Young patients' involvement in a composite endpoint method development on acceptability for paediatric oral dosage...

Area of Science:

• Paediatric medicine development
• Patient engagement in pharmaceutical research
• Drug formulation acceptability

Background:

• The European Medicines Agency (EMA) requires evaluation of medicine acceptability in paediatric drug development.
• A standardized method for assessing 'swallowability' and 'palatability' into a composite endpoint for acceptability was developed.
• Critical review by young patients is needed to validate this new acceptability testing method.

Purpose of the Study:

• To assess the suitability of the composite endpoint on acceptability methodology with young patients.
• To investigate the importance of patient involvement in medicine development and acceptability method creation.
• To determine the most relevant elements for assessing medicine acceptability in children and adolescents.

Main Methods:

• A focus group meeting was conducted with the KIDS Barcelona young people advisory group (ages 16-23).
• Electronic questionnaires were used to investigate patient involvement and preferences for acceptability assessment.
• The relevance of EMA-listed acceptability elements was evaluated using stickers and questionnaires.

Main Results:

• Young people's involvement in method development was highly rated.
• A 5-point smiley Likert Scale was preferred for 6-11 year olds, and a Visual Analogue Scale for adolescents.
• Palatability and swallowability were identified as most relevant, while medicine color was least relevant.

Conclusions:

• The study reinforced the value of involving young people in the medicine lifecycle and acceptability method development.
• Further focus groups with diverse young populations across Europe are planned.
• This research supports the development of more acceptable paediatric medicines.