Real World Adherence of PCSK9 Targeted Therapy

Real World Adherence of PCSK9 Targeted Therapy: A Retrospective Analysis of Japan Databases

Retrospective observational cohort study to compare medication adherence at 12 months between patients in Japan with dyslipidemia and/or hypercholesterolemia (familial and non-familial hypercholesterolemia) who newly initiate inclisiran versus those who newly initiate evolocumab.

Study Overview

• Status: Not yet recruiting
• Conditions: Dyslipidemia, Hypercholesterolemia

• Study Type: Observational
• Enrollment (Estimated): 3600

Contacts and Locations

• Study Contact: Name: Novartis Pharmaceuticals; Phone Number: +41613241111; Email: novartis.email@novartis.com
• Study Contact Backup: Name: Novartis Pharmaceuticals

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Adult patients with dyslipidemia and/or hypercholesterolemia who initiated treatment with inclisiran or evolocumab in the period after first approval of inclisiran in 22-Nov-2023

Description

Inclusion Criteria:

The patients are included if they meet all the following criteria:

1. At least one prescription of inclisiran or evolocumab (140 mg Pens) during the identification period.
2. At least one diagnosis of dyslipidemia and/or hypercholesterolemia (familial and non-familial hypercholesterolemia) without suspicion flag during the look back period.
3. At least 18 years on the index date.
4. At least one claim record within the 210 days prior to the index date (excluding the index date).

Exclusion Criteria:

Those with any of the following criteria will be excluded.

1. Both prescription records of inclisiran and evolocumab 140 mg Pens on the index date.
2. At least one claim data for inclisiran or evolocumab 140 mg Pens within the 210 days prior to the index date (excluding the index date).
3. At least one claim data for evolocumab 420 mg Auto mini-dosers during the observation period.
4. Incomplete data for the one year (365 days) follow-up period due to loss to follow-up (the end of the observation period) or death. (only for adherence cohort).

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort
exposure group; patients who initiated inclisiran as their first treatment
control group; patients who initiated evolocumab as their first treatment

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Medication adherence at 12 months : proportion of days covered (PDC) within a period that the patient is persistent	Adherence is defined using proportion of days covered (PDC) within a period that the patient is persistent: calculated as the number of days covered by the target drug (inclisiran in the exposure group, evolocumab in the control group) during the 12 months follow-up period, divided by 365 days.	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Proportion of patients with good adherence	Proportion of patients with good adherence at 12 months follow-up period, defined as PDC ≥ 80%.	12 months
Number of patients by demographic category		Baseline
Time to discontinuation	The date of discontinuation of first treatment drug will be considered as the date of outcome occurrence.	12 months
Proportion of patients receiving LLTs	Proportion of patients receiving LLTs (statin only/ezetimibe only/statin and ezetimibe) during the baseline period and 12 months follow-up period	12 months

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Estimated): March 30, 2026
• Primary Completion (Estimated): July 30, 2026
• Study Completion (Estimated): July 30, 2026

Study Registration Dates

• First Submitted: March 5, 2026
• First Submitted That Met QC Criteria: March 9, 2026
• First Posted (Actual): March 12, 2026

Study Record Updates

• Last Update Posted (Actual): March 20, 2026
• Last Update Submitted That Met QC Criteria: March 18, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Keywords: adherence; Japan; retrospective; PCSK9i
• Additional Relevant MeSH Terms: Metabolic Diseases; Hyperlipidemias; Lipid Metabolism Disorders; Nutritional and Metabolic Diseases; Hypercholesterolemia; Dyslipidemias
• Other Study ID Numbers: CKJX839A1JP08

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO