Real-World Evidence: Shaping Obesity Treatment Beyond Clinical Trials

Real-world evidence (RWE) is helping researchers better understand how obesity medicines perform outside clinical trials. In observational data, many patients discontinue treatment, and some regain lost weight after stopping therapy.

For people living with obesity and related conditions, the pace of innovation is rapidly accelerating. But so is the need to understand how the latest obesity medicines perform in clinical practice outside of the highly controlled clinical trial setting. RWE is helping to fill that gap, offering insights into how obesity treatments are used by patients day-to-day, and what that means for their health over time.

Real-world evidence refers to clinical insights generated from data collected outside traditional randomized trials, including claims data, electronic medical records and pharmacy records.

Amgen's Center for Observational Research (CfOR) has spent 15 years building the capabilities, data and partnerships needed to generate high-quality real-world evidence across the lifecycle of medicines.

In this Q&A, Paul Dluzniewski, Amgen's executive director of Observational Research, answers important questions about how real-world data is informing obesity research today-and where it can go next.

Q: How is real-world evidence reshaping the way we understand obesity treatment ?

A: Clinical trials are essential because they establish safety and efficacy in well-defined populations under controlled conditions. However, they don't tell the whole story, specifically, what happens when patients take obesity medications in everyday life.

In obesity treatment, we're seeing a lot of new data in both clinical trials and real-world studies, which is exciting. But one thing that stands out across these real-world studies: A lot of patients-some estimates show upwards of 50 to 60%-don't stay on these therapies long-term.

We also see that many people who discontinue treatment regain the weight they have lost. In fact, our research suggests that those who experience the greatest weight loss may also regain the most weight after stopping therapy. These treatment adherence patterns reinforce the continued unmet need in obesity care.

The impact extends beyond weight. We also see changes in cardiometabolic markers tied to long-term health outcomes, including HbA1c, systolic blood pressure and triglycerides.

Q: What is Amgen doing differently to generate and apply real-world evidence in obesity and related conditions?

A: Amgen has spent years building strong data and research capabilities so we can study what's happening in real-world settings at scale, primarily in the U.S., but also globally. And we have a history of successfully using real-world data to evaluate the benefit-risk profiles of our marketed products.

In our obesity research, we continue to use large healthcare datasets, including electronic health records and medical and pharmacy claims with extensive information on patient demographics, clinical measures such as weight and lab values, and major clinical events, to evaluate current management of obesity and obesity-related conditions. This real-world data help us better understand patient characteristics, treatment patterns and how therapies are being used over time.

We're also working to capture the entire patient journey by linking together different datasets using approaches like tokenization, which preserve patient privacy and can create a much more complete view of the patient experience.

At the same time, obesity care is evolving quickly. More patients are receiving telehealth obesity treatment or paying out of pocket for medications-experiences that are not always captured in traditional healthcare datasets. Researchers need data sources that reflect how patients actually receive treatment in everyday life.

To better understand those changes, we're expanding our relationships to include telehealth providers and retail pharmacy organizations. The goal is to better understand how patients are accessing treatment and moving through their care journey. Through this, we remain focused on quality-using fit-for-purpose data and rigorous research methods to generate high-quality RWE.

Q: With the obesity treatment field evolving so quickly, how do you ensure Amgen's real-world evidence stays timely and actionable?

A: There's a constant flow of new obesity data, so our approach is to continuously evaluate new research while also leveraging our internal capabilities to generate insights quickly.

We closely review new studies to understand the data sources, patient populations and study designs. We also have multiple disease- and therapy-specific cohorts that allow us to rapidly analyze treatment adherence patterns and outcomes.

For example, we track groups of patients starting therapy and look at patterns like treatment persistence or how their dosing changes over time, while also analyzing associations between available medicines and published clinical outcomes.

One current finding that is consistent both internally and externally is that many patients either don't start therapy, or don't stay on it. That gap between what clinical benefit these therapies may achieve and what happens in real-world applications is significant.

Q: Looking ahead, how do you see real-world evidence shaping the future of obesity care and treatment decisions?

A: We really are just getting started.

As more long-term longitudinal real-world data becomes available, we'll gain a better understanding of treatment patterns, unmet needs and long-term outcomes, including the experience of patients who may not have been well-studied in the clinical trials. We'll also better understand the impact of patients not remaining on therapy, and build the foundation for conducting comparative effectiveness studies. The goal is to inform healthcare decision-making given the growing number of potential obesity therapies we anticipate will be available to patients over the next five to ten years.

Ultimately, the goal is to generate insights that can help improve care and better support patients living with obesity and obesity-related conditions.

Q: How can real-world evidence evolve to better address the challenges patients are facing?

A: We need to better understand the full patient experience. For example, RWE can show us when patients stop treatment, but it doesn't necessarily tell us why. Access, affordability, tolerability and patient preferences all play a role.

The goal is to turn data into insights that actually help improve care-by making it easier for patients to start, stay on, and benefit from treatment. We also need to be thoughtful about the outcomes we prioritize, ensuring they align with the outcomes that matter to patients and their healthcare providers.

What This Means for Obesity Care

Real-world evidence helps scientists at Amgen understand how treatments work outside of clinical trials, in the daily lives of patients. In a complex chronic disease like obesity, where long-term management and patient behavior play a central role, insights are essential because real-world evidence and clinical trials work together to give a more complete picture of obesity care. Together, they help researchers better understand treatment patterns, patient needs and long-term outcomes. By continuing to invest in data, methods and partnerships, Amgen is working to better understand the realities patients face and find potential solutions that meet their needs.