Survival Gain in Cost-Effectiveness Studies
Authors: Sabrina Trippoli, Monica Vaiani, Andrea Messori, Enrico Tendi, Bruce E. Hillner
Journal: Journal of Clinical Oncology (2000)

Article Tools SPECIAL DEPARTMENTS Article Tools OPTIONS & TOOLS Export Citation Track Citation Add To Favorites Rights & Permissions COMPANION ARTICLES No companion articles ARTICLE CITATION DOI: 10.1200/JCO.2000.18.18.3318 Journal of Clinical Oncology - published online before print September 21, 2016 PMID: 10986065 Survival Gain in Cost-Effectiveness Studies Sabrina TrippolixSabrina TrippoliSearch for articles by this author , Monica VaianixMonica VaianiSearch for articles by this author , Andrea MessorixAndrea MessoriSearch for articles by this author , Enrico TendixEnrico TendiSearch for articles by this author Bruce E. HillnerxBruce E. HillnerSearch for articles by this author Show More Drug Information Center, Policlinico Careggi Hospital Florence, ItalyVirginia Commonwealth University Richmond, VA https://doi.org/10.1200/JCO.2000.18.18.3318 First Page Full Text PDF Figures and Tables © 2000 by American Society of Clinical OncologyjcoJ Clin OncolJournal of Clinical OncologyJCO0732-183X1527-7755American Society of Clinical OncologyResponse18092000In Reply:Trippoli et al have published frequent commentaries about limitations in a variety of published cost-effectiveness issues. Herein, they again question the appropriateness of doing a cost-effectiveness study of a trial without statistically significant results. As Hlatky1 noted in his reply to Jacobson,2 an ongoing debate is about how to distinguish between the concepts of statistically significant and clinically important.Our article3 addressing the cost-effectiveness of pamidronate in advanced breast cancer was based on data from the two trials critical to the United States Food and Drug Administration approval that were statistically significant in the primary end point, skeletal-related complications due to bony metastases.4,5 The determination of a quality-adjusted survival was done in our post-hoc analyses and determining a statistically significant difference in quality-adjusted survival was not possible.In our article6 addressing the role of temozolomide in metastatic melanoma, we previously stated that the trial was underpowered or overly optimistic; that is, too few patients were enrolled. In this trial, the observed 7+ month median survival was almost identical to that of larger community cohorts.7 Therefore, the issue concerning the clinical importance of a 1.1-month survival benefit can be debated when observed in a negative trial. Although we disagree with Trippoli et al that all such analyses are inappropriate, we do hope that in the design of future clinical trials, sample sizes are determined realistically to avoid this debate.1. Hlatky MA: Reply to: Jacobson MW. N Engl J Med 336:: 1840,1997-1841, Crossref, Medline, Google Scholar2. Jacobson MW: Cost-effectiveness of coronary bypass surgery versus angioplasty. N Engl J Med 336:: 1840,1997-1841, (letter) Crossref, Medline, Google Scholar3. Hillner BE, Weeks JC, Desch CE, et al: Pamidronate in prevention of bone complications in metastatic breast cancer: A cost-effectiveness analysis. J Clin Oncol 18:: 72,2000-79, Link, Google Scholar4. Hortobagyi GN, Theriault RL, Lipton A, et al: Efficacy of pamidronate in reducing skeletal complications in patients with breast cancer and lytic bone metastases. Protocol 19 Aredia Breast Cancer Study Group [see comments]. N Engl J Med 335:: 1785,1996-1791, Crossref, Medline, Google Scholar5. Hortobagyi GN, Theriault RL, Lipton A, et al: Long-term prevention of skeletal complications of metastatic breast cancer with pamidronate: Protocol 19 Aredia Breast Cancer Study Group. J Clin Oncol 16:: 2038,1998-2044, Link, Google Scholar6. Hillner BE, Agarwala S, Middleton MR: Post hoc economic analysis of temozolomide versus dacarbazine in the treatment of advanced metastatic melanoma. J Clin Oncol 18:: 1474,2000-1480, Link, Google Scholar7. Barth A, Wanek LA, Morton DL: Prognostic factors in 1,521 melanoma patients with distant metastases [see comments]. J Am Coll Surg 181:: 193,1995-201, Medline, Google Scholar