TY - JOUR
T1 - Demographic representation in clinical trials for cell-based therapy
AU - Saltzman, Russell G.
AU - Jayaweera, Dushyantha T.
AU - Caceres, Lina V.
AU - Tovar, Jairo A.
AU - Vidro-Casiano, Mayra
AU - Karakeshishyan, Vela
AU - Soto, Jeanette
AU - Khan, Aisha
AU - Mitrani, Raul D.
AU - Schulman, Ivonne H.
AU - Hare, Joshua M.
N1 - Funding Information:
This study was supported by National Heart, Lung, and Blood Institute of Health (NHLBI) grants, 1R01 HL134558 , 5UM 1HL113460 , and R01 HL137355 and by the Starr and Soffer Family Foundations . AK and JMH are also supported by grants, NHLBI-ECB-HB-2016-09-JB and 4UM1HL087318-10 , as well as by Production Assistance for Cell Therapy grants, PCT0013-01 , PCT0004-02 , and PCT0009-01 , along with the Marcus Foundation . URIDE was designed and developed by the Software Engineering team at the University of Miami Institute for Data Science and Computing under the Informatics Program of the Miami Miami Clinical and Translational Science Institute and supported by Grant Award Number UL1TR002736 .
Publisher Copyright:
© 2021
Copyright:
Copyright 2021 Elsevier B.V., All rights reserved.
PY - 2021/3
Y1 - 2021/3
N2 - Inclusion of women and minorities in clinical research is critical to fully assess the safety and efficacy of innovative therapies. With inadequate representation of demography, generalizability is impaired since pharmacokinetics and pharmacodynamics differ in these patient populations. This study was designed to analyze the voluntary participation rates of different demographic groups in cell-based therapy clinical trials conducted by the Interdisciplinary Stem Cell Institute (ISCI) at the University of Miami, Miller School of Medicine. ISCI conducted eight clinical trials between 2007 and 2017. The trials enrolled patients with ischemic and non-ischemic cardiomyopathy, idiopathic pulmonary fibrosis (IPF), aging-frailty, and Type-2 Diabetes. Participants received cell-based therapy (n = 218) or placebo (n = 33). Among the 251 participants, 29.5% were Hispanic and 20% were women. The proportion of individuals participating in each trial was compared to that of the respective disease populations attending University of Miami Health System clinics to calculate the participation to prevalence ratio (PPR). Distribution of women accurately reflected the population attending the University of Miami Health System in trials for dilated cardiomyopathy (DCM) and aging-frailty but was under-represented in others. Similarly, Hispanics and whites were accurately represented in three of the five disease fields, with Hispanics under-represented in frailty and diabetes, and whites over-represented in DCM and IPF. Black patients were accurately represented in the diabetes trial but were under-represented in all others. This study provides insight into challenges of achieving representative inclusion in research. Novel community engagement strategies are necessary to improve inclusion of women and under-represented minorities in clinical research of cell-based therapy.
AB - Inclusion of women and minorities in clinical research is critical to fully assess the safety and efficacy of innovative therapies. With inadequate representation of demography, generalizability is impaired since pharmacokinetics and pharmacodynamics differ in these patient populations. This study was designed to analyze the voluntary participation rates of different demographic groups in cell-based therapy clinical trials conducted by the Interdisciplinary Stem Cell Institute (ISCI) at the University of Miami, Miller School of Medicine. ISCI conducted eight clinical trials between 2007 and 2017. The trials enrolled patients with ischemic and non-ischemic cardiomyopathy, idiopathic pulmonary fibrosis (IPF), aging-frailty, and Type-2 Diabetes. Participants received cell-based therapy (n = 218) or placebo (n = 33). Among the 251 participants, 29.5% were Hispanic and 20% were women. The proportion of individuals participating in each trial was compared to that of the respective disease populations attending University of Miami Health System clinics to calculate the participation to prevalence ratio (PPR). Distribution of women accurately reflected the population attending the University of Miami Health System in trials for dilated cardiomyopathy (DCM) and aging-frailty but was under-represented in others. Similarly, Hispanics and whites were accurately represented in three of the five disease fields, with Hispanics under-represented in frailty and diabetes, and whites over-represented in DCM and IPF. Black patients were accurately represented in the diabetes trial but were under-represented in all others. This study provides insight into challenges of achieving representative inclusion in research. Novel community engagement strategies are necessary to improve inclusion of women and under-represented minorities in clinical research of cell-based therapy.
KW - Cell-based therapy
KW - Clinical trials
KW - Diversity
KW - Generalizability
KW - Health equity
KW - Inclusion
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U2 - 10.1016/j.conctc.2021.100702
DO - 10.1016/j.conctc.2021.100702
M3 - Article
AN - SCOPUS:85099435785
VL - 21
JO - Contemporary Clinical Trials Communications
JF - Contemporary Clinical Trials Communications
SN - 2451-8654
M1 - 100702
ER -