Clinical trials are medical research studies that involve the participation of volunteers to help scientists understand and develop new ways to prevent, detect, or treat diseases and their symptoms. These investigational treatments may be new drugs, a combination of drugs, new ways to deliver an already marketed drug, or a medical device.
Before a new medication is approved, it must undergo a series of lengthy and rigorous tests, first in the laboratory, then in animals, and ultimately in humans. A new medication or medical device must not only be safe and effective, but in many cases it must also demonstrate a superior benefit when compared to similarly approved products.
A clinical study is conducted according to a research plan known as a protocol that has been developed by the pharmaceutical company and reviewed by the FDA along with an Institutional Review Board (IRB) representing the public interest. These agencies monitor the progress of these studies.
The protocol determines:
Clinical trials are led by a principal investigator, who is often a medical doctor, along with a research team made up of doctors, nurses and other health care professionals. All of the participants are volunteers. It is important to conduct research in a variety of people to detect different responses to a single investigational product.
Investigational products such as medications, devices or procedures are conducted in various clinical trial phases to find different kinds of information.
Phase 1 studies include a small number (20-80) participants, usually healthy volunteers, in some cases patients with advanced disease (e.g. cancer, mental health disorders) and is often the first time tested in humans. The purpose of Phase 1 studies is to:
Phase 2 studies enroll more people than Phase 1 studies (100-300 participants) with the condition under study. In Phase 2 studies researchers further evaluate safety and determine if the investigational product has the intended effect in humans. These studies are sometimes randomized controlled trials where one group of patients receives the investigational product, while another group (the “control group”) receives a standard treatment or placebo. In order to provide unbiased comparative information, these study are often “blinded” which means neither the patients nor the researchers know who is receiving the investigational product.
Phase 2 studies usually take up to two (2) years to complete. About 33% of Phase 2 conducted successfully move on to Phase 3.
In Phase 3 larger numbers (thousands) of patients with the disease are included to:
These trials are usually randomized and blinded trials that generally takes up to several years (1-4 years) to complete. 25 to 30% of Phase 3 studies are successfully completed and are submitted to the FDA by the pharmaceutical company for approval to market the investigational product in the United States.
Phase 4 studies often called Post-Marketing Surveillance Trials are conducted after the investigational product has been granted approval from the FDA for consumer sale. These studies involve people in various populations and are generally intended to collect additional information after the investigational product is approved and marketed regarding its risks, benefits and use in various populations over a longer period of time. Results from Phase 4 studies can influence whether an investigational product should be taken off the market or if restrictions should be applied to the use of the investigational product.
By taking part in a clinical trial, you have an opportunity to try a new treatment that may or may not be better than those that already exist.
As a study participant you can also help others better understand how the treatment works in people of different races and genders because different people may respond differently to the investigational product. Regulatory agencies such as the FDA seeks to ensure that people of different ages, races, ethnicities, and genders are included in clinical trials.
Some reasons to participate in a clinical trial:
Each clinical research study has specific eligibility criteria which a participant must meet in order to be enrolled in the study. Therefore, not everyone who applies for a clinical research study will be accepted.
There are many different types of clinical research studies, including:
Usually, clinical trials compare a new product or therapy to something else to see if it works as well or better to treat or prevent a disease or condition. In a blinded study, a participant may be randomly assigned to receive the test product, or an existing, approved therapy.
In some studies, participants may be assigned to receive a placebo (a product with no therapeutic action that looks or acts like the test product). Comparison with a placebo can be the fastest and surest way to demonstrate therapeutic effectiveness of new products.
Potential participants are told before they enter a trial whether placebos are going to be used in the study and the risks and benefits of the study medication(s).
A clinical research study is conducted according to a research plan known as a study protocol which is designed to answer specific research questions and to assure the safety of the participants. The protocol includes the following information:
The following process generally occurs during the conduct of a clinical research study:
The clinical research group will review their existing patient databases or medical charts to identify potential patients who may be eligible to participate in the clinical research study. They may also place advertisements on the internet, newspaper, radio, or television to recruit interested participants. They may also conduct seminars, free health screenings, or forums.
Potential participants from these pre-screening efforts are contacted and briefly interviewed to confirm if they are potentially eligible to participate in the clinical research study.
If you are potentially eligible to participate in a clinical research study, an appointment will be scheduled for you to come to the clinical research site and meet with one of the research staff members. Before any study related procedures are performed you will be required to sign an informed consent form which details the study design, risks and benefits, your rights as a study participant and who to contact in case of an emergency. During the informed consent process, you will have an opportunity to ask questions you may have regarding the study and your participation. You should receive a copy of the signed informed consent form for your records and future reference.
Once you agree to participate in the clinical research study and have signed the informed consent form, the study screening procedures will be administered. The purpose of the screening visit is to determine if you meet the specific inclusion and exclusion criteria for the specific clinical research study you agreed to participate in. Generally, during the screening visit you may be asked to answer questions regarding your medical history, medications and other treatments you are taking, and complete questionnaires. A member of the research team may also assess your general health by performing a physical examination, EKG and collect blood/urine samples.
Generally, the investigational product is not dispensed during the screening visit.
If you meet the specific inclusion and exclusion criteria (which includes a study acceptable laboratory and medical results) for the study you will be asked to return to the clinic for a series of study visits sometimes referred to as Treatment Visits. It is generally during these study visits that a study participant will be receive the investigational product or the comparator product (which sometimes can be a placebo). The number of study visits and the interval in which the visits occur varies from study to study.
Once you have reached the end of the treatment visit, an End of Study Visit is conducted. Generally, the same or similar assessments conducted prior to you receiving the investigational product is repeated. The research team will also discuss you follow-up treatment options (which may include receive standard of care treatments for your illness) and if required by the study you may be asked to return to the research site for a follow-up safety visit.
The number of follow-up safety visits and the interval in which the visits occur varies from study to study. The purpose of this visit is to assure you are not experiencing any lingering side-effects from the investigational product or from the overall participation in the study.
To help you decide if you want to be in a study, the FDA requires that study participants are given complete information about the study before they agree to take part.
Informed consent forms should be written so the participant can easily understand it and should include:
If you don’t understand the information included in the informed consent form, be sure to ask the doctor or other research staff member to explain it. This discussion should take place in private, and you must be given enough time to make an informed decision. In most studies, you should be allowed to take the consent form home for further discussion with others, such as family, caregivers and primary care physicians. Make sure you understand all of it before you agree to be in the study.
Before you can be in the study, you must sign the informed consent form, showing that you have been given this information and understand it. The informed consent form is NOT a contract and you can leave the study at any time, for any reason. You may ask questions at any time throughout the study.
In addition, you must also be informed of any new information learned during the study that may affect your willingness to continue to participate in the study.
Here are some questions to ask your doctor to help you decide if you want to take part in a clinical trial:
Every clinical research study is led by a Principal Investigator who is often a licensed medical doctor, and a clinical research team that may include doctors, nurses, social workers, and other health care professionals. In general, the clinical research team roles and responsibilities will include: