FROM EVIDENCE SYNTHESIS TO NATIONAL GUIDELINES & POLICY

1. FROM EVIDENCE SYNTHESIS TO NATIONAL GUIDELINES & POLICY CHIPS Seminar April 4, 2019 Beth Devine, PhD, PharmD, MBA Professor, UW CHOICE Institute Slides courtesy of Marian McDonaugh, PharmD, Professor, OHSU

2. HOW EVIDENCE SYNTHESIS IS USED IN POLICYMAKING • AHRQ Effective Healthcare Program • US Preventive Services Task Force • Drug Effectiveness Review Project • Medicaid Evidence-based Decisions Project • VA Evidence-based Synthesis Program • Professional Society Clinical Guidelines • Other government agencies

4. AGENCY FOR HEALTHCARE RESEARCH AND QUALITY (AHRQ) Evidence-based Practice Centers (EPCs) • Created in 1997; 5-year cycles; now EPC VI • EPCs conduct evidence reports on clinical, behavioral, organization, and financing topics • EPCs conduct research on and produce guidance about methodology of systematic reviews and meta-analyses. • Competitive bid process to be an EPC • Recognizes methodological expertise, experience with evidence synthesis, and depth and breadth of research resources • Only these EPCs can compete to conduct evidence synthesis projects for AHRQ https://www.ahrq.gov/research/findings/evidence-based-reports/overview/index.html

5. CURRENT EPCs (EPC V): • Brown University, Center for Evidence-based Medicine, Providence, RI • Duke University, Durham, NC • ECRI – Penn Medicine Evidence-based Practice Center, PA • Johns Hopkins University, Baltimore, MD. • Kaiser Permanente Research Affiliates, Portland, OR • Mayo Clinic Evidence-based Practice Center, Rochester, MN • Pacific Northwest Evidence-based Practice Center - Oregon Health & Science University, Portland, OR • RTI International--University of North Carolina, Chapel Hill, NC • Southern California Evidence-based Practice Center-RAND, CA • University of Alberta, Edmonton, Alberta, Canada. • Minnesota Evidence-based Practice Center, Minneapolis, MN. • University of Connecticut Evidence-based Practice Center, Storrs, CT • Vanderbilt University Medical Center, Nashville, TN

6. AHRQ EPC REPORTS AND POLICYMAKING • Center for Medicaid and Medicare Services (CMS) • Medicare requests topics typically relevant to a specific policy decision Example: Hyperbaric Oxygen • National Institutes of Health • NIH requests topics, typically for planned Evidence Forums Example: Chronic Fatigue Syndrome • Others: US PSTF, Clinical Guidelines Committees

14. U.S. PREVENTIVE SERVICES TASK FORCE • An independent, non-governmental panel of experts in primary care and prevention that systematically reviews the evidence of effectiveness and develops recommendations for clinical preventive services. • A and B recommendations are currently mandated for insurance coverage under the Affordable Care Act. https://www.uspreventiveservicestaskforce.org/

15. U.S. PREVENTIVE SERVICES TASK FORCE • Makes recommendations on clinical preventive services to primary care clinicians. • Recommendations apply to adults and children with no signs or symptoms. • The USPSTF scope for clinical preventive services includes: • Screening tests • Counseling • Preventive medications

16. USPSTF MEMBERS • 16 volunteer members represent primary care including family medicine, internal medicine, nursing, obstetrics and gynecology, pediatrics, and behavioral medicine. • Serve 4-year terms. • Appointed by AHRQ Director with guidance from Chair & Vice Chairs. • Current members include deans, medical directors, chief health officers, practicing clinicians, and professors.

17. EVIDENCE-BASED PRACTICE CENTER (EPC) • EPCs are funded by AHRQ to conduct systematic reviews for the USPSTF. • The Pacific Northwest EPC at OHSU has worked with the USPSTF since 1998; UW more recently. • Current methods of the USPSTF were developed with the EPC during the initial years and are refined periodically.

18. TOPIC NOMINATION • Anyone can nominate a topic for the USPSTF to consider via its Web site. • The public may suggest a new preventive service topic or recommend reconsideration of an existing topic: • Availability of new evidence • Changes in the public health burden of the condition • Availability of new screening tests supported by new evidence

19. RESEARCH PLAN Draft Research Plan • Task Force members work with researchers from an Evidence-based Practice Center (EPC) to create a draft Research Plan that guides the recommendation process. Opportunity for Public Comment • The draft Research Plan is posted on the USPSTF Web site for public comment. Finalize Research Plan • The Task Force and EPC review all comments, address them as appropriate, and create a final Research Plan.

20. EVIDENCE REVIEW Draft Evidence Review • Using the final Research Plan, the research team independently gathers and reviews the available evidence and creates a draft Evidence Review. Opportunity for Public Comment • The draft Evidence Review and draft Recommendation Statement are posted on the USPSTF Web site for public comment. Finalize Evidence Review • The EPC reviews all comments on the draft Evidence Review, addresses them as appropriate, and creates a final Evidence Review.

21. MAGNITUDE OF BENEFIT VS QUALITY OF EVIDENCE • Quality of evidence considered separately from magnitude of benefits • Need to weigh all important benefits versus all important harms • Use of outcomes tables to summarize benefits and harms

22. QUALITY OF EVIDENCE • The USPSTF grades the quality of the overall evidence for a service on a 3-point scale

23. LEVELS OF CERTAINTY REGARDING NET BENEFIT • The USPSTF defines certainty as "likelihood that the USPSTF assessment of the net benefit of a preventive service is correct." • The net benefit is defined as benefit minus harm of the preventive service as implemented in a general, primary care population. • The USPSTF assigns a certainty level based on the nature of the overall evidence available to assess the net benefit of a preventive service (High, Moderate, or Low).

24. LEVEL OF CERTAINTY REGARDING NET BENEFIT

25. USPSTF RECOMMENDATION GRID

26. USPSTF GRADES

27. RECOMMENDATION STATEMENT Draft Recommendation Statement • The Task Force discusses the draft Evidence Review and the effectiveness of the service. Based on the discussion, the Task Force creates a draft Recommendation Statement. Opportunity for Public Comment • The draft Evidence Review and draft Recommendation Statement are posted simultaneously on the USPSTF Web site for public comment. Finalize Recommendation Statement • The Task Force discusses the final Evidence Review and any new evidence. The Task Force then reviews all comments on the draft Recommendation Statement, addresses them as appropriate, and creates a final Recommendation Statement.

28. ESTIMATED YIELD OF SCREENING FOR HCV IN 1000 AVERAGE RISK ADULTS

29. TASK FORCE RECOMMENDATION ON HCV SCREENING USPSTF Recommendation (Grade B): • The USPSTF recommends screening for hepatitis C virus (HCV) infection in persons at high risk for infection. • The USPSTF also recommends offering 1-time screening for HCV infection to adults born between 1945 and 1965. • The USPSTF recommends the service. There is high certainty that the net benefit is moderate or there is moderate certainty that the net benefit is moderate to substantial.

30. ESTIMATED YIELD OF SCREENING 10000 MEN 55-69 YO FOR PROSTATE CANCER

31. TASK FORCE RECOMMENDATION ON SCREENING FOR PROSTATE CANCER USPSTF Recommendation (Grade D): • The U.S. Preventive Services Task Force (USPSTF) recommends against prostate-specific antigen (PSA)-based screening for prostate cancer. • The USPSTF recommends against the service. There is moderate or high certainty that the service has no net benefit or that the harms outweigh the benefits

36. DRUG EFFECTIVENESS REVIEW PROJECT (DERP) Self-governing collaboration of state Medicaid agencies that: • Pool funds to obtain and synthesize global evidence on the comparative effectiveness, safety, and effects of drugs. • Support policy makers in using the evidence to inform policy in local decision making. • Preferred Drug lists • Prior authorization criteria https://www.ohsu.edu/evidence-based-practice-center/derp-reports

37. DERP ORGANIZATIONS COVER THE MAP

38. DRUG EFFECTIVENESS REVIEW PROJECT (DERP) • DERP GOVERNANCE • Monthly governance meeting by conference call • Weekly written communication by e-mail (Friday Updates) • Biannual face-to-face conferences • Participant website for posting key questions, drafts, governance materials

39. DRUG EFFECTIVENESS REVIEW PROJECT (DERP) • MEDICAID PARTICIPANTS INVOLVEMENT IN REPORT DEVELOPMENT • Topic and update selection • Determination of key questions: • Indications, Interventions & Outcomes • Recommendation of clinical experts • Review of draft report • Review and approval of final report

40. DERP TOPIC AREAS (> 130 REPORTS)

41. USING EVIDENCE:STATE MEDICAID AGENCIES PDLS • Preferred Drug List (PDL) determinations • Independent Pharmacy & Therapeutics Committees review evidence in making recommendations • No drug is excluded, but some are preferred • Cost is considered separately from clinical evidence • Public testimony allowed during committee meetings

42. USE OF EVIDENCE USE BY MEDICAID PARTICIPANTS • States Differ in how they use DERP reports in their PDL process • Most states use DERP reports in addition to other sources • E.g. Pharmacy Benefit Managers (PBM) services • Others use DERP reports almost exclusively • E.g. Washington legislatively mandated to use only DERP reports

43. CHALLENGES IN USING EVIDENCE:STATE MEDICAID AGENCIES PDLS • States differ in what drugs can be considered based on local legislation • E.g. Mental health drugs are exempt from PDL process in many states • States differ in how they use evidence in these situations • Developing criteria for prior authorization • Clinician education initiatives

44. CHALLENGES IN DEVELOPING REVIEW SCOPE: SELECTION OF INCLUSION CRITERIA • Drugs to include in disease-state reviews • States differ on view of products with off-label use • E.g. modafinil for ADHD • States differ in interest in evidence on drugs with limited prescribing • E.g. growth hormone for fibromyalgia • Outcome measures to review • Some variation in view of intermediate or surrogate outcomes • E.g. LDL for statins, radiographic evidence for targeted immune modulators

45. CHALLENGES IN USING EVIDENCE:P&T COMMITTEES AND DERP • Challenges related directly to Comparative Evidence Reviews • Evidence is often complicated, nuanced • Users request high level summaries, easy to read, shorter reports • Rigorous review takes time and $ • Users request faster delivery • Value for money is an issue

46. DISSEMINATING DERP EVIDENCE • Several states directly interact with DERP report authors • Presentation of findings by author to Pharmacy & Therapeutics Committees • Q&A with committee members, respond to public testimony when requested. • Arkansas, Idaho, Washington, New York, Oregon

47. RECENT DEVELOPMENTS IN DERP: WHAT THE STATES NEED NOW • Struggling with high cost drugs, rapid development of new drugs, new evidence • Example: Hepatitis C drugs • Need evaluation of evidence prior to drug approval in some cases • Unpublished evidence • Examples: Hepatitis C and PCSK9 Inhibitor drugs

50. MEDICAID EVIDENCE-BASED DECISIONS PROJECT (MED PROJECT) • Currently eleven state Medicaid collaborative • Started in 2006 • Use evidence to inform benefit and coverage decisions • Over 100 reports produced