PAI Preparation and Readiness PHARMACEUTICAL INDUSTRY Client An emerging gene therapy company. Problem As a start-up biotechnology company with a product that was granted Breakthrough Therapy Designation, the typical clinical and approval timelines were dramatically shortened. This was the company’s first potential marketed product and required the company to grow from a purely clinical focus to a mature commercial biotechnology company. Approach To ensure PAI readiness in Operations, a multi-pronged approach was taken: Gaps were identified and remediated, corrective actions were initiated and preventive actions were implemented For the product, teams were implemented to define and support process capability, develop process characterization Design of Experiments, set specifications, and write SOP’s, protocols and reports For the Quality Management System, through a team-based approach, improvements were made in non-conformance management, supplier qualification / management, document management, training and batch disposition For a successful outcome, it was instrumental to not only identify problems but to identify and implement corrective actions to resolve the underlying problems. The company: Successfully passed FDA and EMA inspections and obtained US approval (EU approval is in progress) Completed all goals and objectives prior to deadline Conducted training courses to ensure team preparation Successfully implemented process for lifecycle approach to process validation for orphan drug Improved the design and management of their Quality Systems Results PT-38