Background

Investigational medications, also called investigational/experimental drugs, are drugs that are being tested in a study to see if a person's condition improves while taking the drug. Patients can join clinical trials to find if an investigational drug works to treat their disease or condition. Clinical trials are used to prove that drugs are safe and effective, how drugs can be used to treat a specific disease, the dosage of the disease that should be used, and the potential benefits and risks of taking a drug. 

Why are studies conducted?

Drugs are complicated! No matter how much we know about them, we really can't be sure that they have a chance of being effective unless we study them. Nor can we know about safety problems without study. Expert researchers can certainly make educated guesses, such as whether this drug might likely help to relieve a particular symptom. But his is just the jumping off point for doing a study.

There are regulations that require certain kinds of studies. While it is easy to complain about the ways these regulations work, the basic idea is sound: drugs should not be allowed on the market until there is evidence that they are safe and effective. But there are some subtle issues to answering those questions!

Is a drug safe? This isn't actually a question that can be answered with a yes or a no. What we really need to know are answers to a number of questions. What negative effects can come from using this drug? How dangerous are they? At what dosage do they occur? How do these risks compare with alternative treatments, including doing nothing?

Is a drug effective? This is also a bit more complicated than it seems. Effective at doing what? How good do its effects have to be to call it effective? Is it more effective than alternative treatments, including doing nothing?

In the end, what we really need to know is whether the potential benefits of the drug are great enough to outweigh the risks.

How are studies conducted?

To answer any of the questions mentioned, a drug being studied has to be compared with other drugs or treatments, and the study has to be designed with specific endpoints in mind. To see why, imagine that we give a new drug to 100 HCM patients. After a year, 97 of them have not had any episodes of atrial fibrillation. Is this drug effective? Safe? We actually can't tell from this information! Think of some of the reasons we can't be sure what this means:

• We don't know whether the drug was intended to treat afib, or something else.
• We don't know if 97 successes and 3 failures for the drug is better than another drug that's already available. Even worse, we don't know if this is better than doing nothing.
• Is 100 patients enough to study for this purpose? Maybe, and maybe not.
• Is a year long enough to study it? Ditto!

 

The bottom line is that studies have to be designed in advance, with a stated goal. There must be large enough samples (and long enough times in treatment). Any study has to involve useful comparisons with other treatments. And all of this must be stated in advance, in writing.

These requirements mean that it takes time and effort to study drugs. There is no way around it. If we are going to have drugs that have a good chance of being helpful to us and a small chance of being harmful this process has to be carried out!!

Should I participate in a study?

We would like to just say "yes," but that is not really good advice. A more serious answer requires some thought on your part. And this is a decision you should make with the advice of your doctors!

When deciding if an investigational drug is the right option for you, consider:

• What is the drug being studied for?
• Is the site of the clinical trial convenient to you?
• How much is known about the drug?
• Is the investigational drug likely to work better for you than an approved treatment?
• What scientific evidence is already available to show the effectiveness of this drug?
• What are the potential risks of the drug?
• Have you already tried other approved treatments for your condition?

How do I find a study to participate in?

Sometimes doctors suggest that some of their patients participate in a study. For example, if you are not responding well to a treatment and they know of a study that might be appropriate, they may suggest that you participate.

 The HCMA sometimes works with investigators to publicize a drug trial, so you may see an announcement about joining the trial from us. 

For those in the United States, visit https://clinicaltrials.gov to find trials.

I volunteered. What happens now?

The researchers are always glad that you volunteered. But they may not always be able to enroll you in the trial because you may not meet their criteria for being included. The criteria can include:

• Being a part of a certain age group
• Medical history and current health status
• Having a certain type/stage of disease
• Having received/not received a certain therapy/treatment in the past.

They may already have enrolled enough total patients or enough at the center for which you volunteered. There are many good reasons for them to say "thanks, but not at this time."

 

Expanded access to experimental drugs and devices

Sometimes people who are not eligible to participate in a clinical trial may be able to get experimental drugs or devices through what is called expanded access (also called compassionate use). For this kind of access in the United States, you can get information from the FDA.