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.

Status and Determinants of Treatment Outcomes Among New Tuberculosis Patients in South Korea: A Retrospective Cohort Study

2021 ◽  
pp. 101053952110005
Author(s):  
Hyunjin Son ◽  
Jeongha Mok ◽  
Miyoung Lee ◽  
Wonseo Park ◽  
Seungjin Kim ◽  
...  
Keyword(s):  
Cohort Study ◽  
South Korea ◽  
Success Rate ◽  
Treatment Outcomes ◽  
Retrospective Cohort Study ◽  
Retrospective Cohort ◽  
Health Care Facilities ◽  
Tuberculosis Patients ◽  
Lost To Follow Up

This is a retrospective cohort study using notification data in South Korea. We evaluated the nationwide status, regional differences, and the determinants of treatment outcomes among tuberculosis patients. Treatment success rate improved from 77.0% in 2012 to 86.0% in 2015. The lost to follow-up rate was higher among older people, males, and foreign nationals. Health care facilities designated for the Public-Private Mix (PPM) project showed higher success rate and lower rate of lost to follow-up. Moreover, municipalities with low regional deprivation index had higher PPM project coverage. Since there is a large regional difference in the coverage of the PPM project, an additional community-based support program should be implemented, especially for tuberculosis patients residing in region with low PPM project coverage.

scholarly journals Pioglitazone use and Parkinson’s disease: a retrospective cohort study in Taiwan

BMJ Open ◽  
2018 ◽  
Vol 8 (8) ◽  
pp. e023302 ◽  
Author(s):  
Hsiu-Feng Wu ◽  
Li-Ting Kao ◽  
Jui-Hu Shih ◽  
Hui-Han Kao ◽  
Yu-Ching Chou ◽  
...  
Keyword(s):  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Cohort Study ◽  
Retrospective Cohort Study ◽  
Retrospective Cohort ◽  
Asian Population ◽  
Secondary Outcome ◽  
Study Cohort ◽  
Patients With Diabetes

ObjectivesMany researchers have expected pioglitazone to serve as an effective neuroprotective agent against Parkinson’s disease (PD). Therefore, we conducted this cohort study to investigate the association between pioglitazone use and PD by using a large Asian population-based dataset in Taiwan.DesignRetrospective cohort study.SettingTaiwan.Participants7906 patients with diabetes who had received pioglitazone were defined as the study cohort, and 7906 matched patients with diabetes who had not received pioglitazone were defined as the comparison cohort.Primary and secondary outcome measuresWe tracked each patient individually over a 5-year follow-up period to identify those diagnosed as having PD during this period. We performed Cox proportional hazard regression analyses to evaluate the HRs for PD between the study and comparison cohorts.ResultsThe findings indicated that among the sampled patients, PD occurred in 257 (1.63%): 119 (1.51%) pioglitazone users and 138 (1.75%) non-users. The adjusted HR for PD within the follow-up period was 0.90 (95% CI: 0.68 to 1.18) in the patients who had received pioglitazone compared with the matched patients who had not received pioglitazone. Moreover, this study revealed that pioglitazone use was not associated with PD incidence in men (HR: 1.06, 95% CI: 0.71 to 1.59) or women (HR: 0.84, 95% CI: 0.61 to 1.15).ConclusionsThis study did not find the relationship between pioglitazone use and PD incidence, regardless of sex, among an Asian population of patients with diabetes.

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