Clinical Trials A Comprehensive Overview

Clinical Trial Definition

A kind of research study that clinical professionals perform in group of people to evaluate a clinical, behavioral, or surgical intervention. This is a technique to find out whether a new device, medicine, or drug is effective or not to cure some disease or condition. They may test the drug or device such as a pacemaker to understand its workability. It also aims to see whether it is safe for people or not. Clinical trials may work to check the usability of a drug. For example, it may proceed trials to see whether a new drug cures the disease early or not? It also analyzes the side effects of the medicine. In short, clinical trials help to understand the significance, drawbacks, and benefits of a particular drug, diet, or device.

Who Is Involved in Clinical Trials

The primary investigator, who is often a medical doctor, conducts clinical trials. Research teams consisting of doctors, nurses, social workers, and other healthcare professionals also conduct clinical trials. Pharmaceutical companies may sponsor or fund such studies. Academic medical centers, social organizations, and voluntary groups also take part in such activities. There are federal agencies such as the National Institute of Health, the U.S. Department of Defense, and the U.S. Department of Veterans affairs that sponsor these trials. Different professionals conduct trials at hospitals, universities, doctors’ offices, and community clinics.

Purpose of Clinical Trials

When we are doing something, a question comes to mind that why are we doing this? What is its significance? What is the need to conduct such activities? There are many mysteries in clinical trials, and people become confused and anxious about becoming a part of such trials. The overall objective of these activities is to discover or invent ways to more effectively prevent, treat, and diagnose the disorder. For example, all the drugs that doctors apply for treating cancers were first passed through clinical trials. There are unlimited myths about these trials. 

Phases of Clinical Trials

Clinical professionals conduct a clinical trial only when there is a cause to believe that a new drug or medicine can improve the way of standard treatment and cure the patient. Before clinical trials, there are some pre-clinical assessments. It does not involve people. It only assesses to analyze the attributes and characteristics of a treatment or test. For example, it may discover whether medicine is harmful for living tissues or not.
 
Another purpose of pre-clinical research may help to understand the chemistry of the drug. After successful completion of this step, the drug passes through a series of clinical trials. Its purpose is to assess if the medicine is safe for people or harmful. Following are four phases of clinical trials. Let’s discuss it one by one. Before going to phase-1, there is a small step in which clinical trial proceeds among the people. It aims to understand how the body processes a particular drug and what kind of effects it produces in the body. It involves 10-15 people. Medical professionals give only a small dose of the drug for observing the changes and getting results.

Phase 1 Clinical Trial And Its Purpose

The objective of phase-1 is to identify the best dose of a newly synthesized drug that creates the fewest adverse effects. They give this drug to 15-30 patients. Professionals start by giving small amounts of the drug. They test it on a small number of patients. They give higher doses to other people and see the effects. They continue the process until they see the desired effect.
 
The medicine may be helpful for patients, but the purpose of phase-1 trials is to analyze the safety of a drug. After confirming that the drug is safe, the drug goes for phase-2 trials. It will let the professional understand the safety of a drug. If it has the fewest side effects and created no harm to people, it will be considered safe. Professionals will make sure that the drug is safe and then pass it for further testing and next phase trials.

Phase 2 Clinical Trial And Its Purpose And How It Differs From Phase 1 Trials

After confirming the safety of a drug, it passes through phase ii trials. The second phase assesses the safety and workability of the drug. It has two functions. It analyzes whether a drug is safe or not and checks its usability. The doctors will test this drug among people with specific kinds of cancer. This phase of trials involves larger groups of patients as compared to phase-1 trials.
 
These tests involve new combinations of drugs. Doctors closely watch the patients and see whether a drug is working or not. They record different symptoms of the disease before and after the consumption of the drug. They notice the changes and make a chart to compare them. It helps them understand the workability of a drug. If a drug creates positive effects and shows that it is working, they process it for phase-3 trials. If it shows no usability to treat some disease, they discard it at this stage.

Phase 3 Clinical Trial And Its Purpose How It Differs From Phase 2 Trials

In phase iii, they compare the current medicine with the standard-of-care drug. This phase of trials analyzes the side effects of each drug and sees which medicine is working better. This phase takes help from 100 or more volunteers. They often randomize the trials. For example, they put patients, by chance, into a treatment group called trial arms. The purpose of such randomization is to ensure that patients in all the treatment arms are similar. It helps the clinical professionals to know that the results of different trials are due to the application of the drug and not because of the differences between the groups. They often use a computer program for randomization. It assigns people to different treatment arms. 
 
In phase-3 trials, there may be more than two trial arms. There is a control small group that gets the standard-of-care treatment. The other groups are treatment arms, and they get treated with a new drug. In this phase, neither your healthcare professional nor you can select your group. You will not know your group until the trial is over. The healthcare professionals watch every patient in the phase-3 trial closely. They can stop the study early if they find severe side effects due to the new medicine. They can also stop the research if one group has much better results. A drug needs phase-3 clinical trials for getting approval from the FDA. After this approval, it will be available for the general public.

Phase 4 Clinical Trial And Its Purpose How It Differs From Phase 3 Trials

Phase iv trials starts after the approval of a drug from the FDA. They test newly developed drugs among people. They test it in several hundred or thousands of people. It helps to conduct better research on long-lasting and short-lived side effects of a drug. It also performs research to analyze the safety of a medicine. For example, some unusual side effects may occur in large groups. This phase also lets the doctors how well a medicine works.
 
This is the closing trial, and after this, the significance and workability of a drug become clear and visible. They see all the recorded data during four phases and compare them. They conclude whether the medicine has proved significant or not. They conclude everything about the drug and make it available for the general public.

Should You Join a Clinical Trial?

All the clinical trials are risky. We must know that any medical treatment, procedure, or test has risks. We should know that risks are higher in clinical trials because there a lot of unknown things. Phase 1 and 2 trials are highly risky. This is the reason that it involves fewer people. Let’s discuss whether we should join a clinical trial or not.

Benefits of Clinical Trials

It is commonly observed that everything has some advantages and some disadvantages. Similarly, clinical trials also have. Their advantages may include that you can have access to new treatments. It may help you get lesser side effects as compared to the standard procedure of treatment. You can have the facility of regular testing. Some people consider this opportunity reassuring. When you are cooperating with a research team during their clinical trials, they will give you a research nurse.
 
This will help you to improve future pancreatic cancer. You will get exceptional respect among the people and research team. It may help you get free treatment for some specific disorder. You will feel proud that you have contributed to saving lives in the future. It will create a feeling that you are playing an active role in healthcare. It will help you access a treatment procedure that is not available yet.

What Are the Risks Involved?

There are many risks for people who participate in a clinical trial. We have discussed that there is randomization, and patients don’t know which treatment they are getting. It may also happen that some medications may work for others but not for you. During phase-1 & 2, you may have to face severe side effects. It may happen that your insurance company may not cover all the costs.
 
We have discussed that doctors will closely monitor the patients, and they will come again and again. They will conduct various tests and examinations to analyze the effects of a particular drug. You will have to answer various questions of a doctor. You may have to travel and spend more time visiting the doctor’s office. It will create a lot of uncomfortable circumstances for you.

Patients Safety

The safety of patients is much important. There is no perfection in the history of clinical research. After many years of experience and learning, Congress has passed laws to protect the patients who participate in the clinical trials. Now, it is the responsibility of every clinical and medical researcher to monitor and ensure that all the participants are safe and sound. The safety of the participants has become an essential part of the research.
 
There are some strict rules that researchers have to follow to make sure that all the participants are doing well. The federal government enforces this legislation. An Institutional Review Board (IRB) at each study site should approve and accept every clinical trial in the United States. This IRB consist of doctors, researchers, and laypeople. They work with dedication and ensure that the lives of the participants are not exposed to risks. The people of IRB keep an eye on the results and outcomes of the study. They make sure that participants are safe.

FAQ About Clinical Trials

Like all other fields, there are some frequently asked questions about clinical trials. People are curious about how the procedures run. Following are different FAQs about clinical trials.
 
Is There Any Advantage Of Participating In A Clinical Trial? 
 
Answer: Participants get free treatment, and they feel proud that they are contributing to the well-being of humans. They get a cure for some disease that is not available yet. 
 
What Risks Can Happen If I Participate In A Clinical Trial?
 
Answer: You may get severe side effects. It may happen that medicine does not affect you but affects others. It may help you recover from some disorder. You may have to face inconvenience due to many tests and examinations by doctors.
 
What Happens During Clinical Trials?
 
Answer: It is a clinical study that helps to get an answer for specific health issues. Medical professionals devise some medicine and pass it through a systematic procedure to understand the mechanism of its workability. They find out whether a medicine is effective or not. 
 
Who Conducts And Sponsors Clinical Trials?
 
Answer: Doctors, medical professionals, nurses, and other research teams from healthcare workers conduct clinical trials. Academic medical centers, social organizations, medicine companies, and governments of the country may sponsor these informed consent trials.
 
Why To Perform Clinical Trials?
 
Answer: It is essential to check the significance and workability of medicine of medical procedure. It helps to collect information such as  side effects or positive effects of a medicine. It helps to identify whether a drug is safe for use or not. Without clinical trials, no medicine can get FDA approval.
 
How Much Risk Is There That I May Get A Placebo?
 
Answer: A placebo is generally a pill or something else that does not produce any therapeutic effect. It is a general consideration that most clinical trials involve placebos. If you are getting a placebo during a clinical trial, you will be informed afterward. They will consider you only when the trial is safe and medically approved. 
 
Are There Any Charges For Participating In A Clinical Trial?
 
Answer: Generally, there are no charges for volunteers participating in a clinical trial. Most clinical trials are sponsored. They bear the charges for all kinds of testing that they need to perform during trials.
 
What Are The Criteria For The Eligibility To Become A Part Of A Clinical Trial?
 
Answer: All the clinical trials have set some criteria and guidelines for participating in a clinical trial. The factors that researchers consider for participating in a clinical trial include gender, age, stage and type of your disorder, records of latest treatment, and other health conditions.
 
When Can I Quit Or Get Out Of A Clinical Trial?
 
Answer: You can watch your conditions from the very first day and see what changes are occurring in your body. You may quit after finding any abnormality or uncomfortable situation. 
 
What About Children? Can They Participate?
 
Answer: Yes, children can participate in a clinical trial. Many pediatric patients have participated in such researches. They have made remarkable contributions in the discovery of treatments for pediatric cancers.
 
What About The Safety Of Clinical Trials?
 
Answer: All the clinical trials are monitored by an Institutional Review Board (IRB) to ensure that the current drug creates the least side effects and is safe for clinical trials. Doctors continuously visit and examine the participants. They keep a close eye to prevent them from any major problem.

Terms For Clinical Trials

What Is Meant By Trial “Phase”?
 
Answer: There are some steps of clinical research. Each step is known as “phase.” Each phase answers a separate research question. For example, phase-1 trials check whether a drug is safe or not. Phase-2 trials calculate and see whether a drug is effective or not. Phase-3 trials test the newly developed medicine against the previously existing medicine. Phase-4 trials try to identify the effectiveness and long-term effects of a drug.
 
What Is “Standard-Of-Care” Or Standard Therapy?
 
Answer: It is a specific treatment that experts of breast cancer consider appropriate and accepted. Healthcare workers must follow the standard therapy. 
 
What Do You Mean By Investigational Treatment?
 
Answer: When doctors are testing some drugs in people to identify their effectiveness among people, this treatment procedure comes under the definition of an investigational study. Such drugs have not got any approval from FDA. They are passing through series of steps and will get approval after passing these trials according to standards.
 
What Is The Significant Value Of The Control Group, And Why We Need It?
 
Answer: In each research, there is a standard that researchers use to compare. For example, during clinical trials, there may be a control group that will get standard therapy, and others will get investigational therapy. They compare the results of both the groups and conclude according to the results.
 
What Is The Need For Randomization During Clinical Trials? 
 
Answer: Randomization ensures that participants may go to any group by chance. Its purpose is to prevent biases in clinical research. 
 
What Is Meant By Randomization?
 
Answer: In a randomized clinical trial, researchers assign different groups to participants. They use a computer program for randomization. It helps to avoid biases because it assigns different groups by chance. Participants may go to any group.
 
What Is The Difference Between A “Blinded” And “Open” Trial?
 
Answer: In a blinded trial, participants may get either a test drug or the approved medicine randomly. They may get either of both by chance. On the contrary, in open trials, both the doctor and the patient know about the type of medicine. They know whether they are using a test drug or a previously existing drug.
 
What Is The Definition Of A “Crossover” Trial?
 
Answer: In this kind of trial, the participant has to get both kinds of drugs. For example, they will ask  people who take part to take a medicine-A for 6 weeks and then take another medicine-B for the next 6 weeks. This kind of trial is a crossover trial.
 
Placebos
 
What Is A Placebo?
 
Answer: A placebo is an ineffective and inactive treatment that looks like an approved drug. The way of its usage is also similar to the previously existing drug. During clinical trials, there is a comparison of the effects of an approved drug and a placebo.
 
Is There Any Use Of Placebos In The Treatment Of Breast Cancer?
 
Answer: There is no use of placebos in the treatment of breast cancer. Cancer patients only get a standard-of-care or newly developed drug. 
 
There are innumerable questions that different people ask about clinical trials. They may have different categories and classes. Participants may ask different questions, whereas professionals ask different ones. Different questions have different significance and origins. We have listed few questions that different professionals and participants frequently ask.