Comparing Options for Management:PAtient-centered REsults for Uterine Fibroids Evan R. Myers, MD, MPH Department of Obstetrics & Gynecology and Duke Clinical Research Institute, Duke University, Durham, NC evan.myers@duke.edu

ORGANIZATION P50 (“Center of Excellence”) Grant Broad aim is to put infrastructure in place to conduct a range of studies Funding by PCORI, administered by AHRQ Data Coordinating Center at Duke Clinical Research Institute 9 Clinical Centers Stakeholder Advisory Group (patients, payers, professional societies, government agencies, industry)

Main Aims Compare duration of symptom relief among available treatments Initial focus on procedures Compare impact of treatments on reproductive outcomes (time to menopause, fertility, pregnancy outcomes) Identify important subgroup effects (heterogeneity of treatment effect) Data elements driven by emphasis on comparative effectiveness

Patients Scheduled to undergo treatment at facility affiliated with Clinical Site Outreach methods vary by site E-mai/letter from attending physician Subsequent contact by e-mail/phone/in-person Electronic portal for E-consent Completion of baseline and follow-up data Other communications with patients Follow-up at post-procedure visit, 12, 24, 36 (and potentially more)

Inherent Challenges—Data Collection Diversity of Symptom type and severity Uterine anatomy Patient goals Hysterectomy still most common procedure (50%), followed by myomectomy (25-30%) Lower numbers of alternatives Substantial differences in patient characteristics by procedure type Limited time between identification of eligible patients and treatment for introduction, consent, collection of baseline PRO data Office-based treatments (ablation, IUD, ultimately drugs) even harder to identify

Inherent Challenges—Data Collection Younger, more mobile patient population Very few Medicare patients (and those unique in some way) State-based all payer claims limited if patient moves out of state Lack of standardization of Symptom description (validated disease-specific QoL available but not widely used in clinical practice) Uterine anatomy (FIGO recommendations in progress) Procedure description/details Devices No analogy to mesh, sterilization in terms of implants Device issues would involve failure or injury at time of procedure, or uncomplicated use in inappropriate patient (morcellation)

Inherent Challenges-Quality No equivalent of “30 day risk-adjusted mortality” Ongoing patient concerns about counseling about all options, availability of all options Constrained by overall lack of comparative effectiveness data Consider “phased” development of registry for QA/QI Did clinician have all relevant information needed for decision making available and documented? Were all appropriate options discussed with patient? Did patient receive treatment from appropriate options? Were outcomes of treatment within expected range given patient characteristics?

Goals for “COMPARE 2.0” Continue collaboration with CRN, ACOG efforts at developing methods for facilitating provider data entry Continue development and enhancement of patient-centered data collection, particularly on reliably patient-reported outcomes over the long-term by incorporating tools for Use of registry data as personal fibroid medical history “How do I compare to other patients in the registry?” feedback Tools for involvement with educating other patients Incorporate medical therapies, both existing off-label uses and drugs currently in Phase II/III