Up to 36 million people in the EU live with one of the more than 7000 distinct Rare Diseases (RD). The evaluation of new medicines for RD and paediatric RD presents several challenges, such as small patient sample sizes, variability among patients and diseases, and differences in disease knowledge. As a result, access to effective treatments and the availability of treatment options for RD are often limited.
The project’s main goal is to provide clinical trial stakeholders, trialists and regulators with generalizable methods, designs and evidence assessment criteria for the future evaluation of treatments for RD and paediatric RD. This evidence-based framework will allow (i) clinical trials’ stakeholders to choose the most appropriate approach(es) for developing treatments for both well-investigated or less-studied RD, and (ii) help regulators make informed decisions about small sample evidence in RD with the help of evidence-tools, such as, workflows and guidelines.
This will be achieved through the development and validation of improved extrapolation models, simulation and in silico trials, model based clinical trial design and evidence synthesis methods, all based on robust and mature computational models and qualified on extensive data from representative selected use cases.
The INVENTS project was divided into 7 Work packages: 1. Improving robustness of model-based treatment effect estimation and extrapolation methods, 2. Developing in silico trials workflow using modelling and simulation, clinical trial and Real-World Data (RWD) to cope with missing knowledge, 3. Increasing robustness of small population confirmatory trials using validated and credible models, 4. Proposing evidence synthesis approaches using computational models, clinical studies, RWD and virtual cohorts, 5. Developing evidence-tools for regulatory decision-making in RD, 6. Integrating patient engagement and regulatory perspectives, and 7. Provide data from use cases to the INVENTS partners.
