Real-World Data and Evidence Generation with Dr. Hilary Marston

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10/01/2024

Photo by Austin Distel on Unsplash

“Q&A with FDA” is the US Food and Drug Administration’s (FDA) podcast that began in 2022 which answers frequently asked questions by interviewing FDA staff members. The most recent episode was released on August 16th, 2024 titled “Real-World Data and Evidence Generation with Dr. Hilary Marston” hosted by Dr. Sara Roach interviewing Dr. Marston, the FDA’s Chief Medical Officer. The interview gives a comprehensive overview of Real-Word Data (RWD), Real-World Evidence (RWE), and the FDA’s perspective on their use in regulatory decision making.  

RWD, as Dr. Marston defines it, is “the information relating to a patients health status or the delivery of health care, that are routinely collected from a variety of sources” and listed examples including electronic health records (EHR), medical claims data (MCD), and data from product or disease registries. RWE “is derived from analysis of RWD, and it’s the clinical evidence about the usage and potential benefits and risks of a medical products”.

Topics covered through the podcast include:

• The history and current uses of RWD and RWE at the FDA:

  • RWD has been used in the FDA since digital health technologies (DHT) made it easier to transmit data.

  • Dr. Marston explained how DHTs can give more information on patient interactions with drugs. She uses the example of “White Coat Hypertension” a phenomenon where patients get anxious being in a clinical setting resulting in an abnormally high blood pressure reading, DHTs can help to mitigate these effects.

  • FDA’s Sentinel System collects RWD and RWE from study collaborators such as Centers for Medicare & Medicaid Services, Veterans Health Administration, and even from foreign sources such as the UK National Health Services.

• The challenges with collecting and evaluating RWD and RWE to assess drug safety and efficacy:

  • Dr. Marston refers to a fragmentation between clinical practices and research clinics in the post-marketing phase of drug development that makes it difficult to acquire RWD.

  • RWD is not collected from controlled clinical research and often EHRs and MCDs do not include the information needed for specific study queries.

  • In multi-site trials, data formatting can differ from each site.

  • Research protocols are often not covered by routine clinical care.

  • In order to support the innovation of trials that rely more on RWD and RWE, the FDA recently launched the CDER Center for Clinical Trial Innovation (C3TI) (For more information on C3TI see our previous blog entry on the topic) and released a new guidance: Conducting Clinical Trials With Decentralized Elements (this guidance will be covered in one of our future blog entries so keep an eye out).

• Resources the public can access for further understanding RWD and RWE:

  • Dr. Marston references multiple Guidances for Industry:

    • Real-World Data: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products (see our blog on this guidance here).

    • Data Standards for Drug and Biological Product Submissions Containing Real-World Data (see our blog on this guidance here).

    • Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices (see our blog on this topic here).

The entire podcast is 21 minutes and is available to listen to on Apple Podcasts, YouTube, and Spotify, you can also listen or read the entire transcript on the FDA website here. To listen to previous episodes or new ones, please click here.

The topic of RWD and RWE is one that is brought up regularly in the Clinical Pathways blog. A list of all of our previous entries on this topic as well as others that are discussed in this podcast (ex. facilitating understanding in informed consent), including those mentioned above, will be listed below. For those interested in staying up to date with FDA activity such as guidance for industry releases, workshops, and programs, consider subscribing to the Clinical Pathways blog and newsletter by clicking here, scrolling down to “Subscribe to Blogs and Newsletters”, filling in the required fields, and clicking “Sign Up”.

-The Clinical Pathways Team

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Previous Blogs:

• FDA Framework for Use of Real-World Evidence

• Real World Evidence Aids Medical Device Approval

• RWE Using Electronic Health Record and Medical Claims Data Draft Guidance

• RWE Data Standards for Drugs and Biologics Draft Guidance

• Another RWE Draft Guidance: Assessing Registries to Support Regulatory Decision-Making for Drug and Biological Products

• Draft Guidance: Using RWE to Support Clinical Trials and Observational Studies

• Real World Evidence in Document Submissions Guidance

• Advancing Real-World Evidence (RWE) Program FAQ

• Improving Clinical Trial Patient’s Comprehension of Informed Consent Forms

• FDA & NIH Work to Clarify Clinical Research Terminology

• New Guidance for Industry on The Use of Real-World Evidence and Electronic Health Records in Regulatory Decision-Making