02/20/2019

Rare Diseases Research (RDR) Challenges Call

EJP RD – RDR Challenges NETWORKING EVENT ANNOUNCEMENT

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 will be organized in which the challenges will be presented and pre-arranged bilateral/multilateral networking meetings will be organized between potential applicants (academia, SMEs, patient organizations) and the involved industry partners that identified the challenges.

 

Summary information on the RDR Challenges Call

The RDR Challenges call will be launched in March 2020.

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.

 

THE NETWORKING EVENT FOR THE RDR CHALLENGES CALL WILL BE HELD

ON 3rd MARCH 2020 IN PARIS

 

The agenda of the full-day event (10:30am – 6:00pm) is as follows:

  • Opening session devoted to the presentation of the EJP RD and the RDR Challenge funding scheme by the coordinator of the EJP RD and RDR Challenges task leader
  • Presentations of the challenges (see below) by industry partners and possibilities for Q&A
  • Buffet lunch and networking break
  • Pre-arranged bilateral/multilateral networking meetings between academia, SMEs and industry with potential partners identified from the information provided by a survey

It is mandatory for future participants to express their interest of participation in the networking event and B2B meetings via the survey. Since the number of places is limited, the organizers may apply a selection process. Priority in participation will be given to entrepreneurs and research organizations with demonstrable relevant expertise, collaborations and initiatives and to representatives from European Federations of Rare Diseases Patient Organizations. Selected participants will be required to attend the whole networking event.

The event will take place in the Hotel Pullman Paris La Défense, 11 avenue de l’Arche, 92400 Courbevoie, FRANCE.

 

RDR Challenges

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


 

PARTICIPANTS

The Networking Event is open to academia, clinical/public health sector, SMEs, and rare disease patient representatives from listed below countries involved in the EJP RD. To ensure active and productive participation at the Networking Event, a maximum of 100 participants will be admitted.

COUNTRYCOUNTRY
ArmeniaAustria
BelgiumBulgaria
CroatiaCzech Republic
DenmarkEstonia
FinlandFrance
GermanyGeorgia
GreeceHungary
IrelandIsrael
ItalyLatvia
LithuaniaLuxembourg
MaltaNorway
PolandPortugal
RomaniaSerbia
SlovakiaSlovenia
SpainSweden
SwitzerlandThe Netherlands
TurkeyUnited Kingdom

Canada (decision still pending. All Canadian applicants are requested to contact call secretariat).

REGISTRATION

Please register to the Networking Event by completing this survey.

Please also note that the survey can be opened in Firefox and Google Chrome, but NOT in Internet Explorer.

SELECTION OF THE PARTICIPANTS

A selection process will be applied based on the participants’ background and expertise. Representatives from the partners in the EJP RD responsible for setting up and implementing the challenges (including the French Foundation for Rare Diseases, EURORDIS, Inserm, DLR, ZonMw, AFM and EATRIS) will select participants according to the relevant interest and expertise in line with one of the four challenges and the type of organization (academics, SME and patient organizations) to ensure fair representation for all challenges and from all type of organizations.

Participants will be informed of the results by email on 10 February 2020.

FEES & COSTS

The Networking Event is free of charge. Participants are expected to arrange their own travel, accommodation and other expenses to attend the Networking Event. Coffee refreshments and lunches will be offered during the day. The organizers will not cover expenses incurred by the participants. A maximum of 8 travel fellowships (up to 350 euros each) for representatives of selected patient organizations are available. Please tick the box in the survey if you are a patient representative and interested in a fellowship. The fellowships will be selected and attributed in accordance to the EURORDIS fellowship evaluation scale available here.

OFFICIAL LANGUAGE

The event will run in English.

CONTACT

For any questions regarding the application form, please contact:

christine.fetro(at)fondation-maladiesrares.com

diana.desir-parseille(at)fondation-maladiesrares.com