ALS, or amyotrophic lateral sclerosis, is a neuron disease that affects over 6,000 Americans each year (Festoff 4). On average, fifteen Americans are diagnosed with amyotrophic lateral sclerosis daily, leading to an annual death toll of two out of every 100,000 Americans (Festoff 4). Amyotrophic lateral sclerosis can kill anyone; amyotrophic lateral sclerosis has no racial, no geographic, and no demographic boundaries. Amyotrophic lateral sclerosis’ progression is amongst the deadliest of any modern day disease. Once diagnosed, amyotrophic lateral sclerosis patients generally are given two to five years to live, with roughly 5% chance of living more than 10 years after diagnosis. Steve Gleason, a former NFL player, was diagnosed with the disease in 2011. Steve Gleason said “Because ALS is underfunded, patients have had no option but to fade away and die. That is not OK” (CBSsports 1). Being diagnosed with amyotrophic lateral sclerosis is a death sentence, and with today’s medicine and technology, this should not be the case. Amyotrophic lateral sclerosis patients have slimmer than a 5% chance of living more than ten years after diagnosis, making amyotrophic lateral sclerosis one of the deadliest diseases in the world today. There is a drug, Genervon, which can treat amyotrophic lateral sclerosis. This drug has caused a lot of controversy because the Food and Drug Association has not approved it, but the European Medicine Agency has approved it. This begs the questions does the FDA have a reason to be withholding this potentially life-saving drug from patients who essentially have no hope already and if so what is the reason? The FDA released a statement about Genervon recently and it raised a lot of controversy because they will not release why the drug has not been approved and only asked the company responsible for Genervon to release all the data from a recent trial. This statement devastated thousands of people with amyotrophic lateral sclerosis because this means that the FDA is still far from approving this drug to treat amyotrophic lateral sclerosis. Ultimately, this begs the question, is the FDA saving lives or ending lives by not releasing this drug? The FDA needs to reevaluate its approval process and improve their communication with the public to ensure the safety of Americans.

The FDA uses a scientifically based process to determine the safety and efficacy of experimental drugs (fda.gov 1). The FDA approval process involves identifying the class of a device and based on the class, goes through one of three approval processes. There are only three types of classes for the devices. The first class are devices that involve the lowest risk and the third class are devices that involve the highest risk (jamanetwork.com 1). The three different types of approval processes are no FDA review, the 510(k) process and the rigorous PMA process. From there, the FDA approves drugs by doing three or occasionally four stages of trials (fda.gov 1). The first stage of trials is used to determine dosing, how the drug is metabolized and excreted, and to identify the possible side effects (fda.gov 1). This is generally the smallest of the trials with between twenty and eighty volunteers and some of these volunteers have the disease and some don’t (fda.gov 2). The second stage of trials involves between one hundred and three hundred participants who have the disease for which the medicine is being tested (fda.gov 2). This stage of trials is used to begin to see if this drug is effective in treating those with the disease and gather additional safety data (fda.gov 2). If the stage two trials indicate that the drug may be effective and the risks are acceptable, the drug moves on to the next phase of trials (fda.gov 2). In the third stage of trials, the drug is tested with between one thousand and three thousand people with the disease (fda.gov 2). This stage of trials is used to further test whether the drug is effective or not, monitor the side effects, and if available, compare its effects to a standard treatment (fda.gov 2). The fourth stage of trials is hardly ever used and when it is, it is after the drug has been approved and on the market (fda.gov 2). The amount of people in this stage of trials varies and used to find out more about long-term risks, benefits and to test the product in different populations of people (Miller 1). Dr. Zuckerman, from the National Research Center for Women and Families, did an experiment to examine the effectiveness of the FDA approval processes. The experiment sought to examine which approval processes were responsible for the majority of Class One recalls, which the FDA defines as the highest risk (Zuckerman, Brown, Nissen 7). From January 2005 to December 2009, there were 113 Class One recalls. Upon further examination, The PMA process was used to approve only 21 of the 113 devices listed as high-risk recalls that could cause serious health problems or death (19%). Eighty were cleared through the 510(k) process (71%), and an additional 8 were completely exempt from FDA regulation and were merely registered with the FDA (7%). In addition, four were counterfeit devices or categorized as other (4%) and did not go through any of the 3 processes for approval, clearance, or registration (Zuckerman, Brown, Nissen 7). In January of 2004, the United States Government of Accountability Office published a critical article of the 510(k) process and the FDA requested that the Institute of Medicine conduct an independent outside review (Zuckerman, Brown, Nissen 3). From this review, in August 2004, the FDA released an internal report that indicated that several changes were made to the approval process (Zuckerman, Brown, Nissen 4). Even after the FDA made these changes, it clearly was not nearly enough because the 510(k) process still causes by far the most recalls. 

The FDA is generally only in charge of ensuring the safety of the United States while the European Medicine Agency is in charge of all twenty-eight countries in Europe. The European Medicine Agency was formed in 1995, which is much newer than the FDA, which was formed in 1938 (fda.gov 3). In this short time, the European Medicine Agency has been able to centralize the agency so all twenty-eight countries are able to work together. As noted earlier, the FDA and European Medicine Agency have similar approval processes but the big difference between the two is that the European Medicine is much slower to approve new medicines. On average, it takes the European Medicine Agency 410 days to get through the three clinical trials while it only takes the FDA half that time (Carroll 1). For people with amyotrophic lateral sclerosis, this can be the difference between life and death.  In a recent study done, researchers sought to compare and contrast how the FDA, European Medicine Agency and Health Canada approved new cancer drugs. In this study, the FDA was by far the fastest to approve the new drugs, but also had the highest number of drugs that needed to be put through the fourth stage of trials to readjust the dosages and ensure their safety (Samuel and Verma 2-3). This shows that although the FDA gets the medicines approved quickly, they are too fast too approve the medicines and don’t take the time to ensure they are safe. 

Former President Barack Obama, on his first day in office, sought to create an unprecedented level of openness in the government (Hamburg and Sharfstein 1). The FDA claims to have embraced this and announced a transparency initiative. This initiative was a start to fixing the problem of transparency at the FDA but was not nearly enough. The FDA still does not disclose whether a drug or device is under development, when an application is withdrawn by a sponsor, whether the agency has placed a hold on clinical studies, whether it agrees with reports published by others about products with pending applications not yet approved by the FDA, and why it does not approve a marketing application (Hamburg and Sharfstein 4). This leaves huge gaps in information that the public needs to know. A perfect example of this is Genervon. The FDA will not release why it has been approved by the European Medicine Agency but has not by the FDA. Additionally, it is unclear whether Genervon is being placed on hold in the approval process or if the company has simply withdrawn the application. For people with incredibly limited time because of a life-threatening disease, this lack of information must be infuriating. By not releasing this information, the FDA is causing more stress in the lives of those affected with diseases who desperately need medicine. When the FDA is not completely transparent about their findings, it leads to a major Public Relations problem because people want to know the full risk before they take medicines and they deserve to have that information.  The FDA needs to be completely transparent with their findings because when they do not release vital information such as this, it leads the public to distrust the FDA. 

Those opposed to changing the 510(k) process would argue that the FDA and all others institutions of similar nature acknowledge that those with illnesses need to be treated quickly and effectively. According to a recent study, the FDA is much faster than other institutions at supplying medicine. The FDA was able to give approvals for new medicines in 184 days. In comparison, it took the EMA almost twice as long to approve the same medicines (Roberts, Allen Sigal 5). The FDA is much more efficient than other agencies. They acknowledge that for many people, even just one day can be the difference between life and death. If the FDA is withholding medicine, there must be a legitimate reason for them doing so. They are looking to keep everyone safe and getting medicine to those in need quickly. From this experiment, it is clear that they do a far better job at getting medicine to those in need than other agencies around the world. Additionally, the FDA defends their current policy of not releasing certain information to the public. Those who are against the FDA being transparent about their findings would defend this by saying that this policy is the best policy because people would be hesitant to take a medicine  

For the FDA to be effective, it must have the perfect balance of speed and safety. It is crucial for the FDA to get medicine out quickly because as sad as it is, it is unclear how much longer somebody has to live but it is also important to ensure that the medicine is safe for use. In many cases, the FDA has to choose whether they are going to approve a drug quickly or hold it longer for further examination. In these situations, the FDA needs to value safety higher than speed. It is true that the extra time taken to approve a new drug can cause some to pass away, but it is more important to fully ensure that it is safe for use. The FDA needs to revise their approval process to make it more stringent because it is far more important to ensure the safety of those taking the drug than the speed at which it is approved. I am not saying that the FDA needs to take an absurd amount of time to approve new drugs, but if it gets down to speed versus safety, safety is more important and instead of using quicker approval methods, the FDA must use stricter and longer approval processes. Additionally, new policy must be implemented for the FDA to be completely transparent because this will help many to understand the approval process and lead to a greater trust between the public and FDA. 

The FDA is a highly-criticized organization but is far more efficient than its counterparts and although one of the approval processes is too expedited, the other two processes appear to be incredibly effective. There are obviously recalls, but nobody is perfect and the FDA is no exception because it is impossible for them to truly see how people react to a new drug with small clinical trials. The main goal of the FDA needs to be to use a tougher approval process to limit the number of recalls to ensure the safety of medicines and Americans. The FDA seems to recognize their current issues and are working towards fixing them. It is critical to ensure the safety of the drug rather than just putting it out on the market and having those taking it experience incredible side effects. It is also important for the FDA to be more transparent in their findings because people need to know exactly what kind of risk is involved in taking a certain type of medicine. Transparency should be an obvious thing but the FDA has not embraced being transparent. They need to take drastic steps in this division to provide the public with all the information needed for them to decide whether to take a drug or not.  The FDA has taken early steps to improve how safe they are but there still is a lot of work to be done. Hopefully the FDA can improve their approval process and be more open to the public because this really is the most effective and safest way for the FDA to go about their business.  
