Work

Analysis of rare events in healthcare intervention using department of defense data: intravenous immune globulin therapy for bullous pemphigoid

Year: 2025

Type: article

Abstract: INTRODUCTION: Rare events data have proven difficult to explain and predict. Standard statistical procedures can sharply underestimate the probability of rare events, such as intravenous immune globulin therapy (IVIg) for bullous pemphigoid. METHODS: This retrospective cross-sectional study used Department of Defense TRICARE data to determine factors associated with IVIg therapy among bullous pemphigoid patients. We used prior and weighted correction methods for logit regression to solve rare event bias. RESULTS: We identified 2,720 individuals diagnosed with bullous pemphigoid from 2019 to 2022, of which 14 were treated with IVIg. Patients who received IVIg therapy were younger (65.07 vs. 75.85, P =.0016) and more likely to be female (13 vs. 1, P =.0036). The underestimation with the standard regression model for event probabilities ranged from 11% to 102% using the prior correction method and from 15% to 107% using the weighted correction method. CONCLUSION: Rare events are low-frequency, high-severity problems that can have significant consequences. Rare diseases and rare therapies are individually unique but collectively contribute to substantial health and social needs. Therefore, correct estimation of the events is the first step toward assessing the burden of rare diseases and the pricing of their therapies.

Source: BMC Medical Research Methodology

DOI: 10.1186/s12874-025-02559-5

Authors: Onur Baser, Huseyin Yuce, Gabriela Samayoa

Institutions: City University of New York, John D. Dingell VA Medical Center, Boğaziçi University, New York City College of Technology

Language: English

Topic: Autoimmune Bullous Skin Diseases

Subfield: Pathology and Forensic Medicine

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: gold

Estimated APC paid: $3,090