The Value of Pharmaceutical Industry-Sponsored Patient Registries in Oncology Clinical Research.

In May 2019, the US Food and Drug Administration (FDA) released the Framework for FDA's Real-World Evidence (RWE) Program, a draft guidance to evaluate the potential use of real-world data in facilitating regulatory decisions. As a result, pharmaceutical companies and medical communities see patient registries, which are large, prospective, noninterventional cohort studies, as becoming increasingly important in providing evidence of treatment effectiveness and safety in clinical practice. Patient registries are designed to collect longitudinal clinical data on a broad population to address critical medical questions over time. With their large sample sizes and broad inclusion criteria, patient registries are often used to generate RWE in the general and underrepresented patient populations that are less likely to be studied in controlled clinical trials. Here, we describe the value of industry-sponsored patient registries in oncology/hematology settings to healthcare stakeholders, in drug development, and in fostering scientific collaboration.

Sequelae of AKI.

Large epidemiologic studies in a variety of patient populations reveal increased morbidity and mortality that occur months to years after an episode of acute kidney injury (AKI). Even milder forms of AKI have increased associated morbidity and mortality. Residual confounding may account for these findings, but considering the huge number of individuals afflicted with AKI, the sequelae of AKI may be a very large public health burden. AKI may simply be a marker for increased risk, but there is increasing evidence that it is part of the causal pathway to chronic kidney disease. These studies have upended the traditional view that AKI survivors who returned to baseline, or near baseline renal function, do not suffer additional long-term consequences. Recovery of renal function after AKI, short of independence from renal replacement therapy, is yet to be clearly defined but may be of significant importance in the management of AKI survivors. The association between AKI in patients who undergo cardiac surgery and clinical outcomes is of considerable importance to clinicians, surgeons, and anesthesiologists alike and is a major focus of this review.

Comparison of the clinical behavior of newly diagnosed stages II-IV low-grade serous carcinoma of the ovary with that of serous ovarian tumors of low malignant potential that recur as low-grade serous carcinoma.

BACKGROUND: Serous ovarian tumors of low malignant potential (STLMP) frequently coexist with low-grade serous carcinoma of the ovary (LGSC) and, when they recur, frequently do so as LGSC. The purpose of this study was to compare the outcomes of patients with these two tumor types. METHODS: All patients with stages II-IV LGSC (group 1) or with STLMP that recurred as LGSC (group 2) seen at our institution from 1973 to 2003 were identified, and demographic data were obtained. For group 1, progression-free and overall survival times were calculated from the date of primary diagnosis to the date of disease progression/recurrence or the date of last contact/death, respectively. For group 2, progression-free and overall survival times were calculated from the date of first relapse as a LGSC to the date of progression or the date of last contact/death, respectively. The method of Kaplan and Meier was used to estimate survival, and the log-rank test was used to compare differences between survival curves. RESULTS: We identified 112 patients in group 1 and 41 in group 2. There were no statistically significant differences between the two groups in median age (42.7 vs. 45.4 years [at relapse]; P=0.37), progression-free survival time (19.5 vs. 25 months; P=0.92), or overall survival time (81.8 vs. 82.8 months; P=0.84). CONCLUSIONS: The age at diagnosis, progression-free survival time, and overall survival time associated with newly diagnosed stages II-IV LGSC of the ovary are similar to those of STLMP that recur as LGSC, providing further evidence of an association between these two tumor types.