Better, Safer Medicines – Paediatric medicines research unit
“The vision of the Paediatric medicines research unit (PMRU) is to improve the health of babies, children and young people through research into safer medicines management and age-appropriate formulations.”
The PMRU is a collaboration between Alder Hey, Liverpool Women’s NHS Foundation Trust, Edge Hill University, Liverpool John Moore’s University, the University of Central Lancashire and the University of Liverpool. It is also supported by industry, clinical experts and children and young people, both nationally and internationally.
Through multiple academic, clinical, and commercial partnerships, the PMRU has carried out research that has international impact, including discovering new drug safety signals of international importance, co-developing novel age-appropriate formulations and evaluating interventions in clinical pharmacy practice designed to improve patient safety and service delivery.
Key Themes
Drug Safety Sciences
Many medicines prescribed for children are not licensed for use in this age group. Many common medicines have not been tested properly or at all in children; frequently medicines are administered to this age group based on how they work in adults and the doses used are often a best guess. This means that children are at a high risk of suffering serious and unexpected side effects, known as adverse drug reactions, from many medicines prescribed by doctors or available at the chemist.
Liverpool has an impressive heritage in drug safety sciences: the PMRU has led an extensive research programme into adverse drug reactions (ADRs) in children. The requirement for validated tools to characterise ADRs in children has grown as clinical trials of new and existing drugs have been driven by the EU and FDA regulations for paediatric medicines.
Medicines Optimisation
Medicines optimisation aims to help patients to make the most out of their medicines and ensure value for money from medicines. The Royal Pharmaceutical Society outlines four principles of medicines optimisation, which are, to understand the patient’s experience of medicines, the use of evidence-based medicines, ensuring medicines use is as safe as possible and making medicines optimisation part of routine practice.
Formulations
Many medicines are only available in dosage forms which are suitable for use in adults. For instance, small children are typically unable to swallow large tablets or capsules. When developing and prescribing medicines for children it is important to consider how suitable the product (or formulation) is for them. One important factor is the acceptability of the medicine to the child who needs to take it. Acceptability includes how straightforward it is to prepare the dose, how easy it is to take and how it tastes.
Due to the lack of licensed ‘authorised’ medicines available for children, medicines often need to be manipulated (e.g. tablets are split or crushed; capsules are opened and their contents sprinkled onto food or into a drink). This is called ‘off-label’ use as the medicine is being used outside the terms of its marketing authorisation. Other considerations in paediatrics include a selection of the most appropriate strength of medicine available and the suitability of its excipients. A careful risk-benefit assessment is needed when age-appropriate formulations (AaFs) are unavailable to ensure safe, effective, and appropriate medicines can be successfully administered to children, in an acceptable manner.
The PMRU team is working with the pharmaceutical industry to help co-design AaFs for children, which are ‘age-appropriate’ and designed with the needs of children in mind.