Abstract

Children are not small adults, but rather a distinct and heterogeneous patient group with specific therapeutic needs. Child development entails dynamic processes inherent to growth from birth into adulthood, and children face a scope of diseases different than those of adults. Accordingly, safe and effective paediatric pharmacotherapy requires medicines adjusted to the needs, acceptability and preferences (of each subpopulation) of children. Moreover, child-specific antibiotics are one key public health area of interest, due to their potential to fight bacterial infections that are among leading causes of death in early life. Despite recent progress, more work lies ahead, and so the aim of the present thesis was to document advancements with respect to priority medicines for children, and conduct additional research on age-appropriate formulations and use of antibiotics in children across different settings.

The adoption of the EU Paediatric Regulation has lead to a fundamental change of culture, as the incentives and regulatory requirements have induced companies to screen new adult products for their potential paediatric value. Due to the modest impact on high priority and unmet paediatric needs (e.g. oncology, pain, neonatal morbidity), a number of corrective measures have now been taken, so that medicines for children are developed independently from adult indications. We observed shifting trends toward oral solid formulations with a focus on innovative flexible, multiparticulate and dispersible preparations. Their advantages include a provision of easy, safe and convenient dose delivery and – in terms of resource-limited settings – superior stability in hot climate zones, easier transport and storage, as well as a reduction in problems with confidentiality and social stigma. However, our research suggests that more age-appropriate antibiotics that facilitate the treatment of children exist globally than currently included on the World Health Organization Model List for Essential Medicines for Children. Hence, it is important to create a global platform to provide the information about the benefits, shortcomings and availability of age-appropriate formulations for children, and advocate for their rational use.

Then again, the rational use of medicines in children has been inadequately studied. Our study on trends in prescribing patterns for acute childhood infections in primary care in developing and transitional countries showed a mixed progress with most of the treatment aspects of infections remaining sub-optimal over time. In high-income countries prescribing is often not rational either, as we found incorrect use of newer broad spectrum antibiotics and/or inappropriate use of antibiotics for viral infections. Moreover, we investigated a specific form of irrational use of antibiotics, self-medication, by patients in Macedonia. This entails either getting antibiotics at the pharmacy without a prescription or using leftover antibiotics from previous treatments. We found that interventions had a small effect on self-medication of children, but not their parents, and the effect disappeared later. Thus, solutions need to focus on multifaceted and multilevel interventions that define local barriers, and integrate the promotion of the rational use of antibiotics for children within health systems.

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