Medable Inc. has introduced a digital-first Long-Term Follow-Up (LTFU) model designed to tackle the significant patient dropout challenges plaguing cell and gene therapy (CGT) trials. The platform addresses the FDA-required 15-year monitoring period for certain CGTs, including those using genome-editing techniques like CRISPR-Cas9, where traditional follow-up approaches have proven inadequate for maintaining patient engagement.
Addressing Critical Dropout Rates in Advanced Therapies
According to a 2024 survey conducted by the Emily Whitehead Foundation and Catalyst Healthcare Consulting, 20% of CAR T-cell therapy patients stop participating in long-term follow-up altogether, with 80% of that group ceasing participation at or after five years post-treatment. The survey identified distance to sites, lack of awareness about local follow-up options, and burdensome travel as key barriers to continued participation.
"Long-term follow-up is essential to ensure the durability and safety of advanced therapies, but the current system isn't working for patients or sponsors," said Dr. Pamela Tenaerts, Chief Medical Officer at Medable. "With our new LTFU model, we're reimagining how post-treatment data is captured - putting patients at the center while ensuring scientific and regulatory rigor."
Digital-First Solution for Complex Monitoring Requirements
The extended 15-year monitoring period is essential for tracking delayed adverse events, understanding therapy durability, and ensuring long-term patient impact. However, traditional LTFU models impose significant logistical and financial challenges on participants, contributing to lower participation and high attrition rates over time.
Medable's digital-first LTFU offering includes four key components:
Remote and Hybrid Patient Interaction: Patients can engage in follow-up through flexible virtual, in-person, or blended formats that meet their individual needs and life circumstances.
Seamless Transition from Parent Trials: Integrated workflows and data continuity ensure a frictionless handoff from interventional studies to long-term observational follow-up.
Patient-First Data Capture and Adherence Monitoring: The platform leverages mobile technology, patient-reported questionnaires, and local healthcare providers to track key endpoints and ensure consistent participation over time.
Personalized Patient Communication: Ongoing engagement through tailored content, reminders, and support tools to build trust and maintain long-term connection.
Addressing Industry-Wide Challenges
CGTs offer transformative potential yet require prolonged safety monitoring, often spanning 15 years post-treatment. By embracing a digital, patient-centric approach, Medable's model addresses key gaps in traditional LTFU execution, improving retention, increasing diversity, and reducing the long-term cost burden for both sponsors and sites.
The platform reduces the burden on patients and sites involved in complex CGT trials while enhancing scientific integrity with sustainable data capture across the FDA-required monitoring period. This approach is particularly relevant for therapies using advanced techniques like CRISPR-Cas9, where long-term safety data is crucial for regulatory approval and clinical adoption.
Medable will showcase its new digital-based LTFU model at the American Society of Clinical Oncology's (ASCO) 2025 Annual Meeting from May 30-June 3 in Chicago at Booth #10105.
