Patient advocacy is where rare disease progress becomes accountable: it keeps innovation anchored to lived experience, focuses attention on equity, and accelerates the translation of science into better outcomes. In this Science/AAAS discussion from December, 2023, Volv Global’s Clinical Innovation Director Léon van Wouwe joined leaders across genomics, policy, and life-sciences strategy to explore how technology can shorten diagnostic odysseys, strengthen evidence, and improve access to the right care at the right time.
The webinar “Advocacy in rare disease: Driving technology advances” was sponsored by Fondation Ipsen and produced by the Science/AAAS Custom Publishing Office.
Panel:
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Léon van Wouwe (Volv Global)
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Heidi L. Rehm, PhD, FACMG (Massachusetts General Hospital)
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Karin Hoelzer, DVM, PhD (National Organization for Rare Disorders, NORD)
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Simon Alfano (McKinsey & Company)
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Erika Gebel Berg, PhD (Science/AAAS) – moderator
“Many of our patients have to go through a diagnostic odyssey of five to seven years… which is horrific for the patients and their families.”
– Karin Hoelzer, Senior Director, Policy and Regulatory Affairs, NORD
Rare diseases expose the limits of siloed data and fragmented pathways. As Karin Hoelzer (NORD) outlines, patients often face a 5–7 year diagnostic odyssey, with scarce natural history data, heterogeneous presentation, and decentralised systems that make patients hard to find and trials hard to run.
Léon was invited as a panel expert alongside leaders from Massachusetts General Hospital, NORD, McKinsey & Company, and Science/AAAS. His perspective speaks directly to a core, recurring gap in rare disease: even when science advances, systems struggle to reflect “true” patient populations, link signals across fragmented records, and translate insight into action. In the discussion, Léon describes his focus as building “bridges” from real-world data into clinical development and patient care – exactly the connective tissue required to move from breakthrough to benefit.
“I am looking to build bridges between real-world data through technology into clinical development and patient care.”
– Léon Van Wouwe, Clinical Innovation Director, Volv Global
For us at Volv Global, advocacy is not peripheral. It is part of what makes technology meaningful and responsibly applied. We see “democratisation of patient data” as ensuring that patient experience is represented in the evidence base (and that insights can be translated into fairer decisions), while still protecting privacy and respecting stewardship.
Volv Global and patient advocacy: “where technology meets human”
At Volv Global, we view patient advocacy as non-optional, because better data without patient context can still miss what truly matters. Our work aims to support the wider rare-disease community by helping close diagnosis gaps and strengthening evidence for change, while respecting privacy and data stewardship.
This philosophy is echoed in voices we’ve hosted and collaborated with:
