FDA EIR - Blueprint Medicines Corporation - September 05, 2019

A comprehensive FDA surveillance inspection of Blueprint Medicines Corporation was conducted from August 28 to September 5, 2019. This routine Prescription Drug User Fee (PDUFA) data integrity sponsor inspection, the first for the company, evaluated Blueprint's oversight of clinical study protocol BLU-285-1101, an investigational drug study supporting IND 125379 and NDA 212608. The inspection adhered to Compliance Program 7348.810, which governs sponsors, contract research organizations, and monitors. Although no formal FDA Form 483 (Inspectional Observations) was issued, the inspection identified several key issues discussed with management. These included the approval of critical study plans, such as the monitoring plan and eCRF Completion Guidelines, occurring after the first study participant had already commenced treatment. Additionally, the inspection highlighted challenges in confirming that Clinical Research Associates received adequate protocol and project-specific training prior to conducting monitoring visits. Most notably, Electronic Case Report Forms (eCRFs) submitted to the FDA as part of the NDA package did not contain adverse events that had been reported and subsequently deleted by clinical sites. Regarding corrective actions, company management did not provide specific responses to the observations about delayed plan approvals or CRA training documentation. However, in response to the eCRF data integrity concern, Blueprint plans to contact the FDA to discuss regenerating and resubmitting eCRFs that fully include all previously deleted adverse event information.