The actual status of drug prices and adjustment factors for drug price calculation: an analysis of ultra-orphan drug development in Japan.

Published in: Orphanet journal of rare diseases (2022)

The status of drug prices and expected sales differed beyond a threshold of 1000 expected patients, indicating that recovering the development cost for ultra-orphan drugs is difficult. In addition, the higher premium for usefulness for ultra-orphan drugs reflects the largely unmet need of the associated diseases. Scarcity among orphan drugs is not considered for marketability, highlighting the need for a new framework to promote the development of ultra-orphan drugs.

Keyphrases

high resolution
drug induced
end stage renal disease
newly diagnosed
ejection fraction
chronic kidney disease
adverse drug
prognostic factors
peritoneal dialysis
emergency department
mass spectrometry