The innovative “Rare Diseases Research (RDR) Challenges” call will be implemented to facilitate and fund collaboration between industry, academia, SMEs, and patient organizations to solve specific research challenges in rare diseases.
Industry partners have been involved in the identification of four challenges that are the topics of the call and that were validated by the EJP RD partners including patients.
Collaborative consortia of applicants (academics, SMEs, patient organizations) and the involved industry partners will address the challenges and provide solutions. It is expected that the creation of new RDR partnerships will enable access to scientific and technological innovations emerging from SMEs and academic research groups for industry and provide a facilitated pathway for academics to exploit their research.
In order to initiate exchanges and/or formation of collaborative consortia of applicants to the call, a Networking Event has been organized in which the challenges have been presented and pre-arranged bilateral/multilateral networking meetings took place between potential applicants (academia, SMEs, patient organizations) and the involved industry partners that identified the challenges.
The RDR Challenges Call is now open, deadline is 30 June 2 pm (CEST)
It foresees selection, evaluation and funding of projects in a 2-stage process. Challenges must be solvable in a short time period of 30 months with first milestones/deliverables at M18.
A first Scientific Evaluation Committee (SEC) meeting will evaluate applications responding to the proposed challenges for the first 18-month phase grant. The aim of this selection process is to fund one project for each proposed challenge.
Following the completion of the first phase (after 18 months), the applicants submit a report demonstrating the work undertaken during the study and, if appropriate, an application for phase 2 funding.
The SEC will examine the results from the 1st phase and evaluate what has been delivered in the specific timeline and how the applicants met the milestones/deliverables. After positive evaluation, the SEC will validate projects for the second phase grant.
The total budget of 1.5 Mio€ funding from the European Commission allows for 4 projects to be funded (375 000 € per project).
The involved industry partners who identified a challenge will join the consortium of applicants once selection is made by the SEC and will co-fund (in cash and in kind) the granted project.
Direct link to the electronic proposal submission
Development of a non-invasive tool for measuring rare disease patient mobility in daily living
INDUSTRY SPONSOR. Chiesi Farmaceutici S.p.A. (Italy), CSL Behring (Australia)
AIM. To develop a set of coordinated non-invasive tools for measuring rare disease patient general movements distinguishing between voluntary and involuntary movements (e.g. by distributing movement-sensors in patients’ home, on their body, on the wheelchair…)
DOWNLOAD THE PDF. RDR Challenge CHIESI CSL BEHRING
Delivery system for intranasal administration of biological drugs to neonates
INDUSTRY SPONSOR. Chiesi Farmaceutici S.p.A. (Italy)
AIM. To develop a delivery system allowing administration of liquids or gels in nostrils of the neonates for intranasal administration of biological drugs.
DOWNLOAD THE PDF. RDR Challenge CHIESI
Characterize Rare Bone Disorders (RBD) Mobility Challenges in Real World Setting
INDUSTRY SPONSOR. Ipsen
AIM. Develop full-body automated mobility assessment tool(s) to assess real-life mobility challenges in people living with RBD, to be compared vs available disease specific patient- and Health Care Professionals (HCP)-reported mobility assessments. Capturing these real-life data could help determine if patient characteristics or environmental conditions could be used to predict mobility outcomes and therefore open possibilities for preventive or corrective interventions, including home and assistive devices design.
DOWNLOAD THE PDF. RDR Challenge IPSEN
Pre-clinical assay to detect instability of microsatellite repeat expansions
INDUSTRY SPONSOR. Pfizer, Cydan
AIM. To develop and validate an assay for screening genes and/or compounds that modulate instability of microsatellite repeats. The rarity of repeat expansion/contraction events, estimated to be <1 per 10,000 DNA molecules, creates many challenges for assay development. The goal of this proposal is to devise, implement, and validate an assay that displays the robustness and sensitivity to detect repeat expansion/contraction events after ≤1 week of compound treatment. The assay should utilize a read-out that is suitable for a mid-scale screen of 100s to thousands of compounds in dose response. If such an assay is developed, it will be transferred to Pfizer for further characterization and validation.
DOWNLOAD THE PDF. RDR Challenge PFIZER CYDAN
The Networking Opportunities is open to academia, clinical/public health sector, SMEs, and rare disease patient representatives from listed below countries involved in the EJP RD.
For any questions, please contact: