BioDrugs最新文献

{"title":"Targeting CD20 for B-cell Depletion in Autoimmune Kidney Disease: Next Generation.","authors":"Federico Yandian, Sanjeev Sethi, Fernando C Fervenza, Fernando Caravaca-Fontán","doi":"10.1007/s40259-025-00742-0","DOIUrl":"https://doi.org/10.1007/s40259-025-00742-0","url":null,"abstract":"<p><p>Anti-CD20 monoclonal antibodies are gaining clinical relevance in the nephrology community due to their demonstrated efficacy and favorable safety profiles across short-, medium-, and long-term use. Initially developed for hematologic malignancies and multiple sclerosis, B-cell depletion therapies are now being investigated across a broader spectrum of autoimmune diseases, including glomerulopathies, both with and without associated podocytopathy. Recent advances have led to the development of novel anti-CD20 agents that are being used not only as potential alternatives to corticosteroids but also as adjunctive therapies in complex clinical settings. However, their efficacy is not uniform across all conditions, whether used for induction therapy, relapse management, or as rescue treatment following first-line therapy failure. A thorough understanding of their mechanisms of action, along with the potential for resistance and therapeutic failure, is essential for advancing precision medicine in this field. This review provides a molecular and clinical overview, incorporating pharmacokinetic and pharmacodynamic insights into the most widely used and emerging anti-CD20 monoclonal antibodies in nephrology. It serves as a practical guide to understand how these agents' function, why they may fail, and what alternative strategies should be considered in cases of adverse reactions or inadequate response. Finally, it highlights their evolving role in precision therapy, both as monotherapy in podocytopathies and as part of a multi-targeted treatment approach for glomerular diseases with systemic involvement.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":""},"PeriodicalIF":6.9,"publicationDate":"2025-09-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145013790","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Bispecific Antibodies in Hematologic Malignancies: Attacking the Frontline.","authors":"Toral Shastri, Asaad Trabolsi, Artavazd Arumov, Jonathan H Schatz","doi":"10.1007/s40259-025-00735-z","DOIUrl":"10.1007/s40259-025-00735-z","url":null,"abstract":"<p><p>Since blinatumomab's approval as the first bispecific antibody (BsAb) in cancer therapy, these immunomodulatory agents have achieved substantial success in lymphoid malignancies. A decade after provisional approval in relapsed settings, blinatumomab became part of first-line induction therapy for patients with B-cell acute lymphoblastic leukemia (B-ALL). Now, six additional BsAbs have FDA approvals for the treatment of B-cell non-Hodgkin's lymphomas and multiple myeloma (MM), achieving high response rates in otherwise refractory scenarios. In lymphoma, epcoritamab, glofitamab, and mosunetuzumab show proof-of-principle for complete remission (CR) without chemotherapy or cell-based treatment. Single-agent remissions do not appear durable, but fortunately, these immunotherapies are readily combined with other treatment modalities. Therefore, their true potential to contribute to cures may be close on the horizon owing to ongoing and future trials. In MM, teclistamab, talquetamab, and elranatamab achieve impressive CR rates in the relapsed setting and similarly, are being investigated in earlier line combinations and in precursor entities such as smoldering myeloma and monoclonal gammopathy of undetermined significance (MGUS). With a unique mechanism of action and continued testing in earlier lines, BsAbs are poised to be among the winners in the race to the frontline treatment of hematologic malignancies.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"793-814"},"PeriodicalIF":6.9,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12354547/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144764437","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Analytical Data and Single-Dose PK are Sufficient to Conclude Comparable Immunogenicity for Biosimilars: An Ustekinumab Case Study.","authors":"Martin Schiestl, Nivedita Roy, Michael Trieb, Joseph P Park, Elena Guillen, Gillian Woollett, Elena Wolff-Holz","doi":"10.1007/s40259-025-00733-1","DOIUrl":"10.1007/s40259-025-00733-1","url":null,"abstract":"<p><p>Comparative immunogenicity is a key regulatory requirement for biosimilars. In a streamlined biosimilar development process, absent a comparative clinical efficacy study, the analytical data and clinical pharmacokinetics (PK) study need to provide sufficient evidence for a conclusion of comparable immunogenicity. In this case study, we have reviewed the role of analytical and clinical data in the immunogenicity assessment of all currently available ustekinumab biosimilars and their reference product. Public information for European Medicines Agency-and US Food and Drug Administration-approved biosimilars reveal that the single-dose clinical PK studies were sensitive in detecting differences in terms of immunogenicity between the biosimilar and the reference product, a finding that was replicated in the comparative clinical efficacy studies. The rates for anti-drug antibodies and neutralizing antibodies were comparable albeit numerically lower for all biosimilars compared to their reference product, which correlates with lower levels of non-human glycans such as α-1,3 galactose known to be potentially relevant for immunogenicity. Our study demonstrates that the single-dose clinical PK studies were sensitive in confirming comparable immunogenicity of ustekinumab biosimilars with their reference product. The comparative clinical efficacy studies revealed no additional information. This finding adds on to the evidence that clinical PK and the comparative analytical assessment, specifically the comparison of quality attributes with potential immunogenic relevance, suffice for the evaluation of immunogenicity of biosimilars in general.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"769-776"},"PeriodicalIF":6.9,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12354544/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144658337","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Influence of Pharmacokinetic-Pharmacodynamic Failure Mechanisms on Outcomes in Biologic Therapy Sequencing in Inflammatory Bowel Disease: A Retrospective Cohort Study.","authors":"Casper Steenholdt, Ruben Due Lorentsen, Jon Henneberg, Pernille Nørgaard Petersen, Jørn Brynskov","doi":"10.1007/s40259-025-00730-4","DOIUrl":"10.1007/s40259-025-00730-4","url":null,"abstract":"<p><strong>Background: </strong>Increasing therapeutic options for inflammatory bowel disease calls for tools to aid choice of sequencing. We investigated if pharmacokinetic (PK) and pharmacodynamic (PD) failure mechanisms prompting therapy change influenced subsequent outcomes when switching to a different biologic drug class.</p><p><strong>Methods: </strong>Retrospective single-center cohort study including patients treated first with tumor necrosis factor (TNF) inhibitors, followed by vedolizumab, and then ustekinumab. Clinical and objective disease remission were conventionally classified by validated indices. PK-PD failure was defined according to maintenance drug concentrations (PK below thresholds and PD above thresholds): infliximab 8.0 µg/mL, adalimumab 12.0 µg/mL, golimumab 1.4 µg/mL, vedolizumab 15 µg/mL. Primary treatment failure despite dose intensification was ascribed to PD. Primary endpoints were steroid-free treatment persistence and 1-year remission.</p><p><strong>Results: </strong>The study included 112 patients switching from TNF inhibitors to vedolizumab (infliximab n = 61, adalimumab n = 32, golimumab n = 16, certolizumab pegol n = 3) and 31 subsequently to ustekinumab. Treatment persistence on vedolizumab did not differ between patients discontinuing TNF inhibitors due to PK (n = 28, 31%) or PD (n = 63, 69%) (mean 989 days [95% confidence interval: 554-1424] vs. 951 [659-1242], p = 0.93). One-year steroid-free clinical and objective remission rates on VDZ were also comparable between PK-PD groups (29% vs. 35%, p = 0.63 and 35% vs. 43%, p = 0.48, respectively). Findings for UST were similar. Sensitivity analyses with exclusion of primary non-responders and multivariate analyses correcting for potential confounders supported findings.</p><p><strong>Conclusion: </strong>PK-PD failure mechanisms do not appear to influence subsequent treatment outcomes when switching to biologics with different modes of action. Sequencing may rather rely on aspects such as efficacy, safety, and costs.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"725-734"},"PeriodicalIF":6.9,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12354565/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144324450","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Lipid-Lowering RNA Therapeutics for Atherosclerotic Cardiovascular Disease Prevention: A State-of-the-Art Review.","authors":"Samuel D Maidman, Robert S Rosenson","doi":"10.1007/s40259-025-00731-3","DOIUrl":"10.1007/s40259-025-00731-3","url":null,"abstract":"<p><p>Despite the modern era of effective and safe high-intensity statins and non-statin agents, a significant portion of patients are still unable to achieve guideline-recommended lipid goals for the prevention of atherosclerotic cardiovascular disease (ASCVD) events. Accordingly, novel strategies are needed to further mitigate residual risk for patients on the background of maximally tolerated lipid-lowering therapies. The past decade has seen an explosion of new agents leveraging ribonucleic acid (RNA)-based technology which reduce plasma lipoprotein levels. In this state-of-the-art review, we examine the ongoing clinical development of lipid-lowering RNA therapeutics. We discuss the efficacy and safety profiles of antisense oligonucleotides and small interfering RNA agents targeting low-density lipoprotein, lipoprotein(a), and triglyceride-rich lipoproteins. We also present challenges future clinical trials must answer to prove RNA therapeutics as a viable strategy for ASCVD prevention among patients with refractory hyperlipidemia.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"753-768"},"PeriodicalIF":6.9,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144483110","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comprehensive Physico-chemical and Functional Similarity Assessment Study of RGB-14-P and RGB-14-X Drug Products as Proposed Biosimilars to Denosumab Reference Products.","authors":"Ágnes Szonja Garai, Dániel Hüse, Ádám Fizil, Zsolt Zólyomi, Gábor Lehoczki, Andrea Kis, Zsuzsanna Kálmán-Szekeres, Viktor Háda","doi":"10.1007/s40259-025-00738-w","DOIUrl":"10.1007/s40259-025-00738-w","url":null,"abstract":"<p><strong>Background and objective: </strong>Denosumab is a fully human monoclonal antibody (IgG2) k subclass that targets and binds with high affinity and specificity to receptor activator of nuclear factor-κB ligand (RANKL). Gedeon Richter's denosumab RGB-14-P and RGB-14-X are proposed biosimilar drug products to the reference medicinal products Prolia^® and Xgeva^® (marketing authorisation holder: Amgen Europe B.V. in the European Union [EU] and Amgen Inc. in USA, respectively). The present study demonstrates the structural, physico-chemical and functional similarity between RGB-14 and reference drug products marketed in the EU and US.</p><p><strong>Methods: </strong>Using an extensive, state-of-the-art analytical and functional panel of 38 methods ensured the comprehensive characterisation of the biosimilar and reference drug products. To assess biosimilarity, physico-chemical and biological functional tests were performed using multiple orthogonal techniques, in addition to the in-depth comparison of the primary and higher-order structures of the therapeutic proteins.</p><p><strong>Results: </strong>It has been demonstrated that the primary and higher order structures of RGB-14-P and RGB-14-X drug products are identical or highly similar to those of EU/US Prolia^® and Xgeva^®. The purity profiles of the biosimilar and reference products were similar. Only minor differences were observed in glycosylation patterns and charge variant profiles. A wide range of bioassays was used demonstrating similarity in terms of potency, ligand and receptor binding. Additionally, during comprehensive analysis of the reference product data as the function of expiry dates, shifts were revealed in certain quality parameters, although these did not impact the biological activity of the products.</p><p><strong>Conclusion: </strong>The extensive analytical and functional similarity assessment study provides robust evidence that the structure and function of RGB-14-P and RGB-14-X are highly similar to those of EU/US Prolia^® and Xgeva^®.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"697-724"},"PeriodicalIF":6.9,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12354141/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144788212","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Individual Prescribing Incentives for Biosimilars in Selected European Countries and the USA: a Scoping Literature Review.","authors":"Félix Lobo, Isabel Río-Álvarez, Ángeles Flores","doi":"10.1007/s40259-025-00736-y","DOIUrl":"10.1007/s40259-025-00736-y","url":null,"abstract":"<p><p>The uptake of biosimilar medicines in Europe and the USA remains highly variable and at times slow, despite the significant potential for cost savings for both patients and healthcare systems. One of the most recommended measures to address this issue is the use of prescribing incentives. On the basis of a well-defined concept of individual prescribing incentives, we conducted a scoping literature review aimed at exploring their role in promoting the uptake of biosimilars in six countries with advanced healthcare systems (the USA, Denmark, England, Italy, France and Germany), with a particular focus on gain-sharing initiatives. Online databases and other sources were used to identify papers published between 2010 and 2023, resulting in the selection of 47 publications. The results suggest that there are few real-world programmes that use provider incentives offered by health systems to encourage prescribing of biosimilars. However, we found gain-sharing schemes of particular interest in England, Italy, France and Germany, where savings are reinvested to improve the quality of care, incentivizing physicians and raising satisfaction, but without financial rewards. In contrast, we found unplanned disincentives hindering the uptake of biosimilars in the USA, as well as very successful top-down strategies that do not rely on individual incentives, including centralized procurement in Denmark, although it remains to be seen whether the success is idiosyncratic to its specific circumstances. In addition, the hypothesis that gain-sharing initiatives with the aforementioned characteristics are more adaptable to different cultural, organizational and political settings to promote biosimilar prescribing merits further research.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"777-791"},"PeriodicalIF":6.9,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144764439","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comprehensive Physico-Chemical and Functional Similarity Assessment of Intravenous and Subcutaneous RGB-19 Drug Products as Proposed Biosimilars to Tocilizumab Reference Product.","authors":"Katalin Solti, Sarolta Timári, Tamás Faludi, Attila Iliás, Rózsa Hegedüs, Zoltán Pataj, Viktor Háda","doi":"10.1007/s40259-025-00734-0","DOIUrl":"10.1007/s40259-025-00734-0","url":null,"abstract":"<p><strong>Background and objective: </strong>Tocilizumab is a recombinant, humanised monoclonal antibody of the immunoglobulin G1 (IgG1) subclass, which specifically targets the interleukin-6 receptor (IL-6R). The RGB-19 product was developed as a biosimilar to the reference medicinal product RoActemra^® (authorised for use in the European Union [EU]). The current study focuses on the demonstration of structural, physico-chemical and functional similarity between RGB-19 (intravenous [IV] and subcutaneous [SC] presentations) and EU-sourced RoActemra^® (IV and SC presentations).</p><p><strong>Methods: </strong>The RGB-19 biosimilar tocilizumab product was comprehensively tested using an extensive state-of-the-art analytical and functional panel of 44 methods to demonstrate similarity to the EU-sourced RoActemra^®. Biosimilarity was rigorously evaluated through an extensive array of orthogonal physico-chemical and functional assays, supplemented by a detailed comparative characterisation of the primary and higher order structures of the therapeutic proteins.</p><p><strong>Results: </strong>Extensive structural analyses confirmed that the primary and higher order structures of tocilizumab proteins in RGB-19 IV and SC drug products are identical or exhibit a high degree of similarity to those of the RoActemra^® reference products. The impurity profiles of RGB-19 and RoActemra^® products were found to be highly comparable, as demonstrated by a series of physico-chemical techniques. A high level of similarity was shown for the most critical (soluble IL-6R binding and cell-based anti-proliferation assay) and for all other bioassay attributes. Based on the statistical evaluation, negligible differences could be detected for sialylation, glycation, fragments and charge variants, which do not affect the functional properties.</p><p><strong>Conclusion: </strong>Based on the similarity study, RGB-19 and RoActemra^® can be considered highly similar drug products. The minor differences found for some physico-chemical attributes do not affect the biological potency, binding and other critical attributes, and are therefore not considered clinically meaningful.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"675-696"},"PeriodicalIF":6.9,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12354568/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144764438","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Introduction of Biopharmaceuticals in Europe: A Cross-Sectional Study of Early Diffusion Patterns and Data Availability.","authors":"Ivar Veszelei, Brian Godman, Katri Aaltonen, Gisbert W Selke, Kristina Garuolienė, Agnese Cangini, Amanj Kurdi, António Teixeira Rodrigues, Caridad Pontes, Carla Torre, Carlotta Lunghi, Edel Burton, Elita Poplavska, Freyja Jónsdóttir, Guenka Petrova, Irene Langner, Irina Iaru, Irina Odnoletkova, Juraj Slabý, Katarina Gvozdanović, Leena Saastamoinen, Ott Laius, Ria Benkö, Silvija Žiogaitė, Stuart McTaggart, Tanja Mueller, Thais de Pando, Tomáš Tesař, Zornitsa Mitkova, Björn Wettermark","doi":"10.1007/s40259-025-00732-2","DOIUrl":"10.1007/s40259-025-00732-2","url":null,"abstract":"<p><strong>Background and objectives: </strong>Biopharmaceuticals add value in the treatment of many diseases but different health systems in Europe face clinical and economic challenges with introducing them. Joint efforts across Europe are therefore essential to ensure their sustainable and equitable use. However, to date few cross-national comparative studies have assessed their introduction. This study aimed to assess the availability of health authority data and variation in the early diffusion of biopharmaceuticals across Europe.</p><p><strong>Methods: </strong>A cross-sectional study was undertaken to analyze the diffusion of 17 biopharmaceuticals, approved between 2015 and 2019, among European countries between 2015 and 2022. The study assessed data availability, diffusion rates measured as accumulated defined daily doses per 1000 inhabitants, as well as relative rankings between countries during the first 4 years following market authorization.</p><p><strong>Results: </strong>Twenty countries and two regions out of 31 European countries provided data on biopharmaceutical utilization for out-of-hospital care, 15 provided wholesaler data, and 14 provided hospital data. Certain countries and regions contributed data in multiple categories, while six did not provide any data. Diffusion rates were assessed for 17 countries and two regions, which showed appreciable variation, with secukinumab and erenumab being introduced in most countries and follitropin delta and tildrakizumab in the least number of countries. Germany, Austria, and Norway demonstrated the highest early diffusion rates, while Lithuania, Romania, and Latvia had the lowest.</p><p><strong>Conclusions: </strong>This study revealed a substantial variation between European countries and regions in the early diffusion of biopharmaceuticals and the availability of data to monitor their use. The reasons behind these patterns require further investigation to support European countries in optimizing the use of biopharmaceuticals to reach an equitable and cost-effective use of medicines across Europe.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":"735-751"},"PeriodicalIF":6.9,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12354550/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144798108","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Data Included in Post-authorisation Measures: A Case Study of Approved Advanced Therapy Medicinal Products in the European Union between 2013 and 2024.","authors":"Diogo Almeida, Diana Mandslay, Peter G M Mol, Bruno Sepodes, Carla Torre","doi":"10.1007/s40259-025-00737-x","DOIUrl":"https://doi.org/10.1007/s40259-025-00737-x","url":null,"abstract":"<p><strong>Background: </strong>Advanced therapy medicinal products (ATMPs) often require long-term monitoring to assess both safety and efficacy post-authorisation due to uncertainties identified during the approval process. This study aims to characterise the use of real-world data (RWD) in post-authorisation measures (PAMs) for ATMPs approved in the European Union.</p><p><strong>Methods: </strong>A systematic extraction of all PAMs from publicly available European Medicines Agency (EMA) regulatory documents for ATMPs approved between January 2013 and December 2024 was performed, followed by the identification of the presence and sources of RWD. Additional databases including the HMA-EMA Catalogue of RWD studies and sources and ClinicalTrials.gov were consulted.</p><p><strong>Results: </strong>Amongst 25 ATMPs approved by the European Commission over the study period, a total of 118 PAMs were identified, of which 49 (41.5%) involved RWD. Most RWD-PAMs were imposed by the EMA (n = 34; 69.4%), secondary data use was the most referenced data use type (n = 28; 57.1%) and registries were the main source of RWD being mentioned (n = 26; 53.1%). Further, 5 (10.2%) included a comparator group and 13 (32.5%) incorporated patient-reported outcomes.</p><p><strong>Conclusions: </strong>This study emphasises the instrumental role of RWD in the post-authorisation monitoring of ATMPs in the European Union. PAMs reflect the regulatory flexibility for these products, shifting some efforts to the post-authorisation phase to address benefit-risk gaps. Enhancing the use of RWD in this context could improve evidence generation, minimise uncertainties and support more informed regulatory decisions.</p>","PeriodicalId":9022,"journal":{"name":"BioDrugs","volume":" ","pages":""},"PeriodicalIF":6.9,"publicationDate":"2025-08-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144941686","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}