In pediatric epilepsy, including rare epilepsy and developmental epileptic encephalopathies (DEEs), complex issues require strategic decisions to enhance clinical trials. DEEs significantly impact the patient, family, and caregivers’ quality of life and, in some cases, result in premature mortality. Despite the rare designation for many conditions, they collectively impact five in every 10,000 children, highlighting the need for effective novel interventions and considerations to address this critical unmet medical need.
Read our white paper for trial design strategies to enhance your outcomes, including:
- Achieving sufficient enrollment despite significant disease heterogeneity
- Working with patient advocacy groups to address retention and protocol adherence
- Ethical and practical factors for consenting minors and their caregivers
