Journal: Clinical and Translational Science
Article Title: Real‐world evidence to support regulatory submissions: A landscape review and assessment of use cases
doi: 10.1111/cts.13903
Figure Lengend Snippet: Real‐world evidence (RWE) and other observational approaches are used to support single‐arm trials.
Article Snippet: Product: Defibrotide sodium (Defitelio), Jazz Pharmaceuticals, 2016 Therapeutic area: Non‐oncology Regulatory use type and purpose: Original marketing application approval Regulatory Agency: FDA , Efficacy/effectiveness data collection , External RWD controls (direct matching) , Retrospective RWD cohort , Site‐based chart data, Registries , Hematopoietic stem cell transplantation (HSCT) 100‐day survival rate.
Techniques: Clinical Proteomics, Biomarker Discovery, Control, Recombinant, Marker, Transplantation Assay, Emulsion, Virus