Gene therapy (hematology)
The Project
Small pharmaceutical company with EMA approved gene therapy in need for support with HTA assessment process in the Nordics.
The Challenge
Positive HTA assessment required for technology adoption and patient access Support requested for:
- Adaptation of global material
(e.g., health economic model) to conform with local requirements (Sweden, Norway, Finland, Denmark) - Consulting KOLs to reflect national perspective (e.g., clinical practice, patient population)
- Compilation of HTA dossier for submission
- Dialogue with HTA bodies during assessment process
The Solution
Project led by Senior Manager with HTA experience from relevant countries.
- Biopharma Excellence Project Leader (PL) with overall coordinating responsibility:
- Main contact point for any questions
- Responsible for timeline, budget and quality of deliverables
- Strategic support
- High level availability with fast response times
- Part of customer market access team during HTA process
- PL working close to customer during HTA process
- Continuous dialogue and flexibility regarding strategy/process
The Outcome
Project successfully and timely completed due to long experience with HTA processes.
- Wide experience from HTA processes in the Nordics
- Good knowledge of HTA system and requirements
- Informed best approach/strategy
- Gap analysis ahead of submission;
- Proactive collection of missing input
- Complete and relevant dossier
- Smooth and timely process
- Post-assessment support at subsequent market access negotiations
- Good knowledge of the therapeutic field and technology from preceding HTA work