Brenda Banwell, MD – The Children’s Hospital of Philadelphia:
When a person is entertaining participation in a clinical trial, there are several important things they need to do. First, they need to understand what the trial is about. Is it a medication or an intervention or a exercise therapy, for example, or is it a counseling and supportive strategy? Second, they need to understand what the inclusion criteria for the trial are. What type of patients can be included? What type of disease activity does the person need to have before the trial? What medications are permitted to have been previously used, or perhaps what medicines are not permitted to be used? And what are the specific characteristics of individuals who are permitted into the study either by age, sex, or other important criteria. Third, it’s important to ask your physician what the trial medication or intervention actually involves. What are the risks, side effects, and potential benefits of the medication or therapy or activity being studied?
Next, it’s important to ask what the study design actually involves. Many medication studies use a placebo-controlled design, which is a study design where one group of patients receive the active new therapy and the other group receive no therapy at all during that particular period of the study. Neither the patient nor their physician will know which group the person is in. This is the most powerful way to prove a new therapy, but it is not an appropriate study designed for every person and each person needs to speak to their physicians about this type of study model.
Other study models might compare two medications, and again in that situation, neither the patient nor their physician would necessarily know whether they were receiving drug A or drug B. In that situation, one needs to know not only the side effects of the new therapy being evaluated, but the ongoing or established risks for the alternate therapy that would be being prescribed.
It’s important for patients to know how long the study is actually going on and at the end of the study whether there is a subsequent extension phase where all individuals will be offered access to the new therapy, assuming that the study showed benefit of that new therapy. The extension phase, duration, and the opportunity to receive access to this new medication is often a particularly important part of the study design, both for the patient and for the science and the people following the new medication, to see how it does in the longterm.
