TY - JOUR
T1 - Evaluation of Treatment Effect in Underrepresented Population in Cancer Trials
T2 - Discussion with International Regulators
AU - Sridhara, Rajeshwari
AU - Marchenko, Olga
AU - Jiang, Qi
AU - Barksdale, Elizabeth
AU - Chen, Jie
AU - Dreyer, Nancy
AU - Fashoyin-Aje, Lola
AU - Garrett-Mayer, Elizabeth
AU - Gormley, Nicole
AU - Gwise, Thomas
AU - Hess, Lorenzo
AU - Mandrekar, Sumithra
AU - Pignatti, Francesco
AU - Rantell, Khadija
AU - Raven, Andrew
AU - Shen, Yuan Li
AU - Singh, Harpreet
AU - Tendler, Craig L.
AU - Theoret, Marc
AU - Pazdur, Richard
N1 - Publisher Copyright:
© 2022 American Statistical Association.
PY - 2023
Y1 - 2023
N2 - This article provides a summary of discussions from the American Statistical Association (ASA) Biopharmaceutical (BIOP) Section Open Forum on April 8, 2021, and May 13, 2021, organized by the ASA BIOP Statistical Methods in Oncology Scientific Working Group in coordination with the United States Food and Drug Administration (U.S. FDA) Oncology Center of Excellence (OCE) and LUNGevity Foundation. In most cancer trials, disproportionately low numbers of older adults and certain racial minority groups are enrolled, even though a high incidence of cancer is observed in these subpopulations. This results in a lack of sufficient information on efficacy and safety of new treatments in such demographic subpopulations. Discussions with a diverse group of stakeholders including oncologists, patient advocates, experts from international regulatory agencies, academicians, and representatives of the pharmaceutical industry focused on designing future pre- and post-market studies to evaluate treatment effect in demographically underrepresented (UR) cancer populations such as racial and ethnic minority groups and older adults. It is noted that often there is poor or no representation of pediatric cancer patients as well. However, not all adult cancers are observed in pediatric patients and vice-a-versa and these discussions mainly focused on adult cancers. It is recognized that inclusion of broader patient populations can introduce heterogeneity and if the trial includes patients with more comorbidities or poorer prognosis may increase the chances of rejecting an effective therapy. However, there are clinical trial designs and statistical methods to include and evaluate treatment effects in UR cancer patients. Importantly, a commitment and a concerted effort from all stakeholders to change the current practice is necessary to better understand the benefit/risk in these demographically UR patients in cancer clinical trials.
AB - This article provides a summary of discussions from the American Statistical Association (ASA) Biopharmaceutical (BIOP) Section Open Forum on April 8, 2021, and May 13, 2021, organized by the ASA BIOP Statistical Methods in Oncology Scientific Working Group in coordination with the United States Food and Drug Administration (U.S. FDA) Oncology Center of Excellence (OCE) and LUNGevity Foundation. In most cancer trials, disproportionately low numbers of older adults and certain racial minority groups are enrolled, even though a high incidence of cancer is observed in these subpopulations. This results in a lack of sufficient information on efficacy and safety of new treatments in such demographic subpopulations. Discussions with a diverse group of stakeholders including oncologists, patient advocates, experts from international regulatory agencies, academicians, and representatives of the pharmaceutical industry focused on designing future pre- and post-market studies to evaluate treatment effect in demographically underrepresented (UR) cancer populations such as racial and ethnic minority groups and older adults. It is noted that often there is poor or no representation of pediatric cancer patients as well. However, not all adult cancers are observed in pediatric patients and vice-a-versa and these discussions mainly focused on adult cancers. It is recognized that inclusion of broader patient populations can introduce heterogeneity and if the trial includes patients with more comorbidities or poorer prognosis may increase the chances of rejecting an effective therapy. However, there are clinical trial designs and statistical methods to include and evaluate treatment effects in UR cancer patients. Importantly, a commitment and a concerted effort from all stakeholders to change the current practice is necessary to better understand the benefit/risk in these demographically UR patients in cancer clinical trials.
KW - Ethnic and racial minority
KW - Oncology drug development
KW - Underrepresented cancer patients
UR - http://www.scopus.com/inward/record.url?scp=85141187364&partnerID=8YFLogxK
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U2 - 10.1080/19466315.2022.2128404
DO - 10.1080/19466315.2022.2128404
M3 - Letter
AN - SCOPUS:85141187364
SN - 1946-6315
VL - 15
SP - 450
EP - 456
JO - Statistics in Biopharmaceutical Research
JF - Statistics in Biopharmaceutical Research
IS - 2
ER -