Estimation of Treatment Eligible Patients With Transthyretin Cardiac Amyloidosis Within a Health Plan
Author(s)
Justin Grodin, MD, MPH1, Lori Roth, MS, PA-C1, Trina Huynh, PharmD2, Liana Hennum, MHA3, Heather Falvey, MSc3;
1University of Texas Southwestern, Dallas, TX, USA, 2University of California San Diego Health, San Diego, CA, USA, 3BridgeBio Pharma, Inc., San Francisco, CA, USA
1University of Texas Southwestern, Dallas, TX, USA, 2University of California San Diego Health, San Diego, CA, USA, 3BridgeBio Pharma, Inc., San Francisco, CA, USA
OBJECTIVES: Transthyretin cardiac amyloidosis (ATTR-CM) is a progressive and fatal disease with considerable burden to health systems. Recent advancements have led to improved disease awareness and earlier diagnosis but there are varying estimates of prevalence and limited data on the number of patients seeking treatment. We sought to estimate the number of treatment eligible patients with ATTR-CM in a hypothetical health plan with 1 million members over a 3-year time horizon.
METHODS: The proportion of Medicare and commercial beneficiaries was an assumption based on claims data (75% Medicare and 25% commercial beneficiaries with ATTR-CM). A literature search was conducted to estimate prevalence rates of heart failure (HF), HF with preserved ejection fraction (HFpEF), and ATTR-CM. Diagnosis rates were calculated based on the literature.
RESULTS: Assuming a hypothetical health plan of 1 million members, an estimated 56 032 patients (5.6%) have HF. Of these patients, an estimated 23 533 patients (42.0%) have HFpEF with an incident population of 2700 patients per year (0.3%). A total of 1513 (6.4%) patients with HFpEF are estimated to have ATTR-CM, with 306 patients (20.2%) expected to be newly diagnosed with ATTR-CM and eligible for treatment in year 1 (prevalent population). An additional 35 incident patients are estimated in each subsequent year.
CONCLUSIONS: In a hypothetical health plan of 1 million members, over a 3-year time horizon, 20.2% of patients with HFpEF and who are estimated to have ATTR-CM, are also expected to be diagnosed with ATTR-CM and be eligible for treatment in year 1. This would be followed by 35 additional patients in years 2 and 3, respectively. The ATTR-CM population seeking treatment may be small when compared with other chronic diseases. With the improvements in ATTR-CM awareness and diagnosis in recent years, early diagnosis and treatment are vital.
METHODS: The proportion of Medicare and commercial beneficiaries was an assumption based on claims data (75% Medicare and 25% commercial beneficiaries with ATTR-CM). A literature search was conducted to estimate prevalence rates of heart failure (HF), HF with preserved ejection fraction (HFpEF), and ATTR-CM. Diagnosis rates were calculated based on the literature.
RESULTS: Assuming a hypothetical health plan of 1 million members, an estimated 56 032 patients (5.6%) have HF. Of these patients, an estimated 23 533 patients (42.0%) have HFpEF with an incident population of 2700 patients per year (0.3%). A total of 1513 (6.4%) patients with HFpEF are estimated to have ATTR-CM, with 306 patients (20.2%) expected to be newly diagnosed with ATTR-CM and eligible for treatment in year 1 (prevalent population). An additional 35 incident patients are estimated in each subsequent year.
CONCLUSIONS: In a hypothetical health plan of 1 million members, over a 3-year time horizon, 20.2% of patients with HFpEF and who are estimated to have ATTR-CM, are also expected to be diagnosed with ATTR-CM and be eligible for treatment in year 1. This would be followed by 35 additional patients in years 2 and 3, respectively. The ATTR-CM population seeking treatment may be small when compared with other chronic diseases. With the improvements in ATTR-CM awareness and diagnosis in recent years, early diagnosis and treatment are vital.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EPH62
Topic
Epidemiology & Public Health
Disease
SDC: Cardiovascular Disorders (including MI, Stroke, Circulatory), SDC: Rare & Orphan Diseases