Challenge
The sponsor of a rare-disease candidate needed a defensible regulatory route and a clear basis for deciding whether to continue the program.
Approach
NGT built a 505(b)(2) roadmap that set out the pathway, the data the pathway would rely on, and the decision points along it.
Outcome
The roadmap enabled data-driven go/no-go decision-making, giving the sponsor a documented basis for its program decision.
Disciplines involved