What it could mean for those battling cancer
Kathryn E. Vinson, MS, CCRC
As many of us are aware, on May 30th, 2018, U.S. President Donald Trump signed the “Right to Try” bill. The goal of this new law is to enable terminally ill patients to bypass the FDA and obtain potentially lifesaving treatments that are still undergoing clinical trials. While this sounds like a fantastic idea, I needed more information to develop an educated opinion on this legislation. Armed with the text of the bill, I was able to decipher some of the legal speak and get a better understanding of what the law does and doesn’t do. Surprisingly, this bill is pretty straight forward, to the point, and easy to read.
What is Compassionate Use?
A minute ago, I said that this bill allows terminally ill patients to bypass the FDA. What does that have to do with it? Well, the FDA has had Compassionate use or Expanded access programs for years. What exactly are these? This is a program already in effect with the FDA that allowed seriously ill patients possible access to treatments that were in clinical trials; however, this program required that the FDA agrees with the request, as well as the physician and the drug manufacturer. The Right to Try bill essentially takes the FDA requirements out of the equation.
Who is eligible?
People that have been diagnosed with a life-threatening disease or condition, and who have exhausted standard treatment options and are not eligible for a current clinical trial, are able to participate in these programs. Okay, so how do you know if you can participate in a trial? Click on over to our Clinical Trials page. On this page you will find instructions on how to get on a list of people wanting to participate in a trial, or you will see a screenshot of clinicaltrials.gov, where you can search for trials yourself. It is vitally important to discuss these trials and the study drug with your physician.
If you remember from our discussion of the clinical trial process in What’s the Hold-Up, clinical trials are designed with a very strict set of criteria called inclusion/exclusion criteria. This is the set of rules that clinical researchers have to follow to enroll a volunteer into a trial. In order to qualify for most late phase clinical trials, patients must have the condition for which the study drug is being targeted, but otherwise healthy. Why is this so important? Well, when the data from the trial comes back to the statisticians, they need be able to make a determination if changes that they see – be it on bloodwork, x-rays or other imaging, symptoms, or myriad other indicators – are a result of the compound that is being tested. If there are a lot of other factors in the picture, such as extra medications or other disease processes, it clouds that picture and makes it difficult to define causality.
I think I’m eligible – what next?
So, let’s say that you or a loved one have exhausted the standard treatment options that are on the market, and after a discussion with your doctor, you have found out that you don’t qualify for any current clinical trials. You do some more research and have found that Drug XYZ has shown promising results in the disease that you are battling. After discussing this with your doctor, he/she agrees that this might help you. According to the bill here is what has to happen:
- Your doctor must be in good standing with licensing boards and will not be getting paid for recommending you to this investigational compound.
- The investigational drug that you will be trying has to have passed through Phase I trials.
The manufacturer of the drug or device also has to agree to your request.
Will this be free of charge?
Maybe. Maybe not. The FDA issued guidance to manufacturers in 2009 about how to go about charging participants in clinical trials or expanded access use for investigational drugs and devices, if they choose to do so. In most clinical trials, the compound or device is being provided free of charge to the patient. Why is that? Well, in a clinical trial, the researchers are able to get a clean set of data that can be analyzed for the effects of the drug or device being studied; thus, while you may be receiving the benefit of a new compound, they are receiving the benefit of data that will help them know if it can be marketed. According to the FDA, some manufacturers provide expanded access free of charge, while others do not. You should be aware that insurance companies do not typically pay for treatments that have not been approved by the FDA.
But won’t they get data from Right to Try use?
Yes and no. As we talked about above, the data that will be obtained from Right to Try use will not be framed by those heavily controlled studies, so the data cannot be analyzed in the same manner. Now then, I’m about to tell you something that will probably have you saying – are you crazy! I am pleased with the fact that the bill does not require the FDA to consider data collected in Right to Try use to negatively impact the ultimate approval of a compound or device.
You think I’m crazy, right? I mentioned this to some family members this weekend and they thought I had lost my mind as well. Let me explain. In the conduct of clinical trials, we don’t use the term “side effect” we talk about adverse events (AEs), then the investigator decides if the adverse event was likely or not likely to have been caused by the investigational product. When you start getting to really bad stuff, such as stroke, heart attacks, and death, these are termed serious adverse events, or SAEs. SAEs have to be reported within 7 calendar days to the study sponsor and to regulatory bodies. Events such as these can understandably slow or even shut down clinical trials.
Remember that in order to qualify for use under “Right to Try”, the patient must be certified by their physician as having a terminal disease and has tried all standard treatments. This use is, for lack of a better term, a Hail Mary pass. Back to our example with Drug XYZ. If a patient gains access to XYZ, but succumbs to the disease despite the treatment – can we know that it was XYZ, or was it the advanced stage of that disease? As I mentioned above – we can’t determine causality.
When will we know more?
Just like with other laws, the agencies in charge of the area (in this case the FDA and Health and Human Services) have to write rules about how the law will be enforced – guidelines for applications, etc. So far, we haven’t seen that timeline for the rules to be published.
You should also know that 38 states in the US already have Right to Try legislation in force; thus, you may already have legal backing to go directly to manufacturers if you have exhausted standard treatment options. Please, always discuss these possibilities with your treatment team to pursue the path that is right for you.