LeadersLink Kapila Viges

Organization Profile

• Kapila Vygis is the Chief Executive of the MPN Research Foundation, a patient-founded entity dedicated to funding research for myeloproliferative neoplasms (MPNs).
• The foundation targets three primary MPN conditions: essential thrombocythemia, polycythemia vera, and myelofibrosis.
• Current estimates indicate approximately 300,000 patients in the U.S. are affected by these bone marrow cancers.
• The only existing curative treatment is a bone marrow transplant, which carries significant risks.

Program Engagement & Networking

• Vygis joined the LeadersLink program (focused on patient/healthcare data resources) to initiate a large-scale data collection initiative.
• The program facilitated peer connections with leaders of other patient-centered research foundations for ongoing support and knowledge sharing.
• Faster Cures provided access to mentors with expertise in building large registries and data science fields.
• Lead mentor David Panzier of the Helmsley Trust helped connect the foundation with reputable service providers.
• The foundation selected IQVIA as an experienced partner to develop a plan for achieving research goals.

Strategic Objectives for MPN Registry

• Primary Goal: Establish a large-scale, real-world evidence (RWE) registry to collect electronic medical records directly from consenting patients.
• Clinical Focus: Identify biomarkers and clinical signals to predict disease progression and distinguish patients likely to worsen versus those who remain stable.
• Trial Design: Collect evidence to define new clinical trial endpoints, specifically "progression-free survival," which currently does not exist as a validated standard in MPN trials.
• Stakeholder Approach: Adopt a multi-stakeholder design process incorporating patient perspectives, physician-scientist expertise, and industry/manufacturer interests.

Identified Challenges

• Data quality and usability concerns regarding the collection of medical records.
• Feasibility of capturing specific data points required to evaluate disease progression.
• Determining cost structures to ensure the sustainability of a large-scale data effort.

Outcomes & Forward-Looking Roadmap

• Established a multi-year roadmap for launching the real-world evidence registry.
• Adopted a phased approach to break down long-term goals into accessible short-term milestones, ensuring financial flexibility and adaptability.
• Prioritized balancing breadth of study with focus to avoid unfocused or unsustainable research scopes.
• Set immediate, valuable outcomes to iterate toward long-term research objectives.

Acknowledgments

• Vygis credited the Milken Institute and Faster Cures leadership, specifically Kristen Raymond and Katie, for the program structure, connections, and supportive environment.