Case study:
Achieving FDA and EMA Alignment for a First-in-Class Gene Therapy Program
Background:
A gene therapy company was working with Boyds on the regulatory strategy for their first in-class treatment for a rare disease that included a complex, novel delivery device and sought Boyds’ advice on how best to gain global regulatory feedback and alignment on their planned pivotal trial.
Boyds approach
Boyds used their existing familiarity with the program and the potential risks to advise the client on the quality, non-clinical and clinical questions that should be posed to regulators globally.
Boyds also conducted a detailed strategic assessment of the best way to gain regulatory feedback in the EU and the US in order to maximise the chances of aligning the requirements from both agencies. Although not always the preferred option, in this instance, parallel scientific advice with the FDA and EMA was advised.
Boyds supported the entire process, including providing input on the operational requirements, authoring and/or reviewing the briefing materials and initial written responses, and managing the submissions. Boyds also worked closely with the client to prepare them for the meeting and attended the online meeting from the client’s global headquarters.
Impact and Outcomes
A highly collaborative meeting was conducted with the two agencies, providing clear and actionable feedback for the client. Although there were areas of misalignment between the agencies, the discussion from each was understood, and the client was able to find a compromise for their protocol that met the needs of both agencies. Boyds continues to work closely with the client.