Clinical trials are research investigations in which volunteers participate to test new treatments or interventions. They can help bridge the gap between scientific discoveries and practical healthcare applications.

The trials typically go through several phases, beginning with a small-scale Phase 1 trial to assess the intervention’s safety and dosage range. Each subsequent trial expands to larger groups of participants over a longer period of time.

The Science

Clinical trials are the main way doctors find out whether a new treatment works in people. They also help researchers learn more about a disease and how to prevent, screen for, diagnose, or treat it. The treatments being studied may be different from what is normally used to treat a condition, like drugs or medical devices. In some cases, the new treatments are compared to an active placebo. The results of a trial are often used to help decide whether the medical product should be approved for use in the United States.

Most clinical studies have strict guidelines about who can participate. These rules are meant to protect participants and make sure that the research is accurate. To join a study, you must agree to a plan for the study that includes details about how it will be conducted, who will be involved, and why each part of the study is important. All trials follow a set of rules called “Good Clinical Practice” that help to keep risks low and safeguard participants’ safety.

Before a drug can be sold to the public, it must pass three phases of clinical testing and be approved by the Food and Drug Administration (FDA). Once a treatment is on the market, researchers continue to monitor its effectiveness and watch for any negative side effects in large groups of people over long periods of time.

In addition to being important for medical breakthroughs, clinical research is a critical component in developing more accurate tests for rare diseases and conditions with few available treatments. Using advanced technologies to improve the way trials are designed and conducted could greatly speed up the development of therapeutic compounds, vaccines, and other medical products for these conditions.

One way to improve the quality of clinical research is to increase the number of people who volunteer to take part in it. In the past, people who wanted to be in a clinical trial had to live near a research site and be willing to travel. That meant that only people from a few geographic areas could participate in trials and that researchers often found it hard to recruit enough people to study a specific disease or condition. Innovative technology can now allow researchers to reach more potential volunteers and make it easier for them to sign up to participate in a trial.

The People

When people participate in clinical trials, they are helping scientists and doctors find better ways to prevent, treat, control, and cure diseases. The information gathered from these studies helps improve medical care for everyone, including future patients. Clinical trials also bring together researchers, doctors, and industry in a collaboration that benefits all.

For some, a medical breakthrough means an easier way to control diabetes, lower cholesterol, or fight cancer. For others, it may mean the chance to receive a life-saving vaccine or new medication that is less risky than current treatments. Many people choose to participate in clinical trials because their existing treatments haven’t worked or they can’t tolerate the side effects of standard treatments.

Before a treatment can be used in clinical trials, it must undergo years of research in the lab and in animals to understand how it works and what side effects might occur. Once the drug or treatment has passed all of these phases, it can be tested in humans.

During the study, people are closely monitored by trained doctors and nurses. Participants are often asked to come to the clinic on a regular basis to have blood and urine samples taken or to answer questions. Some people may be asked to take medication, while other groups will receive the placebo. These groups are chosen by chance, using a computer.

The results of the trial help determine whether the treatment is safe and effective or if more testing is needed. If the treatment is found to be safe and effective, it may go through a final phase before it is able to be sold. The final phase includes larger, more diverse groups of people. It also includes longer-term safety data and collects information on how the treatment affects different parts of the body.

It is important for people to know that every study is carefully reviewed and monitored by an independent group of experts. This group is called a Data and Safety Monitoring Board (DSMB). They ensure that the scientific and ethical standards of Good Clinical Practice are followed and that the people who participate in the study are protected.

The Ethics

Clinical trials are one of the most important steps in testing new treatments to ensure they are safe and effective. The process requires many people from different backgrounds, including researchers at academic medical centers and pharmaceutical companies, doctors who specialize in a particular condition, and Federal offices and agencies that oversee research. All of these groups need to work together to find the right people to participate in a trial and take steps to ensure their safety.

The core ethical question is whether it is acceptable to expose some individuals to research risks in the service of learning more about a specific medical condition or treatment. Proponents of the answer argue that the answer is yes, provided the research satisfies what has come to be known as clinical equipoise. This means that the available clinical evidence does not strongly favor one of the treatments being tested over another, and there are no other medical options for the participants that would be more beneficial than participating in the study.

Critics counter that this view ignores the fact that, even if clinical equipoise does exist, it is not necessarily the case that participation in a particular study will be in the interests of individual research participants. For example, a patient might have a medical condition such as reduced liver function that makes her more vulnerable to harm from certain procedures or drugs than others who might not be affected by the same health issues (Gifford 2007).

Another concern is that the research may not provide enough benefit to offset some patients’ participation risk. The fact is that potential new treatments have to go through a lot of work in laboratories and with animals before they are ready for the next step, which is clinical trials. And once they are completed, it can be years before the treatment becomes widely available.

It is also necessary to ensure that the people who are participating in a trial reflect the populations of patients that will use the medical products once they are approved for commercial distribution. This is why it is essential to have as many different ages, races, and ethnicities as possible in clinical trials.

The Impact

Getting from potential drug to actual treatment involves months, even years, of laboratory studies (including cell and animal models) and clinical trials. Once a new drug makes it to the clinical trial stage, it must pass a multi-phase FDA review and demonstrate safety and efficacy in real-world patients. These clinical trials are critical in the development of innovative medicines and in transforming healthcare.

Choosing to participate in a clinical trial is an important and personal decision that should be made after a careful discussion with your doctor. It is a process that begins with screening, which includes questions and tests to determine whether you are eligible for the study based on your age, sex, health history, family health, and current medications. This may include blood and other lab work, thinking and memory tests, and a physical examination.

All clinical trials have inclusion criteria guidelines about who can volunteer for the study. These guidelines are based on factors such as your age, sex, type and stage of disease, and previous treatment. Some participants will be given a placebo in addition to their normal care, while others will receive the experimental drug. The researchers will analyze the results from all of these volunteers to compare the new treatment with the standard therapy.

Once a medication has passed all phases of the clinical trial process, it is considered a medical breakthrough and can be licensed for use by the FDA or other regulatory agencies in countries around the world. Once a medicine has been licensed, the research team will continue to monitor it in a “real world” setting, to ensure that it performs as expected and to identify any side effects.

The future of clinical trials depends on a paradigm shift that brings routine clinical care and clinical trial research into closer collaboration to generate real-world evidence. This would be achieved by breaking down silos between the fields of clinical trials and medicine and facilitating better access to and participation in clinical trials. This can be done by implementing navigation services and site-agnostic clinical trial matching, by increasing the availability of research funding through mechanisms such as priority reviews, orphan designations, fast-track designations, and breakthrough designations, and by incorporating clinical trial training into medical education. New technologies like digital endpoints (sensor-generated data collected in the context of a patient’s everyday life) can help accelerate the speed of trial execution and improve the external validity of the results.