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Advocate May/June '00:R17

Should I take part in a research study?

Should I take part in a research study?
by Peter Weiden, M.D.

Editor's note: Consumers thinking about taking part in research studies on new medications need a lot of good information before they can make a good decision. There are reasons participation may be right or wrong for a specific person, so knowing and thoroughly understanding what's involved in a clinical trial is extremely important.

For this issue's Partners in Research column, we are posing the basic question all consumers ask if they are invited to join a study: What should I do? The answer is that consumers must ask even more questions and get clear and complete responses from the researchers.

The following suggestions about how to make the final decision-which should not be made too quickly-were put together by Peter Weiden, M.D., a doctor who runs clinical studies, who is a very involved NAMI member, and who currently is the director of the Schizophrenia Research Program for the Department of Psychiatry at the State University of New York Health Science Center, Brooklyn, NY. Dr. Weiden is also an author of Breakthroughs in Antipsychotic Medications: A Guide for Consumers, Families, and Clinicians.

The consumer's question:
I was asked to join a research study testing a new medication for schizophrenia. I'm not happy with the medication I'm on right now, but the idea of research makes me very nervous. What should I do?

The doctor's answer:
There's no right or wrong answer here, but one thing is for sure. There are many new psychiatric medications being developed. To get approval from the FDA, each one must be tested, so there are many research studies out there.

In principle, these studies offer consumers an extra treatment option, which is a good thing because many consumers aren't completely satisfied with the medications available now. Doctors have to find people to take part in these studies, so your joining a research study is a way you can possibly try a new medication. But you need to know that research studies may have some drawbacks. I'll go over these possible drawbacks so you can ask about them when you talk to your doctor about taking part in research.

The following are some of the most commonly asked questions from consumers about participating in research:

Why are there research studies done with new medications?
The most common reason to do a medication study is to show the FDA that the medication is safe and works for a specific condition or illness. If the participants have schizophrenia, that means the medication must work for positive and negative symptoms. To provide that proof, a pharmaceutical company with a new drug sets up large research studies called clinical trials to see how the drug works in real people with real illnesses.

What exactly happens in a clinical trial?
In a clinical trial, the medication being tested is compared to either a placebo (an inactive pill that has no effect) or another medication that is known to work to see what effect it has on the people who take it.

Unlike when a doctor prescribes a medication, the decision about who gets what drug (or a placebo, which is no drug) isn't made by anyone. It's made by a computer, which of course doesn't know anything about who you are. This way to determine who gets what is called random assignment.

You must understand that random assignments mean that you might not get the new treatment you hope to get. Most of the time, neither you nor your doctor will know what you're getting. It's kept secret, and the medication is packaged so you won't be able to figure out your assignment by how it looks. This is called double-blind, which is a term used to describe the fact that both you and your doctor are "blind" to your treatment.

What does this all mean to people taking part in studies?
Most important, you need to know that the main goal of a clinical trial is getting the information needed by the pharmaceutical company or the researcher. Your own treatment is not the main goal of the research study. This fact does not mean you won't get good care. In fact, my experience tells me that many people get better care when they participate in clinical trials than they would otherwise.

Why do doctors (investigators) do clinical trials?
Doctors do clinical trials for two main reasons. One is that they really want to find better medications, or want to learn more about the pros and cons of the medications they study. Therefore, many investigators, or researchers, are experts in the treatment of your condition. When you join a clinical trial, you may get an evaluation from a top specialist in schizophrenia or whatever illness researchers are testing drugs for.

Another reason doctors (investigators) do clinical trials is financial. They are paid to do the research. The investigator usually receives payment from the sponsor of the study if you join the study. This system has its pros and cons. The advantage for you is that you probably won't be charged for your evaluation and treatment. The disadvantage is that you may feel that the investigator is pressuring you to participate for his or her own financial reasons.

Why do people agree to participate in research?
In my experience, the three most common reasons are:

  • to find a medication that works better for the person in the study;
  • to get care that is free of charge from a research staff that is very knowledgeable about the illness the drug is intended to treat; and
  • to help other people who suffer from the same illness by taking part in research that may provide better treatments.

In light of these reasons, let's consider the questions you need to answer before you join a study.

Can I find a better medication?
You should ask the doctor doing the study about what's known about the new medication. A shortcut is to ask which "phase of development" the medication is in. Generally, doctors tend to know a lot more about the medications that are further along in the development process. The key word here is phase-which phase is the medication in? Phase II is pretty early in testing, and phase III means that the medication is pretty far along in development. You'll be more likely to get a medication that works better for you in a phase III study. That doesn't mean you shouldn't enter a phase II study-it simply tells you that if your primary reason for joining is to get a better medication, you've got a better chance with a phase III study than with a phase II study.

Here are some questions about the medication you might want to ask before agreeing to the study:

  • What does the comparison group get? Is it a placebo? Is it a medication I've already tried? What are the chances I'll be in the comparison group?
  • If I respond to the study medication, can I stay on it until FDA approval, or will I have to go off of it once the study ends?
  • If I don't do well on the study medication, will I then have the opportunity to try the new medication?

Can I get better care?
One possible advantage of taking part in a research study is that you'll get free care from clinicians that are experts in your condition. In running my own clinical trials service, I've found that we help many patients simply by doing a careful reassessment of their medical and psychiatric problems. Often these evaluations are even more helpful than the research medication!

Of course, there also may be drawbacks in accepting this care. Sometimes people have to change doctors to join a study, and that can be very hard when you have a good working relationship with your current doctor. And you may face another problem at the end of the study. The research team may refer you to another treatment system, and your treatment is disrupted again. Here are some questions you might want to ask:

  • Have you spoken to my current doctor? Is he or she comfortable with this study?
  • Will I have to change doctors? Will I be able to contact my old doctor during the study? Can I go back to my old doctor at the end of the study?
  • Who will be taking care of me during the study?
  • What happens when the study is over? Will I still get care here, or will I be referred elsewhere?
  • Who will pay for the medications when the study ends?

Will my participation really help others?
People who participate in clinical trials are helping others. Even if a new medication or treatment doesn't work for you, the information researchers learn from your participation always helps others.

Briefly, what must I think about most?
Joining a clinical study isn't for everyone, but it is becoming one of the best ways to get new medications and care from experts. If you do join a research study, you need to keep clearly in mind that the goals of the study may not be the same as your own personal goals. If that's the case, you'll need to review the pros and cons of the study to see if it is worth it to you to take some chances. I know I'm not objective here because I conduct studies, but I believe that people often get excellent care when they join clinical research trials.

I have some advice, though. Ask lots of questions beforehand. Take your time deciding. If you finally decide you will join the study, do your best to stay with the program. It often takes more time than you might think it should to determine for sure if the treatment is good for you. Don't miss out just because you were impatient.

Note: Adapted from an article written by Peter Weiden, M/D., for New York City Voices: A Consumer Journal for Mental Health Advocacy published by Ken Steele.

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