The caronavirus outbreak has caused a great increase in the interest in finding new drugs and new applications of old ones. It has also increased the interest in non-traditional remedies. This diary does not address the latter, only the ones that the FDA may eventually approve. The FDA wants the API or active ingredient, a mechanism of plausible action, and evidence through randomized controlled trials that the drug works. In addition they need to see what indicators that too much drug has been given and what to do about it. There is more but this gives you a pretty good start.
As you do your internet researching of drugs being proposed to treat coronavirus patients, see where the company is in the drug development cycle. Has it been tested in living tissue or is it still in the design stage? Is the testing only in cells or has it been tested on an animal model? Has it been used for a different application and therefore been approved for human use and there is Phase I clinical data? This means that the safety of the drug is known so clinicians know the maximum tolerated dose. How is it being administered (e.g. orally or IV)? Where in the disease cycle is it intervening? Is the drug meant for people who test positive but do not show any symptoms or is it only meant for patients on ventilators? The route of administration gives a clue since IV drugs are not commonly used in outpatient settings. Lastly what is standard of care in the US since information is coming from around the globe? This is important since some studies are simply reaffirming what is done in this country routinely.
One way to learn new applications for a therapeutic is to use it off-label. Most of the above boxes have been checked for the drug and what is being determined is whether or not it might help for a different indication. Physicians may do this out of compassion or even at the request of a patient. For COVID-19, there has been a fair amount of trying drugs that make sense for the patients on ventilators. Once the physicians have had a chance to see what the disease progression looks like they may try drugs that they are familiar with to treat specific symptoms. If the patient improves, then a clinical trial may be proposed and the observation can be studied using a randomized controlled trial design.
One question you might ask is who gathers all of these observations and passes them along to other physicians? Why that would be the CDC! The experts there would be in contact with centers around the globe and document what the disease expression looks like and what has been tried to treat it. This information gives the CDC some of the information they need to prepare the US for the pandemic as well as giving health care centers the knowledge bank they need to prepare their health care practitioners. In the case of COVID-19, the CDC has been MIA for most of its responsibilities. This has left clinicians around the country to contact each other directly and ask for help. This also leads to the cacophony of arguments for a multitude of drugs that may be useful.
This is where the FDA can come in and restore order. The FDA works with companies and investigators to design trials that will show the drug is indeed effective in treating COVID-19 and under what circumstances. That is being done now using randomized controlled trials. When you look at information from the RCT remember to compare what was done vs what is currently being done in the US. For example, US ventilator patients are routinely give antibiotics since the tubes are superhighways for bacterial infections. So a study in Europe that has a study arm without antibiotics would not apply to the US. It would have to have groups on antibiotics with and without the test drug.
So, when you read or hear about a new drug to treat caronavirus patients use the internet and see what you can find out. Search both the trade name and manufacture’s names for the drug. Learn how to use PubMed to find randomized controlled trials for the drug for any indication and what that indication was. Go to manufactures’ websites to see if they have the warning label for that drug. Find out the reasons why that drug can cause a person harm. That alone may help guide you why some therapies are better than others.
The above information should help give you a sense of why it seems to take so long to see if a drug can be useful for COVID-19. People in the pharm industry are highly motivated to find treatments and possible cures. The FDA is highly motivated to make sure the cures actually work and that they are not adding to the damage of the disease. It takes a remarkable amount of discipline by the agency and the test centers to end up with reliable (i.e. reproducible) data.
Have fun and learn more about the stuff we put in our bodies!
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Just as soon as I put this up, MIT Technology Review has to come and spoil all the fun with their article: Which Covid-19 drugs work best? This is not protected by their paywall so enjoy: www.technologyreview.com/… As they point out, all manner of treatments are being studied including vitamins and ones from traditional Chinese medicine. Generally traditional medicines would have to have the active ingredient isolated to be approved in the US. It should make you happy that these studies are going forward without that stipulation.