Pub Date : 2025-12-04DOI: 10.1016/j.drudis.2025.104571
Gui Yao , Leming Shi , Yuanting Zheng
The global oncology drug pipeline is characterized by a tension between the diversification of novel targets and a persistent concentration on a few validated ones. Our analysis of 5127 drugs against 1603 targets categorizes entities as ‘proven’ (10.4%), ‘in development’ (53.6%), or ‘not progressing well’ (36.1%). While single-target agents dominate, dual-target strategies, driven by bispecific antibodies, are the fastest-growing approach. The top 20 proven targets attract over 50 drugs each. Small molecules remain prevalent, but novel modalities are expanding. Clinical validation of a novel target requires a median of 8.0 years with a 22.3% success rate. The USA and China drive 62.7% of global development, with the USA leading in first-in-class (FIC) innovation and China’s role increasing since 2020.
{"title":"The landscape of innovative oncology drug targets","authors":"Gui Yao , Leming Shi , Yuanting Zheng","doi":"10.1016/j.drudis.2025.104571","DOIUrl":"10.1016/j.drudis.2025.104571","url":null,"abstract":"<div><div>The global oncology drug pipeline is characterized by a tension between the diversification of novel targets and a persistent concentration on a few validated ones. Our analysis of 5127 drugs against 1603 targets categorizes entities as ‘proven’ (10.4%), ‘in development’ (53.6%), or ‘not progressing well’ (36.1%). While single-target agents dominate, dual-target strategies, driven by bispecific antibodies, are the fastest-growing approach. The top 20 proven targets attract over 50 drugs each. Small molecules remain prevalent, but novel modalities are expanding. Clinical validation of a novel target requires a median of 8.0 years with a 22.3% success rate. The USA and China drive 62.7% of global development, with the USA leading in first-in-class (FIC) innovation and China’s role increasing since 2020.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"31 1","pages":"Article 104571"},"PeriodicalIF":7.5,"publicationDate":"2025-12-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145695674","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}
Pub Date : 2025-12-04DOI: 10.1016/j.drudis.2025.104572
Yenisetti Rajendra Prasad, Sandeep, Prakash Y. Khandave, Abhay H. Pande
Respiratory syncytial virus (RSV) is a leading cause of lower respiratory tract infections and hospitalizations among vulnerable populations, such as children, older adults, and individuals with weakened immune systems, often causing seasonal epidemics. Although current prophylactics, such as palivizumab, nirsevimab, and clesrovimab, which target the RSV F protein, have enhanced early protection, more effective options with broader impact and longer-lasting efficacy are needed. In response to ongoing research efforts to develop new treatments for RSV, nanobodies have emerged as a promising option. Therefore, in this review, we discuss available therapies and explore the potential of nanobodies in treating RSV, with particular emphasis on the importance of PolyBodies [polyspecific and polyvalent antibodies (PsAbs and PvAbs)] in managing RSV infections.
{"title":"PolyBodies for the Treatment of Respiratory Syncytial Virus Infection","authors":"Yenisetti Rajendra Prasad, Sandeep, Prakash Y. Khandave, Abhay H. Pande","doi":"10.1016/j.drudis.2025.104572","DOIUrl":"10.1016/j.drudis.2025.104572","url":null,"abstract":"<div><div>Respiratory syncytial virus (RSV) is a leading cause of lower respiratory tract infections and hospitalizations among vulnerable populations, such as children, older adults, and individuals with weakened immune systems, often causing seasonal epidemics. Although current prophylactics, such as palivizumab, nirsevimab, and clesrovimab, which target the RSV F protein, have enhanced early protection, more effective options with broader impact and longer-lasting efficacy are needed. In response to ongoing research efforts to develop new treatments for RSV, nanobodies have emerged as a promising option. Therefore, in this review, we discuss available therapies and explore the potential of nanobodies in treating RSV, with particular emphasis on the importance of PolyBodies [polyspecific and polyvalent antibodies (PsAbs and PvAbs)] in managing RSV infections.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"31 1","pages":"Article 104572"},"PeriodicalIF":7.5,"publicationDate":"2025-12-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145695713","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}
Pub Date : 2025-12-03DOI: 10.1016/j.drudis.2025.104570
Satvik Sangai, Dhrumi Patel, Sarika Wairkar
Pulmonary fibrosis (PF) is a chronic interstitial lung disease that causes inflammation and scarring around the lung alveoli. This scarring is called fibrosis and leads to breathing difficulties in patients. The treatment for PF is currently limited to two drugs: nintedanib and pirfenidone. Their use is restricted owing to poor bioavailability and a few contraindications. Biotechnological advancements are leading to the investigation of targeted systems aimed at reversing alveolar damage and alleviating PF. This review provides key insights on biotechnology-based advancements for PF, namely monoclonal antibodies, peptides, nucleic acids and stem cell therapy. We also underscore the obstacles and prospective developments in biotechnology-derived therapeutics for PF.
{"title":"Biotechnology-based therapies for mitigation of pulmonary fibrosis: an update","authors":"Satvik Sangai, Dhrumi Patel, Sarika Wairkar","doi":"10.1016/j.drudis.2025.104570","DOIUrl":"10.1016/j.drudis.2025.104570","url":null,"abstract":"<div><div>Pulmonary fibrosis (PF) is a chronic interstitial lung disease that causes inflammation and scarring around the lung alveoli. This scarring is called fibrosis and leads to breathing difficulties in patients. The treatment for PF is currently limited to two drugs: nintedanib and pirfenidone. Their use is restricted owing to poor bioavailability and a few contraindications. Biotechnological advancements are leading to the investigation of targeted systems aimed at reversing alveolar damage and alleviating PF. This review provides key insights on biotechnology-based advancements for PF, namely monoclonal antibodies, peptides, nucleic acids and stem cell therapy. We also underscore the obstacles and prospective developments in biotechnology-derived therapeutics for PF.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"31 1","pages":"Article 104570"},"PeriodicalIF":7.5,"publicationDate":"2025-12-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145686648","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}
Pub Date : 2025-12-03DOI: 10.1016/j.drudis.2025.104569
Ruth A. Roberts , Mamta Behl , Helena T. Hogberg , Eduardo Dunayevich , Michael Morton , Kimberly L. Rockley , Abigail L Walker , Jennifer B. Pierson
Seizures are the hallmark of treatment-resistant epilepsy and are also a frequent adverse event in drug safety testing. Here we present a critical evaluation of new approach methodologies (NAMs) in epilepsy research and seizure liability. Specifically, recent data have shown that seizure can be profiled using an in vitro seizure liability assay (iSLA) comprising a microelectrode array (MEA) of human induced pluripotent stem cell (iPSC) neurons coupled with a human ion channel panel. Adverse outcome pathways (AOPs) also offer a powerful NAM framework to map how key events can lead to an adverse outcome such as seizure. These NAMs offers an innovative approach to antiseizure medication research as well as to seizure risk testing in drug discovery and development.
{"title":"Use of new approach methodologies (NAMs) in epilepsy research and seizure risk assessment","authors":"Ruth A. Roberts , Mamta Behl , Helena T. Hogberg , Eduardo Dunayevich , Michael Morton , Kimberly L. Rockley , Abigail L Walker , Jennifer B. Pierson","doi":"10.1016/j.drudis.2025.104569","DOIUrl":"10.1016/j.drudis.2025.104569","url":null,"abstract":"<div><div>Seizures are the hallmark of treatment-resistant epilepsy and are also a frequent adverse event in drug safety testing. Here we present a critical evaluation of new approach methodologies (NAMs) in epilepsy research and seizure liability. Specifically, recent data have shown that seizure can be profiled using an <em>in vitro</em> seizure liability assay (iSLA) comprising a microelectrode array (MEA) of human induced pluripotent stem cell (iPSC) neurons coupled with a human ion channel panel. Adverse outcome pathways (AOPs) also offer a powerful NAM framework to map how key events can lead to an adverse outcome such as seizure. These NAMs offers an innovative approach to antiseizure medication research as well as to seizure risk testing in drug discovery and development.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"31 1","pages":"Article 104569"},"PeriodicalIF":7.5,"publicationDate":"2025-12-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145686662","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}
Hepatocellular carcinoma (HCC) is associated with multiple pathological processes. Accumulating evidence implicates circadian disruption in hepatocarcinogenesis, as it disrupts cell cycle regulation, metabolic homeostasis, and tumor immunity. Abnormal activation of clock proteins, particularly circadian locomotor output cycles kaput (CLOCK) and brain and muscle ARNT-like protein 1 (BMAL1), induces HCC cell proliferation, inhibits tumor apoptosis, and facilitates immune evasion, thereby leading to HCC development and progression. Circadian rhythm recovery by means of either pharmacological modulation of clock proteins or chronotherapy emerges as a promising strategy to suppress tumor growth and augment therapeutic efficacy. This review delineates the role of CLOCK and BMAL1 in HCC pathogenesis, discusses emerging circadian-based therapies, and highlights challenges for targeting circadian dysregulation to improve HCC treatment outcomes.
肝细胞癌（HCC）与多种病理过程相关。越来越多的证据表明，在肝癌发生过程中，昼夜节律紊乱会破坏细胞周期调节、代谢稳态和肿瘤免疫。时钟蛋白的异常激活，特别是昼夜运动输出周期衰竭（circadian locomotor output cycles kaput, clock）和脑肌arnt样蛋白1 (brain and muscle ARNT-like protein 1, BMAL1)，可诱导HCC细胞增殖，抑制肿瘤凋亡，促进免疫逃逸，从而导致HCC的发生和进展。通过时钟蛋白的药理调节或时间疗法来恢复昼夜节律是抑制肿瘤生长和增强治疗效果的一种有希望的策略。本文概述了CLOCK和BMAL1在HCC发病机制中的作用，讨论了新兴的基于昼夜节律的治疗方法，并强调了针对昼夜节律失调以改善HCC治疗结果的挑战。
{"title":"Circadian disruption in hepatocellular carcinoma: Current findings and future directions of molecular mechanisms","authors":"Liming Zheng , Xuemei Zhang , Hai Feng , Jianfeng Guo , Zhuo Yu","doi":"10.1016/j.drudis.2025.104566","DOIUrl":"10.1016/j.drudis.2025.104566","url":null,"abstract":"<div><div>Hepatocellular carcinoma (HCC) is associated with multiple pathological processes. Accumulating evidence implicates circadian disruption in hepatocarcinogenesis, as it disrupts cell cycle regulation, metabolic homeostasis, and tumor immunity. Abnormal activation of clock proteins, particularly circadian locomotor output cycles kaput (CLOCK) and brain and muscle ARNT-like protein 1 (BMAL1), induces HCC cell proliferation, inhibits tumor apoptosis, and facilitates immune evasion, thereby leading to HCC development and progression. Circadian rhythm recovery by means of either pharmacological modulation of clock proteins or chronotherapy emerges as a promising strategy to suppress tumor growth and augment therapeutic efficacy. This review delineates the role of CLOCK and BMAL1 in HCC pathogenesis, discusses emerging circadian-based therapies, and highlights challenges for targeting circadian dysregulation to improve HCC treatment outcomes.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"31 1","pages":"Article 104566"},"PeriodicalIF":7.5,"publicationDate":"2025-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145676044","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}
Pub Date : 2025-12-02DOI: 10.1016/j.drudis.2025.104568
Samantha Parker , Jida El Hajjar , Anneliene H. Jonker , Susan R. Kahn , Persefoni Kritikou , Christina Kyriakopoulou , Anthony Haight
There are >6000 rare diseases (RDs), affecting >300 million people worldwide. Despite increases in orphan drug designations (ODDs), approved treatments remain scarce. This study analyzes the outcomes of non-oncology ODs designated by the FDA and European Medicines Agency (EMA) in 2017, providing a 7–8-year window to evaluate progression. This analysis of 292 ODDs examined therapeutic modalities, sponsor size, and funding factors influencing success, as defined by development phase transitions. Biologics showed higher approval rates whereas gene therapies displayed lower phase transition success. Smaller companies and academic groups demonstrated lower success rates. This research identifies drivers and gaps in OD development, emphasizing the need for sustained multistakeholder investment.
{"title":"Non-oncology orphan drug development: Productivity and probability of success","authors":"Samantha Parker , Jida El Hajjar , Anneliene H. Jonker , Susan R. Kahn , Persefoni Kritikou , Christina Kyriakopoulou , Anthony Haight","doi":"10.1016/j.drudis.2025.104568","DOIUrl":"10.1016/j.drudis.2025.104568","url":null,"abstract":"<div><div>There are >6000 rare diseases (RDs), affecting >300 million people worldwide. Despite increases in orphan drug designations (ODDs), approved treatments remain scarce. This study analyzes the outcomes of non-oncology ODs designated by the FDA and European Medicines Agency (EMA) in 2017, providing a 7–8-year window to evaluate progression. This analysis of 292 ODDs examined therapeutic modalities, sponsor size, and funding factors influencing success, as defined by development phase transitions. Biologics showed higher approval rates whereas gene therapies displayed lower phase transition success. Smaller companies and academic groups demonstrated lower success rates. This research identifies drivers and gaps in OD development, emphasizing the need for sustained multistakeholder investment.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"31 1","pages":"Article 104568"},"PeriodicalIF":7.5,"publicationDate":"2025-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145676117","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}
Pub Date : 2025-12-01DOI: 10.1016/j.drudis.2025.104553
Rafael André da Silva , Anna Howell , Kendra Jones-Graham , Shama Parween , Ana Paula Girol , Sandra H.P. Farsky , Cristiane D. Gil , M. Natalia Vergara
Formyl peptide receptors (FPRs) belong to the G protein-coupled receptor family and are involved in regulating inflammatory processes, neuronal survival, and angiogenesis. In tissues such as the retina and cornea, chronic or unresolved inflammation contributes to vision loss. We review FPR2’s dual, stage-dependent roles in ocular diseases, where activation by pro-resolving agonists or inhibition by antagonists can each confer therapeutic benefits. This context-specific ‘on/off’ potential highlights the need for precise, stage-tailored modulation. Preclinical studies demonstrate that targeting FPR2 can improve outcomes in diabetic retinopathy, keratitis, choroidal and corneal neovascularization, and re-epithelialization. We also examine the molecular mechanisms, signaling pathways, and ligand specificity that drive these divergent effects, positioning FPR2 as a promising yet complex therapeutic target in ophthalmology.
{"title":"FPR2 at the crossroads of inflammation and repair in the eye","authors":"Rafael André da Silva , Anna Howell , Kendra Jones-Graham , Shama Parween , Ana Paula Girol , Sandra H.P. Farsky , Cristiane D. Gil , M. Natalia Vergara","doi":"10.1016/j.drudis.2025.104553","DOIUrl":"10.1016/j.drudis.2025.104553","url":null,"abstract":"<div><div>Formyl peptide receptors (FPRs) belong to the G protein-coupled receptor family and are involved in regulating inflammatory processes, neuronal survival, and angiogenesis. In tissues such as the retina and cornea, chronic or unresolved inflammation contributes to vision loss. We review FPR2’s dual, stage-dependent roles in ocular diseases, where activation by pro-resolving agonists or inhibition by antagonists can each confer therapeutic benefits. This context-specific ‘on/off’ potential highlights the need for precise, stage-tailored modulation. Preclinical studies demonstrate that targeting FPR2 can improve outcomes in diabetic retinopathy, keratitis, choroidal and corneal neovascularization, and re-epithelialization. We also examine the molecular mechanisms, signaling pathways, and ligand specificity that drive these divergent effects, positioning FPR2 as a promising yet complex therapeutic target in ophthalmology.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 12","pages":"Article 104553"},"PeriodicalIF":7.5,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145572807","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}
Pub Date : 2025-12-01DOI: 10.1016/j.drudis.2025.104549
Qisi Lu , Tao You , Qian Cheng , Zhe-Sheng Chen , Haiwen Huang
Extranodal natural killer/T-cell lymphoma (ENKTL) is an aggressive non-Hodgkin’s lymphoma associated with Epstein–Barr virus and typically involves extranodal sites. The traditional CHOP regimen is no longer standard owing to drug resistance, whereas asparaginase-based therapies have become the mainstay of treatment. However, resistance and tumor invasion lead to relapses in 40–50% of patients, with 20% progressing to relapsed/refractory ENKTL, resulting in a median survival of <12 months. Management of ENKTL remains challenging, with an unmet clinical need. However, although the prognosis for relapsed/refractory ENKTL is poor, the development of new treatment strategies is progressing with a better understanding of the disease. This review examines factors contributing to treatment resistance, underlying resistance mechanisms and potential strategies to overcome ENKTL resistance.
{"title":"Mechanisms and interventions in relapsed/refractory extranodal natural killer/T-cell lymphoma","authors":"Qisi Lu , Tao You , Qian Cheng , Zhe-Sheng Chen , Haiwen Huang","doi":"10.1016/j.drudis.2025.104549","DOIUrl":"10.1016/j.drudis.2025.104549","url":null,"abstract":"<div><div>Extranodal natural killer/T-cell lymphoma (ENKTL) is an aggressive non-Hodgkin’s lymphoma associated with Epstein–Barr virus and typically involves extranodal sites. The traditional CHOP regimen is no longer standard owing to drug resistance, whereas asparaginase-based therapies have become the mainstay of treatment. However, resistance and tumor invasion lead to relapses in 40–50% of patients, with 20% progressing to relapsed/refractory ENKTL, resulting in a median survival of <12 months. Management of ENKTL remains challenging, with an unmet clinical need. However, although the prognosis for relapsed/refractory ENKTL is poor, the development of new treatment strategies is progressing with a better understanding of the disease. This review examines factors contributing to treatment resistance, underlying resistance mechanisms and potential strategies to overcome ENKTL resistance.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 12","pages":"Article 104549"},"PeriodicalIF":7.5,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145562118","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}
Pub Date : 2025-12-01DOI: 10.1016/j.drudis.2025.104542
Liam Bendicksen , Lawrence King , Edward R. Scheffer Cliff , Aaron S. Kesselheim
Transformative medicines have extended and improved many lives. Few studies, however, have qualitatively investigated how transformative drugs are discovered and developed. We conducted semi-structured interviews to investigate how scientists discovered ibrutinib (Imbruvica), a transformative drug for chronic lymphocytic leukemia. Ibrutinib originated as a tool compound, created to help chemists understand the effects of inhibiting Bruton tyrosine kinase to treat autoimmune diseases. After the corporate owner of ibrutinib sold it to a struggling biotech firm for very little money, the biotech reconceptualized ibrutinib as a cancer therapy. Even after ibrutinib showed preliminary signs of efficacy, internationally accepted response criteria that did not account for its novel mechanism of action threatened to derail its development and ability to reach patients.
{"title":"Discovering a transformative cancer drug: the case of ibrutinib","authors":"Liam Bendicksen , Lawrence King , Edward R. Scheffer Cliff , Aaron S. Kesselheim","doi":"10.1016/j.drudis.2025.104542","DOIUrl":"10.1016/j.drudis.2025.104542","url":null,"abstract":"<div><div>Transformative medicines have extended and improved many lives. Few studies, however, have qualitatively investigated how transformative drugs are discovered and developed. We conducted semi-structured interviews to investigate how scientists discovered ibrutinib (Imbruvica), a transformative drug for chronic lymphocytic leukemia. Ibrutinib originated as a tool compound, created to help chemists understand the effects of inhibiting Bruton tyrosine kinase to treat autoimmune diseases. After the corporate owner of ibrutinib sold it to a struggling biotech firm for very little money, the biotech reconceptualized ibrutinib as a cancer therapy. Even after ibrutinib showed preliminary signs of efficacy, internationally accepted response criteria that did not account for its novel mechanism of action threatened to derail its development and ability to reach patients.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 12","pages":"Article 104542"},"PeriodicalIF":7.5,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145562134","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}
Pub Date : 2025-12-01DOI: 10.1016/j.drudis.2025.104552
Nabasmita Talukdar , Xiaodan Zhang , Shreya Paithankar , Hui Wang , Bin Chen
Electronic health records (EHRs) have been increasingly used as real-world evidence to support the discovery and validation of new drug indications. This paper surveys current approaches to EHR-based drug repurposing, covering data sources, processing methodologies, and representation techniques. It discusses study designs and statistical frameworks for evaluating drug efficacy. Key challenges and opportunities in validation are also discussed, with an emphasis on the role of large language models and target trial emulation. By synthesizing recent developments and methodological advances, this work provides a foundational resource for researchers aiming to translate real-world data into actionable drug repurposing evidence.
{"title":"A survey of the use of EHR as real-world evidence for discovering and validating new drug indications","authors":"Nabasmita Talukdar , Xiaodan Zhang , Shreya Paithankar , Hui Wang , Bin Chen","doi":"10.1016/j.drudis.2025.104552","DOIUrl":"10.1016/j.drudis.2025.104552","url":null,"abstract":"<div><div>Electronic health records (EHRs) have been increasingly used as real-world evidence to support the discovery and validation of new drug indications. This paper surveys current approaches to EHR-based drug repurposing, covering data sources, processing methodologies, and representation techniques. It discusses study designs and statistical frameworks for evaluating drug efficacy. Key challenges and opportunities in validation are also discussed, with an emphasis on the role of large language models and target trial emulation. By synthesizing recent developments and methodological advances, this work provides a foundational resource for researchers aiming to translate real-world data into actionable drug repurposing evidence.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 12","pages":"Article 104552"},"PeriodicalIF":7.5,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145572787","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}
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