What is the purpose of clinical trials?
Clinical trials allow researchers to test formally the efficacy and safety of new drugs for various aspects of MS. There are several stages of clinical trials. Drugs tested in MS trials include those that attenuate the course of MS, such as ones that may decrease the number of MS flares, decrease progression of a person’s disability, and (very recently) drugs that may help with repair (remyelination). Some clinical trials also focus on drugs that help alleviate ongoing MS symptoms. These do not slow the course of MS, but may be added to standard of care with preventive MS treatments.
What are the requirements for someone to participate in trials?
The requirements to participate in a clinical trial are set to maximize patient safety, but they also are designed to help patients remain in the trial until completion, so that all data that is expected to be collected can be used for final analysis.
First, clinical trials have to be approved by an Institutional Review Board (IRB) that evaluates the safety and ethics of a given trial. Based on the approval of this IRB, a consent form will be used that describes the study in lay language so possible candidates can decide if they want to participate. This is to help patients understand the various aspects and safety of being in the trial.
Each clinical trial also has its own set of “inclusion” and “exclusion” criteria that define the population for which the specific drug under study is intended to be tested. For example, trials tend not to enroll patients who have severe health conditions other than MS, as they could jeopardize a patient’s participation in a trial and his or her safety.
In addition, investigators running clinical trials often have some rules based on their experience. For example, patients who live far away from the research center or patients who are not compliant with clinic visits and in taking their medications may not be invited to participate.
Why are clinical trials for MS important?
Clinical trials in MS are critical to help identify new drugs to treat patients safely and effectively. Trials are required before a company or investigator can apply for FDA approval. Randomized clinical trials are the standard way to confirm that a drug has a detectable benefit for patients.
Why is it important to understand how the clinical trial/study is controlled?
The control arm of a trial is usually used to compare the safety and efficacy of the drug being tested. The control drug can be one that has already been approved by the FDA, in which case it is called an “active comparator.” Or it is sometimes a placebo, i.e. an inactive substance. At the end of the trial, data collected in the control arm help determine whether the drug being tested is better than and as safe as an approved drug or an inactive substance. This helps the FDA decide whether the tested drug should be approved and under which conditions it should be used once approved for maximizing safety. For instance, researchers may have found that patients with specific pre-existing conditions proved to be at higher risk for side effects in the trial.
Are there any benefits for people who choose to participate in trials?
Participating in a trial is a big commitment, both for patients and investigators. Some patients are enthusiastic about participating in trials as they like novelty and being part of the leading edge of research. Participating in a trial can also be beneficial for some participants since they are under close medical attention and thus can benefit from early detection of other health issues--as well as improved MS management in terms of symptomatic treatment or medical recommendations.
Being closely-monitored helps some patients feel safer, and this can result in improved overall well-being and may slow disease progression. Some patients also can benefit from receiving drugs that would otherwise not be accessible to them (i.e. before they are approved to be on the market for MS).
Finally, some patients decide to participate in studies, even though they know it may not directly benefit them, because they feel it may help advance treatment for the MS community.
What are the risks of participating in trials?
Risks of participating in a trial include a remote possibility of loss of privacy, although during trials, every precaution is taken to avoid this. In addition, there may be variable risks of injury, depending on the drug being tested and its possible side effects. There is also a remote risk of making an MS condition worse during trials testing new agents. This is why safety is carefully monitored, and there is an oversight committee called data safety monitoring board (DSMB) that is in charge of reviewing regularly all side effects reported in the study, as well as MRI changes. Very few MS trials include potentially dangerous procedures that increase risks of injury such as bleeding, etc.
Does participating in clinical trials cost anything?
Participating in a clinical trial should not cost much, if anything. Depending on the trial, some researchers may offer to cover the cost of travel for study visits and parking, but others may not. Also, the cost of all evaluations (blood tests, clinical examination, MRI and other procedures) should be covered by the trial. In some circumstances, evaluations that are recognized to be the standard of care may be charged to participants’ health insurance (i.e. yearly brain MRI, every six-month neurological examination) if the insurance covers them. this is generally discussed with a patient ahead of study participation.
What role should my doctor have if I choose to participate in a trial?
Your primary care physician or primary neurologist should at the minimum know that you are enrolled in a trial so they know what medication you may be exposed to. This will help them to avoid giving you medications that may be poorly tolerated in association with the drug you are receiving in the trial. In addition, your primary doctors may also identify possible side effect of the study drug when they see you in clinic and bring this to the attention of the doctor running the trial. Finally, your primary doctors can also explain to you potential risks and benefits related to participation in a trial.
How do I search for clinical trials that need participants?
There are several ways to identify trials in which you could participate. First, your doctors may know about ongoing trials that may be occurring. If not, they may be able to give you the name of MS centers that run clinical trials. Second, several websites recapitulate trials that are ongoing in the MS world (National MS Society, Clinicaltrial.org, NIH, local MS center websites).
Waubant, Emmanuelle, M.D., PhD. (2014, Jan 29). E-mail interview.
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