State of the Ecosystem

Despite growing momentum, too few individualized N-of-1 trials have been completed—and with too little longitudinal follow-up—to establish a true gold-standard operational process across institutions. As more groups work to bring gene-targeted therapies to rare disease patients in need (leveraging available FDA guidances for individualized ASO development and other emerging regulatory pathways if appropriate), implementation practices remain highly variable across sites and geographies. This fragmentation drives unnecessary duplication of workflows, governance, and documentation, and it undermines the ability to harmonize and integrate N-of-1 data across studies.

Our Focus

The N1C Institutional Implementation Workgroup serves as a conduit and nucleus for sharing practical, real-world implementation learnings so institutions can launch and run N-of-1 efforts without “reinventing the wheel.” We collate recommendations and curate scrubbed example documents (e.g., protocols, SOPs, manuals, checklists, etc.), and we host open forums to exchange experiences and best practices across programs. Our goals are to develop a long-term “gold standard” implementation framework and to stay abreast of the evolving and globally varied regulatory landscape.