> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Factors in Pivotal Trial Costs
- URL: https://www.fdaweb.com/factors-in-pivotal-trial-costs/
- Published: 2018-09-25T12:00:00.000Z
- Updated: 2026-09-15T00:34:06.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5142430

Researchers from the Institute for Safe Medication Practices and several academic medical centers say that the cost of pivotal clinical trials required by FDA to provide substantial evidence of efficacy for new therapeutic agents increases if more patients are needed to document treatment benefit, if active drug comparators are used, or to measure clinical endpoints rather than a change in a surrogate outcome. Writing online in *JAMA Internal Medicine*, the researchers [say](https://jamanetwork.com/journals/jamainternalmedicine/fullarticle/2702287?ref=fdaweb.com) theirs is the first systematic evaluation of the estimated costs of pivotal clinical trials.

The researchers looked at 59 new therapeutic agents approved by FDA from 2015 to 2016 and found a cluster of trials with cost estimates ranging from $12 million to $33 million, and a median estimated direct cost of $19 million. “At the extremes of the distribution were 100-fold cost differences, and patient enrollment varied from fewer than 15 patients to more than 8,000 patients,” the report says.

Costs were lowest, according to the study, when then-new therapeutic agents were approved by FDA based on uncontrolled trials without any of the major protections against bias. Pivotal trials were expensive when the benefits of a new agent were similar to those of agents already available with well-established clinical benefits. In most instances, the current costs of pivotal clinical trials appeared to comprise a modest portion of published estimates for overall costs of drug development.

“The intent of this study was to put a realistic price tag on one essential component of the drug development process that had not previously been evaluated,” the report says, “not to provide an alternate method to capture the full costs of development or of obtaining approval by FDA.”

The researchers conclude that their study “provides a different perspective to the widely held assumption that elaborate and expensive clinical trials are the main reason for the high costs of developing a new drug. These data suggest that high-cost trials occur, but usually when drug effects are small or a known drug already provides clinical benefit. On the other hand, pivotal trials for novel drugs with substantial clinical benefits can be conducted at lower costs.”