Participating in Clinical Research

Clinical trials are a type of research in which new treatments are studied. Through the clinical trial process, new treatments can be tested to find more effective treatments, to increase survival time, and increase quality of life for people with blood cancer.¹

Types of clinical trials

There are different types of trials: observational or interventional. Observational clinical trials, also referred to as epidemiological studies, observe the participants and monitor their health over a set period of time. They may also be retrospective, or looking at past patient data. No trial drugs or treatments are given to participants in an observational trial. These trials help researchers understand how a disease progresses and provides information that can help them determine how best to treat it. Interventional clinical trials test a new drug, therapy, or experimental treatment. Interventional trials are designed to assess the new treatment's safety and effectiveness.²

In addition, medications must go through four different stages of clinical trials:

• Phase I: the researchers test a new drug on a small group of people (10-80) to evaluate its safety, determine a safe dose, and evaluate any potential side effects
• Phase II: a larger group of people (100-300) are given the drug to determine its effectiveness and further evaluate safety
• Phase III: the drug is given to large numbers of people (1,000-3,000) to confirm its effectiveness, evaluate side effects, compare it to existing treatments, and identify any additional information about how to use the drug safely
• Phase IV: additional studies done after the drug is approved by the U.S. Food and Drug Administration (FDA), and is available in the market, to gather information on long-term use³

The challenges of clinical trials

The clinical trial process is long and expensive - it takes many years for a product to go through all the necessary phases, and many potential treatments fail to show effectiveness to move to the next phase. In addition, approximately 20% of cancer trials fail because they do not have enough participants. Regulatory agencies like the FDA require large groups of participants in trials to ensure the safety and effectiveness of therapies before they are brought to market.⁴

Volunteering for clinical trials

Researchers have demonstrated that patients who enroll in clinical trials often fare better than patients who do not participate.⁴ In addition to the potential for receiving the latest treatment innovations, this benefit could be due to the close monitoring of patients in clinical trials by healthcare professionals.

Before taking part in a clinical trial, you will be provided with an informed consent form, which includes important information about the trial, including its purpose, how long it is expected to last, any tests or procedures that are expected, and potential benefits and risks (like side effects). The informed consent form must be signed before you can take part in a research trial, and your signature confirms that you have been given and understand the information contained in it. In addition, informed consent continues throughout the research trial, as new information about the treatment becomes available, and you have the right to leave the trial at any time.⁵

While you may receive benefit for your health condition by participating in a clinical trial, there is also a chance that you will not receive any benefit, and you may be exposed to unplanned risks. Informed consent aims to give you all the information you need to make a decision about participating in the research trial.⁶