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INTERVIEW
A private sector life in comparative effectiveness research Robert W Dubois joined the National Pharmaceutical Council (Washington, DC, USA) in October 2010 as its Chief Science Officer. In this role, he oversees the National Pharmaceutical Council’s research on policy issues related to comparative effectiveness research, as well as on how health outcomes are valued. Dr Dubois has cofounded and led various healthcare research organizations in developing quality research with practical application. Throughout his career, Dr Dubois’ primary interest has centered on defining ‘what works’ in healthcare and finding ways for that evidence to inform healthcare decision-making. He is a recognized expert in the areas of defining best practice, disease management and appropriateness of care. He has authored more than 100 peer-reviewed articles on comparative effectiveness, evidence-based medicine, the development of practice guidelines and determining the optimal use of high-cost medical services. Dr Dubois received his AB from Harvard College (MA, USA), his MD from the Johns Hopkins School of Medicine (MD, USA) and his PhD in Health Policy from the RAND Graduate School (CA, USA). In addition, he is the associate editor of the Journal of Comparative Effectiveness Research and is on the editorial board for Health Affairs. Could you tell our readers a little bit about your career to date & how you came to your current role?
Robert W Dubois*
QQ
I began with clinical training in internal medicine; however, from the beginning I had a strong interest in macrolevel issues and I decided to also receive training in policy and research methods. I was a Robert Wood Johnson Clinical Scholar at University of California, Los Angeles (CA, USA), and during that time, received a PhD in health policy at the RAND Graduate School (CA, USA). Soon after that, several individuals from RAND founded a company called Value Health Sciences, which was quite a new concept during the latter part of the 1980s, to translate health outcomes or health services research methods into usable tools at the beginning of the managed-care movement. We developed software to apply practice guidelines; we used techniques from the RAND Health Insurance Experiment on building episodes of care to also develop a physician-profiling tool. Taking these methods and turning them into actual day-to-day capabilities for managed care and for physicians was really quite new and exciting. My career has focused on defining what is appropriate care and then finding ways for that care to be implemented. I have endeavored to maintain one foot in the world of methods and another foot in the world of application, and I have pursued that goal within the private sector. Although the work I have carried out has been conducted in the private sector, I have still pursued publication of it in peer-reviewed journals. Over the years, I have worked with managed care, the pharmaceutical industry and makers of electronic health records. I started working for the National Pharmaceutical Council (DC, USA) 3 years ago as it was an opportunity to return to my policy roots and to begin to think about healthcare from a higher level vantage point; that is, not focusing on
10.2217/CER.13.56 © 2013 Future Medicine Ltd
2(5), 429–431 (2013)
“My career has focused on defining what is appropriate care and then finding ways for that care to be implemented.”
*National Pharmaceutical Council, Washington, DC, USA Tel.: +1 202 827 2079 [email protected]
part of
ISSN 2042-6305
429
INTERVIEW Dubois individual conditions or individual treatments, but really considering comparative effectiveness from a much broader perspective. What are your main responsibilities at the National Pharmaceutical Council? QQ
The National Pharmaceutical Council is a health policy research group so we conduct policy research. We are not an advocacy organization and do not lobby. We try to develop research and evidence that can enhance the dialog surrounding policy issues that have relevance to the pharmaceutical industry. As Chief Science Officer, I am involved in setting the strategic direction of the group; that is, identifying research topics and then participating in the research. Our model is to be coinvestigators with other investigators in the field. We have research staff that are actively involved in that research. What is your research focusing on at present? QQ
We currently have three areas of interest. The first is finding the proper balance between populationlevel comparative effectiveness research (CER) assessments and the needs of individual patients. This deals with issues of heterogeneity of treatment effect, incorporating patient preferences and personalized medicine. It is very important to realize that often in CER, the results are made available at the ‘average’ level, and it is assumed that if ‘treatment A’ is better than ‘treatment B’ for the population then everyone should receive ‘treatment A’. In reality, some patients are better served by receiving ‘treatment B’. The key to appropriately implementing CER is to find the right balance and that is one of our main aims. “I am concerned about finding the proper balance in comparative effectiveness research implementation from what is ‘right’ for the population versus what is best for individuals.”
costs. Medications are also very important in achieving the quality benchmarks. The third area is looking at the use of real-world evidence to make assessments about what works in healthcare. In that regard, we are exploring how real-world data can be collected, analyzed and incorporated into decisions in a proper fashion. How successfully do you think CER is currently being translated into policy & practice worldwide? QQ
I think there are two aspects to this question. First, is CER being incorporated into decisions? Second, is it being incorporated correctly? Outside of the USA we are seeing CER being incorporated by groups such as NICE in the UK, Institute for Quality and Efficiency in Health Care in Germany and Pharmaceutical Benefits Advisory Committee in Australia. However, in the USA I would say that we are at the very early stages of CER implementation or its use in decision-making. Whether CER is being used appropriately is a very different question. I am concerned about finding the proper balance in CER implementation from what is ‘right’ for the population versus what is best for individuals. I do not think implementation at NICE or elsewhere has fully incorporated the needs of individual patients when assessments are made on whether to pay for a particular new therapy. At a population level, a new treatment may not meet a given cost–effectiveness threshold, but there may be individual patients who might receive tremendous benefit and they may not have the opportunity to receive that treatment. The classic saying is ‘do not throw the baby out with the bathwater’. I think if you do not carefully look at these issues then this may happen. QQ
How do you think it could be improved?
I think there is both additional research and new considerations on implementation that need to be carried out. On the research side, more work is needed on understanding the heterogeneity of response for individual treatments; that is, when The second area is the evolving payment and we perform CER, it is not nearly enough just to delivery environment, moving from a fee-for- carry out an average assessment, we need to look service world to one that instead provides eco- carefully at the individual subgroups and individnomic bundles to providers, and also has perfor- ual patients. One highly underused approach is mance assessments and bonuses based on quality. crossover designs, which are costly and often comWe are very focused on this performance-based plex but can provide us with important informareimbursement approach and understanding how tion about which patients will respond to a therapy. medications can influence the overall episode of A recent study examined patients with rheumatoid care cost, as well as how proper medication use arthritis and the authors compared biologic thermay reduce hospitalizations or other episode apy with a regimen of three oral drugs [1]. They
430
J. Compar. Effect. Res. (2013) 2(5)
future science group
Dubois
found that one in four patients did not respond to either therapy. However, when they crossed the patients over, many of those nonresponsive patients did respond to the alternative treatment. This study showed that although on average the various regimens seemed similar, for individual patients, there were important differences. The second improvement concerns the policies that are developed. Treatment policies need to consider subgroups; that certain treatment subgroups may receive particular benefit and should have an opportunity to receive therapies that may not be available to other subgroups that receive little benefit. The saying that ‘one size does not fit all’ is very important to keep in mind. What is the pharmaceutical industry’s perspective on CER? QQ
The pharmaceutical industry is very supportive of CER that is carried out correctly, and correctly means to consider heterogeneity as well as assessment of all the impacts on patients resulting from a treatment, including quality of life, functional ability, patient preferences and impact on the overall episode of care. A broad assessment of outcomes is very important. The industry is very supportive of looking at real-world experience and issues such as adherence to therapy, which may differ among treatments. You cannot truly understand that from a clinical trial where everything is tightly controlled, whereas in a real-world environment, you can begin to understand some of these issues. I am quite happy that the PatientCentered Outcomes Research Institute (Washington, DC, USA) has identified their priority topics very broadly. The concern of the industry was that CER would focus primarily on drug–drug comparisons, but, in fact, the vast majority of PatientCentered Outcomes Research Institute’s research funding announcements are focusing on all types of therapeutics, such as the benefits of surgery and different ways to deal with weight loss. What are you excited about working on over the next year? QQ
There has been a flurry of activity around defining ‘good’ CER and there are a number of standards or guidelines concerning how CER should be conducted. What we are concerned or excited about is the need to begin to look at those many different guidelines and come up with some consensus among them so that researchers and policy-makers can agree upon the characteristics of a good study. For example, if I conducted a study with one set of
future science group
INTERVIEW
guidelines, you could look at that study through the lens of another set of guidelines and say that my study was not carried out very well. With a harmonization of guidelines it will become clearer to the field what good looks like. What do you think will be the hot topics in CER over the next 5–10 years? QQ
The focus of the ‘Methods to policy’ piece in this issue of the Journal of Comparative Effectiveness Research, which McElwee and I wrote, describes an area of great interest [2]. There is substantial discussion about ‘big data’ and mining databases to understand the real world, and using those analyses to make clinical and policy decisions. There is a tremendous amount of enthusiasm around this, but we have concerns that the methods for conducting these studies are not yet fully mature and that we need to be cautious before we use these analyses to drive change. In the excitement surrounding rapid learning systems, we need to make sure that there is adequate independent review of what has been carried out and the validity of that since, in the rapid learning environment, there will not be a peer-review process like a manuscript would undergo. We are excited about the use of big data, but want that usage to drive appropriate change, not just any change. Disclaimer The opinions expressed in this interview are those of the interviewee and do not necessarily reflect the views of Future Medicine Ltd.
Financial & competing interests disclosure RW Dubois is an employee of the National Pharmaceutical Council, an industry-funded health policy research group that is not involved in lobbying or advocacy. He has no other 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 apart from those disclosed. No writing assistance was utilized in the production of this manuscript.
References 1
O’Dell JR, Mikuls TR, Taylor TH et al. CSP 551 RACAT Investigators. Therapies for active rheumatoid arthritis after methotrexate failure. N. Engl. J. Med. 369(4), 307–318 (2013).
2
McElwee NE, Dubois RW. Enthusiasm for rapid learning health systems exceeds the current standards for conducting it. J. Comp. Eff. Res. 2(5), 425–427 (2013).
www.futuremedicine.com
431
INTERVIEW
A private sector life in comparative effectiveness research Robert W Dubois joined the National Pharmaceutical Council (Washington, DC, USA) in October 2010 as its Chief Science Officer. In this role, he oversees the National Pharmaceutical Council’s research on policy issues related to comparative effectiveness research, as well as on how health outcomes are valued. Dr Dubois has cofounded and led various healthcare research organizations in developing quality research with practical application. Throughout his career, Dr Dubois’ primary interest has centered on defining ‘what works’ in healthcare and finding ways for that evidence to inform healthcare decision-making. He is a recognized expert in the areas of defining best practice, disease management and appropriateness of care. He has authored more than 100 peer-reviewed articles on comparative effectiveness, evidence-based medicine, the development of practice guidelines and determining the optimal use of high-cost medical services. Dr Dubois received his AB from Harvard College (MA, USA), his MD from the Johns Hopkins School of Medicine (MD, USA) and his PhD in Health Policy from the RAND Graduate School (CA, USA). In addition, he is the associate editor of the Journal of Comparative Effectiveness Research and is on the editorial board for Health Affairs. Could you tell our readers a little bit about your career to date & how you came to your current role?
Robert W Dubois*
I began with clinical training in internal medicine; however, from the beginning I had a strong interest in macrolevel issues and I decided to also receive training in policy and research methods. I was a Robert Wood Johnson Clinical Scholar at University of California, Los Angeles (CA, USA), and during that time, received a PhD in health policy at the RAND Graduate School (CA, USA). Soon after that, several individuals from RAND founded a company called Value Health Sciences, which was quite a new concept during the latter part of the 1980s, to translate health outcomes or health services research methods into usable tools at the beginning of the managed-care movement. We developed software to apply practice guidelines; we used techniques from the RAND Health Insurance Experiment on building episodes of care to also develop a physician-profiling tool. Taking these methods and turning them into actual day-to-day capabilities for managed care and for physicians was really quite new and exciting. My career has focused on defining what is appropriate care and then finding ways for that care to be implemented. I have endeavored to maintain one foot in the world of methods and another foot in the world of application, and I have pursued that goal within the private sector. Although the work I have carried out has been conducted in the private sector, I have still pursued publication of it in peer-reviewed journals. Over the years, I have worked with managed care, the pharmaceutical industry and makers of electronic health records. I started working for the National Pharmaceutical Council (DC, USA) 3 years ago as it was an opportunity to return to my policy roots and to begin to think about healthcare from a higher level vantage point; that is, not focusing on
10.2217/CER.13.56 © 2013 Future Medicine Ltd
2(5), 429–431 (2013)
“My career has focused on defining what is appropriate care and then finding ways for that care to be implemented.”
*National Pharmaceutical Council, Washington, DC, USA Tel.: +1 202 827 2079 [email protected]
part of
ISSN 2042-6305
429
INTERVIEW Dubois individual conditions or individual treatments, but really considering comparative effectiveness from a much broader perspective. What are your main responsibilities at the National Pharmaceutical Council? QQ
The National Pharmaceutical Council is a health policy research group so we conduct policy research. We are not an advocacy organization and do not lobby. We try to develop research and evidence that can enhance the dialog surrounding policy issues that have relevance to the pharmaceutical industry. As Chief Science Officer, I am involved in setting the strategic direction of the group; that is, identifying research topics and then participating in the research. Our model is to be coinvestigators with other investigators in the field. We have research staff that are actively involved in that research. What is your research focusing on at present? QQ
We currently have three areas of interest. The first is finding the proper balance between populationlevel comparative effectiveness research (CER) assessments and the needs of individual patients. This deals with issues of heterogeneity of treatment effect, incorporating patient preferences and personalized medicine. It is very important to realize that often in CER, the results are made available at the ‘average’ level, and it is assumed that if ‘treatment A’ is better than ‘treatment B’ for the population then everyone should receive ‘treatment A’. In reality, some patients are better served by receiving ‘treatment B’. The key to appropriately implementing CER is to find the right balance and that is one of our main aims. “I am concerned about finding the proper balance in comparative effectiveness research implementation from what is ‘right’ for the population versus what is best for individuals.”
costs. Medications are also very important in achieving the quality benchmarks. The third area is looking at the use of real-world evidence to make assessments about what works in healthcare. In that regard, we are exploring how real-world data can be collected, analyzed and incorporated into decisions in a proper fashion. How successfully do you think CER is currently being translated into policy & practice worldwide? QQ
I think there are two aspects to this question. First, is CER being incorporated into decisions? Second, is it being incorporated correctly? Outside of the USA we are seeing CER being incorporated by groups such as NICE in the UK, Institute for Quality and Efficiency in Health Care in Germany and Pharmaceutical Benefits Advisory Committee in Australia. However, in the USA I would say that we are at the very early stages of CER implementation or its use in decision-making. Whether CER is being used appropriately is a very different question. I am concerned about finding the proper balance in CER implementation from what is ‘right’ for the population versus what is best for individuals. I do not think implementation at NICE or elsewhere has fully incorporated the needs of individual patients when assessments are made on whether to pay for a particular new therapy. At a population level, a new treatment may not meet a given cost–effectiveness threshold, but there may be individual patients who might receive tremendous benefit and they may not have the opportunity to receive that treatment. The classic saying is ‘do not throw the baby out with the bathwater’. I think if you do not carefully look at these issues then this may happen. QQ
How do you think it could be improved?
I think there is both additional research and new considerations on implementation that need to be carried out. On the research side, more work is needed on understanding the heterogeneity of response for individual treatments; that is, when The second area is the evolving payment and we perform CER, it is not nearly enough just to delivery environment, moving from a fee-for- carry out an average assessment, we need to look service world to one that instead provides eco- carefully at the individual subgroups and individnomic bundles to providers, and also has perfor- ual patients. One highly underused approach is mance assessments and bonuses based on quality. crossover designs, which are costly and often comWe are very focused on this performance-based plex but can provide us with important informareimbursement approach and understanding how tion about which patients will respond to a therapy. medications can influence the overall episode of A recent study examined patients with rheumatoid care cost, as well as how proper medication use arthritis and the authors compared biologic thermay reduce hospitalizations or other episode apy with a regimen of three oral drugs [1]. They
430
J. Compar. Effect. Res. (2013) 2(5)
future science group
Dubois
found that one in four patients did not respond to either therapy. However, when they crossed the patients over, many of those nonresponsive patients did respond to the alternative treatment. This study showed that although on average the various regimens seemed similar, for individual patients, there were important differences. The second improvement concerns the policies that are developed. Treatment policies need to consider subgroups; that certain treatment subgroups may receive particular benefit and should have an opportunity to receive therapies that may not be available to other subgroups that receive little benefit. The saying that ‘one size does not fit all’ is very important to keep in mind. What is the pharmaceutical industry’s perspective on CER? QQ
The pharmaceutical industry is very supportive of CER that is carried out correctly, and correctly means to consider heterogeneity as well as assessment of all the impacts on patients resulting from a treatment, including quality of life, functional ability, patient preferences and impact on the overall episode of care. A broad assessment of outcomes is very important. The industry is very supportive of looking at real-world experience and issues such as adherence to therapy, which may differ among treatments. You cannot truly understand that from a clinical trial where everything is tightly controlled, whereas in a real-world environment, you can begin to understand some of these issues. I am quite happy that the PatientCentered Outcomes Research Institute (Washington, DC, USA) has identified their priority topics very broadly. The concern of the industry was that CER would focus primarily on drug–drug comparisons, but, in fact, the vast majority of PatientCentered Outcomes Research Institute’s research funding announcements are focusing on all types of therapeutics, such as the benefits of surgery and different ways to deal with weight loss. What are you excited about working on over the next year? QQ
There has been a flurry of activity around defining ‘good’ CER and there are a number of standards or guidelines concerning how CER should be conducted. What we are concerned or excited about is the need to begin to look at those many different guidelines and come up with some consensus among them so that researchers and policy-makers can agree upon the characteristics of a good study. For example, if I conducted a study with one set of
future science group
INTERVIEW
guidelines, you could look at that study through the lens of another set of guidelines and say that my study was not carried out very well. With a harmonization of guidelines it will become clearer to the field what good looks like. What do you think will be the hot topics in CER over the next 5–10 years? QQ
The focus of the ‘Methods to policy’ piece in this issue of the Journal of Comparative Effectiveness Research, which McElwee and I wrote, describes an area of great interest [2]. There is substantial discussion about ‘big data’ and mining databases to understand the real world, and using those analyses to make clinical and policy decisions. There is a tremendous amount of enthusiasm around this, but we have concerns that the methods for conducting these studies are not yet fully mature and that we need to be cautious before we use these analyses to drive change. In the excitement surrounding rapid learning systems, we need to make sure that there is adequate independent review of what has been carried out and the validity of that since, in the rapid learning environment, there will not be a peer-review process like a manuscript would undergo. We are excited about the use of big data, but want that usage to drive appropriate change, not just any change. Disclaimer The opinions expressed in this interview are those of the interviewee and do not necessarily reflect the views of Future Medicine Ltd.
Financial & competing interests disclosure RW Dubois is an employee of the National Pharmaceutical Council, an industry-funded health policy research group that is not involved in lobbying or advocacy. He has no other 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 apart from those disclosed. No writing assistance was utilized in the production of this manuscript.
References 1
O’Dell JR, Mikuls TR, Taylor TH et al. CSP 551 RACAT Investigators. Therapies for active rheumatoid arthritis after methotrexate failure. N. Engl. J. Med. 369(4), 307–318 (2013).
2
McElwee NE, Dubois RW. Enthusiasm for rapid learning health systems exceeds the current standards for conducting it. J. Comp. Eff. Res. 2(5), 425–427 (2013).
www.futuremedicine.com
431
