Jubilee Brown1, R Wendel Naumann2, William E Brady3, Robert L Coleman4, Kathleen N Moore5, David M Gershenson6. 1. Levine Cancer Institute at the Carolinas HealthCare System, 1021 Morehead Medical Drive, Suite 2100, Charlotte, NC 28204, United States. Electronic address: jubilee.brown@carolinashealthcare.org. 2. Levine Cancer Institute at the Carolinas HealthCare System, 1021 Morehead Medical Drive, Suite 2100, Charlotte, NC 28204, United States. Electronic address: wendel.naumann@carolinashealthcare.org. 3. NRG Oncology/Gynecologic Oncology Group Statistics & Data Center, Roswell Park Cancer Institute, Buffalo, NY 14263, United States. Electronic address: bbrady@gogstats.org. 4. Department of Gynecologic Oncology and Reproductive Medicine, The University of Texas MD Anderson Cancer Center, 1155 Herman Pressler Drive, Houston, TX 77030, United States. Electronic address: rcoleman@mdanderson.org. 5. Department of OB/GYN, University of Oklahoma Health Sciences Center, 825 NE 10th Street, Oklahoma City, OK 73104, United States. Electronic address: kathleen-moore@ouhsc.edu. 6. Department of Gynecologic Oncology and Reproductive Medicine, The University of Texas MD Anderson Cancer Center, 1155 Herman Pressler Drive, Houston, TX 77030, United States. Electronic address: dgershen@mdanderson.org.
Abstract
BACKGROUND: Performing clinical trials in rare gynecologic cancers presents specific challenges. Strategies for improving accrual and modifications in clinical trial design are outlined. METHODS: The literature was reviewed in order to present statistical designs pertinent to the study of rare gynecologic cancers. The experience of the Gynecologic Oncology Group/NRG Oncology is outlined as it relates to rare gynecologic cancer clinical trial development. RESULTS: Significant progress has been made in studying rare tumors, both nationally and in gynecologic oncology, but challenges inherent to the study of uncommon diseases remain. Important components of these trials include establishing the standard of care, utilizing the appropriate clinical trial design to effectively answer the question in the trial, accurately estimating sample size, choosing modified and realistic endpoints, and avoiding pitfalls specific to rare tumors. Adaptive trial design and statistical modifications are important components of clinical trial design in rare tumors. CONCLUSION: Strategies for effective study of rare gynecologic cancers must be implemented when designing clinical trials for these patients.
BACKGROUND: Performing clinical trials in rare gynecologic cancers presents specific challenges. Strategies for improving accrual and modifications in clinical trial design are outlined. METHODS: The literature was reviewed in order to present statistical designs pertinent to the study of rare gynecologic cancers. The experience of the Gynecologic Oncology Group/NRG Oncology is outlined as it relates to rare gynecologic cancer clinical trial development. RESULTS: Significant progress has been made in studying rare tumors, both nationally and in gynecologic oncology, but challenges inherent to the study of uncommon diseases remain. Important components of these trials include establishing the standard of care, utilizing the appropriate clinical trial design to effectively answer the question in the trial, accurately estimating sample size, choosing modified and realistic endpoints, and avoiding pitfalls specific to rare tumors. Adaptive trial design and statistical modifications are important components of clinical trial design in rare tumors. CONCLUSION: Strategies for effective study of rare gynecologic cancers must be implemented when designing clinical trials for these patients.