Patient-Reported Outcomes in Pediatric Cancer Registration Trials: A US Food and Drug Administration Perspective
- PROs have been feasible to collect from patients as young as 7 years old and were included in trials from 2013 onward.
- PROs were used as exploratory endpoints in four product applications but not included in product labeling.
- To include PRO data in FDA labeling, early interaction with the FDA, clear research objectives, prospective statistical analysis plans, low levels of missing data, and reliable PRO instruments are crucial.
- Challenges in PRO research include time, logistics, study design issues, discrepancies between clinician/observer and patient self-reports, translation and validation across languages and cultures, and impact on trial recruitment and retention.
- Despite challenges, there's high willingness among children and caregivers to self-report treatment experiences, emphasizing the importance of including the child's perspective in drug development.
The 21st Century Cures Act directed the US Food and Drug Administration (FDA) to systematically incorporate patients’ experiences, needs, perspectives, and priorities into drug development and evaluation. Patients are experts in their disease because of their lived experience with its symptoms and treatment, and this includes children. In completing patient-reported outcome (PRO) measures that are fit for purpose, patients of different ages can provide unique and valuable symptom and functional information to help inform the FDA’s benefit-risk assessment of cancer therapeutics.
The collection of PRO data in adult cancer clinical trials has allowed for enhanced and more accurate reporting of symptomatic adverse events. Emerging evidence also suggests that using PRO assessments to monitor symptoms during routine cancer care can lead to an improvement in clinical outcomes, including survival. Despite these benefits observed in adult patients with cancer, there has been a dearth of work specific to PROs in pediatric oncology drug development.
Studies have demonstrated that clinicians and caregivers frequently under- or overestimate the prevalence, intensity, and burden of symptomatic AEs compared with children’s self-report (6-10). Therefore, pediatric PRO data can provide a more comprehensive assessment of the safety and tolerability profile of cancer therapeutics. Similar to efforts in adult patients, these data can be used in clinical practice to improve communication and shared decision making between clinicians and patients/caregivers about side effect recognition, management, and supportive care, with the goal of maximizing the child’s quality of life (QOL). Thus, patient experience data can help regulators and ultimately prescribers, caregivers, patients, and payers make more informed decisions regarding use of anticancer drugs in pediatric populations...
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