Development and content validation of the Pediatric Oral Medicines Acceptability Questionnaires (P-OMAQ): patient-reported and caregiver-reported outcome measures

Abstract

Background Evolving regulatory guidelines recommend routine assessment of the acceptability of pediatric oral medicines throughout clinical development processes. However, such assessment is problematic owing to a lack of standard methods or criteria that define acceptability for children and their caregivers. This research aimed to identify the attributes of acceptability for targeted oral formulation types that are important to children, and to develop content-valid patient- and caregiver-reported outcome acceptability measures for use in the context of clinical drug development. Methods A concept-focused literature review and two advisory panel meetings involving researchers, clinicians, and measurement scientists were conducted to identify acceptability attributes that may be relevant to children taking targeted oral medicine formulations. The Pediatric Oral Medicines Acceptability Questionnaires (P-OMAQs), including patient (P-OMAQ-P) and caregiver (P-OMAQ-C) versions, were drafted to assess these attributes. Qualitative concept elicitation (CE) and cognitive debriefing (CD) patient and caregiver interviews were conducted to confirm key acceptability attribute concepts for measurement and to evaluate patient and caregiver ability to understand and respond to the questions. Results A full-text review of 40 articles identified 24 acceptability attributes that were categorized into 10 overarching domains and organized into a preliminary conceptual model. Feedback from the advisory panel refined the preliminary model. In total, 14 attributes were reported during the CE phase of the interviews (n = 23 pediatric patients, n = 13 caregivers); six attributes were included in the final model. The draft P-OMAQ was refined over four waves of CD interviews (n = 31 pediatric patients, n = 48 caregivers). The final version of the P-OMAQ-P is a 12-item questionnaire designed for young people aged 8–17 years. The P-OMAQ-C is a 19-item questionnaire designed for adult caregivers of young people aged 6 months to 17 years. There are two versions of each questionnaire: one with a 24-h recall period and one with a 7-day recall period. All items are answered on a 5-point numerical rating scale. Conclusions This research supports the content validity of the patient and caregiver versions of the P-OMAQ. Both questionnaires appropriately assess the acceptability of oral medicine formulations from the perspective of pediatric patients and their caregivers.

Assessing Palatability of a New Amphetamine Extended-Release Tablet Formulation for the Treatment of ADHD

“…Additionally, instruments have been developed to assess patient and/or caregiver acceptability of drug regimens. 23 Palatability as an adherence factor has traditionally focused on pediatric patients, generally in antibiotic development, but research has demonstrated that many of the same acceptability factors that apply to treatments for children also apply to adult patients. 16 As part of the development of the AMPH ER TAB, the palatability was assessed using a protocol that placed emphasis on several acceptance factors considered important to FDA.…”

Acceptability of Prednisolone in an Open-Label Randomised Cross-Over Study—Focus on Formulation in Children

“…A threshold of 80% of acceptance (e.g., acceptability score of 1 or 2) in the population examined is the current standard requirement [ 8 ]. A more patient- and caregiver-centred questionnaire (Paediatric Oral Medicines Acceptability Questionnaire) has recently been developed [ 9 ]. The questionnaire is yet to be validated but can be used in drug development to assess the acceptability of oral formulations [ 9 ].…”

“…A more patient- and caregiver-centred questionnaire (Paediatric Oral Medicines Acceptability Questionnaire) has recently been developed [ 9 ]. The questionnaire is yet to be validated but can be used in drug development to assess the acceptability of oral formulations [ 9 ]. We can expect further work in this area in search of the best methodology [ 8 , 9 ].…”

“…The questionnaire is yet to be validated but can be used in drug development to assess the acceptability of oral formulations [ 9 ]. We can expect further work in this area in search of the best methodology [ 8 , 9 ].…”

“…The questionnaire is yet to be validated but can be used in drug development to assess the acceptability of oral formulations [9]. We can expect further work in this area in search of the best methodology [8,9]. So far, very few studies (four studies were found in PubMed and clinical trial.gov) have investigated the acceptability of prednisolone in children with asthma [10][11][12][13].…”