Therapeutic innovation & regulatory science最新文献_第2页

Patient Preferences for Electronic Versus Paper Patient Information Leaflets: A Survey Among Patients in Sweden. 患者对电子与纸质患者信息传单的偏好：一项瑞典患者调查。

IF 1.9 4区医学

Therapeutic innovation & regulatory science Pub Date : 2026-05-01 Epub Date: 2026-02-07 DOI: 10.1007/s43441-026-00929-9

Annika Forsberg, Wenche Olsen Boström, Klaus Kaae Andersen, Daniel Eek

{"title":"Patient Preferences for Electronic Versus Paper Patient Information Leaflets: A Survey Among Patients in Sweden.","authors":"Annika Forsberg, Wenche Olsen Boström, Klaus Kaae Andersen, Daniel Eek","doi":"10.1007/s43441-026-00929-9","DOIUrl":"10.1007/s43441-026-00929-9","url":null,"abstract":"Objectives: In compliance with legal requirements, medications are mandated to include a patient information leaflet (PIL) in a physical/paper format that serves as a vital source of information about the medication. While the use of an electronic PIL (ePIL) ensures the availability of the most current information for patients, the prevailing preference between the traditional PIL and ePIL remains uncertain. This observational study aimed to address this gap by reporting the findings of an online survey designed to compare patient preferences for an ePIL versus a paper PIL.Methods: Conducted across a total of 15 pharmacies in Sweden, the survey enlisted patients via convenience sampling to rate their preference for an ePIL versus a PIL on a 7-point scale together with other questions on, for example, the ease of information retrieval after their visit to the pharmacy. Data collection commenced in December 2022 and ended in November 2023. The primary hypothesis posited that the ePIL format would emerge as the preferred choice. Most participants were female (66%), and the average age was 56 years.Results: The findings highlighted an inclination towards the ePIL as the preferred choice (58% preferred ePIL, 11% preferred PIL, and 31% were indifferent), with respondents expressing that information retrieval was notably more convenient compared to the paper-based format. Older participants were generally indifferent towards the format. The majority of patients reported familiarity with receiving information in digital formats.Conclusions: These results suggest the readiness of patients in Sweden to transition to ePILs from the traditional paper PIL.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"872-878"},"PeriodicalIF":1.9,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13110224/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146137838","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

The UK's 2026 Clinical Trial Regulations: A Strategic Analysis for Global Pharmaceutical Sponsors. 英国2026年临床试验法规：全球制药赞助商的战略分析。

IF 1.9 4区医学

Therapeutic innovation & regulatory science Pub Date : 2026-05-01 Epub Date: 2026-02-27 DOI: 10.1007/s43441-026-00931-1

Amer Alghabban

{"title":"The UK's 2026 Clinical Trial Regulations: A Strategic Analysis for Global Pharmaceutical Sponsors.","authors":"Amer Alghabban","doi":"10.1007/s43441-026-00931-1","DOIUrl":"10.1007/s43441-026-00931-1","url":null,"abstract":"The United Kingdom is undertaking the most significant reform of its clinical trials legislation in two decades with The Medicines for Human Use (Clinical Trials) (Amendment) Regulations 2025. Set to come into full effect on 28 April 2026, these regulations represent a deliberate national strategy to reposition the UK as a premier global destination for clinical research. This article, informed by insights from the MHRA GCP Symposium in February 2025 and subsequent regulatory guidance, provides a comprehensive analysis of the new framework. It examines the four key pillars of reform: streamlined approvals through a codified Combined Review process; a risk-proportionate approach featuring expedited pathways for low-risk trials and modifications; mandated transparency in trial registration and results publication; and a renewed focus on patient-centricity, including diversity and inclusion. The article further details the simultaneous implementation of ICH E6(R3), discusses the critical transitional arrangements, and outlines the operational imperatives for sponsors. The reforms collectively aim to create a more agile, efficient, and innovative research ecosystem. For global pharmaceutical sponsors, this presents both significant strategic opportunities to accelerate drug development and critical compliance challenges that require immediate operational planning.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"683-687"},"PeriodicalIF":1.9,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147310601","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

Mapping the evolution of cell and gene therapy product research: trends, collaborations, and emerging frontiers. 绘制细胞和基因治疗产品研究的演变：趋势、合作和新兴前沿。

IF 1.9 4区医学

Therapeutic innovation & regulatory science Pub Date : 2026-05-01 Epub Date: 2026-03-05 DOI: 10.1007/s43441-025-00913-9

Yanmei Rao, Li Dong, Zhe Huang

{"title":"Mapping the evolution of cell and gene therapy product research: trends, collaborations, and emerging frontiers.","authors":"Yanmei Rao, Li Dong, Zhe Huang","doi":"10.1007/s43441-025-00913-9","DOIUrl":"10.1007/s43441-025-00913-9","url":null,"abstract":"Advanced therapeutic medicinal products represent a broad spectrum of biopharmaceutical innovations that prominently feature cell and gene therapy formulations. These cutting-edge therapeutic modalities are specifically engineered to intervene in and prevent illnesses that historically necessitated continuous, long-term management. To visually portray the most recent trajectories in cell and gene therapy investigations and to map out prevailing global advancements, a bibliometric assessment was conducted. This analysis meticulously sourced its data from the Web of Science Core Collection database and was significantly enhanced by analytical platforms such as VOSviewer and CiteSpace. Cell and gene therapy products demonstrate considerable promise for both foundational scientific inquiry and diverse clinical interventions, with their utility now extending far beyond cancer treatment to encompass a wide array of other pathological conditions. This paper provides a comprehensive examination of the evolving trends, collaborative endeavors, and burgeoning frontiers within the dynamic landscape of cell and gene therapy products.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"771-781"},"PeriodicalIF":1.9,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147366549","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

Management of Increasing Complexity of Phase I Trials: A Retrospective Analysis from the Niguarda Cancer Center Phase I Unit. I期临床试验日益复杂的管理：来自Niguarda癌症中心I期临床的回顾性分析。

IF 1.9 4区医学

Therapeutic innovation & regulatory science Pub Date : 2026-05-01 Epub Date: 2026-02-17 DOI: 10.1007/s43441-026-00919-x

Anna Bombelli, Noemi Salmistraro, Stefano Stabile, Francesca Pelle, Giovanna Marrapese, Silvia Ghezzi, Sara Mariano, Letizia Monti, Giulia Biamonti, Roberta Carrozzo, Alessio Amatu, Andrea Sartore-Bianchi, Salvatore Siena

引用次数: 0

A Survey on the Implementation of Analytical Quality by Design in Method Development in the Pharmaceutical Industries of Southeast Brazil: Challenges and Opportunities. 巴西东南部制药工业在方法开发中实施设计分析质量的调查：挑战与机遇。

IF 1.9 4区医学

Therapeutic innovation & regulatory science Pub Date : 2026-05-01 Epub Date: 2026-02-24 DOI: 10.1007/s43441-026-00930-2

Bianca C Claro, Erica F Condado, Livia D Prado

{"title":"A Survey on the Implementation of Analytical Quality by Design in Method Development in the Pharmaceutical Industries of Southeast Brazil: Challenges and Opportunities.","authors":"Bianca C Claro, Erica F Condado, Livia D Prado","doi":"10.1007/s43441-026-00930-2","DOIUrl":"10.1007/s43441-026-00930-2","url":null,"abstract":"Analytical Quality by Design (AQbD) is a systematic approach that integrates quality into method design, ensuring robustness, fitness for purpose, and consistency throughout the lifecycle. This study aimed to assess the current scenario, benefits, and challenges of implementing AQbD in analytical method development within the pharmaceutical industry. The methodology included a questionnaire survey applied to pharmaceutical companies, complemented by insights from the literature to contextualize regulatory and scientific challenges. While AQbD offers a promising approach for developing robust methods, its full adoption faces technical, operational, and regulatory barriers. The survey found that although 88.3% of the responding companies are familiar with AQbD, only 50% have begun implementation, mainly in early stages with a focus on tools like design of experiments and risk assessment. Key AQbD components remain underutilized. The main barriers to implementation include a lack of technical resources, qualified personnel, cultural resistance, and unclear regulatory requirements. The literature indicates that AQbD has been widely applied. However, methodological inconsistencies persist. The comparative analysis revealed the alignment between literature and practice regarding benefits but also highlighted gaps in AQbD implementation. By identifying the specific challenges faced by regulated sectors, this study provides valuable insights for regulatory agencies to anticipate future needs, refine guidelines, and promote harmonized standards that ultimately strengthen analytical method quality and ensure consistent product quality throughout the lifecycle.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"879-892"},"PeriodicalIF":1.9,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13110218/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147284984","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

Impurities in Oncology Pharmaceuticals: A Review of Classification, Detection Methods, Regulatory Frameworks and Emerging Trends. 肿瘤药物中的杂质：分类、检测方法、监管框架和新兴趋势的综述。

IF 1.9 4区医学

Therapeutic innovation & regulatory science Pub Date : 2026-05-01 Epub Date: 2026-03-12 DOI: 10.1007/s43441-026-00948-6

Kishan Balehalli Shivananda, Nagarjun Somaprakash, Pradeep Kumar Badiya

{"title":"Impurities in Oncology Pharmaceuticals: A Review of Classification, Detection Methods, Regulatory Frameworks and Emerging Trends.","authors":"Kishan Balehalli Shivananda, Nagarjun Somaprakash, Pradeep Kumar Badiya","doi":"10.1007/s43441-026-00948-6","DOIUrl":"10.1007/s43441-026-00948-6","url":null,"abstract":"Pharmaceutical impurities pose a significant challenge in the development and manufacturing of anti-cancer drugs due to their high potency, narrow therapeutic index, and prolonged administration in most treatment regimens. Even trace-level impurities can compromise drug safety, efficacy, and regulatory compliance. This review provides a comprehensive overview of various types of pharmaceutical impurities organic, inorganic, residual solvents, and genotoxic impurities with a focus on their origins, toxicological significance, and impact on oncology therapeutics. The paper discusses traditional and modern analytical methods used for impurity detection, including chromatographic techniques, spectroscopic tools, mass spectrometry, and capillary electrophoresis and advanced hyphenated systems. Regulatory frameworks from International Council for Harmonization, the U.S. Food and Drug Administration, the European Medicines Agency, Therapeutic Goods Administration, Medicines and Healthcare products Regulatory Agency and the World Health Organization are examined, particularly concerning acceptable limits for genotoxic and elemental impurities. In addition, this study explores recent advancements such as surface plasmon coupled emission technique, AI-assisted data analysis, portable sensors, and real-time monitoring technologies that enhance impurity profiling. The advantages and applications of the modern technologies are discussed, emphasizing their role in improving method efficiency, automation, and sustainability in connection with the impurity profiling.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"756-770"},"PeriodicalIF":1.9,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147445281","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

Ensuring Integrity of the Medicaid Drug Rebate Program. 确保医疗补助药品回扣计划的完整性。

IF 1.9 4区医学

Therapeutic innovation & regulatory science Pub Date : 2026-05-01 Epub Date: 2026-02-11 DOI: 10.1007/s43441-026-00933-z

Priyanka A Abraham, Joseph T Kannarkat, Benjamin N Rome, Jonathan J Darrow

引用次数: 0

Letter to the Editor: Battle Won but War Lost for Barth Syndrome Patients. 致编辑的信：巴斯综合症患者的胜利和失败。

IF 1.9 4区医学

Therapeutic innovation & regulatory science Pub Date : 2026-05-01 Epub Date: 2026-03-14 DOI: 10.1007/s43441-026-00951-x

Norman Stockbridge, Mary Ross Southworth

引用次数: 0

Innovative Multidimensional Quantitative Benefit-Risk Model for Effective Decision Making in Research and Development for Biopharmaceutical Industry. 面向生物制药行业研发有效决策的多维量化效益风险创新模型。

IF 1.9 4区医学

Therapeutic innovation & regulatory science Pub Date : 2026-05-01 Epub Date: 2026-02-21 DOI: 10.1007/s43441-025-00894-9

Maria E Verdugo, Fabio A Lievano, Melvin S Munsaka, Blanca M Martinez-Placencia

{"title":"Innovative Multidimensional Quantitative Benefit-Risk Model for Effective Decision Making in Research and Development for Biopharmaceutical Industry.","authors":"Maria E Verdugo, Fabio A Lievano, Melvin S Munsaka, Blanca M Martinez-Placencia","doi":"10.1007/s43441-025-00894-9","DOIUrl":"10.1007/s43441-025-00894-9","url":null,"abstract":"Medicinal products have benefits and risks that must be carefully balanced to inform decision making. The structured benefit-risk (BR) framework is a powerful approach not only to standardize a holistic BR assessment, but also to incorporate the patient perspective and guide the decisions and discussions of sponsors and regulatory agencies throughout the continuum of drug development. Structured BR assessment has been usually conducted using a qualitative approach during the late development stage. The use of quantitative models that can be applied throughout the drug development process may provide more objective BR information to support scientific recommendations to optimize and inform decisions for critical external and internal development opportunities. A new Multidimensional Benefit-Risk Integrated Evaluation (MBRIE) quantitative model was developed using key attributes of the structured BR assessment. Each attribute was evaluated by assigning a rating score ranging from 1 to 3 (low), 4-6 (medium), 7-10 (high). Also, two dimensions for comparative purposes were considered: standard of care (SOC) and probability of development success (PODS) (likelihood or favorability for development success). Graphical outputs were used to visualize and compare the ranking scores for each of the attributes across the two dimensions. This analysis implements a structured quantitative BR assessment approach earlier in drug development and through the drug lifecycle. The MBRIE model may be an innovative tool to facilitate solutions by fostering a collaborative culture that points to the true objective to improve patient outcomes.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"603-611"},"PeriodicalIF":1.9,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146776690","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

Challenges in Conducting Quantitative Patient-Centered Benefit-Risk Assessments: A Case Study in Ph + ALL with Immature Efficacy Data. 以患者为中心进行定量获益-风险评估的挑战：Ph + ALL疗效数据不成熟的案例研究

IF 1.9 4区医学

Therapeutic innovation & regulatory science Pub Date : 2026-05-01 Epub Date: 2026-03-09 DOI: 10.1007/s43441-026-00935-x

Ajibade Ashaye, Caitlin Thomas, Vamsi Kota, Nicolas Krucien, Kevin Marsh

{"title":"Challenges in Conducting Quantitative Patient-Centered Benefit-Risk Assessments: A Case Study in Ph + ALL with Immature Efficacy Data.","authors":"Ajibade Ashaye, Caitlin Thomas, Vamsi Kota, Nicolas Krucien, Kevin Marsh","doi":"10.1007/s43441-026-00935-x","DOIUrl":"10.1007/s43441-026-00935-x","url":null,"abstract":"Quantitative benefit-risk assessment (qBRA) can reveal how patients balance benefits and risks of cancer treatments. To align with qBRA good practice guidelines, researchers must address challenges including attribute value dependence, double counting, attribute dominance, and uncertainty associated with immature clinical trial outcomes. We present a case study illustrating these challenges in a qBRA of treatment preferences among patients with Philadelphia chromosome-positive acute lymphoblastic leukemia. Preferences were elicited using a discrete choice experiment (DCE). First, we explain how we mitigated potential dominance of survival outcomes by narrowing the range of overall survival (OS) durations that each participant considered. Second, we describe how we acknowledged the conceptual interaction between OS and duration of remission (DOR) attributes and tested for a statistical interaction. Third, we detail how we conducted qBRA with uncertain efficacy data using bivariate sensitivity analysis. Bivariate sensitivity analysis based on DCE-elicited preferences and head-to-head clinical performance data showed that if the considered treatments - ponatinib + chemotherapy and imatinib + chemotherapy - had equivalent efficacy, 52.9% (95% CI: 52.5%-53.4%) of DCE participants would be expected to choose ponatinib over imatinib. If ponatinib offered 10-month longer DOR and 20-month longer OS vs. imatinib, 71.6% (95% CI: 67.2%-76.0%) would choose ponatinib. Probabilistic sensitivity analyses showed that the probability of ≥ 70% of patients preferring ponatinib is 77.5% if ponatinib offers 15-month longer OS and DOR and 93.0% if it offers 45-month longer OS and DOR. Preference heterogeneity analyses identified that the overall choice probability results hold for all subgroups in nearly all scenarios.","PeriodicalId":23084,"journal":{"name":"Therapeutic innovation & regulatory science","volume":" ","pages":"617-628"},"PeriodicalIF":1.9,"publicationDate":"2026-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13110195/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147391030","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