Rare Disease

(157) Childhood cerebral adrenoleukodystrophy (CCALD) in France: epidemiology, natural history, and burden – a real-world study using registry data linked to the French national claims database

(158) Healthcare Resource Utilization from OPERA: A Real-World Study of Pegcetacoplan Treatment in US Adults with Paroxysmal Nocturnal Hemoglobinuria

(159) Prevalence and use of medications for autoimmune diseases during pregnancy

(160) Metabolic Decompensation Events in the Propionic Acidemia Population within a Large EHR-Based Real-World Data Source

(161) Impact of population opt-outs on rare disease prevalence: a simulation study

(162) Rare disease observational research enabled through the federated data network approach: PHederation use case

(163) A Novel Patient-Centered Real-World Evidence Study Designed to Better Understand Short Bowel Syndrome Using Longitudinal Data in the United States

(164) Methodological Approaches for External Control Arms in Rare Diseases

(165) Improving Longitudinally in Rare Disease Data Capture in Real-world Evidence Combining Clinical Trials and Observational Data: A Case Study Using EVOLVE

(166) Novel methods for the identification of a rare disease, hemophagocytic lymphohistiocytosis (HLH)

(167) Drug use in presence of contraindications in MG patients in three Italian Regions: a real-world observational study in Italy - The CAESAR study

(168) Emicizumab wastage estimation for treating people with hemophilia A and inhibitors who failed immune tolerance induction according to the current Brazilian public health protocol

(170) Prevalence and Incidence of Multiple System Atrophy in Real World Setting: Results from US Claims Database

(171) Clinical Outcomes and Population Characteristics in Fibrodysplasia Ossificans Progressiva: A Systematic Literature Review

(172) A Feasibility Evaluation of US Healthcare Claims Data to Describe Patient Healthcare Utilization Patterns for Dengue Infection

(173) Descriptive Epidemiology of Adult T-cell Leukemia/Lymphoma in the United States

(174) An expanded algorithm to estimate prevalence of amyotrophic lateral sclerosis in US claims and UK primary care

(175) Hereditary angioedema case validation using medical claims data from commercially insured patients

(176) Epidemiology and Acute Care Utilization of Patients with Sickle Cell Disease who Present to the Emergency Department

(177) Clinical Characteristics of the Propionic Acidemia Population Identified Within a Large EHR-Based Real-World Data Source (2015-2022)

(178) Real world evidence in Rare Disease – Application of fit for purpose data sources framework in portopulmonary hypertension

(179) Characterizing Patient Care Before And After A Myasthenia Gravis Diagnosis

(181) Descriptive Epidemiology of Peripheral T-Cell Lymphoma in the United States

(182) Alternative Analytic Approaches to Forming a Benchmark Cohort in Rare Diseases

(183) To Estimate the Frequency of Brain Metastases, Presentation of Metastases, Location, Treatment Pattern and paradigm changes in the Brain Metastases Management Among the Population of Southern Asia

(184) Challenges and Mitigations of Conducting a Long-Term Effectiveness Study in a Rare Disease (Spinal Muscular Atrophy)

(185) Descriptive Characteristics and Validation of Fibrotic Interstitial Lung Disease Patients (ILD) with and without progression Using a Claims Based Algorithm

(186) Appropriate Use of Observed Versus Expected Analyses for Newly Emerging Safety Concerns: The Case of Vaccine-Induced Thrombotic Thrombocytopenia Following COVID-19 Vaccination

(187) Prevalence of rare blood cancers in Europe: a population-based network cohort study part of DARWIN EU®