Clinical trials are a necessary step in the process of making new medications generally available. Clinical research trials are conducted by doctors to test the safety and effectiveness of investigational medications. All clinical research is regulated by the federal government and also reviewed by independent ethics committees in order to ensure patient safety.

All medications must go through the clinical trial process. We now have effective treatments and cures for hundreds of diseases thanks to clinical research and the patients who have participated in clinical trials.

As a volunteer in a clinical trial, you help advance the development of new medical therapies that may offer better treatments and even cures for life-threatening and chronic diseases. All clinical research is regulated by the federal government and also reviewed by independent ethics committees in order to ensure patient safety. When you participate in a clinical trial, you receive quality medical care at no charge from local physicians and medical professionals. For most studies, you are also paid for your time and travel.

Prior to beginning the study, you will receive an informed consent document, which includes information about the purpose of the study, the investigational medication, the duration, the study visits and procedures, potential benefits and risks, and if appropriate, what you will be paid for your time and travel. If you choose to consent, physicians and healthcare professionals will then determine if you meet all defined criteria for participation.

During the study, physicians and healthcare professionals are committed to providing you with quality patient care and medical attention. You should feel free to discuss your medical treatment with our research staff members at any time. As a participant, you may be asked to:

-Regularly visit a healthcare provider

-Have laboratory and diagnostic tests

-Take medications

While each study is different, the benefits of participation may include some or all of the following:

-Access to medication, medical care from specialists who know your condition, and laboratory services at no cost

-The opportunity to receive an investigational medication that is not widely available

-The knowledge that you are helping advance medical science.

-You may be compensated for time and travel.

Each trial is different but you should be aware of any potential risks. Some of them may include:

-There are no guarantees that you will benefit from the treatment

-There may be unpleasant or unknown side effects

-In some studies, you may receive a placebo, which contains no medication

It is important to know that as a volunteer, you have the right to leave the study at any time, and for any reason.

Clinical trials are conducted according to a protocol plan designed to safeguard the health of the volunteer participants while aiming to answer specific research questions. The protocol includes the following information:

-Purpose of the study

-How many people will participate

-Who is eligible to participate in the study, and who will be excluded

-Details about schedule of tests, procedures, and medications, and their dosages

-The length of the trial

-The type of information that will be collected

-The end points

A clinical study is led by a principal investigator (PI, in our case, Dr. Vento). The principal investigator runs the clinical trial and makes sure the health team follows the plan. Members of the research team monitor the participants’ health regularly to determine the study’s safety and effectiveness.

Each clinical trial in the United States must be approved and monitored by an Institutional Review Board (IRB) to ensure the risks are minimal and are worth any potential benefits. An IRB is an independent committee of doctors, statisticians, and other members of the community that ensure clinical trials are ethical and that the rights of participants are protected.

A variety of experts review the trial before it is made public for approval. Federal regulation requires all U.S. institutions that conduct or support biomedical research involving people have an IRB initially approve and periodically review the research. Each trial is reviewed many times by different experts before it’s started at a treatment center.

Ethical Guidelines

The goal of clinical research is to develop knowledge that improves human health or advances our understanding of human biology. People who participate in clinical research make this possible. The path to finding out if a new drug or a new treatment is safe or effective is to test it on patient volunteers. The purpose of ethical guidelines is to protect patient volunteers and preserve the integrity of the science.

Informed Consent

Before joining a clinical trial, a participant must qualify for the study. Some research studies seek participants with the illnesses or conditions to be studied in the clinical trial, while others need healthy volunteers. Informed consent is the process of providing potential volunteer participants with the main details about a clinical trial before they decide to participate. The process of informed consent continues providing information updates to enrolled participants during the course of the study. Translation or interpretation assistance can be provided for participants with limited English proficiency.

The research team provides an informed consent document that includes study details, such as study purpose, duration, required procedures, risks and potential benefits, alternatives to the study, and who to contact for more information. The participant then decides whether to sign the document.

Informed consent is not a contract, and volunteers are free to withdraw from the study completely or refuse particular treatments or tests at any time. However, refusing treatment will sometimes make them ineligible to continue the study.tion

Most clinical research that involves the testing of a new drug progresses in an orderly series of steps, called phases. This allows doctors and scientists to ask and answer questions in a way that results in reliable information about the drug and protects the patients. Clinical trials are usually classified into one of four phases:

Phase I trials: Evaluate how a new drug can be provided safely, including proper dosing and potential side effects. This phase also assesses the best way to administer treatment, such as by mouth, injected into the blood, or injected into the muscle. Usually, a phase I trial enrolls a small number of patients or healthy volunteers — sometimes, as few as a dozen. Phase I trials are the first studies in humans, and, as a result, are an important step to transition novel treatments from the bench to the bedside.

Phase II trials: Use the results from Phase I trials regarding maximum tolerated dosages and dose-limited toxicities. Phase II trials continue to test the safety of the drug and begin to evaluate how well the new drug works. Treatment is targeted at the population of volunteer patients that responded most favorably in Phase I. Usually, only a small number of patients participate in Phase II trials, which are focused on a particular type of condition.

Phase III trials: This type of trial may test a promising new drug or a new combination of drugs. It may also test a new surgical procedure that may increase cure rates or decrease side effects or late effects of treatment. A participant is assigned to the standard group or the new group at random, called randomization. Phase III trials often enroll large numbers of patients and may be conducted at many doctors’ offices, clinics, and research centers nationwide.

Phase IV trials: Continue evaluation that takes place after FDA (Food and Drug Administration) approval, when the drug is already on the market and available for general use (post-marketing surveillance). These trials gather more information about long-term side effects.

Once a clinical trial is completed, the information collected during the study is examined carefully by the principal investigator and the research team to determine the meaning of the findings and the potential need for further testing.

When a Phase I or II trial is completed, the investigators decide to move on to the next phase or to stop the study because the drug or intervention being tested was determined to be unsafe or ineffective.

After a Phase III trial is completed, researchers scrutinize the data to decide if the results have medical significance. If the results are important, they may be featured in news media and discussed at scientific meetings and by patient advocacy groups before they are published.

Results from clinical trials and non-intervention research studies are often published in peer-reviewed scientific journals. Peer review is a process by which experts review the report before it’s published to ensure the analysis and conclusions are sound. Once a new approach is proven safe and effective in a clinical trial, it may become the standard of medical practice. Ask the research team investigators if the study results will be published. Usually, a clinical trial’s published results are available in the National Library of Medicine’s PubMed database. You can search for the study using the official name of the study or the Protocol ID number.