FDA seeks input on addressing patient-level data challenges for RWD/RWE
The US Food and Drug Administration (FDA) is requesting feedback on possible solutions that would help sponsors more effectively submit patient-level real-world data (RWD) and real-world evidence (RWE) for drug and biological product applications.
“The rapid advancement of technologies and innovative methods (e.g., super intelligence, digital health technologies, and RWD) in drug development presents both opportunities and challenges for regulatory decision-making. Aligned with the U.S. Department of Health and Human Services (HHS) priorities, the FDA is committed to realizing the full potential of fit-for-use RWD to generate RWE that advances the development and safety of regulated products,” FDA wrote in a request for information (RFI) on Wednesday.
Despite the growing number of submissions to the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) that incorporate RWD and RWE for drugs and biological products, challenges remain in providing patient-level data.
According to FDA, sponsors must ensure they can submit patient-level data for any RWD that has been analyzed as part of clinical studies included in a marketing application. However, often, sponsors do not own the RWD used in these studies and do not have the authorization to submit patient-level information to the agency.
The agency said that when RWD is owned and controlled by third-party entities instead of the sponsor, it is essential for sponsors to establish agreements with those entities. These agreements will ensure that relevant patient-level data can be provided to the FDA and that the source data needed to verify the RWD is available for inspection.
The RFI states that regulatory pathways are available for third parties to provide this data to FDA in support of a sponsor's marketing application. For example, FDA may receive the information through a Type V drug master file (DMF).
In these situations, the sponsor should provide FDA with a letter of authorization from the third-party data provider, allowing FDA to reference the data contained in the third party's DMF or pre-IND for the purpose of reviewing the sponsor’s application.
FDA said it is exploring other potential solutions to address these challenges and is seeking input from the public on four questions related to RWD and RWE.
First, FDA said it wants to know about the opportunities and challenges facing the pharmaceutical industry when it comes to the current requirement to submit patient-level data collected from RWD sources for submissions proposing the use of RWE.
The agency also wants to know what solutions can improve industry practices for sharing patient-level RWD with the FDA. The RFI notes that sponsors or third parties “often encounter challenges” when submitting patient-level RWD to the agency as part of a new drug application (NDA), biologics license application (BLA), DMF or pre-IND submission. These challenges stem from the fact that FDA cannot sign a data-use agreement as these may conflict with FDA’s existing legal obligations.
Next, FDA said it wants suggestions for solutions to help it address challenges related to receiving and retaining patient-level data, ensuring technical interoperability, and performing independent reviews of submissions containing RWD.
Lastly, FDA asks how it can collaborate with external parties to address solutions to the challenges related to patient level data submissions that propose the use of RWE.
