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A Surprising Conclusion: High-Cost Blood Cancer Treatments May Be Cost-Effective

December 30, 2021

Although the past 15 years have seen great progress in the treatment of hematologic malignancies, these innovative therapies have left payers and patients with a serious case of sticker shock. According to a retrospective analysis of 16 years of cost-effectiveness data published in Blood, many of these treatments are actually cost-effective – providing reasonable value for the money spent.

"Our review suggests many new treatments for hematologic malignancies may confer reasonable value for money," noted lead author Cayla Saret, BA, of the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center in Boston, and colleagues. "Despite the high costs of new drugs, the cost-effectiveness ratio distributions are comparable to those for cancers overall and other health-care fields."

Using the Tufts Cost-Effectiveness Analysis Registry, Ms. Saret and colleagues conducted this first-of-its-kind review of cost-effectiveness studies related to hematologic malignancies.

A total of 29 studies published worldwide from 1996–2012 were included in the study, focusing on four types of cancer: chronic myeloid leukemia (CML), chronic lymphocytic leukemia (CLL), non-Hodgkin lymphoma (NHL), and multiple myeloma (MM). Nine treatment types for these cancers were included in the analysis:

  • Alpha interferon
  • Alemtuzumab
  • Bendamustine
  • Bortezomib
  • Dasatinib
  • Imatinib
  • Lenalidomide
  • Rituximab alone or in combination
  • Thalidomide

Hematopoietic stem cell transplant, symptom management, and supportive care were excluded.

The researchers conducted a cost-utility analysis, a type of cost-effectiveness analysis that measures health benefits in quality-adjusted life-years (QALYs). QALY is a generic measure used to assess the value of a medical intervention, based on the number of years of life that would be added by the intervention – taking into account the treatment's impact on both the quality and quantity of a patient's life. A lower cost/QALY ratio means a more favorable result as it represents a more efficient way to achieve health gains.

The studies reviewed included nine from the United States, six from the United Kingdom, and others from Scandinavia and Europe. The pharmaceutical industry sponsored 22 of the 29 studies. Respective currencies were converted to U.S. dollars for the year in which they were published, and cost-effectiveness ratios were inflated to 2012 dollars.

Of the 44 cost-effectiveness ratios published in the 29 studies (some studies reported multiple ratios), most addressed NHL or CML interventions (41% and 30%, respectively); of these, rituximab (43%), alpha interferon (18%), and imatinib (16%) were the most commonly studied therapies.

Interestingly, most ratios fell below, or were more favorable than, the $50,000/QALY (73%) or $100,000/QALY (86%) thresholds for cost-effectiveness. Interventions for CML were the most expensive ($55,000/QALY), while interventions for NHL were the lowest ($21,500/QALY; FIGURE).

"According to the available literature, many treatments for blood cancers may offer good value according to commonly used cost-effectiveness benchmarks," Ms. Saret explained to ASH Clinical News. "Cost-effectiveness is one important input that decision-makers can use alongside other factors such as side effects, affordability, overall budget impact, and ethical and social considerations.

However, she pointed out that her group's analysis did not examine "the question of affordability at the patient level which is a related, but different, issue."

Across the four cancer types, the median ratio reported by industry-funded studies ($26,000/QALY) was lower (more favorable) than the median reported by non-industry-funded studies ($33,000/QALY), but these differences were not statistically significant.
Median ratios fluctuated across the time examined in their research:

  • $35,000/QALY (1996-2002)
  • $52,000/QALY (2003-2006)
  • $22,000/QALY (2007-2012)

The number of years a treatment has been on the market, differences in therapy dose administration, and manufacturers' pricing strategies were cited as possible actors on median ratio variability across time.

"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," senior study author Peter S. Neumann, ScD, Director of the Center for Evaluation of Value and Risk in Health at Tufts, told ASH Clinical News. Although he noted that the current study had a small sample size and cost-effectiveness ratios may have changed over time (as associated costs or benefits have also changed), "the study underscores that debates in health care should consider the value of breakthrough drugs, and not just costs."

Cost-effectiveness studies may help payers create more knowledgeable coverage policies, Ms. Saret added. "These data may be used as a component of the decision-making process when health insurance payers determine coverage," she noted. For instance, she explained, an expensive treatment may be viewed more favorably in a coverage determination if research demonstrates the treatment's favorable cost-effectiveness ratio.

"Rather than looking at cost in isolation, health insurance payers may use such data to consider both the costs and the benefits of treatments," Ms. Saret concluded.

However, the authors noted, the study's implications are limited by a small sample size of analyses in the Tufts Cost-Effective Analysis Registry, and the fact that it included both industry- and non–industry-funded studies. Industry-funded studies – which represented the majority of the studies analyzed – reported a lower, more favorable cost/QALY ratio than non–industry-funded studies.

"The ratios depend on cost and benefit assumptions that may have changed since publication," they noted. "For example, imatinib's price [increased] from about $30,000 per year of treatment in 2001 to roughly three times that amount as of 2012. A 2001 cost-utility analysis would evaluate imatinib as more cost-effective than it is at its higher 2012 price."

Saret C, Winn A, Shah G, et al. Value of innovation in hematologic malignancies: a systematic review of published cost-effectiveness analyses. Blood. 2015 February 5. [Epub ahead of print]


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