Rare Disease

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

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

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

(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)

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

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

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

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

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

(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

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

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

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

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

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

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

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

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

(SLC-034) Trends in type 1 diabetes mellitus onset among youth aged 0 to 20 in Korea before and after COVID-19: A nationwide health insurance claims data analysis, 2003-2022

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

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