Research on the Comparative Management of Uterine Fibroid Disease

1. Research on the Comparative Management of Uterine Fibroid Disease Michelle B. Leavy, MPH Outcome DEcIDE Center September 20, 2011

2. Project Overview • Funded by AHRQ through the DEcIDE program. • Task Order Officer: Shilpa Amin • Completed by the Outcome DEcIDE Center, working collaboratively with the Center for Medical Technology Policy (CMTP). • Timeframe: Sept 2009 – Nov 2010.

3. Uterine Fibroid Disease • Most common gynecological condition among women, occurring most frequently among women ages 30 to 40. • Cumulative incidence approaches 70 percent among white women by age 50 and is even higher among African-American women. • May cause pain, heavy menstrual bleeding, and anemia; also associated with adverse reproductive outcomes.

4. Uterine Fibroid Disease Treatments • Multiple treatment options exist, but evidence on the comparative long-term effectiveness of treatment options is of poor quality. • 2007 AHRQ technology assessment concluded: “The current state of the literature does not permit definitive conclusions about benefit, harm, or relative costs to help guide women’s choices.”

5. Lack of Evidence to Inform Decisions • Lack of evidence on the effectiveness of therapies for uterine fibroids to inform decisions • This disconnect between the needs of decision-makers and the output of clinical research studies is not unique to uterine fibroids. • The disconnect has prompted interest in methods for determining the research priorities of decision-makers.

6. Research Priority-Setting • Goals of research priority-setting: • To ensure that limited research funds are used efficiently, and • To ensure that the end results of the research improve health care quality or shape clinical practice. • But, no clear best practice for priority-setting exists, and few models involving a wide range of stakeholders have been described.

7. Project Goals • To create a new model for stakeholder-driven priority-setting. • As recommended by the IOM Committee on Priority-Setting for CER, “stakeholder” is defined broadly to include patient and consumer representatives, as well as payers, clinicians, and others. • To test that model by developing a prioritized research agenda for uterine fibroid disease. • To develop a research protocol with stakeholder input for the highest priority question.

8. Priority-Setting Process

9. Project Team • Outcome DEcIDE Center • Center for Medical Technology Policy (CMTP) • AHRQ representatives • Clinical experts: • Dr. Evan Myers, Duke • Dr. Wanda Nicholson, UNC

10. Technical Working Group (TWG) • Goals of TWG: to refine and prioritize the initial list of research questions, and to guide discussion at the Stakeholder Committee meeting. • TWG members included: • 7 clinicians with experience in uterine fibroid research • 1 payer representative involved in a pilot study on the relative effectiveness of uterine artery embolization • 2 patient/consumer reps with in-depth knowledge of the disease and available treatment options

11. Stakeholder Committee • Goal of the Stakeholder Committee: prioritize the questions using a modified Delphi approach. • Stakeholder Committee members (37) included: • All members of the TWG • 13 clinician researchers • 5 public and private payers • 7 patient/consumer group representatives • 7 representatives of different federal agencies • 5 industry representatives (pharma and device)

12. TWG Review & Prioritization • TWG prioritized questions using a modified Delphi approach: • Round 1: questions prioritized and results discussed during a conference call. • Round 2: questions re-prioritized and discussed at in-person meeting. • Round 3: questions re-prioritized following meeting. • Final list of top 12 questions developed for Stakeholder Committee review.

13. Stakeholder Committee Prioritization • Stakeholders attended an in-person meeting. • Questions were discussed and scored against priority-setting criteria to come to consensus on the final ranked list. • Research designs for the highest-ranked questions were discussed.

14. Prioritized List of Questions

15. Top Questions by Stakeholder Group

16. Analysis of Prioritization Results • Some variation by stakeholder subtype: • Patient/consumer representatives prioritized the development of patient reported outcome measures, genetic studies, and natural history studies. • Payers prioritized dissemination, factors influencing treatment choice, and shared decision-making. • Largely similar results between TWG and Stakeholder Committee prioritizations.

17. Strengths of the Priority-Setting Model • Use of the TWGto narrow the initial list of questions. • Holding in-person meetings of both the TWG and Stakeholder Committee. • Inclusion of a wide and balanced array of perspectives in both the TWG and Stakeholder Committee.

18. Inclusion of Patients/Consumers • Patient/consumer representatives raised new, patient-focused questions that had not been identified in previous systematic reviews. The questions were added to the prioritized list. • Use of proactive approaches to engage patients and consumers resulted in active participation. • Inclusion of several representatives • Pre-meeting conference calls, background materials • Patient/consumer panel during Stakeholder meeting

19. Areas for Improvement • Refine the initial list of research questions to limit redundancy. • Improve the clarity and context of the research questions (one issue per question). • Reduce the amount of background materials provided to the TWG and Stakeholder Committee and provide hard copies.

20. Challenges in Stakeholder Engagement • Providing sufficient context to engage participants with varying levels of expertise. • Managing conflicts of interest. • Moderating discussions where stakeholders hold strong opinions. • Ensuring that all voices, particularly patient / consumer voices, are heard.

21. Conclusion • We attempted to use a transparent, inclusive process to connect researchers with the practical needs of patients and clinicians to develop a stakeholder-driven research agenda for uterine fibroid disease. • The project demonstrated the importance and feasibility of including patients and consumers in priority-setting activities. • The process for developing the agenda is reproducible and could be adapted for use in other clinical area.

22. For More Information • Read the complete white paper, available on AHRQ’s website at: http://www.effectivehealthcare.ahrq.gov/ehc/products/152/642/DEcIDE31_UterineFibroid_03-07-2011.pdf • Contact the project team: • Email: decide@outcome.com • Phone: 617-715-6955