{"title":"Competition of regulatory ecosystems in approving medicines: policy implications in the case of Europe","authors":"Pedro Franco , Stefan Haefliger","doi":"10.1016/j.drudis.2025.104295","DOIUrl":"10.1016/j.drudis.2025.104295","url":null,"abstract":"<div><div>The competition between business ecosystems is relevant not only for strategic management, but also for health policy and regulators. Regulation is one key factor in ecosystem competition, and government and regulatory bodies implement new pharmaceutical legislations, policies, and guidelines contributing to business environments capable of attracting startups, biotech firms, and pharmaceutical industry investments in innovative medicines and technologies. Implications for patients and societal welfare require a thorough analysis of strategies aimed at enhancing the competitive advantage of the European Union (EU) in attracting pharmaceutical companies to prioritize the submission of their innovative medicines. This analysis is essential for ensuring that patients have timely access to new treatments, that society benefits from advances in healthcare, and could foster the competitive advantage of the European regulatory ecosystem. Here, we present data from 47 interviews with pharmaceutical industry professionals, offering direct insights into regulatory ecosystem competition and global health policy. Our report underscores the necessity for effective strategies that enhance the competitive advantage of the European regulatory system.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 2","pages":"Article 104295"},"PeriodicalIF":6.5,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142997220","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":"The epigenetic impact of fatty acids as DNA methylation modulators","authors":"Meran Keshawa Ediriweera , W.M. Gayashani Sandamalika","doi":"10.1016/j.drudis.2024.104277","DOIUrl":"10.1016/j.drudis.2024.104277","url":null,"abstract":"<div><div>DNA methylation is a key epigenetic mechanism that regulates gene expression. Fatty acids, the building blocks of many essential lipids, play a crucial role in various biological events. Aberrant acetylation and methylation profiles are linked to a number of non-communicable diseases. Various fatty acids have been identified as potential ‘epi-drugs’ because of their ability to correct aberrant acetylation and methylation profiles in a number of non-communicable diseases, enhancing the value of their biochemical properties. This review summarizes the effects of selected saturated and unsaturated fatty acids and fatty-acid-rich food items on disease-associated DNA methylation profiles, aiming to justify the classification of fatty acids as DNA methylation modulators.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 2","pages":"Article 104277"},"PeriodicalIF":6.5,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142875670","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":"Albendazole-induced liver injury in children: a cause for concern?","authors":"Devaraj Ezhilarasan, Mustapha Najimi","doi":"10.1016/j.drudis.2025.104296","DOIUrl":"10.1016/j.drudis.2025.104296","url":null,"abstract":"","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 2","pages":"Article 104296"},"PeriodicalIF":6.5,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142997027","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}
Skylar Connor , Leihong Wu , Ruth A. Roberts , Weida Tong
{"title":"Is ChatGPT Ready for Public Use in Organ-Specific Drug Toxicity Research?","authors":"Skylar Connor , Leihong Wu , Ruth A. Roberts , Weida Tong","doi":"10.1016/j.drudis.2025.104297","DOIUrl":"10.1016/j.drudis.2025.104297","url":null,"abstract":"<div><div>The growing impact of large language models (LLMs), such as ChatGPT, prompts questions about the reliability of their application in public health. We compared drug toxicity assessments by GPT-4 for liver, heart, and kidney against expert assessments using US Food and Drug Administration (FDA) drug-labeling documents. Two approaches were assessed: a ‘General prompt’, mimicking the conversational style used by the general public, and an ‘Expert prompt’ engineered to represent an approach of an expert. The Expert prompt achieved higher accuracy (64–75%) compared with the General prompt (48–72%), but the overall performance was moderate, indicating that caution is needed when using GPT-4 for public health. To improve reliability, an advanced framework,such as Retrieval Augmented Generation (RAG), might be required to leverage knowledge embedded in GPT-4.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 2","pages":"Article 104297"},"PeriodicalIF":6.5,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143021448","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":"Biomedical data analytics for better patient outcomes","authors":"Alireza Ghofrani , Hamed Taherdoost","doi":"10.1016/j.drudis.2024.104280","DOIUrl":"10.1016/j.drudis.2024.104280","url":null,"abstract":"<div><div>Medical professionals today have access to immense amounts of data, which enables them to make decisions that enhance patient care and treatment efficacy. This innovative strategy can improve global health care by bridging the divide between clinical practice and medical research. This paper reviews biomedical developments aimed at improving patient outcomes by addressing three main questions regarding techniques, data sources and challenges. The review includes peer-reviewed articles from 2018 to 2023, found via systematic searches in PubMed, Scopus and Google Scholar. The results show diverse disease-specific applications. Challenges such as data quality and ethics are discussed, underscoring data analytics’ potential for patient-focused health care. The review concludes that successful implementation requires addressing gaps, collaboration and innovation in biomedical science and data analytics.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 2","pages":"Article 104280"},"PeriodicalIF":6.5,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142890875","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":"Multi-layer platform coordination for open innovation in oligonucleotide therapeutics","authors":"Yayoi Kawata , Taisuke Ichimaru , Masakazu Kogami , Kota Kodama , Shuto Miyashita , Shintaro Sengoku","doi":"10.1016/j.drudis.2025.104288","DOIUrl":"10.1016/j.drudis.2025.104288","url":null,"abstract":"<div><div>As technology evolves and medical needs diversify, the pharmaceutical industry must accelerate its openness. This study analysed inter-organizational alliances in R&D for the new modality of oligonucleotide therapeutics to explore the requirements for establishing new markets. The results confirmed that the market has developed in stages, employing open innovation for different purposes according to technological progress. At each stage, it was crucial to form platforms among biotech companies on the drug discovery side, among pharmaceutical companies on the regulatory side and contract development manufacturing organizations (CDMOs) on the manufacturing side. These findings clarify the dynamics of open innovation in the biopharmaceutical industry and provide strategic implications for sustainable value creation in drug R&D.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 2","pages":"Article 104288"},"PeriodicalIF":6.5,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142963408","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}
Sam H.A. Muller , Tessa I. van Rijssel , Ghislaine J.M.W. van Thiel, The Hypermarker Consortium , The Trials@Home Consortium
{"title":"Diffused responsibilities in technology-driven health research: The case of artificial intelligence systems in decentralized clinical trials","authors":"Sam H.A. Muller , Tessa I. van Rijssel , Ghislaine J.M.W. van Thiel, The Hypermarker Consortium , The Trials@Home Consortium","doi":"10.1016/j.drudis.2025.104309","DOIUrl":"10.1016/j.drudis.2025.104309","url":null,"abstract":"<div><div>Innovations such as artificial intelligence (AI) and decentralized clinical trials (DCTs) offer opportunities to enhance trial quality and efficiency. However, these innovations raise ethical questions about key responsibilities in research, such as participant safety and data quality. Using an example of an AI system determining personalized medication dosages in a DCT, we highlight how these technologies diffuse responsibilities among multiple actors and technologies. Diffusion diminishes the ability of actors to fulfill responsibilities. To address this, leading actors, such as sponsors, should prioritize overseeing responsibility attribution and implementation. This involves mapping responsibilities, identifying risks and challenges for fulfilling these responsibilities, and organizing adequate communication channels.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 2","pages":"Article 104309"},"PeriodicalIF":6.5,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143254081","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":"Computational approaches for the identification of novel metal-binding pharmacophores: advances and challenges","authors":"Guoli Xiong , Zhiyan Xiao","doi":"10.1016/j.drudis.2025.104293","DOIUrl":"10.1016/j.drudis.2025.104293","url":null,"abstract":"<div><div>Metalloenzymes are important therapeutic targets for a variety of human diseases. Computational approaches have recently emerged as effective tools to understand metal–ligand interactions and expand the structural diversity of both metalloenzyme inhibitors (MIs) and metal-binding pharmacophores (MBPs). In this review, we highlight key advances in currently available fine-tuning modeling methods and data-driven cheminformatic approaches. We also discuss major challenges to the recognition of novel MBPs and MIs. The evidence provided herein could expedite future computational efforts to guide metalloenzyme-based drug discovery.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 2","pages":"Article 104293"},"PeriodicalIF":6.5,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142976922","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":"Antimicrobial peptides: An alternative strategy to combat antimicrobial resistance","authors":"Zahid Gani , Ajay Kumar , Manoj Raje , Chaaya Iyengar Raje","doi":"10.1016/j.drudis.2025.104305","DOIUrl":"10.1016/j.drudis.2025.104305","url":null,"abstract":"<div><div>Antimicrobial peptides (AMPs) are a diverse group of naturally occurring molecules produced by eukaryotes and prokaryotes. They have an important role in innate immunity via their direct microbicidal properties or immunomodulatory activities against pathogens. With the widespread occurrence of antimicrobial resistance (AMR), AMPs are considered as viable alternatives for the treatment of multidrug-resistant microbes, inflammation, and, wound healing. The broad-spectrum antibacterial activity of AMPs is predominantly attributed to membrane disruption, leading to the formation of transmembrane pores and, eventually, cell lysis. However, mechanisms related to inhibition of cell wall synthesis, nucleic acid synthesis, protein synthesis, or enzymatic activity are also associated with these peptides. In this review, we discuss our current understanding, therapeutic uses and challenges associated with the clinical applications of AMPs.</div></div>","PeriodicalId":301,"journal":{"name":"Drug Discovery Today","volume":"30 2","pages":"Article 104305"},"PeriodicalIF":6.5,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143121675","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}