Innovative designs, extrapolation, simulation methods and evidence-tools for rare diseases addressing regulatory needs

Funding programme: Horizon Europe (N 101136365)

Budget: €6M

Coordinator: INSERM

Duration: 5 years (January 2024 - December 2029)

The overall aim of INVENTS is to provide clinical trial stakeholders, trialists and regulators with a generalisable framework encompassing methods, workflows and evidence-tools to improve the level of evidence in regulatory decision making in rare diseases. This will be achieved through the development and validation of improved extrapolation models, simulation and in silico trials, model based clinical trial design and evidence synthesis methods, all based on robust and mature computational models and qualified on extensive data from representative selected use cases.

The evaluation of new medicines for rare diseases (RD), including paediatric RDs, is challenging for several reasons, among which are the small patient sample sizes, heterogeneity of patients and diseases and heterogeneity in disease knowledge. Due to these difficulties, access to effective treatments and the number of treatment options are often limited. This is where the INVENTS project will apply different models that will be evaluated through simulation studies and tested on extensive data from a range of use cases provided by the industrial partners Roche and Novartis and Real World Data (RWD) from RD registries. 

ECRIN's role

ECRIN will be working together with its Czech node Masaryk University on dissemination activities via training and workshops. Two major stakeholders in the field of clinical trial methodology will be targeted, the clinical trial units (CTUs) and the regulators and health technology assessment (HTA) bodies.