Findings published in Ophthalmology and Therapy can inform the use of patient- and observerreported outcomes (PRO/ObsRO) in retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA). Helena Bradley and colleagues aimed to understand the patient experience of RP and LCA to inform the creation of PRO/ObsRO. First, the study team conducted literature reviews, assessed existing PRO instruments, held interviews with patients, clinicians, and payers, and evaluated a PRO instrument. The findings revealed a range of visual function symptoms that significantly affected vision-related activities and overall QOL. Patient interviews identified additional symptoms not previously reported. Then, the information was used to develop a conceptual model representing the patient experience of RP/LCA. The review of existing PRO instruments highlighted the lack of a comprehensive assessment of all relevant concepts for RP/LCA, emphasizing the need for new PRO instruments.
