TY - JOUR
T1 - A multistakeholder Delphi consensus core outcome set for clinical trials in moderate-to-severe asthma (coreASTHMA)
AU - for the coreASTHMA panel
AU - Tejwani, Vickram
AU - Chang, Hsing Yuan
AU - Tran, Annie P.
AU - Naber, Jennifer Al
AU - Gutzwiller, Florian S.
AU - Winders, Tonya A.
AU - Khurana, Sandhya
AU - Sumino, Kaharu
AU - Mosnaim, Giselle
AU - Moloney, Rachael M.
N1 - Funding Information:
Funding: This project was supported by contributions received from Chiesi, Genentech, Inc, GlaxoSmithKline plc, Novartis Pharma AG, and UCB Biopharma.
Funding Information:
Principal Investigator: Rachael Moloney, MHS (Center for Medical Technology Policy), Core Technical Group (Center for Medical Technology Policy):, Jennifer Al Naber, MS, MSPH; Hsing-Yuan Chang, MD, MPH; Donna Messner, PhD; Rachael Moloney, MHS; Susan Reed, MA; Annie Tran, MPH, Clinical Consultant: Vickram Tejwani, MD (Johns Hopkins Hospital), Advisory Committee: Dave Burnett, PhD (University of Kansas Medical Center); LeRoy Graham, MD (Bridge Atlanta Medical Center, American Thoracic Society); Mitchell Grayson, MD (Nationwide Children's Hospital—The Ohio State University College of Medicine, Asthma and Allergy Foundation of America); Mary Hart, MS, RRT, AE-C (Allergy & Asthma Network [AAN]); Sumita Khatri, MD, MS (Cleveland Clinic Asthma Center, Cleveland Clinic Lerner College of Medicine, American Lung Association); Sandhya Khurana, MD (University of Rochester School of Medicine and Dentistry); Michelle Leavy, MPH (OM1); Giselle Mosnaim, MD, MS (NorthShore University HealthSystem); Rebecca Fortescue, MA, MPH, MB, BChir (Cochrane Airways Group, St. George's University of London, United Kingdom); Samir Shaikh, MBA* (Food and Drug Administration); Kaharu Sumino, MD, MPH (Washington University School of Medicine, American Lung Association); Vickram Tejwani, MD (Johns Hopkins Hospital); Tonya Winders, MBA (AAN), *Observer; all views expressed were his own and did not reflect the opinions of the FDA. Voting Panel: Lorene Alba, AE-C (Asthma and Allergy Foundation of America); Greg Barabell, MD, CPC, FAAP (Clear Bell Solutions); Navdeep Bilkhu (AAN); John Bottrell, RRT (Asthma.net); Sheila Brillhart (AAN); Theresa Cannizzaro, RRT (Parkview Health); Thomas Casale, MD (University of South Florida); Bradley E. Chipps, MD (Capital Allergy & Respiratory Disease Center); Marc Claussen (Chiesi); Geoffrey Crawford, MD, MS (Anthem, Inc); Charnette Darrington Zaskoda (AAN); Emma Dennett, BSc, PhD (Cochrane Airways Group, St. George's University of London, United Kingdom); Rossella Di Bidino, PhD, MSc (Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Graduate School of Health Economics and Management [ALTEMS]); Catherine Drai, PharmD* (European Medicines Agency [EMA]); Tiffany Dy, MD (Washington University School of Medicine in St. Louis); Dmitry Galkin, MD, PhD (Chiesi); Angus Gunn (UCB Celltech Ltd); Florian S. Gutzwiller, MD, MPH (Novartis Pharma AG); Steve Holmes, MD (Somerset Clinical Commissioning Group, Primary Care Respiratory Society); Shawni L. Jackson-Triggs, PhD, MBA, CC (Washington University Asthma Symptom-based Adjustment of Inhaled Steroid Therapy in African American Children [ASIST]); Xavier Jaumont, MD (Novartis Pharma AG); Andrea M. Jensen, CHES, AE-C (Utah County Health Department, Allergy & Asthma Today Magazine); Rabia Kahveci, MD, MScHTA&M (Health Technology Assessment international, Eurasian Health Technology Assessment Initiative); Cara Kraft (AAN); Regan Lloyd (AAN); Kerri MacKay (AAN, Canadian Severe Asthma Network); Peter McQuitty (AAN, European Federation of Allergy and Airways Diseases Patients’ Association); Robert (Bob) Meyer, MD (Greenleaf Health Inc); Divya Mohan, MD (GlaxoSmithKline); Linda Nelsen (GlaxoSmithKline); Rhonda Nelson (AAN); Julie Olsson, MD, MS (Genentech, Inc); Marie Osterberg, PhD (The Swedish Agency for Health Technology Assessment and Assessment of Social Services); Melody Papazis (AAN); David Price, FRCGP (Optimum Patient Care Ltd—Australia and United Kingdom; Observational and Pragmatic Research Institute—Singapore, University of Aberdeen); Yamina Rajput, MSc (Genentech, Inc); David Rind, MD, MSc (Institute for Clinical and Economic Review); Weily Soong, MD (Alabama Allergy and Asthma Center and Clinical Research Center of Alabama); Dia Sue-Wah-Sing (AAN, Canadian Severe Asthma Network); Emily Tsiao, PharmD (Premera Blue Cross); Job F.M. van Boven, PhD, PharmD (University of Groningen, University Medical Center Groningen, Groningen Research Institute for Asthma & COPD [GRIAC], International Primary Care Respiratory Group [IPCRG]); Samantha Walker, PhD (The Asthma UK and British Lung Foundation Partnership); Sophie Werkö (The Swedish Agency for Health Technology Assessment and Assessment of Social Services, International Network of Agencies for Health Technology Assessment); Sara Wright (UCB Pharma); Connie Yang, MD, MSc (University of British Columbia, Canadian Thoracic Society). *Observer; all views expressed were her own and did not reflect the opinions of EMA.
Publisher Copyright:
© 2021 American College of Allergy, Asthma & Immunology
PY - 2021/7
Y1 - 2021/7
N2 - Background: Treatments for long-term control of asthma have improved and include a promising but expensive class of biologic therapies. However, the clinical trials evaluating these and other novel treatments have used a variety of different outcomes to evaluate efficacy. The evolution of asthma care calls for a re-examination of outcomes that are most important to patients and other stakeholders. Objective: To develop a core set of outcomes to be measured in phase 3 and phase 4 clinical drug trials in patients with moderate-to-severe asthma. Methods: We used a robust and in-depth multistakeholder consensus process bringing together patients, clinicians, regulators, payers, health technology assessors, researchers, and product developers to reach consensus on outcomes. We used a modified Delphi method to reach consensus, an approach adapted from the Core Outcome Measures in Effectiveness Trials Initiative aligned with contemporary methodological standards for core outcome set development. Results: The following outcomes were included in the final core set: severe asthma exacerbation, change in asthma control, asthma-specific or severe asthma-specific quality of life, asthma-specific hospital stay (ie, >24-hour stays at any level of care) or admission, and asthma-specific emergency department visit. Conclusion: These 5 outcomes represent a minimum set of core outcomes for use in phase 3 and phase 4 clinical drug trials in moderate-to-severe asthma. Consistent collection of these outcomes as minimum, independent of whether additional heterogeneous primary or secondary outcomes are included, will allow for meaningful comparisons of the effect of asthma therapies across clinical trials.
AB - Background: Treatments for long-term control of asthma have improved and include a promising but expensive class of biologic therapies. However, the clinical trials evaluating these and other novel treatments have used a variety of different outcomes to evaluate efficacy. The evolution of asthma care calls for a re-examination of outcomes that are most important to patients and other stakeholders. Objective: To develop a core set of outcomes to be measured in phase 3 and phase 4 clinical drug trials in patients with moderate-to-severe asthma. Methods: We used a robust and in-depth multistakeholder consensus process bringing together patients, clinicians, regulators, payers, health technology assessors, researchers, and product developers to reach consensus on outcomes. We used a modified Delphi method to reach consensus, an approach adapted from the Core Outcome Measures in Effectiveness Trials Initiative aligned with contemporary methodological standards for core outcome set development. Results: The following outcomes were included in the final core set: severe asthma exacerbation, change in asthma control, asthma-specific or severe asthma-specific quality of life, asthma-specific hospital stay (ie, >24-hour stays at any level of care) or admission, and asthma-specific emergency department visit. Conclusion: These 5 outcomes represent a minimum set of core outcomes for use in phase 3 and phase 4 clinical drug trials in moderate-to-severe asthma. Consistent collection of these outcomes as minimum, independent of whether additional heterogeneous primary or secondary outcomes are included, will allow for meaningful comparisons of the effect of asthma therapies across clinical trials.
UR - http://www.scopus.com/inward/record.url?scp=85105073696&partnerID=8YFLogxK
U2 - 10.1016/j.anai.2021.03.022
DO - 10.1016/j.anai.2021.03.022
M3 - Article
C2 - 33781936
AN - SCOPUS:85105073696
SN - 1081-1206
VL - 127
SP - 116-122.e7
JO - Annals of Allergy, Asthma and Immunology
JF - Annals of Allergy, Asthma and Immunology
IS - 1
ER -