IOM Recommendations Seek to Ensure Evidence-Based, Unbiased Guidelines
Increased emphasis on systematic review of evidencebased data, transparency of process will improve quality of patient care, allow for physician decision making
by Monica Morrow, MD
Clinical practice guidelines (CPGs) are a ubiquitous part of health care. As of February 2012, the United States National Guidelines Clearinghouse (NGC) had 532 guidelines listed under the disease heading of neoplasm, 16 of which were developed by the American Society of Clinical Oncology (ASCO).
The cancer-related guidelines are only a fraction of the 2,200 disease-related guidelines that are available in the NGC, and an additional 247 guidelines are listed as in development. In order to be listed in the NGC, guidelines must meet the following criteria: contain systematically developed statements that include recommendations or information that assist providers with decision making about appropriate care for specific clinical circumstances; be produced by a professional, public, or government group rather than an individual; have documentation verifying that a systematic literature review was performed during guideline development; and the full text must be available in English.
Outside of the NGC, the Guidelines International Network database contains 1,124 cancer-related guidelines in a variety of languages. In addition, multiple other cancer-related guidelines familiar to clinicians, such as those from the National Comprehensive Cancer Center (NCCN), exist which are not included in these databases.
Standardizing Definitions, Improving Transparency
The explosion in guideline development is linked to a number of factors, including recognition of the variation in care across even high-cost, sophisticated medical systems such as those in the United States, the increase in the evidence base physicians must master in order to practice evidence-based medicine, and the often prolonged time required for new knowledge to diffuse within the medical community.
The increased attention to standardized, evidence-based care as a way to improve quality led to enthusiasm for CPGs as a way to address many of the barriers to delivering such care. Unfortunately, many of the existing guidelines contain contradictory recommendations or perform poorly when evaluated with instruments designed to assess guideline quality.1-3 For example, a review of 51 lung cancer guidelines found that only 27 were evidence based.2
The heterogeneity in guideline quality and the lack of transparency in the guideline development process prompted the Institute of Medicine (IOM) to convene a committee to develop standards for creating trustworthy clinical practice guidelines, which resulted in the 2011 report entitled “Clinical Practice Guidelines We Can Trust.”4
The committee began its work by updating the IOM definition of CPGs as follows: “Clinical practice guidelines are statements that include recommendations intended to optimize patient care that are informed by a systematic review of evidence and an assessment of the benefits and harms of alternative care options.”
This definition is considerably more specific than the 1990 IOM definition of CPGs, which described CPGs as “systematically developed statements to assist practitioner and patient decisions about appropriate [health care] in specific clinical circumstances.”
The new definition emphasizes the fundamental role of a systematic review of the evidence in guideline formulation, as well as the importance of assessing not only the benefits, but also the potential harms of a therapy when making management recommendations. The 2011 definition also makes it clear that guidelines that are not based on a systematic review, while they may be informative, should not actually be considered CPGs.
The committee spent some time debating the concept of “trustworthiness,” which was specifically contained in its charge. This was felt to be an important concept because of the existence of guidelines from respected organizations with confl icting recommendations, leaving both clinicians and patients at a loss to explain how a review of the same evidence resulted in different recommendations.
The lack of transparency in most guideline development processes regarding how actual recommendations are derived, particularly for situations where level-1 evidence is lacking, contributes to this problem. The committee also struggled with how best to deal with the inherent confl ict of interest that is present when clinicians who derive substantial clinical income or academic reward from management of the disease process under consideration are involved in guideline development.
The committee felt that clinician experts were necessary for the development of meaningful guidelines but that efforts to achieve balance by including both the spectrum of specialists involved in disease management as well as individuals without confl icts as leaders of the guideline development process were needed. In order to be considered “trustworthy,” the committee ultimately determined that guidelines should meet the following criteria: be based on a systematic evidence review; be developed by a multidisciplinary panel of experts and representatives of key affected groups; consider patient subgroups and preferences as appropriate; be based on a transparent process that minimizes distortion, bias, and confl ict of interest; explain the relationship between care options and health outcomes, and rate both the quality of the evidence and strength of the recommendations; and be revised on the basis of important new evidence.
Individualized Decision Making Still Key
Many of the tenets outlined in the IOM report were already part of the ASCO guideline development process, particularly the recognition that a high-quality systematic review of the existing evidence is the cornerstone of guideline development.5
Ratings of evidence quality and strength of recommendations are now being incorporated into ASCO guidelines, further increasing concordance with IOM recommendations, and hopefully the utility of its guidelines to clinical oncologists. The American Cancer Society has also revamped its cancer screening guideline development process to refl ect IOM recommendations.6
While improvements in the guideline development process that result in improved guideline quality are needed, they will not address one critical problem: how to provide appropriate guidance in areas of uncertainty in which the evidence base is lacking or of poor quality. A decision regarding when benefit outweighs harm is always somewhat subjective and infl uenced by patient preference. Given that the clinical trials that inform our deliberations of benefit versus harm generally exclude patients with multiple comorbidities, decision making for such patients must be individualized.
Guidelines are not intended to replace the “art” of medicine or the ability of an individual physician to decide what is best for his or her individual patient, as their detractors are fond of claiming. Instead, guidelines can be used to establish the boundaries around which decisions should be patient sensitive by defining the circumstances in which it is clear that therapy is beneficial or a therapy is more likely to cause harm. The latter is particularly important because cancer management decisions have traditionally favored aggressive treatment due to our focus on the risk of recurrence rather than on the likelihood of net treatment benefit.
Ratings of evidence quality and strength of recommendations are a much needed step in providing clinicians with the information needed to determine accepted thresholds for therapy, when new treatments should be incorporated into practice, when old treatments should be abandoned, and when therapy may actually be harmful.
The properly developed CPG is not a refl ection of the loudest voice in the room, but a synthesis of the best evidence, and that should be welcome to any provider of cancer care.
About the Author: Dr. Morrow is chief of the Breast Surgery Service, co-chief of the Breast Program, and Anne Burnett Windfohr Chair of Clinical Oncology at Memorial Sloan-Kettering Cancer Center. She is also professor of surgery at Weill Cornell Medical College and the 2012 president of the Society of Surgical Oncology. Dr. Morrow has been Editor of the ASCO Daily News since 2005, and she is this year’s recipient of the Gianni Bonadonna Breast Cancer Award.
1. Dinnes J, Hewison J, Altman DG, et al. The basis for monitoring strategies in clinical guidelines: a case study of prostate-specific antigen for monitoring in prostate cancer. CMAJ. 2012;184(2):169-77.
2. Harpole LH, Kelley MJ, Schreiber G, et al: Assessment of the scope and quality of clinical practice guidelines in lung cancer. Chest. 2003;123 (1 Suppl):7S-20S.
3. Simone B, De Feo E, Nicolotti N, et al: Quality evaluation of guidelines on genetic screening, surveillance and management of hereditary colorectal cancer. Eur J Public Health. 2011; Dec 2. Epub ahead of print.
4. Institute of Medicine. Clinical Practice Guidelines We Can Trust. Washington DC, National Academics Press, 2011. http://www.nap.edu/openbook.php? record_id=13058&page=R1. Accessed February 21, 2012.
5. Somerfield MR, Einhaus K, Hagerty KL, et al. American Society of Clinical Oncology clinical practice guidelines: opportunities and challenges. J Clin Oncol. 2008;26(24):4022-4026.
6. Brawley O, Byers T, Chen A, et al. New American Cancer Society process for creating trustworthy cancer screening guidelines. JAMA. 2011;306(22):2495- 2499.