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

Abstract

ObjectiveTo 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.

Achievements and challenges of the Sakigake designation system in Japan

“…Furthermore, we did not collect the designations of accelerated pathways other than Sakigake, BTD, PRIME, and the orphan designations in Japan, the USA and the EU. A recent study revealed that the therapeutic value rating for approved drugs through expedited programmes in the USA/EU was higher than that for nonexpedited ones 27 . Nevertheless, most expedited drugs approved by the FDA but not the EMA were rated as having low therapeutic value.…”

Next-Generation Bioinformatics Approaches and Resources for Coronavirus Vaccine Discovery and Development—A Perspective Review

“…Before initiating the mass vaccination stage and development process, the volunteer clinical trial is subjected to a code of conduct in terms of safety regulation of the vaccines. This clinical trial plan and approval for general use are strictly regulated by a governing body such as the Food and Drug Administration (FDA) and European Medicines Agency (EMA) [ 45 ]. The doses and schedule for the vaccine are calculated during restricted human studies and tested in phase III studies.…”

Putting patients first in medicines regulation?

“…But research shows this is rarely the case 16. More than two thirds of expedited drugs do not perform better than existing alternatives 17. Some are later found to be ineffective 1819…”