Situation
An exciting biotechnology company that develops gene therapeutic technology based on RNA wanted to develop a baseline, integrated development plan for a lead iron overload (IO) candidate. The client was keen to take a data led approach to understand and document the following:
- Competitor activity, dosing and development status, including relevant input for strategic business development activities and interactions with the regulatory agency
- Lead target indication(s)
- Leading countries, KOLs and trialists
- Protocol design, including baseline patient characteristics, standard of care, outcome measures and inclusion/ exclusion criteria
The Development program should capitalize on the coexistence of 10 patients and maximize ROI
Solution
Within three months Phesi successfully delivered to the Board a baseline, fully integrated development plan document covering:
- Product positioning: TPP and commercial potential, the potential candidate value increasing 5-fold as a result of the analysis
- Clinical development timelines and implementation planning
- Regulatory strategy
- NCD strategy
- Program timelines, resourcing and budget
- Leading authorities from a KOL and trialist stand point
