Clinical practice guidelines must be outcome driven. Indeed, the validity of a guideline rests on showing that following recommendations will lead to projected health and cost outcomes.1 The primary outcomes proposed for guideline use include survival, toxicity, heath-related quality of life (QOL), and cost effectiveness.2 Patient preferences have also been recognized as factor that must be considered in making guideline recommendations.3
This prescription seems fairly straightforward, and in many instances, determining which outcomes to apply is as well. Recommendations for adjuvant regimens typically use overall or disease-free survival, acceptable toxicity profiles, and costs falling into the accepted range as a constellation of outcomes used to judge whether a regimen should be included.
For many guideline users, treatment effectiveness––its impact on a major clinical outcome such as survival––is assumed to trump all other outcomes. In guidelines development, however, this is only true if that treatment's impact on other outcomes is believed to be minimal or inconsequential. In situations in which evaluating multiple outcomes points to opposing views of patient benefit, creating and following guideline recommendations can be problematic. From a purely clinical perspective, balancing effectiveness with toxicity can present difficult value judgments. For example, should adjuvant chemotherapy be recommended to elderly node-negative breast cancer patients; should allogeneic bone marrow procedures be recommended as first-line treatment in several diseases?
The NCCN Myeloid Growth Factor Guidelines present a vivid example of the difficulties of attempting to assess “competing” outcomes. The first question that arises for this supportive care algorithm is what is the...
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Rodger J. Winn is the Editor-in-Chief of JNCCN. He is Clinical Consultant at the National Quality Forum, and his past positions include Associate Professor of Clinical Medicine at the University of Texas M. D. Anderson Cancer Center. Dr. Winn received his medical degree from Jefferson Medical College of Philadelphia. His postgraduate training includes an internship and residency at Jefferson Medical College, and he also completed a medical oncology fellowship at Memorial Sloan-Kettering Cancer Center in New York. He is board certified in internal medicine and holds subspecialty certification in oncology.
NeaseRF, KneelandT, O'ConnorGT. Variation in patient utilities for outcomes of the management of chronic stable angina. Implications for clinical practice guidelines. Ischemic Heart Disease Patient Outcomes Research Team. JAMA1995;273:1185–1190.
NeaseRFKneelandTO'ConnorGT. Variation in patient utilities for outcomes of the management of chronic stable angina. Implications for clinical practice guidelines. Ischemic Heart Disease Patient Outcomes Research Team. JAMA1995;273:1185–1190.