The Role of Comparative Effectiveness in Health Reform

Transcription

1 The Role of Comparative Effectiveness in Health Reform Vivian H. Coates, Vice President ECRI Institute National Congress 0n Health Reform 23 rd September, 2008 Washington, DC ECRI Institute

2 What ECRI does: Evidence-based Practice Center since 1997 AHRQ National Guideline Clearinghouse, National Quality Measures Clearinghouse Conduct health technology assessment and systematic reviews, including comparative effectiveness reviews Methodology research Support for clinical practice guideline development and evidence-based policy making (coverage decisions, national and state policy decisions)

3 ECRI Organizational Experience 40 years experience in laboratory evaluation of healthcare technology, devices and equipment 20 years experience in HTA and systematic review of drugs, devices, procedures and other interventions and practices HTA clients include: Hospitals, health plans, federal and state agencies, drug and device manufacturers worldwide World Health Organization Collaborating Center Nonprofit heath services research agency 300 full time staff in 4 offices (Phila, London, Dubai, KL)

4 Conflict-of-Interest Rules Neither ECRI nor any of its staff has a financial interest in the sale of any medical technology. ECRI and its staff accept no royalties, gifts, finder s fees, or commissions from the medical device or pharmaceutical industries and are not permitted to own stock in or undertake consulting work for such industries.

5 What is Comparative Effectiveness? No standard definition exists Multiple methodologies Variable quality and sophistication of comparative effective research Expensive, resource-intensive But: essential for determining what works best in healthcare ECRI Institute

6 What is Comparative Effectiveness? The comparison of one diagnostic or therapeutic intervention to one or more others Primary comparative effectiveness research: generation of clinical evidence on the outcomes of one intervention in comparison to one or more others (head-to-head trials), prospective observational studies Secondary comparative effectiveness research involves analyzing the results of multiple primary studies to reach conclusions to be drawn. Secondary CER can also utilize analyses of outcomes or claims databases and modeling (Tunis, 2008)

7 Can observational studies be useful in CER? Yes because They include real-world patients, higher risk patients, patients with comorbidities Have external validity--results more generalizable to routine clinical practice

8 Can observational studies be useful? Yes.when Evaluating infrequent treatment-related adverse events/side effects Large clinical registries may be best source for such data e.g. Stent thrombosis in patients with drug-eluting stents or drug-related MI

9 Secondary CER typically includes: Direct analysis of the results of multiple head-to-head studies Indirect analysis, in which the diagnostic or treatment options have not been directly compared to each other in a trial Conclusions utilize inferences based on the relative effect of each intervention to a specific comparison, often a placebo or sham intervention (eg, multiple drug or device trials each compared to a placebo or active comparator). Combinations of direct and indirect analysis ECRI Institute

10 Secondary Comparative Effectiveness Research: Where to find it AHRQ EPC Evidence Reports Health technology assessments (HTA) from many sources (eg, ECRI) Systematic reviews, including meta-analyses Published analyses of outcomes databases Peer-reviewed journal articles, proprietary reports and monographs, government reports (gray literature) Can be hard to find no single place to look ECRI Institute

11 ECRI uses best available evidence approach in CE Pragmatic, used when ideal studies desired (ie, best possible evidence) are not available Uses highest available level of published evidence with appropriate caveats, discussion about reliability Presents the current state of knowledge, even if it is inconclusive

12 Strength of Evidence Domains: Quality Robustness Quantity Size of Effect Consistency

13 Strength of Evidence Strength of evidence characterizes a body of evidence, NOT individual studies A system for rating the stability and strength of medical evidence Jonathan R Treadwell 1, Stephen J Tregear 1, James T Reston 1 and Charles M Turkelson 2 1 ECRI Evidence-Based Practice Center and Health Technology Assessment Group, 5200 Butler Pike, Plymouth Meeting, Pennsylvania 19462, USA 2 American Academy of Orthopaedic Surgeons, 6300 North River Road, Rosemont, Illinois 60018, USA BMC Medical Research Methodology 2006, 6:52 Published 19 October 2006 doi: /

14 Healthcare Reform: The Current Environment Health technology changing faster than clinical practice Pressure for information on the value of new approaches Pressure on providers to deliver care cost effectively Pressure on manufacturers to get reimbursement for new products and services Pressure from govt./payers for less costly services Pressure from reformers to define a benefits packaged that can be offered to all, including uninsured and underinsured

15 Implications of Improper Decision Making (No CER): Inappropriate practices Diffusion of ineffective health technology Excess technology Unnecessary technology Distorted consumer expectations Patient harm Increased costs without improved quality diminished value

16 Why Payers and Providers Need CER for Health Reform: To help control wide variations in practices among clinicians locally, regionally and nationally To control costs and better match resources with needs To make informed purchase decisions about health technology To reduce duplication of services To ensure access to what works best

17 Current Challenges for Assessing Comparative Effectiveness of New Technologies: Hospitals/clinicians offering more and more technologybased services Manufacturers developing more and more products Choices are overwhelming, gaps in evidence huge Comparative effectiveness review/health technology assessment output never enough Large number of topics means that some will be evaluated in lesser depth How to support decision-making when the evidence isn t there?

18 Case Study: ECRI Comparative Effectiveness Review of Lumbar Artificial Disc vs Spinal Fusion ECRI Institute

19 Artificial Intervertebral Discs Insufficient evidence to establish the long-term safety and efficacy of lumbar artificial disc replacement (expected life: 20 years) 2 RCTs flawed (eg, failure to use intent-to-treat, only 2 year follow up)) Patients eligible for lumbar artificial disc replacement only a subset of patients who are candidates for spinal fusion surgery (ie, many contraindications) Revisions/replacement operations for patients with complications are more risky, costly, and debilitating than spinal fusion

20 Implications for Evidence-based Policy: For Charite and other artificial intervertebral discs, need to wait for the results of several ongoing trials (5 year follow up) Comparative effectiveness analysis limited by available data Artificial intervertebral discs may be eclipsed by a less invasive newer procedure: lumbar disc nucleus replacement (if the evidence materializes)

21 Challenges for Health Reform: Many of us already do CER there is demand and capability Need to support ramped-up output we salute Gail Wilensky! But strong countervailing forces and vested interests (eg, manufacturers of products already on the market have no incentive to do head-to-head trials) To what extent will comparative effectiveness be encouraged as part of health reform? Is there really the will to provide what CER findings say should be offered? If not, can health reform be achieved in our society? ECRI Institute