What research is, and what it isn’t: Research is a controlled, experimental exploration of some new drug, device or other medical intervention. An example of the goal of a research study would be to show that the new intervention is better than current standard of care, or at least as good as the current standard of care but perhaps with fewer side effects or faster recovery time.
Research is not your own physician focused on your individual needs, and thus, research is not therapy or treatment for you in particular. This is true even if it is your personal doctor who is leading the study. As a research participant you are essentially volunteering to be the first person to walk in space or explore new lands. No one can say for sure whether the trip will be worthwhile for you in particular, though it may help others in the future.
Fundamentals: Research studies are also referred to as “clinical trials”. A proposed study goes through four trial phases (or stages) on its way to approval by the Food and Drug Administration (FDA) in order to test safety and efficacy, and to identify side effects. However, there is no guarantee that the new intervention (drug, surgery, etc.) will be approved.
A study may require only one visit by the participant; or the study could last for weeks, months or years. As a study participant you may be in the control group or the intervention group. If you are in the control group you will usually be given the standard of care or the placebo (sham intervention). If you are in the intervention group you will be given the exploratory intervention.
How Does a Study Get Approved? Before a clinical trial ever gets to the recruiting stage there is another process it must go through. Each proposed study must be approved (or not approved) by an Institutional Review Board (IRB).
There are federal regulations spelling out the composition and duties of an Institutional Review Board, but essentially it is a group of people with particular expertise who review every proposed study. Every IRB must also have a non-scientist. That is, an average person who can provide a perspective on the study that may be closer to the perspective of potential participants.
The IRB review includes such questions as: Is the study scientifically valid? Are the target population and recruiting processes appropriate? (For example, if a study is investigating diseases of aging it
wouldn’t be appropriate to recruit participants in their 20s.) Is the study recruiting enough participants to produce statistically valid information? Is the study recruiting more people than it needs for statistical validity, thus putting more people at risk than necessary? Are the potential risks of the study proportionate to the potential benefits?
How do you know what is expected of you as a participant in a clinical trial and how do you decide whether or not to participate? Every study must provide a consent form to be signed by each potential participant. In addition to reviewing the protocol (study description) the IRB also reviews the consent form.
Among other things, the form is required to disclose why the study is being done, what is being asked of participants and why, and what the potential risks and benefits may be. It must disclose whether participants will receive compensation for participating and the fact that participation is completely voluntary.
As a potential study participant you have the right to: ask questions of the study team, discuss the form with family and friends before deciding, and withdraw from the study at any time – though there may be specific procedures associated with withdrawal to protect your safety.
While it is possible that your condition will improve if you are in a study, it is also possible that your condition will not improve. Deciding to participate in a research study requires a degree of altruism - the willingness to be the first one through the door even if it is years before anyone benefits from what you’ve done.
For more information about clinical trials visit: www.clinicaltrials.gov or www.nlm.nih.gov