The Institute for Clinical and Economic Review (ICER), a nonprofit organization whose members include drug makers and insurers, has scheduled a meeting next month to review the value of multiple myeloma treatments and to determine a benchmark for payers. The meeting comes as concerns grow over the costs of these drugs, which range from $8,000 to $14,000 a month.
Over the past decade, the treatment of multiple myeloma in the United States has centered on two drugs, often given in combination with dexamethasone, ICER noted in a background statement. The first of these medications to enter use was the proteasome inhibitor bortezomib (Velcade, Takeda Millennium) in 2003, followed by the immune modulator lenalidomide (Revlimid, Celgene) in 2005. More recently, other drugs have become available specifically for the treatment of relapsed or refractory disease, including the immune modulator pomalidomide (Pomalyst, Celgene), the proteasome inhibitor ixazomib (Ninlaro, Takeda), the monoclonal antibody daratumumab (Darzalex, Janssen Biotech), the immunostimulatory antibody elotuzumab (Empliciti, Bristol Myers-Squibb), and the histone deacetylase inhibitor panobinostat (Farydak, Novartis).
Experts are pondering the comparative tradeoffs between the effectiveness and toxicity of these therapies and their various combinations, ICER observed. Cost considerations have also increased along with the list prices and potential for multiple drug combinations in varying sequences.
“Thus there remains substantial uncertainty regarding how best to interpret and apply the available evidence to guide clinical practice and insurance coverage policies,” the group asserted in its statement.
ICER also pointed out that the costs of managing multiple myeloma are substantial, given the use of several treatments over the course of the disease. “The cost of a single course of drug therapy has been estimated to range from $75,000 to $250,000 for patients” who have relapsed or did not respond to previous treatment, the group said in its statement.
ICER has criticized pricing for hepatitis C medications and, more recently, for a new pair of injectable cholesterol drugs. The group has also reviewed medications used to treat obesity, diabetes, and heart failure.
In advance of the pending meeting, Amgen has publicly questioned the extent to which the session will fairly evaluate the medications, according to a report on the STAT website. The company, which manufactures carfilzomib (Kyprolis), is concerned that the upcoming review will hinge too heavily on cost-effectiveness and will make other comparisons the company believes are unwarranted. Sales for carfilzomib totaled $512 million last year.
In a statement, Amgen argued that a “thorough and balanced assessment should rely on direct data from rigorous comparative [clinical] trials,” rather than using “opaque methods” to combine “disparate trials” to determine whether the drugs are useful.
“Results produced by independent organizations should be informed by experts, made fully transparent and available, and undergo complete and independent peer review,” the drug maker said.
In response, an ICER spokesman told STAT that “comparing the results across different drugs is difficult when there are no head-to-head studies [of multiple myeloma medications], but we feel confident that patients, clinicians, and policymakers can benefit from an independent, objective analysis of the existing data.”
Some countries gauge the cost-effectiveness of drugs by determining quality-of-life years (QALYs), which reflect both the quantity and quality of life generated by providing a drug treatment or some other health-care intervention. Specifically, QALYs measure life expectancy and the quality of the remaining years of life when using a treatment.
Amgen developed its own method for assessing a drug’s value—the Kyprolis Global Economic Model (K-GEM). With this approach, the company estimated that using carfilzomib as part of a treatment regimen is “well below” the $150,000 to $300,000 per QALY cited as “reasonable benchmarks” for treating cancer in the United States.