Price and End Points. For Cancer Drugs, There Is a Disconnect, Research Finds

Cancer drugs approved on the basis of overall survival were priced lower than those approved based on progression-free survival (PFS) or overall response rate (ORR), according to research results reported today in JAMA Internal Medicine.

The median annual cost of drugs approved based on overall survival was $185,000 compared with $203,000 for those approved based on PFS and $239,00 for those approved on ORR. Overall survival is considered a more reliable indication of a drug’s worth than other endpoints.

The researchers also found that regardless of the end point, price was not linked to the size of the benefit on that endpoint.

“This suggests that cancer drugs are priced based on what the market will bear,” wrote the researchers.

Vinay Prasad, M.D., M.P.H.

Vinay Prasad, M.D., M.PH., a professor in the Department of Epidemiology and Biostatistics at the University of California, San Francisco, and the corresponding author of this study, has been a prolific and vocal critique of what he sees as an overreliance on surrogate end points such as PFS and ORR in cancer clinical trials.

Defenders of surrogate end points, which include some researchers and the pharmaceutical industry, contend that overall survival as an endpoint presents practical difficulties and that the use of surrogate markers gets valuable therapies on the market and into the clinical practice sooner.

In an accompanying editorial, Isabel R. Ostrer, M.D., also at the University of California, San Francisco, and Cary P. Gross, M.D., a professor of medicine and public health at the Yale University School of Medicine, echoed Prasad and his colleagues’ market-will-bear comment about the pricing of cancer drugs.

“Clearly, cancer treatments in the U.S. are priced at what the market will bear,” they wrote. “Currently, the market seems to be bearing $200,000 a pop. But at what cost to society?”

They go on to say that despite spending twice as much on cancer care compared to other countries, the U.S. does not have a substantially lower cancer mortality rate.

Ostrer and Gross discuss other ways of pricing drugs, including indication-specific pricing and lower prices for drugs approved via the FDA’s accelerated approval pathway until there are positive results from confirmatory trials.

The study by Prasad and his colleagues included 224 FDA drug approvals between Jan. 1, 2015, and Dec. 31, 2020. The study encompasses 119 individual drugs (many drugs have multiple approvals for multiple indications). According to their tally, 46 of the drug approvals were based on overall survival , 71 on PFS, 90 on ORR and 17 on other endpoints.

When the researchers grouped the drugs by end point on which they were approved, they found that differences in efficacy captured less than 15% of the differences in price.