A significant number of gene therapies are expected to come to market imminently, potentially revolutionizing health care systems. Developers will face significant clinical, regulatory and logistical challenges in bringing these therapies to market. Manufacturers and payers must work through the associated administrative, logistical and legal challenges in partnership.

Takeda will soon seek EU approval of its dengue vaccine candidate. The marketing application will be fast-tracked, as will the applications for Regeneron's evinacumab and bluebird bio's eli-cel Lenti-D gene therapy.

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.

The latest drug approval news and highlights from the Pink Sheet’s US FDA Performance Tracker.