The oncologist
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Randomized Controlled Trial Multicenter Study
FDA approval summary: temsirolimus as treatment for advanced renal cell carcinoma.
This report summarizes the U. S. Food and Drug Administration (FDA)'s approval of temsirolimus (Torisel), on May 30, 2007, for the treatment of advanced renal cell carcinoma (RCC). ⋯ Common laboratory abnormalities were anemia, hyperglycemia, hyperlipidemia, and hypertriglyceridemia. Serious but rare cases of interstitial lung disease, bowel perforation, and acute renal failure were observed. Temsirolimus has demonstrated superiority in terms of OS and PFS over IFN-alpha and provides an additional treatment option for patients with advanced RCC.
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In Japan, the history of postoperative chemotherapy for breast cancer started with 5-fluorouracil (5-FU), launched in the 1980s. Currently, oral fluoropyrimidine-based regimens indicated for the treatment of breast cancer in Japan include tegafur plus uracil (UFT); tegafur, gimeracil, and oteracil (TS-1); doxifluridine; and capecitabine. In particular, UFT represents an important option for long-term treatment because of minimal adverse events and the potential for long-term maintenance of effective plasma concentrations of 5-FU to inhibit micrometastasis after surgery. ⋯ This paper reviews the results of clinical studies of UFT conducted in Japan to assess the therapeutic usefulness of this oral 5-FU. The types of patients most likely to benefit from UFT are discussed on the basis of currently available evidence and a global consensus of treatment recommendations. The optimal timing of endocrine therapy and strategies for postoperative adjuvant chemotherapy with UFT in patients with breast cancer are also discussed.
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Neuropathic pain--pain resulting from a lesion, damage, or dysfunction of the somatosensory nervous system--can arise through several distinct etiologies ranging from toxicity, surgery, radiation, and trauma to congenital disorders. Neuropathic pain is widely recognized as a common consequence of cancer and results from administration of several common oncology drugs. It not only impacts quality of life, but it also impacts patient outcomes because of resulting treatment delays, dose reductions, and discontinuations. ⋯ Despite its widely recognized importance, there is a paucity of reliable information available regarding the incidence, prevalence of patient-and physician-reported severity, and time course of cancer-related neuropathic pain. To address this severe knowledge gap, we need new, high-quality, population-based studies of individual cancer pain syndromes and conditions. However, in order to gather this information, we also need substantial improvements in the specific classification of cancer-related neuropathic syndromes and better validated diagnostic tools that can help to elucidate the incidence, prevalence, severity, and potential economic impact of cancer-associated neuropathies.
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Patients, clinicians, payers, and policymakers face an environment of significant evidentiary uncertainty as they attempt to achieve maximum value, or the greatest level of benefit possible at a given level of cost in their respective health care decisions. This is particularly true in the area of oncology, for which published evidence from clinical trials is often incongruent with real-world patient care, and a substantial portion of clinical use is for off-label indications that have not been systematically evaluated. It is this uncertainty in the knowledge of the clinical harms and benefits associated with oncology treatments that prevents postregulatory decision makers from making accurate assessments of the value of these treatments. ⋯ Food and Drug Administration, this situation is exacerbated in the area of oncology. This paper investigates the convergence of incentives and circumstances that lead to widespread uncertainty in oncology and proposes new paradigms for clinical research, including pragmatic clinical trials, methodological guidance, and coverage with evidence development. Each of these initiatives would support the design of clinical research that is more informative for postregulatory decision makers, and would therefore reduce uncertainty and provide greater confidence in conclusions about the value of these treatments.
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The U. S. Food and Drug Administration grants marketing approval for drug products based on a comprehensive review of safety and efficacy data. ⋯ Overall survival is the gold standard for a registration trial designed to gain marketing approval; however, additional endpoints have been used in the approval of oncology drugs. Advantages of specific endpoints are discussed, including the accuracy of an endpoint's measurement and its relation to clinical benefit. Surrogate endpoints may be acceptable for "accelerated" approval, with a sponsor commitment to provide evidence of clinical benefit in a subsequent trial.