Therapeutic innovation & regulatory science最新文献_第8页

Development of a Drug Safety Signal Detection Reference Set Using Japanese Safety Information. 基于日本安全信息的药品安全信号检测参考集的研制。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-03-01 Epub Date: 2024-12-21 DOI: 10.1007/s43441-024-00729-z

Satoru Ito, Mamoru Narukawa

{"title":"Development of a Drug Safety Signal Detection Reference Set Using Japanese Safety Information.","authors":"Satoru Ito, Mamoru Narukawa","doi":"10.1007/s43441-024-00729-z","DOIUrl":"10.1007/s43441-024-00729-z","url":null,"abstract":"Introduction: One of the main objectives of pharmacovigilance activities is to confirm unknown adverse drug reactions (ADRs), and data-mining methods have been developed to detect signals that are candidates for ADRs. Reference sets have been developed to evaluate the performance of the data-mining methods. However, reference sets generated in previous studies are not based on Japanese safety information; therefore, they are not suitable for use in evaluation studies in Japan because some drugs have not been approved or marketed for a long time in Japan. This study aimed to develop a reference set using drug safety information marketed in Japan and to evaluate its performance.Methods: A reference set was developed for 43 drugs and 15 events. For each combination of the selected drug and event, those that were listed as important identified risks in the Japan Risk Management Plan (J-RMP) were set as \"positive controls\" and those that were not listed as adverse reactions in the package insert were set as \"negative controls.\" In addition, we performed data-mining using Japanese Adverse Drug Event Report database (JADER) and evaluated the results against the reference set to empirically confirm its effectiveness.Results: The reference set included 127 positive and 386 negative controls. A comparison of the signals obtained from data-mining using JADER with the reference set revealed higher correlations than those in previous studies.Conclusion: A reference set was developed using the safety information of drugs approved in Japan to promote research on data-mining methods.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"288-294"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142873002","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

Correction: A Global Industry Survey on Post-Approval Change Management and Use of Reliance. 更正：一项关于批准后变更管理和信赖使用的全球行业调查。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-03-01 DOI: 10.1007/s43441-024-00733-3

Andrew Deavin, Aliyah Hossain, Isabelle Colmagne-Poulard, Kum Cheun Wong, Mónica Perea-Vélez, Sonia Cappellini, Susanne Ausborn, Sylvie Meillerais, Céline Bourguignon

引用次数: 0

Basic Considerations for Data Pooling Strategy in Multi-Regional Clinical Trials (MRCTs). 多区域临床试验（mrct）数据池策略的基本考虑。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-03-01 Epub Date: 2025-01-25 DOI: 10.1007/s43441-025-00744-8

Jiali Song, Chen Ji, Meng Chen, Jun Dong, Chao Zhu, Haiyan Wu, Wei Zhang, Kezhou Zhang, Bing Yu, Yun Wang, Hua Zhang, Fan Jia, Yan Hou

{"title":"Basic Considerations for Data Pooling Strategy in Multi-Regional Clinical Trials (MRCTs).","authors":"Jiali Song, Chen Ji, Meng Chen, Jun Dong, Chao Zhu, Haiyan Wu, Wei Zhang, Kezhou Zhang, Bing Yu, Yun Wang, Hua Zhang, Fan Jia, Yan Hou","doi":"10.1007/s43441-025-00744-8","DOIUrl":"10.1007/s43441-025-00744-8","url":null,"abstract":"The National Medical Products Administration of China has been implementing ICH E17, which describes the general principles for planning and designing of multi-regional clinical trials (MRCTs), yet there are several ambiguities in the execution and conduct remains in China or East Asia. In specific, pooling strategy, effect modifiers (EMs), statistical analysis, sample size allocation and their impact in alignment with global trial remains a challenge. In this paper, we explore on the criteria mentioned above under the context of China. EMs need to be determined and identified from intrinsic and extrinsic factors which might have the impact to the drug on specific populations. If no EMs are found, we use pooling by regions to understand whether differences across East Asian population exists, and whether pooling by East Asian is necessary. Statistical models used in the analysis are also listed to estimate the drug effect in pooled populations. In summary, this paper outlines the details of the MRCTs practices in China and provides better insights in practice both domestically and internationally for any future improvements.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"359-364"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11880086/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143042057","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Basic Considerations for the Consistency Evaluation Based on ICH E17 Guideline. 基于ICH E17指南一致性评价的基本考虑。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-03-01 Epub Date: 2025-01-06 DOI: 10.1007/s43441-024-00737-z

Meihua Long, Haiyan Wu, Xiaoni Liu, Pengfei Li, Renxin Lin, Ziwei Zhao, Xiujing Kou, Chao Zhu, Chen Ji, Wei Zhang, Kezhou Zhang, Bing Yu, Yun Wang, Hua Zhang, Fan Jia, Yan Hou

{"title":"Basic Considerations for the Consistency Evaluation Based on ICH E17 Guideline.","authors":"Meihua Long, Haiyan Wu, Xiaoni Liu, Pengfei Li, Renxin Lin, Ziwei Zhao, Xiujing Kou, Chao Zhu, Chen Ji, Wei Zhang, Kezhou Zhang, Bing Yu, Yun Wang, Hua Zhang, Fan Jia, Yan Hou","doi":"10.1007/s43441-024-00737-z","DOIUrl":"10.1007/s43441-024-00737-z","url":null,"abstract":"In the International Council for Harmonisation (ICH) guidance on General Principles for Planning and Design of Multi-Regional Clinical Trials (E17), it is important to evaluate the consistency of treatment effect across regions in a multi-regional clinical trial (MRCT). In this paper, we elaborated on some basic considerations to evaluate consistency. We first list the design considerations, and then provide consistency evaluation and interpretation on pharmacokinetics, pharmacodynamics, efficacy, safety, and benefit-risk. Furthermore, we consider special situations including non-inferiority, multiple primary endpoints, interim analyses with delayed treatment effect, adaptive design, single-arm studies, rare diseases, and statistical methods for regional treatment effect estimate and consistency evaluation. Finally, if potential inconsistency is anticipated or observed in the MRCT, an exploratory framework is provided for further investigations. Overall, this paper elaborates on consistency evaluation in MRCT, discusses possible challenges in reality and also provides strategies and methods to overcome these challenges. This could help consensus across health authorities, industries, and academic societies, which could further facilitate consistency evaluation and MRCT implementation. Effective communication with regulatory authorities is encouraged to obtain an acceptance of a global approach.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":"59 2","pages":"328-336"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11880088/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143543635","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Risk-Based Quality Management: A Case for Centralized Monitoring. 基于风险的质量管理：集中监控的案例。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-03-01 Epub Date: 2024-12-11 DOI: 10.1007/s43441-024-00719-1

Nicole Stansbury, Danilo Branco, Cris McDavid, Jennifer Stewart, Kristin Surdam, Nycole Olson, Joanne Perry, Jeremy Liska, Linda Phillips, Amanda Coogan, Anina Adelfio, Lauren Garson

{"title":"Risk-Based Quality Management: A Case for Centralized Monitoring.","authors":"Nicole Stansbury, Danilo Branco, Cris McDavid, Jennifer Stewart, Kristin Surdam, Nycole Olson, Joanne Perry, Jeremy Liska, Linda Phillips, Amanda Coogan, Anina Adelfio, Lauren Garson","doi":"10.1007/s43441-024-00719-1","DOIUrl":"10.1007/s43441-024-00719-1","url":null,"abstract":"Since 2019, the Association of Clinical Research Organizations has conducted a landscape survey of risk based quality management (RBQM) adoption in clinical trials. Here, we present data from four years of surveys, with an emphasis on the most recent: the 2022 survey included data from 4958 trials across seven contract research organizations, of which 1004 were new studies started in 2022. Results indicate that while overall risk assessment adoption is strong, it is lagging in other risk-based components which suggests companies are not deriving the full expected benefits of performing a risk assessment and mitigation process to their trials. The 2022 study also suggests new study starts showing promising traction, with adoption hovering near 50% for most RBQM elements. At the same time, the survey suggests industry has mixed views on the potential value of quality tolerance limits (QTLs). Ultimately, centralized monitoring is being underutilized despite the potential of increased patient safety oversight and improved data quality. The authors of this paper developed a case study based on a trial in clinicaltrials.gov to demonstrate how RBQM adoption could include the key RBQM elements such as centralized monitoring, reduced source data review and source data verification as well as implementation of QTLs in a real-world scenario. The authors believe the clinical trial industry has an obligation to utilize centralized monitoring to produce more efficient and effective clinical trials and will make a case to do so in this paper.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"199-210"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11880074/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142814222","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Impact of Rule 11 on the European Medical Software Landscape: Analysis of EUDAMED and Further Databases Three Years After MDR Implementation. 规则11对欧洲医疗软件景观的影响：MDR实施三年后EUDAMED和进一步数据库的分析。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-03-01 Epub Date: 2025-01-28 DOI: 10.1007/s43441-025-00747-5

Arndt A Schmitz, Miriam Font-Nieves, Toumani Doucouré, Hans-Peter Podhaisky

{"title":"Impact of Rule 11 on the European Medical Software Landscape: Analysis of EUDAMED and Further Databases Three Years After MDR Implementation.","authors":"Arndt A Schmitz, Miriam Font-Nieves, Toumani Doucouré, Hans-Peter Podhaisky","doi":"10.1007/s43441-025-00747-5","DOIUrl":"10.1007/s43441-025-00747-5","url":null,"abstract":"Medicine is increasingly supported by software, with digital health technologies offering innovative ways to capture insights and drive therapies. Globally, medical device software must follow regulatory processes based on risk classification. The introduction of MDR represents a significant shift in risk-based classification for Medical Devices in Europe, including classification Rule 11 for software, which has caused significant discussions among European regulators. Three years after implementation, we conducted a systematic impact assessment of MDR classification Rule 11 for MDSW through a qualitative and quantitative analysis of over 2000 software entries from the European Medical Device database, complemented by data from other public databases such as the German DiGA directory and mHealthBELGIUM. Our results indicate that classification Rule 11 of the MDR results in a narrow bandwidth for class I software, whereas this used to be the most frequent classification for software under the MDD: while most of legacy software in EUDAMED falls in the lowest risk category as MDD Class I (53%), the situation reverses after the implementation of MDR with the most entries in Class IIa (55%). Analyzing the legacy MDD patient apps in Germany implies that three quarters will have to re-classify as MDR Class IIa at the end of the transition period in 2028. A comparison of the European and US regulatory landscapes, along with a systematic review of software features for Class I vs. Class IIa products, explains our findings and enables us to recommend a regulatory strategy for developing MDSW compliant with MDR Class I rules, ensuring fast access to the European market.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"365-378"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143060821","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

Correction: Consumer Understanding of Prescription Drug Indications in Direct-to-Consumer Television Advertisements. 更正：消费者对直接面向消费者的电视广告中处方药适应症的理解。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-03-01 DOI: 10.1007/s43441-024-00738-y

Helen W Sullivan, Kathryn J Aikin, Mihaela Johnson, Kate Ferriola-Bruckenstein

引用次数: 0

Interim Clinical Trial Data: Who Can See What, and When? 中期临床试验数据：谁能看到什么，什么时候看到？

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-03-01 Epub Date: 2025-01-08 DOI: 10.1007/s43441-024-00728-0

Susan S Ellenberg, Yimei Li

引用次数: 0

The Ethics of the "Right-to-Try" Movement in an Era of Regulatory Flux. 监管变动时代“试权”运动的伦理。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-03-01 Epub Date: 2025-02-06 DOI: 10.1007/s43441-025-00758-2

Neil Jain, David Ralston, Cheryl Erwin

引用次数: 0

A Five-Year Analysis of Market Share and Sales Growth for Original Drugs after Patent Expiration in Korea. 专利到期后韩国原料药市场占有率及销售增长的五年分析。

IF 2 4区医学

Therapeutic innovation & regulatory science Pub Date : 2025-03-01 Epub Date: 2025-01-10 DOI: 10.1007/s43441-025-00741-x

Minyoung Bae, Sung Ryul Shim, Dong-Wook Yang, Kyung-Bok Son, Sang-Won Lee

{"title":"A Five-Year Analysis of Market Share and Sales Growth for Original Drugs after Patent Expiration in Korea.","authors":"Minyoung Bae, Sung Ryul Shim, Dong-Wook Yang, Kyung-Bok Son, Sang-Won Lee","doi":"10.1007/s43441-025-00741-x","DOIUrl":"10.1007/s43441-025-00741-x","url":null,"abstract":"Introduction: The sales patterns of original drugs after patent expiration in Korea show a relatively high market share and continuous sales growth differently from those in the U.S. and European countries. This study aims to investigates a five-year sales pattern of original drugs after patent expiration in Korea using empirical data.Methods: Using data from the Ministry of Food and Drug Safety, original drugs whose patents expired in 2012-2018 were extracted. And we used IQVIA data to determine the market share and sales growth rate of 48 original drugs, whose generic drug launched for the first time in the same molecule market, and whose sales data over five years after first generic entry were available. We analyzed the differences by the attribute of variables.Results: The sales volume of original drugs in the fifth year (Q 20) had an average growth rate of 150.6% compared with that before the first generic drug launched, indicating a continuous growth. The average market share of original drugs in the fifth year (Q 20) decreased to 70.6%, but it was higher than previously reported research results in Korea and other countries. Differences were observed across the category of attribute.Conclusion: This study demonstrated that while market share of original drugs is decreasing, the sales volume increased continuously until the fifth year, differently from those of other countries. Variations in sales patterns by attributes reflect unique dynamics in Korea.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"349-358"},"PeriodicalIF":2.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11880101/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142955501","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0