The experts in the field who prepared the AUR for aducanumab have done a great service for practitioners who are faced with speaking to patients and need to know how to weigh the information that has been provided.
Unfortunately, the information from the trial and from the FDA decision is scant. We have very little information on which to construct these recommendations.
I am so glad that my colleagues were able to step up and summarize what we do know. I hope the company and the FDA will soon become more transparent, and allow us to know critical information such as how the trials were conducted, how many people failed screening and why, and what data was used to allow the FDA to approve on the accelerate basis. Without this information we are truly working in the dark.
In the setting of a broad FDA label, the AUR make an important contribution by providing clear guidelines around which patients may be candidates for treatment and, importantly, which clinical features should be considered exclusionary. I found the suggested algorithm around ARIA monitoring and management particularly helpful, as outside of trialists few clinicians will have had experience managing this common adverse effect of the drug.
That said, there are still many outstanding pragmatic and scientific questions about how to implement this new class of drugs in patient care.
Should APOE genotyping be required to inform the discussion of potential risks, or just recommended as in the AUR?
Will the recommended MRI surveillance schedule be adequate to safely manage ARIA in a “real world” setting?
How can we evaluate whether a particular patient is clinically responding to therapy, beyond comparing to historical, group-level data?
Are plasma biomarkers sufficiently accurate to be used as a “stand-alone” biomarker for establishing amyloid positivity?
Can tau biomarkers (PET, CSF, or plasma) improve the selection of patients who are likely to respond clinically to amyloid-lowering therapy, as suggested by the donanemab data?
Should we be treating continuously, as was done in the aducanumab and lecanemab trials, or can treatment be paused once amyloid PET negativity is achieved, as suggested by the donanemab design?
We are at the beginning of a new era of AD therapy, and it will be exciting to see how we refine our approach to patient care as we gain “real world” experience with molecular therapies.
Comments
Mount Sinai School of Medicine
The experts in the field who prepared the AUR for aducanumab have done a great service for practitioners who are faced with speaking to patients and need to know how to weigh the information that has been provided.
Unfortunately, the information from the trial and from the FDA decision is scant. We have very little information on which to construct these recommendations.
I am so glad that my colleagues were able to step up and summarize what we do know. I hope the company and the FDA will soon become more transparent, and allow us to know critical information such as how the trials were conducted, how many people failed screening and why, and what data was used to allow the FDA to approve on the accelerate basis. Without this information we are truly working in the dark.
View all comments by Mary SanoUCSF
In the setting of a broad FDA label, the AUR make an important contribution by providing clear guidelines around which patients may be candidates for treatment and, importantly, which clinical features should be considered exclusionary. I found the suggested algorithm around ARIA monitoring and management particularly helpful, as outside of trialists few clinicians will have had experience managing this common adverse effect of the drug.
That said, there are still many outstanding pragmatic and scientific questions about how to implement this new class of drugs in patient care.
We are at the beginning of a new era of AD therapy, and it will be exciting to see how we refine our approach to patient care as we gain “real world” experience with molecular therapies.
View all comments by Gil RabinoviciMake a Comment
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