Key Challenges in the Search for Innovative Drug Treatments for Special Populations. Converging Needs in Neonatology, Pediatrics, and Medical Genetics

Children

Stuart MacLeod¹ 

[1] Faculty of Medicine, University of British Columbia, Department of Pediatrics, British Columbia Children’s Hospital Research Institute, 950 West 28th Avenue, Vancouver, British Columbia, Canada V5Z 4H4;

关键词: neonatology;    pediatrics;    rare disorders;    clinical trials;    clinical pharmacology;    pharmacy;    innovative therapy;    human resources;   

DOI  :  10.3390/children4080068

来源: DOAJ

【 摘 要 】

The explosion of knowledge concerning the interplay of genetic and environmental factors determining pathophysiology and guiding therapeutic choice has altered the landscape in pediatric clinical pharmacology and pharmacy. The need for innovative research methods and design expertise for small clinical trials to be undertaken in sparse populations has been accentuated. At the same time, shortfalls in critical human resources represent a key challenge, especially in low- and middle-income countries where the need for new research and education directions is greatest. Unless a specific action plan is urgently developed, there will be a continuing gap in availability of the essential expertise needed to address treatment challenges in special patient populations such as neonates, patients suffering from rare or neglected diseases, and children of all ages.

【 授权许可】

Unknown