Advanced orphan drug trials (four companies), company-reported per-patient trial cost

$137,000 to over $5MM

USD per patient (upper end is "over $5MM"), US

“our analysis found per patient costs to range from $137,000 to over $5MM in advanced orphan drug trials”

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

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

What to know

Built by matching company-reported programme costs to trials on clinicaltrials.gov; the highest costs belong to enzyme replacement and gene therapies tested in 12 to 33 patients.