CONFERENCIA EUROPEA SOBRE ENFERMEDADES RARAS Y PRODUCTOS HUÉRFANOS
Pre-conference tutorials will take place on Thursday 26 May, 2016 from 14:00-17:30
- Provide an overview of orphan designation process
- Discuss incentives, specially protocol assistance and its impact on development and authorisation
- Analyse the requirements for maintenance of orphan designation at the time of marketing authorisation
- Introduce and discuss how new regulatory pathways can support drug development in rare diseases
- Orphan designation and maintenance at MA procedure
- Involvement of patients and external experts
- PRIME and early access tools for orphans
- Learn how to engage with Health Technology Assessment (HTA) organisations earlier.
- Provide an overview of individual countries and for the European Union (EU) as a whole.
- Understand the rapidly evolving regulatory process.
- Look at the process as a whole rather than as several elements in isolation.
- Market Access or Regulatory Directors / Managers with responsibility for HTA preparedness and engagement
- General managers and business leaders considering early and innovative engagement with regulators and HTA organisations
- Patient groups interested in hearing how innovative companies plan for early and innovative engagement with HTA
- This tutorial will demonstrate the scope and current activities of the RD-Connect platform supporting rare disease research.
- Participants will be able to learn how contributing data to the platform can support patient diagnosis.
- They will also receive an update on the most relevant ethical, legal and social issues associated with the movement of patient data within this environment.
- Actions to ensure compatibility of ERNs with the resources of the RD-Connect platform will be explored.
- The Target audience will be prospective coordinators of RD ERNs, or else healthcare providers joining an ERN proposal. The tutorial should also be of interest to patients wishing to learn how data travels in the RD-Connect framework and how it can be used to support research and care.