Path towards efficient paediatric formulation development based on
partnering with clinical pharmacologists and clinicians, a c4c expert
group White paper.
Improved global access to novel age-appropriate formulations for
paediatric subsets, either of new chemical entities or existing drugs,
is a priority to ensure that medicines meet the needs of these patients.
However, despite regulatory incentives, the introduction to the market
of paediatric formulations still lags behind adult products. This is
mainly caused by additional complexities associated with the development
of acceptable age-appropriate paediatric medicines. This position paper
proposes the use of a paediatric Quality Target Product Profile (pQTPP)
as an efficient tool to facilitate early planning and decision making
during the children-centric formulation design for new chemical
entities, or to repurpose/reformulate off-patent drugs. Essential key
attributes of a paediatric formulation are suggested and described.
Moreover, greater collaboration between formulation experts and clinical
colleagues, including healthcare professionals, is advocated to lead to
safe and effective, age-appropriate medicinal products. Acceptability
testing should be a secondary endpoint in paediatric clinical trials to
ensure post-marketing adherence is not compromised by a lack of
acceptability. Not knowing the indications and the related age groups
and potential dosing regimens early enough is still a major hurdle for
efficient paediatric formulation development; however the proposed pQTPP
could be a valuable collaborative tool for planning and decision making
to expedite paediatric product development.