RESEARCH

Rare Disease Research Challenge

The European Joint Programme on Rare Diseases (EJP RD) brings over 130 institutions (including all 24 ERNs) from 35 countries. On March 3rd Dace Liepina and Ingunn Westeheim was supposed to represent OIFE at a networking event in Paris related to the Rare Diseases Research (RDR) Challenges Call.

We were particularly interested in challenge nr. 3 “Characterize Rare Bone Disorders (RBD) Mobility Challenges in Real World Setting” sponsored by the company IPSEN. The aim of the challenge is to develop full-body automated mobility assessment tool(s) to assess real-life mobility challenges in people living with rare bone disorders, to be compared vs available disease specific patient- and Health Care Professionals (HCP)-reported mobility assessments. You can read more about the challenge here.

Because of concerns connected to COVID-19, Dace & Ingunn decided to cancel their trip, but we have followed up with meetings on Zoom and phone conferences with different stakeholders connected to the challenge.

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ADVOCACY

Call for proposals for research projects

The European Joint Programme on Rare Diseases (EJP RD) has made a preliminary announcement about the upcoming 2020 call for proposals for pre-clinical research to develop effective therapies for rare diseases like OI.

A number of national and regional funding organisations will participate in the call, alongside the European Commission and the EJP-COFUND action, to provide the funding for the research projects that are selected. Spread the word and encourage researchers in your network to respond to the call! Patient organisations like the OIFE are also eligible to be partners in the call. This is why the OIFE would like to hear from you on office@oife.org as soon as possible if you and/or your colleagues are planning to respond to this call.

If you have any questions about the call for proposals please contact EJPRDcall@anr.fr.

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EDUCATIONAL

Registries in Rome

OIFE’s honorary member Taco van Welzenis recently attended the International Summer School on Rare Disease Registries and FAIRification of Data in Rome, Italy from Sep. 23rd – 27th 2019. It was a part of a series of training activities proposed by the European Joint Programme on Rare Diseases (EJP RD). You can download the agenda of the course here.

The Course was made up of 5 days of residential training organized by Istituto Superiore di Sanità (ISS) in close collaboration with other stakeholders.

Registries are key resources in order to increase timely and accurate diagnosis, improve patients management, tailor treatments, facilitate clinical trials, support healthcare planning and speed up research.

This course was composed of two training modules:

Module 1:
“Rare Disease Registries” where the participants learned
(a) what resources are needed for the establishment / maintenance of a high quality registry
(b) the features of successful strategies to ensure long-time sustainability of the registry, quality, legal and ethical issues in compliance with the EU General Data Protection Regulation and FAIR principles.

Module 2
“FAIRification of data” enabled the participants to work with IT-trainers learn more about the FAIR principles. The potential of a FAIR registry, as the basis for cross resource questions, were demonstrated by executing a query across the use cases that become FAIR. A time slot was allocated to discuss FAIR data management and FAIR project planning.

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