When I was diagnosed with AFM in 2014, I thought that a vaccine or treatment within a year. Almost 10 years later, a vaccine and treatment are still nowhere to be found. Children diagnosed with AFM are still given the same IVIG and physical therapy treatment I was getting in 2014. Why have treatments for rare neruological conditions stagnated? Was all the struggle I went through with experimental treatments all for nothing?
One of the reasons why treatments for rare neurological diseases are not changing is because of the wait times for clinical trials to take place. A clinical trial is essentially a study to understand whether a treatment is safe or not. There are four steps to a clinical trial, and by understanding these steps, we can learn why new treatments are being created as fast as we might want them to.
The first phase of a clinical trial is to give small doses to see whether or not the treatment is safe. This phase is purely a safety measure to make sure the treatment does not have any adverse effects. After the first phase is completed, there are larger trials with more people to see how the treatment affects people from different backgrounds. After larger trials, which is phase 2, a study is done analyzing the results (phase 3). If the treatment is proven to be safe and effective, there are studies to determine what the long term side effects are, which is phase 4. All these phases must be completed for a treatment to be given to the public.
These 4 phases understandably take a large amount of time, especially since the FDA needs to understand the long term side effects of a treatment. These clinical trials can take as long as 10 years in some cases. AFM's spike lasted for 6 years, so by the time a vaccine or intervention is created, the amount of cases will be so low that the money invested in the treatment would not be worth it. Phase 2 and 3 of clinical trials need large sample sizes to test safety and effectiveness, which is impossible with rare neurological conditions that have less than 1000 cases. AFM only had 751 confirmed cases, and many of these children live across the globe, meaning that a safe clinical trial with a large sample size is impossible to conduct.
I believe that for rare neurological conditions, the clinical trial process should be streamlined in order to help affected children quickly. The aftereffects of AFM include paralysis in nearly all cases, and it is worth taking the risk of streamlining the trial process in order to get help for these children fast. If the children and their families both give consent for an experimental treatment, and they know the risks, the treatment should be given. I do not believe that the FDA should have the power of restricting experimental treatments to children with rare neurological conditions, because at the end of the day, the choice should be given to the children and their families.
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