ACCEPT- Methodology to assess acceptability of paediatric medicines.
Research type
Research Study
Full title
ACCEPT: Methodology to assess acceptability of paediatric medicines. Evaluation of methodology used in the assessment of acceptability of paediatric medicines: A mixed methods approach
IRAS ID
179684
Contact name
Punam Mistry
Contact email
Clinicaltrials.gov Identifier
RG_15-119, Research Governance Number
Duration of Study in the UK
1 years, 0 months, 0 days
Research summary
Children’s pharmacotherapy is a niche market differing from adults, prompting the need to develop unique paediatric formulations.
Any new medicines developed now, are required to consider paediatric use based on European legislation introduced in 2007. Paediatric regulations require medicinal products to be considered and any Paediatric Investigation Plans (PIP) are subject to agreement by the European Medicines Agency (EMA). Pharmaceutical companies have to demonstrate that these new medicines are safe and effective in children. Patient needs must be met when medicines are designed, with the purpose to deliver the intended product performance. With this in mind, there is necessity to assess the acceptability of paediatric medicines during development stages.
Acceptability has been defined as, “an overall ability of the patient and caregiver (defined as ‘user’) to use a medicinal product as intended (or authorised)” (Kozarewicz et al., 2014). Acceptability is determined by the characteristics of the product and the user. Patient acceptability of a medicinal product is likely to have a significant impact on adherence and consequently, on its safety and efficacy. However, there are no instructions or guidance provided by the regulatory authorities of how “acceptability” is measured. Animal studies have proposed that voluntary acceptance rates should be >80% in dogs, and >70%, for all other species, but no values have been calculated for paediatric patients (EMA, 2014).
Pharmaceutical companies play a crucial role in the development of an acceptable formulation for paediatric use; however, determination as to whether medicinal products are acceptable should involve patients; as specified by the EMA guidelines.
Previous literature (Kozarewicz et al., 2014, Thompson et al., 2014) has reported the lack of a standardised method to assess acceptability and highlighted that there is growing need for a standardised approach.
This protocol describes a study that will identify whether commonly used assessment tools are efficient methods to measure the acceptability of routinely administered medicines, for acute illnesses within inpatient paediatric wards.
Outcomes of this study will aid understanding of which tools should be used to evaluate paediatric medicinal products in the future.
REC name
London - Surrey Borders Research Ethics Committee
REC reference
15/LO/1253
Date of REC Opinion
20 Jul 2015
REC opinion
Further Information Favourable Opinion