Abstract
BackgroundThe Food and Drug Administration patient-reported outcome (PRO) guidance provides standards for PRO development, but these standards bring scientific and logistical challenges which can result in a lengthy and expensive instrument development process. Thus, more pragmatic methods are needed alongside traditional approaches.MethodsPartnering with the National Ankylosing Spondylitis (AS) Society, we compared three methods for eliciting patient experiences: 1) concept elicitation (CE) interviews with 12 individuals with AS, 2) “group concept mapping” (GCM) with 16 individuals with AS, 3) a social media review (SMR) of AS online chatrooms. Three conceptual models were developed and compared to explore data breadth/depth, as well as the practicalities and patient-centeredness.ResultsOverlap in concepts was observed between conceptual models; 35% of symptoms were identified by all methods. The SMR approach identified the most concepts (n = 23), followed by CE interviews (n = 18), and GCM (n = 15). Eight symptoms were uniquely identified using GCM and SMR. Eliciting in-depth data was challenging for SMR as detail was not always provided. Insight into the relationships between symptoms was obtained as a “concept map” in GCM, via effective probing within interviews, and through the subject’s descriptions in SMR. Practical investment varied; CE interviews were the most resource intensive, whereas SMR was the least. Individuals in GCM and CE interviews reported high engagement.ConclusionsPrimary CE interviews achieved the greatest depth in conceptual understanding of patient experience; however, novel methods (GCM, SMR) provide complementary approaches for identifying measurement concepts. Each method has strengths and weaknesses and should be selected based on specific research objectives.
Highlights
The Food and Drug Administration patient-reported outcome (PRO) guidance provides standards for PRO development, but these standards bring scientific and logistical challenges which can result in a lengthy and expensive instrument development process
Our research group sought to further the science by conducting an empirical assessment of the relative benefits and limitations of two emerging methods for eliciting and collecting the patient perspective relative to traditional approaches described in the PRO guidance
Analysis of all data collected from the three approaches highlighted 26 symptom concepts as relevant and important to those living with ankylosing spondylitis (AS)
Summary
The Food and Drug Administration patient-reported outcome (PRO) guidance provides standards for PRO development, but these standards bring scientific and logistical challenges which can result in a lengthy and expensive instrument development process. The publication of the Food and Drug Administration’s (FDA) guidance and the European Medicines Agency’s (EMA) reflection paper on patient-reported outcomes (PRO) measurement within clinical drug development are both testament to the enhanced role of the patient in regulatory strategy and decision-making [1,2,3]. Study aims There is limited research exploring the value of traditional (i.e. as set out in FDA guidance) PRO content development methods in comparison with other novel methods [7, 8]. Our research group sought to further the science by conducting an empirical assessment of the relative benefits and limitations of two emerging methods for eliciting and collecting the patient perspective relative to traditional approaches described in the PRO guidance
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