Regulatory Alignment on Surrogate Endpoints for Slowly Progressive Ultra-Rare Neurological Diseases: Bridging the Gap Between Biomarker Science and Global Regulatory Acceptance [0.03%]
罕见神经退行性疾病替代终点的监管协调：弥合生物标志物科学与全球监管机构接受度之间的差距
Shihshuan Fang,Shenghan Chen
Shihshuan Fang
Background: For slowly progressive ultra-rare diseases, hard clinical endpoints such as mortality or sustained functional decline are often impractical within feasible trial timeframes. Surrogate biomarkers, including flu...
Career Paths to Drug Development and Regulatory Science Through the U.S. Food and Drug Administration [0.03%]
美国食品药品监督管理局视角下的药物研发和监管科学的职业路径
Susan M Abdel-Rahman,Gelareh Abulwerdi,Issam Zineh et al.
Susan M Abdel-Rahman et al.
Background: Across the U.S., pharmacy students have limited access to education and experiential training in non-patient facing roles that support the drug development and regulatory sectors. As a result, few cite jobs in...
Schizophrenia Impacts and Treatment Priorities: Results of An Externally-Led Patient-Focused Drug Development Meeting Survey [0.03%]
精神分裂症的影响及治疗优先后次：患者导向的药物开发会议调查结果
Ilse S Peterson,Ramaa Chitale,Theresa Frangiosa et al.
Ilse S Peterson et al.
Objective: Using survey data gathered for an Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting led by the schizophrenia community to inform the US Food and Drug Administration, this publication examines sc...
Guidance on Transparent Sponsorship Reporting in Industry Associated Delphi Studies (TRIAD): Recommendations Based on a Systematic Review [0.03%]
基于系统评价的透明行业相关德尔菲研究资助报告规范建议（TRIAD）
Christopher F Brewer,Thomas J G Chase,Edward Donnely
Christopher F Brewer
Background: The Delphi technique is a structured method for achieving expert consensus and is widely used in health research, including studies involving pharmaceutical sponsors. Although guidance exists for the conduct a...
Analysis of Serious Breach Cases Reported in the EU Clinical Trials Information System: Quantitative and Qualitative Assessment [0.03%]
欧盟临床试验信息系统的严重违规事件分析：定量与定性评估
Ewelina Wodnicka,Damian Swieczkowski
Ewelina Wodnicka
Background: Serious breaches are significant deviations that may compromise participant safety or data integrity in clinical trials. Public reports in the EU CTIS offer a valuable, though limited, opportunity to examine r...
Quality by Design to Mitigate Aggregation: Mechanistic Insights and Analytical Strategies for Biopharmaceutical Manufacturing [0.03%]
质量源于设计(Modi)防止生物制药的聚集：机制见解和分析策略
Anna Fedorko,Iryna Grynyuk,Jesús Alberto Afonso Urich
Anna Fedorko
This review highlights the problem of protein molecule aggregation, which represents a significant challenge in the field of biopharmaceuticals. Protein aggregation is critical because it can affect the efficacy and safety of biopharmaceuti...
35 Years of GLP in Germany: A Brief Overview About Its Successful History [0.03%]
德国GLP的35年历程——简述其成功历史
Sebastian Schmidtsdorff,Christian Strüh
Sebastian Schmidtsdorff
Good Laboratory Practice (GLP) emerged in the 1970s in response to serious deficiencies in the validity and reproducibility of preclinical safety data submitted in the USA. Non-adherence to scientific standards in the use of laboratory meth...
Methods for Assessing Therapeutic Innovation of Drugs: Changes at the Italian Medicine Agency [0.03%]
意大利药品管理局药物治疗创新评估方法的变革
Domenico Motola,Nicola Montanaro,Massimiliano Broccoli et al.
Domenico Motola et al.
Introduction: Therapeutic drug innovation is a fundamental point for patients, healthcare professionals, and national health services. In Europe, the assessment of the therapeutic innovation of drug is establish to the in...
FDA Accelerated Approval Disclosures on Direct-to-Consumer Prescription Drug Websites: Testing Presence, Prominence, and Wording [0.03%]
美国食品和药物管理局对面向消费者处方药网站的加速批准披露：测试存在、显著性和文法
Helen W Sullivan,Amie C O&#x;Donoghue,Mihaela Johnson
Helen W Sullivan
Objectives: The US Food and Drug Administration (FDA) provides a pathway, known as accelerated approval, intended to facilitate and expedite development and review for prescription drugs that treat serious or life-threate...
A Regulatory-Ready Bayesian Evidence Framework across Clinical Development and Pharmacovigilance: Practical Expectations from FDA, EMA, and ICH Guidance [0.03%]
一种监管就绪的贝叶斯证据框架贯穿于临床开发和药品警戒：来自FDA、EMA和ICH指南的实际期望值
Carlos G Bernaus,Maria Lilia Bournissen
Carlos G Bernaus
Bayesian methods are increasingly used in clinical development and pharmacovigilance, especially when evidence is sparse, accrues sequentially, or must incorporate external information. Translation of high-level regulatory principles into a...