细胞与基因治疗的长期随访未来验证：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, IQVIAJIP KLERKS,MSc, Product Strategy Lead 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 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-termjourney. Regulators worldwide mandate that patientstreated with gene therapies are monitored for 5 to15 years to track delayed side effects and sustainedefficacy.2,3Tracking patients over extended periods canbecome costly and lead to operational challenges ifnot managed efficiently. With over 3,000 cell and genetherapies in development globally and dozens of newlaunches expected each year4, the stakes are high forcompanies to optimize the design of their LTFU studies. Sponsors must balance stringent safety oversightwith the realities of patients’ lives post treatment.Traditional clinical trial approaches, anchored toshort-term site-based engagement, struggle underthis scale of time and scope. It’s clear that LTFUprograms demand a modernized playbook that ispatient-centered, data-rich, and accommodateslong-term variability. 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.The overarching theme is thatby rethinkingfollow-up through the eyes of patients andleveraging real-world data and technology, we canensure these studies protect patients and advancescience while also being operationallyand financially sustainable. Long-term patient follow-up is not just a regulatorycheckbox; it’s a scientific, operational, and ethicalimperative in the era of CAGTs.5Regulatory authoritiesand pharmaceutical companies must be able to answercritical questions: Challenges in executing CAGTlong-term follow-up studies •Do the therapeutic benefits persist over time? •Are there delayed adverse effects that only emergeyears later? LTFU studies for CAGTs present a perfect storm ofoperational and scientific hurdles. Some of the majorchallenges that sponsors face when designing andimplementing these programs include: Addressing these questions is essential to validate thelong-term safety and efficacy of CAGTs and to upholdpublic confidence in these innovative treatments. • 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 successmay disengage, while those with poor outcomesmay drop out due to illness or discouragement.Investigators and site staff may lose interest oncethe trial’s active phase ends.6 However, executing LTFU studies is inherently complex.Monitoring patients for a decade or more presentssignificant logistical and financial challenges. It isalso often of limited interest to busy investigatorsand patients.6 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, andaccommodates long-term variability. •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 comefrom patients’ regular doctor visits or personalreports. Data can end up being fragmented acrossdifferent clinics, electronic health record systems,or registries.Furthermore, medical guidelines aresubject to continuous development and refinement.6The burden placed on sites and patients by repeateddata entry and extra visits can strain goodwilland resources if not managed carefully. All of thiscreates a significant data collection challenge: howto assemb