Reflecting on the Promise of Real-World Data, a Fireside Chat with the FDA


Amy Abernathy, Principal Deputy Commissioner & Acting CIO, US Food & Drug Administration

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Syapse hosted its 3rd annual Precision Medicine Council to convene healthcare leaders to discuss applications for real-world insights and common challenges in delivering real-world care.

As a part of the council, we invited Dr. Amy Abernethy, Principal Deputy Commissioner of the FDA, to share her perspective on the complementary role of real-world evidence to prospective clinical trials; the path to establishing the quality bar for the regulatory use of real-world evidence; and the role that collaborative forums such as the Evidence Accelerator, organized by the Reagan-Udall Foundation for the FDA in collaboration with the Friends of Cancer Research, play in bringing us closer to alignment on those standards.

Syapse’s chief medical officer, Dr. Thomas Brown, hosted the fireside chat. As we reflect on the discussion, we remain confident in the FDA’s ability to further the use of real-world evidence and have highlighted three takeaways below. 

The applications of real-world evidence are rapidly expanding 
Real-world evidence is complementary to traditional clinical trials, allowing for insights that are applicable to a much broader population. For example, patients with comorbidities are often excluded from clinical trials; however, doctors still need insight into how they are likely to respond to particular treatments. Recently, the study of real-world evidence during the COVID-19 pandemic demonstrated the value of both building a longitudinal natural history of the disease and using near real-time data to monitor the impact of regulatory decisions and adjust accordingly.

Oncology is the ideal proving ground 
Oncology is fertile ground for advancing the standards and methods that are used to develop high quality real-world evidence for a number of reasons, among them: 

  • consistent and fixed outcomes (e.g. limited spontaneous recovery);
  • a rapidity of innovation driven by the urgency of patient need;
  • a long history of continuous learning, registries and common data models;

While many of the challenges will be worked through in oncology for these reasons, the application of real-world evidence will not end with oncology. The opportunity for continuous learning and real-time monitoring through real-world evidence is incredibly broad.

Expect more guidance from the FDA on the use of real-world data in 2021 
The FDA is the governing body for “defining what good looks like” for drug approval, and by extension patient care. The drug approval process comes with a certain level of risk, and the FDA’s objective is to support the use of science and credible evidence to improve outcomes and access. In 2021, we can look forward to additional guidance from the FDA on using real-world evidence, thus growing its adoption.  With real world data sources continuing to evolve, further definition and validation of quality standards and outcomes will improve development of real-world evidence to benefit the care of all patients. 


About the Syapse Learning Health Network™ 
The Learning Health Network is an international network of healthcare providers enabling improved cancer care by sharing real-world data to support clinical decisions and by fostering collaborations among participants. Healthcare providers, including doctors and nurses, share and learn which cancer treatments produced the best real-world outcomes in clinically and molecularly similar patients. Researchers learn from real-world clinical, molecular, treatment and outcomes data. These collaborations are enabled by a secure platform of shared de-identified data that is standardized and normalized across the Learning Health Network and complies with all applicable federal and state data privacy protection regulations.