Podcast: Non-binding Guidance: Real-World Evidence in Drug Development and FDA Submissions
FDA’s Center for Biologics Evaluation and Research (“CBER”) recently issued a trio of new draft guidances to assist sponsors who are developing and conducting postapproval studies of cell and gene therapies (“CGTs”):...more
The U.S. Food and Drug Administration (FDA) has published its guidance agenda for Fiscal Year 2026, announcing plans for eight guidance documents to be released this upcoming year....more
On September 30, 2025, the Food and Drug Administration (FDA) published a Request for Public Comment to obtain feedback from interested parties on current approaches to measuring and evaluating the performance of AI-enabled...more
In late September, the U.S. Food & Drug Administration (“FDA”) issued three draft guidances related to cell and gene therapy (“CGT”) products: (1) Innovative Designs for Clinical Trials of CGT Products in Small Populations...more
The U.S. Food and Drug Administration (FDA) on Sept. 20, 2025, issued draft guidance, "Expedited Programs for Regenerative Medicine Therapies for Serious Conditions," outlining how sponsors can utilize FDA's expedited review...more
On Wednesday, the U.S. Food and Drug Administration (FDA) published three new draft guidances that aim to offer greater clarity to sponsors of cell and gene therapies (CGTs) on issues related to clinical trial design,...more
Ropes & Gray recently hosted its Third Annual Ropes & Gray Rare Disease Forum on May 1, 2025 in Cambridge, Massachusetts. The event gathered legal, commercial, and patient advocacy experts in the rare disease community to...more
On December 12, 2024, FDA’s Center for Drug Evaluation and Research (CDER) announced a new CDER Center for Real-World Evidence Innovation (CCRI). The CCRI is intended to serve as the focal point to promote more efficient and...more
On July 25, 2024, the US Food and Drug Administration (FDA) announced the release of a final version of its Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making...more
The U.S. Food and Drug Administration (FDA) recently published its criteria for reporting on the use of real-world evidence (RWE) in drug and biologic submissions, as part of its commitments under the Prescription Drug User...more
In December 2023, the U.S. Food and Drug Administration (FDA) issued draft guidance on the use of real-world evidence to support regulatory decision-making for medical devices. While the guidance is currently in draft form...more
On September 19, FDA released a highly anticipated new draft guidance for industry titled Demonstrating Substantial Evidence of Effectiveness Based on One Adequate and Well-Controlled Clinical Investigation and Confirmatory...more
The Consolidated Appropriations Act for 2023 was signed into law on December 29, 2022 and includes the Food and Drug Omnibus Reform Act (FDORA). FDORA authorizes a variety of new and important amendments to the Food, Drug,...more
The Food and Drug Administration (“FDA” or the “Agency”) user fee negotiations may have hit an iceberg, but it did not sink all the legislative riders that accompanied this summer’s House and Senate bills. Buried within the...more
Last month, the U.S. Food and Drug Administration (FDA) issued a final guidance on drug and biologic submissions to the agency that include real-world data (RWD) and real-world evidence (RWE). The procedural guidance aims to...more
Report on Research Compliance 19, no. 10 (October, 2022) - Beginning Oct. 1, Renee Wegrzyn will be the first permanent director of the Advanced Research Projects Agency for Health (ARPA-H), the new HHS agency modeled...more
This month, FDA released the latest in a series of agency guidance to advance the development and use of real-world data (RWD) and real-world evidence (RWE) in clinical trial designs for drug development programs. The draft...more
On December 8, 2021 and in response to the mandate under the 21st Century Cures Act to issue guidance on the use of real-world evidence (RWE) in regulatory decision-making, the U.S. Food and Drug Administration (FDA) released...more
Last week the FDA issued another draft guidance in its series of recent guidance documents setting forth the agency’s views regarding the generation and use of Real-World Data (RWD) and Real-World Evidence (RWE) for...more
The US Food and Drug Administration has issued draft guidance outlining the agency’s recommendations for using real-world data from (and curating such data in) registries to support regulatory decision-making. Pharmaceutical...more
The U.S. Food and Drug Administration (FDA) issued its third draft guidance under the Real-World Evidence (RWE) Program on November 29, 2021. In Real-World Data: Assessing Registries to Support Regulatory Decision-Making for...more
Earlier this week, the Food and Drug Administration released the latest in a series of draft guidance documents on the topic of real-world data (RWD) to support regulatory decision-making for drugs and biological products....more
Showing no signs of food coma, the FDA issued draft guidance on the Monday following the Thanksgiving holiday weekend that outlines considerations for sponsors proposing to design a registry or use an existing registry to...more
The global pandemic has accelerated the Food and Drug Administration’s (FDA) encouragement of real-world data (RWD) and real-world evidence (RWE) (the clinical evidence derived from analysis of RWD), particularly as the FDA...more
Last week, Diana DeGette (D-CO) and Fred Upton (R-MI) introduced in the House highly anticipated bill language for “Cures 2.0”, a follow-up to the transformational 21st Century Cures Act enacted in 2016... The 21st Century...more