Join Mark Kiel, MD PhD, Genomenon’s Founder and Chief Scientific Officer, and Matt Winton, PhD, Chief Commercial and Business Officer at CervoMed and former Chief Operating Officer at Inozyme, for a fireside chat on the role of literature-derived real-world evidence (RWE) in drug development for rare indications.
In this fireside chat, Mark and Matt will 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.
Attendees will learn how literature-derived RWE can systematically address evidence gaps in rare or complex indications enabling more rigorous decision-making across your programs.
What You’ll Learn
Who Should Attend
This fireside chat is designed for teams working in rare or complex indications, including:
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Dr. Mark Kiel is the co-founder and chief scientific officer at Genomenon, where he oversees the company's scientific direction and product development. Mark received his MD/PhD in Clinical Pathology at the University of Michigan. He founded Genomenon to address the challenge of connecting researchers with evidence in the literature to help diagnose and treat patients with rare genetic diseases and cancer.
Dr. Matthew Winton is the Chief Commercial and Business Officer at CervoMed, where he draws on nearly 20 years of global biotech experience to advance therapies for age-related brain disorders. He previously served as COO of Inozyme Pharma and spent almost a decade in senior commercial roles at Biogen, leading major neurology franchises and supporting the launch of SPINRAZA™, the first treatment for spinal muscular atrophy. Earlier in his career, he advised biotech and pharma companies on commercial strategy. Dr. Winton holds a Ph.D. in Neuroscience from the Université de Montréal, an MBA from Boston University, and completed postdoctoral training at the University of Pennsylvania.
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