The move to deny coverage for an approved orphan drug is ‘highly unusual,' NORD VP says. 'We certainly hope this isn’t going to set a precedent.'
National health insurer Anthem Inc. has determined it won’t cover Sarepta Therapeutics Inc.’s drug for Duchenne muscular dystrophy Exondys 51 (eteplirsen) because the drug lacks data showing it is clinically effective.
“The use of eteplirsen is considered investigational and not medically necessary for all indications, including but not limited to the treatment of Duchenne muscular dystrophy,” Anthem
European companies, in particular small and midsize enterprises, spin-offs and start-ups have a hard time expanding within the single market “because of a complex regulatory framework that is perceived as slow and burdensome,” according to the European Commission.
Brensocatib, Insmed's investigational treatment for non-cystic fibrosis bronchiectasis, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.
Industry will fight hard to prevent other states or the federal government from adopting measures similar to Arkansas' law preventing PBMs from owning pharmacies.
