Medable unveils a patient-first remote model to support 15-year safety follow-up and reduce attrition in gene-edited therapies

Cell and gene therapies (CGTs) are reshaping how some of the most challenging diseases are treated, but extended follow-up demands have become a weak point in clinical research. Medable has launched a new digital-first long-term follow-up (LTFU) model designed to address a growing need for patient retention and data integrity over the 15-year FDA-required observation period for some advanced therapies.

The company says the platform was created to alleviate both logistical and financial pressures that patients face during LTFU, particularly those receiving treatments involving genome-editing technologies such as CRISPR-Cas9. These burdens are frequently cited as reasons for declining participation as trials progress into later years.

A 2024 survey conducted by the Emily Whitehead Foundation and Catalyst Healthcare Consulting found that one in five CAR T-cell therapy patients stopped participating in long-term monitoring altogether. Among them, 80% withdrew after five years of follow-up. Key reasons included difficulty traveling to trial sites, a lack of accessible local options, and limited communication.

Medable’s Chief Medical Officer, Dr Pamela Tenaerts, said the current system is falling short for both participants and sponsors.

“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,” she said.

“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.”

The new model integrates remote and hybrid patient engagement, giving participants the flexibility to choose virtual, in-person, or blended follow-up methods. The system also includes tools for adherence monitoring, digital communication, and a seamless transition from the original clinical trial to long-term observation.

Rather than relying solely on trial sites, the model incorporates local healthcare providers and mobile-based patient reporting to reduce the burden of travel and improve long-term compliance. Digital engagement tools provide personalized support and reminders to encourage consistent participation.

According to Medable, this patient-centric structure is designed to increase retention, reduce costs, and support better data quality across a trial’s full lifespan. The system supports regulatory compliance while aiming to improve equity in participation, especially for individuals in rural or underserved areas who are often excluded from extended follow-up.

The move aligns with a broader industry push to decentralize clinical trials and extend digital transformation into post-approval and observational phases of drug development. As CGTs continue to evolve, sustainable models for long-term monitoring will be critical for validating safety and efficacy over time.