Statistical Considerations in Pediatric Cancer Trials: Report of American Statistical Association Biopharmaceutical Section Open Forum Discussions

Author:

Sridhara Rajeshwari1,Marchenko Olga2,Jiang Qi3,Barksdale Elizabeth4,Alonzo Todd A.5,Amatya Anup6,Arons David7,Bliu Alex8,Choo Qiuyi9,Coory Michael10,Donoghue Martha1,Ehrlich Lori6,Fabio Costa Filho Leonardo11,Fox Elizabeth12,Freidlin Boris13,Goodman Nancy14,Hawkins Douglas S.5,Häring Dieter A.15,Karres Dominik16,Kolb E. Anders17,Mao Helen8,Mishra Kalyani Pallavi S.6,Naranjo Arlene518,Pappo Alberto12,Posch Martin19ORCID,Price Karen20,Raven Andrew8,Rantell Khadija Rerhou21,Renfro Lindsay5,Rivera Donna R.1,Roy Pourab6,Shan Minghua2,Simon Richard22,Singh Sonia6,Smith Malcolm13,Theoret Marc1,Thomas Marius15,Thomas Zachary20,Thompson Andrew16,Tian Hong23,Tymofyeyev Yevgen1,Vallejo Jonathon6,Wathen Kyle1,Ye Jingjing23ORCID,Pazdur Richard24,Reaman Gregory25

Affiliation:

1. Oncology Center of Excellence US FDA, Silver Spring, MD

2. Bayer, Leverkusen, Germany

3. Seagen, Bothell, WA

4. LUNGevity Foundation, Chicago, IL

5. Children’s Oncology Group (COG), Monrovia, CA

6. CDER US FDA, Silver Spring, MD

7. National Brain Tumor Society, Newton, MA

8. HC, Ottawa, Canada

9. HSA, Singapore, Singapore

10. TGA, Woden, Australia

11. ANVISA, Brasilia, Brazil

12. St. Jude Children’s Hospital, Memphis, TN

13. NCI, NIH, Bethesda, MD

14. Kids V Cancer, Washington, DC

15. Novartis, Basel, Switzerland

16. EMA, Amsterdam, Netherlands

17. DuPont Hospital for Children, Wilmington, DE

18. University of Florida, Gainesville, FL

19. Institute for Medical Statistics at the Medical University of Vienna, Vienna, Austria

20. Eli Lilly, Indianapolis, IN

21. MHRA, London, UK

22. Simon Consulting, Phoenix, AZ

23. Beigene, Beijing, China

24. J&J, Titusville, NJ

25. Cytel, Waltham, MA

Funder

Innovative Medicines Initiative 2

Publisher

Informa UK Limited

Subject

Pharmaceutical Science,Statistics and Probability

Reference16 articles.

1. Framework for FDA’s Real-world Evidence Program. (n.d.). https://www.fda.gov/media/120060/download

2. Augmenting randomized clinical trial data with historical control data: Precision medicine applications

3. Guidance for Industry E10 Choice of Control Group and Related Issues in Clinical Trials. (n.d.). https://www.fda.gov/downloads/guidances/ucm073139.pdf

4. Power gains by using external information in clinical trials are typically not possible when requiring strict type I error control

5. Statistical Challenges in the Evaluation of Treatments for Small Patient Populations

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