Optimal design and execution of studies in the area of orphan diseases and ATMPs require both cross-functional approach and in-depth scientific expertise.
The services that tranScrip provides include, but are not limited to:
- Design of clinical development programmes, from preclinical through to proof of concept and pivotal studies
- Review and interpretation of study results
- Review of GMP data for assessment of stability of products within the clinical setting
- Acting as member of independent safety boards and ethical review boards
- Preparation for Pre-IND meetings and FDA, EMA and national scientific advice meetings
- Preparation of Orphan Drug designation applications
- Biomarker strategies
This case study outlines the role which tranScrip has played in development of a product for an orphan disease/ATMP.
A small company with novel an X ATMP for Graft vs Host Disease (GvHD) (cell therapy for the treatment and prevention of GvHD).
tranScrip worked with the company to:
- Successfully file an Orphan Drug designation
- Confirm phase 2 design with EMA
- Identify CRO for clinical work
- Seek funding
Orphan Drug designation for prevention of GvHD was achieved Agreement with EMA on phase 2 design and major design elements for phase 3 was reached.