When COVID-19 first emerged, the recommended treatment was supportive care, which means treating the symptoms while the disease runs its course. Though this remains the approved therapy today, many alternative treatment options have been discussed in the news. This can be incredibly confusing for anyone looking to protect themselves.
First of all, let’s define the term “off-label.”
A drug gets approved by the Food and Drug Administration (FDA) based on randomized, controlled clinical trials to be safe and effective to treat a specific disease. That disease is named in the FDA’s product information pamphlet for that drug and the use is considered part of the “label” for that drug’s use. A physician can use drugs that have an approved indication for one disease to treat another disease that is not stated in the FDA’s “label.” This type of use is considered “off-label” because the FDA did not say the drug was studied specifically for that other disease or indication.
The American Lung Association’s Chief Medical Officer, Dr. Albert Rizzo, helped us understand more about some of the “off-label” treatments available for COVID-19.
Q: Why is supportive care still recommended even though many people have died of COVID-19?
A: Most individuals develop mild to moderate COVID-19. Though symptoms vary from person to person here’s a snapshot of what we’ve seen:
|Asymptomatic||Mild disease||Moderate disease||Severe disease|
|Have no symptoms||Low fever||Fever and chills||Difficulty breathing|
|Dry cough||Deep cough||Chest pain|
|Fatigue||Fatigue and body aches||Confusion|
|May spread the disease to others||May spread the disease to others||May spread the disease to others||May spread the disease to others|
For most people, these mild to moderate symptoms can be treated at home with supportive care. That shouldn’t be confused as not being treated, it just means we don’t have a specific targeted medication that makes the disease go away. Resting, staying hydrated, etc. is the best course of action in these cases. A smaller percentage of individuals develop the severe disease and will need to be hospitalized. For these individuals, they may use supportive care AND additional investigational, off-label, unproven therapies to support vital organ functions.
Q: What do you mean investigational, off-label, unproven therapies?
A: Because COVID-19 is a new disease, we are still determining what treatment works best for most people. While we are still learning about COVID-19, healthcare providers have a lot of experience treating other diseases that have some similarities to the disease processes seen in COVID-19 patients. Because a percentage of patients have life-threatening illness, the medical community has launched a worldwide collaborative effort to share therapies that have not been fully tested in COVID-19 cases but offer some potential benefit based on how the drug has worked in other diseases. Some of these therapies are given as part of clinical trials where there is very closely monitored benefits and side effects such that they may ultimately be proven effective for therapy. But many patients receiving these therapies are also receiving other types of treatment and it may be hard to determine which drug is giving the best effect or it may be that the drugs are working better together.
It is hard in this situation to really set up a control group as defined in most clinical trials for drugs being put through the process of meeting FDA approval for a specific treatment. These patients are given multiple drugs in an effort to save lives. This is also why the FDA has used its Emergency Use Access designation at times to allow some drug availability even though a true randomized double-blind controlled trial has not been done.
It’s a lot to keep up with, but there is an amazing amount of information sharing across the world as providers try out different options and share their results with fellow providers. It is vitally important to all that if there are any negative side effects of treatments that we stop using them.
Q: Can you give some examples of investigational, off-label, unproven therapies?
A: You’ve likely heard some about these in the media. First, we are analyzing antibodies present in plasma, the liquid portion of blood. For individuals who recovered from the infection, their plasma now contains COVID-19 antibodies that helped their immune system fight off the virus. It’s possible that plasma with these antibodies might help other people fight COVID-19 as well. The evidence is not clear-cut, but some patients seem to benefit.
Second, we are looking at antiviral medications. In general, antivirals work by stopping the virus from spreading between cells within your body when you have already been infected. Current research recommends further study of one antiviral, remdesivir, in certain patients who are sick with COVID-19 and against using other antiviral medications, such as hydroxychloroquine. But remember that there are multiple ways to kill a virus and using drugs in combination may be the advised route. Again, there is no clinical trial to guide us here, so this is unproven, off-label, investigational but apparently helpful to an extent that ongoing trials are needed.
Next, there is continued interest in anti-inflammatory medications such as tocilizumab or other steroids because COVID-19 is causing a severe immune response in some individuals. It is thought these treatments may be beneficial in some circumstances and is continuing to be studied.
Q: What will it take for one of these therapies to become an approved therapy?
A: Throughout the history of medicine, physicians have been studying treatments to see if they are effective and safe. We use off-label therapies in a variety of circumstances, such as when there are no approved treatments yet for a new disease like COVID-19.
Not every person responds to the same treatment in the same way so it important to observe any treatment in a large number of individuals before making a recommendation to use it as standard care of treatment. Approved treatments have completed extensive testing and have vast scientific proof that they are safe and effective.
Until these benchmarks are met, there is no specific treatment for COVID-19 other than supportive care for the mildly affected and for those hospitalized with critical illness, the doctors on the front-line are using their best judgment to institute therapies at the appropriate time in the course of the disease to give benefit based on the information sharing that has been going on worldwide.
There is no vaccine to prevent COVID-19. The best way to avoid illness is to avoid being exposed to the virus.
Blog last updated: September 28, 2020