Over the last several years, clinician-scientists, regulators, advocates, and industry partners have taken steps to facilitate the development of new efficacy endpoints for clinical trials in preterm neonates. The International Neonatal Consortium (INC), a C-Path/FDA consortium, has formed a working group focused on encouraging therapeutic development for bronchopulmonary dysplasia (BPD), which has been typically defined as the need for oxygen, with or without positive pressure respiratory support, at 36 weeks post-menstrual age (PMA) or “corrected” age. In clinical trials designed to improve pulmonary and respiratory outcomes for preterm neonates, the most commonly used endpoint has been BPD. However, there is no consensus on whether BPD is a clinically meaningful endpoint to predict pulmonary and respiratory outcomes at infancy, childhood, and beyond. Therefore, it can be challenging to encourage the development of products in this space because there is uncertainty over whether BPD is an optimal efficacy endpoint for regulatory submissions.
Efforts are underway to support the development of validated efficacy endpoints, including Clinical Outcome Assessments (COAs), that incorporate longer-term improvement or changes in pulmonary outcomes and are more data-driven. However, there are outstanding questions about how best to facilitate the development of such endpoints.
Under a cooperative agreement with FDA, the Duke-Margolis Center for Health Policy is convening this expert workshop to provide stakeholders the opportunity to explore and discuss the work that has been done to date on efficacy endpoint development for preterm neonates with pulmonary morbidities, highlight gaps and limitations in existing data and research, and identify a path forward to develop validated endpoints and COAs that better represent short- and long-term outcomes associated with pulmonary morbidities of prematurity.