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.
PREA loophole had allowed companies to receive orphan drug designations for diseases common in adults.
Closing cancer health equity gaps require medical breakthroughs made possible by new funding approaches.
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.
Exondys 51 doesn’t cure Duchenne muscular dystrophy and will only help a minority of patients.
The progress in artificial intelligence (AI) is reshaping patient care, across various dimensions, from facilitating faster discharge to curating treatment plans and suggesting lifestyle changes.
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.
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 […]
The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients.