What is Clinical Research
At NYU Langone Health, our researchers and physicians conduct hundreds of research studies and clinical trials to develop new healthcare options for people with a wide variety of medical conditions. We aim to speed up the development of new treatments and enhance medical care.
Our studies help bring new medicines to people who need them and offer volunteers an opportunity to try new, experimental treatment options for their conditions. They receive targeted care in a setting designed to help advance medical knowledge.
All of our clinical trials meet the standards of good clinical practice and comply with strict rules set by the U.S. Department of Health and Human Services’ Office for Human Research Protections (OHRP) and the U. S. Food and Drug Administration (FDA). Adherence to these policies and protocols ensures that participants are treated safely and ensures that the rights, integrity, and confidentiality of research participants are protected.
A clinical trial is a research study involving human volunteers. Clinical trials are designed to answer specific research questions. Carefully conducted clinical trials are the safest and fastest way to find effective treatments and devise new ways to improve health.
A drug being studied in a clinical trial is called an investigational drug, and a device being studied in a clinical trial is called an investigational device. Most clinical trials test products to see how they compare to standard treatments. However, an investigational drug may also be compared with a placebo, which is a substance not expected to have any real medical effect.
Volunteering for a clinical trial is no guarantee of acceptance. Similarly, there’s no guarantee that a person in a clinical trial will receive the drug or medical product being studied.
Clinical trials are conducted according to a plan called a protocol. The protocol describes who may enter the study and the schedules of tests and procedures. A protocol also dictates the devices or drugs to be used, drug dosages (if applicable), possible side effects of treatment, the length of the study, and the outcomes that will be measured.
Many clinical trials are done to see if a new drug or device is safe and effective for people to use. Clinical trials are also performed for other reasons. Some compare existing treatments to determine which is better. The current, approved treatments are called standard treatments.
Clinical trials are also conducted to allow for the study of different ways to use standard treatments, to make them more effective or easier to use or to reduce their side effects. Sometimes studies are done to learn how to best use a treatment in a different population, such as children, in whom the treatment has not previously been tested.
Types of Clinical Trials
You may hear a physician or researchers refer to more than one type of clinical trial. This is because different trials may have different objectives, even if they are studying the same condition.
Treatment trials test experimental treatments, new combinations of drugs, or new approaches to surgery or other interventions, such as radiation therapy for cancer.
Prevention trials look for better ways to prevent a condition in people who have never had it (and in some cases are at risk for it) or to prevent a condition from returning. The approaches tested may include medications; vaccines; vitamins; minerals; or lifestyle changes, such as diet and exercise.
Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular condition.
Screening trials test the best way to detect risk factors or early signs of certain diseases or health conditions.
Quality-of-life trials, sometimes called supportive care trials, explore ways to improve comfort and quality of life in people with a chronic illness.
Clinical trials are sponsored or funded by a variety of sources, including federal agencies, such as the National Institutes of Health (NIH), the Department of Defense, and the Department of Veterans Affairs. They may also be funded by individuals or organizations, such as academic centers, foundations, pharmaceutical or device companies, voluntary groups, or physicians.
Drug Clinical Trial Phases
Clinical trials of new drugs are conducted in phases. Each phase of the trial has a different purpose and helps researchers answer different questions.
In phase I trials, researchers test an experimental drug or treatment for the first time in a small group of healthy volunteers, typically 20 to 80 people, to evaluate its safety, determine a safe dosage range, and document how a drug is metabolized and excreted. Phase I trials are also designed to identify any acute side effects.
In phase II trials, the experimental study drug or treatment is given to a larger group, typically 100 to 300 people who have the disease or condition that the product potentially could treat. Phase II trials are designed to determine if the drug or treatment is effective and to further evaluate its safety.
In phase III trials, the experimental study drug or treatment is given to large groups of people, typically 1,000 to 3,000, to further confirm its effectiveness, monitor side effects, or compare it with commonly used treatments. Ultimately these trials collect information that will allow the experimental drug or treatment to be used safely.
Phase IV trials are conducted on drugs after the FDA has approved them for marketing. This phase provides additional information on the drug’s risks, benefits, and optimal use. Phase IV trials may also be used to test the product in a specific population, such as children.
Benefits and Risks of Participating in a Clinical Trial
People enroll in clinical trials for many reasons. One common reason is that they have an interest in gaining access to novel experimental treatments, especially if all other treatment options have failed.
People also enroll because they want to make a valuable contribution to medical knowledge. Clinical trial participants can play an important role in getting new drugs and devices approved for use in treating other patients.
Volunteers may benefit from participating in a clinical trial if the investigational drug is confirmed as effective, but there is no guarantee that a person in a clinical trial will receive the drug or medical product being studied. Similarly, volunteering for a clinical trial is no guarantee of acceptance into the trial.
Anyone considering enrolling in a clinical trial should know that there are both benefits and risks associated with participation. Benefits include being actively involved in your health care; gaining access to new research treatments; having access to expert medical care for the condition being studied, since investigators are often specialists in the disease area being studied; and helping others by contributing to medical research.
Possible risks of participating in a clinical trial include unpleasant, serious, or even life-threatening side effects resulting from the study treatment; the chance that a treatment may not be effective; or the possibility that a protocol may require more of your time and attention than standard treatment, such as more trips to the study site, more treatments, more or longer hospital stays, or more complex dosage requirements.
All known risks are fully explained by the researchers before the trial begins. Participants are informed if new risk information becomes available during the trial.
Eligibility to Participate in a Clinical Trial
Trial guidelines, or eligibility requirements, are used in research to determine whether a person is or is not allowed to enter a clinical trial. These guidelines help identify appropriate participants and exclude those who may be put at risk by participating. Often, drugs or devices must be tested under very specific conditions, so not all participants with a given disease will be able to enroll in a clinical trial.
For each clinical trial, researchers develop eligibility criteria, such as age, sex, the type and stage of disease, previous treatment history, and the presence or absence of other medical conditions. These criteria help to reduce the amount of variation in the study, without threatening the scientific integrity of the trial, by removing medical variations that might complicate the analysis of the results.
Consequently, not everyone who applies for a clinical trial is accepted. Volunteers may be excluded because of the eligibility criteria or trial design or because the number of participants needed by the researchers to collect enough information to determine the safety and effectiveness of an experimental therapy has already been reached.
Additionally, a clinical trial for a specific drug or device is typically conducted at a limited number of institutions. People who live too far away from a research center may not be able to participate.
The Clinical Trial Process
The process followed in a clinical trial depends on the kind of trial being conducted. A clinical trial team includes physicians, research coordinators, and other healthcare professionals. At the beginning of a trial, potential participants are given complete information about the study. This process is known as “informed consent.”
The informed consent process provides an opportunity for the researcher and participant to exchange information and ask questions. A consent form must be signed by the participant prior to enrollment and before any study procedures are performed.
After the informed consent process is completed, members of the study team check the health of the participant, give specific instructions for participating in the trial, and monitor the participant carefully during the trial.
Participants have the right to leave a study at any time. Conversely, circumstances may arise under which their participation is terminated by the researcher without their consent.
For trials conducted at NYU Langone, researchers, doctors, and other health professionals administer the clinical trials according to good clinical practices, which are strict rules set by the OHRP and the FDA. These rules were created to ensure that the safety, rights, and confidentially of study participants are maintained during their participation in our trials.
At our institution, physicians, investigators, and other healthcare professionals are required to complete rigorous in-person and online research training, available through Research Enterprise Training (requires login using a Kerberos ID) and the Collaborative Institutional Training Initiative, concerning study conduct and management prior to conducting clinical research. In addition, the Office of Science and Research and Human Research Protections provide regulatory services and directly monitor all ongoing studies.
Clinical Trial Safety
Clinical trials are regulated with built-in safeguards to protect participants. The FDA and the OHRP oversee human protection in clinical trials to help protect the rights and welfare of volunteers.
In addition, human research conducted at NYU Langone must be reviewed and approved by an institutional review board (IRB). An IRB is an independent committee of physicians, statisticians, community advocates, and others that reviews research protocols to ensure that appropriate steps are taken to protect the rights and welfare of participants as subjects of research. An IRB reviews the adequacy of the informed consent document to ensure that it includes all elements required by law, the reading level is appropriate, and it is understandable to study participants. Research investigators must regularly update the IRB on a study’s progress, including prompt reporting of any unexpected serious events.
Although efforts are made to control the risks to clinical trial participants, some may be unavoidable because of the uncertainty inherent in research studies involving new medical treatments.
Informed Consent Process
Closely related to the notion of voluntary participation is the requirement of informed consent, which is the process through which potential study subjects learn the key facts about a clinical trial before deciding whether to participate.
Research participants must be fully informed about the procedures and risks involved in research and must give their consent to participate. During this process, the research team provides an informed consent document with details about the study, such as its purpose and duration, required procedures, and key contacts. Risks and potential benefits are also explained in the document.
To help someone decide whether to participate, the doctors, nurses, and research coordinators involved in the trial explain the details of the study. If a potential participant’s native language is not English, the research team should use translated consent forms which can be found in our template library.
This information enables those interested in study participation to make an informed decision about the level of risk they are willing to accept before they enter the trial. The participant then decides whether to sign the document.
Signing the document indicates that the participant has been informed of the study’s risks and possible benefits and that it is a form of research. It also acknowledges that participation is voluntary and formally documents the person’s consent to participate. Research volunteers have the right not to participate or to end their participation in the clinical trial. Participation in research is voluntary. Informed consent is not a contract, and the participant always has the right to withdraw from the study at any time.
After a Clinical Trial Ends
After a study is complete and all collected data are properly entered into the trial database, researchers analyze the data to determine the answers to the specific health questions posed by the study.
The types of questions to be answered depend on the purpose of the study. As an example, with drug trials, results from phase I and phase II trials are used to determine whether a drug is safe or should be tested further or to design later-phase trials. Results from phase III trials may be used by the FDA to determine whether a drug was shown to be safe and effective and therefore approvable for marketing to the public.