27 de Junio, 2023
08 de Noviembre, 2023
11:59 PM
Establishing novel approaches to improve clinical trials for rare and ultra-rare diseases-HORIZON-JU-IHI-2023-04-04-two-stage
Investigación
Escuela de Medicina y Ciencias de la Salud
Comisión Europea
4.500.000 EUR
The topic aims to unravel roadblocks on the current clinical development pathways and deliver methodological solutions for innovative clinical trial designs and analyses, including regulatory considerations.
ExpectedOutcome:
The project generated from the topic should not only develop capacities and capabilities to execute innovative trial designs, but also plan to identify solutions to address scientific gaps as well as technical and operational challenges, and to collaborate / find synergies with relevant existing initiatives to establish a new, dedicated, rare disease specific and sustainable infrastructure. The project is expected to support innovation and optimise drug development for rare diseases with high unmet medical needs by focusing on clinical trials conducted for small populations and clusters of diseases with commonalities.
With a focus on addressing ‘white spots1’ in a subset of the 95% of underserved rare diseases, the R&I action to be supported under this topic should:
1,Deliver novel rare/ultra-rare disease-specific methodological approaches2 to transform the way treatments are developed with a view towards accelerating approval and access.
2,Pressure-test new clinical trial designs by using the playbooks co-created with stakeholders through case studies and modelling, addressing up to four selected paediatric / rare disease (or clusters of ultra‑rare diseases with commonalities) case studies, and different types of interventions (at least one for advanced therapy medicinal products (ATMP)).
Proposal page limits and layout: described in Part B of the Application Form available in the Submission System
at stage 1 of a two-stage Call, the limit for RIA short proposals is 20 pages;
at stage 2 of a two-stage Call, the limit for RIA full proposals is 50 pages.
Documents: (Check them on the webside).
IHI JU Evaluation form for Research and Innovation Actions (single and two-stage Calls)
IHI JU Proposal template (RIA/SP) - Part B
•Colombia is eligible as a third country, for this it must participate with the role of partner together with a consortium with at least three entities from different European/associated countries).
OBJETIVO/DESCRIPCIÓN
The topic aims to unravel roadblocks on the current clinical development pathways and deliver methodological solutions for innovative clinical trial designs and analyses, including regulatory considerations.
REQUISITOS CLAVE
ExpectedOutcome:
The project generated from the topic should not only develop capacities and capabilities to execute innovative trial designs, but also plan to identify solutions to address scientific gaps as well as technical and operational challenges, and to collaborate / find synergies with relevant existing initiatives to establish a new, dedicated, rare disease specific and sustainable infrastructure. The project is expected to support innovation and optimise drug development for rare diseases with high unmet medical needs by focusing on clinical trials conducted for small populations and clusters of diseases with commonalities.
With a focus on addressing ‘white spots1’ in a subset of the 95% of underserved rare diseases, the R&I action to be supported under this topic should:
1,Deliver novel rare/ultra-rare disease-specific methodological approaches2 to transform the way treatments are developed with a view towards accelerating approval and access.
2,Pressure-test new clinical trial designs by using the playbooks co-created with stakeholders through case studies and modelling, addressing up to four selected paediatric / rare disease (or clusters of ultra‑rare diseases with commonalities) case studies, and different types of interventions (at least one for advanced therapy medicinal products (ATMP)).
Proposal page limits and layout: described in Part B of the Application Form available in the Submission System
at stage 1 of a two-stage Call, the limit for RIA short proposals is 20 pages;
at stage 2 of a two-stage Call, the limit for RIA full proposals is 50 pages.
Documents: (Check them on the webside).
IHI JU Evaluation form for Research and Innovation Actions (single and two-stage Calls)
IHI JU Proposal template (RIA/SP) - Part B
•Colombia is eligible as a third country, for this it must participate with the role of partner together with a consortium with at least three entities from different European/associated countries).