Lessons from long-term follow-up of gene and cell therapies approved in the EU
Gene and cell therapies (GCTs) offer transformative potential for patients with rare and complex diseases, yet their long-term safety and efficacy remain under close regulatory scrutiny. In the European Union (EU), mandated long-term follow-up (LTFU) studies form a key component of post-marketing requirements, ensuring outcomes are monitored over extended periods. Understanding how these studies are designed is essential for researchers and developers planning future therapies, as it informs regulatory strategy and evidence generation approaches.
In this interview, Nahila Justo (Executive Director, PPD Evidera Real-World Data and Scientific Solutions, Thermo Fisher Scientific) discusses the research “Mandated long-term follow-up studies of patients treated with gene and cell therapies approved in the European Union (EU): An analysis of commonalities and variations” presented at the 2025 ISPE Annual Meeting, highlighting key trends, methodological insights, and implications for advancing long-term evidence generation.
Interviewee
Nahila Justo
Executive Director, PPD Evidera Real-World Data and Scientific Solutions, Thermo Fisher Scientific
Nahila Justo, PhD, MBA, MPhil, is Executive Director, PPD Real-World Data and Scientific Solutions, Thermo Fisher Scientific. She has over 15 years of experience in the industry where she designed and led numerous pharmacoepidemiology research programs including, but not limited to, comparative effectiveness and safety, drug utilization, and burden and cost of illness studies, leveraging diverse sources of data such as clinical registries, claim and administrative databases, retrospective chart reviews and EMR extractions, national surveys and other surveillance instruments. Dr. Justo has also led and conducted projects such as health-economics models, strategic consulting, stakeholder engagement and management, monitoring or regulatory developments and briefings, advocacy pieces, literature reviews and meta-analyses. Dr. Justo represents PPD in multistakeholder fora specialized in RWE (e.g., GetReal Institute, Duke Margolis Collaborative) and serves as an Expert Evaluator for the European Commission in various initiatives and programs, such as EU4Health and Horizon Europe Programmes, including the Innovative Health Initiative (formerly Horizon 2020 and Innovative Medicines Initiative).
Dr. Justo holds a PhD in Medical Science from the Karolinska Institute with a specialization in Epidemiology and Health Economics, an MBA from the Stockholm School of Economics, an MPhil in European studies from the University of Salamanca, an MSc in Economics from the Inter-American Development Bank and the University Torcuato Di Tella, and a BA and MA in Political Science and International Relations from the National University of Rosario.
Sponsorship for this Peek Behind the Poster was provided by Thermo Fisher Scientific.