Drugs

Expand Real-World Evidence Use in Drug Approvals: Article

David McFarland

— 1 min read
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Real-world evidence (RWE) generated from real-world data (RWD) remains underutilized in drug approvals. That’s the report from several drug company officials, including one former FDAer, writing in Therapeutic Innovation & Regulatory Science. The authors note that beyond the fundamental need for relevant and reliable RWD, three interdependent operational barriers constrain otherwise fit-for-use RWD.

They identify the barriers as:

  • FDA submission requirements and data standard policies are centered on CDISC (Clinical Data Interchange Standards Consortium) and optimized for traditional trials rather than heterogeneous RWD sources;
  • expectations about when patient-level data must be submitted to FDA and how and when FDA may access or inspect source records are unclear; and
  • uncertainty remains about the application of regulations to non-interventional studies.

“On one hand,” the authors explain, “the resulting regulatory ambiguity around these issues may lead sponsors and data providers to expend resources on RWE that is ultimately unfit for regulatory purposes. On the other hand, potentially suitable RWD may be ignored. In either case, patient access to effective therapies may be delayed.”

Among the recommendations offered by the authors, FDA could:

  • continue exploring flexible, fit-for-purpose data exchange and submission standards for RWD;
  • make more systematic use of its existing waiver authority to exempt certain RWD submissions from standard format conversion requirements when appropriate;
  • consider expanding the data standards catalog to include HL 7 FHIR (Health Level Seven International Fast Healthcare Interoperability Resources;
  • amend its 8/2023 RWE guidance to clarify the circumstances where the submission of aggregated data may be acceptable to support regulatory decisions;
  • clarify the purpose, focus, and expectations regarding RWD inspections to help data vendors and sponsors meet regulatory expectations in a pragmatic but scientifically rigorous manner.

“By promoting the development and use of flexible, fit-for-purpose data standards, including expanded use of HL7 FHIR and common data models, clarifying expectations for patient-level data access and considering federated or hybrid approaches to data access, and providing explicit guidance on human subject protections for non-interventional studies, FDA can help advance RWE’s potential in regulatory decision making,” the article concludes. “The recommendations described in this perspective would accelerate evidence generation for rare diseases, underserved populations, and post-market insights while maintaining rigorous standards for relevance, reliability, and ethics. Greater regulatory clarity and innovation in data approaches ultimately enable more efficient, patient-centered drug development and oversight.”

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