scholarly journals Safety related label changes for new drugs after approval in the US through expedited regulatory pathways: retrospective cohort study

BMJ ◽  
2017 ◽  
pp. j3837 ◽  
Author(s):  
Sana R Mostaghim ◽  
Joshua J Gagne ◽  
Aaron S Kesselheim
Keyword(s):  
Cohort Study ◽  
Retrospective Cohort Study ◽  
Retrospective Cohort ◽  
New Drugs ◽  
Regulatory Pathways ◽  
The Us

scholarly journals Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study

BMJ ◽  
2020 ◽  
pp. m3434
Author(s):  
Thomas J Hwang ◽  
Joseph S Ross ◽  
Kerstin N Vokinger ◽  
Aaron S Kesselheim
Keyword(s):  
Cohort Study ◽  
Retrospective Cohort Study ◽  
Retrospective Cohort ◽  
New Drugs ◽  
European Medicines Agency ◽  
Health Authorities ◽  
Therapeutic Value ◽  
The Us ◽  
Value Association

AbstractObjectiveTo characterize the therapeutic value of new drugs approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) and the association between these ratings and regulatory approval through expedited programs.DesignRetrospective cohort study.SettingNew drugs approved by the FDA and EMA between 2007 and 2017, with follow-up through 1 April 2020.Data sourcesTherapeutic value was measured using ratings of new drugs by five independent organizations (Prescrire and health authorities of Canada, France, Germany, and Italy).Main outcome measuresProportion of new drugs rated as having high therapeutic value; association between high therapeutic value rating and expedited status.ResultsFrom 2007 through 2017, the FDA and EMA approved 320 and 268 new drugs, respectively, of which 181 (57%) and 39 (15%) qualified for least one expedited program. Among 267 new drugs with a therapeutic value rating, 84 (31%) were rated as having high therapeutic value by at least one organization. Compared with non-expedited drugs, a greater proportion of expedited drugs were rated as having high therapeutic value among both FDA approvals (45% (69/153) v 13% (15/114); P<0.001) and EMA approvals (67% (18/27) v 27% (65/240); P<0.001). The sensitivity and specificity of expedited program for a drug being independently rated as having high therapeutic value were 82% (95% confidence interval 72% to 90%) and 54% (47% to 62%), respectively, for the FDA, compared with 25.3% (16.4% to 36.0%) and 90.2% (85.0% to 94.1%) for the EMA.ConclusionsLess than a third of new drugs approved by the FDA and EMA over the past decade were rated as having high therapeutic value by at least one of five independent organizations. Although expedited drugs were more likely than non-expedited drugs to be highly rated, most expedited drugs approved by the FDA but not the EMA were rated as having low therapeutic value.

scholarly journals Treatment patterns of melanoma by BRAF mutation status in the US in 2011-2017: A retrospective cohort study

2018 ◽  
Vol 29 ◽  
pp. viii456-viii457 ◽  
Author(s):  
L. Raskin ◽  
S. Shah ◽  
M. Braunlin ◽  
J. Buchanan ◽  
D. Cohan
Keyword(s):  
Cohort Study ◽  
Retrospective Cohort Study ◽  
Braf Mutation ◽  
Retrospective Cohort ◽  
Treatment Patterns ◽  
Mutation Status ◽  
The Us

scholarly journals Association between Race and Cancer-Related Mortality among Patients with Colorectal Cancer in the United States: A Retrospective Cohort Study

2019 ◽  
Vol 16 (2) ◽  
pp. 240 ◽  
Author(s):  
Sayaf Alshareef ◽  
Nasser Alsobaie ◽  
Salman Aldeheshi ◽  
Sultan Alturki ◽  
Juan Zevallos ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
United States ◽  
Cohort Study ◽  
American Indian ◽  
Alaska Native ◽  
Retrospective Cohort Study ◽  
Retrospective Cohort ◽  
The United States ◽  
The Us ◽  
Related Mortality

Colorectal cancer (CRC) is the third most common cause of mortality in the United States (US). Differences in CRC mortality according to race have been extensively studied; however, much more understanding with regard to tumor characteristics’ effect on mortality is needed. The objective was to investigate the association between race and mortality among CRC patients in the US during 2007–2014. A retrospective cohort study using data from the Surveillance, Epidemiology, and End Results (SEER) Program, which collects cancer statistics through selected population-based cancer registries during in the US, was conducted. The outcome variable was CRC-related mortality in adult patients (≥18 years old) during 2007–2014. The independent variable was race of white, black, Asian/Pacific Islander (API), and American Indian/Alaska Native (others). The covariates were, age, sex, marital status, health insurance, tumor stage at diagnosis, and tumor size and grade. Bivariate analysis was performed to identify possible confounders (chi-square tests). Unadjusted and adjusted logistic regression models were used to study the association between race and CRC-specific mortality. The final number of participants consisted of 70,392 patients. Blacks had a 32% higher risk of death compared to whites (adjusted odds ratio (OR) 1.32; 95% confidence interval (CI) 1.22–1.43). Corresponding OR for others were 1.41 (95% CI 1.10–1.84). API had nonsignificant adjusted odds of mortality compared to whites (0.95; 95% CI 0.87–1.03). In conclusion, we observed a significant increased risk of mortality in black and American Indian/Alaska Native patients with CRC compared to white patients.

scholarly journals 1210. Staphylococcal Acute Post-Operative Prosthetic Joint Infection (PJI) Treated With “DAIR” (Debridement and Implant Retention) and Impact of Rifampin: A Retrospective Cohort Study in France

2018 ◽  
Vol 5 (suppl_1) ◽  
pp. S367-S367
Author(s):  
Agathe Becker ◽  
Claire Triffault-Fillit ◽  
Emmanuel Forestier ◽  
Olivier Lesens ◽  
Bertrand Boyer ◽  
...  
Keyword(s):  
Adverse Event ◽  
Cohort Study ◽  
Treatment Failure ◽  
Median Duration ◽  
Retrospective Cohort Study ◽  
Retrospective Cohort ◽  
Methicillin Resistance ◽  
Joint Infection ◽  
New Drugs ◽  
Asa Score

Abstract Background Staphylococci are the most frequent bacteria in PJI. In patients with acute PJI (i.e., &lt;1 month following the implantation), DAIR with exchange of removal components followed by a combination of antibiotics including rifampin (RMP) (particularly RMP + fluoroquinolone) are recommended. Unfortunately, some patients could not receive RMP due to drug–drug interaction or stopped it due to an adverse event. Finally, it is unclear whether the dose and the duration of RMP influenced the prognosis. Methods Retrospective cohort study in four hospitals including patients with staphylococcal acute post-operative PJI treated with DAIR in 2011–2016. Univariate and multivariate Cox analysis and Kaplan–Meier curves were used to determine the risk factors for treatment failure. Results Seventy-nine patients were included (median age: 71 years [IQR 53–89]; 55 men [69.6%]; median ASA score: 2 [IQR 2–3]). Cultures revealed 65 (82%) S. aureus and 15 (19%) coagulase negative staphylococci infections, including 14 methicillin-resistant strains (18%). Among all isolates, only two (3%) were resistant to RMP and 16 (20%) were resistant to fluoroquinolone. The median duration of antimicrobial therapy was 92 days (IQR 31–152). Only 59 patients received RMP (75%), and 35 (44%) the combination RMP + fluoroquinolone. Median duration of RMP was 57 days (IQR 16–86) and median dose 14.6 mg/kg/d (IQR 13–17). Forty patients (51%) received RMP in the first 2 weeks and 43 patients (54%) received at least 2 weeks of RMP. Six patients (8%) developed an adverse event leading to RMP interruption. During a median follow-up of 443 days (IQR 220–791), 21 patients (27%) experienced a treatment failure including 12 persistence of the initial pathogen (57%) and nine superinfections (43%). An ASA score &gt;2 (OR 2.8; 95% CI 1.26–6.15), the use of RMP (OR 0.4; 95% CI 0.71–0.95) and the duration of RMP treatment (OR 0.83; 95% CI 0.75–0.92 per week of treatment) were significant determinants of the outcome (but not methicillin-resistance). Receiving &gt;2 weeks of RMP prevented the failure, but an introduction during the first 2 weeks did not influence the outcome. Conclusion In patients with staphylococcal acute PJI, the use of RMP and its duration strongly influenced the prognosis. As 25% of patients could not receive RMP, new drugs with anti-biofilm activity are required. Disclosures All authors: No reported disclosures.

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