Conducting Drug Treatment Trials in Children: Opportunities and Challenges.

IF 3.1 Q2 PHARMACOLOGY & PHARMACY
Pharmaceutical Medicine Pub Date : 2024-05-01 Epub Date: 2024-05-10 DOI:10.1007/s40290-024-00523-0
Damir Erceg, Marina Jakirović, Luka Prgomet, Marina Madunić, Mirjana Turkalj
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引用次数: 0

Abstract

Children were often referred to as "therapeutic orphans" in the past due to different reasons such as ethical, regulatory, economic, scientific, etc., ones. They were exposed to avoidable risks while missing out on therapeutic advances. Pediatric patients have suffered from a lack of scientific and regulatory standards (e.g., proper drug testing, authorization of medicines for their use, etc.), although the pharmaceutical legislative framework, which ensures the high standards of safety, quality, and efficacy of medicinal products for use in adults, was developed primarily in response to past "drug disasters," mainly involving children. The adoption of pediatric regulatory initiatives first in the USA and then in Europe and other countries and regions has significantly changed the worldwide frameworks and permanently changed pediatric drug research and development. This article tries to give various perspectives with historical context, a review of the different challenges and opportunities as well as important stakeholders in pediatric drug development. The pediatric trial networks are probably the most important stakeholder that enables efficient patient recruitment, access to better resource utilization, and global collaboration of different stakeholders necessary for performing quality and well-designed clinical trials.

在儿童中开展药物治疗试验:机遇与挑战。
过去,由于伦理、监管、经济、科学等不同原因,儿童常常被称为 "治疗孤儿"。他们承受着本可避免的风险,却错失了治疗上的进步。尽管药品立法框架主要是针对过去主要涉及儿童的 "药物灾难 "而制定的,该框架确保了成人用药产品在安全、质量和疗效方面的高标准,但由于缺乏科学和监管标准(如适当的药物测试、用药授权等),儿科患者一直深受其害。首先在美国,然后在欧洲及其他国家和地区,儿科监管举措的采用极大地改变了全球框架,并永久性地改变了儿科药物研发。本文试图从不同角度介绍儿科药物研发的历史背景、不同的挑战和机遇以及重要的利益相关者。儿科试验网络可能是最重要的利益相关者,它能有效招募患者,更好地利用资源,并促进不同利益相关者的全球合作,这是开展高质量和精心设计的临床试验所必需的。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Pharmaceutical Medicine
Pharmaceutical Medicine PHARMACOLOGY & PHARMACY-
CiteScore
5.10
自引率
4.00%
发文量
36
期刊介绍: Pharmaceutical Medicine is a specialist discipline concerned with medical aspects of the discovery, development, evaluation, registration, regulation, monitoring, marketing, distribution and pricing of medicines, drug-device and drug-diagnostic combinations. The Journal disseminates information to support the community of professionals working in these highly inter-related functions. Key areas include translational medicine, clinical trial design, pharmacovigilance, clinical toxicology, drug regulation, clinical pharmacology, biostatistics and pharmacoeconomics. The Journal includes:Overviews of contentious or emerging issues.Comprehensive narrative reviews that provide an authoritative source of information on topical issues.Systematic reviews that collate empirical evidence to answer a specific research question, using explicit, systematic methods as outlined by PRISMA statement.Original research articles reporting the results of well-designed studies with a strong link to wider areas of clinical research.Additional digital features (including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations) can be published with articles; these are designed to increase the visibility, readership and educational value of the journal’s content. In addition, articles published in Pharmaceutical Medicine may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand important medical advances.All manuscripts are subject to peer review by international experts. Letters to the Editor are welcomed and will be considered for publication.
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