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In 2003, the Medicare Prescription Drug, Improvement, and Modernization Act (MMA) was signed into law. Section 1013 of MMA authorized the Agency for Healthcare Research and Quality (AHRQ) to conduct and support research on comparative effectiveness and clinical effectiveness, and to disseminate the findings of that research to stakeholders. AHRQ organized the Effective Health Care (EHC) Program to meet the requirements of Section 1013. The legislation also required that the needs of Medicare, Medicaid, and the Children‘s Health Insurance Program drive AHRQ‘s research priorities. The primary goals of Section 1013 are to develop valid scientific evidence about the comparative effectiveness of different treatments and appropriate clinical approaches to difficult health care problems and to make this information easily accessible to decisionmakers. The conduct of new research in areas where existing scientific evidence is insufficient to inform health care decisions is an important step to meet the goals of Section 1013. As part of the EHC program and Section 1013 of MMA, the AHRQ Effective Healthcare , DEcIDE (Developing Evidence to Inform Decisions about Effectiveness) Program developed an evidence ?generation‘ focused task order designed to revisit the current state of evidence-based research gaps in a priority population condition area of gynecological health as identified in prior AHRQ evidence based-practice center systematic reviews. AHRQ has funded this project to develop a prioritized research agenda and protocol to study the clinical and/or comparative effectiveness of uterine fibroid disease diagnosis, management, care coordination, and treatment.
Uterine fibroids (leiomyomata) are the most common noncancerous tumors in women of childbearing age and the second most common reason women of reproductive age undergo surgery. Uterine fibroids, which result in more than 200,000 hysterectomies every year, have no known cause and limited treatment options. In the United States it is estimated that approximately 1 percent of women with employer-provided insurance have clinically significant uterine fibroids, and the direct costs associated with treating uterine fibroids are more than $1 billion annually. Treatment options for symptomatic uterine fibroids include watchful waiting; nonprocedural treatments (medical therapies) such as hormonal therapies, oral contraceptives, and nonsteroidal anti-inflammatory drugs; and a number of procedural treatments ranging from surgical or incisional such as hysterectomy or myomectomy, to nonsurgical (also called nonincisional or minimally invasive) such as uterine artery embolization and magnetic resonance image-guided focused ultrasound.
Despite the prevalence and possible complications of uterine fibroids, few published studies examining the effectiveness of treatment strategies exist. Little information is available on the long-term effects of procedural or medical therapy on fibroid disease symptoms, recurrence, and patient-reported outcomes. The currently available literature is insufficient to draw conclusions about the relative benefits, harms, or costs of the available choices, making it difficult for decisionmakers (e.g., patients, providers, payers, and others) to select appropriate treatments. New research is needed to address the questions facing decisionmakers. Additionally, a new collaborative model for developing a research plan is needed to ensure that the research meets the needs of decisionmakers. As recommended by the Institute of Medicine (IOM) in its report on comparative effectiveness research, this model should incorporate the perspectives of health care decisionmakers in the strategic planning, priority setting, and research plan development. (See Crossing the Quality Chasm, 2000, available at: http://www.iom.edu/Global/News%20Announcements/Crossing-the-Quality-Chasm-The-IOM- Health-Care-Quality-Initiative.aspx.)
The goals of the project are (1) to develop a consultative partnership with multiple stakeholders to identify clinical problems for which new research will inform treatment decisions for patients and providers (especially beneficiaries of the Medicare and Medicaid programs); (2) to identify, in partnership with stakeholders, clinically or policy-relevant research questions related to the clinical and/or comparative effectiveness of uterine fibroid treatment and management; (3) to design in partnership with stakeholders, researchers, and AHRQ a research protocol for a multicenter prospective study of the comparative effectiveness of diagnosis, management, care coordination, and/or treatment of uterine fibroids; (4) to conduct a preliminary analysis of existing data to inform the protocol development and assess the prioritized research questions identified by stakeholders; and (5) to disseminate, in collaboration with AHRQ, the findings of the project to stakeholder partners and other stakeholder groups.
The Outcome DEcIDE Center has conducted this project for and in collaboration with the AHRQ Effective Healthcare DEcIDE Program and with several individuals and organizations with particular areas of expertise. Drs. Evan Myers of Duke University and Wanda Nicholson of the University of North Carolina at Chapel Hill provided key clinical and epidemiological expertise and cochaired the Technical Working Group (TWG). The Center for Medical Technology Policy (CMTP) provided special expertise and leadership in stakeholder engagement and research prioritization; and JEN Associates performed background analyses on the data sets.
The purpose of this paper is to provide an interim report on the first three objectives of the project and outline the next steps for achieving the fourth objective. This paper is divided into three sections. The first section describes the processes used to engage stakeholders in a collaborative partnership and develop a stakeholder-driven research agenda. The second section presents the stakeholder-driven research agenda. The final section discusses the next steps in the project, including the creation of a research protocol and the use of data analyses to support the protocol development.
The stakeholder engagement and priority-setting phases of the project focused on developing a consultative partnership among multiple stakeholders, identifying key evidence gaps, and prioritizing research questions related to the treatment and management of uterine fibroids. As part of the stakeholder recruitment and engagement process, AHRQ provided guidance to the project team to strive to include a broad spectrum of stakeholders representing patients; consumers; biomedical, clinical translational science investigators; health plans; academic scientists; clinicians; industry; policy makers and state and federal partners all relevant and interested in advancing comparative uterine fibroid disease research. Having potential partners with the capability to disseminate research findings or to integrate directly into decision making and future research and translational activities was a core consideration. The project team, in consultation with the AHRQ EHC DEcIDE program staff, assembled two distinct but interrelated stakeholder groups for these tasks: Technical Working Group (TWG) subcommittee to provide technical expertise and prepare for the priority-setting meeting (eight members), and a diverse 34 member Stakeholder Committee for priority setting.
The primary goal of the TWG was to narrow the list of evidence gaps identified from systematic reviews to a manageable number to present to the Stakeholder Committee. The TWG also assisted the project team in translating the evidence gaps into research questions, identifying current or planned studies that might affect the relative importance of a specific question, and developing appropriate background materials for the Stakeholder Committee. The primary objective of the Stakeholder Committee was to identify the highest priority research questions for uterine fibroid disease treatment and management. The Committee also provided information on issues to consider in developing the research protocol.
The priority-setting process began with the TWG. The TWG reviewed the initial list of questions and scored each question using priority-setting criteria developed by the project team. Next, the TWG met to discuss, refine, and revise the questions. After the meeting, the TWG rescored the questions. The two prior AHRQ Evidence Reports informed the TWG‘s discussions and rankings.1,2 Using the data from the second scoring, the project team assembled a list of the top 12 research questions. The team developed general and question-specific background materials and distributed them to the Stakeholder Committee. Next, the Stakeholder Committee met to discuss and prioritize the list of 12 research questions. This meeting produced a final prioritized research agenda for uterine fibroid disease management and treatment.
The next step in the project was to develop a study protocol to address the highest priority research questions. To develop the protocol, the project team assessed study design options, defined the study objectives, determined the setting and participants, designed the data collection plan, and developed the statistical analysis plan. In selecting a study design option, the project team considered difficulties with randomization raised during the Stakeholder Committee meeting. The team also reviewed the findings from the preliminary data analyses conducted for this project. The data analyses produced descriptive profiles of patient populations with a uterine fibroid diagnosis, using data from the National Inpatient Sample, the State Ambulatory Surgery database, and the Medicaid program. While the data analyses provided valuable insights into variation in treatment patterns, they also highlighted the limitations of retrospective claims-based data analyses, including an inability to adjust data to reflect disease severity or other potential confounders and difficulty in determining initial treatment choices. Due to these factors, the study is planned as a prospective, observational cohort study that will not provide or recommend any treatment.
The prospective, observational study design offers several potential benefits, as well as some limitations. First, the observational nature of the design will allow the study to have broad inclusion criteria and minimal exclusion criteria. The study will also enroll patients from a diverse group of study sites, with the goal of producing results generalizable to a wide range of uterine fibroid disease patients who present to a variety of practice settings nationwide. By leaving treatment decisions up to the patient and provider (and not randomizing patients to a particular treatment protocol), the study may include a larger percentage of eligible patients. Unlike retrospective studies, which are limited by the availability of existing data, the prospective design will allow the study to collect detailed clinical data, as well as patient- reported outcomes. The study design also allows for long-term (5-year) followup with patients. Limitations of the study may include confounding by indication. However, efforts will be made to measure all known predictors of treatment decision and response to therapy and to include key predictive variables in multivariate analysis to minimize the effects of confounding by indication.
The objectives of the planned study are: (1) to evaluate the effectiveness of different treatment pathways in achieving relief from symptoms of uterine fibroids and overall quality of life, with a focus on (a) comparison of hysterectomy as first procedural treatment to uterus- sparing procedural treatments, and(b) comparison of all procedural treatments to nonprocedural (medical) treatments; and (2) to describe the pathways of management and treatment for symptomatic uterine fibroids of greater than 6 months‘ duration among women who have tried and "failed" at least one medical treatment. The draft study protocol will be circulated to the Stakeholder Committee and revised in consultation with the Committee and AHRQ.
Two additional data sources are currently being analyzed to further refine the protocol. These data sources were derived from the Medical Quality Improvement Consortium of General Electric Healthcare and the Fallon Clinic Community Healthplan, Fallon Clinic Research. Appendix 1, Table A.2.
These data sources were selected to provide more information on patterns of care, comorbidities, and predictors of patient care for uterine fibroid disease. Both data sources are equipped to provide a clinically rich, longitudinal picture of patient care. These data sources can also further examine patient treatment by age, race, body mass index, smoking status, and other important patient-level demographic and clinical variables. One source of data is a national electronic health record (EHR) system, with data from 15 million of patients in 43 states. This representative sample of the U.S. population will provide detailed patient profiles of a large sample of women with uterine fibroid disease. The second data source, while restricted to patients in the northeastern United States, provides an integrated picture (EHR data, claims data, pharmacy data) of patient care across multiple settings, thus ensuring that a comprehensive view of patient care is provided. In selecting these data sources, the project team hopes to overcome some of the limitations of the preliminary data analyses. In particular, these data sources are intended to provide a picture of treatment patterns over time in a more representative patient population. The two primary goals of these data analyses are: (1) to provide information on treatment patterns and progression of treatments over time to assess the feasibility of the proposed study design; and (2) to examine the types of data related to uterine fibroid disease that are routinely collected, in order to understand the data collection burden of the proposed study.
This project offered a unique opportunity to involve stakeholders in setting a research agenda for the treatment and management of uterine fibroids. The stakeholders, organized into the small Technical Working Group and larger Stakeholder Committee, provided guidance on developing the initial list of 64 research questions, revising and narrowing the list to 12 questions, and prioritizing the final questions. The stakeholders included patient, consumer, clinician, insurer, and Federal agency representatives. The inclusion of multiple stakeholder groups established a balanced approach to the selection of research priorities. Through transparent interaction with a broad and inclusive Stakeholder Committee, the project attempted to overcome a major flaw in the current clinical research enterprise, where researchers may become disconnected from the practical needs of patients and clinicians. The result of this process is a prioritized research agenda that should reflect the needs of those making decisions related to the treatment and management of uterine fibroids. This paper documents the strengths and limitations of this approach to priority setting and stakeholder engagement and may help to guide similar efforts in the future.