Useful resources
Workshop 1
- European Patient Advocacy Groups (ePAGs) – Introductory video on ERNs
Workshop 2
- Don’t forget to drop your own tips for making an online event engaging here!
- ECRD 2020 learning and recommendations
- Speaker guidelines
- Budget template
- Session script
- Glossary: Suppliers of digital tools
Networking session/Soap box
- Addressing the challenges of diagnosis for a rare disease: How IPWSO is supporting diagnosis for people with Prader-Willi syndrome, Marguerite Hughes and Agnes Hoctor, IPWSO
- International patient registry on the effects of COVID-19 in people with rare diseases, Garrahan Hospital (Buenos Aires) and Sant Joan de Déu Barcelona Children’s Hospital
-
Common challenges in uncommon conditions, Rosanne M. Smits and Chris Verhaak (Amalia Childrens Hospital, Radboudumc, Nijmegen, Netherlands)
Pre-reading material
Before the meeting begins on Wednesday, May 12, we invite you to read the preparatory materials listed below:
Plenary session
Workshop 4
- Key principles for Newborn Screening
- RARE IMPACT EU Report: “Challenges and solutions for improving patient access to advanced therapies medicinal products at the European Union level”
- UNIAMO and PO's Position Paper
Breakout 1:
- Rare 2030 Recommendation #8 (see page 111)
Breakout 2:
- Key principles for newborn screening: A EURORDIS position paper (January 2021)
- European Alliance for Newborn Screening in SMA, Spinal muscular atrophy: Screen at birth, save lives (Whitepaper version 1, 26 March 2021)
Breakout 3:
- Best practice. Taking action at national level: Connecting patient organisations to Centres of Expertise by means of a legal role in the designation process (documents below in Dutch but translatable into English using your browser):
- Letter from the minister of health to the parliament (including several other actions in the field of rare diseases)
- Related publication in the ‘Staatscourant’
- Related publication on the ministry of health’s website
- Instructions (VSOP website) for patient organisations on how to assess the CoEs (and several other related webpages).
- Best practice. Taking action at European Level: