The U.S. Food and Drug Administration (FDA) is asking for a federal investigation into its approval of a controversial Alzheimer’s drug.
Janet Woodcock, acting commissioner of the FDA, has asked for a probe into communications between FDA staff and Biogen representatives in the lead up to the approval of Aduhelm, the company’s Alzheimer’s drug.
The drug received FDA approval on June 7 despite limited data that it worked in clinical trials.
The move comes after a turbulent month for the FDA and Biogen, a Cambridge Massachusetts based drug maker.
In the weeks since approval, three members of an FDA advisory board have stepped down in protest of the drug’s approval, a congressional investigation has been launched and the agency even revised the drug’s label earlier this week.
Now the agency wants to look into its own communications to determine if proper procedure was followed in the lead up to the drug’s approval.
‘Given the ongoing interest and questions, today I requested that [The Office of the Inspector General] conduct an independent review and assessment of interactions between representatives of Biogen and FDA during the process that led to the approval of Aduhelm,’ Woodcock wrote on Twitter.
‘We believe an independent assessment is the best manner in which to determine whether any interactions that occurred between the manufacturer and the agency’s review staff were inconsistent with FDA’s policies and procedures,’
‘We believe this review and assessment will help ensure continued confidence in the integrity of FDA’s regulatory processes and decision-making.
‘If [The Office of the Inspector General] decides to conduct the review, the agency will fully cooperate & should they provide the agency with any recommendations, FDA would review expeditiously to determine the best course of action.
‘The trust of the American public, especially during these difficult times is of the utmost importance to the FDA – and we will continue to exercise transparency around our decision-making as allowed by the law. ‘
She also posted a statement to Twitter, outlining her concerns.
‘I have tremendous confidence in the integrity of the staff and leadership of the Center for Drug Evaluation and Research involved in the [Aduhelm] review and their commitment to unbiased and science-based decision-making,’ the statement says.
‘There continue to be concerns raised, however, regarding the contacts between representatives from Biogen and FDA during the review process, including some that may have occurred outside of the formal correspondence process.
‘To the extent these concerns could undermine the public’s confidence in the FDA’s decision, I believe that it is critical that the events at issue be reviewed be an independent body such as the Office of the Inspector General in order to determine whether any interactions that occurred between Biogen and FDA review staff were inconsistent with FDA policy and procedures.’
Woodcock has previously defended the drug’s approval from critics, saying it was ‘reasonably likely’ that the drug could help slow the cognitive decline associated with Alzheimer’s.
Some disagree, though, due to a rocky trial process by the drug manufacturer.
Biogen launched two clinical trials for Aduhelm, the commercial name of the drug aducanumab, in 2016.
Both were stopped midway because researchers concluded that neither trial would end up reaching its goal.
Later, the company revealed updated data from the second study that showed patients had 22 percent decrease in speed of their cognitive decline.
It also showed that it could remove amyloid beta plaques on the brain that some experts believe can reduce the cognitive decline caused by Alzheimer’s.
Woodcock and others have said that the removal of these plaques can stop cognitive decline, which would make the drug the only available Alzheimer’s treatment to do so.
The FDA has since rolled back a bit on the drug.
Earlier this week, they revised its label, now only recommending it to be prescribed to those in the early stages of Alzheimer’s, or with more mild case of the condition.
Three FDA advisory board members, Dr David Knopman of the Mayo Clinic, Dr Aaron Kesselheim of Harvard University and Dr Joel Perlmutter of Washington University St Louis, stepped down last month.
All three were among the opposition in the board’s 10-0 vote against approving Aduhelm.
Recommendations from the board are not binding, though, and the FDA is allowed to, and often does, make decisions that go against the board’s vote.
It is rare that a unanimous decision by the board is ignored, though, and the agency is generally more conservative than experts on the board – which was not the case with Aduhelm.
Knopman authored a study in November which analyzed the results of the clinical trials, and said he disagreed with Biogen’s claim that the drug was effective.
Kesselheim had some scathing words about the drug’s approval as well.
‘[Aduhelm] is probably the worst drug approval decision in recent U.S. history,’ Kesselheim wrote in a letter to FDA Commissioner Janet Woodcock obtained by Stat News.
‘It is clear to me that FDA is not presently capable of adequately integrating the Committee’s scientific recommendations into its approval decisions.’
The large price tag of the drug has been a point of concern as well.
A year of treatment using the drug will cost $56,000 a year, a huge premium to the $10,000-$20,000 the drug was expected to cost.
Two congressional committees in the House have also launched an investigation into the FDA’s review of the drug.
The House investigations were announced by Rep Carolyn Maloney, chairwoman of the House Committee on Oversight and Reform, and Rep Frank Pallone Jr, chairman of the House Committee on Energy and Commerce.
‘We have serious concerns about the steep price of Biogen’s new Alzheimer’s drug Aduhelm and the process that led to its approval despite questions about the drug’s clinical benefit,’ Maloney and Pallone Jr said in a statement.
Biogen said it will ‘of course cooperate with any inquiry we may receive from these committees,’ in response to a Reuters request for comment.
An analysis published by the Kaiser Family Foundation estimated that if just 500,000 Medicare recipients are prescribed Aduhelm, it would cost Medicare nearly $29 billion a year, far more than any other medication.
The narrower label may ease some of those concerns by shrinking the number of patients likely to get the drug, which requires monthly IVs.
Many hospitals have already stated that they plan to limit the drug’s use to patients with earlier stage disease.
Doctors could still prescribe the drug for more advanced patients, though insurers might refuse to pay for it, citing the FDA label.