We are pleased to announce Fondazione Telethon ETS participation in the EJP RD Joint Transnational Call (JTC) 2023.
The aim of the call is to enable scientists in different countries to build an effective collaboration on a common interdisciplinary research project based on complementarities and sharing of expertise, with expected impact to use the results in the future for benefit of patients.
Projects shall focus on a group of rare diseases or a single rare disease following the European definition i.e. a disease affecting not more than five in 10.000 persons in the European Community, EC associated states, and Canada. Applicants are encouraged to assemble groups of rare diseases based on relevant criteria and commonalities if these leverages added value in sharing resources or expertise.
Topic of the Call: “Natural History Studies addressing unmet needs in Rare Diseases”
The objective of this call is to conduct efficient, innovative and high-quality natural history studies which will facilitate understanding of the disease’s or group of disorders’ progression throughout the lifespan of a patient. The goal of these studies is to collect and analyze comprehensive patient data to define targets for future therapies, taking into consideration innovation, safety, and efficacy.
Research proposals should cover at least one of the following areas:
- Estimation of disease prevalence;
- Identification of biomarkers/companions for the diagnosis/prognosis of a RD;
- Identification of biomarkers/indicators/predictors of a rare disease or group of disorders (e.g. having the same aetiology) onset/progression (including collection of genetic, physiological, environmental data or variables….);
- Identification of relevant endpoints for future studies that include potential biomarkers, querying patient-reported outcomes (PROs) and quality-of-life measures to identify the most;
- Identification of biomarkers/variables for therapeutic approaches (pharmacology, drug repurposing, gene therapy, RNA therapy, cell therapy, medical devices…).
Excluded approaches and topics
The following approaches and topics will be excluded from the scope of the JTC2023:
- Interventional clinical trials to prove efficacy of drugs, treatments, surgical procedures, medical procedures. This also includes studies comparing efficacy, e.g. two surgical techniques or therapies. Projects based on clinical phase IV pharmacovigilance studies cannot be funded either.
- Studies on the exclusive testing of the safety of medical devices and drugs.
- Development of new therapies as covered in EJP RD JTC 2020.
- Development of new analytic tools and pathways to accelerate diagnosis and facilitate diagnostic monitoring of rare diseases as covered in EJP RD JTC 2022.
- Projects focusing only on rare neurodegenerative diseases which are within the main focus of the Joint Programming Initiative on Neurodegenerative Disease Research (JPND). These are: Alzheimer’s disease and other dementias; Parkinson’s disease (PD) and PD-related disorders; Prion diseases; Motor Neuron Diseases; Huntington’s disease; Spinal Muscular Atrophy and dominant forms of Spinocerebellar Ataxia. Interested researchers should refer to the relevant JPND calls. However, childhood dementias/neurodegenerative diseases are not excluded.
- Rare infectious diseases, rare cancers and rare adverse drug events in treatments of common diseases. Rare diseases with a predisposition to cancer are not excluded.
Call Timeline:
| 12th December 2022 | Launch of the call |
| 15th December 2022 | Information webinar for potential applicants |
| 15th February 2023 | Pre-proposal submission deadline |
| End of April 2023 | Invitation to full proposal |
| May 2nd 2023 | Information webinar for applicants invited to submit a full-proposal |
| 14th June 2023 | Full proposal submission deadline |
| 26th July 2023 | Deadline for rebuttals |
| December 2023 | Notification of funding decision |
Forms
Please visit the following link https://www.ejprarediseases.org/jtc2023/ for more details about the topic and guidelines of the call or write to [email protected] .
