Use of appropriate patient-reported outcome measures (PROM) questionnaires in trials can demonstrate the clinically meaningful differences a therapy has on patients’ health and visual outcomes. General questionnaires, which group several conditions together to assess outcomes in general may lead to inaccurate findings.
With a recent influx of clinical trials for gene therapies for patients with inherited retinal diseases (IRD), the need for a validated PROM specific to the targeted population is apparent. In a recent review on the use of PROM in IRD, Selvan K, et al found that most peer-reviewed studies published did not include PROM results. In fact, most did not include any evaluation of the patient experience.
Recently, the Michigan Retinal Degeneration Questionnaire and Michigan Vision-related Anxiety Questionnaire were created to assess the vision ability and associated mental health for adults with IRD, however, these have not been validated for use in children with IRDs.
“Outcome measures used for a specific condition should be rigorously developed with mixed methods input from its target condition and age cohort as advised by FDA and [International Society for Pharmacoeconomics and Outcomes Research],” Selvan K, et al wrote. “As such, the use of general visual impairment PROM is not recommended for garnering significant insight for the purposes of clinical decision-making and evaluating the efficacy of therapies.”
Selvan K, et al concluded that there is a need to create PROMs questionnaires specifically for children with IRD.
For insight on how PROM for this population can be created, click here.
Reference
Selvan K, Abalem MF, Lacy GD, et al. The State of Patient-Reported Outcome Measures for Pediatric Patients with Inherited Retinal Disease. Ophthalmol Ther. 2022;https://doi.org/10.1007/s40123-022-00514-x
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