Pop quiz: If a new drug costs $23.94 per milligram and is given in doses of 3 milligram per one kilogram of body weight for one hour every two weeks, how much would it cost for a 75 kilogram patient to take it for one year?
Even without doing the math, one can guesstimate that the answer is probably a whole lot.
Yet, these types of equations have not been the norm in studies of new therapies. Given the tremendous impact on the economy, comparative-effectiveness researchers are more frequently urging the frank evaluation of both costs and value.
“The reality of exorbitant drug pricing overshadows even the most exceptional stories of drug efficacy,” wrote Dr. Peter Bach, in a commentary posted Wednesday in the New England Journal of Medicine.
Bach, who is director of the Memorial Sloan Kettering Cancer Center's Center for Health Policy and Outcomes, said, “Hand clapping for science is now inextricably linked to hand-wringing over affordability.”
His comments accompany a new NEJM study led by his Sloan Kettering colleague, medial oncologist Dr. Robert Motzer, which compared the survival, response rate and safety profiles of two treatments for advanced kidney cancer.
The condition usually has no symptoms and there are no recommended early detection screening tests. An estimated 61,560 new cases of kidney cancer were expected this year, according to 2015 estimates from the American Cancer Society.
Approximately 30% of patients have advanced disease at the time of diagnosis, according to other estimates. All of the patients enrolled in the Phase III randomized NEJM study had no success with previous therapies.
The study compared outcomes of nivolumab, a newer type of therapy called a checkpoint inhibitor, to everolimus, a rapamycin inhibitor, in 803 patients with advanced disease who had no success with previous treatments. From October 2012 through March 2014, patients were randomly assigned to treatment groups at 146 sites in 24 countries in Asia, Australia and Europe and North and South America.
Patients given nivolumab intravenously in doses of 3 milligrams per kilogram of body weight for one hour every two weeks, lived more than five months longer and had fewer severe adverse events than patients given a 10 milligram oral tablet of everolimus once per day. The research was funded by Bristol-Myers Squibb Co., nivolumab's manufacturer.
The authors note that there has been “considerable progress” in the treatment of advanced kidney disease over the past five years; before then responses to medication were infrequent.
However, the price of promise hurts patients and limits their access, Bach wrote. His team estimated that the monthly cost for one person weighing 75 kilograms, or about 165 pounds, taking nivolumab would be $11,672. The median duration of the treatment in the study was 5.5 months (compared to 3.7 months for everolimus).
The Motzer study is one of two posted in NEJM Wednesday that looks at treatments for advanced kidney cancer.
Dr. William Chin, chief medical officer and executive vice president at Pharmaceutical Research and Manufactures of America, also authored a commentary on the findings. Though he agreed that a delicate balance is needed to make innovation accessible, he points to the effective treatments for HIV-AIDS as proof that financial harm can be avoided.
Observers once warned the disease would bankrupt the system, but instead, new therapies were developed that helped more patients, he said. “Our system recognizes the considerable challenges and expense of the research and development process and the need to reward innovation, and it balances these needs against access.”
