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

Background: In line with the European Paediatric Regulation, the European Medicines Agency (EMA) asks for investigation of a medicine's acceptability in paediatric medicines development. A standardised acceptability testing method combining the outcome of "swallowability" and "palatability" assessments to a "composite endpoint on acceptability" was recently developed. Before this method's suitability for selection of the most acceptable drug formulation of a new medicine for children can be broadly recommended, the acceptance and relevance of such established acceptability needs the critical review and input from young patients with understanding of the medicines development methodology. The benefit of involving patients in drug product development, clinical research and innovation is well established.

Methods: During a focus group meeting with the KIDS Barcelona (young people advisory group, age 16-23 years) the suitability of the "composite endpoint on acceptability" methodology was assessed. Via electronic questionnaires the importance of involving patients in the medicines development and in the acceptability method development was investigated. Questions on how best to determine palatability and swallowability were asked. The relevance of all EMA-listed acceptability elements was assessed via coloured and numbered stickers and questionnaires.

Results: The results showed that the involvement of young people in the medicines and acceptability method development was rated high. The group worked out that a 5-point smiley Likert Scale is preferred for assessing acceptability by 6-11 year old patients, while a Visual Analogue Scale is preferred for collecting adolescents' opinion. The ranking of the EMA-listed acceptability elements showed that palatability and swallowability are the most relevant parameters, while colour of the medicine was rated as least relevant. These results, established face-to-face, were confirmed in a repeat of the ranking through an electronic questionnaire, completed by the participants individually and remotely, 5 weeks later.

Conclusion: This work reinforced the need and value to involve young people in the medicines lifecycle, and specifically in this acceptability method development. As next step other focus group meetings with more young people from different European countries are planned.