Therapeutic innovation & regulatory science最新文献

The Adoption and Use of Artificial Intelligence and Machine Learning in Clinical Development. 人工智能和机器学习在临床开发中的采用和使用。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-05-29 DOI: 10.1007/s43441-025-00803-0

Mary Jo Lamberti, Maria I Florez, Hana Do, Stephanie Rosner, Timothe Menard, Carrie Nielson, Amanda Donovan, Jingjing Ye, Sathish Kaveripakam, Birgit Schoeberl, Alette R Hunt, Helen Yeardley

{"title":"The Adoption and Use of Artificial Intelligence and Machine Learning in Clinical Development.","authors":"Mary Jo Lamberti, Maria I Florez, Hana Do, Stephanie Rosner, Timothe Menard, Carrie Nielson, Amanda Donovan, Jingjing Ye, Sathish Kaveripakam, Birgit Schoeberl, Alette R Hunt, Helen Yeardley","doi":"10.1007/s43441-025-00803-0","DOIUrl":"https://doi.org/10.1007/s43441-025-00803-0","url":null,"abstract":"Background: The use of artificial intelligence (AI) and machine learning (ML) in drug discovery has been well documented, but measures of levels of adoption, investments, and efficiencies gained from its use in clinical development have not yet been developed, captured or published. AI/ML use in clinical development is expected to increase, but its impact has not yet been systematically measured until now.Methods: The Tufts Center for the Study of Drug Development conducted a global online survey among pharmaceutical and biotechnology companies, contract research organizations (CROs), and data and technology vendors servicing drug developers. The survey gathered 302 responses assessing levels of AI/ML implementation across 36 distinct clinical trial planning and design, trial execution, and regulatory submission activities. The survey collected data on US dollar investment, time savings, and challenges and opportunities of AI/ML use in clinical development.Results: Approximately one-third of the sample (36.9%) was not yet using or implementing AI/ML across 36 design and planning, execution, and regulatory submission activities; another 30.3% was beginning their AI/ML implementation (or piloting), 22.1% was partially implementing (or moving beyond pilots), and on average only 10.7% had fully implemented AI/ML (i.e., uses AI in most trials employing a repeatable process).","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144174985","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Overview of Orphan Medicines in European Union: An Analysis of Regulatory and Technical-Scientific Aspects. 欧盟孤儿药概述：监管和技术科学方面的分析。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-05-29 DOI: 10.1007/s43441-025-00810-1

Greta Santi Laurini, Victoria Nikitina, Massimiliano Broccoli, Nicola Montanaro, Domenico Motola

{"title":"Overview of Orphan Medicines in European Union: An Analysis of Regulatory and Technical-Scientific Aspects.","authors":"Greta Santi Laurini, Victoria Nikitina, Massimiliano Broccoli, Nicola Montanaro, Domenico Motola","doi":"10.1007/s43441-025-00810-1","DOIUrl":"https://doi.org/10.1007/s43441-025-00810-1","url":null,"abstract":"Introduction: The Orphan Medicinal Product Regulation was adopted in the EU in 2000 to encourage the implementation of medicines for rare diseases. Providing a current overview of its effects, this study was performed on medicines with active orphan designation authorised in the EU until January 17, 2024.Materials and methods: Based on the Community Register of orphan medicinal products for human use, active orphan designations of medicines that have been granted marketing authorisation (MA) were included in the study. General information on medicines, orphan designations, and MAs was collected from web-based sources and analysed using descriptive statistics.Results: Since 2000, 149 medicines with clinical indications with active orphan designation have been granted MA in the EU, making a total of 184 authorised orphan indications. Most medicines (96;64.4%) received standard MA, while 33 (22.1%) received conditional MA and 20 (13.4%) MA under exceptional circumstances. Sixty-five (43.6%) medicines were biological products, mainly monoclonal antibodies, recombinant human peptides or enzymes, or gene therapies. Active orphan designations with outcome for MA were primarily for indications for neoplasms or endocrine, nutritional or metabolic diseases. Orphan indications were licensed after a mean of 67.2 months (range 6-249 months) from designation date. For 93 (50.5%) orphan designations, the prevalence estimate of the condition in the EU was ≤ 1/10,000.Conclusions: Despite pharmacological advances, a limited number of orphan medicines have been authorised in the EU since the entry into force of the Orphan Regulation, making the lack of available medicines for rare diseases still a public health problem.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144174982","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Letter to the Editor "Research on Core Competency Elements of Clinical Investigators". 致编辑的信“临床研究者核心能力要素研究”。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-05-29 DOI: 10.1007/s43441-025-00815-w

Sadia Farhana

引用次数: 0

Recommendation Paper on Advancing the Use of Decentralised Elements in Clinical Trials. 关于在临床试验中推进分散要素使用的建议文件。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-05-27 DOI: 10.1007/s43441-025-00796-w

Alison Bond, Tracey Robertson, Christine Fletcher, Elizabeth Theogaraj, Greg Jordinson, Scott Askin

引用次数: 0

Review of Recent Pharmacoepidemiologic Post-Market Safety Studies Through the Lens of the Estimand Framework. 从评估框架的角度回顾最近的药物流行病学上市后安全性研究。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-05-27 DOI: 10.1007/s43441-025-00780-4

Yong Ma, Jonathan Haddad, Wei Liu, Ellen Snyder, Dimitri Bennett, Susan Mayo

{"title":"Review of Recent Pharmacoepidemiologic Post-Market Safety Studies Through the Lens of the Estimand Framework.","authors":"Yong Ma, Jonathan Haddad, Wei Liu, Ellen Snyder, Dimitri Bennett, Susan Mayo","doi":"10.1007/s43441-025-00780-4","DOIUrl":"https://doi.org/10.1007/s43441-025-00780-4","url":null,"abstract":"The ICH E9(R1) estimand framework provides a systematic approach to ensure alignment among clinical trial objectives, trial conduct, statistical analyses, and interpretation of results, however, whether it can be readily utilized for the pharmacoepidemiologic safety studies has not been established. We selected articles on drug safety published in the Journal Pharmacoepidemiology and Drug Safety (PDS), during 2020 to investigate whether estimand attributes were well defined in the study design and reporting. We found that among twenty-five articles selected, nineteen were cohort studies and six were nested case-control studies. All studies had well-defined exposure, outcome, target population, and population level summary. The term intercurrent event (ICE) was not mentioned in any of the studies; however, many cohort studies discussed drug discontinuation, treatment modification and terminal events, and strategies to handle them. All studies used methods to control for confounding: propensity score methods or covariate adjustment, or both for cohort studies; matching and covariate adjustment for the nested case-control studies. We conclude that while the estimand framework can serve to add clarity and precision to pharmacoepidemiologic safety studies, more detailed considerations are required for bias assessment to compensate for the lack of randomization and other shortcomings in observational studies. Recent pharmacoepidemiology frameworks, such as Target Trial Emulation, STaRT-RWE, HARPER could be combined with the complementary principals from the estimand framework to help achieve the study objectives.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-05-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144161136","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Advancing AI Ethics Frameworks in Drug Development: Global Applicability, Practical Challenges, and Dynamic Governance. 推进药物开发中的人工智能伦理框架：全球适用性、实际挑战和动态治理。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-05-26 DOI: 10.1007/s43441-025-00814-x

Kai Chen, Zekai Yu

引用次数: 0

The Path To Tarlatamab Approval: Leveraging Innovative Strategies and Global Regulatory Pathways. 塔拉塔单抗获批之路：利用创新战略和全球监管途径。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-05-26 DOI: 10.1007/s43441-025-00809-8

Vandana Pathak, Claudia Arredondo Pimentel, Elli Cooney, Brian Abbott, Jacqueline M Kline

引用次数: 0

Letter to the Editor - Reply to "Advancing AI Ethics Frameworks in Drug Development: Global Applicability, Practical Challenges, and Dynamic Governance". 致编辑的信-回复“推进药物开发中的人工智能伦理框架：全球适用性、实际挑战和动态治理”。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-05-26 DOI: 10.1007/s43441-025-00813-y

Timothé Ménard, Katrina A Bramstedt

引用次数: 0

Keeping the End in Mind: Reviewing U.S. FDA Inspections of Submissions including Real-World Data. 牢记最终目的：审查美国FDA对提交材料的检查，包括真实世界数据。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-05-24 DOI: 10.1007/s43441-025-00791-1

Cheryl Grandinetti, Donna R Rivera, Lee Pai-Scherf, Anna Choe, Paul G Kluetz, Stefanie Kraus, Gabriel K Innes, Kassa Ayalew

{"title":"Keeping the End in Mind: Reviewing U.S. FDA Inspections of Submissions including Real-World Data.","authors":"Cheryl Grandinetti, Donna R Rivera, Lee Pai-Scherf, Anna Choe, Paul G Kluetz, Stefanie Kraus, Gabriel K Innes, Kassa Ayalew","doi":"10.1007/s43441-025-00791-1","DOIUrl":"https://doi.org/10.1007/s43441-025-00791-1","url":null,"abstract":"The increasing use of real-world data (RWD) to generate real-world evidence (RWE) presents unique opportunities and challenges for drug development and regulatory decision-making, particularly in the area of good clinical practice inspections. FDA typically focuses their application review-based inspections on pivotal studies that generate evidence submitted to support new drug and biological product applications. This focus applies regardless of the data sources used in those studies. In this article, we discuss the fundamental role of good clinical practice inspections in verifying the quality, integrity, and reliability of RWD used in regulatory submissions. Through case examples, we highlight specific challenges related to accessing RWD source records, assessing data quality, and evaluating processes for data curation, transformation, and analysis. Our experience underscores the importance of early engagement with regulatory agencies as well as the implementation of robust quality management practices throughout the study lifecycle. As RWD continues to shape the regulatory landscape, these case examples provide insights in navigating the complexities associated with submissions utilizing RWE for drug approval.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144143714","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Cancer Clinical Trial Patients' Perceptions of Reporting Adverse Events Via an Electronic Platform. 癌症临床试验患者通过电子平台报告不良事件的认知。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-05-23 DOI: 10.1007/s43441-025-00804-z

Minna Grahvendy, Bena Brown, Laurelie R Wishart

{"title":"Cancer Clinical Trial Patients' Perceptions of Reporting Adverse Events Via an Electronic Platform.","authors":"Minna Grahvendy, Bena Brown, Laurelie R Wishart","doi":"10.1007/s43441-025-00804-z","DOIUrl":"https://doi.org/10.1007/s43441-025-00804-z","url":null,"abstract":"Background: Accurate adverse event (AE) reporting is critical to the success of clinical trials; over the last 10 years, momentum has been building to collect AE data directly from the patient. In this study, we investigated the use of an electronic, web-based platform by which cancer clinical trial patients self-reported AE data. In this report, we explored the perceptions and experiences of participants using the platform during participation in an interventional clinical trial.Methodology: Patients consenting to an interventional clinical trial run by a cancer clinical trial unit at a tertiary hospital in Australia were eligible for enrolment. Participants used an online platform to report symptomatic data weekly over 26 weeks. Participant feedback on the platform was collected via an implementation survey at baseline, week 12, and week 26 and a qualitative interview at week 26.Results: Participants agreed that the platform was acceptable, appropriate, and feasible in its purpose. This agreement remained throughout their participation on the study. Qualitative analysis of interview data revealed three major themes: accessibility and useability, platform functionality, and personal attributes and benefits/burdens. Participants reported areas for improvement, primarily around platform functionality and consideration of symptom burden limiting participant capacity to engage with the platform.Conclusions: To our knowledge, this is the first study that investigates, in-depth, the participant experience of self-reporting cancer clinical trial AE data via an electronic platform. Our study lends evidence from the participant-perspective to previously-reported studies investigating the feasibility of collecting AE data directly from the patient. Participants agreed that the platform was feasible and would be of benefit to future cancer clinical trial participants.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144128811","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0