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Journal of Clinical Oncology, Vol 26, No 13 (May 1), 2008: pp. 2223-a-2224 © 2008 American Society of Clinical Oncology. DOI: 10.1200/JCO.2008.16.5886
Randomized Clinical Trials, Clinical Use, and Hope: What Relationship?RAND Corp, St Augustine, FL
Department of Health Management and Policy, University of Michigan School of Public Health, Ann Arbor, MI
Department of Obstetrics and Gynecology, University of Auckland, Auckland, New Zealand
University of California San Francisco, Philip R. Lee Institute for Health Policy Studies, San Francisco, CA To the Editor: Questions have been raised once again about the use of high-dose chemotherapy with autologous bone marrow (or hematopoietic stem cell) transplantation to treat early high-risk and metastatic breast cancer by several recent reports—a meta-analysis presented by Berry1 at the 2007 San Antonio Breast Cancer Symposium; the report by Crump and colleagues2 from the National Cancer Institute of Canada in the January 1, 2008, issue of the Journal of Clinical Oncology; and the accompanying editorial by Stadtmauer.3 Stadtmauer, citing our recent book, False Hope: Bone Marrow Transplantation for Breast Cancer,4 dismisses our characterization of the procedure. He argues that it is time to move on from this experience, while remembering its lessons. But he draws few lessons from the experience. There are several to consider. When should a highly toxic and costly procedure be made widely available? After randomized trials, as in the US Food and Drug Administration (FDA) model of drug evaluation, or concurrently with those trials, as was the high-dose chemotherapy case? Stadtmauer suggests that early data in this case were as good as it gets and implicitly rejects the FDA model. Subsequent trials clearly refute that view. Who should finance randomized controlled trials (RCTs) of procedures that do not require FDA review—the federal government through the National Cancer Institute or health insurers? The National Cancer Institute clearly has the lead role, but currently faces severe budgetary constraints. The Philadelphia trial, which Stadtmauer led, was initiated by an unrestricted grant from US HealthCare in 1990, for which it deserves the acknowledged gratitude of many. Only when the Philadelphia trial became an Eastern Cooperative Oncology Group trial did it receive public funds. The BlueCross BlueShield Association established a mechanism to finance RCTs for this investigational treatment. Though limited in important ways, the oncology community should recognize the BlueCross BlueShield Association's effort as a positive step forward and the potential basis for additional negotiations. If health insurers are expected to finance RCTs, should a policy for doing so be hammered out in advance in negotiations between the research community and insurers, or should each new procedure be treated de novo on a case-by-case basis? William Peters pursued the latter route unsuccessfully. But a mechanism for the former does not exist. In our book, we propose a public-private partnership, but other responses may be equally or more promising. If a negotiating mechanism or institution is to be created, what will researchers bring to the table? An entitlement mentality? (Support us, but don't ask questions.) Eternal enmity to insurers? Comis et al5 recently presented data demonstrating that patients trust their physicians the most and insurers the least. Clearly, the basis exists for hostile relations. But a continued dysfunctional relationship among insurers, researchers, and clinicians does not serve the interests of patients, and a mutual concern for evidence of effectiveness provides the best basis for assuring advance evaluation before widespread use. The current work of the Institute of Medicine Roundtable on Evidence-Based Medicine and the Agency for Healthcare Research and Quality's Effective Health Care Program, as well as the debate on establishing a federally funded Comparative Effectiveness Research Institute, indicates clearly that evidence of effectiveness is critically important to multiple stakeholders. Hence our public-private partnership recommendation. We wrote False Hope to promote serious discussion of these issues. Rewriting medical history in a hyper-rationalistic mode is a way to move on without grappling with these issues. AUTHORS’ DISCLOSURES OF POTENTIAL CONFLICTS OF INTEREST The author(s) indicated no potential conflicts of interest. REFERENCES 1. Berry DA, Ueno NT, Johnson MM, et al: High-dose chemotherapy with autologous stem-cell support versus standard-dose chemotherapy: Meta-analysis of individual patient data from 15 randomized adjuvant breast cancer trials. Presented at the 30th Annual San Antonio Breast Cancer Symposium, San Antonio, TX, December 13-16, 2007 2. Crump M, Gluck S, Tu D, et al: Randomized trial of high-dose chemotherapy with autologous peripheral-blood stem-cell support compared with standard-dose chemotherapy in women with metastatic breast cancer. J Clin Oncol 26:37-43, 2008 3. Stadtmauer E: A dramatic story of hope and reality. J Clin Oncol 26:11-12, 2008 4. Rettig RA, Jacobson PD, Farquhar CM, et al: False Hope: Bone Marrow Transplantation for Breast Cancer. New York, NY, Oxford University Press, 2007, pp 1-355 5. Comis RL, Colaizzi DD, Kimmel LG, et al: Barriers to cancer clinical trials (CCT) participation: "We have met the enemy and he is us." J Clin Oncol 25:338s, 2007 (suppl; abstr 6567),[CrossRef] Related Articles
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Copyright © 2008 by the American Society of Clinical Oncology, Online ISSN: 1527-7755. Print ISSN: 0732-183X
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