Adam Gater1, Linda Nelsen2, Sarah Fleming3, J Jason Lundy4, Nicola Bonner5, Rebecca Hall5, Chris Marshall5, Hannah Staunton5, Jerry A Krishnan6, Stuart Stoloff7, Michael Schatz8, John Haughney9. 1. Adelphi Values Ltd., Adelphi Mill, Bollington, Cheshire, UK. Electronic address: adam.gater@adelphivalues.com. 2. GlaxoSmithKline, King of Prussia, PA, USA. 3. Janssen Global Services LLC, Titusville, NJ, USA. 4. Outcometrix, Tucson, AZ, USA. 5. Adelphi Values Ltd., Adelphi Mill, Bollington, Cheshire, UK. 6. University of Illinois Hospital and Health Sciences System, Medical Center Administration, Chicago, IL, USA. 7. University of Nevada, Reno, NV, USA. 8. Kaiser Permanente Medical Center/Kaiser Foundation Hospital, San Diego, CA, USA. 9. University of Aberdeen, King's College, Aberdeen, UK.
Abstract
BACKGROUND: Despite the widespread availability of patient-reported asthma questionnaires, instruments developed in accordance with present regulatory expectations are lacking. To address this gap, the Patient-Reported Outcome (PRO) Consortium's Asthma Working Group has developed a patient-reported asthma daily symptom diary (ADSD) for use in clinical research to assess outcomes and support medical product labeling claims in adults and adolescents with asthma. OBJECTIVES: To summarize the qualitative research conducted to inform the initial development of the ADSD and to provide evidence for content validity of the instrument in accordance with the Food and Drug Administration's PRO Guidance. METHODS: Research informing the initial development and confirming the content validity of the ADSD is summarized. This comprised a review of published qualitative research, semi-structured concept elicitation interviews (n = 55), and cognitive interviews (n = 65) with a diverse and representative sample of adults and adolescents with a clinician-confirmed diagnosis of asthma in the United States to understand the asthma symptom experience and to assess the relevance and understanding of the newly developed ADSD. RESULTS: From the qualitative literature review and concept elicitation interviews, eight core asthma symptoms emerged. These were broadly categorized as breathing symptoms (difficulty breathing, shortness of breath, and wheezing), chest symptoms (chest tightness, chest pain, and pressure/weight on chest), and cough symptoms (cough and the presence of mucus/phlegm). Conceptual saturation was achieved and differences in the experience of participants according to socio-demographic or clinical characteristics were not observed. Subsequent testing of the ADSD confirmed participant relevance and understanding. CONCLUSIONS: The ADSD is a new patient-reported asthma symptom diary developed in accordance with the Food and Drug Administration's PRO Guidance. Evidence to date supports the content validity of the instrument. Item performance, reliability, and construct validity will be assessed in future quantitative research.
BACKGROUND: Despite the widespread availability of patient-reported asthma questionnaires, instruments developed in accordance with present regulatory expectations are lacking. To address this gap, the Patient-Reported Outcome (PRO) Consortium's Asthma Working Group has developed a patient-reported asthma daily symptom diary (ADSD) for use in clinical research to assess outcomes and support medical product labeling claims in adults and adolescents with asthma. OBJECTIVES: To summarize the qualitative research conducted to inform the initial development of the ADSD and to provide evidence for content validity of the instrument in accordance with the Food and Drug Administration's PRO Guidance. METHODS: Research informing the initial development and confirming the content validity of the ADSD is summarized. This comprised a review of published qualitative research, semi-structured concept elicitation interviews (n = 55), and cognitive interviews (n = 65) with a diverse and representative sample of adults and adolescents with a clinician-confirmed diagnosis of asthma in the United States to understand the asthma symptom experience and to assess the relevance and understanding of the newly developed ADSD. RESULTS: From the qualitative literature review and concept elicitation interviews, eight core asthma symptoms emerged. These were broadly categorized as breathing symptoms (difficulty breathing, shortness of breath, and wheezing), chest symptoms (chest tightness, chest pain, and pressure/weight on chest), and cough symptoms (cough and the presence of mucus/phlegm). Conceptual saturation was achieved and differences in the experience of participants according to socio-demographic or clinical characteristics were not observed. Subsequent testing of the ADSD confirmed participant relevance and understanding. CONCLUSIONS: The ADSD is a new patient-reported asthma symptom diary developed in accordance with the Food and Drug Administration's PRO Guidance. Evidence to date supports the content validity of the instrument. Item performance, reliability, and construct validity will be assessed in future quantitative research.
Authors: Clara Padró; Diego Gutiérrez; Francisco Moreno; Antonio Parra; Manuel J Rial; Ramón Lleonart; Carla Torán-Barona; José L Justicia; Albert Roger Journal: Immun Inflamm Dis Date: 2022-05
Authors: Niklas Karlsson; Mark J Atkinson; Hana Müllerová; Marianna Alacqua; Christina Keen; Rod Hughes; Christer Janson; Barry Make; David Price; Helen K Reddel Journal: ERJ Open Res Date: 2021-02-01