Real-World Economic Evaluation of Advanced Therapy Medicinal Products After Market Access

Advanced Therapy Medicinal Products (ATMPs), including gene therapies, cell-based therapies and tissue-engineered products, are transforming the therapeutic landscape by offering the potential to treat, and in some cases fundamentally alter the course of, diseases that have historically been difficult to manage. As these innovative therapies move from clinical development into routine practice, their potentially curative or long-lasting effects, complex administration and uncertain long-term outcomes present significant challenges for healthcare systems, payers, clinicians and policymakers.While regulatory approval is primarily based on evidence of quality, safety and efficacy, decisions regarding continued use and equitable access increasingly require a broader understanding of the real-world clinical and economic value of ATMPs. At the time of market access, evidence may be limited by small patient populations, single-arm or relatively short clinical trials, immature survival data, surrogate endpoints and limited comparator information. Consequently, considerable uncertainty may remain regarding the durability of treatment effects, long-term safety, healthcare resource utilisation, quality of life and the overall economic value of these therapies.Real-world evidence generated after market access can play an important role in addressing these uncertainties. Patient registries, electronic health records, claims databases, long-term follow-up studies and other real-world data sources can provide evidence on treatment effectiveness, resource utilisation, patient-reported outcomes and healthcare costs in routine clinical practice. Such evidence can support the reassessment of cost-effectiveness and inform decisions concerning reimbursement, pricing, managed entry agreements and outcomes-based payment models.The economic evaluation of ATMPs also requires methodological approaches that reflect their distinctive characteristics. Conventional health economic models may not adequately capture the potential for long-term treatment benefit, uncertainty regarding durability, one-time or infrequent treatment costs, delayed adverse events, heterogeneous treatment responses and the consequences of potentially curative interventions. There is therefore a need for innovative approaches to modelling, uncertainty analysis, value assessment and the integration of clinical, economic and patient-centred outcomes.The proposed research topic, “Real-world economic evaluation of Advanced Therapy Medicinal Products after market access,” aims to bring together researchers, clinicians, health economists, regulators, industry representatives, policymakers, patients and other stakeholders to examine how the economic and clinical value of ATMPs can be evaluated in real-world settings following market access. We welcome original research, systematic and narrative reviews, methodological papers, policy and practice reviews, health economic evaluations and perspectives that provide new evidence, critical analysis or innovative approaches to understanding the value, affordability and sustainability of advanced therapies.Particular emphasis will be placed on research that bridges the gap between regulatory approval, reimbursement decisions and the demonstration of real-world value.By integrating perspectives from clinical practice, outcomes research, real-world evidence, health economics, pharmacoeconomics, regulatory science and health policy, this research topic aims to contribute to more robust and sustainable approaches to assessing ATMPs throughout their post-market lifecycle. The Topic is particularly relevant to the Frontiers in Pharmacology – Drugs Outcomes Research and Policies and Pharmacoeconomics, Drug Access & Value Assessment sections, given their focus on outcomes, economic evaluation, access and value assessment in real-world healthcare settings.Contributions may address, but are not limited to:- Real-world effectiveness and economic evaluation of ATMPs after market access- Long-term costs and consequences of ATMP treatment- Durability of treatment effects and its implications for economic models- Patient-reported outcomes, health-related quality of life and utility measurement- Post-market reassessment of cost-effectiveness and clinical value- Equity, affordability and patient access to ATMPs- Health-system preparedness and the economic impact of implementing ATMPs- Long-term safety, pharmacovigilance and their economic implications- Regulatory science, harmonisation and evolving international frameworks for post-authorisation evidence generation- Challenges in low- and middle-income countries and African health systems- Ethical, societal and policy considerations in the economic evaluation and allocation of ATMPs- Innovative approaches to evidence generation, including artificial intelligence, digital health and data-driven methodologies- Methodological challenges associated with small populations, immature evidence, surrogate endpoints and limited comparators- Pharmacokinetics and pharmacodynamics of ATMPs- Exposure-response to ATMPs- Biomarkers- Drug-ATMP interactions and immunogenicity (post-approval)Submissions consisting solely of isolated data-mining analyses using publicly accessible databases, without a clear contribution to the clinical, economic, methodological or policy evaluation of ATMPs, will not be considered. Topic editors: Vanessa Steenkamp, Motlalepula Gilbert Matsabisa Source: https://www.frontiersin.org/research-topics/85541/real-world-economic-evaluation-of-advanced-therapy-medicinal-products-after-market-access