Background and Key Stakeholders in Guidelines Development and Use
Abstract: Before specific consideration of standards for trustworthy clinical practice guidelines (CPGs), the committee examined the history of evidence-based medicine and guideline development. This chapter provides a brief review of modern applications of scientific evidence in the development of clinical care and evolution of CPGs in the United States and internationally, as well as a review of the major guideline developers and users today. The chapter is by no means an exhaustive history of guideline development or a complete list of current stakeholders; instead it aims to present the reader with a general overview of the guideline landscape.
“Clinical practice guidelines are now ubiquitous,” observed Weisz and colleagues in 2007 (p. 691). The Guidelines International Network database currently lists more than 3,700 guidelines from 39 countries. Its U.S.-based counterpart, the National Guideline Clearinghouse (NGC), accepted 722 guidelines to its database in 2008 alone, bringing its total collection to nearly 2,700.1 Furthermore, numerous other clinical guidance statements (e.g., the Centers for Disease Control and Prevention [CDC] rapid response recommen-
dations) and tools (e.g., computerized physician order entry systems) are created to aid clinical care decisions each year. As stated in Chapter 1, clinical practice guideline (CPG) development has evolved dramatically in the 20 years since the Institute of Medicine (IOM) first became involved in this area. This chapter provides a brief review of modern applications of scientific evidence in the development of clinical care and evolution of CPGs. It then offers an overview of many participants in current CPG development and use, providing selected examples.
Healthcare Decision Making Prior to Evidence-Based Medicine (EBM)
Before the end of the 20th century, clinical decisions were based largely on experience and skill (the “art” of medicine); medical teaching and practice were dominated by knowledge delivered by medical leaders (Davidoff, 1999; Eddy, 2005; Evidence-Based Medicine Working Group et al., 1992). Although some form of evidence has long contributed to clinical practice, there was no generally accepted, formal way of ensuring a scientific, critical approach to clinical decision making (Daly, 2005). The 1992 Evidence-Based Medicine Working Group, primarily McMaster University professors, who created a training program to teach EBM to internal medicine residents, described the historical paradigm of medical decision in the following sentences:
“Unsystematic observations from clinical experience are a valid way of building and maintaining one’s knowledge about patient prognosis, the value of diagnostic tests, and the efficacy of treatment.
The study and understanding of basic mechanisms of disease and pathophysiologic principles are a sufficient guide for clinical practice.
A combination of thorough traditional medical training and common sense is sufficient to allow one to evaluate new tests and treatments.
Content expertise and clinical experience are a sufficient base from which to generate valid guidelines for clinical practice.” (Evidence-Based Medicine Working Group et al., 1992, p. 2421)
The modern commitment to EBM dates to the 1970s, when a growing body of health services research refuted long-held assump-
tions about the quality of medical care practiced by U.S. physicians. In 1973, John Wennberg documented wide variations in practice patterns among local provider communities (hospital market areas) with seemingly similar patient populations (Wennberg and Gittelsohn, 1973). Then, the RAND Health Services Utilization Study showed that three common procedures (coronary angiography, carotid endarterectomy, and upper gastrointestinal tract endoscopy) were applied inappropriately at a rate of 17, 32, and 17 percent, respectively (Chassin et al., 1987; Davidoff, 1999). Around the same time, a major study in the New England Journal of Medicine (NEJM) concluded that indications for permanent pacemakers were inadequate or undocumented for 20 percent (77 of 382) of implant cases (Greenspan et al., 1988). To many health policy leaders, the RAND, NEJM, and other complementary investigational findings demonstrated that large proportions of procedures performed by physicians were deemed inappropriate even by experts in associated fields, and that one quarter to one third of all medical care may be unnecessary (Eddy, 2005; Woolf, 1990). In 1990, Steven Woolf wrote in reaction to the above findings that “the perception is that at least some of the variation reflects excessive (or inadequate) use of procedures by physicians in certain areas” (Woolf, 1990, p. 1812).
The Expansion of the Evidence Base
The first randomized controlled trial (RCT) in the health sciences was published in 1948 and demonstrated the efficacy of streptomycin in the treatment of tuberculosis. Since the 1970s there has been an exponential increase in RCTs and in observational research. From 1978 to 2001, 8.1 million journal articles were indexed in MEDLINE, with nearly half occurring from 1994 to 2001. The proportion of MEDLINE RCT articles also grew, from 1.9 percent or 5,174 per year from 1978 to 1985, to 6.2 percent or 24,724 per year from 1994 to 2001 (Druss and Marcus, 2005). Health sciences literature growth was concentrated in clinical research, with an increase in the percentage of studies with human subjects, and Medical Subject Headings (MeSH) in MEDLINE that shifted from basic science to clinical care and public health (Druss and Marcus, 2005). The introduction in the mid-1990s of the ACP (American College of Physicians) Journal Club and the journal Evidence-Based Medicine, which contain quality-evaluated article abstracts selected from hundreds of primary publications, illustrated the dramatic increase in the evidence base and its intended use among practicing physicians (Daly, 2005).
Exploitation of observational research methods as supplements to the RCT has long held traction among EBM’s top proponents. Observational in this context refers exclusively to “quantitative, epidemiological methods and not qualitative, sociological methods. The principal observational epidemiological methods are non-randomized trials, cohort studies (prospective and retrospective), and case control methods,” wrote Black in 1996 (p. 1215). Experimentation may be inadequate as an evidentiary base for clinical practice due to questionable external validity. More recently, Berwick and others have asserted that healthcare has much to gain if the view of EBM is broadened to include sources of observational data such as registries and electronic health records (Berwick, 2005). Overall, study designs and methods of analysis in support of EBM have become increasingly more sophisticated, and now include decision analysis, systematic review of the literature (including meta-analysis), and cost-effectiveness analysis (IOM, 2001).
This increase in knowledge was accompanied by a broad pattern of decentralization, both in sources of funding and authorship. The largest source of nongovernmental funding, by far, was industry in order to support Food and Drug Administration (FDA) approvals of drugs and devices (Druss and Marcus, 2005).
Clinical Epidemiology and Evidence-Based Medicine
Clinical epidemiology and evidence-based medicine emerged as solutions to failings of the traditional approach to medical decision making. Alvan Feinstein and David Sackett were the first to introduce clinical epidemiology as a distinct clinical discipline at Yale University (1968) and McMaster University (1970) respectively. In the late 20th century, the field of clinical epidemiology, defined by Sackett as “the application, by a physician who provides direct patient care, of epidemiologic and biostatistical methods to the study of diagnostic and therapeutic processes in order to effect an improvement in health” (Sackett, 2002, p. 1162), not only survived, but thrived, and its leaders were placed in highly influential positions in departments of medicine, journal editorships, and professional societies (Berwick, 2005).
Major figures in the rise of EBM included Archie Cochrane, Iain Chalmers, Murray Enkin, and Mark Keirse. Cochrane, a British epidemiologist, promoted the RCT as the best means of assessing medical technologies and practices, as early as the 1950s and 1960s. His work later gave rise to the Cochrane Collaboration (IOM, 2001). Chalmers, Enkin and colleagues, created the Oxford Database of
Perinatal trials, which compiled, first in hard copy (1988) and then in an electronic version (1990), all clinical trials data pertaining to effective care in pregnancy and childbirth (Chalmers, 1988, 1990). In addition, Chalmers, Enkin, and colleagues published Effective Care in Pregnancy and Childbirth, an overview of best pregnancy and childbirth evidence with a compendium of systematic reviews, and corresponding summary guide of the results. This work inspired the establishment of the Cochrane Collaboration, described later in this chapter (Chalmers et al., 1989; Enkin et al., 1989). These leaders, as well as others, fostered a new generation of scholars in healthcare, who focused their careers on clinical practice research (Berwick, 2005). “As a result, over the last several decades, the standards for evidence have become more stringent, and the tools for its assembly and analysis have become more powerful and widely available,” according to Davidoff (1999) (IOM, 2001, p. 147).
The term “evidence-based medicine,” coined in 1990, is defined by Daly as “the application of scientific method in determining the optimal management of the individual patient” (Daly, 2005, p. 89). In 1992 the EBM Working Group described the emergent paradigm of Evidence-Based Clinical Decision Making:
While clinical experience and skill are important, systematic attempts to record observations in a reproducible and unbiased fashion markedly increase the confidence one can have in knowledge about patient prognosis, the value of diagnostic tests, and the efficacy of treatment.
In the absence of systematic observation, one must be cautious in the interpretation of information derived from clinical experience and intuition, for it may at times be misleading.
The study and understanding of basic mechanisms of disease are necessary but insufficient guides for clinical practice.
Understanding certain rules of evidence is necessary to correctly interpret literature on causation, prognosis, diagnostic tests, and treatment strategy. (Evidence-Based Medicine Working Group et al., 1992, p. 2421)
Additionally, the EBM Working Group asserted that clinicians must accept uncertainty and the notion that clinical decisions are often made with scant knowledge of their true impact. The EBM paradigm assigns reduced weight to authority of experts, instead valuing physician understanding of underlying rigorous, high quality scientific evidence in patient care provision (Evidence-Based
Medicine Working Group et al., 1992). Importantly, more recent definitions also emphasize that clinical expertise and patient preferences remain vital to clinical decision making, and clarify that scientific evidence refers not only to RCT findings, but to those arising from research designs such as nonrandomized cohort trials and case control studies (IOM, 2001). The development of CPGs, detailed in the next section, is an important extension of EBM (Luce et al., 2010).
Evolution of Clinical Practice Guideline Development
Clinical practice guidelines (CPGs) have enjoyed a presence in medical practice since the early 20th century; many sources cite the American Academy of Pediatrics’ Redbook of Infectious Diseases (1938) as one of the first CPGs produced in the United States (IOM, 2008). Most early guidelines were developed by expert panels or individuals who had gained authority status within specific medical specialties. As research evidence in support of, and methodologies for implementing, EBM had yet to be developed, these CPGs rarely were informed by systematic interpretation of the scientific evidence (IOM, 2008).
By the 1990s, the evolution in research methods and expansion of the scientific evidence base detailed above increased both the need for and ability of CPGs to reflect the latest EBM trend. Physicians could no longer keep up with the growing knowledge base: An internist would have to read 33 articles 365 days a year to stay up to date (Sackett, 2002). Furthermore, the validity of much of the growing body of evidence was suspect. “These two situations combined to place clinicians at increasing risk of ‘drowning in doubtful data,’ ” Sackett wrote in 2002 (p. 1164). Critically appraised, synthesized information such as systematic reviews and CPGs became necessary tools for clinicians desiring to practice EBM (IOM, 2008). The IOM entered the conversation during this time with the two previously mentioned (in Chapter 1) reports, Clinical Practice Guidelines: Directions for a New Program (1990) and Guidelines for Clinical Practice: From Development to Use (1992) (IOM, 1990, 1992), which were requested by Congress to help inform a new government entity, then called the Agency for Healthcare Policy and Research (AHCPR), tasked with CPG development. David Eddy, an early pioneer in guideline development methods, influenced the work of AHCPR, Blue Cross-Blue Shield Association’s TEC program, as well as others. His book, A Manual for Assessing Health Practices and Designing Practice Policies: The Explicit Approach, established a foundation for patient-centered
outcomes based guideline development in the United States (Eddy, 1992).
Early groups applying systematic evidence reviews to CPG recommendations were the Canadian Task Force on the Periodic Health Examination (1976), the U.S. Preventive Services Task Force (USPSTF) (1984), and the American College of Physicians Clinical Efficacy Assessment Project (1980) (IOM, 2008). The involvement of specialty societies in practice guidelines development increased dramatically throughout the 1980s and 1990s. In 1990 Steven Woolf stated that, “Societies with previously limited activity have now revised their internal organizational structure, have established new committees to develop guidelines, and have adopted formal procedures to guide their efforts” (Woolf, 1990, p. 1814). By 1989, more than 35 medical societies and physician organizations had developed at least one CPG. Umbrella organizations such as the American Medical Association (AMA) and the Council of Medical Specialty Societies also became active participants in the guideline development movement, mostly in the role of coordinators and guideline development process “standardizers” (Woolf, 1990).
While U.S. federal agencies such as the USPSTF and the National Institutes of Health’s (NIH’s) Consensus Development Program had been making practice recommendations, federal efforts increased dramatically in 1989, when Congress passed a series of bills that increased funding for “effectiveness research” and called for creation of a formal public health service agency with responsibility for development and dissemination of CPGs (Woolf, 1990). Organized medicine, including the AMA and many medical specialty societies, embraced the legislation and employment of guidelines as alternatives to proposed stricter expenditure targets, endorsed by the George H. W. Bush Administration and certain congressional leaders, for curbing increases in healthcare spending (Woolf, 1990). The newly created AHCPR developed approximately 20 guidelines across a wide spectrum of clinical areas over the next several years (Lohr et al., 1998). The AHCPR’s direct involvement in CPG development was short lived, however, due to political opposition in 1995 from back surgeons who disagreed with the agency’s guidelines for the treatment of lower back pain (IOM, 2009). Following congressional threats to withdraw its funding, the agency, renamed the Agency for Healthcare Research and Quality (AHRQ), limited its responsibilities to financial support of Evidence-based Practice Centers’ (EPCs’) production of systematic reviews. In 2008, AHRQ’s 14 EPCs produced systematic reviews that government agencies (e.g., the NIH and USPSTF), professional societies, and other organiza-
tions could access in their development of CPGs. AHRQ remains involved in guideline dissemination via the National Guideline Clearinghouse, a web-based collection of CPGs from around the world. These activities are discussed in the upcoming section.
CURRENT MAJOR STAKEHOLDERS IN GUIDELINES DEVELOPMENT AND USE
As the preceding background section indicates, numerous stakeholders are involved in the development and use of CPGs. The next two sections discuss examples from stakeholder classes: government agencies, clinical specialty societies, disease-specific societies, and other private and international organizations that develop guidelines and are key players. The examples illustrate the variety of organizations involved with CPGs, and are not intended to be all-inclusive. Also, the examples focus on organizations’ CPG-related activities and do not provide a full description of each organization as a whole.
A few national agencies have contributed significantly to guideline development, both financially and intellectually (NGC, 2010d). The USPSTF has been a CPG developer since the 1980s. It is noted for its early use of transparent standards and inclusion of multidisciplinary experts in the development process. This body was created by the U.S. Public Health Service in 1984 and has been supported by AHRQ staff and funding since 1998. The USPSTF is composed mainly of individuals from primary care specialties, such as internists, pediatricians, nurses, family physicians, methodologists, and some subspecialists. Its preventive care guidelines include topics for screening, counseling, immunizations, and preventive medications and services (AHRQ, 2010a). Because of its prevention focus, its perspective reflects that of primary care for asymptomatic people (Atkins, 2010). The USPSTF relies on systematic reviews (SRs) of relevant literature conducted by AHRQ staff, AHRQ’s EPCs, or outside experts (AHRQ, 2010b). It has 60 CPGs in the NGC (NGC, 2010b).
The USPSTF updates its CPGs at least every 5 years (AHRQ, 2010b). To promote adoption of its guidelines, the USPSTF has developed relationships with a group of partners, including primary care associations and specialty societies; policy, quality improvement,
and population groups (e.g., AARP); and selected federal agencies. These organizations serve as technical experts, peer reviewers, and disseminators (AHRQ, 2010a).
The NIH also has been involved heavily with guidelines and related endeavors through some of its institutes and programs, as part of its mission to translate basic research into medical practice (Simons-Morton, 2010). For example, the National Heart, Lung, and Blood Institute has 12 guidelines listed in the NGC as of October 2009 (NGC, 2010b) and posts 7 CPGs covering 5 health conditions on its website (NHLBI, 2009). It lists four new guidelines and one guideline update in development.
The previously mentioned Consensus Development Program of NIH issues three to five major evidence-based consensus statements annually, extending from highly structured conferences on controversial clinical topics of use to the broad medical community and general public (NIH, 2009). Many aspects of this consensus development process are similar to that used in development of high-quality CPGs: use of an unbiased, independent, expert panel including research investigators, health professionals, methodologists, and representatives of the public without conflicts of interest; a systematic review conducted by AHRQ; and opportunities for public input. Although the statement is not updated after issuance, after 5 years it is considered “historical” and the assumption is that much of the content is of questionable validity. Although the consensus statement may prompt reassessment of medical practice, it differs from a CPG in that it merely synthesizes the latest information, often from current and ongoing medical research; it does not recommend specific clinical actions in particular circumstances. The existence of a current Consensus Statement could be important to the evidence base of a CPG. In fact, the NIH currently posts three such statements in the NGC (NGC, 2010b).
The CDC is a major issuer of guidelines. The CDC Prevention Guidelines database includes more than 400 guidelines for the prevention and control of public health threats such as AIDS, cholera, disasters, dengue fever, suicide, vaccine-preventable diseases, lung cancer, sexually transmitted diseases, birth defects, and malaria. About two thirds of the documents in the database were originally published in the CDC’s Morbidity and Mortality Weekly Report. The others were published as CDC monographs, as books or book chapters, as brochures, or as articles in peer-reviewed journals (CDC, 2007). The CDC currently lists 82 CPGs in the NGC (NGC, 2010b). These guidelines are generally based on a systematic review of the literature and written in consultation with experts in the germane
field. The CDC also produces many recommendations in response to rapidly evolving public health concerns, such as the influenza epidemic. Given the urgent nature of these issues, these recommendations are rarely based on a systematic review of the evidence2 (Briss, 2010).
The Department of Defense (DoD) and the Veterans Administration (VA) develop guidelines for their unique populations, and undertake efforts to implement them within their health systems and cost constraints, while also employing CPGs derived by others (Atkins, 2010). The VA and DoD list 25 CPGs in the NGC, predominantly directed to primary care conditions. The VA uses guidelines to improve quality of care and minimize unjustified variation in practice across geographic areas. Sometimes the VA embeds CPGs in its electronic health record and uses the CPGs to develop clinical performance measures (Atkins, 2010).
In addition to federal agencies, some states, such as Massachusetts, Washington, and New York, have taken responsibility for developing guidelines for their Medicaid and other state programs (NGC, 2010b).
Clinical Specialty Societies
Many medical societies and organizations of other healthcare professionals have taken responsibility for CPG development in responding to members’ requests for guidance on best treatment practices and as an opportunity to provide continuing education. Some societies, such as the American College of Cardiology, or ACC (see discussion below on the American Heart Association, or AHA), are characterized by long histories of CPG development, have established development procedures, and have devoted significant financial resources to underwrite comprehensive SRs, carefully craft guidelines, and promote their implementation. The American Thoracic Society and American College of Physicians are similarly dedicated to development of high-quality guidelines and have recently updated their conflict-of-interest and rating of evidence and recommendation policies respectively, to reflect the latest consensus on that topic (Guyatt et al., 2010; Qaseem et al., 2010). Other societies have more limited resources for guideline development, less experience, and produce fewer CPGs. Most societies work independently on guidelines, although they may address the same conditions and
develop related CPGs. For example, an AHA guideline may have a recommendation for blood pressure treatment in persons with diabetes, as do the American College of Physicians and the American Diabetes Association (Kahn, 2010).
Some societies have collaborated in the development of CPGs, integrating contributions of multiple specialties in the treatment of particular medical conditions. For example, many of the American Society for Clinical Oncology (ASCO) guidelines were developed in partnership with other specialty societies, such as the American Society of Hematology and the College of American Pathologists. ASCO has at least 17 guidelines in the NGC (NGC, 2010b).
Non-physician clinical societies such as occupational and physical therapists and many nursing specialties develop CPGs as well. For example, in 1997 the American Association of Neuroscience Nurses (AANN) created a series of educational tools to aid in patient care, called the AANN Reference Series for Clinical Practice; the series’ name changed in 2007 to the AANN Clinical Practice Guideline Series to reflect the evidence-based nature of the guides. The AANN currently has 10 guidelines downloadable on its website and 5 CPG summaries in the NGC (AANN, 2010; NGC, 2010b).
Disease- or Population-Specific Organizations
Individual disease- or population-specific organizations, some from beyond U.S. borders and some in conjunction with other professional or disease-specific societies, sponsored 202 guidelines in the NGC as of March 2009 (IOM, 2009). The breadth of focus of disease-specific societies as well as their resources and capacity for CPG production vary widely.
For example, the Alzheimer’s Association has produced three guidelines currently listed in NGC, including one entitled Dementia Care Practice Recommendations for Assisted Living Residences and Nursing Homes: Phase 3 End-of-Life Care (NGC, 2010a). This guideline is based on a literature review sponsored by the association and conducted by a consultant, a background paper written by association staff, and a 1-day meeting of representatives from 30 national provider, professional, and consumer advocacy organizations. The guideline’s recommendations reflect expert consensus and are directed at professionals rather than patients and informal caregivers.3
AHA has had a unique relationship with a specialty society, the ACC since 1981 (Jacobs, 2010). Through this partnership the AHA/ACC developed their first joint CPG in 1984. Twenty-two jointly sponsored CPGs are currently available; five new CPGs are in production and five earlier ones are being revised or updated. The guidelines focus on diagnostic procedures, therapeutic interventions, and management therapies for cardiovascular disease. For some of these guidelines, the collaboration has expanded to include other groups, such as the Heart Rhythm Society, European Society of Cardiology (ESC), Society of Cardiovascular Angiography and Interventions, Society of Thoracic Surgeons, and others. The AHA/ACC also gathers guidelines’ endorsements from related organizations to enhance implementation (Jacobs, 2010).
The AHA/ACC guideline development effort is detailed and comprehensive, each requiring an average of more than 2 years to produce and publish; 22 current guidelines include more than 3,000 recommendations, and final products run more than 350 pages. To inform this process, the AHA has published a manual of methodologies and policies. Also, as part of their quality improvement and guideline implementation efforts, the AHA and ACC develop individual physician performance measures based on guideline recommendations (AHA, 2010b).
The American Geriatrics Society has developed three guidelines since 2003, most recently in 2009 on prevention of falls in older persons (an update from 2001). The multidisciplinary panel that developed this update was led jointly by representatives of the American Geriatrics Society and the British Geriatrics Society. Panel participants included members of the American Academy of Orthopedic Surgeons, American Board of Internal Medicine, American College of Emergency Physicians, American Geriatrics Society, American Medical Association, American Occupational Therapy Association, American Physical Therapy Association, American Society of Consultant Pharmacists, British Geriatrics Society, John A. Hartford Foundation Institute for Geriatric Nursing at New York University, and National Association for Home Care and Hospice (AGS, 2010).
Other Private Organizations
Other private organizations, such as large healthcare organizations, academic medical centers, quality improvement organizations, and commercial companies, also develop CPGs. They draw, in part, on existing CPGs from respected specialty societies and related rigorous SRs, but involve their own expert staff and physicians in
evidence review and recommendations derivation befitting their patient and clinician contexts.
For example, Kaiser Permanente staffs a centralized, internal organizational structure dedicated to CPG development. It was originally developed by David Eddy with an explicit standardized methodology, critical appraisal and grading of evidence, and a guideline quality committee of methodologists. It maintains a core set of 19 CPGs related to preventive care and chronic conditions, 8 of which are currently listed in the NGC (Koster, 2010). The Institute for Clinical Systems Improvement (ICSI) produces many CPGs through collaboration in Minnesota of 10,000 predominantly primary care doctors, most hospitals, not-for-profit health plans, and some employers and patients. The NGC includes summaries of 41 ICSI guidelines (Bottles, 2010). (ICSI’s CPG efforts are further detailed in Chapter 6.)
Limited public information about CPGs is produced commercially. The following description is based on information available on the web and a presentation by, and committee discussion with, the editor-in-chief of Milliman’s guidelines. McKesson, a large corporation providing services to healthcare providers and insurers, produces clinical practice guidelines in its Interqual Division. These are the two main purveyors of commercial guidelines.
These proprietary guidelines reportedly concentrate on quality of care, efficient resource expenditure, and reduction in inappropriate care variations. Guidelines are founded on evidence when available and expert opinion when not. What distinguishes these guidelines from most publicly available CPGs is promotion of use through accompanying software that integrates behavior change into real-time management reports usage reviews, workflow and resource controls, and decision tools devoted to quality improvement and cost efficiency. Often providers’ own data as well as larger databases can be integrated for feedback and benchmarking. Both companies provide training and support of clients and are available for additional consulting. The guideline companies support research staffs to continuously mine the literature and consultants to review and revise draft guidelines and provide expert advice and consensus on recommendations when evidence is lacking4 (McKesson Health Solutions LLC, 2004; Milliman Care Guidelines, 2009; Milliman Inc., 2009; Schibanoff, 2010).
Organizations in several countries outside the United States also produce CPGs. Foreign organizations have contributed 895 (36 percent) of the CPGs in the U.S. NGC. Such guidelines may be developed by foreign medical societies, disease organizations, or government-related bodies. Although the attentions of this committee did not extend internationally, several organizations merit highlighting because of the important roles they play.
For example, the National Institute for Health and Clinical Excellence (NICE) is an independent organization that advises the UK National Health Service (NHS) on promoting health and preventing and treating illness (NICE, 2010). It conducts or contracts for technology assessments of new treatments and devices as well as systematic reviews and comparative effectiveness studies used to produce clinical guidance. In addition, NICE supports CPG development through accredited organizations. It accredits organizations that comply with explicit, transparent NICE standards and produce high-quality clinical guidance (see further discussion in Chapter 7). Although the guidelines and accreditation work is designed for use in England by the NHS, these processes potentially could be employed in other countries. NICE staff offer consulting services internationally to assist in adoption of NICE standards and CPG development methodologies, particularly those related to integration of scientific evidence and social values in health care policy and clinical decision making. NICE also issues a suite of products for each review and guideline it produces, including shorter NICE guidelines, full guidelines with systematic reviews, consumer summaries, and implementation assistance (NHS, 2010).
The World Health Organization (WHO) develops a large number of health-related recommendations for a diverse group of target audiences, including the general public, healthcare professionals, health facility managers (e.g., hospital administrators) or regions (e.g., districts), and public policy makers. These recommendations address a wide range of clinical, public health, and health policy topics related to achieving WHO health goals (Oxman et al., 2007). In 2006, the WHO Advisory Committee on Health Research published a series of 16 papers advising WHO guideline developers on how to ensure that their healthcare recommendations are informed by best available research evidence (Schünemann et al., 2006).
Other international developers of CPGs include the European Society of Cardiology and the Kidney Disease Improving Global Outcomes (KDIGO). The ESC Committee for Practice Guidelines was established in 1994 to address new health-related policies in
clinical practice, prevention, quality assurance, research programs, and health economics in cardiovascular diseases. Its committee is composed of 8 to 10 members who are elected for 2 years. It developed 4 guidelines in 2009, and 36 since 2001 (ESC, 2010). KDIGO, an international guideline development organization, was established in 2003 to promote the “coordination, collaboration and integration of initiatives to develop and implement clinical practice guidelines” (Eknoyan et al., 2004, p. 1310). It is currently managed by the U.S.-based National Kidney Foundation and is supported by national kidney disease guideline development initiatives around the world (KDIGO, 2008).
Other Stakeholders Related to Guideline Dissemination or Use
In addition to the organizations cited above that develop CPGs, numerous government, private, and international groups participate in activities vital to guideline dissemination or use. As with the above examples, the organizations below are meant to illustrate the various stakeholders and not be a complete representation. The discussion here, as well, is limited to their CPG-related activities.
As noted previously, AHRQ no longer develops guidelines, but helps other organizations to develop guidelines. Two previously mentioned major functions supported by AHRQ will be discussed below: the support of EPCs and the NGC.
AHRQ’s network of EPCs has expertise in conducting methodologically rigorous, independent SRs and technology assessments of scientific evidence, and research to advance SR methodologies. Reports from these centers encompass comprehensive literature reviews and research syntheses (possibly comprising meta-analyses or cost analyses) on priority health topics conducted by experts with a range of relevant skills. Reports include documentation of process and undergo peer review and public comment before they are made final. These reports are available to all public and private CPG developers (AHRQ, 2008). Further, medical specialty societies such as the American College of Physicians (ACP), the Renal Physicians Association (RPA) and many other guideline developers utilize the EPC’s topic nomination system to encourage the production of high quality SRs in clinical areas relevant to their guidelines (Matchar et al., 2005).
AHRQ, in conjunction with the American Medical Association and America’s Health Insurance Plans, through a contract with ECRI
Institute, created the NGC in 1997 to ensure no-charge availability of CPGs to clinicians, health plans, providers, purchasers, and the general public. The NGC supports dissemination, adoption, and use of CPGs through an online database of summaries of CPGs that satisfy certain minimal quality criteria (see Box 2-1 for a list of current criteria). The criteria were established to promote inclusiveness so potential adopters were granted access to a large proportion of available guidelines (Coates, 2010).
Criteria for Inclusion in The National Guideline Clearinghouse (NGC)
SOURCE: NGC (2010c).
NGC and ECRI staff scrutinize CPGs submitted by developers. If CPGs meet basic criteria, ECRI summarizes them, and posts them on a live, interactive website, www.guideline.gov. Since this website was opened in 1999, ECRI has summarized and posted nearly 8,000 CPGs from more than 300 U.S. and international government agencies, specialty societies, and other nonprofit organizations. Currently, NGC provides more than 2,500 summaries with links to full guideline texts, as well as personal digital assistant downloads of summaries; 25 Guideline Syntheses contrasting CPGs on similar topics; an annotated guidelines bibliography; and a weekly e-mail newsletter update on recent additions (Coates, 2010). To remain in the NGC, a CPG must be updated at least every 5 years; 1,331 CPGs have been withdrawn from the NGC, mainly because they did not satisfy this criterion.
The FDA is involved in developing evidence-based5 recommendations for drug use through its role in approving drug labeling. For example, the FDA now requires pharmaceutical firms to implement Risk Evaluation and Mitigation Strategies, which detail how the benefits of a drug or biological product outweigh its risks (FDA, 2009).
The federal Centers for Medicare & Medicaid Services (CMS) is a particularly important user of guidelines. Because CMS spent billions of dollars on healthcare services for an estimated 118.6 million Medicare, Medicaid, and Children’s Health Insurance Program beneficiaries (including some dual eligibles) in 2009, Medicare and Medicaid policies have a significant impact on patient care and healthcare providers’ and insurers’ revenue (HHS, 2010). CMS employs guidelines in several ways:
A guideline from the USPSTF or a Technology Assessment conducted through AHRQ’s EPC Program could support an expansion or reduction of Medicare coverage to include, for example, a screening test for beneficiaries. Under the Patient Protection and Affordable Care Act, Medicare (and commercial insurers) will be required to include coverage of preventive services that receive a highly rated recommendation from a CPG of the USPSTF.6
Medicare’s quality improvement program, contracted through Quality Improvement Organizations in each state,
can concentrate collaboration with hospitals, physicians, and other providers on guideline-recommended actions and apply guidelines-driven quality measures to track improvements in practice and outcomes over time.
CMS requires participating hospitals, home health agencies, dialysis facilities, and nursing homes to report quality measures, frequently based on CPGs. Full reimbursement or an incentive payment is linked to this reporting for selected providers. A voluntary Physician Quality Reporting Initiative provides incentives to physicians and group practices reporting measures. The agency then posts these measures and other quality data on its website, www.medicare.gov/nhcompare/, to encourage beneficiaries and other consumers to make careful provider choices (Jacques, 2010).
Medicare additionally has supported experimentation with Pay-for-Performance schemes that are based on quality measures derived in part from well-accepted CPGs (Jacques, 2010).
Along with organizations in many countries around the world developing CPGs, there are support organizations with functions of interest to U.S. developers too. For example, Guidelines International Network (GIN), a Scottish-based, not-for-profit, international association of 50 individuals and 91 organizations representing 39 countries, was founded in 2002 to promote development and use of CPGs, in part by collecting published CPGs (Guidelines International Network, 2010a). In addition to compiling a publicly accessible library of more than 3,700 guidelines and 3,000 related documents (guideline clearing reports, methodologies, implementation tools and systematic reviews) from members (Guidelines International Network, 2010c), the organization holds annual conferences to encourage collaboration among guideline-producing organizations and experts and enhance dissemination of guidelines-related research. GIN also has a Patient and Public Involvement Working Group (G-I-N PUBLIC) of consumers, developers, and researchers that supports effective patient and public involvement in guideline development and implementation, and has recently approved a request to establish a G-I-N affiliated U.S. interest group to foster the development and use of high-quality CPGs in the United States (Guidelines International Network, 2010b).
Another international group, the Cochrane Collaboration, is dedicated to improving healthcare decisions through development of systematic reviews of healthcare outcomes evidence. The SRs are conducted by a network of 10,000 individuals around the globe, predominantly volunteers. CPG developers in the United States often include these SRs in their evidence bases. The Collaboration has a small staff and is supported by donations, including extensive contribution of in-kind services from individuals and institutions and support form governments in many countries, and subscriptions to the Cochrane Library; commercial funds for SRs are prohibited. In addition to producing reviews and related abstracts and derivative products, the Collaboration has created a library of SRs and their scientific evidence foundations (The Cochrane Collaboration, 2010a). The Collaboration also has developed extensive guidance, a reviewers’ handbook, and training programs on how to conduct rigorous SRs (The Cochrane Collaboration, 2010b).
Funders of CPGs
Funders of CPG development play a critical role in the process; however, the committee found no systematic sources of information regarding CPG funders or their levels of financial support. Although CPGs developed by federal agencies would likely be funded from federal tax dollars, the amount spent on that function in the various agencies is unknown. A reasonable assumption is that commercial guidelines are funded, at least in part, through their sale and sales of related support products and services. Many guidelines developed by medical societies and other private organizations are self-funded, through membership dues, donations, or other means. CPGs funded by medical societies dependent on membership dues may be cause for concern regarding conflict of interest if their recommendations would likely affect their members’ income.
Guidelines listed in the NGC may report support from government, a parent professional organization, private foundations or individual donors, sales of guidelines, payers, or major health systems. Funding from medical imaging, device, or pharmaceutical industries associated with a guideline topic may also be suspect due to Conflict of Interest (COI), yet it may be difficult to discern that financial presence. A guideline might report to the NGC that it was fully funded by its sponsoring organization, but that body might or might not receive significant portions of revenue from industry. For example, the Alzheimer’s Association’s listing in the NGC for its
guideline, Dementia Care Practice Recommendations for Assisted Living Residences and Nursing Homes: Phase 3 End-of-Life Care, a 2007 submission (NGC, 2010a), indicates that it was funded by the Association. That, in turn, is supported by national corporations (including pharmaceutical firms), foundations, and private donations (Alzheimer’s Association, 2010).
In another case, funding for the AHA’s 2008 CPG on adults with congenital heart disease came totally from AHA and ACC Foundations (AHA, 2010a). AHA’s funding primarily arises from individual donations (46 percent) and corporate funds (28 percent), with less than 4 percent from the medical device and drug industries; according to AHA policy, CPG development receives no direct industry support (AHA, 2010a).
This chapter has briefly detailed the history of evidence-based medicine and the variety of organizations historically and currently involved with CPG development. The next chapter examines the ongoing challenges undermining the trustworthiness and impact of CPGs and potential ways to overcome them.
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