Blood Cancer Drugs May Offer 'Reasonable Value for Money'

Zosia Chustecka

February 10, 2015

Despite the high cost of many of the new drugs used in the treatment of hematologic malignancies, which has led to an outcry from experts in the field, a new analysis of published data suggests that many of the innovative treatments "may provide reasonable value for money."

This is the conclusion from a cost-effectiveness review published online February 5 in Blood.

"The main takeaway is that we need to think about value and not just price," commented senior author Peter J. Neumann, ScD, director at the Center for the Evaluation of Value and Risk in Health, Institute for Clinical Research and Health Policy Studies, Tufts Medical Center, Boston.

"When you look at cost relative to value, it makes these drugs look more attractive," added first author Cayla Saret, BA, from the same department. However, she added that "any particular ratio should be used with caution. For example, some may be outdated now."

The study calculated cost-effectiveness from published clinical trials for nine drugs (alemtuzumab, bendamustine, bortezomib, dasatinib, imatinib, interferon, lenalidomide, rituximab alone or in combination, and thalidomide) in the treatment of four malignancies: chronic myeloid leukemia (CML), chronic lymphocytic leukemia, non-Hodgkin's lymphoma (NHL), and multiple myeloma.

The authors conducted a systematic review of peer-reviewed cost-effectiveness analyses published between 1996 and 2012, specifically seeking out published studies in the medical and economic literature examining the cost utility of innovative treatment agents for blood cancers.

However, this 2012 cut-off date means that many of the newer drugs launched for the treatment of CML, for example, were not included.

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, the authors explain. This unit of measurement incorporates a treatment's impact on patients' length of life and quality of life into its benefit. A lower cost/QALY ratio means a more favorable result, as it represents a more efficient way to achieve health gains.

Most of the cost-effectiveness ratios calculated for the blood cancer drugs fell below $50,000/QALY (73%), and only a minority were over $100,000/QALY (14%), they report.

There was little difference between the results obtained for studies that were sponsored by industry and those that were not, and the difference was not statistically significant. The industry-funded studies (n = 22) reported a lower median ratio ($26,000/QALY) than others (n = 7) ($33,000/QALY).

Most of the cost-effectiveness ratios fell below the $50,000/QALY (73%) or $100,000/QALY (86%) threshold, the researcher emphasize. "These thresholds are commonly used in the United States as benchmarks for cost-effectiveness," they comment.

They are also used as benchmarks elsewhere — for instance, in the United kingdom, the National Institute for Clinical Excellence (NICE) uses a value of around $55,000/QALY as a cut-off for allowing drugs to be available on the National Health Service (NHS).

The review found only four of these studies found a cost-effectiveness ratio that was higher than $100,000/QALY, including two studies pertaining to treatment of multiple myeloma with bortezomib, one study pertaining to treatment of CML with interferon, and one study pertaining to treatment of CML with imatinib.

"Despite the high cost of the new drugs, the cost-effectiveness ratio distributions are comparable to those for cancers overall and other healthcare fields," say the authors.

They cite a study from 2010 from the Tufts Medical Center (J Natl Cancer Inst. 2010;102:82-88), which looked at cost-effectiveness ratios for several cancer types, and found (in 2008 US$) ratios of $27,000 for breast cancer, $22,000 for colorectal cancer, $34,500 for prostate cancer, $32,000 for lung cancer, and $48,000 for hematologic cancers. However the authors of that study, which include Dr Neumann, noted that these calculations were based on data published in 2007, and so did not include expensive drugs such as bevacizumab (Avastin, Genentech/Roche), cetuximab (Erbitux, Bristol-Myers Squibb), sunitinib (Sutent, Pfizer), and sorafenib (Nexavar, Bayer/Onyx).

In addition, the Tufts Medical Center has a league table of cost-effectiveness ratios for selected measures and treatments.

Dr Neumann commented to Medscape Medical News that the calculation for most of the drugs used in hematologic malignancies of $50,000/QALY appears to offer better value for money than the calculations that have been made for screening for lung cancer with annual CT scans in heavy smokers, which has been calculated at around $80,000/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," Dr Neumann said in a statement.

"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 healthcare should consider the value of breakthrough drugs and not just costs," he added.

Review Did Not Include Recent Expensive Products

Across the hematologic malignancies that were studied, the median reported ratio was highest for CML ($55,000/QALY) and lowest for NHL ($21,500/QALY).

"Treatments for NHL may appear more cost-effective than treatment for CML, because innovative agents such as rituximab (Rituxan, Roche) for NHL were introduced in earlier years than tyrosine kinase inhibitors (TKIs) for CML, which may have affected the manufacturers' pricing strategies," the researchers comment.

"In addition, unlike rituximab, the TKI market has experienced a flurry of agents approved for the same target," they add. "Churning in this market based on loss of efficacy, concern about gene mutation, differences in dosing administration (e.g., daily vs twice daily), and side-effect profiles may result in increasing costs for CML treatment as compared with NHL."

The TKIs for CML have, in particular, come in for harsh criticism from experts in the field for being "priced at astronomical levels." These comments appeared in a special article in Blood in 2013, which was signed by more than 100 international experts in hematologic malignancies, headed by Hagop Kantarjian, MD, from the Department of Leukemia at the University of Texas M.D. Andersen Cancer Center in Houston.

Three of these drugs were approved by US Food and Drug Administration in 2012: ponatinib (Iclusig, Ariad), priced at $138,000 annually; omacetaxine (Synribo, Teva), at $28,000 for induction and $14,000 for a maintenance course; and bosutinib (Bosulif, Pfizer), at around $118,000 per year.

None of these were included in the above cost-effectiveness review, as the review looked at trials that had been published up to 2012. But the review did look at the original drug in this class, imatinib (Gleevec, Novartis), which was launched in 2001 at a price of $30,000 per year. However, since then, it has had a huge price hike, so that in 2012, it cost $92,000 per year, a price increase that outraged the experts.

So, the review's conclusions about offering reasonable value for money cannot be applied to these newer TKIs in CML or the newly high priced imatinib in CML, as these products were not included in the review.

In the United Kingdom, many of the newer drugs for hematologic malignancies have been rejected by NICE as not being cost-effective. This means they are not available on the NHS, and would normally only be available to patients who can afford to pay for them privately.

However, the somewhat controversial government-funded Cancer Drugs Fund is, at present, providing funds to cover the use of some of these drugs. The latest list of drugs covered by the Cancer Drugs Fund, updated in January, includes the following: bendamustine (Treanda, Cephalon), bosutinib, brentuximab (Adcetris, Seattle Genetics), clofarabine (Clolar/Evoltra, Genzyme), dasatinib (Sprycel, Bristol-Myers Squibb), ibrutinib (Imbruvica, Pharmacyclics/Johnson & Johnson), idelalisib (Zydelig, Gilead Sciences), ofatumumab (Arzerra, Genmab), pomalidomide (Pomalyst/Imnovid, Celgene), ponatinib, and ruxolitinib (Jakafi/Jakavi, Incyte/Novartis). All of these drugs are being covered by the special fund because they have been rejected by NICE as not being cost-effective.

Two multiple myeloma drugs were removed from this latest list — lenalidomide (Revlimid, Celgene) was removed because it is now NICE approved (i.e., deemed to be cost-effective), but bortezomib (Velcade, Millennium) was removed from the Cancer Drugs Fund list as it will no longer be funded (after government support for fund was reduced earlier this year). This means the only way now that patients can get bortezomib is to pay for it themselves.

The Center for the Evaluation of Value and Risk in Health receives funding from government, private foundation, and industry sources. The authors have disclosed no relevant financial relationships.

Blood. Published online February 5, 2015. Abstract

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