As the clinical research landscape progresses, so do the expectations of regulators. The U.S. Food and Drug Administration (FDA) has made it clear: real-world data (RWD) has the potential to enhance the efficiency and relevance of clinical studies, but only if it’s collected and used responsibly.
While the FDA has released several guidance documents on the use of RWD across a range of settings, for Sponsors involved in access programs like post-trial access (PTA) or expanded access (EA), it’s not always clear how this guidance applies in practice. So, the question remains: how should these standards be interpreted through the lens of access?
In this article, we link and break down three key FDA guidance documents and what they mean in practical terms for sponsors collecting and applying RWD in the context of access.
1) Framework for FDA’s Real-World Evidence Program (2018):
This foundational document sets the stage for how the FDA views RWD and its potential role in regulatory decision-making. It introduces the idea of assessing whether data is “fit for purpose,” highlights the importance of study design, and explores early-use cases, such as supporting effectiveness data for drug approvals. If you’re new to the space, this FDA framework is a good place to start.
2) Considerations for the Use of RWD and RWE to Support Regulatory Decision-Making for Drug and Biological Products (2013):
This is one of the FDA’s most practical resources for sponsors designing real-world studies that can withstand regulatory scrutiny. It outlines exactly what the agency wants to see: a clearly defined study objective, a pre-specified protocol, and reliable, clinically meaningful data.
It also calls out common missteps, like overlooking confounding factors or failing to properly document where and how the data was collected. It reinforces that real-world evidence isn’t just about the data itself, but also how it’s sourced, cleaned, and analyzed. If you plan to take RWD into a regulatory conversation, it’s worth exploring the full document.
3) Clinical Trials Using Real-World Data (Draft Guidance 2023):
This draft guidance is aimed at Sponsors designing and running clinical trials that rely on RWD, like electronic health records- as a key data source. It’s particularly relevant for those exploring pragmatic or hybrid trial designs conducted in routine healthcare settings.
The document covers how to ensure consistent, verifiable data capture; what documentation is needed to maintain transparency; and how to manage trial elements like site selection, consent, and monitoring. If you’re looking to move beyond traditional trial models and into real-world infrastructure, this guidance is a must-read.
How Can Sponsors Apply This Guidance in the Context of Access Programs?
If you’re already managing early access or follow-up programs, you’re in a prime position to collect RWD that reflects how your product works in practice. These programs often involve more diverse patient populations and real-world treatment conditions, making the insights even more valuable when done right.
But don’t assume any RWD will do: The data needs to be relevant, reliable, and clearly tied to your study’s objective. Such data can support health technology assessments (HTAs) and pricing negotiations by demonstrating the drug’s value in real-world use.
Having an all-hands approach to determining what data is most important to collect and analyze is key. Different stakeholders in your organization may have input that is necessary to determine whether a particular data point is relevant to the objectives of the program. Having these discussions early in the program development process is essential.
Here are some key considerations when collecting RWD in your access program:
- Design with intent: A solid protocol and thoughtful methodology are essential if you want the data to support a regulatory decision.
- Choose an effective collection tool: Data collection should be conducted using an EDC tool that is CFR 21 Part 11 compliant and ensures the security of the patient’s data. WEP utilizes REDCap Cloud, which is a compliant, cost-effective, data collection tool that is custom designed for the collection and management of access program data. This EDC is designed to extract data in the exact format required for FDA submission.
- Start the conversation early: The FDA’s Advancing RWE Program gives sponsors a chance to get early feedback- don’t wait until your data is already collected.
- Ensure a diverse country mix: When diversifying your country mix, make sure to account for country- specific regulations and guidance. Access programs can have different regulations per country, so it’s important to understand how those regulatory differences can influence the collection of data and what types of data/assessments are appropriate.
- Understand access program intricacies: While there are many similarities between data collection in a Clinical Trial setting and an Access setting, there are also some very important differences. These differences require close monitoring and understanding of the ever-changing landscape of RWD in the context of access programs. Reading and understanding the guidance that is put out by the FDA is a great start but when pulling together a team, choose a partner (Like WEP Clinical) that has many years of expertise specific to access programs.
Conclusion
FDA guidance sets the standard, but applying it in the world of access programs takes more than just reading the rules. At WEP Clinical, we know how to turn regulatory expectations into practical, workable solutions – helping sponsors collect RWD that’s not only compliant, but genuinely valuable for decision-making and patient care.
