On 7th of June 2021, the American Food & Drug Administration (FDA) officially approved the first new Alzheimer’s drug in almost 20 years. The drug is called Aducanumab and it was developed by Biogen, a U.S. pharmaceutical company. Aducanumab sticks to amyloid – a protein that creates large chunks of plaques in the brain – and helps break up these plaques.
The problem is that couple of years earlier, in 2019, the drug’s trials came to an end, as it was shown that it did not help with memory deterioration. After re-analysing the results, Biogen came back and said that in fact higher doses did result in the slowing of cognitive decline.
The FDA eventually ignored the results of its advisory committee (causing multiple members to resign from it in protest). It has now approved the drug using its “accelerated approval” pathway. The process is used for drugs that are not yet confirmed to be advantageous for patients, but that are “reasonably likely to be”.
FDA on its decision: “…treatment with Aduhelm (the drug) was clearly shown in all trials to substantially reduce amyloid beta plaques. This reduction in plaques is reasonably likely to result in clinical benefit.”
This raises all kinds of questions, the main one being: are we in the new era of research and development?
Why is this considered to be a controversial decision?
1. It has been shown that amyloid lowering has not led to cognitive benefits, and this has made it a sticking point for researchers. The “juggling of data” done by Biotech has infuriated quite a few members of the pharmaceutical community and has caused a serious split. After all, if the drug does not work, what right does the parent company have from benefiting from it?
2. Half-finished products. While at the moment pharmaceutical companies fight for their place under the sun by producing drugs that work, the Aducanumab decision by FDA means that more of them may adapt to the changing market landscape, by releasing products that are not yet “fully functional”. This means an increase of cost for the end-consumers, who would be buying newer “versions” of drugs as they come out, instead of the ultimate solution right away. (Think iPhone but paracetamol instead).
3. Side effects and public safety. While speeding up the process on paper looks great, you can’t always speed up nature. Some side effects become apparent only after a longer period of time. Resorting to “accelerated approval” could mean bypassing such extensive testing, and may end up raising the number of cases where patients suffer from these side effects in the future.
What’s the good news?
1. Sometimes people simply can’t wait. When it comes to cancer, the FDA has been known to be more lenient when it comes to approving certain drugs. And given the choice between giving up or possibly buying yourself more time, how many people would choose the former?
2. More funding for research. At the end of the day, it’s all about funding. Many investors shy away from the medical field due to long ROI cycles. Adopting a model where there is more risk taken, but results become apparent more quickly means shortening these cycles. That means the inflow of more capital. Ultimately humanity benefits from more research done as the result of that – a win-win. (Notwithstanding the above “safety” argument).
3. Alzheimer’s and generally, dementia is a condition that is horrible and that is currently incurable. Recent approval of the drug, means once again that more attention is paid to this space. Which in turn, once again, means more attention from potential investors. Could this problem be solved in our lifetime thanks to the recent news? We will have to wait and see.
More risk – quicker reward?
Over the last 20 years the increase of venture capital available to companies has rewarded risk takers. After all, every time you start a new business/company you are taking a risk. A lot of times, such ventures simply do not work out, but when they do, investors can get over 100x on their investment, and it makes their efforts worth it.
Because of the increase of capital inflow, more and more ideas are getting funded. Even the governments get involved in the process – just look at the tax grants received by Elon Musk’s SpaceX. Is anyone complaining? Certainly not the space enthusiasts. Are we all winners when it comes to space exploration? Potentially.
With medicine, things get trickier. Nobody wants to risk someone’s life or serious damage to their health. Another potential problem – approving a drug through the “sped-up” process means that we may get to the point where fewer effective treatments are pushed ahead of the ones that actually make a difference.
Then again, free market is a thing to behold. It is not always perfect, but in time it may balance itself out and we may yet see regulators adjust according to the effectiveness of certain processes when it comes to approving drugs. After all, these things are driven by capital and public’s perception. If the latter is managed well, and the former is distributed where it’s needed, we are in for quite a ride in the next 20 years.