Pharmaceutical regulators are open to new kinds of data to support new drug applications.  However, that doesn't mean that applicants can ignore the quality of that data.

That's the topline message of a recent report from Cardinal Health that highlights the challenging journey from RWE to U.S. Food and Drug Administration (“FDA”) approval. In recent years, the FDA has embraced the potential of RWE as a supplementary tool to traditional clinical trial data. RWE is derived from the analysis of real-world data and has garnered attention for its potential to expedite drug development while reducing costs.

The FDA issued its proclamation of a commitment to RWE in 2018 and use of this pathway has surged. The number of RWE studies increased by 162 percent between 2016 and 2021. The appeal of RWE lies in its capacity to fill knowledge gaps left by clinical trials, enabling companies to make more informed decisions throughout drug development.

How could there be gaps in clinical trial information?  Don't the trials involve thousands of patients in most cases?

Think about it. The clinical trials are typically supervised by experienced physicians.  Patients who participate are motivated.  The clinicians and their staff monitor the patients to try to ensure they follow the clinical protocol.  Most things are highly scripted.

But, outside the clinical trials – “life happens”.  And they happen to hundreds of thousands to millions of patients.  There's little or no direct clinical supervision.  And the commitment of patient may vary widely.  So, RWE takes account of all those diverse factors based on what really happens.

For example, the drug Prograf achieved FDA approval for a new indication in 2021 based on an observational study supported by RWE. This success story demonstrates that when RWD is robust and fit-for-purpose.  It can pave the way for groundbreaking advancements in drug development.

Yet, as the report from Cardinal Health underscores, the quality of RWD often doesn't receive the attention it deserves. In the industry's initial excitement over RWE, data reliability and relevance frequently took a backseat, leading to many RWE studies falling short of the FDA's standards. Though awareness about data quality has improved, the report suggests that early issues persist.

To enhance the quality of RWE submissions and increase the likelihood of FDA approval, the report offers practical recommendations:

1. Engage with the FDA Early: Sponsors should collaborate with the FDA during the protocol development phase to ensure alignment on study objectives and methodology. Early engagement enables strategic planning and helps prevent the need for course correction later.
2. Ask Unasked Questions: Exploring uncharted territory within RWE studies can yield creative insights and strengthen submissions. Thinking beyond the predefined roadmap can lead to more comprehensive and impactful outcomes.
3. Match Data to Questions: Align the research question with the appropriate data source, rather than retrofitting available data to fit the question. This approach ensures accuracy and relevance.
4. Diversify Data Streams: Combining diverse RWE inputs like EMR, claims, and chart reviews can provide a holistic perspective. Building multiple complementary studies enhances the case presented to regulatory agencies.

While the path to utilizing RWE effectively is challenging, it remains a pivotal tool in modern-day drug approval processes.

FDA itself has announced that it is conducting an Advancing Real-World Evidence Program that will meet with selected sponsors to discuss how best to incorporate RWE into its review processes.  FDA's stated goal is to “improve the quality and acceptability of RWE-based approaches.”  In the meantime, by approaching RWE strategically and partnering with knowledgeable consultants, sponsors can optimize their RWE submissions to meet the FDA's standards and usher in a new era of pharmaceutical innovation.

In the complex landscape of pharmaceutical development and regulatory approvals, navigating the intricacies of RWE can be a daunting task. This is where Crowley Law steps in as your trusted partner. With our deep experience in pharmaceutical law and regulatory affairs, we help guide clients through the challenges of FDA submissions. Whether you're seeking to optimize your RWE strategy, engage with the FDA early on, or align your research questions with the right data sources, Crowley Law and its network of experts in the industry are here to empower you throughout the journey.

For personalized assistance and strategic consultation, reach out to us today at 844-256-5891.