EJP RD Launches First Rare Diseases Research Challenges Call

The European Joint Programme on Rare Diseases (EJP RD) was established to help in coordinating the research efforts of European, Associated and non-European countries in the field of rare diseases and to implement the objectives of the International Rare Disease Research Consortium (IRDiRC).

The newly implemented Rare Diseases Research (RDR) Challenges call aims to facilitate collaboration between industry, academia, SMEs, and patient organisations 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.

The challenges are:

1. Development of a non-invasive tool for measuring rare disease patient mobility in daily living (Sponsors: Chiesi Farmaceutici SpA, Italy, CSL Behring, Australia)

The aim of this challenge is to develop a set of coordinated non-invasive tools for measuring rare disease patient general movements distinguishing between voluntary and involuntary movements (eg by distributing movement-sensors in patients’ home, on their body, on the wheelchair etc.)

2. Delivery system for intranasal administration of biological drugs to neonates (Sponsor: Chiesi Farmaceutici SpA, Italy)

The aim of this challenge is to develop a delivery system allowing administration of liquids or gels in nostrils of the neonates for intranasal (IN) administration of biological drugs.

3. Characterise Rare Bone Disorders (RBD) Mobility Challenges in Real World Setting (Sponsor: Ipsen

The aim of this challenge is to 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.

4. Pre-clinical assay to detect instability of microsatellite repeat expansions (Sponsor: Pfizer, Cydan)

The aim of this challenge is 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 characterisation and validation. A total budget of €1.5 million from the European Commission allows for four projects (one for each challenge) to be funded at €375,000 per project. The involved industry partners who identified a challenge will join the consortium of applicants once selection is made by the CEC and will co-fund (in cash and in kind) the granted project. Transnational consortia consisting of a minimum of two (and a maximum of six) eligible applicants from at least two different countries participating in the call are eligible to apply. The call is open to academia, clinical/public health sector, SMEs, and rare disease patient representatives from the following countries: Armenia, Austria, Belgium, Bulgaria, Croatia, Czech Republic, Denmark, Estonia, Finland, France, Georgia, Germany, Greece, Hungary, Ireland, Israel, Italy, Latvia, Lithuania, Luxembourg, Malta, Norway, Poland, Portugal, Romania, Serbia, Slovakia, Slovenia, Spain, Sweden, Switzerland, The Netherlands, Turkey and United Kingdom. In order to initiate exchanges and/or formation of collaborative consortia of applicants to the call, a Networking Event took place in Paris on 3 March 2020 where the challenges were presented. Pre-arranged bilateral/multilateral meetings were also organised between potential applicants (academia, SMEs, PAOs) and the involved industry sponsors. If a potential applicant was not able to join the networking event it is mandatory to contact the industry sponsor in order to validate relevance and adequacy of the concept. The deadline for applications is 30 June 2020 (14:00 CET). Funding decisions will be notified in December 2020. More information about this research funding opportunity and the application process is available on the RESEARCHconnect funding information platform. RESEARCHconnect provides up-to-the minute content, insight and analysis on research funding news and policy. To find out more about how RESEARCHconnect can keep you in the know, and subscription fees, contact us today.