OBJECTIVES: Goal attainment scaling (GAS) is a patient-centered outcome that quantifies the extent of goal attainment after an intervention. The recent FDA patient-focused drug development draft guidance 4 saw GAS as valuable for conditions with heterogeneous symptoms, where standardized measures often can be insensitive to meaningful change. We reviewed a decade’s use of this personalized endpoint in clinical trials.

METHODS: We searched ClinicalTrials.gov for “goal attainment scale” and “goal attainment scaling” between January 1^st, 2014 to December 18^th, 2023. Trials which were terminated, suspended, or withdrawn were excluded. Information on trial status, trial type, disease or condition, and GAS implementation method was reviewed. For consistency, diseases or conditions were consolidated under umbrella terms.

RESULTS: Of 263 trials identified, we excluded 16 in which GAS was not used as an outcome. Nearly half (112, 45%) of the trials were completed; 35% were ongoing (pre-recruitment, recruiting, enrolling, or active). Most were interventional (225, 91%); 22 (9%) were observational studies. Of the 21 interventional trials with phases listed, most were phase III (9, 43%), 7 (33%) were phase II, 2 (10%) were phase I, and others were one phase IV and combined trials. GAS was the primary outcome in 99 trials (40%) , secondary in 130 (53%), and ‘other’ in 18 (7%). In 2 (1%) trials, GAS was also part of the intervention. GAS was most often used in Cerebral Palsy (32, 13% of trials), Mental Health (27, 11%), Autism (25, 10%), Stroke (19, 8%) and Spasticity (15, 6%). Novel uses of GAS were identified in several rare diseases (e.g. Rett Syndrome, Prader-Willi Syndrome).

CONCLUSIONS: Increased interest in GAS has increased across multiple disciplines. Most studies that employed GAS were interventional, where it was used as a primary or secondary outcome. Interest in GAS is likely to increase further with the FDA’s endorsement of personalized endpoints.