FDA System For Approving New Drugs Found Lacking By Study

A new study by federal researchers is highly critical of the U.S. Food and Drug Administration (FDA) process for approving new medications, such as the recent approval of the Alzheimer’s disease drug, Aduhelm, indicating that the agency regularly fails to rely on reasoning or precedents set in previous drug approvals.

According to findings published on September 21 in the Annals of Internal Medicine, FDA researchers indicate their own agency lacks an apparatus for looking at how it ruled on previous drug approvals, when the approval of a new drug is in doubt.

In the standard drug approval process, new medications must go through a series of clinical trials and FDA reviews, as well as advisory committee hearings to show safety, efficacy and need before gaining final approval by the FDA. In this study, researchers with the FDA sought to “understand the FDA’s evidentiary standards when flexible criteria are employed.” In other words, they sought to determine how the FDA makes decisions on drug approvals when the drug’s safety or efficacy are in question.

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The researchers looked at applications submitted from 2013 to 2018, which went through multiple review cycles due to a lack of evidence for clinical efficacy, to see how the FDA made its final approval decisions. This involved looking at advisory committee minutes, approval packages, FDA reviews and complete response letters.

According to the findings, which involved 912 application reviews, 117 went through multiple review cycles. Of those, 22 faced additional reviews due to issues related to clinical efficacy. When these drugs were rejected, it was usually due to concerns about the clinical meaningfulness of the observed effects of the drug, concerns about the primary endpoint of the studies, and inconsistent results.

However, the researchers found that seven of the 22 cases resulted in approvals which did not require new evidence, but just re-interpretations of the original evidence. They found no cases where the FDA decisions cited reasons used in previous drug approval decisions.

Aduhelm Approval Concerns

Researchers noted the recent controversy over the approval of the Alzheimer’s drug Aduhelm as an example of the confusion and lack of trust such “bespoke” decisions may cause.

The FDA granted accelerated approval of Aduhelm in early June, as the first Alzheimer’s disease therapy drug. However, the decision came after an FDA advisory panel nearly unanimously recommended against approval.

The agency’s approval led to several members of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee resigning in protest, and several congressional committees are looking into the decision. The agency has since faced a number of serious questions, such as why the FDA approved the drug when its own experts said it should not, and whether there was an improper working relationship between the agency and the drug’s manufacturer, Biogen.

Aduhelm’s approval has led to inquiries into the FDA’s approval process, and its relationship with manufacturers, by outside critics, lawmakers, and the agency itself.

“The FDA has no mechanism to find or tradition to cite similar cases when weighing evidence for approvals, resulting in standalone, bespoke decisions. These decisions show highly variable criteria for ‘substantial evidence’ when flexible evidential criteria are used, highlighted by the recent approval of aducanumab (Aduhelm),” the researchers determined. “A precedential tradition and suitable information system are required for the FDA to improve institutional memory and build upon past decisions. These would increase the FDA’s decisional transparency, consistency, and predictability, which are critical to preserving the FDA’s most valuable asset, the public’s trust.”


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