Lupus Researchers Now Have A Three-Pillar RWD Platform To Support Discovery And Development

A conversation between the Lupus Research Alliance Lupus Nexus Director Devon Kelly and Clinical Leader Executive Editor Abby Proch

Lupus, a complex heterogeneous autoimmune disease, needs more than isolated datasets or sample collections to drive drug discovery and development.

That’s why the Lupus Research Alliance launched Lupus Nexus — a platform that brings together three essential resources: a longitudinal registry, a repository of matched biological samples, and a centralized data exchange platform. By connecting clinical and molecular information with biospecimens — and making those resources accessible to the wider research community — the platform provides a source of longitudinal real-world data (RWD) that can generate real-world evidence (RWE) and help investigators pursue research questions that would otherwise require years of infrastructure-building.

In this Q&A, Devon Kelly explains why the platform’s three pillars are most powerful together, how stakeholder and patient feedback shaped its development, and what researchers and industry sponsors should consider when using —and sustaining— registries and biorepositories.

Clinical Leader: What problem is the Lupus Nexus platform designed to solve?

Devon Kelly: There are many registries out there. There are many biorepositories out there.  But there was no community-wide access to a longitudinally curated dataset that included not only clinical information but also patient-reported outcomes, molecular information, and matched biological samples. Lupus Nexus has three pillars: registry, biorepository, and a data exchange platform. It’s the combination that really makes Lupus Nexus powerful.

What, then, is the Lupus Landmark Study, and how is it connected to the platform?

The Lupus Landmark Study (LLS) is a longitudinal observational study and is the first study seeding the registry and biorepository. The study acts as a core data-gathering engine inside the Lupus Nexus platform and collects data from study participants for up to 5 years. People with lupus have played a key role in the design of this study and will continue to be at the core of this effort through their participation. Their engagement will generate the data needed to drive patient-centric treatments as well as allow participants greater visibility into their own personal health data.

What input led to the platform?

The Lupus Research Alliance asked researchers what would really catalyze lupus research, and they said a centrally-managed registry and biorepository open to all researchers. From there, we established eight working groups, comprised of both industry and academic experts.  These working groups developed various aspects of the program, such as protocol design, core and specialty analyses, patient engagement, technology, and others. We then had individuals living with lupus weigh in on many of these aspects and share what concerns they had and what was missing from their perspective.

Do you recall any feedback that led to a change?

One of the more sensitive aspects of the study is our request for stool samples. The microbiome, as we well know, is central to our immune system, and lupus being an autoimmune disorder, that's a key focus.

Well, our patient advisory group was like, "Ew, gross." We asked them, "This is critical, and this is the reason why it's critical. How can we make it better?"

We sent each of them an at-home collection kit, and we talked through how to use it. Then, we asked for feedback. They suggested putting gloves and clearer instructions in the kit. So, we had the vendor add gloves, and we created a half-page of instructions. To further normalize the idea of stool collection, we also developed a fun, simple video that talks about the importance of the biospecimen, how to use the kit, and how to mail it in.

How did you develop patient trust so that patients would be comfortable with or confident in participating?

We rely on study coordinators to develop that relationship, and a lot of our participants have a very trusted relationship with their clinicians. When a trusted clinician tells them that they are working with the Lupus Research Alliance for this or that study, we generally get more ‘yes’s’ than ‘no’s.’

The Lupus Research Alliance, as a patient advocacy organization, is inherently a trustworthy source.  

We got a lot of feedback from a lot of different people and naturally could not implement every suggestion. But we have success in saying, “We're listening to what you’re telling us. We did our operational assessment. We like this. We're implementing this. We really like that, but we can't for X, Y, and Z reasons." Being transparent and honest closes the loop and also reinforces trust.

How do you hope researchers will use the platform?

The Lupus Nexus’s value is in shortening the time, lessening the cost, and reducing uncertainty from research and development.  We are looking for researchers to gain access to the samples and/or data, to return raw data to us, and to publish their findings.  This is an off-the-shelf offering that will improve R&D decisions, improve clinical trial feasibility, and make clinical development faster and more efficient. It will save researchers money because they're not having to set it up from scratch. 

How can a researcher access the Lupus Nexus?
To gain access to the Lupus Nexus data and/or biospecimens, researcher can fill out a form on our Researcher Landing page. From there, a Lupus Nexus team member will reach out to them to confirm project requirements. While a license agreement is under negotiation with legal teams, we will work with them to complete case selection and prepare for the data/biospecimen transfer.

When should a study sponsor start looking for a registry, and how do they determine the one that best fits their needs?

Many registries are developed to answer a specific research question or have some specific aim. There are broader ones out there, mostly governmental, like the UK Biobank and All of Us. But the smaller registries and biorepositories have a narrower focus and tend to collect less data. It's really important for researchers to understand the data collection and management schema because if they don’t, they will not be able to use the data meaningfully. If you've got inconsistent, incomplete, or just bad data, you're not going to make a discovery based on that.

How can pharma support registries?

The biggest thing that biotech and pharma can do to support registries and biorepositories is to actually use them. For as long as I've been involved in this field, one of the key concerns of every registry and biorepository is sustainability. The smaller institutional ones driven by a principal investigator’s R01 — that one the researcher is just holding the data in an Excel file or the samples in a freezer — don't necessarily need funding to sustain operations.

Seems straightforward, but do they sometimes need convincing?

Biotech and pharma are big organizations, and research funding decisions often involve several internal reviews and priority discussions. Sometimes that means promising ideas take longer to gain traction. At the same time, many companies aren't used to having access to registries and biorepositories because these resources are often restricted.

That’s one of the biggest questions I get- is whether the Lupus Nexus can be accessed by any researcher; yes, this is open to all researchers! If you have a research question, submit a request through the official Lupus Nexus Request Center, and we’ll work through the process. That's the reason why we created it, to support as many researchers as we possibly can.

About The Expert:

Devon Kelly is the director of the Lupus Nexus at the Lupus Research Alliance, the largest nonprofit, private funder of lupus research in the world. She is accountable for the general oversight and strategic direction of the Lupus Nexus. After over 26 years in industry and academia, Kelly transitioned to the nonprofit sector in 2022. Her background includes basic and translational research in a variety of therapeutic areas, as well as clinical operations and building biorepository capabilities with a focus on data management. She received her BS in Zoology from The George Washington University and her M.S. in Forensic Science from the University of New Haven. For any questions, contact LupusNexus@lupusresearch.org.