Company-reported per-patient cost in advanced orphan trials against the average Phase 3 per-patient cost

$137,000 to over $5MM against $42,000

ratio of per-patient trial cost, advanced orphan to average Phase 3 (minimum), US

“per patient costs to range from $137,000 to over $5MM in advanced orphan drug trials, an analysis published by PhRMA and Battelle estimated average per patient phase 3 trial costs to be $42,000”

Health Advances, Orphan Drug Development: What are the Real Costs? (10 April 2019; read from an Internet Archive copy), 2019-04

Kind of source

Named press report

Read on

2026-11-06

Daily check

Source re-read on 2026-11-06 needs a browser (PDF or protected page)

What to know

The upper end is a floor ("over $5MM"); the highest costs are gene and enzyme therapies in 12 to 33 patients, so the ratio shows rarity, not an average rare disease.

Arithmetic

Low: $137,000 / $42,000 = 3.26; high: $5,000,000 / $42,000 = 119.