The team conducted an environmental scan to explore the patient-reported outcome measures (PROMs) that could be used in new and emerging sickle cell disease (SCD) therapies. In SCD clinical trials, PROMs may be used as clinical outcome assessments (COAs) to assess patient-reported outcome (PRO) endpoints related to how patients feel and function as a result of treatment and to inform regulatory evaluations. The development of new cell-based gene therapies introduces substantially different treatment processes, risks, and expected outcomes that may not be fully captured by PROMs used in trials for traditional SCD treatments. We conducted an environmental scan to identify and characterize PROMs that could be used in clinical trials for both existing and emerging cell-based gene therapies for SCD. Activities included a peer-reviewed literature search using PubMed and EMBASE and a targeted web scan of grey literature, with a focus on sources documenting PROM use in trials (e.g., ClinicalTrials.gov) and SCD-focused advocacy organization websites. The environmental scan identified that PRO endpoints historically recommended in SCD trials by the American Society of Hematology and the FDA primarily focused on specific aspects of pain, affect, and function. In contrast, PRO endpoints used in trials of cell-based gene therapies for SCD addressed some of these same areas but more commonly emphasized global measures of health-related quality of life with substantial variability in the domains assessed across measures. The environmental scan of PROMs used as COAs in SCD cell-based gene therapy clinical trials identified a wide range of instruments in a rapidly evolving field.
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