Volv Global is delighted to be attending the ISPOR International Annual Conference 2026 in Philadelphia, presenting new research conducted in collaboration with Sanofi on Pompe disease – a rare, chronically debilitating metabolic disorder where advances in treatment have fundamentally changed the clinical picture for patients, and where the evidence frameworks the field relies upon are still catching up.
Real-World Endpoint Mapping and Identification of Evolving Phenotypes of Pompe Disease using Machine Learning
As enzyme replacement therapy extends patient survival, those living with Pompe disease may experience new long-term manifestations that were not fully captured by clinical trial endpoints established earlier in its treatment history. At the same time, many clinically meaningful endpoints are difficult to observe directly in routine healthcare records, creating a gap between what patients experience and what the evidence base reflects.
Working with a large-scale US administrative claims database, this research used machine learning to assess whether pre-specified clinical endpoints could be reliably mapped to codes present in real-world data, and to identify novel disease manifestations in treated patients that pre-specified frameworks had not anticipated. The findings demonstrate that a data-driven approach can surface a richer, more complete picture of how Pompe disease manifests in the real world – with direct implications for natural history studies, endpoint selection, and the design of future clinical trials.
The full research will be presented at the ISPOR International Annual Conference 2026. This page will be updated with the research following the conference.
If you would like to meet our team at ISPOR 2026, please contact us to arrange a meeting.
About the ISPOR International Annual Conference 2026
ISPOR is the leading international forum for health economics and outcomes research, bringing together researchers, payers, regulators, and industry leaders to advance the science and practice of real-world evidence, value assessment, and health technology evaluation.
- Dates: 17–20 May 2026
- Location: Pennsylvania Convention Center, Philadelphia, PA, USA
- Find out more about the ISPOR International Annual Conference 2026
Links
- Volv Global: Value & Access, HEOR
- Volv Global: ATS 2025 – Alpha-1 Antitrypsin Deficiency research with Takeda
Frequently Asked Questions
What is ISPOR Global 2026? ISPOR Global is the world’s leading conference for health economics and outcomes research, uniting experts from pharma, payers, regulators, HTA bodies, and academia to share advances in real-world evidence, value assessment, and health technology evaluation.
What is Volv Global presenting at ISPOR 2026? Volv Global is presenting research conducted in collaboration with Sanofi, examining how machine learning can be used to map clinical endpoints to real-world claims data and identify evolving disease phenotypes in Pompe disease – a rare metabolic disorder where treatment advances have changed the clinical picture for patients over time.
Why does this research matter? As treatments extend patient survival, new unmet needs emerge and established evidence frameworks may no longer reflect the full reality of how a disease is experienced. This research demonstrates how a data-driven approach can close that gap, with implications for natural history studies, endpoint design, and clinical trial planning that extend well beyond Pompe disease.
How does this relate to Volv Global’s broader capabilities? This project reflects Volv Global’s expertise in applying machine learning to population-scale real-world data to generate evidence that informs the full pharmaceutical development lifecycle – from clinical strategy and trial design through to HEOR and lifecycle management.
Can I meet Volv Global at ISPOR 2026? Yes – please contact us to arrange a meeting in Philadelphia.
