Patient Guide to Clinical Trials

The development of a clinical trial often begins in the laboratory. Researchers first test new treatments or procedures in lab settings and in animals to evaluate their potential. If the results are promising, the treatment may advance to clinical trials involving human volunteers. As the treatment progresses through a series of defined phases, researchers gather increasing amounts of information about its safety, potential risks, and effectiveness.

Clinical drug trials play an important role in improving how doctors treat and prevent illnesses. These trials are typically conducted in four phases. Phases I, II, and III involve investigational drugs that have not yet been approved by the U.S. Food and Drug Administration (FDA) for general medical use. These early phases focus on evaluating the drug’s safety, appropriate dosage, and effectiveness. Phase IV trials take place after a drug has received FDA approval and is available by prescription. In this phase, researchers continue to monitor the drug’s long-term safety and effectiveness and may explore its potential use for treating additional conditions. 

Clinical trials are conducted by a dedicated research team composed of scientists, physicians, advanced practice practitioners (APPs), nurses, and research coordinators. These professionals work together to ensure that studies are conducted ethically, safely, and in accordance with regulatory standards.
 
Clinical trial sponsors can be individuals, institutions, companies, government agencies, or other organizations responsible for initiating, managing, or financing a clinical trial. However, they do not directly conduct the research itself.

Clinical Trial Participants
People who take part in clinical research come from the general public and choose to participate for many reasons. Participants may be healthy volunteers or individuals with a medical condition.

Healthy Volunteers
Healthy volunteers play an important role in clinical research, particularly in early-stage studies and in the development of diagnostic tools such as blood tests or imaging technologies. By providing information about what is considered “normal,” they help researchers establish baseline data for comparison with patient results. Healthy volunteers are often matched to patient participants based on factors such as age, sex, or family relationship and may undergo the same tests, procedures, or treatments to help researchers better understand disease processes.

Patient Volunteers
Patient volunteers are individuals with a known health condition who participate in research to advance understanding, diagnosis, or treatment of their disease. Their involvement is essential to evaluating new drugs, devices, or treatment strategies. While some studies—particularly later-phase trials—may offer direct benefit, others may not provide immediate therapeutic value.

Study Commitment and Informed Consent
The time commitment, level of discomfort, and potential risks vary by study. Some trials involve minimal participation, while others require more time or include procedures with greater risk. All participants complete an informed consent process, during which the study’s purpose, procedures, expectations, and potential risks are fully explained to support an informed decision about participation

Clinical trials follow a detailed plan called a protocol. The protocol is carefully designed to balance potential benefits and risks to participants while addressing specific research questions. It includes important information such as:

•    The goal of the study
•    Eligibility criteria for participation
•    Measures to protect participants from risks
•    Details about tests, procedures, and treatments involved
•    The expected duration of the trial
•    The types of information and data that will be collected

The protocol ensures that the trial is conducted safely, ethically, and effectively to produce reliable results.

Informed consent is the process through which potential participants learn the essential details about a clinical trial before deciding whether to take part. This exchange of information continues throughout the study to keep participants informed. The research team explains the purpose, duration, required procedures, risks, and potential benefits of the trial. Participants receive an informed consent document outlining all these details, along with contact information for questions or concerns. Signing this document is required to enroll in the trial. Importantly, participation is voluntary, and you may choose to withdraw from the study at any time without penalty.

The time and effort required from participants can vary widely. Some clinical trials may only require a short-term commitment with minimal involvement, while others can span weeks or even months and demand a longer-term dedication.

The time and effort required from participants can vary widely. Some clinical trials may only require a short-term commitment with minimal involvement, while others can span weeks or even months and demand a longer-term dedication.

In many clinical trials, the study sponsor provides the investigational drug, device, or other study-specific intervention at no cost. However, routine medical care—such as standard doctor visits, hospital stays, or treatments that you would receive even if you were not participating in a clinical trial—is usually the responsibility of the patient or their health insurance.

While some tests, procedures, or visits that are related to the research study may be covered by the study sponsor, many are considered routine care and are billed to you and/or your health insurance. You are responsible for any applicable copayments, coinsurance, or deductibles required by your insurance plan.

Coverage for routine care related to a clinical trial can vary by insurance plan. Participants are encouraged to contact their insurance company before enrolling to confirm what costs are covered and to determine whether there are any restrictions, limitations, or prior authorization requirements related to participation in a clinical trial.

The primary goal of clinical research is to generate knowledge that improves human health and deepens our understanding of biology. This progress is only possible because individuals—both patients and healthy volunteers—choose to participate in clinical trials. Ethical guidelines exist to protect these participants and to uphold the integrity of the scientific process.