Conference programme

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Programme - 10 April

Address: 2A Księcia Trojdena Street, 02-109 Warsaw

08:00 | REGISTRATION

Moderator of the conference: Jarosław KULCZYCKI, Chief Public Relations Officer, Medical University of Warsaw, Poland 

09:00-09:30 | OPENING SESSION

Welcome: Rafał KRENKE, Rector, Medical University of Warsaw, Poland

  • Izabela LESZCZYNA, Health Minister, Poland (video message) introduced by Urszula DEMKOW, Undersecretary of State, Ministry of Health, Poland

  • Baiba MILTOVIČA, President of the Section for Transport, Energy, Infrastructure and the Information Society, European Economic and Social Committee

  • Olivér VÁRHELYI, European Commissioner for Health and Animal Welfare (video message)

  • Adam JARUBAS, Chair of the European Parliament Committee on Public Health (online)

09:30-10:45 | SESSION 1 - Towards an EU action plan on rare diseases: national plans and European cooperation – state of play and future actions

Moderator: Enrique TEROL, Health Counsellor, Permanent Representation of Spain to the EU

  • Urszula DEMKOW, Undersecretary of State, Ministry of Health, Poland: "Poland’s national strategy on rare diseases"
  • Anne-Sophie LAPOINTE (PhD), Project Manager of Rare diseases mission, Ministry of Labour, Health, Solidarity and Families, France: "Main outcomes and lesson learned from the French national strategy on rare diseases"
  • Martina BRZKOVA, Department of Healthcare Services. Ministry of Health, Czech Republic (online) “Towards a second Czech National Strategy on Rare Diseases”

  • Donata MERONI, Head of Unit 'Health monitoring and cooperation, Health networks', Directorate-General for Health and Food Safety, European Commission: "The EU action for rare diseases" 

    Debate 

10:45-11:15 | COFFEE BREAK

11:15-12:30 | SESSION 2 - Organisation and management of healthcare systems for patients with rare diseases

Keynote Speech: Krystyna SZYMAŃSKA, Professor at the Medical University of Warsaw, Poland: "Showcase of rare disease patient"

Moderator: Josep FIGUERAS, Director Emeritus of the European Observatory on Health systems and policies, Ambassador for the Health Union Fellowships

  • Luca SANGIORGI, ERN BOND Coordinator, Coordinator of the ERNs Coordinators Group: "ERNs 8 years’ experience on integration into the national healthcare systems: barriers and enablers"
  • Eileen TREACY, Clinical Professor of Medicine of Rare Disorders, Trinity College Dublin, Ireland: "Development of efficient patient pathways and referral system: the EU dimension: JARDIN (The Joint Action on Integration of ERNs into National Healthcare Systems)"
  • Juan Fernando MUÑOZ MONTALVO, Secretary General of Digital Health and Innovation, Ministry of Health, Spain: "ÚNICAS: A network of coordinated care and expert knowledge to support children with rare diseases and their families"

  • Lori RENNA LINTON, EuroHSP: "The rare diseases patient journey experience"

    Debate

12:30-13:30 | SESSION 3 - Access to treatments: key issues and new approaches

Moderator:  Alexis ARZIMANOGLOU, Professor, Coordinator of the European Reference Network for Rare and Complex Epilepsies (ERN EpiCARE)

  • Kaja KANTORSKA, Policy Officer, Unit 'Medicines: Policy, Authorisation and Monitoring', Directorate-General for Health and Food Safety, European Commission: "Regulatory options and pull and push incentives" 
  • Daria JULKOWSKA, ERDERA scientific coordinator, INSERM, France: "The example of the European partnership ERDERA to boost clinical research and innovation"
  • Eulàlia OLESTI, Regulatory Affairs Lead, Hospital Clínic Barcelona, Spain: "Academic CAR-T Clinical Development: The Case of ARI-0001"

  • Katarzyna PIOTROWSKA-RADZIEWICZ, Director, Department of Drug Policy and Pharmacy, Ministry of Health, Poland: "Orphan drugs in Poland - access, new developments, key issues"

    Debate

13:30-15:00 | LUNCH

15:00-15:30 | SESSION 4 - Solidarity and cooperation on rare diseases with Ukraine

Moderator:  Urszula DEMKOW, Undersecretary of State, Ministry of Health, Poland

  • Tacjana WILCZYŃSKA, Head of Clinic of Children Palliative Care, Consultant for Mobile Palliative Care Unit, Kyiv City Children Diagnostic Centre, Ukraine
  • Tatiana MAKAREVICH, Public Health Support Officer, WHO Country Office for Poland: "The initiatives and outcomes of Poland’s support to Ukraine"

  • Ruth LADENSTEIN, Professor, ERN PaedCan Coordinator, Vice-Chair of the ERNs Coordinators Group  

    Debate

15:30-16:15 | SESSION 5 - Towards reducing the time for an early and accurate diagnosis for patients with rare diseases

Moderator: Virginie BROS-FACER, CEO, EURORDIS 

  • Maurizio SCARPA, Coordinator of MetabERN: "Promises and challenges of genomic new-born screening"
  • Felipe GÓMEZ MARTÍN, Policy Maker, Ministry of Health, Spain: "Screening, Genetic Tests and Reference Centres for Patients with Rare Diseases in the Spanish National Health System"
  • Bruno RODRIGUES, Head of Stats and Data Strategy, Ministry of Research and Higher Education, Luxembourg: "The European “1+ million genomes” initiative" (online)

  • Lene CIVIDANES, Head of Health, Research and International Relations, Danish National Genome Center: "Discuss national initiatives improving access to genetic and genomic testing"

    Debate

16:15-17:15 | SESSION 6 - Patient data as a fundamental element in addressing the needs of patients with rare diseases

Moderator:  Hélène LE BORGNE, Policy Officer, Unit 'Health Innovations & Ecosystems', Directorate-General for Research & Innovation, European Commission

  • Tomislav SOKOL, Member of the European Parliament, Rapporteur on the e-Health Data Space Regulation: "Expected benefits for patients with rare diseases" (online)
  • Holm GRAESSNER, Coordinator of ERN-RND and JARDIN WP7 leader: "Patient registries and data sharing in the ERN environment"
  • Joni KOMULAINEN, Senior Ministerial Adviser, Ministry of Social Affairs and Health, Finland: "Implementation of the regulation at the national level in the case of rare diseases: Is there a need for a particular approach?" 

  • Szymon BIELECKI, Head of Sector - Research and Innovation, Unit 'eHealth, Well-Being and Ageing', Directorate-General for Communications Networks, Content and Technology, European Commission: "Opportunities of data technologies in tackling rare diseases"

    Debate

17:15-17:30 | CLOSING SESSION

  • Urszula DEMKOW, Undersecretary of State, Ministry of Health, Poland
  • Ágnes CSER, Rapporteur of the European Economic and Social Committee's exploratory opinion "Leaving No One Behind: European Commitment to Tackling Rare Diseases" 

Simultaneous interpretation provided from English and Polish into English, Polish and French.