This drug, aducanumab, engineered by Biogen, would be the first new Alzhiemer’s treatment in almost twenty years. However, the FDA’s panel of medical experts declined to endorse it, saying there wasn’t enough evidence that showed its effectiveness in slowing cognitive decline. While the non binding vote of the Food and Drug Administration’s panel doesn’t signify a complete rejection of the drug by the agency, it shows that many experts do not see its effectiveness. This disagreement starkly contrasts a presentation by Dr. Billy Dunn, the director of the FDA’s office of neuroscience, who believes that “the evidence supporting its approval appears strong”. Many of the agency’s reviewers also agree with Dr. Dunn, a development that raised Biogen’s stock price by over 40 percent.
Currently, almost six million US residents and 30 million people across the globe suffer from Alzheimer’s. This statistic is expected to double by 2050. Aducanumab, Biogen’s Alzheimer's drug, would be the first medicine that would act against Alzheimer’s by attacking the biology of the disease. It is designed to clear the amyloid plaques in the brain that are associated with Alzheimer’s. This drug is different from the ones made twenty years ago because it treats the disease process rather than simply reducing the symptoms. It is considered to be most helpful to patients with mild cognitive decline. The panel did not support it because it did not meet their requirement of showing two successful studies - one shows that aducanumab slowed the progression of Alzheimer’s while the other study did not. To make up for the failure of the second study, the FDA presented only positive evidence and analysis that was favorable to the drug. Dr. Scott Emerson, a biostatistician at the University of Washington said: “This analysis seems to be subject to the Texas sharpshooter fallacy, a name for the joke of someone first firing a shotgun at a barn and then painting a target around the bullet holes”. This did not go over well with the FDA's advisory panel. Additionally dr. Lon Schneider who directs the California Alzheimer's Disease Center at the University of Southern California said the FDA should not approve of aducanumab because “1 identical study was flat-out negative. The other one was biased and unblinded, and whatever effects it showed were truly trivial.”
Despite these varying results and the pushback from the advisory panel, the agency has said that it still wants to approve aducanumab. Medical experts are now concerned about the possible harm of Alzheimer’s patients taking this drug that might not even help them.
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While it surely seems that an improved drug to treat Alzheimer's could be very beneficial, more studies and statistical significance must be proven before any drug is hastily administered to the public. Since the drug has not received FDA approval, it will be interesting to see what steps will be necessary to secure the trust that the drug is actually capable of treating the disease. It is also extremely concerning that the results and testing not only do not prove that success of the drug, but have been obviously manipulated in order to receive quick approval. Hopefully, if Aducanumab ever does get approved and eventually widely available to patients, it will have met the necessary criteria and evaluated by multiple experts across the field, to ensure medical safety and eliminate any corporate bias.
It was said that Biogen had halted Aducanumab trials because of disappointing intern trial analysis back in March 2019. However, to everyone’s shock, it was restarted due to new favorable results from one of the 2 studies. The other study was said to have had a slightly worse result than the placebo group. Like Rosella mentioned, I also believe that Aducanumab is a step in the right direction for Alzheimer treatment, but it should definitely be retested. There will be major consequences if this product were to be pushed out prematurely, especially when it has questionable and fabricated results. Brain swelling was one of the common symptoms of the drug, which might have unpredictable effects on the patients wellbeing. From the company’s perspective, they just want to see profits after they’ve invested all this time, money and effort into the drug pipeline. However, quality control is also a component of the drug pipeline that the company is responsible for. As referenced by the FDA committee, it is quite unreasonable to disregard the results of the 2nd study. That slight margin of error may not seem small, but it will definitely foster public distrust in pharmaceutical companies and the FDA's credibility.
Although it would have been fantastic to see a new Alzheimer's drug reach patients, I have got to agree with the previous commentators and the FDA's advisory panel. As incredibly frustrating as it must be for the producers of Aducanumab, not to mention the millions of people suffering from the disease, the rejection to endorse it was the right decision. We all have to believe that the FDA exists for one reason and one reason only- to protect the safety and health of the American people, or as they say on their website to protect"public health by ensuring the safety, efficacy, and security of human and veterinary drugs." Since these first two trials have shown mixed results, the drug ought to go through more testing before it is released on the larger scale for hospitals to give to patients. WIth the biotechnology industry growing by the day, I have no doubt that in the next couple years Aducanumab will reach the level of proper protocols and efficacy or another comparable drug will become available. I think under the Biden administration, more focus will be put back on science and hopefully pharmaceutical companies will also feel that push.
It makes sense that the drug was rejected, as only one experiment projected successful data. Like the previous commentators, I agree that it is a shame that this drug failed to pass when it could have helped many people with problems associated with Alzheimer's, but the risk of a drug that has the possibility of causing harm (because it is not 100% guaranteed to improve Alzheimer's) should not be distributed out to the public, nonetheless be approved by the FDA. But with work similar to this, there is definitely a possibility in the future for a drug to improve the lives of others who suffer from Alzheimer's, and I'm excited to see what comes!
It is not uncommon for drugs to fail to receive FDA approval, and in fact, most drugs ultimately fail. While the direction that the Aducanumab takes is a step forward from previous methods, there just does not seem to be enough evidence to truly allow it to be used in people beneficially. It does not make sense for the drug to be continually retested, as some tests already indicated that it is not effective and the company would essentially be fishing for any sort of positive result just to make money. No drug is guaranteed to be successful 100% of the time, and while there are many drugs that appear promising, they are more likely than not going to fail. The FDA exists in order to maintain a high standard for the development of drugs, and letting Aducanumab slip by approval would be the opposite of what the FDA is meant to do. Similarly, vaccine development for COVID-19 may appear promising, and many are often quick to jump to conclusions, for drugs require extensive testing in order to be determined as safe, and even then, there is still a chance for them to be recalled if new information arises.
While people want to see a treatment for Alzheimer's, it shouldn’t be hasty to approve a drug that is giving varying results. It makes sense that the FDA rejected this drug since it was only proven positive in one test while the other was negative. With the risk of the drug not being effective 100%, the FDA clearly doesn't want to approve and distribute something that is dangerous to the public. But seeing that it isn’t completely a failure, there is a chance of a successful drug that will help treat Alzheimer's.
Even though there might have been positive results shown in the tests, in my opinion, a good thing that it was not distributed because it did not have a 100% success rate. While its generally not possible that it will have 100% success over millions of trials, even a 1% failure rate across millions of patients could be devastating. It is a good step however, as the information learned from this iteration could help the success rate for the next drug produced for Alzheimers
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