The report is available here.

The aim of this policy brief consists of setting up a methodological approach to understanding translation of research into innovative outcomes. Studies on knowledge transfer and innovation processes often come across the idea of “the valley of death”, that is the critical phase where one step of the innovation process ends and a new one begins. In this perspective, one of the roles of partnerships is to bridge this valley of death by linking excellent research with technology and solution-minded business undertakings hence generating virtuous cycles of research and innovation.

The report, to which IRDiRC members Takeya Adachi, Gareth Baynam and Hugh Dawkins contribute as an expert panel, takes a look at the challenges patients with rare disease face in accessing treatments. It also considers where healthcare and health systems have made progress in helping people living with rare diseases, as well as identifying ongoing areas that need improvement.

The full report is available for reading here.

In this report, the Foundation undertook a comprehensive review of the approved and investigational drugs and therapies for Dravet syndrome. The report also summarises the activities and achievements in 2021 as a Foundation dedicated to research and the patient.

For more information on the report: https://dravetfoundation.eu/dravet-syndrome-therapeutic-pipeline-report-end-of-2021-for-families/

The report contains summaries and quotes from the event’s 70 online live sessions and workshops, as well as from over 200 consultations between participants and Commission representatives.

Notable take-ways of the report include:

• Europe’s collaborative research and innovation at the global scale are key drivers for a greener, healthier and more digital future for all;
• Europe needs more synergies between policies, programmes and instruments at EU, global, national and local level to remain at the forefront of research and innovation, and to be effective at delivering a sustainable future;
• Citizens need to continue having the opportunity to add their voice to the conversation about the future of European research and innovation.

The report’s output will feed into the next co-creating steps of European policy-making in research and innovation, followed by co-implementation.

For more information:

• Event recordings remain available on the event platform.
• The European Research and Innovation Exhibition showcases ground-breaking EU-funded research.

Click here to read the full report

In order to improve care and get insights to enhance future research, optimal (re)use of data collected from Duchenne and Becker patients is of the essence. For this reason, the Duchenne and Becker Muscular Dystrophy (DMD and BMD) community has been working on making DMD/BMD data FAIR since 2018. FAIR Data means your data is Findable, Accessible, Interoperable, and Reusable for both humans and machines. Patient organizations are key drivers of the FAIRification process in practice, and dialogue with stakeholders is critical to success.

Data of people living with Duchenne and Becker Muscular Dystrophy (DMD and BMD) are collected by researchers, in the health system and by themselves through Patient Reported Outcomes and wearables. In 2019, the World Duchenne Organization published the Duchenne FAIR Declaration, highlighting the need for optimal (re)use of data. Currently, the data are often kept in different data systems. Use and reuse is prevented by technical barriers, or problems with ownership, control, data protection and security. The failure of connecting data hampers the discovery of new diagnostics, treatments, healthcare policies, and the use of data by the patient themselves. Each, which can benefit people living with DMD and BMD greatly.

The report of the second virtual meeting summarizes the presentations and discussions of the meeting on March 3. In addition to this, it provides an overview of the key lessons learned since the first meeting, and outlines the next steps. Participants included representatives from patient organizations, clinicians, clinical and academic researchers, pharmaceutical companies, regulators, FAIR Experts and EU organizations. The wide spectrum of stakeholders shows convergence of interests, alignments of efforts and willingness to overcome any obstacles in order to build a solid ecosystem for FAIR data sharing of patient-derived data (e.g. disease or patient registries) at a European level – this is what rare disease patients like DMD/BMD really want.

For any additional information, please contact Nawel van Lin (nawel [at] worldduchenne.org).