Cancer drugs approved based on OS vs. surrogate outcomes have lowest median annual cost
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Cancer drugs that received approval based on increased OS compared with other measures had the lowest median annual cost, according to a research letter published in JAMA Internal Medicine.
The findings suggest cancer drugs are predominantly priced based upon what the market will bear, researchers concluded.
Background and methods
“The past 5 years have seen a marked increase in the number of cancer therapies on the market and greater interest in value-based pricing,” Vinay K. Prasad, MD, MPH, academic oncologist in the department of epidemiology and biostatistics at University of California, San Francisco, and colleagues wrote.
However, it is unclear whether the value proposition has shifted and whether cancer drugs started to rely on improved benefit and decreased use of surrogate endpoints to justify skyrocketing costs, according to the letter.
For this reason, Prasad and colleagues analyzed all FDA cancer drug approvals between 2015 and 2020 to determine whether an association existed between cost and efficacy. Researchers rounded all prices to the nearest $1,000 and used the Kruskal-Wallis test for categorical variables to determine differences between median annual cost of subgroups.
Findings
The analysis included 224 FDA cancer drug approvals across 119 individual agents. Median annual cost for a course of each cancer drug was $196,000 (interquartile range [IQR], $170,000 to $277,000).
Results showed a significantly lower median annual cost of the 46 cancer drugs approved based on OS ($185,000; IQR, $159,000 to $206,000) compared with the 71 PFS-based approvals ($203,000; IQR, $183,000 to $248,000; P = .02) or the 90 drugs approved based on overall response rate ($239,000; IQR, $185,000 to $341,000; P < .01).
Researchers observed a weak correlation between cancer drug cost and the most common approval endpoints (R2 = 0.14 for OS; R2 = 0.16 for PFS; and R2 = 0.02 for ORR).
Moreover, researchers observed no significant difference between the median price of drugs approved after randomized clinical trials ($191,000) and those without randomized clinical trial data ($206,000; P = .06).
Limitations of the study included the restriction of the analysis to a 6-year period and restriction of analyzed factors impacting drug price to endpoints only, researchers noted.
Implications
“Among the drugs approved on the basis of response rate, there was only a weak correlation between cost and the magnitude of the response rate gain; the same was true in the categories of drugs approved on the basis of PFS and OS gains,” Prasad and colleagues wrote. “Correcting this trend is vital for the solvency of health care and pharmaceutical development.”
“There is no shortage of innovative ideas to tie cancer-drug price to value,” according to an accompanying editorial by Isabel R. Ostrer, MD, researcher at University of California, San Francisco, and Cary P. Gross, MD, researcher at Yale School of Medicine.
“For example, indication-specific pricing would allow for different prices for the same drug, depending on how effective it is for different indications,” they wrote. “Alternatively, cancer drugs approved based on the FDA accelerated approval pathway, which relies on surrogate outcome data, could be priced lower until postmarketing confirmatory trials are complete showing clinical benefit.”
However, the challenge of value-based pricing is not the lack of creative ideas, but about the lack of political will, they added.
“The landmark Inflation Reduction Act legislation has cracked open a window to the prospect of paying for cancer treatments in a manner that is commensurate with their value,” they wrote. “We need to break the glass.”
References :
- Miljkovi MD, et al. JAMA Intern Med. 2022;doi:10.1001/jamainternmed.2022.4924.
- Ostrer IR, et al. JAMA Intern Med. 2022;doi:10.1001/jamainternmed.2022.4921.
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