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Outdated research infrastructures and approaches 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

INVENTS Consortium Holds Successful First Annual Meeting in Göttingen

The INVENTS consortium gathered at the Historic Observatory - Georg-August Universität Göttingen, Germany, for its first annual consortium meeting from November 14th-15th, 2024. This event offered a great platform for reviewing progress, sharing knowledge, and fostering collaboration. Pre-Meeting Workshop Before the main event, participants attended a specialized workshop on “External Controls for Clinical Trials,” expertly [...]