Project description
Innovative designs and tools for rare disease medicines evaluation
Assessing the efficacy and safety of new medicines for rare diseases, particularly paediatric conditions, poses several challenges. These include small patient sample sizes, patient and disease heterogeneity, and limited disease knowledge, which hinder effective treatment access and limit available options. In this context, the EU-funded INVENTS project seeks to refine longitudinal model-based disease trajectories, improve extrapolation models, and implement in silico trials, considering patient’s needs, to enhance regulatory decision-making. The project’s outcomes are expected to enable researchers, regulators and the European pharmaceutical industry to leverage innovative clinical trial designs, in silico trials, and real-world data analysis approaches. Patients with rare diseases stand to benefit from increased and expedited access to effective and safe treatments.
Objective
The evaluation of new medicines for rare diseases (RD) including rare 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 in RDs.
INVENTS aims to provide clinical trial trialists, researchers and regulators with a global framework encompassing methods, workflows and evidence assessment tools to be implemented in orphan and paediatric drug development. Our ambition is to significantly improve the evaluation of evidence and regulatory decision-making through the development and validation of: refined longitudinal model-based diseases trajectories and treatment effect, improved extrapolation models, in silico trials (e.g. virtual patient cohorts), optimised model-based clinical trial designs and evidence synthesis methods. These will be evaluated through simulation studies and tested on extensive data from a range of use cases provided by our industrial partners Roche and Novartis and Real World data (RWD) from RD registry. The INVENTS framework will improve consistency and efficiency of the drug evaluation process for RD by augmenting clinical evidence without compromising its scientific integrity and providing regulators assessment credibility criteria.
At the end of this 5 years project, the European industry will be able to exploit novel and improved clinical trial designs, in silico trials and RWD analysis approaches supporting drug development in RD. The European Medicine Agency and European national regulators (including Health Technology Assessment bodies) will be supplied with a general framework allowing better informed decision-making. Most importantly, RD patients will benefit from an increased and faster access to efficacious and safe treatments.
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HORIZON-RIA - HORIZON Research and Innovation ActionsCoordinator
75654 Paris
France