Below are listed articles and PDF documents relating to the Council of European Disease Federation, including:
CEF Workshops:
Workshop 14, 9-10 November 2021, online
Presentations
- Revision of the general pharmaceutical legislation – Eliciting comment form NAs and EFs for the public consultation, Simone Boselli
- Revision of the general pharmaceutical legislation – Focus on unmet needs, access to treatment, Table of Negotiation and EU Fund, Simone Boselli
- UN Resolution – What it means? What is next? Clara Hervas
- Including rare cancers in Europe’s Beating Cancer Plan, Ariane Weinman
- Covid-19, François Houyez
- New CABs programme, François Houyez
- A new training by EURORDIS on how patients can take part in HTA, Matteo Scarabelli
- How can federations prepare themselves for a new pilot at the EMA, when the evaluation of a new medicine starts? François Houyez
- Access to orphan medicinal products: where do we stand, Simone Boselli
- Update on Newborn Screening, Valentina Bottarelli, Gulcin Gumus
Workshop 13, 26-27 November 2020, online
Presentations
- Brussels Rare Disease Week 2021, Kostas Aligiannis
- EU4Health
- Advocating toward a new European policy framework for rare diseases and how we could join all our forces to campaign together, Valentina Bottarelli
- Recommendations to progress towards a mature ERN system, Ines Hernando, Matt Johnson
- Update on RDI and International activities, Flaminia Macchia
- Update on Newborn Screening, Gulcin Gumus
- Access to orphan medicinal products: where do we stand, Simone Boselli
Workshop 12, Brussels, Belgium, 08 November 2019
Presentations
- ERN and ePAG update_Matt Johnson, Ines Hernando
- Prevalence Paper, Ana Rath, Orphanet
- Horizon Europe – DG Research €100 billion research and innovation programme to succeed Horizon 2020, Valentina Bottarelli
- New European Commission & Parliament, EU Presidencies, analysis of where rare diseases stand, risk & opportunities, our priority actions, Valentina Bottarelli
-
Rare Impact – better access to advanced therapies in Europe, Karolina Hanslik,
Simone Boselli
Workshop 11, Paris, France 11-12 December 2018
Presentations
- ERN update: Outcome conference November & next steps_Matt Johnson, Ines Hernando
- ePAG Training, Lenja Wiehe, Ines Hernando
- Social Position Paper, Raquel Castro, Dorica Dan
- Rare Barometer Voices surveys: How you can get infographics for the results of your country or group of diseases, Sandra Courbier & Erwan Berjonneau
- Patient Engagement in research_Virginie Bros‐Facer
- Community Advisory Boards (CAB)_Rob Camp
- Paradigm, Elisa Ferrer
- Horizon scanning – Methods_François Houÿez
- The HTx project ‐ Can we inform clinicians with real world evidence in real time when prescribing a treatment?_François Houÿez, Matteo Scarabelli
- Drug Repurposing_François Houyez
CAB Training
Workshop 10, Paris, France 26-27 October 2017
Presentations
- RareConnect_Marta Campabadal
- RareBarometer_Sandra Courbier
- Orphanet codificiation_Ana Rath & Annie Olry
- ERN deployment_Matt Bolz-Johnson
- EPAG update_Lenja Wiehe
- European Pillar of Social Rights_Annette Dumas
- Position Paper on Social Policy_Raquel Castro
- ECRD 2018_Emilie Zingg
- EuNetHTA_Matteo Scarabelli
- How to engage in HTA_Matteo Scarabelli
- HTA done properly_Russell Wheeler
- Patient Engagement_Elisa Ferrer
CAB Training
Workshop 9, Paris, France 3-4 Nov 2016
Presentations
Workshop 8 – Paris, 28-29 oct. 2015
Presentations
- ECRD 2016 Edinburgh- Get involved
- Rare Connect
- In Kind Contributions in Financial Reports
- European Year of Rare Diseases
- Patient Voices Survery Panel – Programme Plan
- EURORDIS Membership Criteria
- RARE – Bestpractices
- Rare Diseases International – Progress and Membership of International Federations
- Awareness Days
- INNOVCare – Innovation in social care provision to complex diseases
- ERNs – Overview of Assessment Process
- ERNs – Engaging your Organisation
Workshop 7 – Paris, Registry training 14-15 oct. 2014
Presentations
- RareConnect: An opportunity for partnership
- Rare Diseases International
- Debrief: EUCERD Joint Action Workshop Guiding Principles for Social Care in RDs
- European Year for Rare Diseases 2019
- Organisation of Centres of Expertise and European Reference Networks for RDs
- Benefit/risk evaluation : patients now invited to oral explanations @ CHMP
- Registry training: Anil Mehta
-
New Resources for Registries
Training Documents
- Fact Sheet: Rare Disease Patient Registries
- The how and why of disease registers
- The European Cystic Fibrosis Society Patient Registry: valuable lessons learned on how to sustain a disease registry
- Comparative demographics of the European cystic fibrosis population: a cross-sectional database analysis
- More evidence to favour newborn screening for Cystic Fibrosis
- Economic implications of newborn screening for Cystic Fibrosis: a cost of illness retrospective cohort study
- Cystic Fibrosis Registry Patient Information Sheet
- EUCERD core recommendations on rare disease patient registration and data collection
- EURORDIS-NORD-CORD Joint Declaration of 10 Key Principles for Rare Disease Patient Registries
- OpenApp Registry: The Evolution of a Patient Registry
- OpenApp Registry core common data elements
- User’s Guide to Registries Evaluating Patient Outcomes: Summary
- Patient Crossroads: Connect for Advocacy
- Patient Crossroads: Connect for Industry
Workshop 6 – Paris, France 29-30 October 2013
- Agenda
- Participants list
- European Year for Rare Diseases
- Access Campaign
- Specialised Social Services and Integration of Rare Diseases into Social Policies: State of the art & Training Social Services Providers
- Registries and Research Infrastructures
- RARE-Bestpractices project A platform for sharing best practices for the management of rare diseases
- Innovative Medicines Initiative
- Horizon 2020 and Rare Diseases Upcoming Opportunities for Research on Rare Diseases
- Discovering drugs for and with patients
- Lysogene, A Leading Gene Therapy Company
Workshop 5 – Paris, France 30-31 October 2012
- Report CEF WS 671KB
- Participants list CNA_CEF 57KB
- Specialised social services
- Understanding Off Label use 2M
- Rareconnect 1,8 M
- Federations Exchange Program 274 KB
- Support to European Federations 209 KB
- TIRCON 833 KB
- Euro Wabb 1,8 KB
Workshop 4 – Paris, France 30 June – 1 July 2011
Presentations:
Workshop 3 – Paris, France 1-2 July 2010
Presentations:
Workshop 2 – Brussels, Belgium 3 December 2009
Presentations:
Workshop 1 – Athens, Greece 7 May 2009