Think about it. We want to know how the child experiences his or her illness and therapies, and we can just ask, right? Well, some children are too young to even understand or respond to questions, infants and young toddlers, others go through a “no” stage and answer “no” to every question, and others may be unable to respond to questions for a range of other reasons.
The recent IOM workshop, Comprehensive Cancer Care for Children and their Families, touched on some key issues around use of PROs in children with cancer, and much of what was said is applicable to children with other diseases and conditions. I’m hoping that video of the presentations will be posted (they often are).
The research imperative to develop pediatric Patient Reported Outcomes (PROs)
Try presentation by Lillian Sung, The Hospital for Sick Children, Toronto has the bland title “Academic Perspective on Clinical Research”, but is a hard hitting overview of the issues around implementing Patient Reported Outcomes (PROs) in pediatric cancer patients, and includes a full list of action items and recommendations. She notes the FDA 2009 Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims and its statement,
We discourage proxy-reported outcome measures for this population (i.e., reports by someone who is not the patient responding as if that person were the patient). For patients who cannot respond for themselves (e.g., infant patients), we encourage observer reports that include only those events or behaviors that can be observed.
Pediatric Patient Reported Outcomes (PROs) to improve estimates of toxicity and adverse events
Bryce Reeve made a critical point in favor of use of PRO’s in pediatric cancers. He presented evidence that there is a low correlation between clinicians’ report and patient report for a range of symptoms. The consequence of relying on clinicians’ report for identifying adverse events is an underestimate of adverse events, something that can be corrected by broader use of PRO’s in cancer research. Presumably this is relevant to other diseases, as well.