Regulators Offer Fresh Guidance on Using Real-World Data to Support Regulatory Decisions

Becky Wood in Washington, D.C., Emily Marden in Palo Alto, and Josefine Sommer in Brussels look at new FDA guidance on the use of real-world data to support new indications and post-approval study requirements.

Real-world data (RWD), data generated by patients and doctors outside of clinical trials, are increasingly being used by the U.S. Food and Drug Administration (FDA) and life sciences companies to support a range of regulatory decisions.

In the U.S., the FDA has had a congressional mandate to advance the regulation of RWD for some years, since the 21st Century Cures Act 2016 set up a framework for how the agency would make more use of RWD in regulatory decisions.

The trend toward using RWD accelerated during the COVID-19 pandemic, given the sudden obstacles to conducting on-site trials. The urgent need for tests, treatments, and vaccines created by the pandemic also encouraged flexible approaches to clinical studies and data.

In this context, the FDA has issued new draft guidance on RWD, on which comments from interested stakeholders are invited:

• Data Standards for Drugs and Biological Product Submissions Containing Real-World Data
• Use of Real-World Data and Real-World Evidence to Support Effectiveness of New Animal Drugs
• Real-World Data: Assessing Electronic Health Records and Medical Claims Data to Support Regulatory Decision-Making for Drug and Biological Products

These documents refine the FDA’s framework for incorporating RWD into regulatory decision-making, guide the process of sourcing RWD from electronic health records and medical claims, and address the use of RWD in regulatory filings for the approval of therapeutic human and animal drugs. The new guidance also sets out the FDA’s thinking about study design elements and the use of RWD to satisfy post-approval study requirements. RWD is also becoming an increasingly important part of post-market surveillance to help track how the product is actually performing in the field.

European regulators Heads of Medicines Agencies and European Medicines Agency (EMA) have also in recent years launched several initiatives aimed at establishing the place of real-world evidence in regulatory decision-making. A Big Data Taskforce was set up in 2017 (since superseded by a Big Data Steering Committee) and has made recommendations, which include the promotion of data discoverability through the identification of metadata.

There are also several pilot projects currently being undertaken by the EMA’s scientific committees and working parties, including a pilot looking at how rapid analytics of RWD might support regulatory decision-making. Further EMA pilot studies that are slated to begin running in 2022 include providing the first catalogue of RWD sources.

This post is as of the posting date stated above. Sidley Austin LLP assumes no duty to update this post or post about any subsequent developments having a bearing on this post.