Cancer Clinical Trials beyond Pandemic: Report of an American Statistical Association Biopharmaceutical Section Open Forum Discussion

Abstract

This article provides a summary of discussions from the American Statistical Association (ASA) Biopharmaceutical (BIOP) Section Open Forum organized by the ASA BIOP Statistical Methods in Oncology Scientific Working Group in coordination with the US FDA Oncology Center of Excellence and LUNGevity Foundation on January 14, 2021, and February 8, 2021. Diverse stakeholders including oncologists, patient advocates, experts from international regulatory agencies, academicians, and representatives of the pharmaceutical industry engaged in a discussion on how best to incorporate lessons learned during the COVID-19 pandemic into the design of future oncology trials. While recognizing that decentralized or hybrid cancer trials may increase variability associated with measurement error and potentially increase bias in treatment effect estimation, panel discussions highlighted the importance of flexibility for decreasing patient burden, which has the potential to increase access to and retention in cancer clinical trials and may broaden the representation of real-world patients in the trial setting.

Keywords:

• COVID-19 pandemic
• Decentralized trials
• Oncology drug development
• Telemedicine

Acknowledgments

Authors thank Ms. Joan Todd (OCE FDA), Dr. Weidong Zhang (Jounce Therapeutics), and Hong Tian (Janssen) for taking the meeting minutes.

Disclosure Statement

No potential competing interest was reported by the authors.

Martin Posch is a member of the EU Patient-centric clinical trial platform (EU-PEARL). EU-PEARL has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement N${}^{°}$

853966. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA and Children’s Tumor Foundation, Global Alliance for TB Drug Development nonprofit organization, Springworks Therapeutics Inc. This publication reflects the authors’ views. Neither IMI nor the European Union, EFPIA, or any Associated Partners are responsible for any use that may be made of the information contained herein.

Kit Roes and Martin Posch are affiliated with EMA’s Biostatistics Working Party. Filip Josephson and Sinan Sarac are affiliated with EMA’s Committee for Medicinal Products for Human Use. The views expressed in this article are the personal views of the authors and may not be understood or quoted as being made on behalf of or reflecting the position of the regulatory agency/agencies or organizations with which the authors is/are employed/affiliated.

Funding

The author(s) reported there is no funding associated with the work featured in this article.