The U.S. Food and Drug Administration (FDA) recognizes that common standards and a shared infrastructure for conducting and evaluating real-world data (RWD) studies are essential to determine whether substantial evidence exists—or is even sufficient to make decisions.

A lack of standards in working with RWD continues to challenge advances across specialty care and regulatory review of studies in these areas. Oncology, for example, lacks agreement on how progression and other core measures are defined and applied. Because definitions vary in sources of data, from clinical notes to radiology and pathology, definitions vary across data sets. These data sets, therefore, cannot be easily combined and used, limiting their value.

“To make data interoperable and comparable, we need good standards,” said Deborah Schrag, M.D., Dana Farber’s chief of population sciences, medical oncology, at the Aetion Summit 2019: Evidence at the Apex. “Real-world progression and real-world response needs to mean something consistent.”

The good news is that FDA has launched a process to create an infrastructure to support standardization in working with and submitting real-world evidence (RWE). On September 18, 2019, the FDA’s Office of Information Management and Technology (OIMT) released a Technology Modernization Action Plan. Led by Vid Desai, the FDA’s new Chief Technology Officer, the project’s primary goal is to build a formalized process for RWE submissions and reviews. The 12- to 24-month plan advances the FDA’s draft guidance encouraging sponsors and applicants to include a uniform cover letter when RWE is part of a regulatory submission and the Framework for FDA’s Real-World Evidence Program released in 2018.

With that groundwork in place, the FDA can scale review of RWE submissions and industry can increase the utility of data collected in routine health care encounters.

Technology upgrades are on the way at the agency, says Amy Abernethy, M.D., Ph.D., principal deputy commissioner at FDA, to support standardized processes and definitions for RWE. Speaking at the FDA’s 7th annual Scientific Computing Days symposium in September, she confirmed that the agency is moving toward “centralized data resources … that allow us to have real-time data access, data exchange, and data utilization in a much better way than we have now.

These near-term actions are likely to shape the FDA’s long-term guidance for its staff and industry on using RWE in regulatory decision-making. It’s worthwhile, therefore, to consider their implications for pharma now: 

What you can expect:

  • Stakeholder engagement via public meetings and workshops to better inform the FDA’s policy making process;
  • Collaboration between the FDA, other government agencies, and the data and technology sector on targeted use cases, which may expand support/the set of use cases; and,
  • A strategic plan to be released between March 2021 and September 2022, likely to be followed by a comment period. 

What you can do now to prepare:

Capitalize on this window of time before the plan is released to demonstrate your company’s capabilities in RWE submissions in the following ways:

  1. Use FDA’s proposed RWE cover letter template in product submissions.
  2. Consider the use of a rapid-cycle analytics platform for RWD analyses. Platforms enable increased speed to insight, adoption of widely accepted metrics and statistical procedures, testing against FDA’s modular programs such as Sentinel, and capturing of complete reporting according to ISPE/ISPOR and RECORD-PE guidelines.
  3. Grant FDA access to your platform’s review environment. FDA can then test a real-world study’s ability to identify true effects and can disregard spurious associations.


As FDA takes time for infrastructure development that enables standardization, stakeholders across health care can begin their own planning. This is a critical opportunity to learn and assess your own capabilities in moving from real-world data to real-world evidence. This is the time to prepare your organization for a common approach that meets the regulatory standards of the FDA.