FDA’s Draft Real-World Evidence Program Will Help Evaluate New Drug Indications but More Details Required

ASCO proposes changes to draft framework in comment letter to FDA Commissioner
February 7, 2019

The U.S. Food and Drug Administration (FDA) recently issued a draft Framework for a Real-World Evidence Program, as required under the 21st Century Cures Act. The proposed program would help evaluate the potential for using real-world evidence (RWE) to support the approval of a new indication of already approved drugs, or to help support or satisfy drugs’ post-approval study requirements.

In a letter to FDA Commissioner Scott Gottlieb, MD, ASCO indicated its support for the multifaceted RWE program and offered comments to ensure the RWE framework is usable and interoperable.

In particular, ASCO highlighted the need to develop appropriate data standards and a common format to maximize the utility of real-world data (RWD) .  

“In general, the lack of data standards presents a problem even beyond the contemplation of their use in the RWE framework. This is especially true in specialty fields such as oncology,” said ASCO President Monica Bertagnolli, MD, FACS, FASCO. Dr. Bertagnolli also noted that ASCO is actively working on the mCODE™ project, an effort designed to result in a set of consensus-developed oncology data elements necessary for critical information exchange between EHRs for clinical care, quality reporting, and other use cases. It is possible that the results of these efforts could feed into the RWE framework over time, helping to establish agreed-upon data elements for oncology.

ASCO also asked FDA to:

  • Consider using an existing common data model (CDM) (e.g. the PCORnet Common Data Model)
  • Include pharmacy databases and social media as examples of RWD sources
  • Clarify the types of clinical trials and observational studies that will be covered by the RWE program, and in which situations observational data will be accepted to inform RWE
  • Provide specific examples of how FDA has used RWE data in the past for regulator decision-making
  • Include examples of oncology-specific outcomes that can be collected using RWD
  • Clarify how FDA expects to use study monitoring to determine if a study meets FDA regulatory requirements
  • Provide more detail about FDA’s expectations for data validity with respect to efficacy endpoints
  • Specify if the review of existing RWD/RWE-driven work will be directed by one of the existing standards-developing organizations in healthcare, or by an internal and external stakeholder collaboration.
  • Explain the types of stakeholder engagement that would be most useful to facilitate learning and collaboration

Read the full comment letter to FDA.

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