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Outdated research infrastructures and approaches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 – Using Stochastic Simulation-Estimation and Automated Model Development to Assess Power and Accuracy for Covariate Identification
Using Stochastic Simulation-Estimation and Automated Model Development to Assess Power and Accuracy for Covariate Identification Publication When study designs are evaluated using clinical trial simulations for their ability to identify covariate effects in population pharmacokinetic (PopPK) modeling, it is typically assumed that the true model will be known at the data analysis stage. In this [...]
Conference – Strong presence of INVENTS at PAGE 2026 !
INVENTS well represented at PAGE 2026 ! The INVENTS consortium was strongly represented at PAGE2026, with several consortium members attending and contributing to the scientific program through poster presentations. The conference provided an excellent opportunity to showcase ongoing work across the consortium, exchange ideas with the broader research community, and strengthen collaborations around our shared [...]
Publication – Validating Physiologically-Based Pharmacokinetic Models Using the Continuous Ranked Probability Score: Beyond Being Correct on Average
Validating Physiologically-Based Pharmacokinetic Models Using the Continuous Ranked Probability Score: Beyond Being Correct on Average Publication Physiologically-based pharmacokinetic (PBPK) models have become increasingly popular for model-informed drug development (MIDD) over the past decade. While several guidelines for model evaluation exist, these are by design often of a general and non-specific nature. It is clear what [...]