OBJECTIVES: Recently, the US Food and Drug Administration (FDA) has hosted Patient-Focused Drug Development (PFDD) meetings with the objective of identifying: symptoms/impacts on daily living most important to patients and their experiences with treatments. Voice-of-the-Patient (VoP) summary reports are published following each meeting. This pilot study examined whether symptoms prioritized by patients during the sickle cell disease (SCD) meeting (February 2014) were assessed as endpoints for indicated treatments (iTX) and treatments under study (TXus). METHODS: Symptoms identified by patients as “most important” were extracted from the SCD VoP report. Product labels and clinicaltrials.gov were used to identify endpoints for iTX, and Phase 2 or 3 trials for TXus over the past 5 years. Endpoints were pooled for treatments with multiple studies. To examine uptake of the SCD VoP information, a sub-analysis was performed for “post-PFDD” studies with initial clinicaltrials.gov dates later than 2015. RESULTS: Hydroxyurea is the only SCD iTX (N=1); ten treatments under study (TXus, N=10). VoP reported “most-important symptoms” include: acute pain crises (iTX=1/1; TXus=5/10); chronic pain (iTX=1/1; TXus=4/10); fatigue (TXus=1/10); difficulty concentrating/cognitive issues (none); intolerance to cold/weather changes (none); cardiovascular events (none specifically, adverse events generally); organ damage (TXus1/10); Chronic leg/skin ulcers (TXus=1/10); Bone/osteopathic disorders (none); Hearing loss (none); Loss of sight/eye issues (none); Pregnancy complications (none); Complications of long-term treatment (none). Four treatments were studied post-PFDD; of these, three included VoP outcomes, including two beyond pain: organ damage (TXus 1/10) and chronic leg ulcers (TXus 1/10). CONCLUSIONS: This pilot study demonstrates that PFDD meetings may be having an impact on endpoint selection for trials. As patient engagement becomes more widespread, incorporation of additional patient-prioritized endpoints can be anticipated. Additionally, not all patient-prioritized symptoms are ethical for evaluation during intervention studies and may be good candidates for observational studies.