Emerging Technology Reports

The Emerging Technology Report provides regular updates about emerging and potentially disruptive technologies that are likely to have a significant impact in healthcare. This “early warning” report monitors new health technologies from 6 months to 2 years within a regulatory submission and tracks those as they go through clinical trial development and the regulatory approval process.

For each emerging technology, our analysts describe the technology and intended use, its prospects relative to the regulatory process and FDA information, competing technologies, relevant clinical guidelines, high-level look at the evidence such as published abstracts in the medical literature and available meeting abstracts, and available clinical trials.

Valoctocogene Roxaparvovec (Roctavian; BioMarin Pharmaceutical) for Hemophilia A

Valoctocogene roxaparvovec (Roctavian; Biomarin Pharmaceutical Inc.) is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 international unit/deciliter [IU/dL]) without pre-existing antibodies to adeno-associated virus serotype 5 detected by a U.S. ...