Clinical trials are crucial to the research and development of new therapies to address a wide variety of conditions – from relatively common health conditions, including diabetes and heart disease, to rare indications that only affect a very small percentage of the population.
However, many of these potential therapies are slower to reach patients who need them the most, frequently due to challenges in getting patients to enroll in clinical trials. For people with rare conditions, the problem is compounded by the low number of people with the condition, lack of awareness about active studies and potential new treatments, as well as perceived barriers to enrollment– such as costs and travel.
Choosing to enroll in a clinical trial is an important decision with many potential benefits. By participating in a clinical trial for affected individuals, patients are provided access to the latest promising therapies, while contributing to overall research and understanding of the condition.
Each person’s experience in a clinical trial, including those evaluating the potential benefit of a new therapy for a rare condition, is different.
For more information on clinical trials you can visit the FDA website:
Before joining a clinical trial, it is important to learn as much as possible. Discuss your questions and concerns with members of your health care team to determine if the trial is a good option based on your current condition and options available. Be sure you understand:
For more information please visit the FDA website: https://www.fda.gov/ForPatients/ClinicalTrials/ClinicalvsMedical/ucm20041761.htm
Clinical trials, also called clinical research studies, are conducted by clinicians and medical researchers to understand the potential benefits and risks of new, unapproved medical treatments.
Phase 1 and 2 studies are early studies intended to obtain initial data about the safety of the new investigational therapy and its potential effectiveness. If Phase 1 and 2 studies produce positive data, Phase 3 studies may be conducted. Phase 3 studies are larger studies intended to obtain more extensive data about the safety and effectiveness of the treatment.
A Natural History Study (NHS) is a long-term study to gather information about the progression of a condition and its impact on a patient’s quality of life. Individuals may undergo routine testing to determine the progression of their condition but no investigational therapies are administered.
A rare condition, as defined by the U.S. Food and Drug Administration (FDA), is a condition affecting fewer than 200,000 out of the U.S. population of more than 325 million.
All clinical trials have requirements for who can participate in the study, known as inclusion and exclusion criteria. These criteria are based on factors such as age, gender, location, type and severity of condition, medical history, laboratory test results and other health related categories. These criteria are used to identify eligible participants for the clinical trial and to ensure their safety.
Informed consent is the process of understanding the key facts about a clinical trial before deciding if to participate.
At any point of the clinical trial, a participant can leave or withdraw from the study. When withdrawing from the trial, the participant should let the research team know about it, and if they feel comfortable, share the reason for leaving the study.
You are free to leave a clinical trial at any time, even after you have signed an informed consent or received the investigational drug or standard of care. Additionally, the quality of your care received will not be affected by your decision to participate in or withdraw from the study. However, you should always inform the research team of your decision to withdraw from the study to ensure treatment is discontinued safely. The research team may also ask to continue monitoring you for a period of time to assess long-term effects of the treatment.
For more information on clinical trials please visit the FDA website: https://www.fda.gov/ForPatients/ClinicalTrials/default.htm