RWE 201 – FDA’s Advancing RWE Program

Real-world evidence is transforming the regulatory landscape, enabling the FDA to make informed decisions based on robust data from real-world settings. In line with this, the FDA has launched the Advancing Real-World Evidence (RWE) Program, aiming to harness the benefits of RWD to support new labelling claims, expand indications for approved medical products, and fulfil post-approval study requirements.

Goals of the Program:

  1. Identify strategies for creating RWE that satisfies regulatory needs for labelling effectiveness or post-approval study obligations.
  2. Establish agency protocols for consistent RWE decision-making and shared knowledge.
  3. Encourage public understanding of RWE’s attributes for informed regulatory choices through public discussions.


  1. Sponsors must have a pre-IND or IND number for their medical product
  2. The RWE proposal should aim at supporting labelling for effectiveness or meeting post-approval study demands
  3. Both FDA and sponsor must consent to publicly disclose the study design


  1. The FDA welcomes all fitting RWE proposals. Selection is based on data’s suitability, study design quality, regulatory conduct, and diverse data sources and methodologies

Submission Process:

  1. Semi-annual submission deadlines: 31 March and 30 September, continuing until 31 March 2027
  2. Sponsors will be told whether their meeting request has been accepted within 45 days post-deadline. Successful applicants can apply for an additional 3 follow-up meetings.

Initial Meeting Request Content – Sponsors must provide:

  1. Product name
  2. IND number
  3. Study purpose (new labelling/post-approval study)
  4. Proposed indication
  5. Product development history
  6. Rationale for RWE approach
  7. Study design overview
  8. Non-disclosable design elements and reasons for their omission
  9. Study design details: objectives, design, eligibility, endpoints, treatment, comparator, concomitant therapies
  10. Data sources: category, description, validation, linkage, additional data collection.
  11. Analysis plan: sample size, analytic plan, confounding factors, follow-up period, data handling
  12. Other factors: study pre-specification, patient data availability, human subject protection

Disclosure Agreement:

Before initial meetings, FDA and sponsors decide on public data disclosure. Certain details, like sponsor name and patient data, are excluded from disclosure. However, unique disclosure considerations are allowed. Participation is voluntary.

In conclusion, the FDA’s Advancing RWE Program offers a structured approach for sponsors to collaborate with the FDA and harness the potential of RWE in label extensions and post market studies.

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