Orphan medicinal product regulation for haemophilia criticised due to the paediatric clinical trial requirement
An opinion piece published in Haemophilia states that when it comes to treating haemophilia, medications do not need orphan drug designation to be profitable. According to the authors current global market for haemophilia products is worth in excess of US $ 7 billion. According to the author, the orphan drug designation and marketing exclusivity should be reserved only for very rare bleeding disorders such as FII, FV, FX and FXIII deficiencies, while providing new haemophilia treatment market exclusivity can “set a dangerous precedent and gravely damage patients’ rights to access”. This is mainly because the author perceives the requirement of paediatric clinical trials and sequential testing of some haemophilia drugs before approval for adults as unnecessary. The author also states that the “new longer acting products are not similar and that each protein modification should be treated as distinct and therefore be granted marketing authorisation”, without the paediatric testing requirement.
