We are pleased to present the first in a three-part series on clinical trials sponsored by Vertex Pharmaceuticals Incorporated. This article is written by Jacqueline (Jacquie) Curtiss, Vice President, Global Clinical Operations, Vertex Pharmaceuticals Incorporated.
As the Vice President of Vertex Pharmaceutical’s Global Clinical Operations team, I work every day with my colleagues to help advance over 50 active clinical trials at over 1,100 study sites across 19 countries. I recognize that while we spend every day trying to advance clinical trials so we can potentially bring transformative medicines and therapies to patients, there are many – maybe even you – who are not familiar with clinical trials and why they are important, so today I’d like to share some information on this topic.
Companies like Vertex conduct clinical research, or clinical trials, to advance medical knowledge. We conduct this research to find better ways to prevent, treat, and cure disease in people. The clinical trials conducted by pharmaceutical and biotech companies are carefully designed and regulated. Patients volunteer to participate in these trials to test the efficacy and safety of new medical interventions. Patients make a very personal decision when they opt to participate in a clinical trial, and there are regulations and guidelines in place to protect participants from unreasonable risks. Prior to joining a clinical trial, patients are provided an informed consent document, which describes their rights as a participant and the details about the clinical study, including potential benefits and risks. By signing the informed consent, the patient acknowledges they understand that the trial is research and that they may leave the study at any time for any reason. When a patient chooses to participate in a clinical trial, their participation is kept confidential. Personal identifying information and medical information of clinical trial participants is known only to the individual patient and researchers conducting the study.
Clinical trials are conducted in a series of steps, also known as “phases”. Each phase has a different purpose and helps researchers answer different questions.
- Phase I trials: Researchers test a drug or treatment in a small group of people for the first time; typically 20-80 people, but for cell and gene therapies, the groups during Phase 1 can be even smaller. The purpose is to study the drug or treatment to learn about safety and identify side effects.
- Phase II trials: The new drug or treatment is given to a larger group of people (100-300) to determine its effectiveness and to further study its safety.
- Phase III trials: The new drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it with standard or similar treatments, and collect information that will allow the new drug or treatment to be used safely if it is approved by the FDA.
- Phase IV trials: After a drug is approved by the FDA and made available to the public, researchers track its safety in the general population, seeking more information about a drug or treatment’s benefits, and real-world use.
The National Institutes of Health (NIH) has a helpful resource center for people who want to learn more about clinical trials. Additionally, if you are someone living with type 1 diabetes and are interested in learning more about clinical trials or locating a trial that might be right for you, JDRF has a clinical trial matching tool that helps match individuals with trials.