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The State of Patient-Reported Outcome Measures for Pediatric Patients with Inherited Retinal Disease
Kavin Selvan1,2,3, Maria F Abalem4,5, Gabrielle D Lacy4
1Genetics and Genome Biology (GGB) Program, The Hospital for Sick Children Research Institute, Toronto, ON, Canada.
Insights
Patient-reported outcome measures (PROMs) are crucial for assessing patient health. This study highlights the need for specialized PROMs for pediatric inherited retinal disease (IRD) patients, as current options are insufficient.
Area of Science:
- Ophthalmology
- Patient-Reported Outcomes
- Pediatric Health
Background:
- Patient-reported outcome measures (PROMs) assess health outcomes important to patients.
- PROMs aid clinical decisions, disease impact understanding, and therapeutic evaluation.
- Existing PROMs lack specificity for pediatric patients with inherited retinal diseases (IRD).
Purpose of the Study:
- To identify and evaluate existing pediatric PROMs for relevance to IRD.
- To assess the suitability of current PROMs based on FDA guidelines.
- To propose standards for developing IRD-specific pediatric PROMs.
Main Methods:
- Literature search of MEDLINE and Embase databases.
- Evaluation of identified pediatric PROMs against FDA guidelines.
- Review of International Society for Pharmacoeconomics and Outcomes Research (ISPOR) standards.
Main Results:
- No existing pediatric PROMs are specifically tailored for inherited retinal disease.
- Current pediatric PROMs show limited applicability for IRD patients.
- Significant need exists for IRD-specific PROMs in pediatric populations.
Conclusions:
- Development of IRD-specific PROMs for pediatric patients is necessary.
- ISPOR and FDA guidelines can inform the creation of tailored PROMs.
- Improved PROMs will enhance care and research for pediatric IRD.
Abstract:
Patient-reported outcome measures (PROMs) are questionnaires that assess health outcomes meaningful to the patient. PROMs have multiple applications, such as supporting clinicians' decision-making for patient care, understanding the impact of disease on patient functioning, and evaluating the efficacy of therapeutics. Though PROMs were developed for various eye conditions, no PROM was tailored to pediatric patients with inherited retinal disease (IRD). Hence, a literature search was conducted using MEDLINE and Embase to identify PROMs potentially relevant to this patient population. This review evaluated selected pediatric PROMs against the US Food and Drug Administration (FDA) guidelines and found restricted use in the context of IRD. As there is a need for PROMs tailored to pediatric patients with IRD, we provide a perspective on applying the International Society for Pharmacoeconomics and Outcomes Research and FDA standards on the development of PROMs specific to IRD.
