Helping Others by Contributing to Medical Research
What is a Clinical Trial?
A clinical trial, also called a research study, is a scientific evaluation that helps medical professionals learn about an investigational medication or vaccine.
But there’s much more to a clinical trial than just that. To find out what we mean, watch this short video that will:
- Describe a clinical trial in more detail
- Explain investigational medication
- Give you an idea of what it’s like to participate in a clinical trial
What are the phases in a Clinical trial?
A clinical trial, also called a clinical research study, is a carefully designed scientific evaluation conducted by doctors and researchers to determine if an investigational medicine, device, or procedure is safe or effective.
Trials are conducted in four phases:
Examine the safety of an investigational medicine in healthy people.
Assessment of safety and additional efficacy and establishing the best dosing regimen while testing safety in patients.
Confirm the efficacy and safety of an investigational medicine in patients.
Phase 4 trials are conducted after an investigational medicine has been approved to gather additional efficacy and safety information.
Why is clinical research important?
Clinical research studies help doctors and scientists determine if investigational medicines are safe for people to take and whether they are effective in treating a condition, disease, or disorder. In rare diseases where the population is limited and not as much is known in comparison to other more common diseases, clinical research may be even more impactful toward improving researchers’ understanding of the disease.
What you can expect to happen in a clinical trial?
Clinical trials are rarely the same. They can last from weeks to years, study different doses of a medication, or even compare multiple medications at once. While the details change, all trials are structured in a similar way. Typically, there are four main parts of a trial, with each part serving a different purpose.
Screening
Before you can join a trial, the trial doctor and staff must determine if you meet the requirements to participate and you will need to sign an Informed Consent Form (ICF). To do this, they will conduct a number of tests and assessments to see if you qualify.
Enrollment
Once you complete screening and it’s determined that you qualify, you will have the option to enroll in the trial. If you choose to enroll, you will begin to participate in the trial.
Investigational Treatment Phase
In this part of the trial, you will have study visits and take your assigned trial medication. The number of visits and length of time you receive trial medication will depend on the specific trial. While you’re receiving investigational medication, you will attend clinic visits and undergo tests and assessments.
Follow-Up
Once Treatment ends, you will enter Follow-Up. You won’t take any investigational medication, but you may continue attending clinic visits. Additionally, the trial doctor and staff will stay in touch with you to continue monitoring your health. The length of time you spend in Follow-Up will depend on the specific trial.
Why are participants needed?
Clinical research studies often require many volunteers to participate in a single study; sometimes thousands of participants are needed to obtain reliable information. This can be especially be challenging in rare diseases, as it can be difficult to find enough patients to enroll.
Is a clinical trial right for you?
Each clinical trial has a specific set of eligibility criteria that you must meet before you can join. Most trials will require you to be in a certain age range and have a specific disease or condition.
Here is an example of eligibility criteria:
- 18 years of age or older
- Diagnosed with type 2 diabetes
- Taking medication for type 2 diabetes
Prior to joining a trial, the trial doctor and staff will review the eligibility criteria with you.
Patients tell all: why clinical trials were right for them
If you’ve never participated in a clinical trial, it’s common to have questions about what the experience is like. While My Trial Community is here to answer any questions, another good way to learn about clinical trials is to talk with people who’ve volunteered as trial participants themselves. It might be a friend, a family member, a classmate, or a coworker. As a starting point, check out the four patients below and hear how clinical trials made a difference in their lives.
Amy
Amy has spent the past 20 years living with the challenges of systemic scleroderma, a rare disease that affects only about one in 10,000 people. Hear what first led her to clinical trials and why she thinks they played a huge role in developing multiple treatment options that benefit her disease community today.
George
A retired doctor himself, George was taken by surprise to learn he had mantle cell lymphoma, a type of cancer affecting the immune system. Facing a difficult prognosis, he quickly realized that joining a clinical trial offered him the chance to once again be a contributing member of the scientific community. Now, some three years later, he’s only looking ahead.
Lucy
With no cancer history in her family, Lucy had little reason to believe that a small lump in her breast could be cancerous. Until it was. Listen as Lucy, a young teacher, does her own research to decide if a clinical trial of two similar cancer treatments is right for her.
Alicia
When Alicia was diagnosed with HIV, she largely kept it to herself. It was a different time, and the stigma of HIV was unforgiving. But as times changed, she began to have a different outlook. Hear how clinical trials became one of the many ways that Alicia could give back to her community.
Interested?
Clinical research study participation offers an opportunity to be part of something bigger - research that may advance understanding of a disease or help get a new treatment approved. However, participation may also involve risks. Always make sure to discuss any potential benefits and risks with your study doctor. If you are interested in participating in a clinical trial and you would like to learn more - your doctor, nurse, or other healthcare professional could help you in searching for trials that are available.
Benefits
- Getting actively involved in your own healthcare
- Regularly scheduled medical attention from a team of healthcare professionals
- Helping others by participating in research that may result in advancing the understanding and treatment of the disease
Risks
- The investigational treatment may not work
- The investigational treatment may cause side effects
- Potential to be excluded from other clinical trials
Terms you should know
Arm
An arm is a group of people in a clinical trial who receive the same intervention or medication. A trial may have two or more arms to compare research outcomes. For example, one arm may receive an investigational medication or vaccine and the other arm may receive a placebo and/or the standard of care.
Disease Disparity
When specific patient populations (i.e. age, ethnic, racial, regional, sex) have certain diseases and conditions at higher rates than others, this is known as a disease or health disparity. Disease disparities within these patients may be caused by differences in genetic or environmental factors.
Enroll
When you enroll in a clinical trial, that means the trial doctor and staff determined that you are eligible, and you have agreed to participate.
Informed Consent Form (ICF)
The ICF is a document that will be provided to you prior to joining a trial. You must read the ICF as it helps to explain the trial details in easy-to-understand language to fully inform you about trial participation. Your trial doctor will discuss the ICF with you and will take the time to answer any question you may have. You can also take the time to discuss with your family and/or your general practitioner. After you have no more questions and you have decided you want to participate in the trial you must sign the ICF before you can enroll in the trial.
Investigational Medication or Vaccine
The investigational medication or vaccine is what is being evaluated in the trial. Typically, it is not approved for public use for the condition being studied and may only be available in clinical trials.
Phases
Phases are the series of trials (i.e. phase I, II, etc.) an investigational medication or vaccine will go through to determine if there is scientific evidence that an investigational medicine may prevent or treat a disease or condition. Each phase generates data to assess if an investigational medication or vaccine may be approved by the regulatory authorities (such as FDA or EMA) for public use.
Placebo
A placebo is an inactive substance that looks like the investigational drug or treatment being studied in some clinical trials used in some clinical trials. It is used in the control group or arm of a trial to compare with the investigational medicine/vaccine being studied. When there is standard treatment available for a disease or condition (see Standard of Care), in most trials placebo is added to the standard of care.
Plain Language Summary
A Plain Language Summary (PLS) is a summary of the key results of a trial provided after a trial is complete. Each PLS is written in a way that’s easy to read, and helps participants understand more about the research they have contributed to.
Randomization
When you are randomly assigned to one of the different arms or groups in the clinical trial, this is known as randomization. This typically occurs when you are enrolled in a trial comparing an investigational medication or vaccine to the standard of care or placebo.
Regulatory Authorities
Each country has a set of regulatory authorities that reviews the results of clinical trials. Based on the information gathered about an investigational medication in clinical trials, these authorities will determine whether it will be approved and be made available to the public.
Standard of Care
The standard of care is the most accepted and standard treatment for a disease or condition. This may vary by country or region.
Tests and Assessments
You will undergo various tests and assessments during trial participation. The types of tests and assessments will depend on the trial, but these usually include vital signs measurements, physical exams, blood and urine sample collections, and questions about how you are feeling.
Trial Clinic Visits
You will be required to attend clinic visits, also called study visits, throughout a trial. The number of visits will depend on the trial, but at each clinic visit, the trial doctor and staff will perform various tests and assessments to monitor your health.
Trial Doctor
The trial doctor is in charge of the trial at a clinic. He or she will determine whether patients can be in a trial and whether they should continue in a trial. A trial doctor may also be called a study doctor or investigator. It can be that your regular doctor is the trial doctor, but it can also be that you need to visit another doctor.
Trial Sponsor
The trial sponsor, which is typically a pharmaceutical company, academic center, or non-profit organization, conducts a clinical trial and pays the trial doctor and staff to conduct the trial.
Voluntary
Participation in a clinical trial is your decision. That means you don’t have to participate if you don’t want to. Even if you participate, you can leave a trial at any time and for any reason.