A Multi-Stakeholder Approach to Designing Studies of Emerging Medical Technologies

1. A Multi-Stakeholder Approach to Designing Studies of EmergingMedical Technologies Donna M. Schaffer Promoting Evidence-Based Medicine Session AHIP Medical Leadership Forum October 23, 2007 Phoenix, AZ

2. Summary of Presentation • a new model for designing studies of emerging medical technologies • Center for Medical Technology Policy (CMTP) • CMTP in the continuum of evidence generation and technology assessment • CMTP – facilitated research projects

3. Evidence Development sponsored by manufacturers and NIH FDA Regulatory Approval Coverage and Payment Decisions Studies designed to promote market penetration OR to advance science No review of medical and surgical procedures Safety and efficacy studies on drugs and devices conducted in limited trial populations No mechanism to bridge evidence gaps Limited ability to deny coverage for established (non-EB) technologies Current Research Model

4. Results In • Gaps in: • Evidence • Standards • “Real World” research methods • Outdated models for funding research in emerging technologies • Inappropriate care for patients • Underused, overused and misused health care technologies and treatments

5. Rationale for Collective Action • Insufficient resources and incentives • Conflict of interest • Too costly for individual private payers • Public sector research subject to statutory and regulatory constraints • MedPAC recommendation to Congress

6. CMTP’s New Model • “Decision-based evidence-making” Collaboratively-designed prospective studies to fill gaps identified by systematic reviews • Multi-stakeholder workgroups Patients, clinicians, payers, industry, regulators, and researchers at the same table • Comparative effectiveness discussion Strong, broad relationships with key organizations in the national dialogue

7. Where CMTP Fits: Evidence Generation and Technology Assessment 1. Systematic review of existing evidence 2. Identify critical gaps in evidence 3. Establish priorities for evidence development 4. Convene stakeholders to design studies 5. Identify funding mechanisms CMTP 10. Coverage and payment decisions 9. Create clinical guidelines based on evidence 8. Model cost effectiveness 7. Dissemination of evidence 6. Oversee study implementation Adapted from Steve Pearson Building Blocks 9

8. What CMTP Does… Products and Functions Priorities for Evidence Development-Research Agenda Identify & Prioritize Questions Guidance Documents Develop Condition-Specific Evidence Standards CMTP-Facilitated Research Develop Protocols, Secure Funding, & Implement Studies Mission-Critical Support Activities

9. CMTP-Facilitated Research2006-2007 Pilot Projects Diagnostic Test: • Cardiac CT Angiography (64-slice CT) Therapeutic Intervention: • Radiation Therapy for Prostate Cancer Genetic Application: • Gene Expression Profile Tests for Early Stage Breast Cancer

10. Aetna Kaiser Permanente UnitedHealth MN Medicaid GE Healthcare Siemens Medical Phillips Medical Toshiba AHA ACC FDA AHRQ ACRIN University researchers Patient representative Effectiveness of Cardiac CT Angiography Versus Usual Care in the Management of Coronary Artery Disease A Randomized Controlled TrialWorkgroup Members:

11. Aetna ASTRO Blue Shield California Camino Medical Group Siemens Medical Varian Medical Systems Wellpoint AHRQ CET Cancer Center Fred Hutchinson CRC ICER NCI MD Anderson CRC University Researchers Patient Representative Effectiveness of Radiation Therapy in the Treatment of Prostate CancerA Prospective Cohort Study Comparing the Side Effect Profiles of IMRT, Brachytherapy, and Proton Beam Workgroup Members:

12. Kaiser Permanente Independence Blue Cross National Heritage Insurance Company Genomic Health Agendia Foundation for Informed Decision-Making AHRQ Blue Cross Blue Shield Association TEC University Researchers Gene Expression Profile Tests for Early Stage Breast Cancer(Oncotype DX and Mammaprint)Study Question(s) in ProgressWorkgroup Members:

13. Lessons from the Pilot Studies: Multi-Stakeholder Workgroups • Charter • Clearly define the role of the multi-stakeholder members • Priorities for Evidence Development • Separate the selection of study topic from protocol development • Early Engagement • Convene the workgroup during product development / launch

14. Lessons from the Pilot Studies: Criteria for Study Selection • Promising but unproven clinical indication • Addresses evidence needs of payers, clinicians, patients • Can be conducted on abbreviated timeline • Sample size estimates achievable (< 5,000)

15. Lessons from the Pilot Studies: Criteria for Study Selection (con’t) • Funding level can be secured from non-governmental sources • Employs “real world” design (appropriate comparisons, limited restrictions, meaningful outcomes) • Unlikely to receive funding from traditional grantor agencies or product developers • Reasonable expectation of conclusive results

16. A new model of “private sector coverage with evidence development” (CED) Balancing methods of “real world” design against the gold standard (RCT) Transitioning from study protocol design to funding and implementation of studies Informing the policy debate on comparative effectiveness research Future Challenges & Opportunities

17. For more information: www.cmtpnet.org Donna M. Schaffer Center for Medical Technology Policy 524 Second Street San Francisco, CA 94107 415-537-6971