Real World Evidence (RWE) 201 – FDAs RWE Framework

RWE 201: https://rwr-regs.com/rwe-201/

The FDA’s Real-World Evidence (RWE) Program framework, established under the 21st Century Cures Act, focuses on several key aspects:

[1] Scope of RWE Program Under 21st Century Cures Act: The program aims to evaluate the potential use of RWE to support the approval of a new indication for a drug already approved under section 505(c) of the FD&C Act or to help support or satisfy drug post-approval study requirements (e.g., FDAs Advancing RWE Program).

[2] Current Use of Real-World Data (RWD) for Evidence Generation: RWD sources, such as electronic health records, medical claims, and billing data, can be used for data collection and to develop analysis infrastructure to support various study designs, including randomized clinical trials and observational studies.

Current Uses of RWE:

• Complementing traditional clinical trial data.
• Supporting post-market commitments or requirements.
• Label expansions (e.g., adding new indications for approved drugs).
• Observational studies to understand product safety and effectiveness.

[3] Generating Evidence Regarding Safety and Effectiveness: Historically, there has been limited use of RWE to support effectiveness. Advances in the availability and analysis of RWD have increased the potential for generating robust RWE to support FDA regulatory decisions.  However, the value of using RWE to support regulatory decisions on drug efficacy and effectiveness has yet to be established.  To be explored further when we move to the EU and the EMAs RWE 2025 Vision.

[4] Framework for Evaluating RWD/RWE for Use in Regulatory Decisions: The FDA’s framework for evaluating RWD and RWE involves assessing the fitness of RWD (sources) for use in regulatory decisions, potential for study designs using RWD to support effectiveness, and regulatory considerations for study designs using RWD.

Data Quality and Reliability:

• Not all RWD is fit for regulatory decisions. The FDA assesses the relevance and reliability of RWD.
• Sources of RWD must be transparent, and any transformations to derive RWE must be robust and valid.

Methodological Challenges:

• There are inherent biases in RWD because they are observational in nature
• FDA focuses on minimising biases and promoting the use of appropriate statistical methods.

The framework aims to facilitate the acceleration of medical product development and the delivery of medical innovations for patients in need. It also distinguishes between the sources of RWD and the evidence derived from that data, as evaluating RWE in the context of regulatory decision-making depends on the evaluation of the methodologies used to generate the evidence and the reliability and relevance of the underlying RWD.