细胞与基因治疗的长期随访未来保障：以患者为中心、数据驱动的方法

Future-Proofing Long-TermFollow-Up in Cell & Gene Therapy A patient-centered, data-driven approach DIEGO CORREA,VP and Global Head, CAGT Center of Excellence, IQVIAIAN BONZANI,PhD, Senior Principal Real World Technology Solutions, IQVIABARBARA ARONE,MSc, VP Medical Affairs, Real World Solutions, IQVIA Table of contents Introduction3Challenges in executing CAGT long-term follow-up studies3How to overcome LTFU challenges6Patient-centered, journey-driven design6Enriched data collection using real-world sources and registries8Digital and decentralized tools to reduce burden10Designing for adaptability and longevity12Future vision: Toward more efficient and scalable LTFU13Industry collaboration and shared registries13Seamless integration with routine care14Smarter analytics and AI14Lifecycle-based LTFU models14Regulatory leadership and advocacy15References16 Introduction Sponsors must balance stringent safety oversightwith the realities of patients’ lives post treatment.Traditional clinical trial approaches, anchored toshort-term site-based engagement, struggle under As cell and gene therapies (CAGTs) advancetoward curative potential, long-term follow-up(LTFU) has become a regulatory cornerstone forensuring sustained efficacy and safety. CAGTs carryuncertainties that only years of observation canresolve.1When a patient receives cell or gene therapy, the treatment marks the beginning of a long-term This paper is structured in three parts: first, we outlinethe key challenges in executing LTFU for CAGTs;second, we describe IQVIA’s current strategies toaddress these challenges; and third, we present afuture vision for scalable, patient-centered LTFU. Long-term patient follow-up is not just a regulatorycheckbox; it’s a scientific, operational, and ethicalimperative in the era of CAGTs.5Regulatory authorities Challenges in executing CAGT •Do the therapeutic benefits persist over time? •Are there delayed adverse effects that only emerge LTFU studies for CAGTs present a perfect storm ofoperational and scientific hurdles. Some of the major Addressing these questions is essential to validate thelong-term safety and efficacy of CAGTs and to uphold • Sustaining engagement over a decade or more.Keeping patients and investigators committedto a study for 5–15 years is inherently difficult.Patients who experience therapeutic success However, executing LTFU studies is inherently complex.Monitoring patients for a decade or more presentssignificant logistical and financial challenges. It is Sponsors must balance stringent safety oversight with the realities ofpatients’ lives post treatment. It’s clear that LTFU programs demanda modernized playbook that is patient-centered, data-rich, and •Data collection is fragmented and burdensome.LTFU means capturing a wide range of data acrossmultiple healthcare systems and providers overmany years. Unlike a controlled trial, where everydata point is collected per protocol, LTFU blursthe line with routine care: some data comes fromscheduled study visits, but the rest might come The result is high risk of attrition, e.g. missingdata, and lost patients, which can bias outcomes if those doing well are the most likely to be lostto follow-up.7Maintaining motivation and contact • Geographic dispersion and continuity of care.It’s common for rare disease patients to travel greatdistances to receive a one-time gene therapy at aspecialized center. After treatment, expecting themto travel back to that center regularly for years isoften impractical.6Patients resume their normal • Patients age and their health evolves. Over afollow-up period of more than ten years, a patient’scircumstances will inevitably change. New healthissues unrelated to the original disease can emergewhich could confound interpretation of long-termsafety or effectiveness signals. For example, if a Since CAGTs target rare diseases or niche cancers,LTFU studies already have limited sample sizes,leaving little room for missing data. Additionally,rare disease patients may not be captured withina single healthcare system or registry, making •Similarly, the effectiveness of a therapy may wane aspatients age or as the disease progresses naturally;distinguishing treatment failure from expected The challenge is how to “meetpatients where they are,” gatheringrequired data through healthcare • Technology and data access standards willchange midstream. Over the past five years, therehave been notable developments in digital healthtechnologies, connected devices, and tools usedacross consumer health, research, and healthcaredelivery. We are currently only beginning to see thepower of Artificial Intelligence (AI) when applied toremote monitoring systems. These changes haveoccurred alongside ongoing policy initiatives aimed opportunities or non-compliance with evolvingstandards. Thus, LTFU studies face a movingtarget and must be designed from the start with • Integrating trial and post-commercial follow-up.An often-over