Beyond Compassionate Use: Building a Master Protocol to Advance Individualized Medicines at Scale скачать в хорошем качестве

Beyond Compassionate Use: Building a Master Protocol to Advance Individualized Medicines at Scale

Individualized medicines for patients with ultra-rare, life-threatening genetic conditions have historically been developed through singular, case by case efforts focused on one or few unique genetic variants. While this approach has enabled lifesaving treatments for individual patients, it has not generated the systematic evidence needed to support scalability, regulatory learning, or broader access to individualized therapies. This session will describe a collaborative model in which EveryONE Medicines and Great Ormond Street Hospital (GOSH) have developed a master protocol to advance the development of individualized medicines beyond one-off compassionate use. The first patient treated under the Master Protocol represents a critical milestone demonstrating the operational feasibility of this approach. This webinar will introduce the Master Protocol (EOM-MP1), a prospective clinical framework designed to enable standardized data collection and prespecified analyses across individualized antisense oligonucleotide (ASO) therapies for patients with unique genetic variants. By incorporating structured run-in periods, within-participant comparisons, and harmonized safety, biomarker, functional, and quality-of-life assessments, the protocol is designed to generate a robust evidence base to inform regulatory decision-making and support the advancement of individualized medicines at scale, while preserving the flexibility required for N-of-1 therapies.