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Qualifying Biomarkers to Support Rare Disease Regulatory Pathways: Afternoon Sessions

On February 21 we convened "Qualifying Biomarkers to Support Rare Disease Regulatory Pathways," a day-long hybrid public workshop. This workshop explored primary disease activity biomarkers in rare genetic diseases using heparan sulfate in neuronopathic mucopolysaccharidoses (MPS) as a case study for a biomarker to support accelerated approval. Featured perspectives included FDA representatives, patient advocates, researchers, and representatives from regulated industry. Chapters: 0:00 Case Study: Animal Model Translation to Human Application 33:48 Case Study: Relationship Between Cerebrospinal HS Levels and Clinical Outcomes 1:17:35 Q&A Session with Afternoon Case Study Presenters 1:55:03 Panel Discussion: Challenges in Qualifying Biomarkers to Support Rare Disease Approvals 3:11:58 Closing Remarks