EURORDIS internal call for interest

Dear Members,

We’re pleased to share the following information with you for this month’s Member News:


EURORDIS internal call for interest – patient representatives for the Committee for Orphan Medicinal Products

The European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) is composed of around 30 experts including three patient representatives, whose mandate will end in July 2018. The European Commission (EC) has yet to issue the call for expression of interest but EURORDIS would already like to hear from you if you are interested in applying for the role of patient representative (read the job description). Please send your CV and a short email explaining your motivation to and Further details on the application process will be available once the EC has officially launched the call.

Petition calling on the EU to do more for health

The European Public Health Alliance, with the support of EURORDIS and around 40 other groups, is leading a call on the European Commission to step up coordinated EU action to tackle cross-border health challenges. A letter to Jean-Claude Juncker, President of the European Commission, calls for more EU health collaboration. Sign the petition!

EPF campaign: universal health coverage for all

The European Patients’ Forum campaign ‘Universal Health Coverage For All’ is an opportunity to raise awareness about the barriers patients face in accessing healthcare and to build on current political momentum to foster more EU cooperation on access to healthcare. Get involved!

Watch now – genome editing workshop

A recording of a EURORDIS workshop on gene therapy and genome editing is now available to watch! The workshop brought together patient representatives from across Europe with experts to discuss the scientific progress of these technologies and their relevance for rare diseases, as well as associated ethical, legal and social issues. Watch part 1 (What is genome editing? The science and beyond ), part 2 (Ethical, legal and social issues ) and part 3 (Perspectives of the biotech sector and regulatory aspects ).


Your feedback on final position paper on access to medicines

A work-in-progress EURORDIS reflection paper on possibilities for patients’ full and equitable access to rare disease therapies was published earlier in 2017. This first draft was developed taking into account the feedback of EURORDIS members submitted by email or during several consultative webinars, and was presented at EURORDIS’ Second Multi-Stakeholder Symposium on Improving Patient Access to Rare Disease Therapies in February 2017.

The final draft paper of the paper is in development and we would like to collect your feedback on its content; to receive details on how to participate in a consultative webinar in late September please email


Charlie Gard case

Over the past weeks EURORDIS has been closely following the story of Charlie Gard. The tragic situation this little boy and his family face resonates very strongly within the rare disease community, and also more broadly with the general public. Charlie’s parents have now announced that they are ending their legal battle to take Charlie to the US to receive an experimental treatment.

EURORDIS does not comment publicly on individual situations and has shared a letter explaining the decision not to communicate on this individual situation with national rare disease alliances. Our thoughts are with Charlie and his family at this difficult time.

Video testimonials: what do ERNs mean for patients?

In 2017 over 900 highly specialised medical teams from all over Europe joined forces to tackle rare or complex diseases within 24 European Reference Networks (ERNs). In four new videos, Elisa (also available with subtitles in Italian), Paula (with subtitles in Spanish), Jasper (with subtitles in Dutch) and Daniel (with subtitles in French) explain how the networks will help them.

In 2016 EURORDIS launched the ePAGs (European Patient Advisory Groups) to ensure patients are integrated in the development, governance and operations of the European Reference Networks (ERNs). Learn about recent ePAG activities and how to get involved.

Welcome to new members!

EURORDIS welcomes six new member organisations: Shoqata e Semundjeve te Rralla – Rare Disease Association (Albania), Solidarité Handicap autour des maladies rares (France), Association internationale maladies kystes Tarlov (France), Association of Cancer Patients in Finland (Suomen syöpäpotilaat ry) (Finland), AIBWS ONLUS (Italy), and Interregional Public Organisation of patients with Fabry Disease (Russia).

New, the online portal for rare disease patients and families, has launched a new look version of the website and a new feature – patient organisations as well as individuals can now register for RareConnect! RareConnect is a safe, easy-to-use platform where rare disease patients, families and patient organisations can develop online communities and conversations across continents and languages.

EURORDIS Therapeutic Report

Read the June edition of the EURORDIS Therapeutic Report to get the latest news about important activities at the European Medicines Agency on orphan medicines, medicines safety and patient involvement in various committees.


Please contact me if you have any enquiries or need additional information.

Kind regards,
Anja Helm

Anja Helm

Anja Helm
Manager of Relations with Patient Organisations.
+(33) 1 56 53 52 17

Add me to your contact list 


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This publication (or activity) has been funded with support from the European Union’s Health Programme. This material only reflects the views of the author, and funders cannot be held responsible for any use which may be made of the information contained herein.

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