FDA Commissioner Denies Wrongdoing in Approval Process for Biogen Alzheimer’s Drug Aduhelm

“Nobody’s in trouble,” Dr. Califf said, while acknowledging the agency made mistakes in failing to properly documenting all of its interactions with Biogen. “These meetings need to be documented. Period.”

Dr. Califf said Billy Dunn, the agency’s neuroscience leader, made the right call to work closely with Biogen to reconsider the company’s data after the company halted its trials.

“Billy, he should have thought more about documentation. But, you know, that guy’s smart, and he’s very dedicated to the field,” Dr. Califf said. “A couple of years from now, people are going to look back and say, ‘OK, he should have documented these meetings, but basically, the general picture he got right.’” 

The 18-month investigation by the committees had found that the FDA and Biogen had an atypical number of meetings, calls or substantive emails, while the company was preparing to seek approval for its drug, and that at least 66 of those weren’t properly documented. The committee had also found that the agency and the company worked so closely on a document prepared for outside advisers that at least one paragraph of Biogen’s submission was written by FDA officials. 

The commissioner said a high level of interaction between agency officials and drug companies helps cut down on applications for drugs that don’t work and waste investment. In every clinical trial, he said, there will be questions about how to interpret the data.

“The way you resolve this is you get on the phone, look at the data jointly and see where you agree and disagree. That is normal,” Dr. Califf said. “Billy had access to data from early-phase trials that weren’t publicly available. That sort of confirmed the hypothesis that he had. But he couldn’t tell anybody.”

The committees also found that the FDA and Biogen pursued a broad label for Aduhelm indicating that it was intended for all Alzheimer’s patients, even though clinical trials had been limited to a smaller subset of patients. The label was later narrowed. 

The FDA last week granted accelerated approval to a drug similar to Aduhelm, Leqembi by

Eisai Co.

and Biogen. That drug, like Aduhelm, targets amyloid plaque in the brain, which researchers have long hoped would hold the key to slowing the progress of Alzheimer’s disease.

Leqembi is the first drug to clearly show in a more recent study that reducing amyloid results in clinical benefits to patients, though doctors have said its effects are relatively modest and far from a cure. 

The agency is poised to also make a decision on yet another Alzheimer’s drug, Eli Lilly & Co.’s donanemab, in the coming months.

Dr. Califf also defended the agency’s accelerated approval process, which allows companies to sell their drugs based on preliminary data. He said it gets treatments faster to patients who may have no other options and that his own mother added several years to her life thanks to a cancer drug granted accelerated approval.

Congress at the end of last year passed legislation giving the agency more power to require companies to do follow-up trials, after critics said too many companies were getting accelerated approval and then failing to do the work to confirm their drugs’ benefits.

Write to Liz Essley Whyte at [email protected]

Corrections & Amplifications
Leqembi is a drug similar to Aduhelm. An earlier version of this article misspelled Leqembi as Leqeumbi.

“Nobody’s in trouble,” Dr. Califf said, while acknowledging the agency made mistakes in failing to properly documenting all of its interactions with Biogen. “These meetings need to be documented. Period.”

Dr. Califf said Billy Dunn, the agency’s neuroscience leader, made the right call to work closely with Biogen to reconsider the company’s data after the company halted its trials.

“Billy, he should have thought more about documentation. But, you know, that guy’s smart, and he’s very dedicated to the field,” Dr. Califf said. “A couple of years from now, people are going to look back and say, ‘OK, he should have documented these meetings, but basically, the general picture he got right.’” 

The 18-month investigation by the committees had found that the FDA and Biogen had an atypical number of meetings, calls or substantive emails, while the company was preparing to seek approval for its drug, and that at least 66 of those weren’t properly documented. The committee had also found that the agency and the company worked so closely on a document prepared for outside advisers that at least one paragraph of Biogen’s submission was written by FDA officials. 

The commissioner said a high level of interaction between agency officials and drug companies helps cut down on applications for drugs that don’t work and waste investment. In every clinical trial, he said, there will be questions about how to interpret the data.

“The way you resolve this is you get on the phone, look at the data jointly and see where you agree and disagree. That is normal,” Dr. Califf said. “Billy had access to data from early-phase trials that weren’t publicly available. That sort of confirmed the hypothesis that he had. But he couldn’t tell anybody.”

The committees also found that the FDA and Biogen pursued a broad label for Aduhelm indicating that it was intended for all Alzheimer’s patients, even though clinical trials had been limited to a smaller subset of patients. The label was later narrowed. 

The FDA last week granted accelerated approval to a drug similar to Aduhelm, Leqembi by

Eisai Co.

and Biogen. That drug, like Aduhelm, targets amyloid plaque in the brain, which researchers have long hoped would hold the key to slowing the progress of Alzheimer’s disease.

Leqembi is the first drug to clearly show in a more recent study that reducing amyloid results in clinical benefits to patients, though doctors have said its effects are relatively modest and far from a cure. 

The agency is poised to also make a decision on yet another Alzheimer’s drug, Eli Lilly & Co.’s donanemab, in the coming months.

Dr. Califf also defended the agency’s accelerated approval process, which allows companies to sell their drugs based on preliminary data. He said it gets treatments faster to patients who may have no other options and that his own mother added several years to her life thanks to a cancer drug granted accelerated approval.

Congress at the end of last year passed legislation giving the agency more power to require companies to do follow-up trials, after critics said too many companies were getting accelerated approval and then failing to do the work to confirm their drugs’ benefits.

Write to Liz Essley Whyte at [email protected]

Corrections & Amplifications
Leqembi is a drug similar to Aduhelm. An earlier version of this article misspelled Leqembi as Leqeumbi.