Clinical trials are studies in which people volunteer to test new drugs or procedures. Doctors use clinical trials to learn whether a new treatment works and is safe for people. These kinds of studies are needed to develop new treatments for serious diseases such as cancer.
Deciding to take part in a clinical trial can be hard if you have cancer. But it is your choice to make, if there is a clinical trial for which you qualify. A lot has changed over the past few decades, and many people want to know as much as possible about all of their options before they make up their mind.
There is always uncertainty when you're thinking about a clinical trial. Part of it is that the doctors in charge of a clinical trial don't know ahead of time how things will turn out. If they did, there would be no need for the study in the first place. Because of this, there's no simple answer to the question, "Should I take part?"
Most people don't pay much attention to clinical trials until they have a serious illness like cancer. Medical breakthroughs (the results of clinical trials) often make the news, but you usually don't hear about clinical trials themselves unless something has gone wrong in one of them. The media is quick to report an instance when a volunteer in a study is harmed. Although it is very rare, people have been harmed, and have even died, while taking part in clinical trials. Reports of these tragic outcomes are important, because they help to expose problems in the system. These problems can then be corrected so that they don't happen again. Because of bad things that happened in the past, there are now laws, requirements, and procedures in place to protect the rights and the health of human volunteers.
What you usually don't hear about in the news are the thousands of people who are helped each year because they decided to take part in a clinical trial. You also aren't likely to hear about the millions who will benefit from others' participation in clinical trials.
There is no right or wrong choice when it comes time to decide on taking part in a clinical trial. The decision is a very personal one and depends on many factors, including the benefits and risks of the study, what the person hopes to achieve by taking part, and other preferences and priorities.
Knowing all you can about clinical trials in general -- and the ones you are looking at in particular -- can help you feel better about your decision. If you do decide to take part, knowing what to look for and what to expect ahead of time can be helpful, too.
This guide will address many basic questions and concerns so that you will be better prepared to discuss clinical trials with your doctor and your family. It should help you decide which questions you need to ask and what the answers may mean for you. But in the end, only you can decide if taking part in a clinical trial is right for you.
One last note: this guide focuses on clinical trials for people who are being treated for cancer. But most of the information here applies to other types as well.
Why do we need clinical trials?
Clinical trials show us what works (and what doesn't) in medicine. They are the best way for doctors to learn what works best in treating diseases such as cancer. Clinical trials are designed to answer 2 important questions:
- Does the new treatment work in humans? Is it better than what's now being used to treat a certain disease? If it's not better, is it at least as good, perhaps while causing fewer side effects? Or does it work in some people who aren't helped by current treatments? In other words, is it a step forward? A treatment that doesn't offer anything new probably isn't worth studying.
- Is the new treatment safe? This must be answered while realizing that no treatment or procedure -- even one already in common use -- is entirely without risk. But do the benefits of the new treatment outweigh the possible risks?
Answering these questions, while exposing as few people as possible to an unknown treatment, often requires several different clinical trials. They are usually grouped into "phases." Clinical trials in each phase are designed to answer certain questions, while trying to make sure the people taking part are safe. Every new treatment is tested in several phases of clinical trials before being considered reasonably safe and effective. These phases are discussed in the section "What are the phases of clinical trials?"
How long have clinical trials been used?
Some doctors and scientists conducted what would now be thought of as clinical trials as far back as the late 1700s, but clinical trials were not used widely until the middle of the 20th century. Up until that time, doctors relied on their own experience in similar cases and on the teachings of those who came before them. Progress was slow, and very few medicines could even be tested.
With the discovery of the first antibiotics and other drugs, doctors needed a reliable way to tell what worked from what didn't. They also needed ways to find out which of the countless remedies available at that time were safe for people to use. So they came up with ways to test and compare treatments in certain groups of people. The results of these early clinical trials proved to be more useful than relying on whether or not something worked for one person or a few people.
In the United States, the Food and Drug Administration (FDA) must approve new drugs and medical devices (but not dietary supplements) before they can be advertised or sold to the public. The FDA began overseeing the safety of new treatments in the late 1930s, but didn't require proof that they actually worked until the early 1960s. Today, new drugs and medical devices must go through several phases of clinical trials (discussed later) before they can be approved for use.
Based on what we have learned about cancer in recent years, researchers can now develop new treatments in a more logical way and much faster than in the past. But it's still a hard process that takes a long time.
New treatments have to pass many tests before they get to you
Clinical trials are only a small part of the research that goes into developing a new treatment. Drugs of the future, for example, first have to be discovered or created, purified, described, and tested in labs (in cell and animal studies) before ever reaching human clinical trials. About 1,000 potential drugs are tested before just one reaches the point of being tested in a clinical trial. On average, a new cancer drug has at least 6 years of research behind it before it even makes it to clinical trials. But the major holdup in making new cancer drugs available is how long it takes to complete clinical trials themselves. It takes an average of about 8 years from the time a cancer drug enters clinical trials until it is approved.
Why so long? To be sure it is safe and effective, researchers look at each new treatment in several different studies. Only certain people are eligible to take part in each clinical trial. And cancer clinical trials take years to complete. It takes months, if not years, to see if a cancer treatment works in any one person. And figuring out if a drug really improves survival can take a very long time.
The biggest barrier to completing studies is that not enough people take part in them. Fewer than 5% of adults (less than 1 in 20) with cancer will take part in a clinical trial. According to the Pharmaceutical Research and Manufacturers of America (PhRMA), more than 800 cancer medicines were being tested in clinical trials in 2009. Not all of them will prove to be useful, but those that are may be delayed in getting approved because so few adults volunteer.
The main reason people give for not taking part in a clinical trial is that they didn't know the studies were an option for them. But there are many other reasons. Some people may want to take part but aren't eligible. Some people are uncomfortable with the idea of being a volunteer in a study. Others worry that they won't be treated fairly or could be harmed by an unproven treatment. All of these are valid concerns. We have addressed them in more detail in the section, "Should I think about taking part in a clinical trial?"
One of the most important points that must be decided before a clinical trial can be done is if it is ethical to ask patients to volunteer for the experimental treatment. Has the study been designed, as much as possible, to make sure the people involved will be safe? Will the volunteers get a treatment that is at least as good as, and maybe even better than, what they would get if they did not volunteer for the study? Scientific panels are set up to review and approve all clinical trials to make sure questions like these are answered before the researchers are allowed to sign-up patients. - Does the new treatment work in humans? Is it better than what's now being used to treat a certain disease? If it's not better, is it at least as good, perhaps while causing fewer side effects? Or does it work in some people who aren't helped by current treatments? In other words, is it a step forward? A treatment that doesn't offer anything new probably isn't worth studying.
- Is the new treatment safe? This must be answered while realizing that no treatment or procedure -- even one already in common use -- is entirely without risk. But do the benefits of the new treatment outweigh the possible risks?
Answering these questions, while exposing as few people as possible to an unknown treatment, often requires several different clinical trials. They are usually grouped into "phases." Clinical trials in each phase are designed to answer certain questions, while trying to make sure the people taking part are safe. Every new treatment is tested in several phases of clinical trials before being considered reasonably safe and effective. These phases are discussed in the section "What are the phases of clinical trials?"
How long have clinical trials been used?
Some doctors and scientists conducted what would now be thought of as clinical trials as far back as the late 1700s, but clinical trials were not used widely until the middle of the 20th century. Up until that time, doctors relied on their own experience in similar cases and on the teachings of those who came before them. Progress was slow, and very few medicines could even be tested.
With the discovery of the first antibiotics and other drugs, doctors needed a reliable way to tell what worked from what didn't. They also needed ways to find out which of the countless remedies available at that time were safe for people to use. So they came up with ways to test and compare treatments in certain groups of people. The results of these early clinical trials proved to be more useful than relying on whether or not something worked for one person or a few people.
In the United States, the Food and Drug Administration (FDA) must approve new drugs and medical devices (but not dietary supplements) before they can be advertised or sold to the public. The FDA began overseeing the safety of new treatments in the late 1930s, but didn't require proof that they actually worked until the early 1960s. Today, new drugs and medical devices must go through several phases of clinical trials (discussed later) before they can be approved for use.
Based on what we have learned about cancer in recent years, researchers can now develop new treatments in a more logical way and much faster than in the past. But it's still a hard process that takes a long time.
New treatments have to pass many tests before they get to you
Clinical trials are only a small part of the research that goes into developing a new treatment. Drugs of the future, for example, first have to be discovered or created, purified, described, and tested in labs (in cell and animal studies) before ever reaching human clinical trials. About 1,000 potential drugs are tested before just one reaches the point of being tested in a clinical trial. On average, a new cancer drug has at least 6 years of research behind it before it even makes it to clinical trials. But the major holdup in making new cancer drugs available is how long it takes to complete clinical trials themselves. It takes an average of about 8 years from the time a cancer drug enters clinical trials until it is approved.
Why so long? To be sure it is safe and effective, researchers look at each new treatment in several different studies. Only certain people are eligible to take part in each clinical trial. And cancer clinical trials take years to complete. It takes months, if not years, to see if a cancer treatment works in any one person. And figuring out if a drug really improves survival can take a very long time.
The biggest barrier to completing studies is that not enough people take part in them. Fewer than 5% of adults (less than 1 in 20) with cancer will take part in a clinical trial. According to the Pharmaceutical Research and Manufacturers of America (PhRMA), more than 800 cancer medicines were being tested in clinical trials in 2009. Not all of them will prove to be useful, but those that are may be delayed in getting approved because so few adults volunteer.
The main reason people give for not taking part in a clinical trial is that they didn't know the studies were an option for them. But there are many other reasons. Some people may want to take part but aren't eligible. Some people are uncomfortable with the idea of being a volunteer in a study. Others worry that they won't be treated fairly or could be harmed by an unproven treatment. All of these are valid concerns. We have addressed them in more detail in the section, "Should I think about taking part in a clinical trial?"
One of the most important points that must be decided before a clinical trial can be done is if it is ethical to ask patients to volunteer for the experimental treatment. Has the study been designed, as much as possible, to make sure the people involved will be safe? Will the volunteers get a treatment that is at least as good as, and maybe even better than, what they would get if they did not volunteer for the study? Scientific panels are set up to review and approve all clinical trials to make sure questions like these are answered before the researchers are allowed to sign-up patients.
by cancer.org, 10/3/10
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