Full length article Ulipristal acetate for pre-operative management of uterine fibroids: Modeling outcomes and costs Brigitta Badiani a , Marco Chiumente b, *, Andrea Messori b a Post-graduate School of Hospital Pharmacy, University of Firenze, Firenze, Italy b Scientific Direction, SIFACT, Italian Society for Clinical Pharmacy and Therapeutics, Milano, Italy A R T I C L E I N F O Article history: Received 21 August 2017 Received in revised form 8 January 2018 Accepted 16 January 2018 Available online xxx Keywords: Costs and cost analysis Economic model Leiomyoma Markov chains Quality-adjusted life years A B S T R A C T Objectives: The aim of this study was to evaluate the pharmacoeconomic profile in Italy of preoperative treatment with ulipristal acetate at the dose of 5 mg/day for 13 weeks in comparison with placebo prior to surgical management of symptomatic uterine fibroids. Study design: The pharmacoeconomic analysis was based on the calculation of incremental cost- effectiveness ratio (ICER). Effectiveness data were derived from the randomized-controlled trial PEARL-1, whilst costs data were retrieved from the published literature. A Markov model was employed to simulate the pattern of costs and two univariate sensitivity analyses tested the robustness of the results. Results: In comparison with placebo, ulipristal acetate 5 mg for presurgical therapy was estimated to be associated with an incremental cost of s351 per patient. Costs per patient were s3836 for ulipristal acetate vs s3485 for placebo. The incremental effectiveness was 0.01931 QALYs per patient (around 7 quality-adjusted days per patient). Hence, the cost effectiveness ratio was calculated to be s18,177 per QALY gained. Conclusions: Preoperative use of ulipristal acetate 5 mg in patients with uterine fibroids has a favourable pharmacoeconomic profile. © 2018 Elsevier B.V. All rights reserved. Introduction In women in reproductive age, uterine leiomyomas (or fibroids) may cause several symptoms including excessive bleeding, pelvic pain, iron deficiency anemia, infertility and reduced quality of life. Apart from surgical intervention, there are no medical treatments that completely eliminate fibroids. However, medical treatments (e.g. GnRH analogues, oral progestogens and the levonorgestrel intrauterine system) can effectively be employed in the pre- operative period for the reduction of symptoms, in particular heavy bleeding and anemia. Results of a pivotal randomized controlled trial (the PEARL-I study [1]) have shown that treatment with ulipristal acetate 5 mg daily for 12-weeks before planned surgery is effective in controlling bleeding, decreasing fibroid volume, and reducing discomfort in women with menorrhagia and anemia. Therefore, ulipristal acetate 5 mg has been approved for pre-surgical treatment by the European Medicines Agency (EMA) in 2012 and thereafter by the Italian Medicines Agency (AIFA)[2]. Surgical treatments, currently indicated for these benign tumors, include hysterectomy and myomectomy and both are invasive procedures. At present, the drugs approved for the pre- surgical treatment include Gonadotropin-Releasing Hormone (GnRH) analogues and ulipristal acetate [3]. Choice of the treatment is closely related to the attitudes of gynecologists [4]. The main aim of our work was to evaluate the pharmacoeco- nomic profile in Italy of a single-13-week cycle of ulipristal acetate 5 mg daily in comparison with placebo before surgery for the management of symptomatic uterine fibroids. Our pharmacoeconomic analysis was aimed at determining the costs incurred by patients treated or not treated with ulipristal acetate 5 mg/day; a Markov model was developed for this purpose. Materials and methods PEARL-I trial In this randomized trial, women with symptomatic fibroids, excessive uterine bleeding (a score of >100 on the pictorial blood- loss assessment chart [PBAC, an objective assessment of blood loss, * Corresponding author at: Scientific Direction, Italian Society for Clinical Pharmacy and Therapeutics, Via Carlo Farini 81, Milano, Italy. E-mail address: marcochiume@yahoo.it (M. Chiumente). https://doi.org/10.1016/j.ejogrb.2018.01.020 0301-2115/© 2018 Elsevier B.V. All rights reserved. European Journal of Obstetrics & Gynecology and Reproductive Biology 222 (2018) 84–88 Contents lists available at ScienceDirect European Journal of Obstetrics & Gynecology and Reproductive Biology journal homepage: www.else vie r.com/locat e/e jogrb