INTERVIEW
Improving the efficiency and outcomes of medical care Alvin I Mushlin, MD, ScM, is Chair of the Department of Public Health at Weill Cornell Medical College and Public Health Physician-in-Chief of the New York-Presbyterian Hospital/Weill Cornell Medical Center in New York City, USA. Dr Mushlin’s interests focus on outcomes research to improve clinical decision-making and policy formulation. His studies have sought to quantify the value and accuracy of diagnostic tests and procedures, to understand the efficacy and cost–effectiveness of medical interventions, and to measure the quality of medical care. He is a leader in the development of comparative effectiveness research methods and the use of economics in medical research and was one of the first to systematically evaluate the accuracy of MRI. He also led a landmark study in the evolution and adoption of cardiac defibrillators. Dr Mushlin was recently the Principal Investigator of the Weill Cornell Centers for Education and Research Therapeutics (CERT) grant from the Agency for Healthcare Research and Quality to evaluate medical and orthopedic devices. He is the Weill Cornell Clinical and Translational Science Center’s representative on the NIH Comparative Effectiveness Research Key Function Committee. He holds an MD degree from Vanderbilt University (TN, USA), a ScM in Epidemiology and Medical Care (completed as a Robert Wood Johnson Clinical Scholar) from Johns Hopkins University (MD, USA) and completed his clinical training at The New York Hospital/Cornell Medical Center. Dr Mushlin is board certified in internal medicine and a Fellow of the American College of Physicians. Could you tell our readers a little about your career to date & how you came to your current role? QQ
For my entire career, I have thought that we should be able to make medical care more efficient and thereby expand our ability to help more people and do a better job for the patients under our care. Although I have appreciated and recognized the contributions that biomedical research can make toward that aim, I have also thought that just having more knowledge about diseases and what causes them was not enough. Research to help understand the effectiveness of diagnostic tests, treatments and clinical strategies, as well as how to deliver care most efficiently, was largely missing when I finished my clinical training. I wanted to improve medical practice, not just for my patients, but for medicine overall. To my knowledge then, the type of career I was envisioning did not exist. I didn’t see role models in academic medicine who were doing this. Fortunately, I was wrong, there was more going on in the field than I recognized. One of my mentors helped me to learn about such efforts in the fields of clinical epidemiology and health services research. I discovered and entered a training program that included concomitant graduate education to gain the research skills to evaluate medical interventions and clinical decisions; and I have done this kind of research for my entire career. I initially was fascinated by the challenge of using diagnostic tests appropriately. Not only did it seem to me that diagnostic tests were being used both too much
10.2217/CER.12.40 © 2012 Future Medicine Ltd
1(5), 387–389 (2012)
Alvin I Mushlin*
“[Comparative effectiveness research] ... should ... make medical care more efficient and thereby expand our ability to help more people...”
*Weill Cornell Medical College/New York Presbyterian Hospital, Department of Public Health, 402 East 67th Street, New York, NY 10065, USA [email protected]
part of
ISSN 2042-6305
387
News & Views
Mushlin
and sometimes not enough, but also that the way we thought about diagnostic information and the ‘calculus’ we used to quantify their impact on clinical decisions was incompletely developed and inadequate for informing their selection and interpretation. I first tried to understand and then to develop new methods for diagnostic test evaluation. However, I soon recognized that without the science behind the optimal use of scarce resources, one could rarely be definitive about test use recommendations or guidelines. To further develop these skills, I devoted a sabbatical to learning the principles of cost–effectiveness analysis and clinical economics. During that year, my colleagues and I developed a strategy that used economic methods and decision analysis to provide insights and to set priorities for diagnostic test evaluations. Among other things, I completed a series of studies to help inform the use of MRI, then a new diagnostic tool. We set up experiments to measure the accuracy of brain MRI in patients with nonspecific neurological symptoms and decision analytic models using these data to help understand if and when it would improve outcomes and in whom MRI would be most useful. We finished this line of research via a cost–effectiveness analysis to inform MRI’s uses for these clinical indications. Later, I had the opportunity to measure resource use and the costs associated with another new technology: implantable cardiac defibrillators (ICDs) for patients with coronary artery disease at risk for fatal arrhythmias. I was the principle investigator for the economic component of a multisite, multinational, randomized clinical trial (RCT) of ICDs versus medications for patients who had episodes of nonsustained ventricular tachycardia. This became the largest economic analysis alongside an RCT to date and still represents a model for how to perform such studies. For the past decade, my career has shifted toward more administration and leadership. I have had the opportunity here at the Weill Cornell Medical College in New York City to develop an academic department of public health almost from scratch. Of course, I had been thinking for a long time about the importance of the social and public health sciences, in addition to the biomedical sciences, as part of the full spectrum of disciplines for medical research. Developing the department here has required recognizing the required elements and putting them into place. I hope that the contributions of our department to the research portfolio of this institution will
388
J. Compar. Effect. Res. (2012) 1(5)
demonstrate the importance of including these disciplines in medical research more generally. How is your time balanced between clinical & research work? QQ
The main balancing act I have had to master is between administration and research. I am still active in clinical care, supervising students and residents in our general medicine clinic, but that is a relatively small time commitment. My major challenge is leading a department while still remaining an active investigator. I have found that one of the most natural ways for me to do that is to assist, mentor and collaborate with the junior investigators in my department. What is your research focusing on at present? QQ
My current research interests and activities are broad. One major recent focus is on the evaluation of medical devices. I have been interested in developing registries and databases of patients in whom new technologies are being used, carefully cataloging the types and characteristics of such devices and then tracking how patients respond after they receive them. This parallels an interest I have had for a long time about methods to improve our understanding of the effectiveness and comparative effectiveness of drugs and devices that can be used when RCTs are not practical. I still have a major interest in cost–effectiveness analysis and I am doing projects using this methodology to define clinical use recommendations. I think that we need to do more to inform the intelligent use of medical resources and to reduce the growth in the costs of medical care, particularly in the USA. For example, toward this end, I have been working with an economist in our department to track and evaluate efforts at clinical cost containment, through decreasing incentives for the use of diagnostic tests for coronary artery disease. How successfully do you think comparative effectiveness research is currently being translated into policy & practice worldwide? QQ
I have always thought that when the evidence was clear and compelling, doctors would use it to change what they are currently doing, and that hospitals and healthcare systems would follow their lead. My thinking is now changing, as it seems increasingly clear to me that this is not always the case. I recently completed a study with a young cardiologist colleague on the
future science group
Improving the efficiency & outcomes of medical care
use of medications as compared with stents or angioplasties for patients with coronary artery disease following the results of the COURAGE trial, which showed that drugs were basically as effective as angioplasty or stents. However, we found that many patients coming to percutaneous cardiac intervention had not been given a trial of medical therapy and that their medical regimens were less than what is currently recommended. We have also looked at the rates of antianginal medication use in regions throughout the USA and did not find a correlation with the rates of percutaneous cardiac intervention, suggesting that more medical therapy does not always translate into fewer invasive procedures. All of this has convinced me that we need to do much more to understand and then to influence clinical behavior. We are just now starting a study to systematically track the diffusion of research findings from comparative effectiveness research (CER) and their impact. Although I don’t think CER is being translated into practice as well as it should be, the main problem is that there is still not enough CER to influence care in a major way, and some of the CER that is out there is not solid and convincing enough to bring about change. QQ
How do you think this can be improved?
First, we need more and better CER. The ‘better’ issue is worth expanding upon. In my mind, it’s more than a lack of funding and thereby, lack of research opportunities that is the problem; we also need better methods, study designs and statistical techniques to carry out this kind of research. Particularly important are two issues: we need to change the basic thinking about RCTs and their emphasis on preserving ‘internal’ validity at any cost. There is a natural tension between maximizing internal validity and clinical relevance, and generalizability. We need to create a better balance. Doing so will require that we get more comfortable with ‘practical’ clinical trials with their emphasis on effectiveness. At the same time, we also need to develop new methods and approaches that preserve their ability to assess efficacy. However, we will always need observational research designs that do not require experiments and random assignment. We have made significant progress through creating a variety of databases and making them available for such research and through developing new statistical and econometric approaches, such as propensity scores and instrumental variables, but we need to do even
future science group
News & Views
more. There is still too much skepticism about our ability to inform clinical practices from observational studies. That must change; however, even the best evidence will not change behavior until we have better strategies to influence thinking and to change healthcare systems – that will always require a mix of science and politics. What are you excited about working on over the next year? QQ
I would like to be involved in studies that develop and use methods for conducting highquality observational studies and convincingly showing that they provide new and valid insights about the way clinical medicine should be practiced and new technologies used. Finally, what do you think will be the hot topics in CER in your field over the next few years? QQ
I think that developing and refining methods to make CER truly credible and capable of influencing clinical practice and health policy in significant and meaningful ways is of the utmost importance. The Institute of Medicine here and other groups around the world have already come out with priority lists of topics to receive new funding for CER. The Patient-Centered Outcomes Research Institute (PCORI) in the USA has just issued a request for new grant applications based on their priorities for funding. Included in their announcement is the need for new ways of engaging patients and other stakeholders in CER. We should not underestimate the challenge that this represents or lose track of this important dimension as we proceed over the next few years. Of course, we all think that as long as studies include healthrelated outcomes that CER will be useful, but really addressing patients’ concerns and the elements of care that are critical to their physicians is much more complicated than that. We should all recognize the challenge, but also value the opportunities in this line of research. Financial & competing interests disclosure AI Mushlin 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.
www.futuremedicine.com
389
Improving the efficiency and outcomes of medical care Alvin I Mushlin, MD, ScM, is Chair of the Department of Public Health at Weill Cornell Medical College and Public Health Physician-in-Chief of the New York-Presbyterian Hospital/Weill Cornell Medical Center in New York City, USA. Dr Mushlin’s interests focus on outcomes research to improve clinical decision-making and policy formulation. His studies have sought to quantify the value and accuracy of diagnostic tests and procedures, to understand the efficacy and cost–effectiveness of medical interventions, and to measure the quality of medical care. He is a leader in the development of comparative effectiveness research methods and the use of economics in medical research and was one of the first to systematically evaluate the accuracy of MRI. He also led a landmark study in the evolution and adoption of cardiac defibrillators. Dr Mushlin was recently the Principal Investigator of the Weill Cornell Centers for Education and Research Therapeutics (CERT) grant from the Agency for Healthcare Research and Quality to evaluate medical and orthopedic devices. He is the Weill Cornell Clinical and Translational Science Center’s representative on the NIH Comparative Effectiveness Research Key Function Committee. He holds an MD degree from Vanderbilt University (TN, USA), a ScM in Epidemiology and Medical Care (completed as a Robert Wood Johnson Clinical Scholar) from Johns Hopkins University (MD, USA) and completed his clinical training at The New York Hospital/Cornell Medical Center. Dr Mushlin is board certified in internal medicine and a Fellow of the American College of Physicians. Could you tell our readers a little about your career to date & how you came to your current role? QQ
For my entire career, I have thought that we should be able to make medical care more efficient and thereby expand our ability to help more people and do a better job for the patients under our care. Although I have appreciated and recognized the contributions that biomedical research can make toward that aim, I have also thought that just having more knowledge about diseases and what causes them was not enough. Research to help understand the effectiveness of diagnostic tests, treatments and clinical strategies, as well as how to deliver care most efficiently, was largely missing when I finished my clinical training. I wanted to improve medical practice, not just for my patients, but for medicine overall. To my knowledge then, the type of career I was envisioning did not exist. I didn’t see role models in academic medicine who were doing this. Fortunately, I was wrong, there was more going on in the field than I recognized. One of my mentors helped me to learn about such efforts in the fields of clinical epidemiology and health services research. I discovered and entered a training program that included concomitant graduate education to gain the research skills to evaluate medical interventions and clinical decisions; and I have done this kind of research for my entire career. I initially was fascinated by the challenge of using diagnostic tests appropriately. Not only did it seem to me that diagnostic tests were being used both too much
10.2217/CER.12.40 © 2012 Future Medicine Ltd
1(5), 387–389 (2012)
Alvin I Mushlin*
“[Comparative effectiveness research] ... should ... make medical care more efficient and thereby expand our ability to help more people...”
*Weill Cornell Medical College/New York Presbyterian Hospital, Department of Public Health, 402 East 67th Street, New York, NY 10065, USA [email protected]
part of
ISSN 2042-6305
387
News & Views
Mushlin
and sometimes not enough, but also that the way we thought about diagnostic information and the ‘calculus’ we used to quantify their impact on clinical decisions was incompletely developed and inadequate for informing their selection and interpretation. I first tried to understand and then to develop new methods for diagnostic test evaluation. However, I soon recognized that without the science behind the optimal use of scarce resources, one could rarely be definitive about test use recommendations or guidelines. To further develop these skills, I devoted a sabbatical to learning the principles of cost–effectiveness analysis and clinical economics. During that year, my colleagues and I developed a strategy that used economic methods and decision analysis to provide insights and to set priorities for diagnostic test evaluations. Among other things, I completed a series of studies to help inform the use of MRI, then a new diagnostic tool. We set up experiments to measure the accuracy of brain MRI in patients with nonspecific neurological symptoms and decision analytic models using these data to help understand if and when it would improve outcomes and in whom MRI would be most useful. We finished this line of research via a cost–effectiveness analysis to inform MRI’s uses for these clinical indications. Later, I had the opportunity to measure resource use and the costs associated with another new technology: implantable cardiac defibrillators (ICDs) for patients with coronary artery disease at risk for fatal arrhythmias. I was the principle investigator for the economic component of a multisite, multinational, randomized clinical trial (RCT) of ICDs versus medications for patients who had episodes of nonsustained ventricular tachycardia. This became the largest economic analysis alongside an RCT to date and still represents a model for how to perform such studies. For the past decade, my career has shifted toward more administration and leadership. I have had the opportunity here at the Weill Cornell Medical College in New York City to develop an academic department of public health almost from scratch. Of course, I had been thinking for a long time about the importance of the social and public health sciences, in addition to the biomedical sciences, as part of the full spectrum of disciplines for medical research. Developing the department here has required recognizing the required elements and putting them into place. I hope that the contributions of our department to the research portfolio of this institution will
388
J. Compar. Effect. Res. (2012) 1(5)
demonstrate the importance of including these disciplines in medical research more generally. How is your time balanced between clinical & research work? QQ
The main balancing act I have had to master is between administration and research. I am still active in clinical care, supervising students and residents in our general medicine clinic, but that is a relatively small time commitment. My major challenge is leading a department while still remaining an active investigator. I have found that one of the most natural ways for me to do that is to assist, mentor and collaborate with the junior investigators in my department. What is your research focusing on at present? QQ
My current research interests and activities are broad. One major recent focus is on the evaluation of medical devices. I have been interested in developing registries and databases of patients in whom new technologies are being used, carefully cataloging the types and characteristics of such devices and then tracking how patients respond after they receive them. This parallels an interest I have had for a long time about methods to improve our understanding of the effectiveness and comparative effectiveness of drugs and devices that can be used when RCTs are not practical. I still have a major interest in cost–effectiveness analysis and I am doing projects using this methodology to define clinical use recommendations. I think that we need to do more to inform the intelligent use of medical resources and to reduce the growth in the costs of medical care, particularly in the USA. For example, toward this end, I have been working with an economist in our department to track and evaluate efforts at clinical cost containment, through decreasing incentives for the use of diagnostic tests for coronary artery disease. How successfully do you think comparative effectiveness research is currently being translated into policy & practice worldwide? QQ
I have always thought that when the evidence was clear and compelling, doctors would use it to change what they are currently doing, and that hospitals and healthcare systems would follow their lead. My thinking is now changing, as it seems increasingly clear to me that this is not always the case. I recently completed a study with a young cardiologist colleague on the
future science group
Improving the efficiency & outcomes of medical care
use of medications as compared with stents or angioplasties for patients with coronary artery disease following the results of the COURAGE trial, which showed that drugs were basically as effective as angioplasty or stents. However, we found that many patients coming to percutaneous cardiac intervention had not been given a trial of medical therapy and that their medical regimens were less than what is currently recommended. We have also looked at the rates of antianginal medication use in regions throughout the USA and did not find a correlation with the rates of percutaneous cardiac intervention, suggesting that more medical therapy does not always translate into fewer invasive procedures. All of this has convinced me that we need to do much more to understand and then to influence clinical behavior. We are just now starting a study to systematically track the diffusion of research findings from comparative effectiveness research (CER) and their impact. Although I don’t think CER is being translated into practice as well as it should be, the main problem is that there is still not enough CER to influence care in a major way, and some of the CER that is out there is not solid and convincing enough to bring about change. QQ
How do you think this can be improved?
First, we need more and better CER. The ‘better’ issue is worth expanding upon. In my mind, it’s more than a lack of funding and thereby, lack of research opportunities that is the problem; we also need better methods, study designs and statistical techniques to carry out this kind of research. Particularly important are two issues: we need to change the basic thinking about RCTs and their emphasis on preserving ‘internal’ validity at any cost. There is a natural tension between maximizing internal validity and clinical relevance, and generalizability. We need to create a better balance. Doing so will require that we get more comfortable with ‘practical’ clinical trials with their emphasis on effectiveness. At the same time, we also need to develop new methods and approaches that preserve their ability to assess efficacy. However, we will always need observational research designs that do not require experiments and random assignment. We have made significant progress through creating a variety of databases and making them available for such research and through developing new statistical and econometric approaches, such as propensity scores and instrumental variables, but we need to do even
future science group
News & Views
more. There is still too much skepticism about our ability to inform clinical practices from observational studies. That must change; however, even the best evidence will not change behavior until we have better strategies to influence thinking and to change healthcare systems – that will always require a mix of science and politics. What are you excited about working on over the next year? QQ
I would like to be involved in studies that develop and use methods for conducting highquality observational studies and convincingly showing that they provide new and valid insights about the way clinical medicine should be practiced and new technologies used. Finally, what do you think will be the hot topics in CER in your field over the next few years? QQ
I think that developing and refining methods to make CER truly credible and capable of influencing clinical practice and health policy in significant and meaningful ways is of the utmost importance. The Institute of Medicine here and other groups around the world have already come out with priority lists of topics to receive new funding for CER. The Patient-Centered Outcomes Research Institute (PCORI) in the USA has just issued a request for new grant applications based on their priorities for funding. Included in their announcement is the need for new ways of engaging patients and other stakeholders in CER. We should not underestimate the challenge that this represents or lose track of this important dimension as we proceed over the next few years. Of course, we all think that as long as studies include healthrelated outcomes that CER will be useful, but really addressing patients’ concerns and the elements of care that are critical to their physicians is much more complicated than that. We should all recognize the challenge, but also value the opportunities in this line of research. Financial & competing interests disclosure AI Mushlin 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.
www.futuremedicine.com
389
