Dr. Cara O’Neill, the Foundation’s co-founder and Chief Science Officer, has been selected by the U.S. Food and Drug Administration (FDA) and the Clinical Trials Transformation Initiative (CTTI) as a representative for the Patient Engagement Collaborative (PEC).
O’Neill will serve a two-year term on the PEC, a collaboration of patient organizations and individual representatives established by FDA and CTTI in 2018 that discuss ways to enhance patient engagement. The group of 16 patients, caregivers, and patient group representatives meet with the FDA several times a year to discuss topics such as communication, transparency, and the best ways for patients to engage the FDA about medical product regulation.
Members of the PEC include patients who have personal disease experience, caregivers who have personal experience supporting someone with a health condition, and representatives from patient groups who have direct or indirect disease experience. The selection committee, which included patient advocates, FDA staff from multiple centers, CTTI staff and several outgoing PEC members, worked to identify individuals with diverse perspectives and experiences who could meaningfully contribute and express the patient voice.
FDA and CTTI collaborate to involve representatives with a variety of perspectives including patients, caregivers and representatives from diverse patient organizations and communities. The PEC is run by the FDA’s Patient Affairs, which is dedicated to providing an inviting, welcoming and meaningful experience for patient communities to engage with the FDA.
O’Neill has worked to get the Sanfilippo caregiver and patient voice heard by leading publication of the first-ever “Caregiver Preference Study for Sanfilippo Syndrome,” presenting to the FDA on mulitple occassions, and taking the lead in developing of the Sanfilippo community’s response to the FDA regarding its draft guidance on drug development for Sanfilippo Syndrome.