I SSUE 4 DECEMBER 2019

NEWSLETTER

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ERN CRANIO 2019 ANNUAL MEETING , ROME 29 -30 NOVEMBER The ERN CRANIO 2019 annual meeting was hosted by Fondazione Policlinico Universitario Agostino Gemelli in Rome, Italy on

29-30 November 2019. Many thanks to all who attended.

Each ERN CRANIO annual meeting features an educational session on a specific topic relevant to the diseases covered by the

network. This year the educational session was focused on difficult airway in craniofacial disorders.

We welcomed speakers to present on the following topics:

Diagnostic tools, causes and consequences

The long term outcome of laryngotracheal stenosis

The role of the ENT surgeon in airway management for children with craniofacial anomalies

Perioperative care for craniofacial patients with a difficult airway

Swallowing disorders in craniofacial disorders

Thank you to all of our speakers for your insightful presentations which stimulated interesting discussion.

The meeting continued with a presentation by the ERN CRANIO management team (coordinator & project managers). Key network updates and future opportunities within the ERN framework were presented. Other presentation topics included:

Patient registries (and the recently submitted funding application for an

ERN CRANIO patient registry)

The European Joint Programme on Rare Diseases (EJP RD) and

opportunities for ERNs within this programme

Patient representation and ‘Patient Journeys’

The ERN CRANIO 2020 annual meeting will be held on 13-14 November at Vall d’Hebron hospital, Barcelona. Save the date!

Patient representation and ‘Patient Journeys’ This year and with the support of EURORDIS, our patient representatives have worked on developing ‘Patient Journey’ documents. Creation of these documents asks patient representatives to reflect on their own personal journey with the rare disease they represent and map what their needs were against what would have been the 'ideal clinical care' at each stage. These journeys are then reviewed and added to by others in their rare disease community. On Friday 29th, Matt Johnson (from EURORDIS) presented on this initiative and its potential benefits. On Saturday 30th attendees met in their diagnosis-specific work streams/working groups. ERN CRANIO patient representatives had the opportunity to present their patient journeys here, stimulating valuable discussion.

Patient journeys were presented for: Craniosynostosis, Craniofacial Microsomia, Cleft lip/palate & Genetic hearing loss. A patient journey on Pierre Robin Sequence has also been developed. These journeys will continue to be shared, added to and developed. The clinical content may also be reviewed by clinicians within the network.

The second call for new healthcare centres to join the ERNs as full members officially closed on 30th November 2019. Across all 24 ERNs, a total of 841 applications were received. ERN CRANIO received 24 applications.

THE 2ND CALL FOR FULL ERN MEMBERSHIP– CLOSED!

1 2 3 4 5 ~ End of 2021

European Commission eligibility check (1-1.5 months)

ERN CRANIO assessment committee review and provision of opinion (3 months for draft ERN decision + 1 month for applicants’ comments + 1 month for final ERN decision)

Independent Assessment Body (IAB) review and preparation of draft assessment report

Independent Assessment Body review and preparation of final assessment report (6 months for draft and final assessment report)

Board of member states (BoMS) approval of applications (1-2 months)

For more detailed information on the assessment process click here.

The call for affiliated partners is also now closed. These partnerships are now being formalised and an integration strategy is being prepared (European Commission deadline: end of January 2020).

INIT IATIVE FOR SURGI CAL ERNS TO COLLABORATE

On 15 November, ERN coordinators and project managers met in Brussels for the 9th internal ERN coordinators meeting. At this meeting Prof. dr. Irene Mathijssen (Chair of the ERN coordinators group & ERN CRANIO coordinator) proposed the idea to set up a surgical-based ERN working group. Many ERNs were interested in this initiative. Interested ERNs will nominate a point of contact to be involved in this collaboration. The working group will begin collaborating in the new year.

OTHER RELEVANT MEETINGS , EVENTS & DEADLINES FOR YOUR INFORMATION:

JANUARY 2020 20th: Deadl ine for ERN CRANIO cent res to submi t the i r 2019 moni tor ing data

FEBRUARY 2020 18th: European Jo int Programme on Rare Diseases Jo int Transnat ional Cal l —pre -proposal submiss ion deadl ine.

MARCH 2020 11 -14th: The In ternat ional Congress of Research on Rare and Orphan Diseases (Re[act ] congress & IRDiRC conference, Ber l in) 15th: EJP RD Internal ca l l : Cl in ica l t r ia ls methodology demonstrat ion pro jects —ini t ia l deadl ine.

MAY 2020 15 -16th: The 10th European Conference on Rare Diseases and Orphan Products , Stockholm

Possible Board of Member States

action on an unfavourable decision