OBJECTIVES

To assess the context in which single-item patient global assessments (PGAs) or clinician global assessments (CGAs) have been used to support the evaluation of treatment benefit in new products approved by US FDA.

METHODS

Review of the Drugs@FDA database identified new drugs approved by FDA between January 2017 and December 2019. For all identified drugs, product labels were reviewed for references to global assessments. Drug approval packages were further reviewed to assess endpoint status of global assessments.

RESULTS

Of the 153 new drugs approved during these years, a total of 14 drugs (9.1%) had label language that referred to either PGA or CGA within 9 unique indications. CGAs or PGAs were most prominent in drugs approved to treat diseases of the nervous systems (n=4) or skin (n=3) and served as primary or co-primary endpoints in 10 of 14 (71.4%) approvals. Static items were more commonly referred to than dynamic. PGA and CGA were components of primary endpoint to assess the efficacy of drugs for treating diseases of the musculoskeletal system (n=2).

CONCLUSIONS

Global assessments were shown to support approximately 10% of new drugs approved between 2017-2019. The FDA PRO Guidance (2009) states that a “single-item global question usually would be inadequate as an endpoint to support labeling claims and would be uninformative about the effects on each specific symptom and sign.” This review illustrates flexibility by the FDA in considering global assessments in the evaluation of treatment benefit.