Rare diseases

(298) Are Costs Decisive in the Incorporation of Medicines for the Treatment of Ultra-rare Diseases in Brazil? Trends in Healthcare Coverage for Ultra-rare Diseases

(299) Assessing the agreement between open and closed medical claims in a rare disease cohort

(300) Assessing the feasibility of TREAT-NMD registries for characterising adult patients with SMA receiving risdiplam treatment

(301) Can riluzole use for motor neuron disease reduce risk of hospitalisation: A common data model study using Self-Controlled Case Series

(302) Canadian Pustular Psoriasis Study (CAPPS): Examining disease burden, treatments and healthcare resource utilization for palmoplantar pustulosis in Canada

(303) Estimation of period-based costs associated with individual chronic complications of rare inherited diseases, in the context of multimorbidity, using real-world data.

(304) Healthcare resource utilization among patients with multiple system atrophy in US claims database

(305) High Misclassification of Glycogen Storage Disease Type 1a in a US Electronic Medical Records Database – Insights from a Physician Note Review

(306) Incident Treatment and Adverse Liver Outcomes among Patients with Primary Sclerosing Cholangitis: An Analysis of Real-World Data

(308) Prevalence, Comorbidities, and Healthcare Costs of Rare Genetic Diseases in Germany: A Claims Database Study

(309) Race and sex differences in healthcare resource utilization among patients with myasthenia gravis in the United States

(310) Retrospective Chart Review Data Collection to Generate External Comparator Arm Populations: Considerations and Suggestions for Reducing Key Biases

(311) Sickle Cell Disease: Comorbidities, Treatment Patterns, and Healthcare Resource Utilization

(312) Strategy for identifying data sources in a rare disease through data landscaping and maximizing the usefulness of the results for researchers

(313) To be an Orphan Disease or Not to Be: An Evaluation of Prevalence Estimates for Cancers Near the Orphan Status Threshold and Implications for Orphan Drug Planning

(314) Treatment of systemic lupus erythematosus: analysis of treatment patterns in adult and paediatric patients across four European countries

(315) Treatments of multiple myeloma in Europe from 2012 to 2022: a population-based network cohort study

(316) Understanding the Prevalence of Transthyretin Amyloidosis (ATTR) in Europe: A Comprehensive Literature Review

(317) Use of European rare disease registries to describe the natural history and disease progression of Spinal Muscular Atrophy (SMA) over time