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E2 – Real-World Evidence: Is It a Game Changer for the FDA?

E3 – Real-World Evidence: Is It a Game Changer for the FDA?

Transcription:

Nancy: Hello and welcome to our first podcast in a three-part series entitled “Real-World Evidence: Is it a Game Changer for the FDA?” I’m Dr. Nancy Beck, a director in Avalere Health’s Regulatory Science and Commercial Strategy practice. I’m joined today by my colleague Dr. Brenda Huneycutt, a vice president in the practice, and we will be your moderators. Thank you for joining, Brenda.

Brenda: Thank you, Nancy. Happy to be here discussing real-world evidence, also referred to as RWE, which is the term we’ll be using throughout our conversation. I’m excited to be launching this podcast series with you because I see RWE as a disruptive force in healthcare.

Nancy: There’s particular interest right now in how RWE can improve the development and approval of drugs, biologics, and devices, given the legislative mandates in 21^st Century Cures and FDARA, the current user fee law, requires FDA to incorporate RWE into its decision-making processes. RWE represents a new and exciting approach in assessing the safety and efficacy of medical products, prompting us to dive deeper into the conversation and share our observations and insights on this topic.

Brenda: The influx of questions we’ve been receiving shows there is a lot of curiosity and a lot of confusion about what RWE is, it’s utility, and how it might be used in the regulatory context—for FDA decisions regarding product approval, labeling changes, and post-market safety monitoring.

Nancy: Our goal is to cover three key topics over the podcast series:

1. The promise of RWE for improving the efficiency of product development and approval.
2. The drivers of RWE and the FDA’s evolving approach to RWE.
3. The implications for the industry, considering both the challenges as well as the opportunities, using cases to illustrate ways you can capitalize on RWE.

Brenda: Let me start by defining RWE. RWE is evidence generated from data collected outside of a clinical trial, such as insurance claims data, health records, registries, and patient-reported outcomes. RWE has long been used to inform decision-making for healthcare delivery and reimbursement, and now the FDA is determining how RWE can be more broadly applied in the regulatory context.

Nancy: Brenda, can you give me an example of how this RWE could be applied?

Brenda: First and foremost, the collection of data generated during the normal course of care represents a far more diverse group of patients than those who participated in clinical trials on which FDA generally bases its decisions. When carefully collected and analyzed, the greater representativeness provided by RWE can provide a more accurate picture of a product’s safety and efficacy.

Using a variety of data types and sources can provide insight that you can’t get from clinical trials alone. It adds layers and additional detail to the picture presented by clinical trial data.

Nancy: I also think the move toward RWE will help advance patient-focused drug development. The FDA is opening the door further to consideration of patient generated data to inform its decisions, like patient preference data and patient reported outcomes, which can be more meaningful to patients than standard clinical trial endpoints.

Brenda: A takeaway here is that RWE creates a lot of opportunities to improve the efficiency of drug development and speed access to patients. It’s also a tremendous opportunity to leverage existing data for multiple audiences and apply RWE more broadly across the product life-cycle. FDA’s willingness to consider RWE could allow companies to develop coordinated evidence generation strategies, where data could be leveraged to meet the needs of regulators, payers, and other healthcare stakeholders. Ultimately, this could help streamline drug approval, patient access, and reimbursement processes, making it a significant trend for multiple groups across your company to watch.

Nancy: Because RWE is new territory for the FDA, we’re getting a lot of questions from our pharma and biotech clients about the implications to their portfolio. What they should be doing now to prepare for changes in FDA’s decision-making paradigm, and how they can engage patients in the development of RWE to make the patient voice a more prominent part of regulatory decisions.

Brenda: That and much more is coming your way. Before we sign off, one thing with which I want to leave our audience is that to propel generation and use of RWE in this new way is to foster the cross-sector, cross-disciplinary conversations that are needed.

Nancy: That’s a great point. The more people who are conversational in RWE, the better the likelihood of successfully overcoming challenges and integrating RWE into regulatory decision-making.

Brenda: So, Nancy, is RWE a game changer?

Nancy: Tune in to see if we uncover the answer in our next episode when we examine the factors driving the use of RWE, the steps the FDA is taking, and where those are likely to lead.

Thanks to everyone for listening.