What It Actually Means to Embed the Patient Voice in Trial Design

In a recent video interview with Applied Clinical Trials, Amélie Lothe, global medical community head for rare epilepsies at UCB Pharma, discussed what it truly means to embed the patient voice in clinical trial design—and why the distinction between genuine partnership and checkbox consultation matters enormously in rare and severe neurological conditions. She opened by framing patient and caregiver involvement not as a late-stage review activity but as a foundational input that should begin before endpoints are selected or protocols are written. In conditions like developmental and epileptic encephalopathies, Rett syndrome, and Dravet syndrome, she argued, families are the experts on what the disease actually feels like to live with—and that knowledge is irreplaceable.

On endpoints, Lothe was direct about the limitations of a seizure-centric view of success. While seizure frequency remains a well-established and regulatory-accepted measure, families living with conditions like CDKL5 deficiency disorder or Dravet syndrome describe a much wider disease burden—behavioral challenges, loss of communication and motor skills, disrupted family life, 24-hour supervision. Incorporating caregiver-reported quality of life measures, sleep and behavioral assessments, and caregiver burden scales alongside traditional seizure counts, she argued, is not optional if sponsors want their outcomes to reflect what families are actually hoping for.

Lothe traced the most consequential timing failure in patient engagement to the protocol feasibility stage—a point at which families are asked to react to decisions already made, rather than help shape them. Earlier engagement, she said, shifts patients from subjects to co-designers and improves both data quality and participant experience. She grounded that argument in firsthand learning from the epilepsy community, noting that feasibility is ultimately determined by the everyday realities of caregivers—travel burden, visit frequency, and appointment scheduling—not scientific ambition alone.

She closed by describing UCB's approach to building trust-based relationships with patient advocacy organizations, which she distinguished from transactional engagement around single trials. Joint working groups, structured surveys, listening sessions co-developed with organizations like the Dravet Syndrome Foundation, and a commitment to sharing results back in accessible language are all part of treating advocacy organizations as long-term partners in the science rather than stakeholders to be managed.