Top 10 Rheumatology Clinical Trial Topics at the Upcoming Edition of ACR 2024
The global rheumatology community is gearing up for The American College of Rheumatology (ACR) Convergence 2024 in Washington, DC, the largest rheumatology conference in the Americas and one of the biggest worldwide. This event brings together experts in general and pediatric rheumatology, rare diseases, patient advocacy, and other healthcare professionals. This article highlights key topics and emerging trends that could reshape the future of rheumatology clinical trials and patient care. Let’s dive into the transformative ideas set to be presented at this year’s conference.
1. Efforts on Optimizing Clinical Trials Design and Addressing Diversity, Inclusion, and Racial Disparities in Rheumatology
This session will address the current shortcomings in clinical trials within rheumatology. It aims to explore strategies for improving trial design and participation, particularly focusing on diversity, inclusion, and racial disparities. Panelists will discuss methods to overcome patient accrual challenges by broadening eligibility criteria and increasing access for underrepresented minorities. The session will highlight examples from systemic sclerosis and systemic lupus erythematosus (SLE) trials, emphasizing meaningful endpoint selection, reducing placebo exposure through external arms, utilizing adaptive trial designs to enhance efficiency. This session promises to offer critical insights for advancing clinical trials in rheumatology.
This session will tackle the challenges associated with heterogeneity in clinical trial responses. It will present innovative trial designs aimed at enhancing precision and optimizing outcomes. Key topics include the Multiphase Optimization Strategy (MOST), which facilitates smaller, more efficient trials, and the Sequential Multiple Assignment Randomized Trial (SMART) design, which adapts treatment based on patient response to maximize effectiveness. This session will cover topics such as intervention optimization for varied treatment responses, the application of the MOST methodology, and the use of the SMART design to improve response rates. This session is particularly valuable for those involved in developing or redefining clinical trials, offering practical guidance into addressing treatment variability.
This session will explore cutting-edge technological approaches to improve clinical trials. The highlight will be on the work of the National Institute of Allergy and Infectious Diseases (NIAID)’s Autoimmunity Centers of Excellence (ACE), which integrates basic and clinical research to accelerate the development of therapies for autoimmune diseases. By combining clinical interventions with detailed mechanistic studies, ACE aims to identify patient subsets with shared medication responses and utilize circulating exosomes as disease biomarkers. The session will feature several key presentations:
- Predicting Responses to Therapy in SLE: A discussion on how high-level OMICs data can be used to predict therapeutic responses in SLE.
- Predicting Progression to Rheumatoid Arthritis (RA): Presentation methods for forecasting the progression of RA.
- Monitoring Disease Activity with Exosomes: Exploration of the potential of exosomes as biomarkers for monitoring disease activity.
This presentation will provide a comprehensive overview of how mechanistic studies can enhance the precision and effectiveness of clinical trials in autoimmune diseases.
Lupus disproportionately affects diverse racial and ethnic minority populations, yet there is a significant disparity in their representation in clinical trials. This session will address this gap, highlighting the role of the Lupus Research Alliance’s Lupus Clinical Investigators Network (LuCIN) in advancing lupus treatment across 50 academic medical center sites. Key barriers include socioeconomic and logistical challenges, such as transportation, compensation, and visit time. Building long-term patient relationships and involving trusted messengers, such as primary care providers and community health workers, are essential for promoting equity.
The New York City Lupus Clinical Trials Education Program will present a study on an SLE randomized educational intervention modeled after the Lupus Research Alliance Patient Advocates for Lupus Studies (PALS) program. The study provides outreach to a racially and ethnically diverse group of SLE patients from 3 New York City academic lupus centers. Clinical trial participation is essential for advancing treatment paradigms and should be considered part of clinical care in SLE. However, clinical trials often fail to reflect the racial and ethnic diversity of SLE patients. Historically, minority groups harbored negative attitudes toward clinical research, partly due to a lack of knowledge about clinical trial protections. This patient-centered study suggests that the educational program was effective in increasing patient understanding of SLE clinical trials. While education alone showed a trend toward altering attitudes, self-efficacy, or intentions, patients provided positive feedback about the education they received and demonstrated improved attitudes toward clinical trials after the program.
In SLE clinical trials, selecting primary efficacy measures like the Systemic Lupus Erythematosus Responder Index 4 (SRI4) and the BILAG-based Composite Lupus Assessment (BICLA) is crucial for evaluating treatment efficacy. Findings from the EXPLORER and ATHOS trials underscore key distinctions between these measures. Disease activity in EXPLORER was assessed using BILAG and SELENA-SLEDAI, while ATHOS utilized the updated BILAG-2004 and SLEDAI-2K indices, which demonstrated higher concordance between BICLA and SRI4. BICLA, with its focus on gradual, organ-specific improvements, exhibited greater sensitivity in manifestations such as arthritis, contributing to increased BICLA+/SRI4- discordance. Baseline serological markers, including complement levels and anti-dsDNA antibodies, also influenced the concordance between the 2 indices, with active serology associated with greater alignment. These findings highlight the critical importance of carefully selecting or combining outcome measures based on the trial design and patient population. The use of updated disease activity indices, such as BILAG-2004 and SLEDAI-2k, further enhances the precision of treatment effect assessments in lupus trials.
2. Clinical Trials in Knee Osteoarthritis
Colchicine, a medication approved for gout treatment, has been investigated for its potential benefits in osteoarthritis (OA) due to its anti-inflammatory properties. In the largest double-blind controlled study of colchicine for knee OA, the medication failed to improve knee pain, function, or the size of synovial effusions. This outcome suggests that while colchicine is effective for gout, its benefits do not extend to knee OA, highlighting the need for continued research into effective treatments for this condition.
Ongoing research with lorecivivint demonstrates the urgent need for validated surrogate markers in OA clinical trials. After 3 years of treatment with 3 doses, results suggest that lorecivivint may have the potential to modify the progression of knee OA over the long term. This situation invites comparisons to the field of cardiovascular disease, where surrogate markers—such as managing hypertension or hyperlipidemia—are widely accepted by regulatory bodies due to their established links with major clinical outcomes like heart attacks, strokes, or cardiovascular mortality. These cardiovascular events, much like joint failure in OA, often take years to even decades to develop. To accelerate progress in OA treatment, the identification and validation of reliable surrogate markers for joint failure are essential. Recognizing the significant unmet need in OA, the US Food and Drug Administration (FDA) has designated OA as a serious disease, opening the door for accelerated approvals of therapies aimed at altering the disease’s course.
3. Potential of CAR-T Cell Therapies and T-Cell Engagers for Autoimmune Diseases
This session will explore the emerging role of CAR-T and other cellular therapies in treating systemic autoimmune diseases, particularly in rheumatology. The expert-led discussion will offer attendees a comprehensive understanding of the evolution and potential of these therapies, critically examining the scientific rationale, logistical challenges, and necessary data before widespread clinical adoption. The session will highlight both the promise and limitations of CAR-T therapies, exploring how these approaches could redefine the management of severe autoimmune conditions.
- Historical Overview: Opening with a historical overview of cellular therapies in rheumatic diseases.
- Critical Examination: Discussing whether the promise of CAR-T therapies may be overstated for most patients with rheumatic diseases, identifying the patient populations that may benefit most and the potential barrier to broader application.
- Future Clinical Trials: Addressing critical considerations for future clinical trials, emphasizing the design and data needed to make CAR-T a mainstream treatment option for severe autoimmune rheumatic diseases.
The abstract on the safety and efficacy of CABA-201, a fully human autologous 4-1 BB anti-CD19 CAR T cell therapy, highlighted promising results from the RESET-Myositis™ and RESET-SLE™ clinical trials. Two subjects were dosed: one with non-renal SLE and the other with SRP+ immune-mediated necrotizing myopathy. Both subjects tolerated the treatment well, with no serious adverse events (SAE), cytokine release syndrome (CRS), or immune effector cell-associated neurotoxicity syndrome (ICANS). The administered dose resulted in CAR T cell expansion, peripheral B cell depletion, and biomarker and clinical improvements consistent with previous data.
This abstract will explore the potential of T cell-engaging bispecific antibodies (BsAbs) to target autoreactive 9G4 idiotope B cells in SLE. While CAR-T cell therapies hold promise for SLE, they are limited by scalability and long-term safety concerns. BsAbs offer an alternative by engaging T cells to eliminate autoreactive B cells with precision. The study will showcase the development of a precision T cell-engaging BsAbs therapy that selectively eliminates 9G4 B cells, a major source of disease-relevant autoantibodies in active SLE.
This abstract will detail the development of RO7507062, a CD19-targeting T-cell bispecific antibody (CD19TCB), and the design of its ongoing phase 1 trial in SLE. RO7507062 represents a significant advancement in T cell engager therapies, mediating potent B cell depletion with a minimal cytokine release profile, reducing the risk of CRS. The ongoing phase 1 trial explores both single ascending and fractionated dosing strategies, focusing on safety, tolerability, pharmacokinetics, and pharmacodynamics, including T-cell activation and cytokine release. RO7507062 offers a promising therapeutic approach by leveraging T cell engagers to deliver targeted B-cell depletion in autoimmune disease like SLE.
4. Clinical Trials in Sjögren’s Syndrome
The phase 2 DAHLIAS trial will present pivotal data on nipocalimab, an anti-FcRn monoclonal antibody, in primary Sjögren’s Disease (SjD). This multicenter, randomized, placebo-controlled study enrolled 163 patients with active SjD, assessing the efficacy of nipocalimab at 5 mg/kg and 15 mg/kg doses. The primary endpoint was the change in the Clinical European League Against Rheumatism Sjögren’s Syndrome Disease Activity Index (clinESSDAI) at week 24. Results showed that the 15 mg/kg group achieved a significant improvement versus placebo (-2.65;90% Cl: -4.03, -1.28; p=0.002). Secondary endpoints, including Physician Global Assessment and ESSDAI, also favored the 15 mg/kg dose. Safety was acceptable, with similar adverse event rates across groups. This study confirms the potential of FcRn inhibition in treating SjD and supports further development of nipocalimab in this population.
Recent findings from 3 randomized controlled trials highlight the importance of biomarker-based patient selection for clinical trials in SjD. These studies suggest that different clusters, or “endotypes,” of SjD patients—identified through transcriptomic analysis—respond differently to treatments like hydroxychloroquine, leflunomide, rituximab, and abatacept. For instance, patients with a pronounced interferon signature (Cluster 1) were significantly more likely to respond to therapy compared to placebo, while those in a “healthy-like” cluster (Cluster 2) showed minimal treatment response. This research highlights the critical need for precise biomarker-driven patient stratification in future trials, enabling targeted therapies and improving the chances of achieving meaningful clinical outcomes.
5. Clinical Trials in Systemic Vasculitis
The TAPIR trial provides key insights into the use of low-dose glucocorticoids (prednisone) for maintaining remission in granulomatosis with polyangiitis (GPA), an ANCA-associated vasculitis characterized by small vessel inflammation and granulomatous tissue infiltration. GPA typically affects the paranasal sinuses, organs such as lungs, and kidneys, but it can present with a wide range of symptoms and even mimic cancer or infection. The trial’s findings show that continuing prednisone at 5 mg/day reduces the likelihood of minor disease relapses over 6 months compared to tapering off prednisone completely. This benefit was particularly evident in patients not receiving rituximab, suggesting a more tailored approach for non-rituximab regimens. Importantly, no significant differences were observed in patient-reported outcomes or the risk of major relapses, regardless of prednisone dose. These results have critical implications for future clinical trial design, emphasizing the need for careful tapering strategies and patient selection, particularly for regimens that do not include rituximab.
In the SELECT-GCA phase 3 trial, the efficacy and safety of Upadacitinib (UPA) were evaluated in patients with giant cell arteritis (GCA), with the positive results reaffirming its potential as a new treatment option. The study, conducted across 24 countries, compared UPA (7.5 mg and 15 mg daily) combined with a 26-week glucocorticoid (GC) taper regimen to placebo with a 52-week GC taper. The primary analysis of the first 52-week period demonstrated that UPA15 achieved sustained remission in 46% of patients compared to 29% in the placebo group (P=0.0019). UPA15 also met 9 of 11 secondary endpoints, including a significant reduction in disease flares, improved FACIT-Fatigue scores, and notably lower cumulative GC exposure (1615 mg vs. 2882 mg, P>0.0001). Safety outcomes were generally consistent with the known safety profile of UPA, although numerically higher rates of herpes zoster and lymphopenia were observed in the UPA15 group. Importantly, no major adverse cardiovascular events (MACE) were reported in the UPA groups. These findings, first presented at EULAR 2024, confirm UPA15’s superior efficacy and reduced GC burden, positioning it as a promising new oral therapy for GCA.
6. Clinical Trials in Spondyloarthropathies and Early Rheumatoid Arthritis
Trial A, B and C investigate therapies targeting the interleukin-17 (IL17A and IL-17F) pathway, a key mediator of inflammation in axial spondyloarthritis (axSpA) and psoriatic arthritis (PsA). These trials reflect the growing interest in dual IL-17 inhibition, highlighting its potential to modify disease outcomes in chronic inflammatory conditions.
This trial investigates the efficacy and safety of XKH004, a recombinant anti-human IL-17A/F humanized monoclonal antibody, in patients with active ankylosing spondylitis. The study highlights the potential of dual IL-17 inhibition in modifying disease outcomes in axSpA. The findings from this 24-week phase 2 multicenter trial demonstrate the growing interest in targeting the IL-17 pathway to manage chronic inflammatory conditions.
This phase 3 open-label extension study examines the long-term effects of bimekizumab on spinal radiographic progression in patients with radiographic axSpA. Bimekizumab demonstrated minimal spinal radiographic progression, reinforcing its role as dual IL-17A/F inhibitor. The study suggests that bimekizumab could lead to significant advancements in the treatment of axSpA by offering a new therapeutic option that targets both IL-17A and IL-17F.
This global randomized, double-blind, placebo-controlled phase 2 trial evaluates the efficacy and safety of sonelokimab, a novel IL-17A and IL-17F inhibiting nanobody, in patients with active psoriatic arthritis (PsA). The 24-week results indicate that sonelokimab effectively reduces disease activity, highlighting the therapeutic potential of IL-17A/F inhibition. The emergence of sonelokimab as a competitive alternative to existing therapies like bimekizumab points to a new era in the management of PsA and axSpA.
The TREAT EARLIER trial provides compelling evidence that a 1-year course of methotrexate can prevent the development of RA in ACPA-negative individuals with clinically suspect arthralgia (CSA) at increased risk. This double-blind, placebo-controlled trial enrolled 236 participants and found that methotrexate significantly benefits suitable candidates. However, there are challenges for widespread implementation. The trial’s findings highlight the potential of methotrexate as a preventive strategy for a niche population, though broader considerations on the accessibility and feasibility of MRI as a screening technique are important for its adoption in clinical practice.
This phase 1 double-blind, randomized clinical trial explores the potential of allogeneic mesenchymal stem cells (MSCs) in treating early RA. The results show promising improvements in patient-reported outcomes (PROs) and disease activity over a 28-day period post-infusion. Patients receiving MSCs reported significant improvements in physical function, reduced fatigue, and less pain interference. Disease activity, measured by DAS28-CRP, also showed notable improvement. These findings suggest that MSCs may offer a novel therapeutic option in early RA, supporting the need for larger phase 2 trials to further explore their efficacy. The results from this trial have significant implications for future clinical trial design in RA, particularly in targeting early disease stages with innovative biologic therapies.
7. Clinical Trials in Fibromyalgia
In this confirmatory phase 3 trial, bedtime sublingual cyclobenzaprine (TNX-102 SL) demonstrated significant efficacy in treating fibromyalgia (FM), a complex nociplastic syndrome characterized by widespread pain, nonrestorative sleep, fatigue, and cognitive dysfunction. Conducted across 33 US sites, the trial enrolled 457 patients who were randomized to receive TNX or placebo for 14 weeks. TNX significantly reduced FM pain intensity at week 14 (P=0.00005) and improved key secondary outcomes, including patient global impression of change (PGIC), fibromyalgia impact scores (FIQR), sleep disturbance, fatigue, and sleep quality. Additionally, exploratory endpoints suggested that TNX improved depressive symptoms, cognitive function, and sexual function in females. TNX was generally well tolerated, with transient and mild administration site reactions being the most frequent adverse event. These findings demonstrate TNX’s potential to address core FM symptoms and improve overall patient well-being, positioning it as a promising treatment option for fibromyalgia.
8. Clinical Trials in Idiopathic Inflammatory Myophathies
In this randomized, delayed-start phase 3 clinical trial, baricitinib, a JAK1/2 inhibitor, was evaluated for its efficacy in treating idiopathic inflammatory myopathies (IIM), specifically dermatomyositis (DM) and polymyositis (PM). The trial included 15 adult patients who had active disease despite previous treatment with glucocorticoids or other disease-modifying drugs. Patients were randomized to either receive immediate treatment with baricitinib or a delayed-start regimen after 12 weeks of standard care. At 24 weeks of active treatment, 93% of patients achieved the primary outcome of at least minimal clinical response, as defined by the ACR/EULAR 2016 response criteria for adults PM and DM. Secondary outcomes showed 60% of patients achieved moderate clinical response, and significant improvements were noted in muscle strength and other disease activity measures, such as physician and patient visual analog scores (VAS) and manual muscle testing (MMT-8). There was no significant difference in response rates between the immediate—and delayed—start groups. Importantly, the safety profile of baricitinib was favorable, with no serious adverse events directly related to the study drug. These findings suggest that baricitinib may offer an effective treatment option for IIM patients, improving both clinical response and muscle strength with a manageable safety profile.
Another highlight is the early-phase trial results of umbilical lining-derived stem cells (ULSC) in adult DM and PM. This first-in-human, open-label trial demonstrated that ULSC infusions were generally well tolerated, with only one adverse event reported, and showed promising efficacy signals. Notably, 6 out of 9 patients achieved moderate improvement in disease activity within 6 months, as measured by the composite total improvement score (TIS), and there was a significant gain in muscle strength (nearly 10 points on the MMT8 scale). Additionally, patient experienced a 35% reduction in prednisone dosage, indicating the potential of ULSC to mitigate both disease activity and corticosteroid reliance in DM/PM. These results represent an early and small but encouraging step forward in regenerative medicine for treating complex inflammatory myopathies.
9. Clinical Trials in Gout—Crystal Arthropathies
A relevant trial to be showcased is the Gout Treatment Strategy (GO TEST) Overture Trial, which provides clinical practice-changing evidence supporting the Treat to Target (T2T) approach for gout management. This trial demonstrated that patients managed with T2T—using urate-lowering therapy (ULT) to achieve and maintain serum urate (sUA) levels below 0.36 mmol/L—had significantly better outcomes compared to those managed with a Treat to Avoid Symptoms (T2S) strategy. After 1 year, 77% of patients in the T2T group achieved target sUA levels, compared to only 29% in the T2S group. Additionally, the incidence of gout flares was 54% higher in the T2S group. These findings confirm the superiority of the T2T approach, highlighting its potential to reduce disease progression and improve long-term outcomes for patients with gout.
10. Clinical Trials in Systemic Lupus Erythematosus
Long-term results from a phase 2 study of Ianalumab (VAY736) SLE will be presented, extending data to 68 weeks. Ianalumab, a human IgG1 monoclonal antibody, depletes B-cells and blocks B-cell activating factor receptor. The study included 67 patients who were randomized to receive subcutaneous Ianulamab (300 mg) or placebo every 4 weeks, switching to open-label treatment after week 28. At week 28, 44.1% of Ianalumab-treated patients met the SLE Responder Index (SRI-4) plus corticosteroid tapering endpoint, compared to 9.1% of those on placebo. By week 52, response rates for patients continuing Ianalumab were 45.5% and 40.6% for those who switched from placebo to Ianalumab. At week 68, further improvements were seen, with sustained reductions in disease activity across multiple measures, including BILAG flares and autoantibody levels. The proportion of patients achieving Lupus Low Disease Activity State (LLDAS) and SRI-6 and SRI-8 scores increased by week 68, maintaining clinical benefits. Ianalumab was well tolerated, with no new safety concerns. These findings reinforce the potential of Ianalumab for long-term SLE management, with ongoing phase 3 trials anticipated to confirm these results.
Two significant studies will present advances in SLE therapies, both targeting critical immune mechanisms. Dapirolizumab pegol (DZP) AND Ianalumab (VAY736) share a common goal of modulating key immune pathways involving B cells and T cells, but through distinct mechanisms of action. DZP, a CD40L inhibitor, was evaluated in a 24-week phase 2b trial where it blocked the CD40-CD40L interaction essential for activating T cells, B cells, and antigen-presenting cells (APCs). This inhibition resulted in significant down regulation of T cell activation markers and pro-inflammatory cytokines, such as IFN—γ and IL12B, with effects observed as early as week 2 following a single dose. By week 24, these reductions were sustained, highlighting DZP’s potential to interrupt key immune processes involved in SLE. Ianalumab, a B cell activating factor receptor (BAFFR) targeting mAb, depletes B cells via both antibody-dependent cellular cytoxicity and blockade of BAFF: BAFFR meditated survival signals. This resulted in a durable reduction of circulating B cells and associated down regulation of IFN pathways, with effects observed as early as week 28 and sustained through week 52. The long-lasting impact on B cell and neutrophil profiles further supports its potential for prolonged disease modification. Both therapies demonstrate a significant ability to disrupt T cell and B cell interactions, crucial in SLE immunopathology, offering promising timelines for achieving durable, long-term disease control.
This abstract will focus on the latest developments in the creation of a novel clinical outcome assessment (COA) for SLE, known as the Treatment Response Measure for SLE (TRM-SLE). An international task force, comprising clinicians, patients, and industry representatives, is leading this initiative to improve the outcome measures used in SLE randomized controlled trials (RCTs). The task force has reached consensus on 8 key organ-specific domains—alopecia, arthritis, hemolytic amenia, thrombocytopenia, mucosal ulcers, nephritis, rash, and serositis—based on their clinical significance and impact on patient outcomes. Key milestones include the shortlisting of over 180 candidate measures across these domains, which are currently undergoing systematic literature review. These efforts mark significant progress in refining outcome measures for SLE, with the goal of creating a more robust and clinically meaningful assessment tool, to be validated in upcoming studies.
Best wishes go out to all attendees for a productive and inspiring conference. This event showcases the dedication and innovation within the rheumatology community, promising to unveil transformative research and ideas that will shape the future of patient care.
BONUS — Pediatric Selection
11. Clinical Development
Achieving complete inactive disease (CID) in Juvenile Dermatomyositis (JDM) remains a significant hurdle, with only 25% of patients reaching this target by 12 months. While remission off medication is the goal in JDM, timely achievement of clinically inactive disease (CID) is an important interim outcome. Data from the CARRA JDM Registry, a multi-center North American JDM inception cohort, was evaluated to quantify the number of patients who met CID at 12 months. The baseline characteristics associated with achieving CID were also assessed.
The 24-week data on olokizumab (OKZ) in treating pcJIA was exclusively gathered from Russian sites and previously presented at the 31st European Pediatric Rheumatology Congress held in Gothenburg, Sweden, from September 11 to 14, 2024. OKZ, a direct interleukin-6 inhibitor used for RA, is currently under investigation in an open-label phase 2 trial involving adolescents with pcJIA who have not responded adequately to methotrexate. This study aims to evaluate the pharmacokinetics, effectiveness, and safety of OKZ in these patients.
The 16-week data on ixekizumab in treating juvenile psoriatic arthritis (JPsA) and enthesitis—related arthritis (ERA) was part of the COSPIRIT-JIA study. JPsA and ERA are classified under juvenile idiopathic arthritis (JIA) by the International League of Associations for Rheumatology and are pediatric equivalents of PsA and axSpA, respectively. IXE, an anti-interleukin-17A monoclonal antibody, has shown efficacy and safety in adults with PsA and axSpA. This face-to-face trial against adalimumab aimed to assess the efficacy and safety of IXE in pediatric patients with active JPsA and ERA over a 16-week period. The study reported an ACR30 treatment response of 88.9%, highlighting the potential of IXE in this young patient population.
12. Sessions
In recent years, the options for treating uveitis in both children with JIA and adults have expanded significantly. This session will explore the latest on the use of biologics and other immunomodulatory medications for uveitis treatment, as well as guidelines for monitoring and managing non-infectious uveitis in high-risk JIA patients. Additionally, it will cover the diagnosis and treatment of uveitis associated with JIA, describe the long-term outcomes of these patients, and apply new data on biologic medications for treating non-infectious uveitis.
This session will explore recent discoveries in the pathophysiology of pediatric lupus, focusing on the roles of genetic risk factors, specific cell populations, variants, enhancers, and gene expression in disease development and management. It will cover the application of current sequencing techniques and—omics methodologies to understand the pathogenesis of pediatric lupus, provide insights into newly identified genetic risk factors and their relation to disease etiology and biological pathways, and discuss the implications of multi-omics studies for personalized medicine in pediatric lupus.
The speakers will explore the FDA’s strategies for ensuring the availability of safe and effective drugs, with a particular focus on new initiatives relevant to pediatric rheumatology. They will review the efficacy and safety of recently approved treatments for pediatric rheumatic diseases, discuss the growing presence of biosimilars and interchangeable products, and examine the supporting data for these approvals. Additionally, the session will cover the mechanisms by which these agents were approved and the FDA’s approach to developing biosimilar and interchangeable products.
Juvenile systemic sclerosis, one of the rarest autoimmune diseases affecting children, will be the focus of this session. Attendees will receive updates on the latest discoveries in the disease’s pathogenesis and assessment methods. Additionally, the session will cover current treatments and explore emerging therapies, including innovative cell-based approaches such as autologous stem cell transplants and CAR-T cell therapy.
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