The Lupus Research Alliance (LRA), which formed and administers the Lupus Accelerating Breakthroughs Consortium (Lupus ABC)—a public-private partnership with the U.S. Food and Drug Administration (FDA)—is leading efforts to ensure that the voices of people living with lupus are central in the development of new therapies.
Lupus ABC unites members of academia, industry, patient advocacy organizations, professional societies, federal agencies, and people living with lupus in a precompetitive environment aimed at overcoming the longstanding challenges in lupus drug development.
“One of Lupus ABC’s key objectives is to ensure that the perspectives of people living with lupus are incorporated in the drug development process. Its governance structure reflects the critical role of the patient voice. The Lupus Voices Council, comprised of people living with lupus and their caregivers, is one of the two governing bodies of Lupus ABC and has a vote in endorsing all [of] Lupus ABC’s initiatives,” Hoang Nguyen, PhD, Assistant Vice President of Research at the LRA, tells BlackDoctor.
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Through the integration of patient-reported outcomes (PROs)—direct reports from patients about how they feel and function—into clinical trials, this initiative ensures that treatments target what truly matters to those living with the disease. The latest research was presented at the 2025 American College of Rheumatology (ACR) Convergence in Chicago.
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Why Now?
Historically, lupus drug approvals have relied primarily on clinician assessments of disease activity. However, patient surveys have revealed that the symptoms most burdensome to daily life—such as fatigue, pain, and cognitive dysfunction (“brain fog”)—often differ from what clinicians measure.
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Recognizing this gap, Lupus ABC convened an in-person meeting on October 16–17, 2024, bringing together people living with lupus, academic experts, industry representatives, and the FDA. The group set out to:
- Assess the current state of PROs used in lupus clinical trials,
- Develop a roadmap to advance their use, and
- Prioritize next steps for integrating PROs into the design and evaluation of lupus studies.
This meeting marked a pivotal step toward aligning research priorities with patients’ lived experiences.
Identifying What Matters Most
Researchers determine which symptoms to measure by listening directly to patients. Through multiple large-scale surveys and studies, people with lupus have consistently identified the top areas affecting their quality of life:
- Fatigue
- Pain
- Mental health
- Physical function
- Cognition and “brain fog”
These insights are guiding efforts to ensure future trials capture meaningful improvements in these domains.
Measuring “Brain Fog” in Lupus
While there is currently no lupus-specific tool for measuring “brain fog,” researchers use a range of approaches to assess cognitive function. These include:
- Self-reported questionnaires capturing patients’ experiences with concentration, memory, and mental clarity.
- Neuropsychological tests assessing learning, reasoning, reading, and processing speed.
- Advanced brain imaging, such as functional magnetic resonance imaging (fMRI), which has recently shown promise in detecting reduced attention, working memory, and emotional recognition among lupus patients with cognitive symptoms.
Ensuring Surveys Are Feasible for Patients
A major discussion point during the PRO meeting centered on minimizing patient burden. Participants agreed that:
- 15 minutes was an acceptable duration for monthly questionnaires,
- 1–2 minutes was ideal for daily check-ins.
To make participation easier, the group recommended digital tools, mobile access, and training resources to help patients complete surveys comfortably—even during lupus flares or fatigue.
Challenges in Regulatory Acceptance
While the FDA recognizes the importance of PROs and has published four Patient-Focused Drug Development (PFDD) Guidance documents, two of these—those most relevant to selecting and incorporating clinical outcome assessments—remain in draft form.
These guidance documents outline best practices for:
- Collecting accurate patient experience data,
- Balancing risks and benefits important to patients,
- Selecting or developing meaningful clinical outcome assessments, and
- Integrating these measures into regulatory decision-making.
Three of the four Guidances are in the final forms, while the remaining one is still in drafts.
“Guidance 1 describes methods to collect accurate patient experience data. Guidance 2 discusses risks and benefits and the approaches to identifying what is most important to patients. Guidance 3 gives guidelines on how to select, modify, or develop clinical outcome assessments (COAs) to measure outcomes important to patients. Guidance 4 discusses how to incorporate the assessments into endpoints for regulatory decision making, including interpreting the meaningfulness of the benefits,” Dr. Nguyen explains.
Patient Involvement in Trial Design
Meeting participants reached a strong consensus that people with lupus should be actively involved early in the trial design process—specifically in identifying which symptom domains are most relevant to measure. Their insights will help researchers select the most meaningful outcomes and develop suitable assessment tools.
Sharing Data Back with Participants
While PRO results are typically included in published clinical trial findings, these data are not always shared directly with participants. Many people with lupus expressed a desire for greater transparency, asking that PRO results be communicated to them and their physicians.
However, researchers noted that sharing interim PRO data could risk compromising trial integrity. As a compromise, the group agreed it is essential to explain at the start of each trial how and why PRO data will be collected, used, and eventually shared.
A New Era of Patient Partnership
By prioritizing patient-reported outcomes, Lupus ABC and the Lupus Research Alliance are leading a transformative shift in lupus research—ensuring that future therapies reflect what truly matters to patients.
As Dr. Nguyen notes, this initiative represents more than just improved measurement; it signifies a commitment to partnership, empowering patients as co-creators in the journey toward better lupus treatments.
