Advancing Clinical Trial Design for Non-Muscle Invasive Bladder Cancer

Author:

Chang Elaine1,Hahn Noah M.2,Lerner Seth P.3,Fallah Jaleh1,Agrawal Sundeep1,Kamat Ashish M.4,Bhatnagar Vishal5,Svatek Robert S.6,Jaigirdar Adnan A.7,Bross Peter7,Shore Neal8,Kates Max9,Sachse Karen10,Brewer Jamie R.1,O’Donnell Michael A.11,Steinberg Gary D.12,Viviano Charles J.13,Bloomquist Erik1,Ribal Maria J.14,Galsky Matthew D.15,Oliver Richard,Black Peter C.16,Al-Ahmadie Hikmat17,Brothers Kenneth18,Pohar Kamal19,Dinney Colin P.4,Feng Zhou1,Downs Tracy M.20,Porten Sima P.21,Smith Angela B.22,Bangs Rick10,Psutka Sarah P.23,Agarwal Neeraj24,Amiri-Kordestani Laleh1,Suzman Daniel L.1,Pazdur Richard5,Kluetz Paul G.5,Weinstock Chana1

Affiliation:

1. Center for Drug Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA

2. Johns Hopkins Greenberg Bladder Cancer Institute, Johns Hopkins School of Medicine, Baltimore, MD, USA

3. Scott Department of Urology, Baylor College of Medicine, Houston, TX, USA

4. Department of Urology, University of Texas MD Anderson Cancer Center, Houston, TX, USA

5. Oncology Center of Excellence, Food and Drug Administration, Silver Spring, MD, USA

6. Department of Urology, University of Texas Health San Antonio, San Antonio, TX, USA

7. Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA

8. Department of Urology, Carolina Urologic Research Center, Myrtle Beach, SC, USA

9. Department of Urology, Johns Hopkins Greenberg Bladder Cancer Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA

10. Bladder Cancer Advocacy Network, Bethesda, MD, USA

11. Department of Urology, University of Iowa, Iowa City, IA, USA

12. Goldstein Bladder Cancer Program, NYU Langone Health, NYU Urology Associates, New York, NY, USA

13. Center for Devices and Radiological Health, Food and Drug Administration, Silver Spring, MD, USA

14. Uro-Oncology Unit, Hospital Clinic, University of Barcelona, Barcelona, Spain

15. Hematology-Oncology, Icahn School of Medicine at Mount Sinai/Tisch Cancer Institute, New York, NY, USA

16. Department of Urologic Sciences, University of British Columbia, Vancouver, BC, Canada

17. Department of Pathology and Laboratory Medicine, Genitourinary Pathology Service, Memorial Sloan Kettering Cancer Center, New York, NY, USA

18. National Cancer Institute Bladder Cancer Task Force, Bethesda, MD, USA

19. Department of Urology, The Ohio State University, Columbus, OH, USA

20. Department of Urology, University of Virginia, Charlottesville, VA, USA

21. Department of Urology, University of California San Francisco, San Francisco, CA, USA

22. Department of Urology, University of North Carolina, Lineberger Comprehensive Cancer Center, Chapel Hill, NC, USA

23. Department of Urology, University of Washington, Seattle Cancer Care Alliance, Seattle, WA, USA

24. Huntsman Cancer Institute, University of Utah, Salt Lake City, UT, USA

Abstract

BACKGROUND: Despite recent drug development for non-muscle invasive bladder cancer (NMIBC), few therapies have been approved by the US Food and Drug Administration (FDA), and there remains an unmet clinical need. Bacillus Calmette-Guerin (BCG) supply issues underscore the importance of developing safe and effective drugs for NMIBC. OBJECTIVE: On November 18–19, 2021, the FDA held a public virtual workshop to discuss NMIBC research needs and potential trial designs for future development of effective therapies. METHODS: Representatives from various disciplines including urologists, oncologists, pathologists, statisticians, basic and translational scientists, and the patient advocacy community participated. The workshop format included invited lectures, panel discussions, and opportunity for audience discussion and comment. RESULTS: In a pre-workshop survey, 92% of urologists surveyed considered the development of alternatives to BCG as a high drug development priority for BCG-naïve high-risk patients. Key topics discussed included definitions of disease states; trial design for BCG-naïve NMIBC, BCG-unresponsive carcinoma in situ, and BCG-unresponsive papillary carcinoma; strengths and limitations of single-arm trial designs; assessing patient-reported outcomes; and considerations for assessing avoidance of cystectomy as an efficacy measure. CONCLUSIONS: The workshop discussed several important opportunities for trial design refinement in NMIBC. FDA encourages sponsors to meet with the appropriate review division to discuss trial design proposals for NMIBC early in drug development.

Publisher

IOS Press

Subject

Urology,Oncology

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线,采集、加工和组织学术论文而形成的新型学术文献查询和分析系统,可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容,当前同舟云学术共收录了国内外主流学术期刊6万余种,收集的期刊论文及会议论文总量共计约1.5亿篇,并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询!咨询电话:010-8811{复制后删除}0370

www.globalauthorid.com

TOP

Copyright © 2019-2024 北京同舟云网络信息技术有限公司
京公网安备11010802033243号  京ICP备18003416号-3