A biotech needed FDA approval for their macular disease drug. But combining data from multiple trials was harder than expected… one wrong decision, and years of work could’ve gone to waste.
1) The Challenge
A biotech company was preparing a New Drug Application (NDA) for a treatment targeting Geographic Atrophy.
The results across individual trials looked good. However, the FDA requires the full picture for approval: safety and efficacy across the entire program.
The client needed a robust ISS (Integrated Summary of Safety) and ISE (Integrated Summary of Effectiveness), using data from multiple studies with different designs, standards, and patient populations.
They couldn’t afford delays – the summaries had to be accurate and delivered on time.
2) The Solution
IDDI led the Integrated Summary of Safety and Effectiveness.
Our biostatisticians:
- Aligned data from multiple ophthalmology trials, each with different designs and patient groups.
- Focused the analysis on the key 2mg dose for Geographic Atrophy.
- Designed and executed a pooling strategy that gave regulators the necessary clarity.
- The work was conducted in parallel with the clients’ internal teams, to keep timelines on track.
3) The results
- Despite the constraints, the proactive steps taken by IDDI’s team ensured that the ISS/ISE submissions provided robust evidence to support the FDA’s evaluation of the drug.
- The integrated analysis helped demonstrate the drug’s safety and efficacy across trials.
- FDA approved the drug for GA on August 5, 2023.
- EMA accepted the MAA two weeks later.