This is a recording of the webinar held on April 29, 2024.

This is a conversation about a promising clinical trial model for new epilepsy treatments that accelerates access to the wider community of DEEs. A “basket trial” design is used in clinical trials for medicines aiming to treat symptoms of those living with DEEs, including seizures. This broad approach is different than the current standard clinical trial design singling out individual disorders, and would mean increased access to clinical research for a wider range of families interested in participating. With this more inclusive model, treatments approved using this method could include DEEs in the “label” (i.e. approved by the FDA for use), reducing the need for families to have to fight with doctors and insurance companies for the use of drugs off-label.

Joining us for this discussion about this new model in clinical trials for epilepsy treatments was Randall Kaye, Chief Medical Officer at Longboard Pharmaceuticals who has applied this model in their innovative Phase I/II PACIFIC trial design, which was inclusive of all DEEs.

This webinar was also held in partnership with the Sjeldent epilepsinettverk, represented by Christina SanInocencio, the current REN Chair.

DEE-P Connections is actively advocating with the FDA to be more supportive of pharmaceutical companies in utilizing this basket trial model and we welcome more families to help speak out about the need to accelerate improved treatments for the dozens of rare epilepsies. Reach out if you want to get involved – info@deepconnections.site