Clinical Evidence Generation During a Pandemic

Abstract

Because of significant adaptations forced by the COVID-19 pandemic, resultant changes within health care delivery and clinical research introduced the potential for evaluation of novel evidence generation approaches in oncology. On July 26 and 27, 2021, the National Academies of Science, Engineering, and Medicine, National Cancer Policy Forum hosted a virtual workshop entitled "Cancer Care and Cancer Research in the Context of the COVID-19 Pandemic: A Workshop on Lessons Learned." This workshop examined changes in cancer care and cancer research that occurred in response to the COVID-19 pandemic and considered lessons learned from that experience. The goal was to identify what changes could improve the delivery of high-quality cancer care and the conduct of cancer clinical trials in the postpandemic era, with an emphasis on health equity. How can we sustain the valuable lessons learned that might accelerate progress and enhance clinical evidence generation for patients and clinicians? In this overview, we discuss ways in which the COVID-19 experience has catalyzed research efficiencies as well as fostered a broader array of trial design and research methods that may facilitate improved cancer drug development during the pandemic and beyond.

Regulatory and HTA Considerations for Development of Real‐World Data Derived External Controls

“…This adoption has accelerated since the emergence of the coronavirus disease 2019 (COVID-19) pandemic, which both made traditional trials more difficult and motivated new efforts and collaborations to use RWE to inform decision making. 6 Despite recent guidance on the appropriate use of RWE to support regulatory and HTA approvals, [7][8][9][10][11][12][13][14][15][16][17] best operational and methodological practices for executing an RWD-derived external control study are still being defined. [18][19][20] The aim of this review is to delineate a set of key takeaways to support the responsible generation of RWD-derived external controls for stakeholders submitting evidence to regulatory and HTA decision-making bodies to support evidence of product effectiveness and safety.…”

“…RWE, generated from analysis of RWD, is increasingly leveraged to support evaluation of product effectiveness. This adoption has accelerated since the emergence of the coronavirus disease 2019 (COVID‐19) pandemic, which both made traditional trials more difficult and motivated new efforts and collaborations to use RWE to inform decision making 6 . Despite recent guidance on the appropriate use of RWE to support regulatory and HTA approvals, 7–17 best operational and methodological practices for executing an RWD‐derived external control study are still being defined 18–20 …”

A call to action to advance patient‐focused and decentralized clinical trials

“…17 In March 2020, the FDA released interim guidance for conducting clinical trials during the coronavirus disease 2019 (COVID- 19) public health emergency, 18 which allowed more people to participate in trials locally by receiving treatments and completing testing through their local oncologist, accessing telemedicine for remote monitoring, and using nearby laboratory and imaging centers. [18][19][20][21][22][23][24] This decentralized approach meant that a patient in an underserved setting no longer bore the time, financial, and logistical burdens of traveling to a designated trial center to receive studyrelated treatment and/or testing. These provisions eliminated a known impediment to clinical trial participation and retention, and information suggests there was little to no impact on data quality.…”