Core Outcome Measures in Effectiveness Trials

coreUF

General Information

Summary:
The goal of this project is to develop multi-stakeholder consensus on a core outcome set (COS) for symptomatic uterine fibroids that is patient-important, clinically meaningful to payers, and relevant to other health care decision-making entities. Adoption of the COS in pivotal and other late phase clinical trials, including post-market observational registries/studies, will ultimately result in research-generated evidence that is not only relevant to multiple stakeholders, but also comparable for assessing the value of various uterine fibroid interventions.

Uterine fibroids are the most common benign pelvic tumors in women. The estimated cumulative incidence is 80 percent in black women and 70 percent in white women by age 50. Symptomatic fibroids account for 25 to 50 percent of cases, causing heavy menstrual bleeding, pelvic pain, reproductive complications, and bulk symptoms. These symptoms can substantially affect quality of life and daily functioning. Uterine fibroids are the underlying cause of approximately one-third of all hysterectomies performed in the U.S. and 29 percent of gynecological hospitalizations among 15 to 54-year-old women. The burden of these symptoms is economically palpable, with annual direct costs of treatment estimated between $3.2 to $9.4 billion and indirect costs up to $34.4 billion.

A burgeoning movement in the treatment of symptomatic uterine fibroids seeks to provide women with effective alternatives that reduce morbidity and financial burden while improving quality of life. Though traditional management via hysterectomy remains the most commonly used technique, newer procedures are shifting toward minimally invasive, uterine-preserving strategies. At the same time, novel, orally-active therapeutics targeting progesterone pathways are being developed to provide long-term, etiologic care as an alternative to surgery.

As innovation increases the number of interventions potentially available to women who suffer from fibroids, it is imperative to capture the most relevant data needed for patients, providers, payers, and other stakeholders to make well-informed decisions in a post-regulatory context. Existing reviews have found the quality of evidence across studies to be very low to moderate, caused by heterogeneous outcomes and imprecise measurements. Substantial evidence gaps exist for both medical and surgical treatments, particularly pertaining to short- and long-term relative effectiveness and safety. Development of a COS will address these issues, increasing the ability to consistently gather the most patient-relevant outcomes and assure their consideration by regulators, payers, clinicians, and the patients they all serve.

Nature/Type of Intervention: Any, omitting hysterectomy

Contributors:
This project will be conducted through the Green Park Collaborative (GPC). GPC is a major initiative of the non-profit Center for Medical Technology Policy (CMTP) that aims to improve clinical research by cultivating transparency and mutual understanding on clinical evidence needs between drug and device developers, private and public payers, clinicians, researchers, regulators, and the patients that they all serve. The extensive expertise, networks, and partnerships that have been established within the GPC program provide the necessary underpinnings and create an ideal forum to successfully accomplish this work. GPC will partner with CARE About Fibroids, the White Dress Project, and Dr. Evan Myers of Duke University School of Medicine’s Department of Obstetrics and Gynecology and the Duke Clinical Research Institute. Dr. Myers also leads COMPARE-UF, a patient registry dedicated to capturing real treatment experiences and outcomes important to uterine fibroid patients.

Further Study Information

Current Stage:
Ongoing
Date:
Funding source(s):
TBC

Health Area

Disease Category
Gynaecology

Disease Name
Uterine fibroids

Target Population

Age Range
18 - 100

Sex
Female


Nature / type of Intervention
Any

Method(s)

Consensus meeting
Delphi process
Focus group(s)
Interview
Literature review
Semi structured discussion
Survey

Intervention: Any, omitting hysterectomy

Consensus on a core set will be developed through an iterative survey and engagement process called a modified Delphi survey. Through this process, stakeholders gain mutual understanding and develop a shared sense of the most important outcomes to include in all studies, especially those outcomes reflecting the greatest concerns of patients. The Delphi will be conducted using methods and practices based on the COMET Initiative (Core Outcome Measures in Effectiveness Trials) approach but adapted for robust multi-stakeholder participation and timelier project completion. While the current project scope is limited to developing consensus on what outcomes should be measured, a second phase of the project may be added to reach consensus on how the agreed-upon core outcomes should be measured.


Stakeholders Involved

Clinical experts
Consumers (patients)
Governmental agencies
Journal editors
Patient/ support group representatives
Pharmaceutical industry representatives
Policy makers
Regulatory agency representatives
Researchers
Service providers

Study Type

COS for clinical trials or clinical research

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