Research Learning Center

What is a clinical trial?

Clinical trials are medical research studies performed with the help of patients who volunteer to try new diagnostic techniques or new therapies, or to be observed as part of a group of people with a specific health issue. The doctors conducting the research seek to answer specific medical questions — ultimately to improve health and patient care.

Clinical trials are needed to develop or test new drugs, devices, treatments, and procedures and help answer questions like:

• Does it work?
• Does it work better than other treatments?
• What amount is safe and effective?
• What are the side effects?

Some of these treatments have not been approved by a government agency, which is why they are called “investigational” treatments. If clinical trials show that a new treatment works and is safe, then it can be approved to be used by the people who need it. If you have ever taken medicine or gotten a vaccine, then you have benefited from clinical trials.

How do clinical trials work?

For most clinical trials a participant is assigned to either an intervention or a control group.

Intervention Group: Participants in this group receive the intervention that is being tested.

Control Group: Participants in this group do not receive the intervention. Instead they might receive the standard treatment for the disease or condition, or they might receive a “placebo”. A placebo looks or feels like the intervention, but it is not an active medicine or treatment. The control group helps the researchers understand the effects of the experimental intervention.

Research participants are often assigned randomly to a group. This means your group assignment happens by chance (like tossing a coin). If you take part in a clinical trial, you might not get the experimental treatment that is being tested. In some cases, you won’t know the group to which you are assigned.

What are clinical trials used for?

Clinical trials are important because they are the best way to study (investigate) new treatments and understand their safety and efficacy (how well the drug or treatment works).

Types and Phases of Clinical Trials

Clinical trials can be used for much more than testing new drugs. Types of clinical trials include:

• Treatment trials – Test new drugs, devices, therapies or surgical techniques.
• Prevention trials – Look for ways to prevent disease.
• Diagnostic or screening trials – Find better tests for identifying or screening for certain health conditions.
• Quality of life trials (or supportive care trials) – Explore ways to improve quality of life for people living with chronic disease.

What are different types of study designs?

Interventional: Participants receive experimental treatment — which can include a drug, device, surgical procedure or an existing therapy used in a new way — to determine whether it’s safe and effective.

Observational: Participants receive diagnostic or therapeutic care through their regular care providers and researchers observe and note the health outcome, sometimes through review of patient medical charts.

Chart Review: Doctors use data from existing patient health charts to answer the research question.

Registry: Researchers study a specific patient population by observing participants with a particular disease, condition or exposure to something that may be affecting their health.

Specimen Collection and Research Repository: Participants donate biologic samples for analysis, including blood, solid tissues, cells, cell cultures or molecules derived from tissues — such as DNA, RNA and proteins — and body fluids. Participants may consent to use of their samples for a particular study or for unspecified future research or testing.

Behavioral Studies: These are studies that test how people act (behave) in different ways. For example, completing a survey about your experience with an illness, or being part of a focus group to talk about ways a program is working or not working.

Genetic Research: These are studies that look at the role of genes in different diseases. For example, a genetic research study discovered that the BRCA gene mutation is a risk factor for breast cancer.

Public Health Research: This is research that focuses on the prevention and treatment of illness and disease in a community or population. A media campaign that encourages healthy behaviors is an example of public health research.

Community-Based Participatory Research (CBPR): This is research that engages community partners as equal participants in the research. CBPR is an effective method for community members and researchers to combine knowledge to improve community health.

How do clinical trials test new treatments for FDA approval?

The idea for a clinical trial often starts in the lab. After researchers test new treatments or procedures in the lab and in animals, the most promising treatments are moved into clinical trials. As new treatments move through a series of steps called phases, more information is gained about the treatment, its risks, and its effectiveness.

Drug Trial Phases

For the study of new drugs, or of existing drugs used in new ways, pre-clinical and clinical trials are divided into different phases, numbered from 0 to 4. The earliest phases may look at safety or side effects. A later phase may test whether a new treatment is better than existing treatments:

• Pre-Clinical: Research using In Vitro (i.e., test tube or petri dish) or In Vivo (i.e., live organism/animals) to find out if a drug, procedure, or treatment is likely to be useful. Preclinical studies take place before any testing in humans is done.
• Phase 0: An exploratory study that requires very limited human exposure to the drug and has no therapeutic or diagnostic goals.
• Phase 1: Conducted with an emphasis on safety, the goal is to discover how the drug is processed, toxicity and any side effects.
• Phase 2: Gathers preliminary data on effectiveness — whether the drug works in participants with a certain condition, compared with participants receiving a different treatment or a placebo (a medicine that has no effect).
• Phase 3: Measures safety and effectiveness by examining different populations taking different dosages of the drug or in combination with other drugs.
• Phase 4/Post Market Approval: Occurs after the Federal Drug Administration (FDA) has approved a drug, but researchers want more information about its safety, efficacy or best use.

Medical Device Trial Phases

Clinical trials are also used to test medical devices — healthcare products that don't achieve results through chemical action or by being metabolized. A device can range from something as simple as a tongue depressor or thermometer to a complex instrument, such as a specialized heart valve or robotic surgical device. Device trial phases include:

• Pilot/Feasibility – May be used to answer basic research questions or provide support for a future study. It's an initial, exploratory stage to determine whether the potential benefit justifies any risk.
• Pivotal – Evaluates the safety and effectiveness of the device for the intended use, which may require one or more studies.
• Post-market – Assesses safety of the device after it’s been used commercially, such as investigating rare adverse events or long-term effectiveness.

How are research studies funded and led?

The people or organizations that initiate studies are known as research sponsors. Doctors, medical institutions, foundations, voluntary groups, device and pharmaceutical companies sponsor medical studies. Federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD) and the Department of Veterans Affairs (VA) also sponsor research. Trials can take place in hospitals, universities, doctors’ offices or community clinics.

Every study has a principal investigator, or lead researcher, who takes direct responsibility for completion of a clinical trial. The principal investigator directs the research and reports to the sponsor.

Who participates in clinical trials?

People of all ages may participate in clinical trials. Some participants have a disease or condition that the researchers are studying; other participants are healthy volunteers. For each clinical trial, there are requirements regarding who can participate (inclusion criteria) and reasons why someone might not qualify to participate in the trial (exclusion criteria). Trial criteria may include age, sex, the type and stage of the disease, and previous treatment history.

Why is diversity important in clinical research?

Diversity in clinical trial participation helps ensure new treatments are safe and effective for all people. People may experience the same disease differently. It’s essential that clinical trials include people with a variety of lived experiences and living conditions, as well as characteristics like race and ethnicity, age, sex, and sexual orientation, so that all communities benefit from scientific advances.Learn more about why diversity in clinical research matters here(video). 

Can children participate in research?

Yes. It’s important that children be involved in research so that researchers can understand the health and behavior of children. Treatments may work differently in children than they do in adults.

There are special protections and laws in place that apply to any research that involves children. Children (any person under 18) cannot agree to participate in research for themselves, and must have a parent or guardian give permission (consent) for their child to participate in a research study.

What are the potential benefits or risks of participating in research?

Benefits

• Having access to experimental treatments that are not yet available to the public
• Helping others by contributing to the development of new medical treatments or procedures

Risks

• Receiving an experimental treatment when its effects are not yet fully understood
• Experiencing unpleasant, serious, or even life-threatening side effects or complications

What questions should I ask my doctor or the study staff?

Below are some questions you can ask your doctor or study staff when considering participation in a clinical trial.

• What is the main purpose of this study?
• Has the study been approved by an IRB?
• What are the risks and benefits of joining the study?
• Does the study involve a placebo or a treatment that is already on the market?
• How will the treatment be given to me?
• How long is the study going to last and what will I be asked to do as a participant?
• What has been learned about the study treatment and are any study results published?
• Do I have to pay for any part of the study? Will my insurance cover these costs?
• Is there any reimbursement for travel costs or childcare?
• Will I be able to see my own doctor?
• If the treatment works for me, can I keep using it after the study?
• Can anyone find out whether I’m participating in the clinical trial?
• Will I receive any follow-up care after the study has ended?
• What will happen to my medical care if I stop participating in the study?
• Does the physician/investigator have any financial or special interest in the clinical study?
• What are the credentials and research experience of the physician and study staff?
• When and how will the results of the clinical trial be provided to me?
• Will I learn my treatment assignment?

Do I get paid to be in a clinical trial?

Some trials pay for travel costs, time, and commitment. But not all do. Each trial will have its own guidelines that tell you if you will be paid, what costs will be covered, and what expenses you can be reimbursed for. Check with your trial doctor and the research team to confirm.

Payments in trials are made for your time and possible inconvenience of participating in the trial. These payments are also called compensation or stipends. The amount is usually based on the trial length, number of visits, the types of procedures, and other factors.

Will my insurance cover any costs?

Some costs may be covered by your health insurance. These may include travel costs for taxis, buses, gas mileage, or hotels. There could also be costs for the care you receive in a trial.

Usually, most of the costs in a trial are covered by the trial sponsor. The sponsor is the organization that funds and conducts the trial. Every trial has an Informed Consent Form that explains the possible costs, payments you may receive, and expenses that will be covered by the sponsor. You can also ask the trial staff for more information about costs.

What are my rights as a research participant?

As a research participant, you have the following rights:

• To be treated in a caring and polite way.
• To be told what the study is trying to find out.
• To be informed what will happen, and whether any of the procedures, drugs or devices are different from what would be used in standard medical care.
• To be told about possible side effects or discomforts that may occur during the study.
• To be told if you can expect any benefit from being in the study and, if so, what the benefit might be.
• To be told of other choices for treatment you have, and how they might be better or worse than being in the study.
• To be told what sort of treatment is available if any medical problems arise.
• To be allowed to ask any questions about the study both before agreeing to be involved and during the course of the study.
• To be free from pressure when deciding if you want to be in the study.
• To be told about new information learned during the study that might affect your safety or your willingness to continue to take part in the study.
• To refuse to be in the study, or to change your mind about being in the study after it has started. This decision will not affect the care you receive at the hospital.
• To receive a copy of your signed consent form.

If you are not fluent in English or prefer to communicate with your study team in a language other than English, study materials will be presented in language understandable to you and an interpreter will be provided for conversations with the study team. Boston Medical Center has a dedicated Interpreter Services Department that staffs certified medical interpreters who speak most of the languages spoken across the Boston area.

View Participant Bill of Rights in:

• English
• Español
• Kreyòl ayisyen
• Français 
• Português
• Kriolu 
• Tiếng Việt

What are clinical trial protocols?

Clinical trials follow a plan known as a protocol. The protocol is carefully designed to balance the potential benefits and risks to participants, and answer specific research questions. A protocol describes the following:

• The goal of the study
• Who is eligible to take part in the trial
• Protections against risks to participants
• Details about tests, procedures, and treatments
• How long the trial is expected to last
• What information will be gathered

A clinical trial is led by a principal investigator (PI). Members of the research team regularly monitor the participants’ health to determine the study’s safety and effectiveness.

What is informed consent?

Informed consent is the process of providing you with key information about a research study before you decide whether to accept the offer to take part. The process of informed consent continues throughout the study.

To help you decide whether to take part, members of the research team explain the details of the study. If you do not understand English, an interpreter will be provided. Boston Medical Center has a dedicated Interpreter Services Department that staffs certified medical interpreters who speak most of the languages spoken across the Boston area.

The research team provides an informed consent document that includes details about the study, such as its purpose, how long it’s expected to last, tests or procedures that will be done as part of the research, and who to contact for further information. The informed consent document also explains risks and potential benefits. You can then decide whether to sign the document. Taking part in a clinical trial is voluntary and you can leave the study at any time.

It’s not a contract and it’s not binding. You may withdraw from a trial at any time.

What is IRB?

In the United States, an Institutional Review Board (IRB) approves and monitors clinical research to ensure that risks are reduced and are outweighed by potential benefits. IRBs typically include people with varying backgrounds, including community member(s), to make sure that research activities are completely and adequately reviewed.

The Office of Human Research Affairs (OHRA) is responsible for overseeing human research conducted at BMC, to ensure that it is held to the highest ethical standards and meets all regulatory requirements.

Our local BMC/BU IRB needs community members to serve on the IRB and provide their opinions. To learn more about volunteering with the IRB, call or email the IRB at 617-358-5372 or email the IRB.

Learn more about BMC's IRB.

What are different ways I can be involved in research?

Become a Research Advocate – The Office of Human Research Affairs (OHRA) offers trainings to community members about getting involved in research. Email them here for more information.

Participate in a study – Please see the section below to learn more about participating in a study.

Join our Institutional Review Board (IRB) – An IRB is a group of people who review research to make sure it is ethical and safe. Our IRB needs community members to serve on the IRB and provide their opinions. Please click here to learn more about volunteering with the IRB, and call the IRB at 617-358-5372 or email the IRB for more information.

How can I learn more about the outcome of completed trials?

You can find results from past clinical trials on the website. Some sponsors post results on their own websites. The European Union (EU) also posts plain language summaries for trials that take place in any EU member state. You can search for these summaries on this website.

As a participant, you are entitled to a plain language summary of the results of your clinical trial once the trial is finished. If you do not have access to a summary, check with the trial doctor or research team or contact the Clinical Research Network.