Study type

Study topic

Human medicinal product

Study type

Non-interventional study

Scope of the study

Effectiveness study (incl. comparative)
Non-interventional study

Non-interventional study design

Cohort
Study drug and medical condition

Name of medicine

TAVNEOS

Study drug International non-proprietary name (INN) or common name

AVACOPAN

Anatomical Therapeutic Chemical (ATC) code

(L04AJ05) avacopan
avacopan

Medical condition to be studied

Granulomatosis with polyangiitis
Microscopic polyangiitis
Population studied

Age groups

Adults (18 to < 65 years)
Adults (18 to < 46 years)
Adults (46 to < 65 years)
Adults (65 to < 75 years)
Adults (75 to < 85 years)
Adults (85 years and over)

Estimated number of subjects

305
Study design details

Main study objective

To describe characteristics associated with avacopan prescription and initiation, and outcomes observed among avacopan initiators. Among avacopan initiators: (1) investigate factors associated with glucocorticoid discontinuation and relapse risk; and (2) evaluate healthcare resource utilization.

Outcomes

Primary Outcomes:
• Number or participants achieving discontinuation of glucocorticoids for treatment of GPA or MPA 1 month before Month 6 and disease remission within 6 months of avacopan initiation.
• Number of participants achieving sustained remission at Month 12, defined as remission at Month 6 and remission at Month 12, with no glucocorticoid treatment for GPA/MPA 1 month before Month 12, and no relapse between Months 6 and 12.

Data analysis plan

There are three primary analyses. Analysis 1 will describe the baseline characteristics of participants by avacopan prescription status (yes/no), as well as the baseline characteristics of participants prescribed avacopan by initiation status (yes/no). Analyses 2 and 3 will examine the 6- and 12-month outcomes among initiators of avacopan, respectively. Analysis 1 will be performed at least six months after the study start date when the final cohort of avacopan initiators is identified and baseline data are collected. Analyses 2 and 3 will begin once at least 6 months after the last avacopan participant is identified during the inclusion period and at least 6 months of follow-up data are collected. The primary outcomes will be analysed using a descriptive approach. Remission will be based on the Birmingham Vasculitis Activity Score version 3 with/out glucocorticoids for the treatment of GPA/MPA in the 4 weeks prior to the month 6 or 12 visits. Summary statistics for continuous variables, including the number of patients, mean, standard deviation (SD), median, Q1, Q3, minimum and maximum, will be calculated after excluding missing/unknown values.