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Comparative Effectiveness Research:: Threat or Opportunity

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Edward Berger
Edward Berger

Comparative effectiveness research (CER) aims to compare different treatment options for health conditions. While controversial, CER has long been conducted by government agencies and is integral to regulatory approval processes. Recent healthcare reforms have significantly increased funding for CER through the Patient-Centered Outcomes Research Institute and require dissemination of findings. However, limitations remain on how findings can directly influence coverage and clinical practice may be slow to change. If conducted rigorously and findings are well-communicated, CER has potential to improve health outcomes and lower costs over time by informing treatment choices.

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COMPARATIVE EFFECTIVENESS RESEARCH:Threat or Opportunity?September 22, 2010Edward E. Berger, Ph.D.Larchmont Strategic Advisors
DEFINITION (1)Comparative Effectiveness Research (CER) is:the conduct and synthesis of systematic research comparing different interventions and strategies to prevent, diagnose, treat and monitor health conditions* *Federal Coordinating Council for Comparative Effectiveness Research2
DEFINITION (2)3 an analysis of comparative effectiveness is simply a rigorous evaluation of the impact of different options that are available for treating a given medical condition for a particular set of patients.** Congressional Budget Office
CER IS NOTHING NEWNational Center for Healthcare TechnologyWithin DHHS 1978 1981Several major studies + 75 coverage recommendationsOffice of Technology AssessmentAdvisory agency to Congress 1979 1995Agency for Healthcare Research & QualityWithin DHHS 1989 present300 staff members, $300 mm annual budget4
CER IS INGRAINED IN REGULATORY PROCESSFDA review routinely demands controlsOptimal medical therapyEstablished alternative devices/drugsExample: LVAD for destination therapyCMS Coverage Analysis Group utilizes CER principles in decision makingNETT study of Lung Volume Reduction SurgeryDaily hemodialysis study Focus on inclusion of > age 65 patient data 5
CER CONTROVERSY IS LONG-ESTABLISHED The justification for most medical practices used in the United States today rests on the experience and expertise of clinicians and patients rather than on objective evidence that these practices can measurably improve peoples health. Compiling objective evidence is considered by somehighly controversial, because the evidence might be applied in ways that would limit individuals choices of medical treatments.** Office of Technology Assessment, 19946
PERCEIVED CER THREAT HAS SEVERAL ELEMENTSThe entry wedge for Cost Effectiveness AnalysisMedicare effort (1989) to back-door limited CEA principlesAgencies will not interpret findings properly and subtly in making policyFear of blanket either-or decisionsGovernment role will lead to restriction on physician practice of medicine7
RECENT DEVELOPMENTS INTENSIFY INTERESTNeed to control healthcare system costsDocumentation of startling differences in geographic area utilization ratesIncreasing patient involvement in therapy choiceWeb portalsPersonalized medicine revolutionTherapies affect sub-populations differently8
CER COMMITMENT IN 2009 STIMULUS Federal Coordinating Council for Comparative Effectiveness Research$400 million allocated to DHHS for CERResearchHuman and scientific capitalData infrastructureTranslation and adoptionMandated IOM review of initial priorities9
IOM RECOMMENDATION: TOP CER PRIORITY AREASHealth Care Delivery SystemsRacial and Ethnic DisparitiesCardiovascular and Peripheral Vascular DiseaseGeriatricsFunctional Limitations and DisabilitiesNeurologic DisordersPsychiatric DisordersPediatrics10
CER PROVISIONS OF 2010 HEALTH REFORM LAWCreated PCORI (Patient Centered Outcomes Research Institute)Commissions and funds CER studiesAHRQ, with NIH assistance, responsible for dissemination of findingsWithin 90 days of receiptTo all key stakeholdersWith specification of relevant populations, research methods, and limitations of findings11
FOCUS ON DISSEMINATION OF FINDINGSSignificant role for AHRQs Office of Communication and Knowledge TransferDirect outreach to promote incorporation of CER findings into clinical decision support toolsCreation of informational tools to support dissemination to physicians, patients, payors and policy makersDevelop mechanisms for stakeholder feedback on utilization and value of studies12
LAW RESTRICTS CMS USE OF CER FINDINGSCMS is prohibited from using CER findings in making coverage policy determinationsPolitical sop to industry?Defensive of physician prerogatives and patient right to choose?Legitimate questions about methodology and limitations of findings?Parallels long-standing prohibition on consideration of cost13
A MINDS AND HEARTS STRATEGY FOR IMPACT14Central development of research priorities, agenda, methodological principlesFunding from the national levelMandated system-wide distribution of findingsPush information to stakeholdersReliance on voluntary incorporation of findings into clinical practices
PRIVATE INSURERS HAVE MORE FLEXIBILITYThey can directly incorporate CER findings into coverage policyCompetitive pressures impose limits in fee-for-service environmentCapitated models and integrated delivery systems are more fertile groundMayo, Geisinger, VA already have learning systems in placeClinical experience, reflected via HIT, informs evolving treatment protocols15
CHANGING CLINICAL PRACTICE IS HARD TO DOFragmentation of systemTens of thousands of decision makersAlready too much information to evaluate and incorporateRapid change and instability of findingsShortage of time and methodological expertiseChallenge to unlearn what you learned in medical school16
ENABLING CER IMPACTSystem reforms to control cost and enhance quality also enable CERSophisticated HIT and decision support capabilities of integrated delivery systemsShift in financial incentives accompanying full or partial capitation modelsIncreasing importance of widely-promulgated protocols approved by specialty societies Internet-enabled explosion in consumer role in clinical decision making 17
LIMITING FACTORSMethodological uncertaintiesContinuing distrust of meta-analysesPace of systemic move toward integrationGenerational resistance from physiciansFear of shift from information resource to mandate and/or denial of accessRisk of turf wars among stakeholder groups18
Coordination of PCORI agenda with privately sponsored CEREstablishment and promulgation of widely recognized methodological standards for conduct and evaluation of CER studies Most effective methods for communicating results to diverse stakeholder groupsWith useful reflection of strength and stability of findings19PRACTICAL ISSUES TO ADDRESS
CER WILL -> BETTER CHOICESThis is a threat toIneffective therapies/tests/technologiesOver-utilized therapies/tests/technologiesThose with mismatch between demonstrated utility and current utilizationUnder-researched and unproven Positive impact on health outcomes and healthcare resource utilizationSlowly accretive over extended timeframe20
OPPORTUNITIES FOR SOMEIndependent support forTherapeutics and diagnostics that address designated high priority areasThose with well-defined target treatment sub-groupsThose with unequivocally positive support from empirical research data Currently underutilized treatment options21
CER AFFECTS THE COMPETITIVE LANDSCAPELevels the playing field for newer and smaller companiesPublic funding for studies that are hard for young companies to financeData banks and registries facilitate data mining for strategy and product development3rd party research, widely disseminated, can help counter sales/marketing muscle of larger and well-established competitors22
CER AND PERSONALIZED MEDICINECER facilitates the right treatment for the right patient at the right timeCER reinforces the companion diagnostics non-blockbuster business modelSegment patients by likelihood of responseUse segmentation to structure qualifying research and to lower development costsTarget utilization to achieve higher success rates and better command of smaller market23
FINAL THOUGHTSCER does have the potential to improve outcomes and resource utilizationThe losers from a well-designed and managed CER initiative deserve to lose Resource differentials between competitors will have diminished effectDevice and diagnostics developers will need to join Pharma in adapting to a new business and product development model24
Thank YouEdward E. Berger, Ph.D.Larchmont Strategic Advisors2400 Beacon St., #203Chestnut Hill, MA 02467Tel: 617-645-8452Email: eberger@larchmontstrategic.com25

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Comparative Effectiveness Research:: Threat or Opportunity

  • 1. COMPARATIVE EFFECTIVENESS RESEARCH:Threat or Opportunity?September 22, 2010Edward E. Berger, Ph.D.Larchmont Strategic Advisors
  • 2. DEFINITION (1)Comparative Effectiveness Research (CER) is:the conduct and synthesis of systematic research comparing different interventions and strategies to prevent, diagnose, treat and monitor health conditions* *Federal Coordinating Council for Comparative Effectiveness Research2
  • 3. DEFINITION (2)3 an analysis of comparative effectiveness is simply a rigorous evaluation of the impact of different options that are available for treating a given medical condition for a particular set of patients.** Congressional Budget Office
  • 4. CER IS NOTHING NEWNational Center for Healthcare TechnologyWithin DHHS 1978 1981Several major studies + 75 coverage recommendationsOffice of Technology AssessmentAdvisory agency to Congress 1979 1995Agency for Healthcare Research & QualityWithin DHHS 1989 present300 staff members, $300 mm annual budget4
  • 5. CER IS INGRAINED IN REGULATORY PROCESSFDA review routinely demands controlsOptimal medical therapyEstablished alternative devices/drugsExample: LVAD for destination therapyCMS Coverage Analysis Group utilizes CER principles in decision makingNETT study of Lung Volume Reduction SurgeryDaily hemodialysis study Focus on inclusion of > age 65 patient data 5
  • 6. CER CONTROVERSY IS LONG-ESTABLISHED The justification for most medical practices used in the United States today rests on the experience and expertise of clinicians and patients rather than on objective evidence that these practices can measurably improve peoples health. Compiling objective evidence is considered by somehighly controversial, because the evidence might be applied in ways that would limit individuals choices of medical treatments.** Office of Technology Assessment, 19946
  • 7. PERCEIVED CER THREAT HAS SEVERAL ELEMENTSThe entry wedge for Cost Effectiveness AnalysisMedicare effort (1989) to back-door limited CEA principlesAgencies will not interpret findings properly and subtly in making policyFear of blanket either-or decisionsGovernment role will lead to restriction on physician practice of medicine7
  • 8. RECENT DEVELOPMENTS INTENSIFY INTERESTNeed to control healthcare system costsDocumentation of startling differences in geographic area utilization ratesIncreasing patient involvement in therapy choiceWeb portalsPersonalized medicine revolutionTherapies affect sub-populations differently8
  • 9. CER COMMITMENT IN 2009 STIMULUS Federal Coordinating Council for Comparative Effectiveness Research$400 million allocated to DHHS for CERResearchHuman and scientific capitalData infrastructureTranslation and adoptionMandated IOM review of initial priorities9
  • 10. IOM RECOMMENDATION: TOP CER PRIORITY AREASHealth Care Delivery SystemsRacial and Ethnic DisparitiesCardiovascular and Peripheral Vascular DiseaseGeriatricsFunctional Limitations and DisabilitiesNeurologic DisordersPsychiatric DisordersPediatrics10
  • 11. CER PROVISIONS OF 2010 HEALTH REFORM LAWCreated PCORI (Patient Centered Outcomes Research Institute)Commissions and funds CER studiesAHRQ, with NIH assistance, responsible for dissemination of findingsWithin 90 days of receiptTo all key stakeholdersWith specification of relevant populations, research methods, and limitations of findings11
  • 12. FOCUS ON DISSEMINATION OF FINDINGSSignificant role for AHRQs Office of Communication and Knowledge TransferDirect outreach to promote incorporation of CER findings into clinical decision support toolsCreation of informational tools to support dissemination to physicians, patients, payors and policy makersDevelop mechanisms for stakeholder feedback on utilization and value of studies12
  • 13. LAW RESTRICTS CMS USE OF CER FINDINGSCMS is prohibited from using CER findings in making coverage policy determinationsPolitical sop to industry?Defensive of physician prerogatives and patient right to choose?Legitimate questions about methodology and limitations of findings?Parallels long-standing prohibition on consideration of cost13
  • 14. A MINDS AND HEARTS STRATEGY FOR IMPACT14Central development of research priorities, agenda, methodological principlesFunding from the national levelMandated system-wide distribution of findingsPush information to stakeholdersReliance on voluntary incorporation of findings into clinical practices
  • 15. PRIVATE INSURERS HAVE MORE FLEXIBILITYThey can directly incorporate CER findings into coverage policyCompetitive pressures impose limits in fee-for-service environmentCapitated models and integrated delivery systems are more fertile groundMayo, Geisinger, VA already have learning systems in placeClinical experience, reflected via HIT, informs evolving treatment protocols15
  • 16. CHANGING CLINICAL PRACTICE IS HARD TO DOFragmentation of systemTens of thousands of decision makersAlready too much information to evaluate and incorporateRapid change and instability of findingsShortage of time and methodological expertiseChallenge to unlearn what you learned in medical school16
  • 17. ENABLING CER IMPACTSystem reforms to control cost and enhance quality also enable CERSophisticated HIT and decision support capabilities of integrated delivery systemsShift in financial incentives accompanying full or partial capitation modelsIncreasing importance of widely-promulgated protocols approved by specialty societies Internet-enabled explosion in consumer role in clinical decision making 17
  • 18. LIMITING FACTORSMethodological uncertaintiesContinuing distrust of meta-analysesPace of systemic move toward integrationGenerational resistance from physiciansFear of shift from information resource to mandate and/or denial of accessRisk of turf wars among stakeholder groups18
  • 19. Coordination of PCORI agenda with privately sponsored CEREstablishment and promulgation of widely recognized methodological standards for conduct and evaluation of CER studies Most effective methods for communicating results to diverse stakeholder groupsWith useful reflection of strength and stability of findings19PRACTICAL ISSUES TO ADDRESS
  • 20. CER WILL -> BETTER CHOICESThis is a threat toIneffective therapies/tests/technologiesOver-utilized therapies/tests/technologiesThose with mismatch between demonstrated utility and current utilizationUnder-researched and unproven Positive impact on health outcomes and healthcare resource utilizationSlowly accretive over extended timeframe20
  • 21. OPPORTUNITIES FOR SOMEIndependent support forTherapeutics and diagnostics that address designated high priority areasThose with well-defined target treatment sub-groupsThose with unequivocally positive support from empirical research data Currently underutilized treatment options21
  • 22. CER AFFECTS THE COMPETITIVE LANDSCAPELevels the playing field for newer and smaller companiesPublic funding for studies that are hard for young companies to financeData banks and registries facilitate data mining for strategy and product development3rd party research, widely disseminated, can help counter sales/marketing muscle of larger and well-established competitors22
  • 23. CER AND PERSONALIZED MEDICINECER facilitates the right treatment for the right patient at the right timeCER reinforces the companion diagnostics non-blockbuster business modelSegment patients by likelihood of responseUse segmentation to structure qualifying research and to lower development costsTarget utilization to achieve higher success rates and better command of smaller market23
  • 24. FINAL THOUGHTSCER does have the potential to improve outcomes and resource utilizationThe losers from a well-designed and managed CER initiative deserve to lose Resource differentials between competitors will have diminished effectDevice and diagnostics developers will need to join Pharma in adapting to a new business and product development model24
  • 25. Thank YouEdward E. Berger, Ph.D.Larchmont Strategic Advisors2400 Beacon St., #203Chestnut Hill, MA 02467Tel: 617-645-8452Email: eberger@larchmontstrategic.com25

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