Effectiveness of PCSK9 Inhibitors in Familial Hypercholesterolemia: Feasibility Analysis

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/1000000387
• EU PAS number:
  EUPAS1000000387
• Study ID:
  1000000387
• Official title and acronym:
  Effectiveness of PCSK9 Inhibitors in Familial Hypercholesterolemia: Feasibility Analysis
• DARWIN EU® study:
  No
• Study countries:
  Canada
• Study description:
  Proprotein convertase subtilisin/kexin type 9 inhibitors (PCSK9i) are a relatively novel class of drugs used in the treatment of heterozygous familial hypercholesterolemia (hereafter, FH). However, data are limited on the real-world use and effectiveness of PCSK9i for FH in Canada. The aims of this study are 1) to determine the percentage of patients with FH achieving Canadian public drug plan treatment goals for PCSK9i, and 2) to estimate the incidence of major adverse cardiovascular events among patients with FH who are prescribed PCSK9i. Using administrative healthcare data from the provinces of Alberta, British Columbia, and Ontario, we will identify patients who began treatment with a PCSK9i (alirocumab or evolocumab) between 2015 and 2023. PCSK9i initiators will be identified using prescription drug claims, and FH status will be inferred using a combination of public drug plan reimbursement (which is limited to those with FH) and laboratory test values for low-density lipoprotein cholesterol (LDL-C). Patient characteristics at the time of PCSK9i initiation will be described. We will determine the percentage of patients achieving the public drug plans’ recommended treatment goal of at least 40% reduction in LDL-C levels during treatment with PCSK9i. The incidence of major adverse cardiovascular events during treatment will be estimated to support assessment of the feasibility of future observational studies of PCSK9i.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Lady Davis Institute

University of British Columbia

University of Alberta, Edmonton, Canada
ICES, Toronto, Canada

Networks

Canadian Network for Observational Drug Effect Studies (CNODES) and Alberta Drug and Technology Evaluation Consortium (ADTEC)

Study timelines

Date when funding contract was signed

Planned: 08/08/2024

Actual: 08/08/2024

Study start date

Planned: 08/08/2024

Actual: 08/08/2024

Date of final study report

Planned: 14/04/2025

Sources of funding

Other

More details on funding

CNODES and ADTEC are collaborating core network partners of CoLab funded for query-related activity by Canada’s Drug Agency (CNODES CDA grant number C222 360).

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable