RealiseD

The aim of new IHI project RealiseD is to change the paradigm for clinical trials in ultra-rare diseases. By bringing together all stakeholders, including clinicians, methodologists, pharmaceutical industry, patients, regulators and HTA body, RealiseD will catalyse the development and acceptance of innovative approaches for designing trials, (re-)using, analysing and interpreting data. These resources will be incorporated into easy-to-use playbooks and digital tools designed to facilitate the set-up and conduct of innovative trials focusing on white spots (conditions for which there is no approved treatment option and where development is not currently commercially viable).

Operational Strategies:

- Developing systematic patient referral procedures.
- Certifying clinical sites and addressing gaps in conducting U(RD) clinical trials, using 4 diseases from 4 European Research Networks (ERNs) as case studies.

Methodological Innovation:

- Optimising statistical and quantitative methods for U(RD)-CT design and analysis refined over the last decade.
- Streamlining the regulatory pathway for drug development through these approaches

Collaborative Framework:

- Engaging regulators, HTA bodies, and other stakeholders in a public-private consortium to co-create methodologies and operational frameworks through iterative processes.
- Reducing uncertainties in U(RD) drug development to incentivise pharmaceutical industry involvement.

Global Impact and Sustainability:

- Leveraging international efforts to address challenges in U(RD) drug development beyond geographical boundaries.
- Creating and deploying playbooks to ensure visibility, readiness, and stakeholder acceptance of innovations.

ERN EpiCARE Involvement:

Carmen Fons from Hospital Sant Joan de Deu and Rima Nabbout from Hôpital Necker AP-HP are participating in:

WP 2: Clinical Use Case in the group focusing on pediatric Developmental and Epileptic Encephalopathies
WP3: Certified Clinical site.

Goal:

Ensure real-world applicability and transferability of outcomes and methodologies for broader use in rare epilepsies.
Integration of new measures and methodologies into a comprehensive playbook
Mapping of the EU research centres and national directives

The ERN EpiCARE is also involved in the WP 11 on External Communications and Dissemination.

RealiseD

Transforming Clinical Trials for Ultra-Rare Diseases

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Sébile Tchaicha

For more information, please do not hesitate to contact Sébile Tchaicha, the ERN EpiCARE Research project manager ([email protected])