Pre-conference tutorials require an additional registration and fee to attend.
Industry representatives: 425 EUR
Academia/researchers/healthcare professionals: 140 EUR
Registration can be made online for the above categories or by completing the registration form here: http://www.rare-diseases.eu/registration/
Patient group representatives are invited to inform DIA in writing if they would like to attend a tutorial session at [email protected] There is no charge for patients and their representatives to attend these sessions but pre-registration is required.
Tutorial 1: EMA regulatory routes supporting orphan drug development
Tutorial Instructors: Kristina Larsson, Head of Orphan Medicines, European Medicines Agency; Jordi Llinares Garcia, Head of Product Development Scientific Support Department, European Medicines Agency
Developers of drugs in the field of rare diseases
Overview of tutorial
Following topics will be covered:
Tutorial 2: Health Technology Assessments (HTA) – How to Prepare & Engage Early to support Market Access
Tutorial instructors: Christian Hill – Managing Director MAP BioPharma Limited
Mark Harries – Chief Operating Officer, MAP BioPharma Limited
Overview of Tutorial:
Market Access for BioPharmaceutical innovation is often described as confusing and full of risk for the innovative companies trying to bring new products to patients. However, in many cases, this is more perception than reality when we consider the risks and opportunities involved. In this tutorial, we help you to understand how companies can engage with HTA and regulatory organisations earlier than has usually been the case, and what are the potential benefits and risks of such an approach.
Tutorial 3: How could the RD-Connect platform support Rare Disease European Reference Networks?
Tutorial Instructors: Hanns Lochmuller, Sergi Beltran/Steven Laurie, Matts Hanson/Pauline McCormack
Overview of Tutorial:
The first European Reference Networks will be operational by late 2016/early 2017. Although the primary purpose of an ERN is to facilitate access to highly specialised healthcare in domains requiring a particular concentration of expertise, research will be a cornerstone of each network – ERNs and their constituent Healthcare Provider members need to demonstrate research excellence. The RD-Connect platform has been designed to support the linking of RD patient data from databases, registries, biobanks and bioinformatics, to support research and increase the potential of precious RD data. The tutorial will demonstrate how the RD-Connect platform could support ERNs in conducting their research activities. It will also address the most relevant ethical, legal and social issues related to sharing patient data in this way.