Otolaryngology http://oto.sagepub.com/ -- Head and Neck Surgery
Individualized Clinical Practice Guidelines: The Next Step in the Evidence-based Health Care Evolution? Scott E. Brietzke Otolaryngology -- Head and Neck Surgery published online 10 January 2014 DOI: 10.1177/0194599813517864 The online version of this article can be found at: http://oto.sagepub.com/content/early/2014/01/10/0194599813517864 A more recent version of this article was published on - Feb 24, 2014
Published by: http://www.sagepublications.com
On behalf of:
American Academy of Otolaryngology- Head and Neck Surgery
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Commentary
Individualized Clinical Practice Guidelines: The Next Step in the Evidence-based Health Care Evolution?
Otolaryngology– Head and Neck Surgery 1–4 Ó American Academy of Otolaryngology—Head and Neck Surgery Foundation 2014 Reprints and permission: sagepub.com/journalsPermissions.nav DOI: 10.1177/0194599813517864 http://otojournal.org
Scott E. Brietzke, MD, MPH1
No sponsorships or competing interests have been disclosed for this article.
Abstract Clinical practice guidelines (CPGs) are playing an increasing role in the evolution of evidence-based health care. CPGs involve a process in which the best evidence in translated into best practice principles that are then applied on a wide range of patients. Although there are clear benefits in terms of simplicity, applying broad recommendations on a diverse group of patients can lead to wide variations in outcomes on an individual patient level. The individualized guideline (IG) is an approach that builds on the concept of the CPG by specifying treatment recommendation based on important individual baseline characteristics. The IG approach could potentially lead to superior outcomes but necessitates a substantial increase in complexity to the CPG development process. Keywords clinical practice guidelines, evidence-based health care, individual guidelines Received November 11, 2013; revised November 19, 2013; accepted December 3, 2013.
C
linical practice guidelines (CPG) are being produced with increasing frequency in all areas of health care. Otolaryngology is no exception, as the National Guidelines Clearinghouse (www.guideline.gov) as of September 2013 lists 83 guidelines under ‘‘Otolaryngology’’ that include all areas of the specialty, on topics such as otitis media, sleep apnea, and head and neck cancer. The CPG is the current pinnacle in the evolving quest of evidence-based health care. The simple goal of a CPG is to ‘‘help clinicians translate best evidence into best practice.’’1 This is accomplished by developing simple, direct clinical action statements stemming from a rigorous review of all of the available biomedical evidence. The goal of simplifying complex and sometime controversial patient care management decisions into concrete steps is useful and beneficial and can be expected to improve patient care outcomes. However, just as the widespread development and implementation of CPGs reflects the latest centerpiece of
the evolving process to continually improve patient care, one must wonder and look ahead to what will be the next step in this incremental, evolving process.
Clinical Practice Guidelines and the Ecological Fallacy CPGs can undoubtedly simplify and improve patient care at the population level. This is expected as the clinical action statements of the CPG are developed from the best available biomedical evidence that is inferred from the highest quality clinical studies (Figure 1). Clinical studies in turn are increasingly valued as they adhere to the most respected principle within the hierarchy of biomedical evidence: avoidance of bias and error (Centre of Evidence Based Medicine, www.cebm.net). This is best accomplished with the use of large randomized clinical trials to study and optimize knowledge of comparative treatment options. In this way, the discernment of what are the ‘‘best’’ treatment practices for the individual patient are determined from the study of large groups of patients. Enter the essential concept of the ecological fallacy: ‘‘The ecologic fallacy is a logical fallacy in the interpretation of statistical data where inferences about the nature of individuals are deduced from inference for the group to which those individuals belong.’’2 The ecological fallacy is not an ignored concept within the evidence-based health care context, and its acknowledgement was recognized early on as an essential component for an appropriate evidencebased approach. ‘‘Good doctors and health professionals use both individual clinical expertise and the best available external evidence, and neither alone is enough. Without clinical expertise, practice risks becoming tyrannized by evidence, for even excellent external evidence may be inapplicable to or inappropriate for an individual patient.’’3 Yet, 1
Walter Reed National Military Medical Center, Bethesda, Maryland, USA
The views herein are the private views of the author and do not reflect the official views of the Department of the Army or the Department of Defense. Corresponding Author: Scott E. Brietzke, MD, MPH, Dept. of Otolaryngology, Walter Reed National Military Medical Center, 8901 Wisconsin Ave. Bethesda, MD 20889, USA. Email: [email protected]
Downloaded from oto.sagepub.com at MEMORIAL UNIV OF NEWFOUNDLAND on August 3, 2014
2
Otolaryngology–Head and Neck Surgery XX(X)
Figure 1. Simple schematic showing the process for the development of a typical clinical practice guideline in which biomedical evidence is critically assessed and ‘‘averaged’’ leading to a homogeneous treatment recommendation for a wide range of patients.
as the evolution of evidence health care toward optimal care continues, what are the implications of the ecological fallacy in the development and implementation of CPGs?
Individualized Guidelines as a Potential Next Step toward Optimized Care Individualized guidelines (IG) could represent the next evolutionary step in the endless quest to achieve optimal patient care. Simple, uncomplicated CPGs are naturally easier to remember and easier to implement in real patient care scenarios.4 However, simplification often comes at a price. To produce simple action statements, individual patient baseline characteristics often have to be combined or averaged. The fact that the majority of patients in that averaged group will benefit from the given treatment remains true, but the reality that an individual patient’s benefit could be much more or much less than this average is also true.5 Although CPGs are thus undeniably valuable, they can be somewhat unrealistic in the context of direct application to individual patients and have been perhaps appropriately criticized in this light.6 The concept of an individualized guideline includes a process in which the baseline characteristics and risk factors of the individual patient are acknowledged and assessed and resulting specific, individualized treatment recommendations are thereby formulated. Individualized guidelines are not necessarily a radical new idea but really represent a more detailed and complex form of the current CPG process as depicted in Figure 1 versus Figure 2. Certainly the individualized guideline approach can be expected to increase data requirements and complicate the entire process of guideline development and implementation. This of course goes directly against the simplification principle of a useful
guideline discussed previously. However, could an individualized guideline approach lead to better outcomes at both the individual patient level and the population level than using the current ‘‘averaged’’ approach? There is no definite answer to this question (yet), but initial investigations into individualized guidelines suggest the potential gains of an individualized approach could be large. In an attempt to quantitatively assess the potential benefits of individualized guidelines, Eddy et al utilized prospectively collected cohort data and applied both the current accepted hypertension guidelines (Seventh Joint National Committee on Prevention, Detection, Evaluation, and Treatment of High Blood Pressure; JNC-7) and an individualized guideline approach using individualized treatment recommendation adjusted by baseline risk factors and assessed stoke and myocardial infarction (MI) outcomes between the different guideline approaches.4 They showed that an individualized guideline approach could either reduce stroke and MI by 43% at the same cost as the standard guidelines or reduce the same number of strokes and MI as the JNC-7 standard guidelines at a 67% overall cost saving. These are impressive results that can be expected to further increase interest in the individualized guideline approach with the omnipresent goal of improving treatment outcomes.
Individualized Guidelines and Otolaryngology What might an individualized guideline in otolaryngology look like? Consider a simplistic example using the recent tympanostomy tube CPG.7 This is a well-written and comprehensive CPG on a very common medical problem. The key action statements are succinct and plainly stated. They
Downloaded from oto.sagepub.com at MEMORIAL UNIV OF NEWFOUNDLAND on August 3, 2014
Brietzke
3
Figure 2. Simple schematic showing the process in which an individualized guideline is developed where important baseline risk factors and characteristics are accounted for in the development of individualized treatment recommendations. Clearly this process is more complicated than the typical clinical practice guideline but produces action statements that are tailored to important patient characteristics.
are intended to be applied to a wide range of pediatric patients presenting with otitis media. Although the importance of individual patient factors are clearly referenced in the tympanostomy tube guideline, a hypothetical individualized guideline might take a more direct approach to highlight the importance of these differences and how they could impact treatment decisions. Key action statement #8 from the tympanostomy tube CPG states: ‘‘Clinicians should determine if a child with recurrent AOM or with OME of any duration is at increased risk for speech, language, or learning problems from otitis media because of baseline sensory, physical, cognitive, or behavioral factors’’ with a supporting table listing these factors such as Autism-Spectrum Disorder, craniofacial syndromes, cleft palate, and so on.7 An individualized guideline would potentially integrate these factors into the other treatment recommendations. For example, a hypothetical individualized guideline might have an action statement that directly states ‘‘Clinicians should strongly consider placement of tympanostomy tubes at an early age in children with cleft palate to ensure optimal hearing during the critical years of speech and language development.’’ This statement in turn would be based on existing, high-quality evidence specifically regarding outcomes with cleft palate children and tympanostomy tube placement.8
Individual Guidelines and the Potential of Real-time Point of Care Impact As an increasing number of baseline risk factors is considered, patient assessment and its integration with evidence-based
individualized guidelines resultantly becomes exponentially complex and cumbersome. In fact, even producing an extensive individualized guideline in a publishable text format could prove difficult and become a limiting factor. This eventuality could be counteracted by developing automated risk calculators that assess and weight multiple baseline factors simultaneously and instantaneously before delivering individualized treatment recommendations. These ‘‘calculators’’ could furthermore be built directly into future electronic medical record system to further streamline the process in which the appropriate baseline patient factors are entered as part of the history and physical exam during the clinic visit and individualized, evidence-based treatment recommendations are immediately available to guide the clinician in real time. Time will tell whether the individualized guideline approach will produce enough additional benefit to be widely embraced. Critically important, unanswered questions, including ‘‘Is the increased complexity required for the individual guideline approach worth the effort?’’ and ‘‘Will an individual guideline approach actually lead to superior individual and population level outcomes?,’’ remain. Given the perpetual desire to improve outcomes, increase quality, and decrease costs, it is likely the individualized guideline approach will be embraced at some level and future assessments will provide data to address these critical questions. Individualized guidelines could ultimately end up being looked upon as simply the next evolution in evidence-based health care to produce higher quality guidelines that will increase quality of care and improve patient outcomes.
Downloaded from oto.sagepub.com at MEMORIAL UNIV OF NEWFOUNDLAND on August 3, 2014
4
Otolaryngology–Head and Neck Surgery XX(X)
Acknowledgment The commentary was solicited as part of the GIN Scholars Program.
Author Contributions Scott E. Brietzke, conception and design, drafting of article, final approval.
Disclosures Competing interests: None. Sponsorships: None. Funding source: None.
References 1. Richard RM, Shiffman RN, Robertson P.Clinical practice guideline development manual, third edition: a quality-driven approach for translating evidence into action. Otolaryngol Head Neck Surg. 2013;148:S1. 2. Ecological fallacy. http://en.wikipedia.org/wiki/Ecological_fallacy. Accessed December 11, 2013.
3. What is EMB?http://www.cebm.net/index.aspx?o=1914. Accessed December 11, 2013. 4. Eddy DW, Adler J, Patterson B, et al. Individualized guidelines: the potential for increasing quality and reducing costs. Ann Intern Med. 2011;154:627-634. 5. Kent DM, Hayward RA. Limitations of applying summary results of clinical trials to individual patients. JAMA. 2007;298: 1209-1212. 6. Sniderman AD, Furberg CD.Why guideline-making requires reform. JAMA. 2009;301:429-431. 7. Rosenfeld RM, Schwartz SR, Pynnonen MA, et al. Clinical practice guideline: tympanostomy tubes in children. Otolaryngol Head Neck Surg. 2013;149:S1. 8. Valtonen H, Dietz A, Qvarnberg Y. Long-term clinical, audiologic, and radiologic outcomes in palate cleft children treated with early tympanostomy for otitis media with effusion: a controlled prospective study. Laryngoscope. 2005;115:1512-1516.
Downloaded from oto.sagepub.com at MEMORIAL UNIV OF NEWFOUNDLAND on August 3, 2014
Individualized Clinical Practice Guidelines: The Next Step in the Evidence-based Health Care Evolution? Scott E. Brietzke Otolaryngology -- Head and Neck Surgery published online 10 January 2014 DOI: 10.1177/0194599813517864 The online version of this article can be found at: http://oto.sagepub.com/content/early/2014/01/10/0194599813517864 A more recent version of this article was published on - Feb 24, 2014
Published by: http://www.sagepublications.com
On behalf of:
American Academy of Otolaryngology- Head and Neck Surgery
Additional services and information for Otolaryngology -- Head and Neck Surgery can be found at:
P> OnlineFirst Version of Record - Jan 10, 2014 What is This?
Downloaded from oto.sagepub.com at MEMORIAL UNIV OF NEWFOUNDLAND on August 3, 2014
Commentary
Individualized Clinical Practice Guidelines: The Next Step in the Evidence-based Health Care Evolution?
Otolaryngology– Head and Neck Surgery 1–4 Ó American Academy of Otolaryngology—Head and Neck Surgery Foundation 2014 Reprints and permission: sagepub.com/journalsPermissions.nav DOI: 10.1177/0194599813517864 http://otojournal.org
Scott E. Brietzke, MD, MPH1
No sponsorships or competing interests have been disclosed for this article.
Abstract Clinical practice guidelines (CPGs) are playing an increasing role in the evolution of evidence-based health care. CPGs involve a process in which the best evidence in translated into best practice principles that are then applied on a wide range of patients. Although there are clear benefits in terms of simplicity, applying broad recommendations on a diverse group of patients can lead to wide variations in outcomes on an individual patient level. The individualized guideline (IG) is an approach that builds on the concept of the CPG by specifying treatment recommendation based on important individual baseline characteristics. The IG approach could potentially lead to superior outcomes but necessitates a substantial increase in complexity to the CPG development process. Keywords clinical practice guidelines, evidence-based health care, individual guidelines Received November 11, 2013; revised November 19, 2013; accepted December 3, 2013.
C
linical practice guidelines (CPG) are being produced with increasing frequency in all areas of health care. Otolaryngology is no exception, as the National Guidelines Clearinghouse (www.guideline.gov) as of September 2013 lists 83 guidelines under ‘‘Otolaryngology’’ that include all areas of the specialty, on topics such as otitis media, sleep apnea, and head and neck cancer. The CPG is the current pinnacle in the evolving quest of evidence-based health care. The simple goal of a CPG is to ‘‘help clinicians translate best evidence into best practice.’’1 This is accomplished by developing simple, direct clinical action statements stemming from a rigorous review of all of the available biomedical evidence. The goal of simplifying complex and sometime controversial patient care management decisions into concrete steps is useful and beneficial and can be expected to improve patient care outcomes. However, just as the widespread development and implementation of CPGs reflects the latest centerpiece of
the evolving process to continually improve patient care, one must wonder and look ahead to what will be the next step in this incremental, evolving process.
Clinical Practice Guidelines and the Ecological Fallacy CPGs can undoubtedly simplify and improve patient care at the population level. This is expected as the clinical action statements of the CPG are developed from the best available biomedical evidence that is inferred from the highest quality clinical studies (Figure 1). Clinical studies in turn are increasingly valued as they adhere to the most respected principle within the hierarchy of biomedical evidence: avoidance of bias and error (Centre of Evidence Based Medicine, www.cebm.net). This is best accomplished with the use of large randomized clinical trials to study and optimize knowledge of comparative treatment options. In this way, the discernment of what are the ‘‘best’’ treatment practices for the individual patient are determined from the study of large groups of patients. Enter the essential concept of the ecological fallacy: ‘‘The ecologic fallacy is a logical fallacy in the interpretation of statistical data where inferences about the nature of individuals are deduced from inference for the group to which those individuals belong.’’2 The ecological fallacy is not an ignored concept within the evidence-based health care context, and its acknowledgement was recognized early on as an essential component for an appropriate evidencebased approach. ‘‘Good doctors and health professionals use both individual clinical expertise and the best available external evidence, and neither alone is enough. Without clinical expertise, practice risks becoming tyrannized by evidence, for even excellent external evidence may be inapplicable to or inappropriate for an individual patient.’’3 Yet, 1
Walter Reed National Military Medical Center, Bethesda, Maryland, USA
The views herein are the private views of the author and do not reflect the official views of the Department of the Army or the Department of Defense. Corresponding Author: Scott E. Brietzke, MD, MPH, Dept. of Otolaryngology, Walter Reed National Military Medical Center, 8901 Wisconsin Ave. Bethesda, MD 20889, USA. Email: [email protected]
Downloaded from oto.sagepub.com at MEMORIAL UNIV OF NEWFOUNDLAND on August 3, 2014
2
Otolaryngology–Head and Neck Surgery XX(X)
Figure 1. Simple schematic showing the process for the development of a typical clinical practice guideline in which biomedical evidence is critically assessed and ‘‘averaged’’ leading to a homogeneous treatment recommendation for a wide range of patients.
as the evolution of evidence health care toward optimal care continues, what are the implications of the ecological fallacy in the development and implementation of CPGs?
Individualized Guidelines as a Potential Next Step toward Optimized Care Individualized guidelines (IG) could represent the next evolutionary step in the endless quest to achieve optimal patient care. Simple, uncomplicated CPGs are naturally easier to remember and easier to implement in real patient care scenarios.4 However, simplification often comes at a price. To produce simple action statements, individual patient baseline characteristics often have to be combined or averaged. The fact that the majority of patients in that averaged group will benefit from the given treatment remains true, but the reality that an individual patient’s benefit could be much more or much less than this average is also true.5 Although CPGs are thus undeniably valuable, they can be somewhat unrealistic in the context of direct application to individual patients and have been perhaps appropriately criticized in this light.6 The concept of an individualized guideline includes a process in which the baseline characteristics and risk factors of the individual patient are acknowledged and assessed and resulting specific, individualized treatment recommendations are thereby formulated. Individualized guidelines are not necessarily a radical new idea but really represent a more detailed and complex form of the current CPG process as depicted in Figure 1 versus Figure 2. Certainly the individualized guideline approach can be expected to increase data requirements and complicate the entire process of guideline development and implementation. This of course goes directly against the simplification principle of a useful
guideline discussed previously. However, could an individualized guideline approach lead to better outcomes at both the individual patient level and the population level than using the current ‘‘averaged’’ approach? There is no definite answer to this question (yet), but initial investigations into individualized guidelines suggest the potential gains of an individualized approach could be large. In an attempt to quantitatively assess the potential benefits of individualized guidelines, Eddy et al utilized prospectively collected cohort data and applied both the current accepted hypertension guidelines (Seventh Joint National Committee on Prevention, Detection, Evaluation, and Treatment of High Blood Pressure; JNC-7) and an individualized guideline approach using individualized treatment recommendation adjusted by baseline risk factors and assessed stoke and myocardial infarction (MI) outcomes between the different guideline approaches.4 They showed that an individualized guideline approach could either reduce stroke and MI by 43% at the same cost as the standard guidelines or reduce the same number of strokes and MI as the JNC-7 standard guidelines at a 67% overall cost saving. These are impressive results that can be expected to further increase interest in the individualized guideline approach with the omnipresent goal of improving treatment outcomes.
Individualized Guidelines and Otolaryngology What might an individualized guideline in otolaryngology look like? Consider a simplistic example using the recent tympanostomy tube CPG.7 This is a well-written and comprehensive CPG on a very common medical problem. The key action statements are succinct and plainly stated. They
Downloaded from oto.sagepub.com at MEMORIAL UNIV OF NEWFOUNDLAND on August 3, 2014
Brietzke
3
Figure 2. Simple schematic showing the process in which an individualized guideline is developed where important baseline risk factors and characteristics are accounted for in the development of individualized treatment recommendations. Clearly this process is more complicated than the typical clinical practice guideline but produces action statements that are tailored to important patient characteristics.
are intended to be applied to a wide range of pediatric patients presenting with otitis media. Although the importance of individual patient factors are clearly referenced in the tympanostomy tube guideline, a hypothetical individualized guideline might take a more direct approach to highlight the importance of these differences and how they could impact treatment decisions. Key action statement #8 from the tympanostomy tube CPG states: ‘‘Clinicians should determine if a child with recurrent AOM or with OME of any duration is at increased risk for speech, language, or learning problems from otitis media because of baseline sensory, physical, cognitive, or behavioral factors’’ with a supporting table listing these factors such as Autism-Spectrum Disorder, craniofacial syndromes, cleft palate, and so on.7 An individualized guideline would potentially integrate these factors into the other treatment recommendations. For example, a hypothetical individualized guideline might have an action statement that directly states ‘‘Clinicians should strongly consider placement of tympanostomy tubes at an early age in children with cleft palate to ensure optimal hearing during the critical years of speech and language development.’’ This statement in turn would be based on existing, high-quality evidence specifically regarding outcomes with cleft palate children and tympanostomy tube placement.8
Individual Guidelines and the Potential of Real-time Point of Care Impact As an increasing number of baseline risk factors is considered, patient assessment and its integration with evidence-based
individualized guidelines resultantly becomes exponentially complex and cumbersome. In fact, even producing an extensive individualized guideline in a publishable text format could prove difficult and become a limiting factor. This eventuality could be counteracted by developing automated risk calculators that assess and weight multiple baseline factors simultaneously and instantaneously before delivering individualized treatment recommendations. These ‘‘calculators’’ could furthermore be built directly into future electronic medical record system to further streamline the process in which the appropriate baseline patient factors are entered as part of the history and physical exam during the clinic visit and individualized, evidence-based treatment recommendations are immediately available to guide the clinician in real time. Time will tell whether the individualized guideline approach will produce enough additional benefit to be widely embraced. Critically important, unanswered questions, including ‘‘Is the increased complexity required for the individual guideline approach worth the effort?’’ and ‘‘Will an individual guideline approach actually lead to superior individual and population level outcomes?,’’ remain. Given the perpetual desire to improve outcomes, increase quality, and decrease costs, it is likely the individualized guideline approach will be embraced at some level and future assessments will provide data to address these critical questions. Individualized guidelines could ultimately end up being looked upon as simply the next evolution in evidence-based health care to produce higher quality guidelines that will increase quality of care and improve patient outcomes.
Downloaded from oto.sagepub.com at MEMORIAL UNIV OF NEWFOUNDLAND on August 3, 2014
4
Otolaryngology–Head and Neck Surgery XX(X)
Acknowledgment The commentary was solicited as part of the GIN Scholars Program.
Author Contributions Scott E. Brietzke, conception and design, drafting of article, final approval.
Disclosures Competing interests: None. Sponsorships: None. Funding source: None.
References 1. Richard RM, Shiffman RN, Robertson P.Clinical practice guideline development manual, third edition: a quality-driven approach for translating evidence into action. Otolaryngol Head Neck Surg. 2013;148:S1. 2. Ecological fallacy. http://en.wikipedia.org/wiki/Ecological_fallacy. Accessed December 11, 2013.
3. What is EMB?http://www.cebm.net/index.aspx?o=1914. Accessed December 11, 2013. 4. Eddy DW, Adler J, Patterson B, et al. Individualized guidelines: the potential for increasing quality and reducing costs. Ann Intern Med. 2011;154:627-634. 5. Kent DM, Hayward RA. Limitations of applying summary results of clinical trials to individual patients. JAMA. 2007;298: 1209-1212. 6. Sniderman AD, Furberg CD.Why guideline-making requires reform. JAMA. 2009;301:429-431. 7. Rosenfeld RM, Schwartz SR, Pynnonen MA, et al. Clinical practice guideline: tympanostomy tubes in children. Otolaryngol Head Neck Surg. 2013;149:S1. 8. Valtonen H, Dietz A, Qvarnberg Y. Long-term clinical, audiologic, and radiologic outcomes in palate cleft children treated with early tympanostomy for otitis media with effusion: a controlled prospective study. Laryngoscope. 2005;115:1512-1516.
Downloaded from oto.sagepub.com at MEMORIAL UNIV OF NEWFOUNDLAND on August 3, 2014
