questionable expensive Alzheimer's drug may get FDA approval

Crushed

It would become a blockbuster drug within several years, analysts predict, costing tens of thousands of dollars annually per patient and bringing a windfall to its manufacturer, Biogen.

Patient groups, desperate for treatments, are pushing for approval. But greenlighting the drug would fly in the face of objections from several prominent Alzheimer’s experts and the F.D.A.’s independent advisory committee.

In November, the committee voted overwhelmingly against recommending approval, saying data failed to demonstrate persuasively that aducanumab slowed cognitive decline. Three advisory committee members later wrote a point-by-point critique of the evidence. Other scientists, and an independent think tank, say aducanumab hadn’t shown convincing benefit to outweigh its safety risks.

“This should not be approved, because substantial evidence of effectiveness hasn’t been shown,” said Dr. Lon Schneider, director of the California Alzheimer’s Disease Center at the University of Southern California and one of many site investigators who helped conduct one of the aducanumab trials. “There’s very little potential that this will address the needs of patients.”

  https://www.nytimes.com/2021/06/05/health/alzheimers-aducanumab-fda.html?action=click&module=Top%20Stories&pgtype=Homepage

  

Lane Simonian

Thank you for posting this, Crushed.  It is important to know that any numbers are averages in these clinical trials.  Some people do better and some people do worse.  What other anti-amyloid trials (even though they are attacking different forms of amyloid and at times in different ways) is that attacking amyloid hardly slows down the disease at all in non-ApoE4 carriers.  For some carriers with two copies of the gene, Biogen's drug aducanumab may slow down the disease in a way that is barely clinically significant, but it is also this group of patients who may suffer serious side effects from the drug including brain swelling and micro-bleeds.  For those who don't suffer these side effects, aducanumab may slow down  early disease progress more along the lines of non-ApoE4 carriers.  Potentially it is a big price to pay for little gain.

I understand how everyone wants a drug that will help even if a little bit.  But a little bit does not justify an expensive drug that may do more harm than good.  Biogen should have been much more transparent in presenting its data, but knew that doing so would result in a limited and conditional approval at best.  And adovocacy organizations wanting to show some progress against the disease rallied patients and their caregivers to try to pressure a partly-captured FDA to approve a drug that should never have made it this far.

Lane Simonian

The FDA just approved aducanumab for Alzheimer's disease.  Now we have another almost useless drug to "treat" the disease.

dayn2nite2

Treatment theater.  How sad.  And so many will be prescribed this and spend lots of money taking it to no avail.

Rescue mom

It’s so sad to me that a big money-maker for a big company

Jeff86

This is yet one more decision that tarnishes the FDA’s reputation.  It appears as if politics trumped science.  The decision required the FDA to set aside or ignore its independent advisory commission’s thumbs-down verdict.

While the unmet need is enormous, that doesn’t justify the approval of a medicine that raises both efficacy and safety issues.  The advocacy of patient groups (like the host of this website!) may have been a factor.  To me, that advocacy is regrettable as the drug’s benefits are clinically negligible and risks of brain bleeds or swelling are concerning.

Even if the drug were more effective, it wouldn’t help most of our LO’s, as it’s targeted for early stage.  But giving false hope to patients and families, at high cost and with substantial risks, isn’t good science, it isn’t good medical practice, and it isn’t good healthcare policy.

Rennbird

I am just a layperson but I have always thought that in my mother and husband’s cases, Aricept was a waste of money and a false hope.  It ranked right up there with coconut juice.

JJ401

I read the cost will be about $50,000 per year. That's prohibitive for private pay. Even with a Medicare drug plan if it's a tier four drug, I think you'd need $8,000 to $10,000 for your part of the cost. 

DH is past the MCI stage, so I don't think it would be even recommended for DH. I am concerned that spouses and caregivers will get family pressure to get this drug for their loved one even if it is not indicated for their level of disease.

Jeff86

There’s a big leap, a big assumption, in the FDA’s approval.  From their decision letter:

 Although the Aduhelm data are complicated with respect to its clinical benefits, FDA has determined that there is substantial evidence that Aduhelm reduces amyloid beta plaques in the brain and that the reduction in these plaques is reasonably likely to predict important benefits to patients.

The reduction in plaques will not necessarily result in a clinical benefit.  There is considerable uncertainty whether plaques are a result of the disease or a cause of the disease or its symptioms.  

This drug is delivered monthly, via intravenous injection.  Follow up MRI’s will be necessary to monitor side effects.

This is not the home run some are touting it to be.

Larrytherunner

No way aducanumab is going to be approved. Not effective and possible serious side effects. I think this is an an effort by Biogen top executives to manipulate the stock price.

jfkoc

another disappointment ....sigh

Larrytherunner

Ok. I am wrong. I guess spending a billion dollars on developing a drug is all it takes to get partial approval. Big pharma gets its way again with big money. 

Thanks MPSunshine for posting this article on the Clinical Trials board

https://www.washingtonpost.com/health/2021/06/07/fda-approves-alzheimers-drug-aducanumab/

JulieB46

Thanks for posting this, Crushed.  I haven't been on in a while, DH now has a diagnosis of AD, instead of Unspecified Dementia.  This came across my phone this morning, and then my DH's sister called me and said, "I have something to tell you"  I told her I already knew about the drug.  She acted like she had to keep me in the loop.  Are you kidding me????  I get notifications from Alz.org etc. daily.  She didn't know that it is indicated for MCI, not AD that has progressed to mid stage or beyond.  DH was diagnosed with MCI in 2014.  She also doesn't know the side effects or the cost or the number of cases that didn't show improvement.  I just get sick of people who don't live this on a daily basis, giving unsolicited advice.  Of course, I came home and my DH is excited about the drug because he saw it on CNN and his sis called him.  I had to explain that it is important news, but he might not be a candidate because he is beyond MCI.  That was fun trying to explain.  I told him I'd call his doc and see what he thought.  Who ever thought we wouldn't be ecstatic to have a new AD drug?

JJ401

 On the NPR website there is a discussion of this drug. At the end of the article a leading expert says he will not prescribe the drug. 

...the drug's benefits are ambiguous at best and not worth this cost..

 https://www.npr.org/2021/06/07/1003964235/fda-approves-controversial-alzheimers-drug-aducanumab

1962ART

I am interested to see if my DH's neurologist, even brings the topic up.  I can't imagine DH would be a candidate for this, but I guess I'll keep an open mind.  From reading the articles, the side effects are many and DH's medical condition is a complicated one.  So, for me, no rush to judgment, but highly skeptical it will have any value for us.  DH has not mentioned reading about this and I have no intention of mentioning it.

John1965

JJ401 is right. I’m already getting texts from friends and former colleagues who “saw a new drug on GMA” and think our prayers have been answered. Ugh!

French

We will see if it will be approved here. I read news on it. It was in an economic newspaper and was criticizing the decision because of very weak proofs of efficiency and price.

It has also been tested in Europe, so our researchers will certainly give their opinion