TL;DR: Nonrandomized clinical trials with definitive end points can yield US Food and Drug Administration approvals, and these drugs have a reassuring record of long-term safety and efficacy.
Abstract: Purpose To approve a new anticancer drug, the US Food and Drug Administration often requires randomized trials However, several oncology drugs have been approved on the basis of objective end points without a randomized trial We reviewed the long-term safety and efficacy of such agents Methods We searched the Web site of the US Food and Drug Administration’s Center for Drug Evaluation and Research and MEDLINE for initial applications of investigational anticancer drugs from 1973 through 2006 Results Overall, 68 oncology drugs, excluding hormone therapy and supportive care, were approved, including 31 without a randomized trial For these 31 drugs, a median of two clinical trials (range, one to seven) and 79 patients (range, 40 to 413) were used per approval Objective response was the most common end point used for approval; median response rate was 33% (range, 11% to 90%) Thirty drugs are still fully approved United States marketing authorization for one drug, gefitinib (an epidermal growth factor receptor [EGFR] inhibitor), was rescinded after a randomized trial showed no survival improvement; however, this trial was performed in unselected patients, and it was subsequently demonstrated that patients with EGFR mutation are more likely to respond Nineteen of the 31 drugs have additional uses (per National Comprehensive Cancer Network or National Cancer Institute Physician Data Query guidelines), and subsequent formal US Food and Drug Administration approvals were obtained for 11 of these (range, one to 18 new indications) No drug has demonstrated safety concerns Conclusion Nonrandomized clinical trials with definitive end points can yield US Food and Drug Administration approvals, and these drugs have a reassuring record of long-term safety and efficacy
TL;DR: A review of the current standard of care and recent advances in therapy for newly-diagnosed and recurrent glioblastomas, based on the most authoritative guidelines, the National Cancer Institute's comprehensive cancer database Physician Data Query (PDQ®), and the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology(TM) for central nervous system cancers (V.1.2010), to elucidate the current position and in what direction we are advancing as mentioned in this paper.
Abstract: Glioblastoma is the most common primary malignant brain tumor in adults and is a challenging disease to treat. The current standard therapy includes maximal safe surgical resection, followed by a combination of radiation and chemotherapy with temozolomide. However, recurrence is quite common, so we continue to search for more effective treatments both for initial therapy and at the time of recurrence. This article will review the current standard of care and recent advances in therapy for newly-diagnosed and recurrent glioblastomas, based on the most authoritative guidelines, the National Cancer Institute's comprehensive cancer database Physician Data Query (PDQ®), and the National Comprehensive Cancer Network Clinical Practice Guidelines in Oncology(TM) for central nervous system cancers (V.1.2010), to elucidate the current position and in what direction we are advancing.
TL;DR: Regardless of the stage at diagnosis, men who were younger and lived in a neighborhood with higher income and education levels were the most likely to receive a prostatectomy as opposed to other treatments.
Abstract: Objectives: Data from the California Cancer Registry were used to model the effect of race/ethnicity, census-derived socioeconomic status (SES), age, year, and stage at diagnosis on prostatectomy utilization in men diagnosed with prostate cancer from 1990 through 1993. Treatment received was compared with the National Cancer Institute's Physician Data Query (PDQ) to evaluate concordance. Methods: Odds ratios (OR) and 95% confidence intervals (CI) were estimated to assess the likelihood of (a) receiving a prostatectomy and (b) receiving a treatment in concordance with the PDQ. Non-concordance was defined as a prostatectomy performed on a patient who was either diagnosed with AJCC stage III or IV prostate cancer, or was older than 70 years. All other treatments were considered compliant with the PDQ. Results: Regardless of the stage at diagnosis, men who were younger and lived in a neighborhood with higher income and education levels were the most likely to receive a prostatectomy as opposed to other treatments. Black men were the least likely to be treated with prostatectomy (OR = 0.6, CI = 0.5–0.6), and the differential was evident within all income levels examined. With respect to the PDQ, black men were 1.4 times more likely to receive concordant treatment than white men (OR = 1.4, CI = 1.3–1.5). Conclusions: California black men are receiving less aggressive treatment (that is more concordant with the PDQ) when diagnosed with prostate cancer.
TL;DR: A system for clinical trial eligibility determination where patients or primary care providers can enter clinical information about a patient and obtain a ranked list of clinical trials for which the patient is likely to be eligible and returns a numerical score.
Abstract: We have developed a system for clinical trial eligibility determination where patients or primary care providers can enter clinical information about a patient and obtain a ranked list of clinical trials for which the patient is likely to be eligible. We used clinical trial eligibility information from the National Cancer Institute's Physician Data Query (PDQ) database. We translated each free-text eligibility criterion into a machine executable statement using a derivation of the Arden Syntax. Clinical trial protocols were then structured as collections of these eligibility criteria using XML. The application compares the entered patient information against each of the eligibility criteria and returns a numerical score. Results are displayed in order of likelihood of match. We have tested our system using all phase II and III clinical trials for treatment of metastatic breast cancer found in the PDQ database. Preliminary results are encouraging.
TL;DR: There exist deviations from NCI established treatment recommendations among rural breast cancer patients, and more research is needed to develop better methods for dissemination of state‐of‐the‐art cancer information to rural physicians and patients.
Abstract: Background. Research shows that rural populations are more likely than their urban counterparts to be diagnosed with late‐stage cancer, but less is known about appropriateness of cancer treatment in rural locations after diagnosis. The objective of this analysis was to assess the degree to which rural breast cancer treatment was received in concordance with national recommendations. Methods. Data came from 251 stage I and II breast cancer patients residing in rural North Carolina. State‐of‐the‐art care was defined using the National Cancer Institute's (NCI) physician data query (PDQ) database, and cases were categorized into appropriate primary and/or adjuvant treatment. Chi‐square and Fishers' exact tests were used to assess changes in appropriate treatment over time (1991–1996) and between stage. Multiple logistic regression was used to determine whether any patient or disease characteristics were associated with receipt of appropriate treatment. Results. Most (81–90%) of the breast cancer cases received the appropriate primary therapy (mastectomy or lumpectomy followed by radiation therapy); of these, the majority received a mastectomy (66–72%). Fewer women received adjuvant therapy as recommended (27–61%), although significantly more stage II than stage I cases did so (p≤0.05). Regression showed that stage and estrogen‐receptor (ER) status were associated with appropriate therapy. Conclusions. The findings suggest that there exist deviations from NCI established treatment recommendations among rural breast cancer patients. More research is needed to develop better methods for dissemination of state‐of‐the‐art cancer information to rural physicians and patients, and to understand how treatment decisions are made.