, Winn A, Shah G, et al. Value of innovation in hematologic malignancies: a systematic review of published cost-effectiveness analyses. Blood 2015; Feb 5. pii: blood-2014-07-592832. [Epub ahead of print]