OBJECTIVES: Goal Attainment Scaling (GAS) is a patient-centric outcome measure that quantifies the impact of an intervention on individualized goals. Clinicians work with patients and/or caregivers to identify goals and build goal scales unique to each patient and clinically meaningful. Here we investigated the feasibility of using a goal quality assessment inventory to ensure a standardized approach to high-quality goal development across patients with heterogeneous symptoms.

METHODS: Four clinician experts were trained in setting psychometrically adequate goals with caregivers of individuals with SCN2A-associated developmental and epileptic encephalopathies. The inventory was developed to be used in real-time goal quality assurance within a fast-paced clinical trial environment and included items (n=20) such as language specificity, outcome orientation, patient-friendly language etc. Two assessors used the inventory to independently evaluate all the goal scales. Interrater reliability (IRR) was evaluated with Cohen’s kappa (95% confidence intervals, CI) and Scott's Pi. Percentage agreement between the two raters was also calculated.

RESULTS: Ten participants (Mean age =8.2 years, SD=5.62, range 3.4-20.4; Nmale=8) and their caregivers took part in goal-setting interviews with four GAS raters. Across all goals (n=30), Cohen's Kappa was 0.395 [CI 0.275 0.515] and Scott’s Pi was 0.319, suggesting fair IRR. The overall percent agreement between raters was 88.8%.

CONCLUSIONS: There is a critical need to standardize implementation of personalized endpoints like goal attainment scaling to ensure consistency across sites/patients. We recommend implementation of a two-step goal setting process and real-time goal quality assessment using the inventory, providing GAS interviewers with feedback tailored to each scale which can be used to finalize goals with patients before treatment administration. Although additional studies are needed, this work suggests that goal quality assessment inventories may be a helpful component in a standardized approach to goal-setting in clinical development.