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More Than 100-Fold Cost Differences Found for Pivotal Trials


The costs of pivotal trials ranged from $2.1 million to $346.8 million, with a central cluster of trials with estimated costs of $12.2 million to $33.1 million.

Pivotal trials leading to FDA approval of novel therapies have a median cost of $19 million, with factors such as disease target and type of control cohort influencing costs, according to researchers.

The study of 138 pivotal trials assessing 59 new therapies that received FDA approval between 2015 and 2016 found more than 100-fold differences in the costs of clinical trials, along with the finding that a central cluster of trials has estimated costs of $12.2 million to $33.1 million.

“Pivotal clinical trial costs increased if more patients were needed to document treatment benefit, if active drug comparators were used, or to measure clinical end points rather than a change in surrogate outcome,” wrote the researchers.

To read about efforts to diversify clinical trials, click here.

They identified the novel therapeutic agents using the FDA Center for Drug Evaluation and Research annual summary reports on “Novel Drugs.” The characteristics of the trials were obtained through public sources, including ClinicalTrials.gov and the FDA Summary Review.

Of the 59 approved therapies, the most common disease target was cancer (30.5%), followed by endocrine and metabolic diseases (15.2%) and central nervous system disorders (13.6%). Just 16 agents were approved based on a standard review and 2 or more clinical trials, whereas 43 were granted 1 or more forms of expedited reviews or legal exceptions to the standard of 2 well-controlled trials.

The costs of trials ranged from $2.1 million for a trial that enrolled 4 patients to test uridine triacetate for a rare hereditary metabolic disorder to $346.8 million for a noninferiority trial that assessed the impact of a new combination cardiovascular drug for chronic heart failure on hospitalization and cardiovascular mortality. The clinical trials cost a median of $41,117 per patient and $3562 per patient visit.

The number of patients enrolled in the trial, as well as the type of control involved, also played a role in cost. Although median enrollment was 488 patients, trials had wide arrays of enrollment numbers, ranging from fewer than 15 patients for 3 trials for orphan drugs to more than 1000 patients in 21 other trials. Estimated costs increased from a mean of $5.9 million for trials with 100 or fewer patients to a mean of $77.2 million for the trials with more than 1000 patients.

Those without a control cohort had an estimated mean cost of $13.5 million, compared with placebo-controlled trials that had more than double the cost ($28.8 million) and active drug—controlled trials that had a mean cost of nearly $50 million ($48.9 million).

Other factors influencing costs included duration of treatment and type of end point. “An end point of clinical outcome more than doubled the mean trial cost compared with trials using either surrogate outcomes or clinical scales,” noted the researchers.


Moore T, Zhang H, Anderson G, Alexander GC. Estimated costs of pivotal trials for novel therapeutic agents approved the US Food and Drug Administration, 2015-2016 [published online September 24, 2018]. JAMA Intern Med. doi: 10.1001/jamainternmed.2018.3931.

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