At Truveta, our mission is Saving Lives with Data, and we are thinking about the HHS leadership mission of making American’s healthier, and how to help the talented scientists of HHS do more with less. We’d like to contribute to this important mission – starting with reflecting on the use of representative real-world health data for advancing public health.
First of all – congrats to Jay Bhattacharya, MD, PhD and Martin Makary, MD, MPH on their approvals to lead the NIH and FDA, respectively, and join Secretary Robert F. Kennedy Jr’s leadership team!
For decades, randomized controlled trials (RCTs) and registries have been heralded as the gold standard for clinical research. These approaches are meant to advance new therapies, determine treatment protocols, and define best practices for care. But the reality? They are slow, expensive, and often fail to reflect the diversity and uniqueness of America’s population.
A few examples:
- NIH spends up to $1.44 billion per approval, according to a cross-sectional study of 356 drugs approved by the US Food and Drug Administration from 2010 to 2019.
- Clinical trials carry the highest burden of cost for bringing new drugs to market – an estimated $117.4 million per drug. They can also suffer from small or unrepresentative samples.
- Registries can cost anywhere from $500,000 to $5 million+ and take significant time to set up as they require sophisticated data collection systems. They can also suffer from missing or incomplete data, and for rare diseases or specific patient populations, they may have small or unrepresentative samples.
As we consider how many lives are impacted by cancer, autoimmune disease, neurological conditions, and more, the cost to humanity is real. The good news? There is a better way. Delivering representative real-world data to researchers can advance innovation and help ensure a healthier America.
Imagine an America where timely, representative, and cost-effective insights drive faster innovation and inform healthcare decisions from yesterday’s care, today. This is possible using real-world evidence (RWE), an approach increasingly embraced by agencies like the FDA and CMS. RWE can deliver larger, more representative populations that reflect all of us, enabling faster innovation and greater precision in care. As a faster, more representative alternative, RWE is better suited for modern healthcare challenges.
Let’s look at a few examples.
Validating results in weeks, not years
A groundbreaking application of real-world data comes from a study on pulmonary hypertension associated with interstitial lung disease (PH-ILD). The INCREASE trial, a phase 3 placebo-controlled study, evaluated inhaled treprostinil for this condition. The trial included 326 patients and took years to complete. Using real-world data, Truveta analyzed the outcomes of 699 patients—more than double the trial’s size—and replicated the findings in less than two weeks.
The takeaway? Real-world data can validate findings on a larger, more representative population at a fraction of the time and cost of traditional trials. This efficiency can lead to faster access to life-saving treatments and better health outcomes for all Americans.
Real-world data can generate results faster than clinical trials
At Truveta, we are also showing how real-world data can help reduce the need for costly and time-intensive clinical trials for new clinical applications of therapies that are already approved and in use for other purposes —saving years and millions of dollars.
Take GLP-1 therapies like tirzepatide and semaglutide, which are widely used for weight loss. Truveta’s real-world study, published in JAMA Internal Medicine, compared the two therapies in a cohort of over 8,800 patients—more than 10 times the sample size of the traditional SURMOUNT-5 clinical trial. Truveta’s results were consistent with the trial’s findings: tirzepatide outperformed semaglutide. Even better, Truveta’s study delivered these insights a full year before the SURMOUNT-5 trial was completed (the results of the trial are still awaiting publication and currently in peer review).
Why representative data matters
Real-world data isn’t just about speed and cost. It’s about representation. America is diverse, but traditional clinical trials often fail to reflect that diversity. By leveraging electronic health records (EHRs) from over 120 million patients and closed claims data from more than 200 million patients across the US, Truveta Data ensures that every community—from rural towns to urban centers—is represented in the evidence that shapes healthcare policies.
This representative approach ensures that therapies and treatments work not just for the average patient, but for everyone. It’s the key to closing healthcare gaps and building a healthier America.
The time for real-world evidence is now
We can no longer afford to wait years for clinical trials that only tell part of the story. Every day lost impacts the lives of people we love. Representative real-world data offers a better way forward, one that’s faster, more representative, and more cost-effective. From GLP-1 therapies to lung disease treatments, the examples are clear: real-world evidence doesn’t just complement traditional research; it has the potential to replace it.
By embracing this shift, we can ensure that America’s healthcare system truly serves all its people. It’s time to make Americans healthier, one data-driven decision at a time.
