Clinical Trial Size in Orphan and Rare Diseases Some orphan drugs were approved by the European Medicines Agency based on studies with as few as 12 patients.
Studies supporting (post) marketing authorisation (post accelerated approval / Phase 3 confirmatory studies) included several hundred patients.
As stated in the “Guideline on clinical trials in small populations” by the European Medicines Agency/Committee for Medicinal Products for Human Use (EMA/CHMP), most orphan indications submitted for regulatory approval are based on randomised controlled trials (RCT).
ONCY pelareorep is being trialed in Orphan and Rare Diseases/Cancers, such as pancreatic, CRC MSI high, and SCCA, for example, using randomised controlled trials (RCTs).
https://ojrd.biomedcentral.com/articles/10.1186/s13023-017-0597-1
https://www.irdirc.org/wp-content/uploads/2017/12/SPCT_Report.pdf
https://www.ncbi.nlm.nih.gov/books/NBK56176/
https://listenandlearn.alexion.com/Regulatory_policy_Reform