Pediatric Research in Office Settings (PROS), Department of Research, American Academy of Pediatrics, Elk Grove Village, Illinois, USA.
Paediatr Drugs. 2011 Apr 1;13(2):119-24. doi: 10.2165/11584240-000000000-00000.
The recently renewed Best Pharmaceuticals for Children and Pediatric Research Equity Acts (BPCA/PREA) have continued industry incentives and opportunities for pediatric drug trials (PDTs). However, there is no current assessment of the capacity to perform PDTs.
The aim of this study was to deepen understanding of the capacity for US PDTs by assessing PDT infrastructure, present barriers to PDTs, and potential approaches and solutions to identified issues.
Pediatric clinical research experts participated in semi-structured interviews on current US pediatric research capacity (February-July 2007). An initial informant list was developed using purposive sampling, and supplemented and refined to generate a group of respondents to explore emerging themes. Each phone interview included a physician researcher and two health researchers who took notes and recorded the calls. Health researchers produced detailed summaries, which were verified by the physician researcher and informants. We then undertook qualitative analysis of the summaries, employing multiple coding, with the two health researchers and the physician researcher independently coding each summary for themes and subthemes. Coding variations were resolved by physician researcher/health researcher discussion and consensus achieved on themes and subthemes.
The 33 informants' primary or secondary roles included academia (n = 21), federal official (5), industry medical officer (8), pediatric research network leader (10), pediatric specialist leader (8), pediatric clinical pharmacologist (5), and practitioner/research site director (9). While most experts noted an increase in PDTs since the initial passage of BPCA/PREA, a dominant theme of insufficient US PDT capacity emerged. Subthemes included (i) lack of systems for finding, incentivizing, and/or maintaining trial sites; (ii) complexity/demands of conducting PDTs in clinical settings; (iii) inadequate numbers of qualified pediatric pharmacologists and clinician investigators trained in FDA Good Clinical Practice; and (iv) poor PDT protocol design resulting in operational and enrollment difficulties in the pediatric population. Suggested potential solutions for insufficient PDT capacity included (i) consensus-building among stakeholders to create PDT systems; (ii) initiatives to train more pediatric pharmacologists and educate clinicians in Good Clinical Practice; (iii) advocacy for PDT protocols designed by individuals sensitive to pediatric issues; and (iv) physician and public education on the importance of PDTs.
Insufficient US PDT capacity may hinder the development of new drugs for children and limit studies on the safety and efficacy of drugs presently used to treat pediatric conditions. Further public policy initiatives may be needed to achieve the full promise of BPCA/PREA.
最近更新的《儿童最佳药物和儿科研究公平法案》（BPCA/PREA）延续了激励制药行业为儿科临床试验（PDT）提供机会的政策。然而，目前还没有对开展 PDT 能力的评估。
本研究旨在通过评估 PDT 基础设施、目前 PDT 面临的障碍，以及识别问题的潜在方法和解决方案，深入了解美国 PDT 的能力。
2007 年 2 月至 7 月，儿科临床研究专家参与了关于美国当前儿科研究能力的半结构式访谈。采用目的性抽样方法制定了初始知情者名单，并进行了补充和完善，以生成一组受访者，以探索新出现的主题。每次电话访谈都包括一名医师研究员和两名健康研究员，他们负责记录和记录通话内容。健康研究员制作了详细的总结，由医师研究员和知情者进行核实。然后，我们对总结进行了定性分析，采用多重编码，两名健康研究员和医师研究员分别对每个总结进行主题和子主题编码。通过医师研究员/健康研究员的讨论解决编码差异，并就主题和子主题达成共识。
33 名受访者的主要或次要角色包括学术界（21 名）、联邦官员（5 名）、工业医疗官（8 名）、儿科研究网络负责人（10 名）、儿科专家负责人（8 名）、儿科临床药理学家（5 名）和从业者/研究基地主任（9 名）。尽管大多数专家指出自 BPCA/PREA 最初通过以来，PDT 的数量有所增加，但仍出现了美国 PDT 能力不足的主要问题。子主题包括（i）缺乏发现、激励和/或维持试验基地的系统；（ii）在临床环境中开展 PDT 的复杂性/需求；（iii）接受过 FDA 良好临床实践培训的合格儿科药理学家和临床研究员人数不足；以及（iv）PDT 方案设计不佳，导致儿科人群在操作和入组方面存在困难。针对 PDT 能力不足的潜在解决方案包括（i）利益相关者之间达成共识，建立 PDT 系统；（ii）开展更多儿科药理学家培训和临床医生良好临床实践教育的举措；（iii）倡导由对儿科问题敏感的个人设计 PDT 方案；以及（iv）对 PDT 的重要性进行医生和公众教育。
美国 PDT 能力不足可能会阻碍儿童新药的开发，并限制对目前用于治疗儿科疾病的药物的安全性和疗效的研究。可能需要进一步的公共政策举措来实现 BPCA/PREA 的全部承诺。
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