Pediatric Clinical Trials in Mainland China Over the Past Decade (From 2009 to 2020)

In mainland China, there remains a shortage of pediatric drugs. The Chinese government has recently launched policies and incentives to encourage pediatric drug development and clinical trials. However, data on the characteristics or development trends of these trials are limited. In this review, we extracted source data from the Chinese Clinical Trials Registry and Information Transparency Platform and systematically reviewed the pediatric clinical trials conducted in mainland China from 2009 to 2020, a comprehensive process evaluation of the pediatric drug clinical trials development in the past decade, providing data support to policy makers and industry stakeholders. We included 487 pediatric clinical trials. Over the past decade, the number of pediatric trials has increased, especially since 2016. The most common therapeutic areas were infectious diseases (n = 108, 22.2%), agents for preventive purpose (n = 99, 20.3%), and neurological and psychiatric diseases (n = 71, 14.6%). The number of clinical trials involving epilepsy (39, 10.1%), asthma (33, 8.5%), and influenza (24, 6.2%) were the highest. The distribution of leading institutions is unbalanced in mainland China, with most units in East China (34.0%) and few in Southwest China (6.9%). China has made progress in improving the research and development environment of pediatric drugs and increasing pediatric trials. However, a wide gap in pediatric drug development and clinical trials quality exists between China and the developed countries. The pharmaceutical industry in China has faced grim setbacks, including study duplication, lack of innovation, poor research design, and unbalanced resource allocation. Thus, we suggest that the Chinese government should adjust their policies to improve innovation and clinical design capacity, and optimize resource allocation between regions.

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Ushering in the next generation of precision trials for pediatric cancer

Science ◽

10.1126/science.aaw4153 ◽

2019 ◽

Vol 363 (6432) ◽

pp. 1175-1181 ◽

Cited By ~ 15

Author(s):

Steven G. DuBois ◽

Laura B. Corson ◽

Kimberly Stegmaier ◽

Katherine A. Janeway

Keyword(s):

Clinical Trials ◽

Drug Development ◽

Pediatric Cancer ◽

Case Reports ◽

Precision Oncology ◽

Genomic Profile ◽

Key Factors ◽

Pediatric Drug ◽

The Past ◽

Pediatric Cancers

Cancer treatment decisions are increasingly based on the genomic profile of the patient’s tumor, a strategy called “precision oncology.” Over the past few years, a growing number of clinical trials and case reports have provided evidence that precision oncology is an effective approach for at least some children with cancer. Here, we review key factors influencing pediatric drug development in the era of precision oncology. We describe an emerging regulatory framework that is accelerating the pace of clinical trials in children as well as design challenges that are specific to trials that involve young cancer patients. Last, we discuss new drug development approaches for pediatric cancers whose growth relies on proteins that are difficult to target therapeutically, such as transcription factors.

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Physiologically Based Pharmacokinetic Modeling: Methodology, Applications, and Limitations with a Focus on Its Role in Pediatric Drug Development

Journal of Biomedicine and Biotechnology ◽

10.1155/2011/907461 ◽

2011 ◽

Vol 2011 ◽

pp. 1-13 ◽

Cited By ~ 55

Author(s):

Feras Khalil ◽

Stephanie Läer

Keyword(s):

Clinical Trials ◽

Drug Development ◽

Age Groups ◽

Modeling Technique ◽

Pbpk Modeling ◽

Pediatric Drug ◽

Pediatric Drug Development ◽

Pbpk Models ◽

Physiologically Based Pharmacokinetic ◽

Physiologically Based

The concept of physiologically based pharmacokinetic (PBPK) modeling was introduced years ago, but it has not been practiced significantly. However, interest in and implementation of this modeling technique have grown, as evidenced by the increased number of publications in this field. This paper demonstrates briefly the methodology, applications, and limitations of PBPK modeling with special attention given to discuss the use of PBPK models in pediatric drug development and some examples described in detail. Although PBPK models do have some limitations, the potential benefit from PBPK modeling technique is huge. PBPK models can be applied to investigate drug pharmacokinetics under different physiological and pathological conditions or in different age groups, to support decision-making during drug discovery, to provide, perhaps most important, data that can save time and resources, especially in early drug development phases and in pediatric clinical trials, and potentially to help clinical trials become more “confirmatory” rather than “exploratory”.

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