TY - JOUR
T1 - Are Pivotal Clinical Trials for Drugs Approved for Leukemias and Multiple Myeloma Representative of the Population at Risk?
AU - Casey, Mycal
AU - Odhiambo, Lorriane
AU - Aggarwal, Nidhi
AU - Shoukier, Mahran
AU - Garner, Jamani
AU - Islam, K. M.
AU - Cortes, Jorge E.
N1 - Publisher Copyright:
© American Society of Clinical Oncology.
PY - 2022/11/10
Y1 - 2022/11/10
N2 - PURPOSEThere are significant disparities in care and outcomes for patients with leukemias and multiple myeloma (MM). To evaluate the extent to which clinical trials (CTs) match the demographic and geographic diversity of populations affected by leukemias and MM.METHODSCTs leading to drug approval were identified from the US Food and Drug Administration databases. Demographic and geographic data were collected from ClinicalTrials.gov and primary manuscripts. Standard descriptive statistics were used to summarize the data in frequencies and proportions, including 95% CIs, by race, ethnicity, sex, and malignancy subtypes.RESULTSA total of 41 (67.2%) trials leading to drug approval reported data on race and 20 (48.8%) on ethnicity. These trials included 13,731 patients, of whom 11,209 (81.6%) were White. Among minorities, Asian-Pacific Islanders and Blacks had the highest representation in chronic myeloid leukemia, 147 (12.7%) and 61 (5.3%), and lowest in chronic lymphocytic leukemia, 55 (3%) and 20 (1.1%), respectively. Proportions for Blacks, Native Americans, and Hispanics were significantly low, reflecting under-representation in trials compared with the proportion in the general population. Females were also under-represented in acute myeloid leukemia (44.7% v 60.5%, P <.0001), and males in MM (55.3% v 60.2%, P <.0001) and chronic myeloid leukemia (55.2% v 62.9%, P <.0001). The geographic distribution of trials showed inadequate regional and state participation compared with mortality for all malignancies except MM.CONCLUSIONThere are significant demographic and geographic under-representation and imbalances in pivotal CTs leading to drug approvals for leukemias and MM compared with the population affected. These disparities need to be addressed to make results applicable to all relevant populations.
AB - PURPOSEThere are significant disparities in care and outcomes for patients with leukemias and multiple myeloma (MM). To evaluate the extent to which clinical trials (CTs) match the demographic and geographic diversity of populations affected by leukemias and MM.METHODSCTs leading to drug approval were identified from the US Food and Drug Administration databases. Demographic and geographic data were collected from ClinicalTrials.gov and primary manuscripts. Standard descriptive statistics were used to summarize the data in frequencies and proportions, including 95% CIs, by race, ethnicity, sex, and malignancy subtypes.RESULTSA total of 41 (67.2%) trials leading to drug approval reported data on race and 20 (48.8%) on ethnicity. These trials included 13,731 patients, of whom 11,209 (81.6%) were White. Among minorities, Asian-Pacific Islanders and Blacks had the highest representation in chronic myeloid leukemia, 147 (12.7%) and 61 (5.3%), and lowest in chronic lymphocytic leukemia, 55 (3%) and 20 (1.1%), respectively. Proportions for Blacks, Native Americans, and Hispanics were significantly low, reflecting under-representation in trials compared with the proportion in the general population. Females were also under-represented in acute myeloid leukemia (44.7% v 60.5%, P <.0001), and males in MM (55.3% v 60.2%, P <.0001) and chronic myeloid leukemia (55.2% v 62.9%, P <.0001). The geographic distribution of trials showed inadequate regional and state participation compared with mortality for all malignancies except MM.CONCLUSIONThere are significant demographic and geographic under-representation and imbalances in pivotal CTs leading to drug approvals for leukemias and MM compared with the population affected. These disparities need to be addressed to make results applicable to all relevant populations.
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U2 - 10.1200/JCO.22.00504
DO - 10.1200/JCO.22.00504
M3 - Article
C2 - 35944216
AN - SCOPUS:85141893218
SN - 0732-183X
VL - 40
SP - 3719
EP - 3729
JO - Journal of Clinical Oncology
JF - Journal of Clinical Oncology
IS - 32
ER -