From Pre-competitive Collaboration to Regulatory-Ready Endpoints: A Bayer–DEEP Measures Case Study in Late-Onset Pompe Disease

About this session

Rare disease programs face a distinct measurement problem: small patient populations, few established endpoints, and very little room to get it wrong. Bayer and DEEP Measures will share how a focused effort helped move novel digital endpoints for late-onset Pompe disease closer to regulatory readiness. Representatives from Bayer and DEEP Measures will discuss: Why Bayer engaged in this work, the gap it was meant to close ahead of regulatory interactions, and why a systematic, evidence-first approach carries more weight in a rare disease context than in a large-population indication. How evidence was structured following the stack model in collaboration with a leading KOL. Next steps in the collaboration journey: pressure testing with patients and a wider group of KOL's, pre-competitive alignment, defining shared regulatory pathways and building complementary evidence.

Speakers

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  John Batchelor

  Speaker

• JK

  Julien Kroll

  Clinical Trial Technology Strategist · Bayer HealthCare

  Speaker