What was the event?

PMS mom and PMSF rep Anna Williams participated at an annual Epilepsy Foundation Research Roundtable in Washington D.C. on March 24th. The meeting is attended by industry leaders, medical experts, regulatory agencies which approve drugs,  patient advocacy representatives and families living with epilepsy. The goal is to improve diagnosis and treatment of epilepsies by addressing major barriers.

This year’s roundtable discussed the possibility of increasing remote assessments for clinical trials and other studies. This would involve bringing a medical professional to the homes of families to conduct tests to assess if a treatment is working, rather than having families travel to medical centers.

What did Anna say?

Many families face barriers to traveling due to medical complexity, scheduling conflicts, financial difficulty, and lack of support.

Anna advocated on behalf of her daughter, Lily, and PMS families everywhere. She shared the following points about her experience with travel for clinical studies:

• Her daughter experiences motion sickness, movement disorders and behaviors. The stress and travel has caused seizures and severe vomiting. Wouldn’t the outcome data collected for a study/trial be tarnished by the impacts of travel on her body?

• How impossible it feels to set aside time to engage in a clinical trial or study onsite when there are so many other medical and therapy appointments that take priority. (Anna created a graphic to help drive home this point).

• How much more effectively Anna can communicate with a provider or researcher via telehealth while her child is being cared for and she can focus on the questions/reporting at hand. This comes from direct experience with the NIH Natural History Study for Phelan McDermid Syndrome.

Why are at-home assessments important?

At-home assessments have the power to lower barriers for families to participate in clinical studies and reduce inequities. It may allow trials to be done faster, on more people, and with less infrastructure. All these things could improve the quality and number of clinical trials in epilepsy.

We are so thankful to Anna for being a fantastic voice for PMS families. We appreciate the time and effort it took for her to participate in this event. PMSF will continue to prioritize advocacy for remote assessments.

Findings from the roundtable will be published.

For more about the Roundtable, see: https://www.epilepsy.com/research-funding/research-roundtable