Uterine fibroids (UF) are common benign tumours of the uterus (1,2) with bleeding and pain symptoms negatively affecting quality of life of women (3,4,5). Treatment options include surgeries and/or medical treatment. Since 2012, a new drug, ulipristal, can be prescribed for repeated-intermittent use in women with uterine fibroids.
Bayer is developing a drug for long-term treatment of UF. We propose to study the current use of ulipristal i using routinely collected health information from the Clinical Practice Research Datalink. We will use anonymised data that is routinely collected by general practitioners. The results will help us to understand the target population and treatment patterns in a large and well-characterised sample of women.
The results of our study are expected to help ascertain a better understanding of current healthcare resource utilisation in women with symptomatic UF.
Background
Bayer is developing a new selective progesterone receptor modulator (SPRM), Vilaprisan, for long-term pharmacological UF treatment. To prepare access and health-economic evidence, a detailed description of the ulipristal target population and treatment pathways in a large and well-characterized sample of women are needed.
Objectives
This population-based descriptive study will characterize first-time users of ulipristal and utilisation patterns including switching to other medications and invasive gynaecological treatments following ulipristal treatment
Methods
This is a retrospective cohort study with prospective data collection, designed to assess the characteristics of women and patterns of drug utilization in new users of ulipristal in the UK. Data will be obtained by merging the The Health Improvement Network (THIN) and the Clinical Practice Research Datalink (CPRD), with the latter supplemented by data from HES database. The enrolment period will be from January 1, 2012 (year ulipristal became available in the UK) to April 30, 2017 (THIN) and the latest available version of CPRD. All women with at least one prescription of ulipristal during the study period will be included.
Data analysis
The study is of descriptive nature. The analyses will be conducted using standard summary statistics such as means and proportions; continuous data will be presented as means with standard deviation (SD) and as medians with inter quartile range (IQR, min and max) when appropriate; categorical variables will be presented as counts (n) and proportions (%). Missing data will not be imputed. Survival analyses will be used for time-to-event outcomes (e.g. time to invasive gynaecological treatments after the last ulipristal course, time to pregnancy, etc.); women will be censored at the date of last practice data collection. The analyses will be performed for the total and sub-cohorts with respective number of ulipristal courses (i.e. women with 1, 2, 3, and 4 courses during the study period). The alternative sub-cohort's cuts may be considered after a review of ulipristal treatment pattern.
The details on reporting and analysis of individual outcomes (incl. subgroups, handling of competing risk/events in survival analysis) will be described in the statistical analysis plan (SAP).
Statistical analyses will be performed using STATA package version 12.0 (StataCorp LP, College Station, TX, USA).
To describe utilization patterns of ulipristal, including switching to other medications and the invasive gynecological treatments following treatment with ulipristal
- To provide detailed information on baseline demographic and clinical characteristics of women newly exposed to ulipristal, including its indication (uterine fibroids, endometriosis, heavy menstrual bleeding, other)
- To describe gynaecological diagnostic procedures, pharmacological treatments and healthcare resource use (primary, secondary, hospitalizations) within 1 year and invasive gynaecological treatments within the 5 years prior to receiving the first prescription of ulipristal
- To describe occurrence of pregnancy events in women newly exposed to ulipristal from the start of ulipristal use to the study end
Renate Schulze-Rath - Chief Investigator - Bayer AG
Renate Schulze-Rath - Corresponding Applicant - Bayer AG
Charles Kreilick - Collaborator - Bayer AG
Kamonthip Wichmann - Collaborator - Bayer AG
Lucia Soriano - Collaborator - University Complutense of Madrid
Luis Alberto Garcia Rodriguez - Collaborator - Centro Español de Investigaciones Farmacoepidemiológicas S.L. ( CEIFE S.L. )
HES Admitted Patient Care;Patient Level Townsend Score;Pregnancy Register