Improving medicines for children

As a parent or carer of a child have you ever considered the following:

• How children’s medicines are made?
• How are they tested and whether they are tested in children?
• Does mixing/crushing medicines into foods/drinks change the products efficacy and whether it can be harmful to your child?

My point is do you ever think about the science behind medicine development in children? I can’t speak for every parent, or carer but it’s not something that was at the forefront of my mind when my children had raging temperatures, vomiting bugs, or chest infections. My goal was to give them whichever medicine that the doctor prescribed and hoped they got better quickly, regardless of how the medicine tasted or how it was administered. I was raised by the saying “the worse the medicine tasted, the quicker you get better!”

Of course, I now know that this was a tactic just to get me to take my medicine. There are hundreds more tactics and tricks to do this, especially from parents who have children with chronic conditions and need to take multiple medicines on a daily basis. But, how are these medicines developed and what are scientists and researchers doing to improve better and safer medicines for children? Before we begin addressing some of these questions, here are some facts about paediatric medicines:

FACTS ABOUT PAEDIATRIC MEDICINES

·      Due to the lack of age-appropriate formulations, children are commonly given medicines that are licensed for use in adults.

·      Although flexible solid oral dosage forms of medicines are an attractive option for children as they reduce the chances of unpleasant medicine tastes, the cost may be 2 to 3 times higher than the pill form for adults

·      Healthcare professionals and parents often use fractions of adult dosages forms or prepare makeshift prescription of medicines by crushing tablets or dissolving portions of capsules in water

·      There is little knowledge about the effects certain medicines can have on children. This is partly because fewer clinical trials are conducted in children than in adults.

www.who.int/childmedicines/facts/en/

ADVANCES IN PAEDIATRIC MEDICINE DEVELOPMENT

I believe as a parent or carer looking after a child, we would want them to be able to take medicines that are adapted to their needs. So out with the ‘tactics’ and in with the more child acceptable formulations.

This has been recognised by the European Union and US Regulations and the amount of work done to study medicines for children is significantly greater than it was 10 years ago.  However advancing research also involves gaining a better understanding of what children and young people think about taking their medicines and to assess their requirements and acceptability of formulations.

DEVELOPING FORMULATIONS THAT ARE ACCEPTABLE

The European Medicine Agency (EMA) guidance[1] now states that patient acceptability must be an integral part of paediatric formulation development.

But what do we mean by ‘acceptable?’ and ‘acceptable’ to whom?

Acceptability is an overall ability of the patient and caregiver to use a medicinal product as intended (or authorised) [2]. Acceptability of a medicine is likely to impact hugely on the patient’s adherence and consequently is likely to have an impact on safety and efficacy of the product.  As a general rule, acceptability aspects should be embedded in the early development of the medicines and evaluated during the clinical study with patients from the target age group(s).

WHAT IS ACCEPTABLE TO CHILDREN AND YOUNG PEOPLE?

A key aspect of formulation success is developing a medicine that is acceptable to children and young people. Formulation factors that could impact acceptability in children include:

• Taste
• Smell
• Mouthfeel
• Aftertaste and swallowability

”By addressing these factors individually, we can understand which are most important to children and young people when identifying acceptable, age-appropriate formulations for this population. Researchers from the Paediatric Medicines Research Unit (a collaboration between Alder Hey, Liverpool Women’s NHS Foundation Trust, the University of Liverpool, Liverpool John Moores University and the University of Central Lancashire) have been doing exactly that.  Their vision is to improve the health of babies, children and young people by research and development of better and safer medicines, in collaboration with children, young people and families.

In my next blog we focus on what children and young people have to say about the medicines they take and what type of formulations they would like researchers to develop in the future.

For further information about paediatric drug formulations click here.

[1] EMA, 2013. Guideline on pharmaceutical development of medicines for paediatric use. Available at: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2013/07/WC500147002.pdf

[2] Kozarewicz P. Regulatory perspectives on acceptability testing of dosage forms in children. Int J Pharm 2014; 469: 245-248