Check out this Ask the Experts feature on our sister site Neuro Central, delving into the controversial recent approval of aducanumab.
Here, key thought leaders form a panel to discuss the Alzheimer’s drug that was approved for use by the US FDA in June 2021, a decision that caused much debate within the neuroscience community. To what extent has amyloid been shown to be a causative characteristic of Alzheimer’s? Does – and should – the ‘patient voice’ play a role in these kinds of approval decisions? How may this approval impact future Alzheimer’s drug approvals?
Experts for this feature include Robert Howard (University College London; UK), Mark Dallas (University of Reading; UK) and David Whitrap (Institute for Clinical and Economic Review; BS, USA). These experts provide their opinions on the questions below as we try to decipher the impacts and implications of this decision and how it was reached. Follow the links below to read the answers to each question.
- Given that Alzheimer’s is believed to exist across a continuum and isn’t a single disease, could aducanumab provide clinical benefit to individuals across that entire spectrum?
- Before this decision, the Alzheimer’s research space seemed to be moving towards tau as a more stringent predictor of Alzheimer’s disease and the next target to focus on for therapeutics – where do you stand on the tau versus amyloid debate?
- Central to the controversy regarding aducanumab is the decision to approve the drug based on its ability to reduce amyloid plaques, rather than a clear demonstration of its clinical benefit. To what extent has amyloid been shown to be a causative – rather than resultant – characteristic of Alzheimer’s?
- Do you think it is possible that reducing plaques could be a purely cosmetic result; removing a produced physical characteristic of Alzheimer’s without addressing the root pathogenic mechanism of the disease?
- Some stakeholders argue that this surrogate endpoint (the reduction in amyloid plaques) is not very person-centered – what are some of the challenges and implications associated with using surrogate endpoints in clinical research and approval decisions?
- To what extent does – and should – the ‘patient voice’ play a role in these kinds of approval decisions?
- The cost of the drug is believed to be high. How do you evaluate the cost–effectiveness of a therapy for which there are uncertain and contradictory data available? Could the cost of the drug exacerbate access disparities in dementia care?
- What kinds of post-approval data collection and analyses will be needed to maintain market access of aducanumab?
- How may this approval impact future Alzheimer’s drug approvals – with all the controversy, does it set a lower standard? Could this open up a role for real-world evidence in supplementing clinical trial data?