When Every Patient Counts: A Fireside Chat on Literature-Derived RWE for Rare Indications

Mark Kiel, MD PhD, Genomenon’s Founder and Chief Scientific Officer, and Matthew Winton, PhD, Chief Commercial and Business Officer at CervoMed and former Chief Operating Officer at Inozyme, come together for a fireside chat on the role of literature-derived real-world evidence (RWE) in drug development for rare indications.

Mark and Matthew explore why the peer-reviewed literature remains a valuable and often underused source of RWE. They will dive into how it fits into rare-disease drug development, the practical challenges of generating literature-derived RWE at scale, and how its role is expected to evolve in the coming years.

Moving beyond just the definitions of RWE, this session will highlight the practical realities of leveraging the literature for rare-disease programs. Drawing on their extensive real-world experiences, this discussion will explore how literature-derived RWE differs from traditional sources such as EHRs, registries, and claims; how it can support trial design and other key decisions in rare indications; and what it takes to extract, standardize, and synthesize data from the literature into actionable evidence, leveraging AI and expert curation.

Viewers will learn how literature-derived RWE can systematically address evidence gaps in rare or complex indications enabling more rigorous decision-making across your programs.
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What You’ll Learn

• How literature-derived RWE differs from traditional sources in terms of coverage of rare diseases as well as data granularity, structure, and longitudinality.

• Practical ways literature-derived RWE can expand small rare-disease cohorts, refine inclusion/exclusion criteria, and inform endpoint selection.

• Where literature-derived RWE can add value across the lifecycle - from pre-IND hypothesis generation to post-approval label expansion.

• The real-world challenges of extracting and synthesizing data from the literature, and how AI plus expert curation can make that tractable.

• How to design literature-derived RWE so that it meets the expectations of regulators, investors, and internal stakeholders for scientific rigor and reliability.