Estimating Rare Disease Incidence and Prevalence of Essential Thrombocythemia in Major Health Plans in the United States
Author(s)
Andrew Howe, BA, PharmD1, Paul Walden, PhD2, Noemi Hummel, PhD3, Craig Zimmerman, PhD2.
1Pharmaessentia, Woodstock, GA, USA, 2Medical, Pharmaessentia USA, Burlington, MA, USA, 3Certara GmbH, Lörrach, Germany.
1Pharmaessentia, Woodstock, GA, USA, 2Medical, Pharmaessentia USA, Burlington, MA, USA, 3Certara GmbH, Lörrach, Germany.
OBJECTIVES: Essential thrombocythemia (ET) is a rare myeloproliferative neoplasm (MPN) with risks of thromboembolic events and progression to myelofibrosis or acute myeloid leukemia. Current ET treatment landscape is limited despite the lack specificity of prior ICD-10 diagnosis coding and ET patient medical expenditures being 2.3 times greater versus matched comparisons. The study objective was to estimate the ET incident and prevalent patient populations eligible for National Comprehensive Cancer Network (NCCN) guideline driven treatments among major US health plans.
METHODS: A systematic literature assessment identified epidemiologic estimates to determine the prevalence and incidence of ET, the revised IPSET-thrombosis risk stratification (rIPSET-T) distribution and identify the patients eligible to receive NCCN 1.2025 recommended ET cytoreductive therapy options. Health plan membership data from the eight largest commercial health plans was determined from Earning releases, Annual and 10-K reports. The data was incorporated into Microsoft Excel to create model inputs and calculate the base-case analysis of a clinically treatment eligible population that may potentially receive NCCN recommended treatments.
RESULTS: Sixteen references identified the epidemiology, rIPSET-T distribution, and utilization of NCCN recommended treatment options in separate systematic literature searches. Of the 8 health plans assessed, the mean and median lives covered were 38,725,462 and 27,847,800 respectively. A mean 426 (SD±362) and 7,612 (SD±5,507) incident and prevalent ET NCCN cytoreductive therapy eligible patients among the US health plans were identified. Among the ET health plan(s) population, a mean of 192 (SD±109), and 8,797 (SD±7,479) in incident high-and prevalent intermediate and high-rIPSET-T eligible patients were identified respectively.The percentage of NCCN identified treatment eligible patients was 72.5% representing a mean of 128 (SD±109) and 5,101 (SD±3,690) of incident and prevalent patients, respectively.
CONCLUSIONS: Wide variation exists in both prevalent and incident patient estimates across US commercial health plans effecting eligibility for NCCN recommended ET cytoreductive therapies and formulary policy recommendations.
METHODS: A systematic literature assessment identified epidemiologic estimates to determine the prevalence and incidence of ET, the revised IPSET-thrombosis risk stratification (rIPSET-T) distribution and identify the patients eligible to receive NCCN 1.2025 recommended ET cytoreductive therapy options. Health plan membership data from the eight largest commercial health plans was determined from Earning releases, Annual and 10-K reports. The data was incorporated into Microsoft Excel to create model inputs and calculate the base-case analysis of a clinically treatment eligible population that may potentially receive NCCN recommended treatments.
RESULTS: Sixteen references identified the epidemiology, rIPSET-T distribution, and utilization of NCCN recommended treatment options in separate systematic literature searches. Of the 8 health plans assessed, the mean and median lives covered were 38,725,462 and 27,847,800 respectively. A mean 426 (SD±362) and 7,612 (SD±5,507) incident and prevalent ET NCCN cytoreductive therapy eligible patients among the US health plans were identified. Among the ET health plan(s) population, a mean of 192 (SD±109), and 8,797 (SD±7,479) in incident high-and prevalent intermediate and high-rIPSET-T eligible patients were identified respectively.The percentage of NCCN identified treatment eligible patients was 72.5% representing a mean of 128 (SD±109) and 5,101 (SD±3,690) of incident and prevalent patients, respectively.
CONCLUSIONS: Wide variation exists in both prevalent and incident patient estimates across US commercial health plans effecting eligibility for NCCN recommended ET cytoreductive therapies and formulary policy recommendations.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EPH85
Topic
Epidemiology & Public Health, Real World Data & Information Systems, Study Approaches
Topic Subcategory
Disease Classification & Coding
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology, Personalized & Precision Medicine, Rare & Orphan Diseases