The United States Food and Drug Administration (FDA) approved a new drug called aducanumab (Aduhelm). The new drug is said to delay clinical decline in patients with Alzheimer’s disease. There, however, are concerns over the absence of evidence to substantiate the claims.
The drug which reportedly cost the US government $56 000 (£40 000; €46 000) per year per patient was approved through the ‘accelerated approval pathway.’ The accelerated approval pathway is employed when uncertainties abound over a drug’s efficacy but it’s believed it could provide “meaningful therapeutic benefit over existing treatments” for serious or life threatening illnesses.
The approval process consists of procedures where the manufacturer must conduct post-approval studies known as phase IV confirmatory trials to “verify the anticipated clinical benefit.” The FDA reserves the right to remove the drug from circulation If these trials do not verify the anticipated benefit.
In this instance, the FDA did not specify which patients are eligible for treatment with aducanumab.
The root causes of Alzheimer’s have eluded researchers but there is broad agreement that the brain plaque targeted by aducanumab is just one factor. There is, however, increasing evidence that suggests family history, education and chronic conditions, such as diabetes and heart disease, may be responsible.
“This is just one piece of the puzzle and I think all these other options need to be explored and amplified,” Dr Ronald Petersen, a Mayo Clinic dementia specialist who has consulted for Biogen and other drug makers told The Associated Press.
An FDA outside panel of neurological experts voted “no” to a series of questions in November on whether reanalysed data from a single study submitted by Biogen showed that the drug was effective.
Biogen halted two studies of the drug in 2019 after disappointing results suggested aducanumab, marketed by Biogen as Aduhelm, would not meet its goal of slowing mental and functional decline in Alzheimer’s patients.
