A proliferation of new data sources and sophisticated analytic methods are helping researchers transform real-world data (RWD) into real-world evidence (RWE) to address important healthcare questions. Regulators and policymakers recognize the power of policy to realize the full potential of data to improve patient care and outcomes.
In January 2025, FDA issued a draft guidance titled “Considerations for the Use of Artificial Intelligence to Support Regulatory Decision-Making for Drug and Biological Products.” This draft guidance describes considerations on the use of AI to support regulatory decision making in a wide variety of contexts, including by processing and analyzing large sets of RWD.
In December 2024, FDA issued draft guidance on the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Guideline titled “E6(R3) Good Clinical Practice: Annex 2.” The ICH guideline addresses GCP considerations that arise from the use of a wider range of design elements and data sources, focusing on examples of trials that incorporate decentralized elements, pragmatic elements, and/or real-world data.
In June 2024, FDA issued draft guidance titled “Diversity Action Plans to Improve Enrollment of Participants from Underrepresented Populations in Clinical Studies.” This draft guidance describes considerations for the submission and content of Diversity Action Plans.
In September 2024, FDA issued draft RWE Program guidance titled “Integrating Randomized Controlled Trials for Drug and Biological Products Into Routine Clinical Practice.” This draft guidance provides recommendations for the conduct of randomized controlled trials with streamlined protocols and procedures that focus on essential data collection, allowing integration of research into routine clinical practice.
In July 2024, FDA issued draft guidance on the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) Guideline titled “M14 General Principles on Plan, Design, and Analysis of Pharmacoepidemiological Studies That Utilize Real-World Data for Safety Assessment of Medicines.” The ICH guideline describes recommendations for post-marketing safety studies that use RWD.
In September 2021, FDA issued draft guidance titled “Real-World Data: Assessing Electronic Health Records and Medical Claims Data To Support Regulatory Decision-Making for Drug and Biological Products.” This draft guidance provided recommendations for using real-world evidence derived from electronic health records and medical claims data for regulatory decisions across therapeutic areas. The RWE Alliance commented on the draft guidance in January 2022, and FDA issued final guidance in July 2024.
In December 2023, FDA issued draft guidance titled “Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices.” This draft guidance builds on the Agency’s earlier 2017 final guidance and provides expanded recommendations on considerations for the use of RWE for medical devices.
In March 2024, FDA issued draft guidance titled “Real-World Evidence: Considerations Regarding Non-Interventional Studies for Drug and Biological Products.” This draft guidance provides recommendations for sponsors planning to submit a non-interventional study to FDA to help demonstrate substantial evidence of effectiveness or evidence of safety of a drug.
In September 2023, FDA issued draft guidance titled “Demonstrating Substantial Evidence of Effectiveness Based on One Adequate and Well-Controlled Clinical Investigation and Confirmatory Evidence.” This draft guidance complements the Agency’s previous guidance for industry on effectiveness by describing considerations for assessing whether a single adequate and well-controlled clinical investigation and confirmatory evidence are sufficient to demonstrate substantial evidence of effectiveness, and by providing examples of sources of confirmatory evidence, including RWD/E.
In December 2021, FDA issued draft guidance titled “Considerations for the Use of Real-World Data and Real-World Evidence To Support Regulatory Decision-Making for Drug and Biological Products.” This draft guidance outlined various regulatory considerations for submissions containing RWD/E. The RWE Alliance commented on the draft guidance in March 2022, and FDA issued final guidance in August 2023.
In March 2023, FDA issued draft guidance titled “Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics.” This draft guidance provides recommendations to sponsors of oncology products on considerations for designing trials intended to support accelerated approval.
In February 2023, FDA issued draft guidance titled “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products.” This draft guidance provides recommendations for using externally controlled trials to demonstrate the safety and effectiveness of drugs.
In November 2021, FDA issued draft guidance titled “Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products Guidance for Industry.” This draft guidance provided recommendations for using RWD derived from data registries to support regulatory submissions. The RWE Alliance commented on the draft guidance in February 2022, and FDA issued final guidance in December 2023.
In October 2021, FDA issued draft guidance titled “Data Standards for Drug and Biological Product Submissions Containing Real-World Data.” This draft guidance provided recommendations for applying FDA’s currently supported data standards to submissions containing RWD. The RWE Alliance commented on the draft guidance in February 2022, and FDA issued final guidance in December 2023.