OBJECTIVES: Patient reported outcomes (PROs) help in assessing the risks and benefits of oncology and haemato-oncology therapies and foster patient-centricity. However, data on PROs in oncology and haemato-oncology studies are sparse. The objective of this study was to evaluate the reporting of PROs endpoints in recently approved oncology and haemato-oncology treatment-related studies.

METHODS: We conducted a systematic review of the FDA Office of Hematology and Oncology Products archives for all haemato-oncology and oncology drug approvals issued between January 2020 and June 2023 (data cut-off, 20 June 2023). We retrieved the pivotal clinical trial(s) used for FDA approval from PubMed and ClinicalTrials.gov and evaluated the PRO endpoints in study design and subsequent publication using the Consolidated Standards of Reporting Trials (CONSORT)-PRO checklist. All results were analyzed descriptively.

RESULTS: Total 157 out of 180 (87%) FDA approvals were treatment approvals (117 [74%] regular approvals, 39 [25%] accelerated approvals, 1 expand access approval). Of these, 70 studies (45%) incorporated PRO endpoints (as secondary endpoints) in their study designs (14 accelerated approvals; 56 regular approvals; single agent studies: 70%; combination treatment studies: 30%). Most studies (42/70, 60%) involved solid tumors while 28 (40%) studies involved hematological cancers. The most common PRO endpoints were change from baseline to different timepoints in European Organization for Research and Treatment of Cancer (EORTC) 30-item core quality of life questionnaire (QLQ-C30) (69%), European Quality of Life-5 Domain 5 Level Scale (EQ-5D-5L) (46%) and Functional Assessment of Cancer Therapy (FACT) (23%) total and subscale scores. Only 34 of 70 studies (48%) incorporating PRO endpoint in their study design published their PRO results; no study complied to all 5 CONSORT-PRO checklist items.

CONCLUSIONS: There is suboptimal reporting of PROs in oncology and haemato-oncology. Increased awareness and relevant trainings may help to maximize patient-centricity in the development and approval of various cancer treatments.