RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:RE:Clinical Trial Size in Orphan and Rare Diseases It has been shown that orphan/ rare disease trials have smaller sample sizes than non-rare disease trials. Indeed some orphan drugs were approved by the European Medicines Agency based on studies with as few as 12 patients.
https://ojrd.biomedcentral.com/articles/10.1186/s13023-017-0597-1
Drugs for common orphan indications were more often biomarker directed.
The rare disease market is set for major growth in the decade to come, and pharma companies are securing their footholds now.
https://www.pharmavoice.com/news/rare-disease-market-orphan-drug-pharma/642983/