Pillar 4 supports researchers and clinicians to turn knowledge into true patient benefit for the rare diseases community. To do this, research projects will be provided with expert mentoring, and those with promising results will be selected for their translational potential and specifically supported in further developing their plans. Importantly, Pillar 4 will also foster the implementation and development of innovative statistical design and analysis methodologies tailored for clinical studies in rare diseases. This specifically will include supporting ERNs to use the most adapted methodologies improving clinical trials in rare diseases.
Pillar 4 will improve rare diseases research and development by tackling the following challenges:
- Translating knowledge into patient benefit is a long, complex and risky process – academia is often not well equipped to navigate the technical and regulatory processes involved in translation
- Clinical research on small populations has particular methodological challenges – while there are high potential methodologies in need of validation and dissemination
The key outputs from Pillar 4 are:
- Promising academic research will have higher chance of being translated into the clinic for real patient benefit, in the form of new diagnostics and medicines
- Disseminate innovative statistical design and analysis methodologies tailored for clinical trials in rare diseases to the rare diseases research community by validation of the methods to practical problems
- Develop innovative statistical methodologies for tailored rare diseases clinical trials
The goals will be achieved by:
- Providing expert mentoring to project teams performing high quality translational research
- Creating a toolbox of guidelines, best practices and other resources for researchers to perform translational research more confidently
- Support successful projects in finding further funding for the next stage of development
- Under the auspices of an expert Task Force select high potential study methodologies, to be validated in collaboration with the ERNs, using their existing datasets for validation
- Dissemination through a broad push in EJP and beyond for community uptake of successful methodologies.
- Implementing a Task Force for identification of most promising areas for demonstration and development of innovative statistical methodologies.
- Launching 6 demonstration projects for implementing the most attractive methodologies to practical situation and 6 development projects for mostly needed statistical methodologies.