INNOVATIVE DESIGNS, EXTRAPOLATION,
SIMULATION METHODS AND EVIDENCE-TOOLS
FOR RARE DISEASES ADDRESSING REGULATORY NEEDS
The INVENTS program focuses on advancing the future evaluation of treatments and the regulatory decision-making for rare diseases (RD) including paediatric rare diseases.
About rare diseases
European key figures
Up to
36 million
people are concerned in the EU
95%
of rare diseases
have no treatment option
There is more than
6000
distinct rare diseases
70%
of genetic rare diseases
starts in childhood
Challenges
Future assessments for rare diseases treatments face challenges :
A lack of clinical trials and sufficient evidence to decide for treatment
The heterogeneity of rare diseases
Outdated research infrastructures and approaches
In a 5 years project, INVENTS aims to enhance decision-making by incorporating patient experiences, creating in silico evaluation models from clinical trial or real-world data, and developing regulatory decision support tools for regulatory agencies.
Ambition
INVENTS’ framework will enhance drug development, treatment safety and robustness in rare diseases.
News
Publication – In Silico Clinical Trials in Drug Development: A Systematic Review
In Silico Clinical Trials in Drug Development: A Systematic Review Publication In the context of clinical research, computational models have received increasing attention over the past decades. In this systematic review, we aimed to provide an overview of the role of so-called in silico clinical trials (ISCTs) in medical applications. Exemplary for the broad field [...]
Publication – Addressing Causality and Homogeneity Assumptions in Exposure-Response Analyses
Addressing Causality and Homogeneity Assumptions in Exposure-Response Analyses Publication Exposure-response, or pharmacokinetic–pharmacodynamic (PKPD), analyses support many drug development decisions. It is typically applied without assessment of causality and homogeneity, where the latter refers to the assumption that the reason for variability in exposure is unimportant for the impact on response. Randomized dose is the ideal [...]
INVENTS at the World Orphan Drug Congress Europe 2025 !
INVENTS at the World Orphan Drug Congress Europe 2025 ! On October 27th 2025, INVENTS members took part in the pre-conference workshop at WODC2025 in Amsterdam, organized by ERDERA. Sarah Zohar, Vincent Damotte, Laurens Sluijterman presented the objectives of the project, with a particular focus on the methods, data sharing strategy, and the evaluation framework [...]