Spark Therapeutics, Inc.
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Also, deals between Boehringer Ingelheim/Oxford BioTherapeutics, Bausch/Eyenovia, Ligand/HitGen, Moderna/Merck & Co., Vincera/Bayer, Genentech/Kineta, Selecta/IGAN, RedHill/AstraZeneca, Urovant/Sunovion, Biogen/Scribe, BridgeBio/Eidos, Elevar/Taiba
A Phase III trial of Swedish Orphan Biovitrum’s Doptelet in solid tumor cancer patients with chemotherapy-induced thrombocytopenia has failed. The setback could affect the firm’s growth expectations.
Value-based contracting in the US is moving toward high-cost drugs for rare disease and away from lower cost chronic treatments as payers plan for the coming pipeline of cell and gene therapies. Marketed gene therapies all have some kind of risk sharing deal in place but further progress on the most innovative payment models is hampered by regulatory and operational challenges. The Centers for Medicare and Medicaid Services has taken a first step toward addressing regulatory obstacles but there is a long way to go.
With gene therapies for sickle cell disease looming and two new expensive therapies of to a slow start, access challenges could prevent a large portion of patients from receiving treatment, a report from the US National Academies of Sciences, Engineering and Medicine concludes. The report urges development of new payment models, offering five ideas.
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