When I was in graduate school, I took a class in how to design and run a clinical trial. I was so fascinated by the process, that I chose to participate in a trial that was going on at the university (this was before my diagnosis with multiple sclerosis – the trial was an open-label trial of a new birth control pill to look at side effects and acceptability).
I thought I knew exactly what would happen when I signed the informed consent form. I was given so much information that I became overwhelmed. It turns out that the process was different than I expected.
I wish I had asked more questions at the beginning of the trial. It turned out that I was expected to do much more than I anticipated (daily recording of side effects and more frequent blood tests) and there was no mechanism for reporting side effects that did not fall within the form that I was provided. I experienced so many side effects that I could not really discuss with anyone in a meaningful way that I had to drop out of the study. There was not even a mechanism to tell anyone why I was dropping out. I hated dropping out, as this can hurt the quality of the data. I especially was upset that my side effects were not recorded.
I recommend learning what you can about the trial in advance by visiting the Clinical Trials Database. After reading through the posted description and any additional materials that you may obtain from the study staff, make your list of questions. That way, you are ready for your intake visit and can engage in the informed consent process more effectively.
The questions below are adapted from my book, The Multiple Sclerosis Manifesto. These are just to get you started thinking about the different aspects of clinical trials and how they might impact your life. Undoubtedly, you will be adding many of your own questions to this list. There are no "right" or "wrong" answers or particular "red flags" to be looking for – each trial is different and each participant has their own priorities and concerns.
What are the basics of the study design?
- How many people will be participating in this trial?
- Will some people be getting placebo or will the control arm be getting a current therapy?
What is the purpose of the study?
- Is this trial to see if an untried medication is safe?
- Is it a trial of a drug that is already on the market for another use, to see if it is an effective MS therapy?
- Is it comparing one well-known drug to another well-known drug to see which one is better?
- Is it combining two already used drugs to see if they can be more effective together?
- Is it trying out different doses of the same drug to see what the balance of benefits to side effects are at the different doses?
What are the possible risks and benefits from the medication or treatment?
- What are the known side effects of this drug?
- What are the possible long-term risks from this drug?
- What are the possible short-term benefits?
- What are the possible long-term benefits?
What will I have to do if I participate?
- How will the therapy be administered?
- Where will I get it?
- What kinds of tests will be run during the trial and how often will this happen?
- Will the tests hurt or will I have to be in the hospital?
- Will I be able to stay on my regular medications?
- Who will be in charge of my regular medical care?
- Will my care change during the trial?
- How long will my participation in the trial last?
What can I find out?
- Will I be able to find out results of tests that were run on me and get copies of these (like MRI films)?
- Will I be able to find out if I was on treatment or placebo at the end?
- How can I find out the results of the study?
Will I be paid?
- Will I be reimbursed for expenses, including non-medical expenses, such as lodging, meals and transportation?
- Will I be reimbursed for such expenses to cover a caregiver to accompany me, if medically necessary?
Other QuestionsBesides the above questions, you may have additional things you want to know, especially as things are happening in the wider world of MS treatment. These are a couple of things that I have been asked about recently:
- What will happen when the trial ends to my treatment? What if the treatment is working for me but is not covered yet by insurance – will the trial sponsors or manufacturers continue to pay for it? Will I have access to it at all?
- What if I decide to get treated for CCSVI during the trial? Will I get kicked out of the trial?
You have the right to have your questions answered before you start a trial. You also can ask questions that come up during the trial. When I was a trial participant, I should have insisted that the questions that arose during the trial were answered to my satisfaction. I often took an "I don't know" for an answer, even when it was something that did not compromise the data or the study.
You should know, however, that there are some questions that you will not get an answer to. You cannot ask if you will be (or are) on drug or placebo, for instance, if it is a blinded trial, although you can find this out after the trial ends. You cannot ask what others are experiencing until the trial is closed out, the data is analyzed and the results are made public.
Remember, a clinical trial is, by nature, experimental. If the results were known beforehand, there would be no point in conducting a trial. You will usually not know many things about how you will react to the medication (or if you are even receiving drug or placebo in many trials). However, there should not be any surprises involving the protocol that is followed.
While learning about MS clinical trials you may have a few questions and run across some unfamiliar terms. Below is a list to help you further understand MS clinical trials: