Archive | Patient Reported Outcomes (PROs)

The challenge of pediatric Patient Reported Outcomes (PROs)

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.

Pediatric patient reported outcomes (PROs)

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.

Other resources

Learning module – Patient Reported Outcomes (PROs)

Just released by ISPOR’s Distance Learning Program, the module, “Patient Reported Outcomes – Analysis and Interpretation”, Tamar Lasky, PhD, faculty.

Course Description

The module will review definitions of patient reported outcomes and the contexts in which they are used, including applications to health quality improvement, clinical trials and drug safety. The module will provide an overview of methodologic issues to consider when using patient reported outcomes or research with patient reported outcomes, and the concepts that are assessed by government agencies such as FDA and NIH. Measurement concepts such as content validity will be introduced, and limitations of discussed.

After the overview, the module will walk through all issues related to statistical analysis of patient reported outcomes after the instrument has been validated, calibrated, approved and finalized. Concepts related to development will be introduced only as they relate to ultimate analysis of the data (development will be covered more thoroughly in a separate module). The use of multiple endpoints and composite endpoints will be covered, as well as approaches to continuous, categorical and time to event analysis. Approaches to analysis of missing data will be introduced.

Three case studies will be used to illustrate analysis of PRO data in different contexts. The first case study will describe the work being done by PROMIS, and the status of the outcome measures available for measuring health care quality. The second case study will describe work being done in the United Kingdome National Health Service (NHS) in PROMs, and analysis of data within a health system. The final case study will illustrate the use of PROs in clinical trials, in support of FDA labeling claims. All modules will discuss statistical analysis, and interpretation of statistical results.

Learning Objectives

By the end of the Patient Reported Outcomes – Analysis and Interpretation module, you will be able to describe what patient-reported outcomes are, general statistical issues to consider when analyzing data collected from PRO instruments, and three case studies demonstrating use of PRO instruments.

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