There have never been more opportunities to help develop new drugs for cystic fibrosis than there are today. Because of all the promising new research opportunities, as many individuals with CF as possible are needed to participate in clinical trials.
Under-enrollment is one of the biggest challenges for researchers, and it can slow the development of a potential treatment. Because there are only around 30,000 people with CF in the United States, there is a limited pool of people to draw upon for a clinical trial. That is why it is critical that those who are interested and able to participate find an appropriate clinical trial.
Watch this video to hear CF clinicians talk about:
- Why research and clinical trials are important
- Patient safety
- How to learn more about participating in a clinical trial
The Benefits of Participating in a Clinical Trial
Participating in a clinical trial can be a very satisfying and worthwhile experience. People with CF choose to participate in clinical trials for a number of different reasons. Some possible benefits include:
- Taking an active role in managing your own CF care
- Gaining access to new treatments before they are more widely available
- Helping advance our knowledge of CF
- Receiving a treatment that works for you or your child
"I've enjoyed participating in clinical trials basically because I know that they are helping the greater good. ... Participating in a clinical trial knowing that something in the future is going to be a benefit, it's a huge gift, and it's such a blessing for me to be able to do that."
- Linda Bowman in Reflections on Clinical Trials
Watch Dr. David Orenstein, a pediatric pulmonologist and CF doctor at Children's Hospital in Pittsburgh, emphasize the need for people with CF to participate in clinical trials.
What About the Risks?
Although there are many benefits to participating in a clinical trial, it is important to remember there also are risks. Possible risks include:
- Side effects of the medications or treatments being studied
- Unwanted events during the trial that may or may not be related to the study drug
- Failure of a treatment to work
The research team will continuously monitor your or your child's health and safety throughout the trial, whether you are receiving the drug being studied or a placebo.
If the research team notes any worsening of your health during the trial, they will notify your regular CF care team and the study sponsor to determine if it is related to the study drug. You should contact the study team if you notice any worsening of your or your child's health or have any concerns during the clinical trial.
Remember: By signing an informed consent document, you are not signing a contract. You can leave a study at any time. In addition, those who are monitoring the trial for safety -- such as the principal investigator or the study medical monitor -- can pull you from a study if they believe your health is declining.
We call people with CF who participate in clinical trials, and all those who support them, trailblazers. Help us blaze a trail to better treatments and a cure for CF.