Rare Disease

((SLC-033)) (SLC-033) Establishing standing cohorts of refractory lupus patients in the fit-for-purpose Real-World Data (RWD) sources to facilitate contextualization of upcoming clinical trial safety data: A feasibility project

((SLC-035)) (SLC-035) Effect of Anemia on Mortality in Janus Kinase Inhibitors Users with Myelofibrosis: A Nationwide Cohort Study

((SLC-036)) (SLC-036) Characteristics and mortality of patients with Pulmonary Arterial Hypertension in France: A 2010-2023 Nationwide Study from the French Health Data System (SNDS)

((SLC-037)) (SLC-037) Implementing and Validating an Algorithm for Identifying ATTR-CM patients in the PHARMO Data Network

((SLC-038)) (SLC-038) Diagnosis Patterns of Retinal Vasculitis in Real-World Data

((SLC-039)) (SLC-039) Rare Findings: Characterizing External Control Arm Studies Assessed by FDA for Drugs and Biologics in Rare Disease

((SLC-040)) (SLC-040) Application of observational research methods to real-world studies for rare disease treatments: a scoping review

((A-303)) (A-303) Evolution of the treatment landscape for patients diagnosed with amyotrophic lateral sclerosis in the United States

((C-190)) (C-190) Disease burden and unmet needs of patients with amyotrophic lateral sclerosis in the United States

((C-192)) (C-192) Is There a Lack of Dietary Data Collection in Real-World Pediatric Rare Disease Studies? Literature Review and Future Considerations

((C-193)) (C-193) Therapeutic Horizon in Multiple Myeloma: Emerging Therapies and Future Perspectives

((C-194)) (C-194) Methodology and Framework for Estimating the Burden of Rare Diseases Using Regional Healthcare Data

((C-195)) (C-195) External Control Arm Studies in Marketing Authorisation Applications Submitted to the European Medicines Agency: A Review of Study Design Features and Methods

((C-196)) (C-196) Varying period prevalence of idiopathic inflammatory myopathies by enrollment factors and case definition: an exploration in a U.S. administrative claims database

((C-197)) (C-197) Prevalence of congenital and acquired generalized lipodystrophy in the EU

((C-198)) (C-198) Decoding IgA Nephropathy: Large Language Model-Powered Case Report Analysis of Etiologies and Sequential Treatment Strategies in Primary and Secondary Forms

((C-199)) (C-199) A Framework for Combining Diverse and Complementary Datasets to Create External Comparator Arms

((C-200)) (C-200) Current Status, Challenges and Opportunities in the Study of Minimum Data Set for Rare Diseases

((C-201)) (C-201) Validation of a claims algorithm for identifying patients with Duchenne muscular dystrophy in US administrative claims with genetic laboratory data

((C-202)) (C-202) A Comparative Analysis of Premarket and Postmarket Safety Data Between Non-Oncology Externally Controlled and Internally Controlled Orphan Drug Approvals