Assessing and Improving Value in Cancer Care Workshop Summary (2009) / Chapter Skim
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4 Generating Evidence About Effectiveness and Value
4 Generating Evidence About Effectiveness and Value
Pages 23-32
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operates at the introduction of cancer care drugs and biological therapeutics into the market, Dr. Janet Woodcock of the FDA's Center for Drug Evaluation and Research said.
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Many drugs reaching the market in recent decades have been approved after regulations were implemented allowing approval based on surrogate endpoints (accelerated approval)
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While pharmaceutical discovery and candidate selection in cancer is driven by recent scientific discoveries, much more of clinical oncology treatment development is empirical -- trial and error -- compared to other disease therapeutics areas. This approach limits understanding of cancer drug benefits, since there are no means of assessing drugs' pharmacodynamic effect.
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From page 26... ...
Among the 53 new indications approved by the FDA in cancer therapeutics between July 2005 and December 2007, 38 clearly showed clinical benefit and proceeded through the approval process using regular approval indications, while 10 used accelerated approval, and 5 previous accelerated approvals were converted to regular approvals upon completion of confirmatory trials with a new indication. With respect to the measures used to support these approvals, 10 indications showed benefits in overall survival, 5 indications were approved based on evidence of disease-free survival, 12 indications were approved based on evidence of time-to-progression or p rogression-free survival, 17 indications were approved based on response rates, and other, novel endpoints, such as reduction in hepatic iron and depletion of asparagines, were used in the remainder of cases.
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Dr. Daniel Sargent of the Mayo Clinic defined a few key terms to begin to address the question he set out to answer through his presentation, "What constitutes reasonable evidence of efficacy and effectiveness in cancer care?
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While there is input from many parties, collection and reporting of treatment effectiveness data are disorganized, and effectiveness is often unclear because therapies are used in situations beyond those examined in clinical trials. Randomized controlled trials (RCTs)
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Large, simple trials use streamlined trial designs with no extra investigations and minimal extra workload. One such trial, the QUASAR (QUick And Simple And Reliable)
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When evaluating evidence for treatments, true clinical efficacy measures, such as overall survival, are always the gold standard. In some cases a validated surrogate endpoint showing effectiveness can predict the true clinical benefit endpoint measure, but this represents a weaker source of evidence than true clinical efficacy measures.
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Predictive biomarker validation will require randomized clinical trials, either through targeted selection trials (where only patients who express a given biomarker are enrolled) or through unselective enrollment trials with prospectively specified biomarker analysis.
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2007. End points for colon cancer adjuvant trials: Observations and recommendations based on individual patient data from 20,898 patients enrolled onto 18 randomized trials from the ACCENT group.
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