Risks and Benefits

The potential harms and benefits of a study depend on what sort of study it is, and what sort of treatment or intervention is being evaluated.  Keep in mind that,
  • Not all clinical trials study treatments. Many clinical trials study new ways to detect, diagnose, or learn the extent of disease. Some even look at ways to prevent the disease from happening in the first place. These studies typically have minimal potential harm. 
  • Even among clinical trials that do study treatments, not all of them study drugs. Many clinical trials test other forms of treatment, such as physical therapy techniques, that may have different benefits and harms from a drug trial. 
  • When clinical trials do look at drugs, not all of them study new ones. Even after a drug has been approved for use, doctors sometimes find it works better when given a certain way or when combined with other treatments. It may even work on a different condition. Clinical trials are needed to study these possibilities as well. 
  • Very few clinical trials involve a participant getting only a placebo. A placebo is an inactive ingredient or “sugar pill” used in some types of clinical trials to help make sure that the researchers can tell the difference between the group getting the study treatment and those not getting the study treatment. With the possibility of getting a placebo, people can't tell whether they are getting the treatment being studied or not, which makes the results more likely to be valid. Instead of using a true placebo, most clinical trials compare the new treatment against the current standard treatment, so everyone gets some form of treatment. 
Each clinical trial offers its own opportunities and risks, but most have some things in common. Generally, clinical trials have some of the same potential benefits:
  • You may help others who have the same condition in the future by helping to advance research. 
  • You may have access to treatment that is not otherwise available, which might be safer or work better than current treatment options. 
  • You may increase the total number of treatment options available to you, even if you haven't yet received all of the standard treatments. 
  • You may feel you have more control over your situation and are taking a more active role in your health care. 
  • You may get more attention from your health care team and more careful monitoring of your condition and the possible side effects of treatment. 
  • Some study sponsors may pay for part or all of your medical care and other expenses during the study. This is not true for all clinical trials. Be sure you know who is expected to pay for your care before you enroll in the study. 
  • Some of the possible downsides of being in a study can include the following: 
  • The new treatment may have unknown side effects or other risks, which may or may not be more severe than those from existing treatments. This is especially true of early phase trials. 
  • As with other forms of therapy, the new treatment may not work for you, even if it helps others. 
  • There may be inconveniences such as more frequent testing, plus time and travel commitments. 
  • Sometimes it is emotionally difficult to participate in a clinical trial. 
  • If you take part in a randomized clinical trial, you may not have a choice about which treatment you get. If the study is blinded, you will not know which one you are getting (although this information is available if it is needed for your safety). This will be explained to you before you decide to take part.

Adapted from www.cancer.org
Document adapted from Duchenne Connect written by Holly Peay (Parent Project Muscular Dystrophy)