Mr Torben Grönnebaek, Rare Disorders Denmark and Co-chair of the Programme Committee

Mr Marek Twardowski, Undersecretary of State, Ministry of Health Poland

Dr Andrzej Rys, Director of Public Health, European Commission

The Added Value of Centres of Expertise

Centres of Expertise, linked with European Reference Networks, form the backbone of EU and national strategies on Rare Diseases. This session will revisit the basic concepts developed over the past 4 years and will discuss the current state of play and its potential to improve quality of care for patients.

Co-chairs:

• Dr Edmund Jessop, Medical Adviser, NHS National Commissioning Group, United Kingdom

Introduction

• Yann Le Cam, Chief Executive Director, Eurordis

   

Round table with:

• Prof Fons Gabreels, Department of Child Neurology, Radboud University Hospital, The Netherlands

   

• Stein Are Aksnes, Directorate for Health and Social Affairs, Norway

   

• Guillaume Le Henanff, Ministry of Health, France (invited)

   

• Cristina Rusu, University of Medicine and Pharmacy, Lasi, Romania

Orphan Drug Development, Paediatric Investigation Plans and Advanced therapies

This session will examine the different committees centred around one common objective: to bring innovative medicines to a broader set of patients.

• Michele Lipucci di Paola, Committee of Advanced Therapies, European Medicines Agency, and Eurordis

• Prof Josep Torrent i Farnell, COMP, Fundacio Doctor Robert

   

Speakers

• Dr Kerstin Westermark, COMP, European Medicines Agency: the EMEA workshop May 2010 for the 10th anniversary of the Orphan Drug Regulation and challenges for the future.

   

• Dr Fernando Andrés Trelles , PDCO, European Medicines Agency: Paediatric Committee and Paediatric Investigation Plan: What is relevant to rare diseases?

   

   

• Dr Fabrizia Bignami, Eurordis : Committee for Advanced Therapies: What is relevant to rare diseases?

The Added Value of European Reference Networks?

Co-chairs:

• Antoni Monserrat, DG Sanco, European Commission

• Dr Frits Lekkerkerker, WGM, Netherlands

• European perspective

  • Dr Frederic Sicard (Co-chair High Level Group on Care and Medical Services)

• Examples of Reference Networks

  • Prof Krystyna Chrzanowska, Poland - Dyscerne Project
     
  • Prof Thomas Wagner, ECORN-CF, Germany

Making the Best use of Funds for Genetic Testing

Is what is possible always desirable? This session will explore the utility of genetic testing.

Co-chairs:

• Pol Gerits, Federal Service of Health, Food Chain Safety and Environment, & Europlan, Belgium (invited)

• Prof Milan Macek, Department of Biology and Medical Genetics, Charles University Prague, Czech Republic

 Speakers:

• Prof Bruno Dallapiccola, cientific director of Bambino Gesù Children Hospital, Italy

   

   

Cross Border genetic testing

• Prof David.E Barton, National Centre for Medical Genetics, Ireland: Cross Border genetic testing

   

Undiagnosed Rare Diseases

• Prof. Anil Mehta, University of Dundee, UK: Undiagnosed rare diseases, the EuroCareCF project

Improving Access to Orphan Drugs

Authorised but not always accessible to patients! From the European Pharmaceutical Forum recommendations on improving access to orphan drugs, we now need to consider implementation through international collaboration

Co-chairs:

• Prof Hans Georg Eichler, Senior Medical Officer, European Medicines Agency

• Lia van Ginneken, EMP, Netherlands (invited)

Speakers:

EUnetHTA working group on Monitoring emerging/new technology development and prioritisation of HTA

Claudia Wild,  Ludwig Boltzman Institute of Health Technology Assessment, Vienna, Austria

The point of view of the industry

Dr Andras Fehervary, Novartis

How can interaction between regulators and payers facilitate access to Orphan Drugs?

Prof Hans Georg Eichler, Senior Medical Officer, European Medicines Agency

Pawel Wojtowicz, Cystic Fibrosis Poland

Bozenna Dembovska, COMP member Poland