By Richard Gliklich, MD, Founder, OM1
A Historical Perspective on Registries
In 2005, I had the honor of leading the first Agency for Healthcare Research and Quality (AHRQ) effort to develop a handbook on patient registries. The impetus for the handbook, which we published in 2007, was the growing need to demonstrate real-world safety, effectiveness, and value of newly approved treatments that had only been tested through randomized clinical trials. “Registries for Evaluating Patient Outcomes: A User’s Guide” has gone through four successive editions, with the most recent led by our team at OM1 released in September 2020. Used in the U.S. and internationally by regulators, payers, manufacturers, academicians, healthcare and patient organizations, the comprehensive and case-study filled guide has been cited thousands of times in the medical literature and regulatory guidance documents.
The goal of collecting real-world data to produce evidence of safety, effectiveness, and value has only grown since that time as the extent of differences between clinical trial participants and real-world patients has become better understood. The use of real-world evidence has become a cornerstone in drug and device development as leading organizations develop integrated evidence plans with their molecules and products. But not all real-world data and its analytic product and evidence are created equal.
Why Registries are Regaining Ground
Over the last decade, as manufacturers struggled to identify where to spend initially flush and now increasingly limited dollars on real-world data, patient registries are once again rising in importance. Existing data sources have tremendous value for many use cases because they are broad, representative, large, and can be linked with many other de-identified data sources for a range of purposes from drug development through commercialization.
Where existing data sources fall short are when specific outcomes are inconsistently captured in real-world practices, such as standardized clinical outcomes assessments and patient reported outcome measures; when data transparency, traceability and ultimately auditability are necessary for regulatory submissions; when the disease is rare; and, when long-term follow-up with limited attrition is required and so forth. While it is obvious in certain circumstances that a registry approach needs to be used when there is a definitive post-marketing commitment or to establish an external control arm, registries are again rising in use because they are proving to be a more cost-effective and versatile means to accomplish all the goals of an integrated evidence plan. This is especially true with modern data automation technologies that are dramatically lowering the costs and site burden associated with these programs, as well as pragmatic trials.
Today, registries serve many purposes, from understanding safety and effectiveness to measuring quality and value. They also serve as a source of ‘fit for purpose’ real-world data for post-marketing commitments, external control arms, and for embedding pragmatic trials. Where registries have evolved most is in their ability to leverage the collection of passive data, meaning data that already exists in source document systems. This dramatically reduces the cost of running these registries, and, equally important, drives down the burden at the site and even patient level.
The Strategic Value and Explosive Growth of Registries
At the same time, sponsors find that these efforts are not only cost-effective over the long term but have substantial side benefits beyond evidence generation. These include creating a community among key opinion leaders and patient groups, putting sponsors in thought leadership positions within their respective markets, and providing unique insights into the management of conditions and the impact of different products. These insights are also beginning to appear as personalized medicine tools that can also be tested within the context of the registry. The latter, driven by AI models such as OM1 PhenOM®, will likely represent the largest single benefit of registry participation to patients and the centers that care for them.
As shown in Figure 1 below, 10 years ago, the number of new patient registries was contracting. Today, they are rapidly expanding. The Registry of Patient Registries (RoPR), which our team developed with funding from AHRQ to catalogue registries as unique study types and which was ultimately embedded within Clinicaltrials.gov (CT.gov), provides useful data. In 2017, 886 registries were first posted on CT.gov, but by 2024, that number had risen to 2068. More impressively, from 2023 to 2024, the number grew an amazing 39%, from 1493 to 2068. It is heartening to see the value of patient registries once again being recognized as one of the most critical tools for understanding patterns of care and resulting outcomes in the real world.
# of New Registries Posted on CT.gov
Data Source: Clinicaltrials.gov
Conclusion
Patient registries have returned to the forefront as vital platforms not just for data collection but for advancing real-world evidence, enabling stakeholder collaboration, and personalizing patient care. OM1’s Aspen platform is leading this transformation, offering an AI-powered, streamlined, and auditable approach to registry development that reduces the burden for sites while increasing transparency for sponsors. Beyond clinical value, modern registries create opportunities for sponsors to establish thought leadership, engage deeply with key opinion leaders, and uncover actionable insights that shape better healthcare decisions. The data is clear, registries aren’t just growing, they’re reshaping the future of evidence generation.