OBJECTIVES

Patient experience (PE) data is a broad term that can include many aspects of a patient’s experience with a disease or condition. One common type of PE data submitted to FDA are PRO assessments of symptoms and function that can complement existing safety and efficacy data. In some cases this data has been included in FDA product labels. The purpose of this review was to characterize the number and type of PRO data included in FDA labels reviewed by the Office of Hematology and Oncology products from 2010 to 2018.

METHODS

FDA labeling changes in hematology and oncology products between the years 2010-2018 were obtained from an internal FDA database and the Drugs@FDA website. FDA labels were reviewed for inclusion of PRO symptom and function data. The following were collected: year of label update, abbreviated indication, location of PRO data in label (label section), PRO concept measured and whether the PRO endpoint was included in the statistical hierarchy for multiplicity adjustment (type I error control).

RESULTS

Between 2010 and 2018 the Office of Hematology and Oncology approved 575 labeling changes. Of the 575 labeling changes, 9 incorporated PRO results. Of the 9 label updates that contained PRO results, the PRO concepts that were labeled included disease symptoms (5), disease symptoms and physical function (1), fatigue (1), patient preference (1) and symptomatic side effects (1). PRO endpoints were included in the statistical hierarchy in 2 of 9 cases.

CONCLUSIONS

PRO information was included in eight percent of label changes between 2010-2018, with the majority of those capturing patient-reported disease symptoms, symptomatic side effects or aspects of physical function. Efforts are underway to improve the collection, analysis, interpretation and communication of PRO data in cancer trials to better communicate the patient experience.