Pre-conference tutorials | ECRD 2016 : The European Conference on Rare Diseases & Orphan ProductsPre-conference tutorials

Pre-conference tutorials | ECRD 2016 : The European Conference on Rare Diseases & Orphan ProductsPre-conference tutorials



EUROPEAN CONFERENCE ON RARE DISEASES & ORPHAN PRODUCTS

Register for ECRD 2016 pre-conference tutorials on orphan drug development, HTA & rare disease research platforms

Pre-conference tutorials

Pre-conference tutorials will take place on Thursday 26 May, 2016 from 14:00-17:30

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 ECRD@DIAglobal.org. 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

Objectives:

• 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

Target Audience:

Developers of drugs in the field of rare diseases

Basic/Intermediate level

Overview of tutorial

Following topics will be covered:

• Orphan designation and maintenance at MA procedure
• Incentives
• Involvement of patients and external experts
• PRIME and early access tools for orphans

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

Objectives:

• 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.

Target Audience:

• 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

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

Objectives:

• 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.

Target Audience:

• 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.

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.