Yen Elizabeth, Davis Jonathan M, Milne Christopher-Paul
1 Department of Pediatrics, The Floating Hospital for Children at Tufts Medical Center, Boston, MA, USA.
2 The Tufts Clinical and Translational Research Institute, Boston, MA, USA.
Ther Innov Regul Sci. 2019 Sep;53(5):609-614. doi: 10.1177/2168479019837522. Epub 2019 Apr 14.
Surveys evaluating industry experience with performing pediatric studies under the Best Pharmaceutical for Children Act (BPCA) and Pediatric Research Equity Act (PREA) regulatory regime were conducted by Tufts Center for the Study of Drug Development (Tufts CSDD) in 2000, 2006, and 2016. These survey results are being used to assess the future impact of regulatory incentive programs on generating pediatric specific labeling information and development of age-appropriate drug formulations. A second perspective will be provided through the experience and expertise of neonatal/pediatric clinicians and researchers with a focus on the urgent need for the study of new and existing drugs in this vulnerable population (especially with 90% of drugs in neonates still being used off-label). This group will also address the impact of existing regulations and the likely trajectory of future pediatric drug development efforts after nearly 2 decades of regulatory incentives (both mandatory and voluntary). Finally, this review will provide input on approaches that are needed to continue to advance pediatric drug development with an emphasis on rare diseases.
塔夫茨药物开发研究中心（Tufts CSDD）于2000年、2006年和2016年开展了多项调查，评估制药行业在《儿童最佳药物法案》（BPCA）和《儿科研究公平法案》（PREA）监管制度下开展儿科研究的经验。这些调查结果正被用于评估监管激励计划对生成儿科特定标签信息以及开发适合儿童年龄的药物剂型的未来影响。另一个视角将通过新生儿/儿科临床医生和研究人员的经验与专业知识来呈现，重点关注在这一脆弱人群中研究新药和现有药物的迫切需求（尤其是90%的新生儿用药仍为非标签用药）。该小组还将探讨现有法规的影响以及经过近20年的监管激励（包括强制性和自愿性）后未来儿科药物开发努力可能的发展轨迹。最后，本综述将为继续推进儿科药物开发（重点是罕见病）所需的方法提供意见。
