For US biotech companies developing rare disease therapies, decisions about clinical evidence, patient populations and regulatory advice can have consequences in Europe long before a marketing application is submitted.
Drug developers need policies and technological infrastructure to execute on their goal of being patient-focused. At a minimum, this means a compliant way to engage patients and truly hear their voices; at its most effective, patient engagement also meets patients where they are – whenever and wherever that may be.
Veradigm examines key clinical trends, comorbidity profiles, and treatment trends across adolescence, reproductive years, and peri-/post-menopause. Download it today!
PREA loophole had allowed companies to receive orphan drug designations for diseases common in adults.
The story of Korlym highlights how America’s drug development system can turn an old drug into a new one that treats relatively few — but often very desperate — patients.
The AveXis treatment for spinal muscular atrophy type 1 generated positive clinical data from a study in November last year — the 15 infants treated at 20 months with AVXS-10 suffered no adverse events.
Promising Phase 3 study results for its RNAi treatment, called Patisiran, for a rare, genetic nerve disorder have increased the chances that Alnylam could be the first to commercialize a RNAi drug.
In an interview, Kyan Health Co-Founder and Chief Commercial Officer Konstantin Struck discussed how Kyan gives mid-market and enterprise employers access to premium workforce mental healthcare, at a price point that is affordable.
Exondys 51 doesn’t cure Duchenne muscular dystrophy and will only help a minority of patients.
The orphan drug industry is somewhat static in growth, but significantly outpaces the mass market drug industry, according to a new report from EvaluatePharma.
Right on the heels of a fast track designation from the FDA, San Diego startup Mapp Biopharmaceutical gets orphan drug status from European regulators for its Ebola drug, ZMapp.
Drugs that treat rare disease are on the rise, which is enormously important to patient communities. But when treating such small population, companies are running out of patients for their clinical trials.
Enterprise EHR boosts scalability, interoperability, and governance for large healthcare systems.
No mutation left behind – that’s the concept of Perlstein Lab, a Bay Area biotech and public benefit corporation that explores precision orphan drug delivery. Its thesis is that though 1 in 12 people have some variety of orphan disease, the vast majority – some 95 percent – have no treatment. Because each orphan condition is caused by a “spectrum […]