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FROM METHODS TO POLICY

Past as prologue: how comparative effectiveness research became patient-centered outcomes research Eugene C Rich*

The debate over comparative effectiveness research (CER) in the USA has resulted in the coining of a new term – ‘patient-centered outcomes research (PCOR)’ – codified in the 2010 Affordable Care Act (ACA). In the ACA, the purpose of the Patient-Centered Outcomes Research Institute was described as assisting “…patients, clinicians, purchasers and policy-makers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored and managed through research and evidence synthesis that considers variations in patient subpopulations, and the dissemination of research findings with respect to the relative health outcomes, clinical effectiveness and appropriateness of the medical treatments, services and [other] items…” [1]. In 2012, the Institute defined PCOR through a process led by their Methodology Committee and informed by diverse stakeholder input. This definition begins with a statement of the ultimate purpose of the research: “PCOR helps people and their caregivers communicate and make informed healthcare decisions, allowing their voices to be heard in assessing the value of healthcare options”. It then goes on to illustrate this purpose by offering examples of the types of patients’ questions answered by this form of research [101]: ■■ “Given my personal characteristics, conditions and preferences, what should I expect will happen to me?” ■■

“What are my options and what are the potential benefits and harms of those options?”

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“What can I do to improve the outcomes that are most important to me?”

“How can clinicians and the care delivery systems they work in help me make the best decisions about my health and healthcare?” The reasons for this evolution of political discourse from CER to PCOR are grounded in debates that have occurred inside the Washington DC beltway over the 3 years leading up to the passage of the ACA. Certainly, the principle of research to better inform patient decision-makers was included in various definitions of CER developed prior to the ACA. In 2007, the Institute of Medicine’s Roundtable on Evidence-based Medicine emphasized the central focus of CER on improved outcomes for patients. The Roundtable’s report ‘Learning What Works Best’ noted: “…a core objective for the nation is achieving the best health outcome for every patient. This objective simply cannot be accomplished until we have better evidence on which to base healthcare decisions, as well as ■■

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“…be it termed patient-centered outcomes research or comparative effectiveness research … researchers must do a better job of finding clear answers to the tough questions that patients and their clinicians grapple with every day.”

*Center on Health Care Effectiveness, Mathematica Policy Research, 1100 First Street NE, Washington, DC 20002, USA Tel.: +1 202 250 3544 [email protected]

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FROM METHODS TO POLICY more effective application of the knowledge we have.” [2]. The Medicare Payment Advisory Commission (MedPAC), while charged with advising Congress on the policies relevant to the improving payment policy, focused on patients as a key stakeholder for the information to be generated by CER. In their June 2007 report calling for a publicly funded entity to produce comparative effectiveness information, MedPAC noted: “…there is not enough credible, empirically based information for healthcare providers and patients to make informed decisions about alternative services for diagnosing and treating the most common clinical conditions” [3]. Accordingly, Congressional policy work on CER emphasized that it should inform patient and clinician decision-makers. Legislation establishing a CER center passed the US House of Representatives in 2007 (but not the Senate). This legislation required that the information produced by this CER center be “clinically relevant to decisions made by clinicians and patients at the point of care…” [4]. This emphasis on research that answers real-world questions for patients and clinicians acknowledged the need to modify the wellestablished process of healthcare research in the USA. Various policy makers had noted that far too few studies provided clear answers regarding the proper role of innovations in typical clinical settings, resulting in systematic reviews of available evidence that were often not able to provide clear guidance on important clinical questions [5]. Prominent analyses observed that many authoritative clinical practice guidelines relied on weak evidence [6], and even widely applied quality metrics incorporated controversial assumptions regarding benefits of interventions in under-studied populations [7]. So there was little controversy over the concept that additional research was needed to answer important questions for patients and their caregivers and clinicians. Indeed, policy makers noted that the research market place was unlikely to produce as much information on comparative effectiveness as was needed by patient and clinician decisionmakers. In 2007, the Congressional Budget Office (CBO) provided a report on the rationale for public support of CER. As the agency charged with advising Congress on the fiscal impact of legislation, CBO provides dispassionate nonpartisan economic ana­lysis to inform

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many contentious political debates. In this report, CBO emphasized that “the private sector generally will not produce as much research on comparative effectiveness as society would value” [8]. The CBO report, however, also reflected the emerging debate over the role of costs and cost– effectiveness ana­lysis in CER. True to its responsibility as a steward of US fiscal wellbeing, the CBO had been emphasizing that the trajectory for healthcare cost growth was unsustainable [9]. Not surprisingly, its report on CER focused on the potential benefits for healthcare cost growth of producing more of this type of evidence. While CBO noted that “hard evidence is often unavailable about which treatments work best for which patients”, the report went on to note that the research should also help determine “whether the added benefits of more effective but more expensive services are sufficient to warrant their added costs…”. CBO thus described CER as providing “better information about the costs, risks and benefits of different treatment options” and suggested that cost–effectiveness ana­lysis and the calculation of benefits in terms of quality-adjusted life years might be appropriate work for this entity [8]. Although various professional groups, including the American College of Physicians, indicated that cost–effectiveness ana­lysis would provide useful information for clinicians and for patients [10], the assessment of costs became equated with rationing in political discourse [102]. This concern was exacerbated by the debates over the 2009 Recovery Act investments in CER, where an early draft of the Congressional rationale for CER focused on the potential benefits of this research in controlling healthcare costs [103]. In subsequent debates some opponents of CER began referring to it as ‘cost–effectiveness research’. Perhaps to underscore the ‘patientcentered’ aspect of this information, as well as to distance it from the conflation of ‘comparative effectiveness’ with ‘cost–effectiveness’, the US Senate found the phrase ‘patient-centered outcomes research’ less problematic. In 2009, it passed healthcare reform legislation whereby the CER provisions employed the term PCOR; meanwhile, the House version of health reform, building on the legislation from 2007 and the 2009 Recovery Act, continued with the term CER [11]. Exactly how the two bodies might have compromised on this terminology difference can

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FROM METHODS TO POLICY never be known. In early 2010, the Democrats lost the 60 votes needed to assure the supermajority required to pass a revised version of the ACA through the Senate. Accordingly, the previously passed Senate version of the ACA ultimately became law, and a new trust fund and a new Institute were established to support ‘patientcentered outcomes research’. Acknowledging the anxiety over the use of cost analyses, the ACA also prohibited cost–effectiveness ana­lysis: the PCOR Institute “shall not develop or employ a dollars-per-quality-adjusted life year (or similar measure that discounts the value of a life because of an individual’s disability) as a threshold to establish what type of healthcare is cost effective or recommended” [1]. This debate in the USA has certainly shaped the nature and scope of the work that can and will be supported by the PCOR Institute. The References 1

111th Congress of the United States. Patient Protection and Affordable Care Act. HR 3590, 1, 1–906 (2010).

2

Institute of Medicine. Learning what works best: the nations need for evidence on comparative effectiveness in health care. 1–80 (2007).

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debate has also highlighted the central role of the patient perspective in this scholarship, be it termed PCOR or CER. Clearly, healthcare researchers must do a better job of finding clear answers to the tough questions that patients and their clinicians grapple with every day. Fortunately, substantial new resources and infrastructure are now available in the USA to help investigators with this challenging work. Financial & competing interests disclosure The author has no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. No writing assistance was utilized in the production of this manuscript.

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Tricoci P, Allen JM, Kramer JM, Califf RM, Smith SC Jr. Scientific evidence underlying the ACC/AHA clinical practice guidelines. JAMA 301(8), 831–841 (2009).

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Hayward RA. Performance measurement in search of a path. N. Engl. J. Med. 356(9), 951–953 (2007).

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Medicare Payment Advisory Commission. Report to congress: promoting greater efficiency in medicare. 1(June), i–297 (2007).

Research on the comparative effectiveness of medical treatments: issues and options for an expanded federal role. Congressional Budget Office, Washington DC, USA, 1–48 (2007).

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Rep Dingell JD. Children’s Health and Medicare Protection Act. HR 3162(110) (2007).

Congress of the USA, Congressional Budget Office. The long-term outlook for health care spending. Washington DC, USA (2007).

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American College of Physicians. Information on cost–effectiveness: an essential product of a national comparative effectiveness program. Ann. Intern. Med. 148(12), 956–961 (2008).

Tunis SR, Carino TV, Williams RD 2nd, Bach PB. Federal initiatives to support rapid learning about new technologies. Health Aff. (Millwood) 26(2), w140–w149 (2007).

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Dingell J, Rangel C, Waxman H, Miller G, Stark P, Pallone F, Andrews R. America’s Affordable Choices Act of 2009. HR 3200(11th Congress; 1st Session), 1–2454 (2009).

■■ Websites 101 PCORI. Patient-centered outcomes research

2012. www.pcori.org/what-we-do/pcor/ (Accessed 17 September 2012) 102 Economic Scene – rhetoric of rationing health

care overlooks reality. www.nytimes.com/2009/06/17/business/ economy/17leonhardt.html?_r=1&emc=eta1 (Accessed 17 June 2009) 103 The Stimulus Bill: why the senate must fix

the health care provisions. www.heritage.org/Research/HealthCare/ wm2267.cfm (Accessed 5 February 2009)

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From methods to policy: past as prologue: how comparative effectiveness research became patient-centered outcomes research.

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