Exploring and Leveraging Known Resources to Support Pediatric Clinical Outcomes Assessment (COA) Development

Background: Best practices for pediatric clinical outcomes assessment (COA) development rely on guidelines that have been developed for adult populations. While some useful resources are available to support pediatric COA development, this information has primarily come from within the measurement development field. To our knowledge, no research has explored the experiences of professionals from other disciplines who interact with children on a routine basis. Aim and Objective: The goal of this research was to explore the experiences of professionals from outside of the measurement science field, who work closely with children every day, in settings relevant to the context of concept elicitation and cognitive debriefing interviews for pediatric COA development. The objectives were to 1) learn new ways to engage children in conversations regarding their health state; 2) understand how methods used in other disciplines can be used to improve the amount and quality of data emerging from pediatric qualitative interviews; and 3) generate a list of references to support pediatric COA development. Methods: Individual, one-to-one expert advice meetings were conducted over the phone or in person and lasted approximately 60 minutes in duration. One child life specialist, one speech/language pathologist, and three reading specialists were consulted, given their role in evaluating children’s comprehensive abilities as well as their daily interactions with children. Two experienced COA researchers conducted the expert advice meetings using a semi-structured interview guide to provide a framework for discussion. Results: These experts reported that factors such as interview setting, time taken to build rapport with the child, the child’s comfort level, presence or absence of caregiver during the interview, the child’s communication style, disease-related factors, and the child’s developmental age may influence the amount and type of information that is possible to elicit during qualitative interviews. Several of these factors are also important for cognitive debriefing interviews. In addition, experts provided input that may improve the debriefing procedure, such as having the child read the text aloud in small increments, re-read text, and highlight text that they do not understand. Best practice tips from the experts were consolidated into a set of references for use by those conducting pediatric COA development research. Conclusions: Incorporating interdisciplinary perspectives into pediatric COA development may improve both the methods used to elicit information from children and the quality of the resulting questionnaires.

Validation and equivalency of electronic clinical outcomes assessment systems

<p>The US food and drug administration (FDA) has long called for clinical outcomes assessments (COA), such as patient-reported outcomes (PRO), to be ‘fit-for-purpose’ meaning the COA has been validated to support the context of use. The FDA’s recent patient-focused drug development guidance series has renewed the importance of ensuring that COA is ‘fit-for-purpose’ and valid. In addition, the FDA has recommended that COA be collected electronically and that the electronic (eCOA) system and devices also be validated. Advancing technology requires eCOA systems and devices to evolve; eCOA devices may change over time. As bring your own device (BYOD) models gain popularity and acceptance, devices may also be mixed within trials. Changes in eCOA devices or mixing devices may require equivalence testing to prove validity across platforms. The aim of this article is to provide an overview of the different types of validation at both the assessment level and the eCOA system (device) level to help clinical trial sponsors determine the appropriate level of validation or equivalence testing required for COA used in their clinical trials. </p>

Novel Approaches to Air Pollution Exposure and Clinical Outcomes Assessment in Environmental Health Studies

An accurate assessment of pollutants’ exposure and precise evaluation of the clinical outcomes pose two major challenges to the contemporary environmental health research. The common methods for exposure assessment are based on residential addresses and are prone to many biases. Pollution levels are defined based on monitoring stations that are sparsely distributed and frequently distanced far from residential addresses. In addition, the degree of an association between outdoor and indoor air pollution levels is not fully elucidated, making the exposure assessment all the more inaccurate. Clinical outcomes’ assessment, on the other hand, mostly relies on the access to medical records from hospital admissions and outpatients’ visits in clinics. This method differentiates by health care seeking behavior and is therefore, problematic in evaluation of an onset, duration, and severity of an outcome. In the current paper, we review a number of novel solutions aimed to mitigate the aforementioned biases. First, a hybrid satellite-based modeling approach provides daily continuous spatiotemporal estimations with improved spatial resolution of 1 × 1 km2 and 200 × 200 m2 grid, and thus allows a more accurate exposure assessment. Utilizing low-cost air pollution sensors allowing a direct measurement of indoor air pollution levels can further validate these models. Furthermore, the real temporal-spatial activity can be assessed by GPS tracking devices within the individuals’ smartphones. A widespread use of smart devices can help with obtaining objective measurements of some of the clinical outcomes such as vital signs and glucose levels. Finally, human biomonitoring can be efficiently done at a population level, providing accurate estimates of in-vivo absorbed pollutants and allowing for the evaluation of body responses, by biomarkers examination. We suggest that the adoption of these novel methods will change the research paradigm heavily relying on ecological methodology and support development of the new clinical practices preventing adverse environmental effects on human health.