Using Real-World Evidence to Derive an External Control Arm for a Rare Disease Drug Trial
Our client was developing a new anti-viral drug for a rare, life-threatening viral infection that affects Hematopoetic Stem Cell Transplantation (HSCT) patients. This rare disease had no approved therapies.
Feedback confirmed that the control arm should include European patients with specific comments on design requirements. A multi-country chart review in Europe was commissioned to derive an external control arm via a European natural history study, as well as to validate the US based external control arm.
Certara used a two-phase approach to this project.
Phase 1 included a selection of transplantation centers through feasibility assessment, and creation of an “allo HSCT registry” of adult and paediatric patients.
Phase 2 was implementation of the medical chart review to identify patients who could serve as external controls. More than 500 patients were identified with the rare viral infection post-transplant, and a detailed chart review was conducted for 241 patients who met the viral load criteria as external controls.