TriNetX, driven by its vision of a connected world where data and intelligence power improved human health, has officially published the results from its latest CSO Perspectives: Research Impact report i.e., Unlocking Rare Disease Insights.
Going by the available details, this particular study was designed to generate greater understanding in the context of how life sciences organizations are using real-world data (RWD) to drive innovation across the rare disease research space.
More on the same would reveal how, taking inspiration from real-world studies conducted on the back of TriNetX LIVE™ platform, the report in question really goes the distance to reveal the ways in which advanced data science is helping the healthcare industry to overcome chronic evidence gaps and deliver actionable insights across the product lifecycle, right from early feasibility to regulatory submission and market access.
“Rare diseases are chronically underserved but collectively common,” said Jeffrey Brown, PhD, Chief Scientific Officer at TriNetX. “Our data show that when applied thoughtfully, real-world evidence can do more than fill gaps. It can guide entirely new approaches to treatment and care.”
Talk about the whole report on a slightly deeper level, we begin from how it discovered that, RWD is no longer a marginal but a central component. This translates to how regulatory bodies across the board are now increasingly embracing RWD to inform decisions. The stated factor is especially evident in areas where traditional rare disease clinical trials fall short due to small, heterogeneous populations.
Next up, the report found electronic health records (EHRs) to power expanded clinical trial possibilities. You see, thanks to EHR-derived data, there is potential to also access longitudinal, real-time insights that can help pharma teams identify eligible patients, test protocols, and at the same time, streamline trial site identification.
Another detail worth a mention relates to how, as far as Paroxysmal Nocturnal Hemoglobinuria (PNH) is concerned, RWD revealed persistent disease activity in patients treated with C5 inhibitors, highlighting unmet needs and supporting the rationale for next-generation therapies like C3 inhibitors.Â
On the other hand, when it came down to Eosinophilic Granulomatosis with Polyangiitis (EGPA) and Hypereosinophilic Syndrome (HES), RWD was able to support some of the most comprehensive real-world analyses till date. By doing so, it offered greater clarity where traditional studies could not.
To understand the significance of such a study, we must take into account a separate report, where it was claimed that 263-446 million persons are affected globally at any point in time by 7,000 distinct conditions, turning rare disease research more of an essential.
Founded in, TriNetX’s rise up the ranks stems from providing the world’s broadest federated network of real-world data which accelerates innovation across the healthcare ecosystem.Â
The company’s self-service, HIPAA-, GDPR-, and LGPD-compliant platform of federated de-identified and anonymous electronic health record datasets is presently focused on improving clinical trial protocol design, streamlining trial operations, fine-tuning safety signals, and enhancing real-world evidence generation.Â
TriNetX’s excellence in what it does can also be understood once you consider it has, thus far, provided support for more than 250 million patients, empowered 200+ healthcare organizations to provide continuous and comprehensive up-to-the-month data, and made 70 billion patient specific clinical observations available for download.Â
“By combining deep clinical data with scalable analytics, we can illuminate what has long been invisible in rare disease research,” said K. Arnold Chan, MD, ScD, SVP, Office of the Chief Scientific Officer at TriNetX. “These studies demonstrate how real-world data can capture the complexity of care and reveal patterns that traditional research often misses.”
