title: Modeling strategy to identify patients with primary immunodeficiency utilizing risk management and outcome measurement.
abstract: This study seeks to generate analytic insights into risk management and probability of an identifiable primary immunodeficiency defect. The Jeffrey Modell Centers Network database, Jeffrey Modell Foundation's 10 Warning Signs, the 4 Stages of Testing Algorithm, physician-reported clinical outcomes, programs of physician education and public awareness, the SPIRIT® Analyzer, and newborn screening, taken together, generates P values of less than 0.05%. This indicates that the data results do not occur by chance, and that there is a better than 95% probability that the data are valid. The objectives are to improve patients' quality of life, while generating significant reduction of costs. The advances of the world's experts aligned with these JMF programs can generate analytic insights as to risk management and probability of an identifiable primary immunodeficiency defect. This strategy reduces the uncertainties related to primary immunodeficiency risks, as we can screen, test, identify, and treat undiagnosed patients. We can also address regional differences and prevalence, age, gender, treatment modalities, and sites of care, as well as economic benefits. These tools support high net benefits, substantial financial savings, and significant reduction of costs. All stakeholders, including patients, clinicians, pharmaceutical companies, third party payers, and government healthcare agencies, must address the earliest possible precise diagnosis, appropriate intervention and treatment, as well as stringent control of healthcare costs through risk assessment and outcome measurement. An affected patient is entitled to nothing less, and stakeholders are responsible to utilize tools currently available. Implementation offers a significant challenge to the entire primary immunodeficiency community.
authors.affiliation: Jeffrey Modell Foundation, 780 Third Avenue, 47th Floor, New York City, NY, 10017, USA.
authors.forename: Vicki
authors.initials: V
authors.lastname: Modell
authors.affiliation: Jeffrey Modell Foundation, 780 Third Avenue, 47th Floor, New York City, NY, 10017, USA.
authors.forename: Jessica
authors.initials: J
authors.lastname: Quinn
authors.affiliation: Jeffrey Modell Foundation, 780 Third Avenue, 47th Floor, New York City, NY, 10017, USA.
authors.forename: Grant
authors.initials: G
authors.lastname: Ginsberg
authors.affiliation: Jeffrey Modell Foundation, 780 Third Avenue, 47th Floor, New York City, NY, 10017, USA.
authors.forename: Ron
authors.initials: R
authors.lastname: Gladue
authors.affiliation: Texas Children's Hospital, Baylor College of Medicine, Houston, TX, USA.
authors.forename: Jordan
authors.initials: J
authors.lastname: Orange
authors.affiliation: Jeffrey Modell Foundation, 780 Third Avenue, 47th Floor, New York City, NY, 10017, USA. fmodell@jmfworld.org.
authors.forename: Fred
authors.initials: F
authors.lastname: Modell
mesh_terms: D000465:Algorithms; D003362:Cost-Benefit Analysis; D016208:Databases, Factual; D042241:Early Diagnosis; D006801:Humans; D007153:Immunologic Deficiency Syndromes / Q000175:diagnosis* / Q000453:epidemiology; D007231:Infant, Newborn; D015997:Neonatal Screening; D017063:Outcome Assessment, Health Care; D015995:Prevalence; D011788:Quality of Life; D012041:Regional Medical Programs; D012306:Risk; D014481:United States / Q000453:epidemiology
keywords: Diagnosis; Immunology; Jeffrey Modell Centers Network (JMCN); Jeffrey Modell Foundation (JMF); Modeling; Outcome measurement; Primary immunodeficiencies (PI); Risk management; Treatment