Participate in Clinical Studies

A clinical study is a research program that tests whether a potential new medicine, treatment, or approach is safe and effective in people. Studies follow strict scientific and ethical guidelines to protect participants while adding to medical knowledge. Every treatment approved after 1962 was once tested in a clinical study.

Studies are how researchers discover better ways to prevent, diagnose, and treat illness. Without patients and health volunteers willing to participate, medical progress would not be possible.

There are several potential benefits of clinical study participation. They include:

• Becoming involved in testing an investigational drug that would not otherwise be available.
• Contributing to medical science, which may help other patients in the future.

In most countries, including the US, clinical research must follow federal regulations intended to protect the welfare, decisions, and rights of all people interested in joining a clinical study. These regulations establish key ethical principles for how clinical studies should be designed and run. They are intended to protect all people who have agreed to participate in a clinical study, or may be thinking about participating.

In addition to these regulations, clinical studies have independent oversight by entities such as an Institutional Review Board (IRB) and the US Food & Drug Administration (FDA). Oversight helps ensure that clinical research is conducted, analyzed and reported in accordance with federal law. You can learn more about patient safety and your rights as a potential participant here.

Studies can be led by different entities, including government agencies, universities, and pharmaceutical or biotechnology companies like Amgen. They are conducted primarily at research sites, which may include hospitals, clinics, or universities. Although some visits or study activities may be completed at home or near a participant's home. Either way, a trained study team monitors your participation throughout the entire study.

The length of a study depends on what medical intervention is being researched or what the study team is attempting to determine. Some studies last a few weeks, while others may continue for several years. The study team will explain the expected timeline before you decide to participate.

Each study has a specific set of criteria — called “eligibility criteria” — that determine who can participate. These criteria are based on factors like age, medical history, and current health status. They are used by the study team during the screening process to determine whether potential participants fit the criteria for a particular study.

Participants may stop participating in a clinical study for many reasons. You may simply change your mind, or you might run into certain challenges:

• Lack of understanding about what was involved in study participation
• Too many study visits or difficulty fitting visits into your schedule
• Too far to travel to the study location
• Lack of improvement or side effects
• Anxiety about participating

Don’t be afraid to ask questions up front and consider carefully whether you would like to participate.

Learn more about patient safety and informed consent.

This is the first stage of testing an investigational medicine in people. Researchers study how the investigational medicine affects the body and look for early signs of safety, side effects, and how well people tolerate it. This phase is generally conducted in healthy volunteers, with the exception of cancer, and in a small group of participants.

Sometimes studies combine Phase 1 and 2 into a single study. This allows researchers to first assess clinical safety and tolerability of the investigational medicine, usually with a small number of participants. The study is then expanded as researchers focus on the impact of the investigational treatment on patients.

The efficacy (how well it works) and safety of the investigational medicine are evaluated in a larger group of patients with the condition that the therapy could treat. This phase also tests a range of doses and regimens (how the study treatment is given) in people with the condition being studied.

Like Phase 1/2 studies, these studies allow researchers to combine testing of an investigational medicine. First, they adjust the dosage and regimens for the investigational medicine, optimizing for the best course of treatment with an acceptable amount of potential side effects. Then they work to confirm the investigational treatment is both safe and effective.

This phase confirms the efficacy (how well it works) and safety of an investigational medicine or treatment. Phase 3 studies are the largest studies performed and are considered the primary standard of evidence used to assess benefit and risk. It is often after this phase, where the data are given to the regulatory agency to review for approval.

Once a medicine is approved and available for use, Phase 4 studies are sometimes used to further observe the medicine’s safety and collect additional information about study treatment benefits and safety.