NorthWest Biotherapeutics Inc (NWBO)

[Margin Buu]

Use of External Controls in DCVax-L’s Phase III Trial: The DCVax-L Phase III trial, published in JAMA Oncology, used matched contemporaneous external controls to evaluate efficacy, as approved in the Statistical Analysis Plan (SAP) by the MHRA. The trial demonstrated significant improvements in median overall survival (mOS) for both newly diagnosed and recurrent glioblastoma patients, with external controls independently selected by experts. The MHRA’s acceptance of the SAP suggests they already agreed to the methodology, including the use of external comparison groups. A request for patient-level data from these groups would likely have been addressed during the SAP approval or early in the MAA review, not as a late-stage demand.