Letters
In Reply Drs Saugel and Reuter challenge the description of the OPTIMISE trial as a pragmatic trial. As applied to clinical trials, the term pragmatic has a particular meaning. Pragmatic trials are designed to evaluate the clinical effectiveness of a treatment in the context of routine clinical practice.1 This distinguishes them from explanatory trials, which are designed to evaluate the efficacy of a treatment under ideal conditions. However, rather than describing dichotomous alternatives, the 2 terms represent different ends of a qualitative continuum. 2 Investigators designing pragmatic trials are faced with the challenge of balancing competing pressures to maintain internal validity (efficacy of the intervention) and the feasibility of implementation into widespread clinical use, the latter ensuring external validity (generalizability of findings). The OPTIMISE intervention was developed from an evidence base consisting of several clinical trials and other supporting data, which were described in the article. We evaluated this intervention in an explanatory pilot trial, the findings of which indicate a clear mechanistic basis through improvements in systemic oxygen delivery, tissue microvascular flow, and tissue oxygenation.3 Our laboratory studies also suggest that dopexamine may have important anti-inflammatory actions.4 Given the evidence available when the trial was designed, the high levels of protocol adherence, and our findings, we are satisfied that we achieved an appropriate balance between efficacy and feasibility in the design of this trial. Drs Latif and Faraday raise questions relating to the systematic review incorporated into the OPTIMISE article. They correctly describe unit-of-analysis issues that were highlighted in our discussion. These relate to evaluation of the primary outcome measure (number of patients developing complications), which was not reported for the trial by Sandham et al.5 However, mortality data were reported for this trial and were included both in the previous Cochrane systematic review6 and in analyses of mortality reported in the main text of our article. The results of the systematic review in detail are presented in the supplementary material. Comments in the report of the Cochrane review regarding sensitivity to analytical technique 6 relate to the choice of methods of metaanalysis and not to the design or outcome measures of component trials. The findings of the OPTIMISE trial and our systematic review require careful interpretation. The contrasting views of these correspondents highlight that uncertainty remains regarding the benefits of this treatment. They also reinforce the importance of pragmatic studies, such as the OPTIMISE trial, and emphasize the need for much larger trials to address this and other key uncertainties in perioperative medicine. Rupert M. Pearse, MD Neil MacDonald, FRCA Michael P. W. Grocott, MD; for the OPTIMISE Study Group
Author Affiliations: Queen Mary University of London, London, England (Pearse, MacDonald); Integrative Physiology and Critical Illness Group, University of Southampton, Southampton, England (Grocott). Corresponding Author: Rupert M. Pearse, MD, Queen Mary University of London, Adult Critical Care Unit, Royal London Hospital, Whitechapel, London E1 1BB, England ([email protected]). Conflict of Interest Disclosures: The authors have completed and submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest. Dr Pearse reported receiving equipment loans from LiDCO Ltd; receiving a grant from the National Institute for Health Research; receiving personal fees from Edwards Lifesciences, Covidien Inc, and Massimo Inc; and being a named inventor on a lapsed patent application relating to the perioperative use of dopexamine. Dr Grocott reported receiving grant funding from Deltex Medical Ltd; and receiving fees for lecturing from Fresenius Kabi and Edwards Lifesciences. No other disclosures were reported. 1. Roland M, Torgerson DJ. What are pragmatic trials? BMJ. 1998;316(7127):285. 2. Thorpe KE, Zwarenstein M, Oxman AD, et al. A pragmatic-explanatory continuum indicator summary (PRECIS): a tool to help trial designers. J Clin Epidemiol. 2009;62(5):464-475. 3. Jhanji S, Vivian-Smith A, Lucena-Amaro S, Watson D, Hinds CJ, Pearse RM. Haemodynamic optimisation improves tissue microvascular flow and oxygenation after major surgery: a randomised controlled trial. Crit Care. 2010; 14(4):R151. 4. Bangash MN, Patel NS, Benetti E, et al. Dopexamine can attenuate the inflammatory response and protect against organ injury in the absence of significant effects on hemodynamics or regional microvascular flow. Crit Care. 2013;17(2):R57. 5. Sandham JD, Hull RD, Brant RF, et al; Canadian Critical Care Clinical Trials Group. A randomized, controlled trial of the use of pulmonary-artery catheters in high-risk surgical patients. N Engl J Med. 2003;348(1):5-14. 6. Grocott MP, Dushianthan A, Hamilton MA, Mythen MG, Harrison D, Rowan K; Optimisation Systematic Review Steering Group. Perioperative increase in global blood flow to explicit defined goals and outcomes following surgery. Cochrane Database Syst Rev. 2012;11:CD004082.
Primary Care Physician–Led Health Reform To the Editor Dr Mostashari and colleagues1 described a vexing problem—creating incentives that encourage efficiency, not volume. Invoking an ambiguous role model, the corporate CEO, they postulated that primary care physicians, who account for 5% of health care spending, should make the tough calls on the rest. Supporting their solution proves hard. To set primary care’s contribution equal to estimated avoidable costs, they ignored a key finding: there is an association between costs and primary care physician supply.2 To support physician-led over hospital-led accountable care organizations (ACOs), which must absorb volume-related revenue declines that are off practice balance sheets, they cited similar cost-reduction success rates. The authors asserted that primary care physicians can best pick specialists, diagnostics, and institutions “that provide evidence-based high-value care.” Putting primary care physicians in charge of reducing costs is neither new nor supported by experience. As gatekeepers in both earlier health maintenance organization and more recent payer-driven cost containment efforts, they had, at best, modest success in improving outcomes3 and reducing costs. 4 So why again nominate them as quality change agents? How does primary care prepare one for, say, comparing cancer treatment pathways or reducing surgical errors? Treating specialists are better informed about processes, outcomes, and tradeoffs.
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JAMA October 8, 2014 Volume 312, Number 14
Copyright 2014 American Medical Association. All rights reserved.
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1471
Letters
Crafting a solution that neutralizes perverse incentives but engages clinical expertise will be hard, beginning with how quality is defined. If solely by cost reduction, nonphysicians can be deployed to untangle inefficient processes, stanch revenue leaks, and identify cheaper labor. The broader Institute of Medicine definition, which adds using effective treatments, avoiding errors, absorbing innovations, including patients in dec ision making and reducing disparities, 5 requires an inclusive strategy that engages all clinicians, including specialists. But to declare the quality conundrum solved by making primary care physicians first among equals is just magical thinking. James A. Talcott, MD, SM Author Affiliation: Mt Sinai Beth Israel Medical Center, Mt Sinai School of Medicine, New York, New York. Corresponding Author: James A. Talcott, MD, SM, Center for Health Care Quality and Outcomes Research, Mt Sinai Beth Israel Medical Center, 10 Union Square E, New York, NY 10003 ([email protected]). Conflict of Interest Disclosures: The author has completed and submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest and none were reported. 1. Mostashari F, Sanghavi D, McClellan M. Health reform and physician-led accountable care: the paradox of primary care physician leadership. JAMA. 2014;311(18):1855-1856. 2. Joynt KE, Gawande AA, Orav EJ, Jha AK. Contribution of preventable acute care spending to total spending for high-cost Medicare patients. JAMA. 2013; 309(24):2572-2578. 3. Vedsted P, Olesen F. Are the serious problems in cancer survival partly rooted in gatekeeper principles? an ecologic study. Br J Gen Pract. 2011;61(589):e508e512. 4. Pati S, Shea S, Rabinowitz D, Carrasquillo O. Does gatekeeping control costs for privately insured children? findings from the 1996 medical expenditure panel survey. Pediatrics. 2003;111(3):456-460. 5. Committee on Quality of Health Care in America. Crossing the Quality Chasm: A New Health System for the 21st Century. Washington, DC: National Academy Press; 2001.
In Reply Dr Talcott raises the concern that physician ACOs merely repackage the failed strategies of primary care physician as gatekeeper of the 1990s, but there are fundamental differences. First, the incentive structure of physician ACOs is different because they benefit from simultaneous reduction in overall spending and improvement in quality and patient experience. This creates stronger motivation for them to provide enhanced, patient-centered services that can reduce costs. Payment models that reward total cost and quality achieve lower total health care cost with more intense—and redesigned— primary care services.1,2 In addition, under these models, as well as in the primary care physican–led ACOs, patients have enhanced access to primary and preventive care, and their care is coordinated with specialists and hospitals that must compete for referrals and admissions based on quality, cost, and communications. Primary care physicians are not directly providing high-value specialist care or reducing surgical errors but are collaborating with specialists and hospitals that can demonstrate that they are. 1472
Second, traditional methods for paying physicians, including fee-for-service payments with quality-related bonuses, do not provide the financial, data, and technology support for coordinating care with specialists to lower costs. This is a likely reason that increasing the supply of primary care physicians seems to be associated with better patient outcomes but not necessarily associated with reduced total health care costs3 and why evidence on the overall effect of medical home reforms has been mixed, as we noted in our article. In addition, many of the managed care cost reduction efforts of the 1990s did not provide such support either. Because Medicare beneficiaries can choose care outside of their ACO, primary care physicians cannot reduce costs through gatekeeping. Instead they must use these other tools for patient loyalty and retention. We recognize that specialists play an essential role in improving the bulk of patient care. Nearly 40% of elderly patients have 7 or more conditions, and these patients see an average of 8 different specialists. Other payment reforms that can be implemented along with primary care–ACO payments can more directly align specialist support, 4,5 and many physician-led ACOs directly include collaborating specialists. However, these points reinforce that primary care physicians are uniquely situated to take on a new version of their traditional role as health care quarterbacks for their patients, using new business models and 21st century data tools. We agree that the evidence on ACOs and related reforms that shift more accountability for better results and lower costs to physicians is limited so far. That is why multiple models are being tried now, and not all physician-led ACOs will succeed. But primary care physician–led ACOs, which require effective collaboration with specialists, are showing early promise in many settings and present a unique opportunity for primary care physicians today. Farzad Mostashari, MD, ScM Darshak Sanghavi, MD Mark McClellan, MD, PhD Author Affiliations: Aledade Inc, Bethesda, Maryland (Mostashari); Brookings Institution, Washington, DC (Sanghavi, McClellan). Corresponding Author: Mark McClellan, MD, PhD, Brookings Institution, 1775 Massachusetts Ave NW, Washington, DC 20036 ([email protected]). Conflict of Interest Disclosures: The authors have completed and submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest and none were reported. 1. Cohen R, Lemieux J, Schoenborn J, Mulligan T. Medicare Advantage Chronic Special Needs Plan boosted primary care, reduced hospital use among diabetes patients. Health Aff (Millwood). 2012;31(1):110-119. 2. Tanio C, Chen C. Innovations at Miami practice show promise for treating high-risk Medicare patients. Health Aff (Millwood). 2013;32(6):1078-1082. 3. Chang CH, Stukel TA, Flood AB, Goodman DC. Primary care physician workforce and Medicare beneficiaries’ health outcomes. JAMA. 2011;305(20): 2096-2104. 4. Landon BE, Roberts DH. Reenvisioning specialty care and payment under global payment systems. JAMA. 2013;310(4):371-372. 5. Pham HH, Schrag D, O’Malley AS, Wu B, Bach PB. Care patterns in Medicare and their implications for pay for performance. N Engl J Med. 2007;356(11): 1130-1139.
JAMA October 8, 2014 Volume 312, Number 14
Copyright 2014 American Medical Association. All rights reserved.
Downloaded From: http://jama.jamanetwork.com/ by a Otterbein University User on 06/03/2015
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In Reply Drs Saugel and Reuter challenge the description of the OPTIMISE trial as a pragmatic trial. As applied to clinical trials, the term pragmatic has a particular meaning. Pragmatic trials are designed to evaluate the clinical effectiveness of a treatment in the context of routine clinical practice.1 This distinguishes them from explanatory trials, which are designed to evaluate the efficacy of a treatment under ideal conditions. However, rather than describing dichotomous alternatives, the 2 terms represent different ends of a qualitative continuum. 2 Investigators designing pragmatic trials are faced with the challenge of balancing competing pressures to maintain internal validity (efficacy of the intervention) and the feasibility of implementation into widespread clinical use, the latter ensuring external validity (generalizability of findings). The OPTIMISE intervention was developed from an evidence base consisting of several clinical trials and other supporting data, which were described in the article. We evaluated this intervention in an explanatory pilot trial, the findings of which indicate a clear mechanistic basis through improvements in systemic oxygen delivery, tissue microvascular flow, and tissue oxygenation.3 Our laboratory studies also suggest that dopexamine may have important anti-inflammatory actions.4 Given the evidence available when the trial was designed, the high levels of protocol adherence, and our findings, we are satisfied that we achieved an appropriate balance between efficacy and feasibility in the design of this trial. Drs Latif and Faraday raise questions relating to the systematic review incorporated into the OPTIMISE article. They correctly describe unit-of-analysis issues that were highlighted in our discussion. These relate to evaluation of the primary outcome measure (number of patients developing complications), which was not reported for the trial by Sandham et al.5 However, mortality data were reported for this trial and were included both in the previous Cochrane systematic review6 and in analyses of mortality reported in the main text of our article. The results of the systematic review in detail are presented in the supplementary material. Comments in the report of the Cochrane review regarding sensitivity to analytical technique 6 relate to the choice of methods of metaanalysis and not to the design or outcome measures of component trials. The findings of the OPTIMISE trial and our systematic review require careful interpretation. The contrasting views of these correspondents highlight that uncertainty remains regarding the benefits of this treatment. They also reinforce the importance of pragmatic studies, such as the OPTIMISE trial, and emphasize the need for much larger trials to address this and other key uncertainties in perioperative medicine. Rupert M. Pearse, MD Neil MacDonald, FRCA Michael P. W. Grocott, MD; for the OPTIMISE Study Group
Author Affiliations: Queen Mary University of London, London, England (Pearse, MacDonald); Integrative Physiology and Critical Illness Group, University of Southampton, Southampton, England (Grocott). Corresponding Author: Rupert M. Pearse, MD, Queen Mary University of London, Adult Critical Care Unit, Royal London Hospital, Whitechapel, London E1 1BB, England ([email protected]). Conflict of Interest Disclosures: The authors have completed and submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest. Dr Pearse reported receiving equipment loans from LiDCO Ltd; receiving a grant from the National Institute for Health Research; receiving personal fees from Edwards Lifesciences, Covidien Inc, and Massimo Inc; and being a named inventor on a lapsed patent application relating to the perioperative use of dopexamine. Dr Grocott reported receiving grant funding from Deltex Medical Ltd; and receiving fees for lecturing from Fresenius Kabi and Edwards Lifesciences. No other disclosures were reported. 1. Roland M, Torgerson DJ. What are pragmatic trials? BMJ. 1998;316(7127):285. 2. Thorpe KE, Zwarenstein M, Oxman AD, et al. A pragmatic-explanatory continuum indicator summary (PRECIS): a tool to help trial designers. J Clin Epidemiol. 2009;62(5):464-475. 3. Jhanji S, Vivian-Smith A, Lucena-Amaro S, Watson D, Hinds CJ, Pearse RM. Haemodynamic optimisation improves tissue microvascular flow and oxygenation after major surgery: a randomised controlled trial. Crit Care. 2010; 14(4):R151. 4. Bangash MN, Patel NS, Benetti E, et al. Dopexamine can attenuate the inflammatory response and protect against organ injury in the absence of significant effects on hemodynamics or regional microvascular flow. Crit Care. 2013;17(2):R57. 5. Sandham JD, Hull RD, Brant RF, et al; Canadian Critical Care Clinical Trials Group. A randomized, controlled trial of the use of pulmonary-artery catheters in high-risk surgical patients. N Engl J Med. 2003;348(1):5-14. 6. Grocott MP, Dushianthan A, Hamilton MA, Mythen MG, Harrison D, Rowan K; Optimisation Systematic Review Steering Group. Perioperative increase in global blood flow to explicit defined goals and outcomes following surgery. Cochrane Database Syst Rev. 2012;11:CD004082.
Primary Care Physician–Led Health Reform To the Editor Dr Mostashari and colleagues1 described a vexing problem—creating incentives that encourage efficiency, not volume. Invoking an ambiguous role model, the corporate CEO, they postulated that primary care physicians, who account for 5% of health care spending, should make the tough calls on the rest. Supporting their solution proves hard. To set primary care’s contribution equal to estimated avoidable costs, they ignored a key finding: there is an association between costs and primary care physician supply.2 To support physician-led over hospital-led accountable care organizations (ACOs), which must absorb volume-related revenue declines that are off practice balance sheets, they cited similar cost-reduction success rates. The authors asserted that primary care physicians can best pick specialists, diagnostics, and institutions “that provide evidence-based high-value care.” Putting primary care physicians in charge of reducing costs is neither new nor supported by experience. As gatekeepers in both earlier health maintenance organization and more recent payer-driven cost containment efforts, they had, at best, modest success in improving outcomes3 and reducing costs. 4 So why again nominate them as quality change agents? How does primary care prepare one for, say, comparing cancer treatment pathways or reducing surgical errors? Treating specialists are better informed about processes, outcomes, and tradeoffs.
jama.com
JAMA October 8, 2014 Volume 312, Number 14
Copyright 2014 American Medical Association. All rights reserved.
Downloaded From: http://jama.jamanetwork.com/ by a Otterbein University User on 06/03/2015
1471
Letters
Crafting a solution that neutralizes perverse incentives but engages clinical expertise will be hard, beginning with how quality is defined. If solely by cost reduction, nonphysicians can be deployed to untangle inefficient processes, stanch revenue leaks, and identify cheaper labor. The broader Institute of Medicine definition, which adds using effective treatments, avoiding errors, absorbing innovations, including patients in dec ision making and reducing disparities, 5 requires an inclusive strategy that engages all clinicians, including specialists. But to declare the quality conundrum solved by making primary care physicians first among equals is just magical thinking. James A. Talcott, MD, SM Author Affiliation: Mt Sinai Beth Israel Medical Center, Mt Sinai School of Medicine, New York, New York. Corresponding Author: James A. Talcott, MD, SM, Center for Health Care Quality and Outcomes Research, Mt Sinai Beth Israel Medical Center, 10 Union Square E, New York, NY 10003 ([email protected]). Conflict of Interest Disclosures: The author has completed and submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest and none were reported. 1. Mostashari F, Sanghavi D, McClellan M. Health reform and physician-led accountable care: the paradox of primary care physician leadership. JAMA. 2014;311(18):1855-1856. 2. Joynt KE, Gawande AA, Orav EJ, Jha AK. Contribution of preventable acute care spending to total spending for high-cost Medicare patients. JAMA. 2013; 309(24):2572-2578. 3. Vedsted P, Olesen F. Are the serious problems in cancer survival partly rooted in gatekeeper principles? an ecologic study. Br J Gen Pract. 2011;61(589):e508e512. 4. Pati S, Shea S, Rabinowitz D, Carrasquillo O. Does gatekeeping control costs for privately insured children? findings from the 1996 medical expenditure panel survey. Pediatrics. 2003;111(3):456-460. 5. Committee on Quality of Health Care in America. Crossing the Quality Chasm: A New Health System for the 21st Century. Washington, DC: National Academy Press; 2001.
In Reply Dr Talcott raises the concern that physician ACOs merely repackage the failed strategies of primary care physician as gatekeeper of the 1990s, but there are fundamental differences. First, the incentive structure of physician ACOs is different because they benefit from simultaneous reduction in overall spending and improvement in quality and patient experience. This creates stronger motivation for them to provide enhanced, patient-centered services that can reduce costs. Payment models that reward total cost and quality achieve lower total health care cost with more intense—and redesigned— primary care services.1,2 In addition, under these models, as well as in the primary care physican–led ACOs, patients have enhanced access to primary and preventive care, and their care is coordinated with specialists and hospitals that must compete for referrals and admissions based on quality, cost, and communications. Primary care physicians are not directly providing high-value specialist care or reducing surgical errors but are collaborating with specialists and hospitals that can demonstrate that they are. 1472
Second, traditional methods for paying physicians, including fee-for-service payments with quality-related bonuses, do not provide the financial, data, and technology support for coordinating care with specialists to lower costs. This is a likely reason that increasing the supply of primary care physicians seems to be associated with better patient outcomes but not necessarily associated with reduced total health care costs3 and why evidence on the overall effect of medical home reforms has been mixed, as we noted in our article. In addition, many of the managed care cost reduction efforts of the 1990s did not provide such support either. Because Medicare beneficiaries can choose care outside of their ACO, primary care physicians cannot reduce costs through gatekeeping. Instead they must use these other tools for patient loyalty and retention. We recognize that specialists play an essential role in improving the bulk of patient care. Nearly 40% of elderly patients have 7 or more conditions, and these patients see an average of 8 different specialists. Other payment reforms that can be implemented along with primary care–ACO payments can more directly align specialist support, 4,5 and many physician-led ACOs directly include collaborating specialists. However, these points reinforce that primary care physicians are uniquely situated to take on a new version of their traditional role as health care quarterbacks for their patients, using new business models and 21st century data tools. We agree that the evidence on ACOs and related reforms that shift more accountability for better results and lower costs to physicians is limited so far. That is why multiple models are being tried now, and not all physician-led ACOs will succeed. But primary care physician–led ACOs, which require effective collaboration with specialists, are showing early promise in many settings and present a unique opportunity for primary care physicians today. Farzad Mostashari, MD, ScM Darshak Sanghavi, MD Mark McClellan, MD, PhD Author Affiliations: Aledade Inc, Bethesda, Maryland (Mostashari); Brookings Institution, Washington, DC (Sanghavi, McClellan). Corresponding Author: Mark McClellan, MD, PhD, Brookings Institution, 1775 Massachusetts Ave NW, Washington, DC 20036 ([email protected]). Conflict of Interest Disclosures: The authors have completed and submitted the ICMJE Form for Disclosure of Potential Conflicts of Interest and none were reported. 1. Cohen R, Lemieux J, Schoenborn J, Mulligan T. Medicare Advantage Chronic Special Needs Plan boosted primary care, reduced hospital use among diabetes patients. Health Aff (Millwood). 2012;31(1):110-119. 2. Tanio C, Chen C. Innovations at Miami practice show promise for treating high-risk Medicare patients. Health Aff (Millwood). 2013;32(6):1078-1082. 3. Chang CH, Stukel TA, Flood AB, Goodman DC. Primary care physician workforce and Medicare beneficiaries’ health outcomes. JAMA. 2011;305(20): 2096-2104. 4. Landon BE, Roberts DH. Reenvisioning specialty care and payment under global payment systems. JAMA. 2013;310(4):371-372. 5. Pham HH, Schrag D, O’Malley AS, Wu B, Bach PB. Care patterns in Medicare and their implications for pay for performance. N Engl J Med. 2007;356(11): 1130-1139.
JAMA October 8, 2014 Volume 312, Number 14
Copyright 2014 American Medical Association. All rights reserved.
Downloaded From: http://jama.jamanetwork.com/ by a Otterbein University User on 06/03/2015
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