Ever wonder how your doctors know what drugs to use and how often you should take them? Well, they don’t just guess and hope for the best! Your treatment protocols have been carefully designed based on past clinical trials.
What is a Clinical Trial and why should I care?
A clinical trial is a research study to determine the most effective treatment for a particular disease. Before a new treatment is made available to the public, it must be tested to be sure it is safe and effective. Your medical team might talk with you about participating in a clinical trial and you need to know what it is and what it might mean to you .
Types of Trials
Any new treatment must successfully complete three phases of trials before the Food and Drug Administration (FDA) approves it for general use.
Phase I Trials
Help determine the best way to give a new treatment (by mouth, injection or IV drip, for instance) and the most appropriate dosage. These trials also find out if there are any potentially harmful side effects. Usually only a small number of patients participate in this stage. Phase I studies basically ask: “ Is this treatment safe?”
Phase II Trials
Evaluate whether the new treatment actually has a positive effect against a particular disease. Usually fewer than 100 participants participate in Phase II trials. If at least 20 percent of the patients get better with the treatment, the new therapy goes through more testing. Phase II studies ask: “Is this treatment safe and effective?
Phase III trials
Compare the new treatment (which has already gone through Phases I and II) to the best existing treatment. Many patients, from a few hundred to several thousand, receive either the new treatment or the best existing treatment to see which one works better. Phase III studies ask: “Is this new treatment better than what is currently being used?”
Understanding how a Phase III trial works can be a little tricky:
- Each treatment comparison is referred to as an “arm”. Example : Let’s say your particular trial involves adding a new drug to an already-proven combination of drugs to see if the new combination is more effective . There would be two arms of the study: one arm using the standard drugs as treatment, and the other arm using the new drugs. Some clinical trials have multiple arms.
- Each patient who participates in a trial is randomly picked to receive one treatment or the other. This process assures that each patient has a fair and equal chance of being assigned to any of the treatment arms. This is called randomization.
- It’s important to know that every patient in a Phase III trial receives either the standard treatment for a specific cancer or the new treatment being investigated. If you are participating in a Phase III trial, you don’t have to worry about getting appropriate treatment.
- After the results of the study are analyzed, the researchers will determine if the new therapy is beneficial. At this point, the FDA will approve the drugs for general use.
Who is involved in a clinical trial?
In many clinical trials, the main researcher (PI or principle investigator) is a doctor, but nurses, psychologists, social workers, dietitians, and therapists conduct clinical trials in their area of specialty. All trials testing new drugs include one or more licensed physician who order the medications and laboratory tests. Many other people including nurses, lab and x-ray technicians, research coordinators, nutritionists, physical therapists and statisticians are vital members of the research team. In other words, if you’re participating in a trial, be prepared for a lot of people to be asking you questions!
What else should I know?
Like many decisions in our lives, there are two sides to consider. Before agreeing to participate in a clinical trial, you need to understand all the potential benefits and risks:
Clinical trials offer access to new approaches that might not be available otherwise. The National Institutes of Health (NIH), which fund many clinical trials, offer these benefits:
- The new approach may be more effective or safer than the standard approach.
- Participants receive regular and careful medical attention from a research team that includes doctors and other health professionals.
- Participants may be the first to receive the new method under study.
- Results from the study may help others in the future.
- Some clinical trials provide all treatment and follow-up care at no cost to the family.
You should understand that clinical trials are still experiments that involve risks. The NIH urges you to consider these possible risks before joining a clinical trial:
- New drugs or procedures under study are not always better – and could be worse – than the standard care to which they are being compared.
- New treatments may have unexpected side effects which may be permanent.
- Participants in randomized trials can not choose the approach they receive and may worry that they are not receiving the best treatment.
- Participants may be required to make more visits and undergo more procedures than they would if not in the clinical trial.
- Insurance may not cover all costs in a study. Your parents need to carefully explore this issue prior to signing the consent form ( see informed consent ).
After reading about the possible risks, you might feel nervous about saying “yes” to a clinical trial. That’s understandable! Keep reading and maybe you will feel better. By federal law, researchers must take several steps to make clinical trials as safe as possible:
- Many pre-clinical studies are conducted before the clinical trial is begun . In other words, researchers have done their best to “ get all the bugs out .”
- When a patient is “on study” (research-speak for being in a clinical trial!) , your medical team will use a strict protocol which must be followed exactly. This is a good thing because your case will get lots of attention with less chance of things falling through the cracks! Now, don’t you feel special?
- Each hospital has an Institutional Review Board (IRB), a group of at least five experts, and selected non-medical community members, who evaluates the merits of a trial. They ensure that the clinical trial poses no unnecessary or inappropriate risks.
Do I get a say in this?
Most definitely yes! Here’s how it goes:
A very important aspect of clinical trials is something called “ Informed Consent ” . You will get all the information you need to make an informed decision about participating in a trial.
- If you are under 18 years old :
You are not old enough to legally give your consent for a clinical trial, so the responsibility will fall to your parents. hey will be given an Informed Consent Document (link to medical dictionary) to read and sign that describes everything that will happen in the study. It’s really important that your parents completely understand everything that’s written in this document because they will be acting as your advocate – making sure that you are getting the very best care. They should keep a copy of the Informed Consent document in case they have questions later.
- Even though you can’t legally consent to being part of a trial, you still have many important rights and responsibilities. Your medical team will ask for your “ Assent ” – an informed desire to participate in the trial . Remember – participating in a clinical trial is completely voluntary and your parents and medical team need to respect your opinion.
- You should join a clinical trial only when you understand it fully. If parts of the trial are complicated or unclear, ask your doctors and nurses to explain until you completely understand. Together with your parents and your medical team, make the decision that offers the best chance to cure your disease.
- Of course, no one has a crystal ball that will accurately foretell the future! All you can do is make the best decision possible and trust that your team will always be working in your best interest.
Some Questions to Ask
Before joining a clinical trial, you should feel comfortable and fully informed. You should know the answers to all of these questions before you agree to participate.
It’s often tough to remember the answers to all these questions . Idea: Print out this page, and ask your mom or dad to take notes when you talk with your doc. You can also bring a tape recorder so you can review the answers afterward.
- What are the researchers trying to learn in this study?
- What do researchers already know about the different experimental methods in this study? Have they been tested before? Are they already being used?
- What exactly will they do during this study?
- Who will be in charge of my care during the study?
- What are the differences between what I would get in this study and my current care? Are there different side effects? What are the risks and benefits?
- How will this trial affect my daily life?
- How long will this study last?
- Who will pay for the various parts of the study?
- Who will pay for my medical care if I am injured during the study?
- How will I know if the study was successful? Will I get the results?
Clinical trials are important for at least three reasons:
- You may be helped by the treatment you receive. You will have access to the latest drugs, procedures and other types of treatment. Although there is no guarantee that a new treatment will be effective, many trial participants conclude that the possible benefits outweigh the risks. If a new treatment is effective, patients in trials are among the first to receive it.
- Clinical trials contribute to the overall knowledge and progress against cancer. Many of today’s standard treatments for cancer began in clinical trials.
- Teens have not participated in clinical trials as much as younger kids or older adults. With more research into the types of cancer that teens get, a cure may be found. Isn’t this a pretty good reason?
Remember, no one else can decide whether participation in a clinical trial is right for you. Doctors, nurses, other patients and family members can only advise – the ultimate decision is up to you.
There are many helpful websites that can give you more information. These are just a few that may be helpful: