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By: Jennifer Walsh

Jennifer WalshSince the introduction of pediatric regulations in the USA (Pediatric Research Equity Act and Best Pharmaceuticals for Children Act) and Europe, there has been a huge  increase in the number of medicines that have information about their use for children. However, despite the expansion of research in the development of pediatric medicinal products, there is still an unmet need for off-patent medicines for children. Incentives in the form of funding support and data protection have not markedly increased the number of off-patent pediatric product licences. 

Due to major changes in growth and development that occur during infancy and childhood, the needs of pediatric patients are very different than those of adult patients, which adds to the complexity of pediatric product formulation. The selection of dosage form that must be suitable for the intended patient population is of key importance as it is well recognized that the ability to take certain dosage forms is dependent on age; a newborn baby or infant is not able to swallow a large tablet or use a dry power inhaler.

Age-related changes in pharmacokinetics and pharmacodynamics also occur and can lead to differences in dose requirements between adults and children as they grow. Hence, there is a need for flexible dosing. These physiologic differences can also affect the way in which pediatric patients handle excipients, and as a result, their inclusion in the pediatric formulation must be justified.

In addition to physiologic differences, many parents, caregivers, and health care professionals will also be familiar with the difficulty in getting a child to take an aversive tasting medicine, and so the palatability and overall patient acceptability of pediatric medicines are important aspects that must be addressed during the formulation development program. Another factor to consider is how the pediatric medicine will be given to the patient, including the need for and testing of an administration device.

It is encouraging to note that to help address some of the challenges described above, a number of work groups have been established (e.g., Institute of Pediatric Innovation, Global Research in Paediatrics, European Paediatric Formulation Initiative) that bring together industry, academia, clinicians, and regulators to facilitate the development of pediatric medicines. However, it is vital that the activities of these groups are supported and their outputs disseminated to avoid duplication of research.

A recently published article in the Pediatric Drug Development and Dosage Form Design special theme of AAPS PharmSciTech describes some of the key formulation challenges that were faced during the development of a pediatric dosage form of the off-patent drug enalapril as part of a European Union (EU) Paediatric Investigation Plan. The Labeling of Enalapril from Neonates to Adolescents (LENA) project has received funding from the EU’s Seventh Framework Programme (FP7/2007-2013) under grant agreement n°602295. Research on formulation of therapeutics for special populations continues to be an important topic. We must do the research today to affect tomorrow.

The author has provided consultancy support to the LENA project but received no financial support for the creation of this blog or manuscipt.

Jennifer Walsh, Ph.D., is director of Jenny Walsh Consulting Ltd. and offers pharmaceutical development support especially in the area of paediatric product development. She is a member of the European Paediatric Formulation Initiative (EUPFI).