OBJECTIVES: Existing clinical outcome measures of the core features of autism, social communication and interaction, as well as restricted and repetitive behaviours have shortcomings in assessing treatment effects. Goal attainment scaling (GAS) offers the potential to measure individual experiences, while Global Impression (GI) items provide a holistic assessment of treatment benefit. The objective of this study was to develop and cognitively debrief a novel GAS and GI items in paediatric autism.

METHODS: A comprehensive literature review was first completed to understand potential treatment benefits and goals in autism. This was followed by a qualitative study with two waves of interviews. The first ‘Concept Elicitation’ interview round (N=40) included autistic adolescents aged 12-17 years, parents of autistic children aged 5-17 years and clinicians, the purpose was to generate personalised goals and GI items. The second ‘Cognitive Debriefing’ interview round (N=39) was to obtain feedback on a draft GAS manual and GI items. An external expert panel provided input into all aspects of the study.

RESULTS: Twenty-five personalised goals were generated for core characteristics (communication, socialisation, restrictive and repetitive behaviours) and associated characteristics of autism for children and adolescents. Feedback from the cognitive debriefing supported the use of personalised goals. Adolescents/parents found these helpful, and clinicians recommended using these goals in clinical research. Nine GI items were generated for assessing impact/difficulty and change in symptoms; these can be rated by adolescents, parents of children with autism and clinicians.

CONCLUSIONS: As autism is a heterogeneous condition, use of personalised goal measures and tailored GI items may overcome some of the challenges with existing outcome measures. The FDA’s Patient-Focussed Drug Development Guidance-4 highlights the patient focused nature of outcomes like GAS in clinical trials. These findings will inform future measurement strategies for clinical studies in paediatric autism.