Md Mamun-Ur-Rashid
Quality of government secondary school services in regional Bangladesh
Heliyon
one challenges rare disease clinical trials probably availability small patient population great concern conduct clinical trials small number subjects available obtaining substantial evidence regarding safety effectiveness approval rare disease drug product investigation fda however intention create statutory standard approval orphan drugs different standard approval drugs common conditions thus suggested innovative trial designs complete nof1 trial design adaptive design used accurate reliable assessment rare disease drug products investigation article basic considerations innovative trial designs statistical methods data analysis discussed addition innovative thinking evaluation rare disease drug products proposed