FROM NATURAL HISTORY TO POST-APPROVAL EVIDENCE: AN INTEGRATED MULTI-SOURCE REAL-WORLD PROGRAM CHARACTERIZING IGA NEPHROPATHY IN THE ERA OF ORAL TARGETED-RELEASE FORMULATION OF BUDESONIDE
Author(s)
Giancarlo Pesce, PhD1, Lobat Hashemi, PhD2, Ramin Tolouian, MD3, Margaret Helmuth, PhD4, Artak Khachatryan, MPH, PhD, MD5, Ananth Kadambi, MS, PhD6, Terri Madison, PhD7;
1Certara Italy, Milan, Italy, 2Veloxis Pharmaceuticals, Cary, NC, USA, 3Calliditas Therapeutics, New York, NY, USA, 4University of Michigan, Ann Arbor, MI, USA, 5Certara UK, London, United Kingdom, 6Certara USA, Radnor, PA, USA, 7Madison Epi Solutions LLC, Ann Arbor, MI, USA
1Certara Italy, Milan, Italy, 2Veloxis Pharmaceuticals, Cary, NC, USA, 3Calliditas Therapeutics, New York, NY, USA, 4University of Michigan, Ann Arbor, MI, USA, 5Certara UK, London, United Kingdom, 6Certara USA, Radnor, PA, USA, 7Madison Epi Solutions LLC, Ann Arbor, MI, USA
OBJECTIVES: To generate complementary perspectives on immunoglobulin A nephropathy (IgAN) disease burden, management, and real-world therapeutic safety and effectiveness by integrating registry, electronic health records (EHR), and chart-review data across the pre- and post-approval landscape of oral targeted-release budesonide therapy.
METHODS: Three independent US-based observational studies were conducted. First, an ancillary study using the CureGN registry evaluated disease characteristics, treatment patterns, and long-term outcomes among 653 biopsy-confirmed IgAN patients (2015-2024). Second, a retrospective EHR study using the TriNetX network (2011-2022) compared safety outcomes, healthcare resource utilization, and costs among propensity-score-matched IgAN patients treated with systemic glucocorticoids (SGC, n=401) versus untreated patients (non-SGC, n=401). Third, a retrospective chart review of 45 IgAN patients treated with oral targeted-release formulation of budesonide (2022-2023) at the Chinatown Kidney Care (New York) assessed changes in estimated glomerular filtration rate (eGFR), urine protein-creatinine ratio (UPCR), and adverse events (AEs) up to 12 months.
RESULTS: In the registry analysis, median follow-up was six years. SGC use (≈60%) was not associated with slower eGFR decline; younger adults and adolescents experienced the most rapid progression, underscoring an early-intervention window. In TriNetX, SGC therapy was associated with higher rates of serious infections (3.5% vs 0.3%), greater healthcare costs (USD 37,526 vs 12,957 per patient-year), and no significant benefit effects in kidney function preservation. In the chart review, eGFR remained stable and UPCR declined by ~20% at nine months following targeted-release budesonide initiation, with greater benefits among patients completing 9-month therapy cycle. AEs were mild and consistent with the known safety profile.
CONCLUSIONS: Registry and EHR analyses highlighted unmet needs and the limitations of systemic corticosteroids, while chart-level data demonstrated the favorable real-world effectiveness and tolerability of oral targeted-release formulation of budesonide. Triangulating evidence from diverse RWD sources yields a comprehensive understanding of IgAN and supports regulatory, clinical, and payer decision-making in rare kidney diseases.
METHODS: Three independent US-based observational studies were conducted. First, an ancillary study using the CureGN registry evaluated disease characteristics, treatment patterns, and long-term outcomes among 653 biopsy-confirmed IgAN patients (2015-2024). Second, a retrospective EHR study using the TriNetX network (2011-2022) compared safety outcomes, healthcare resource utilization, and costs among propensity-score-matched IgAN patients treated with systemic glucocorticoids (SGC, n=401) versus untreated patients (non-SGC, n=401). Third, a retrospective chart review of 45 IgAN patients treated with oral targeted-release formulation of budesonide (2022-2023) at the Chinatown Kidney Care (New York) assessed changes in estimated glomerular filtration rate (eGFR), urine protein-creatinine ratio (UPCR), and adverse events (AEs) up to 12 months.
RESULTS: In the registry analysis, median follow-up was six years. SGC use (≈60%) was not associated with slower eGFR decline; younger adults and adolescents experienced the most rapid progression, underscoring an early-intervention window. In TriNetX, SGC therapy was associated with higher rates of serious infections (3.5% vs 0.3%), greater healthcare costs (USD 37,526 vs 12,957 per patient-year), and no significant benefit effects in kidney function preservation. In the chart review, eGFR remained stable and UPCR declined by ~20% at nine months following targeted-release budesonide initiation, with greater benefits among patients completing 9-month therapy cycle. AEs were mild and consistent with the known safety profile.
CONCLUSIONS: Registry and EHR analyses highlighted unmet needs and the limitations of systemic corticosteroids, while chart-level data demonstrated the favorable real-world effectiveness and tolerability of oral targeted-release formulation of budesonide. Triangulating evidence from diverse RWD sources yields a comprehensive understanding of IgAN and supports regulatory, clinical, and payer decision-making in rare kidney diseases.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
SA20
Topic
Study Approaches
Topic Subcategory
Registries
Disease
SDC: Rare & Orphan Diseases, SDC: Urinary/Kidney Disorders