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Outdated research infrastructures and approaches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] Bayesian Methods Incorporating Baseline Covariates for Borrowing Historical Control Data in Randomized Clinical Trials: A Neutral Comparison Study

Bayesian Methods Incorporating Baseline Covariates for Borrowing Historical Control Data in Randomized Clinical Trials: A Neutral Comparison Study Publication The idea of leveraging historical control data in randomized clinical trials (RCTs) has gained a lot of attention, in particular for rare disease and pediatric indications where conducting traditional and fully powered RCTs can be challenging. [...]

[Conference] INVENTS at the ISBS2026 Conference

A successful Week in Madrid for the INVENTS Consortium at ISBS 2026 conference ! Last week, the INVENTS consortium made a significant impact at the International Society for Biopharmaceutical Statistics (ISBS) conference held in Madrid. The event provided a premier platform for our members to present cutting-edge research and engage with global leaders in biopharmaceutical [...]

[Publication] Overview of Regulatory Guidelines Related to Methods and Models for Small Populations

Overview of Regulatory Guidelines Related to Methods and Models for Small Populations Publication This paper provides an overview of approaches and related guidelines from the EMA, FDA, and ICH that are relevant for obtaining clinical evidence for small populations to support regulatory decision making. The approaches are grouped into three categories: (1) maximizing the attainable [...]