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. 2020 Oct 1;4(1):80.
doi: 10.1186/s41687-020-00246-1.

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

Diane M Turner-Bowker  1 , Kristina An Haack  2 , Meaghan Krohe  3 , Andrew Yaworsky  3 , Norma Vivas  4 , Masami Kelly  3 , Godhuli Chatterjee  5 , Emily Chaston  3 , Erin Mann  6 , Matthew Reaney  7
Affiliations

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

Diane M Turner-Bowker et al. J Patient Rep Outcomes. .

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.

Keywords: Acceptability; Caregivers; Clinical outcome assessments; Cognitive debriefing; Concept elicitation; Oral formulations; Pediatric oral medicines; Questionnaires; Systematic literature review.

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Conflict of interest statement

DMT-B, AY and MKr are employed by Adelphi Values, which has received funding from Sanofi to conduct the qualitative research reported in this paper. EC, EM and MKe were paid employees of Adelphi Values at the time of this study. KA, NV, and GC are paid employees of Sanofi. MR was a paid employee of Sanofi at the time of this study. KA and MR are also stockholders of Sanofi. Editorial support, funded by Sanofi US Services Inc., was provided by Alice Fodder, Alistair Ray, and Jamie Singer of PharmaGenesis Oxford Central, Oxford, UK. Adelphi Values conducted the qualitative research, funded by Sanofi US Services Inc.

Figures

Fig. 1
Fig. 1
Literature-based preliminary conceptual model of oral formulation acceptability attributes
Fig. 2
Fig. 2
Draft conceptual model of targeted oral formulation acceptability attributes following advisory panel
Fig. 3
Fig. 3
Final conceptual model of targeted oral formulation key acceptability attributes following patient and caregiver interviews
See this image and copyright information in PMC

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