Pivotal trial of uridine triacetate for a rare hereditary metabolic disorder, 4 patients

$2.1 million

USD per patient, US

“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”

AJMC, More than 100-fold cost differences found for pivotal trials (September 2018), reporting Moore et al., JAMA Internal Medicine 2018, 2018-09

Kind of source

Peer-reviewed study

Read on

2026-11-06

Daily check

Quote found at the source on 2026-11-06

What to know

The cheapest of 138 pivotal trials, yet far above the $41,117 median per patient because only 4 patients were enrolled.

Arithmetic

$2,100,000 / 4 patients = $525,000 per patient.