FH Optimal Care in the US (FOCUS) Real-World Data Analysis Report – 2018
The FH Foundation’s FOCUS Report quantifies gaps in diagnosis and treatment, and the unfortunate corresponding cardiovascular event rates in individuals with familial hypercholesterolemia (FH). The FOCUS Report leverages a national healthcare database built by the FH Foundation that includes healthcare encounter data on over 272 million individuals in the United States being treated, or evaluated for, cardiovascular disease. All data is anonymous at the individual level.
The new 2018 FOCUS Report includes healthcare encounter data from January - December 2018, from 45 of the largest health plans and other payers (for example, pharmacy benefit managers, self-insured employers). The results are presented by types of insurance: Commercial, Medicare, or Other (for example, Medicaid). Data may be filtered by gender, race/ethnicity, age and by health plan/payer.
These gaps in care highlight the important and meaningful opportunity we have to work together within the healthcare system to improve outcomes for people with FH by improving FH diagnosis and uptake of guideline-recommended treatment.
Opportunity to Improve FH Diagnosis
The number one barrier to FH care is lack of diagnosis. The table below shows the total number of individuals with FH, either Diagnosed FH or Undiagnosed Probable FH, in 45 of the largest health plans and other payers. Results highlight the important and significant opportunity to improve care and outcomes by proactively addressing underdiagnosis.
Individuals with FH are at High Risk
Left untreated, individuals with FH are at high risk for early cardiovascular disease. This table shows that individuals with FH, and those with diagnosed ASCVD, had a higher rate of cardiovascular events during the 12 month period from January to December 2018, compared to the total population in the database covered by these 45 health plans and other payers. There is an urgent need to improve FH diagnosis and treatment in order to prevent heart attacks and other cardiovascular events, both in the long-term and also in the short-term.
FH Treatments are Underused
There is tremendous room for improvement when it comes to using safe and effective lipid lowering treatment across each of the populations in this database. The 2018 ACC/AHA Cholesterol Guideline recommends high-intensity statin treatment for each of these populations – individuals with FH or with ASCVD – adding ezetimibe and then PCSK9i treatment for those who cannot get their LDL-C below the recommended 100 mg/dL for those with no cardiovascular disease or below 70 mg/dL for those with cardiovascular disease. Use of all LDL-C lowering therapies is generally even lower among women, non-whites, and younger people. Underuse of safe and effective treatments – starting with statins - represents a missed opportunity for prevention of heart attacks, the need for bypass or stents, and sudden cardiac death.
PCSK9 inhibitor Prescriptions are Rejected or Unfilled at High Rates
The FDA approved Proprotein Convertase Subtilisin Kexin Type 9 Inhibitors (PCSK9i) in 2015 as a treatment for people with FH or ASCVD who need additional LDL-C lowering. For individuals with FH whose LDL-C remains above 100 mg/dL (no ASCVD) or above 70 mg/dL (with ASCVD), on maximally tolerated lipid lowering therapies (statins and ezetimibe), a PCSK9i is recommended according to the 2018 ACC/AHA Cholesterol Guideline.
However, this table shows that PCSK9i are prescribed for a small percentage of individuals with FH or ASCVD, and that coverage is denied by payers at a high rate. In addition, a substantial percentage of individuals who are approved by their health plan do not fill their prescription, in many cases because of high out-of-pocket costs. This disproportionately impacts the Medicare population, as copay assistance is not allowed, by law, for this population.
Diagnosed FH: This category represents individuals with the ICD-10 diagnosis code for Familial Hypercholesterolemia, E78.01. E78.01 has been in effect since October 2016. While FH diagnosis and use of the diagnosis code E78.01 is improving, at the end of 2018 the total number of individuals in the database with this code was 197,479, or approximately 15% of the estimated 1.3 million people in the US with FH.
Undiagnosed Probable FH: This category represents individuals identified with the FH Foundation’s FIND FH® Machine Learning model, but who do not have E78.01 in their record. The Undiagnosed Probable FH prevalence is higher in the FH Foundation’s database than in the general population because this is a database of individuals with diagnosis or procedure codes related to the evaluation or treatment of cardiovascular disease and risk factors.
ASCVD: Atherosclerotic Cardiovascular Disease, defined as myocardial infarction (heart attack), stable or unstable angina, need for revascularization (stent or coronary artery bypass graft surgery), stroke, transient ischemic attack, abdominal aortic aneurysm, and peripheral arterial disease.
Rejected: Final prescription coverage status was rejected by insurance.
Paid: Individuals were approved by insurance and received 338 or more days of therapy within 12 months.
Unfilled: The prescription was approved by insurance but not filled.
Cardiovascular Event: Myocardial Infarction (MI), ischemic stroke, coronary artery bypass graft (CABG), Percutaneous Coronary Intervention (PCI – stents), Cardiac Arrest, acute ischemic heart disease (Acute Coronary Syndrome), or unstable angina.
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