Evaluating Demographic Representation in Clinical Trials: Use of the Adaptive Coronavirus Disease 2019 Treatment Trial (ACTT) as a Test Case.

Autor: Ortega-Villa AM; Biostatistics Research Branch, Division of Clinical Research, National Institute of Allergy and Infectious Diseases, Rockville, Maryland, USA., Hynes NA; Division of Infectious Diseases, Department of Medicine, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA., Levine CB; Division of Infectious Disease, Department of Internal Medicine, University of Texas Medical Branch, Galveston, Texas, USA., Yang K; Department of Clinical Pharmacy, University of California, San Francisco, San Francisco, California, USA., Wiley Z; Department of Medicine, Emory University School of Medicine, Atlanta, Georgia, USA., Jilg N; Department of Medicine, Massachusetts General Hospital, Boston, Massachusetts, USA.; Brigham and Women's Hospital, Harvard Medical School, Boston, Massachusetts, USA., Wang J; Clinical Monitoring Research Program Directorate, Frederick National Laboratory for Cancer Research, Frederick, Maryland, USA., Whitaker JA; Molecular Virology and Microbiology, Baylor College of Medicine, Houston, Texas, USA., Colombo CJ; Department of Virtual Health and Department of Medicine, Madigan Army Medical Center, Tacoma, Washington, USA.; Department of Medicine, Uniformed Services University of the Health Sciences, Bethesda, Maryland, USA., Nayak SU; Division of Microbiology and Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA., Kim HJ; Department of Community Health Systems, School of Nursing, University of California, San Francisco, San Francisco, California, USA.; National Patient Care Services, Kaiser Permanente, Oakland, California, USA., Iovine NM; Division of Infectious Diseases and Global Medicine, Department of Medicine, University of Florida Health, Gainesville, Florida, USA., Ince D; Division of Infectious Diseases, Department of Internal Medicine, University of Iowa, Iowa City, Iowa, USA., Cohen SH; Division of Infectious Diseases, University of California, Davis, Sacramento, California, USA., Langer AJ; COVID-19 Emergency Response Team, Centers for Disease Control and Prevention, Atlanta, Georgia, USA., Wortham JM; COVID-19-Associated Hospitalization Surveillance Network, Centers for Disease Control and Prevention, Atlanta, Georgia, USA., Atmar RL; Department of Medicine, Baylor College of Medicine, Houston, Texas, USA., El Sahly HM; Molecular Virology and Microbiology, Baylor College of Medicine, Houston, Texas, USA., Jain MK; Department of Internal Medicine, University of Texas Southwestern Medical Center, Dallas, Texas, USA., Mehta AK; Division of Infection Diseases, Emory University School of Medicine, Atlanta, Georgia, USA.; National Emerging Special Pathogens Treatment and Education Center, Atlanta, Georgia, USA., Wolfe CR; Division of Infectious Diseases, Department of Medicine, Duke University Medical Center, Durham, North Carolina, USA., Gomez CA; Division of Infectious Diseases, Department of Internal Medicine, University of Nebraska Medical Center, Omaha, Nebraska, USA., Beresnev T; Division of Microbiology and Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA., Mularski RA; Department of Pulmonary and Critical Care Medicine, Northwest Permanente, Kaiser Permanente Northwest, Portland, Oregon, USA.; The Center for Health Research, Kaiser Permanente Northwest, Portland, Oregon, USA., Paules CI; Division of Infectious Diseases, Penn State Health Milton S. Hershey Medical Center, Hershey, Pennsylvania, USA., Kalil AC; Division of Infectious Diseases, Department of Internal Medicine, University of Nebraska Medical Center, Omaha, Nebraska, USA., Branche AR; Division of Infectious Diseases, Department of Medicine, University of Rochester Medical Center, Rochester, New York, USA., Luetkemeyer A; Department of Medicine, University of California, San Francisco, San Francisco, California, USA., Zingman BS; Department of Medicine, Montefiore Medical Center, University Hospital for Albert Einstein College of Medicine, Bronx, New York, USA., Voell J; Laboratory of Immunoregulation, National Institute of Allergy and Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA., Whitaker M; COVID-19-Associated Hospitalization Surveillance Network, Centers for Disease Control and Prevention, Atlanta, Georgia, USA., Harkins MS; Division of Pulmonary and Critical Care, Department of Internal Medicine, University of New Mexico Health Sciences Center, Albuquerque, New Mexico, USA., Davey RT Jr; Laboratory of Immunoregulation, National Institute of Allergy and Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA., Grossberg R; Division of Infectious Diseases, Department of Medicine, Montefiore Medical Center/Albert Einstein College of Medicine, Bronx, New York, USA., George SL; Department of Internal Medicine, Saint Louis University and St Louis Veterans Affairs Medical Center, St Louis, Missouri, USA., Tapson V; Division of Pulmonary and Critical Care, Cedars-Sinai Medical Center, Los Angeles, California, USA., Short WR; Division of Infectious Diseases, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA., Ghazaryan V; Division of Microbiology and Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA., Benson CA; Division of Infectious Diseases and Global Public Health, University of California, San Diego, San Diego, California, USA., Dodd LE; Biostatistics Research Branch, Division of Clinical Research, National Institute of Allergy and Infectious Diseases, Rockville, Maryland, USA., Sweeney DA; Division of Pulmonary, Critical Care and Sleep Medicine, Department of Medicine, University of California, San Diego, San Diego, California, USA., Tomashek KM; Division of Microbiology and Infectious Diseases, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland, USA.
Jazyk: angličtina
Zdroj: Open forum infectious diseases [Open Forum Infect Dis] 2023 May 27; Vol. 10 (6), pp. ofad290. Date of Electronic Publication: 2023 May 27 (Print Publication: 2023).
DOI: 10.1093/ofid/ofad290
Abstrakt: Background: Clinical trials initiated during emerging infectious disease outbreaks must quickly enroll participants to identify treatments to reduce morbidity and mortality. This may be at odds with enrolling a representative study population, especially when the population affected is undefined.
Methods: We evaluated the utility of the Centers for Disease Control and Prevention's COVID-19-Associated Hospitalization Surveillance Network (COVID-NET), the COVID-19 Case Surveillance System (CCSS), and 2020 United States (US) Census data to determine demographic representation in the 4 stages of the Adaptive COVID-19 Treatment Trial (ACTT). We compared the cumulative proportion of participants by sex, race, ethnicity, and age enrolled at US ACTT sites, with respective 95% confidence intervals, to the reference data in forest plots.
Results: US ACTT sites enrolled 3509 adults hospitalized with COVID-19. When compared with COVID-NET, ACTT enrolled a similar or higher proportion of Hispanic/Latino and White participants depending on the stage, and a similar proportion of African American participants in all stages. In contrast, ACTT enrolled a higher proportion of these groups when compared with US Census and CCSS. The proportion of participants aged ≥65 years was either similar or lower than COVID-NET and higher than CCSS and the US Census. The proportion of females enrolled in ACTT was lower than the proportion of females in the reference datasets.
Conclusions: Although surveillance data of hospitalized cases may not be available early in an outbreak, they are a better comparator than US Census data and surveillance of all cases, which may not reflect the population affected and at higher risk of severe disease.
Competing Interests: Potential conflicts of interest. N. J. reports salary support to his institution by Sagent Pharmaceuticals. D. I. has received clinical trial funding paid to her institution from Gilead Sciences. M. K. J. has received grant funding paid to her institution from Gilead Sciences. R. A. M. has received grants and research funding to his institution from Gilead Sciences, Pfizer, Sanofi, and GlaxoSmithKline (GSK). A. R. B. has received grant funding to her institution from Pfizer, Merck, and Cynavac, and has consulted for Janssen and GSK. A. L. has received research grant support to her institution from Gilead. R. G. has received research funding from Gilead Sciences and GSK. W. R. S. has received clinical trial funding paid to his institution from Gilead Sciences. C. A. B. has received contracts and grants to her institution from Gilead Sciences. All other authors report no potential conflicts.
(Published by Oxford University Press on behalf of Infectious Diseases Society of America 2023.)
Databáze: MEDLINE
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