Introduction to Clinical Research:
What is Clinical Research and What are Clinical Research Trials?
Clinical research trials are an important part in drug development and medical devices.
In order for a medication to be available for physicians to prescribe or over the counter, they must pass extensive checks that are put in place.
The Food and Drug administration as well as other regulatory agencies, have to ensure that medications meet efficacy and safety requirements before they can be sold.
Even though medications are first trialed with animals such as rodent and monkey studies, they must also go through human trials if the end recipient of the drug will be a human.
Not only must the drug be proven to work but it must also be proven safe for a diverse population and demographics.
If you’re interested in what clinical research trials are, keep reading to learn about the different phases of clinical trials!
Please keep in mind that everything in this blog is written as if I was explaining it to a friend. You should always verify that this information is accurate for your region and check with your doctor before proceeding with any type of new/investigation medication.
In order to fully understand what clinical trials are and how to participate in clinical research lets go over the phases in clinical research studies!
Phase I Clinical Research Trials
The first part of human trials are phase one trials. These trials are conducted at clinical research sites such as hospitals, private clinics and universities.
Since this is the first time that the drug or medication will be used by a human, these trials are done on healthy individuals.
People can volunteer to trial medications in exchange for monetary compensation for their time and efforts.
For phase one, people who wish to participate are screened for the study based on different parameters that might include or exclude them. This can include medical history, family history as well as lab and other diagnostic results. There can be questionnaires, assessments and other requirements involved.
This step is done for every trial regardless of the phase they are in, in order to find a group of people who can participate in the trial with minimal variations in the results.
Once the willing participant is deemed a good fit for healthy trials, they will be asked to give permission by signing consents. This officially starts the clinical trial participation!
The participant or subject will now be asked to follow the protocol or rules that were written out by the scientists and physicians who are developing the medication. This might include guidance as to how many and how frequently the visits to the clinical research site must occur. As well as what treatments and medications should not be taken while the subject is participating.
The participant is followed closely to ensure that the medication does not cause any side effects or unwanted changes. Since this is the first time that a medication is trialed by a human, many of these studies require inpatient visits. Meaning that the participant will have to stay overnight for a predetermined amount of time. This could range from a few days to weeks, depending on the protocol and the study that the participant signed consent for.
The research site will have accommodations for the participants to sleep, eat, and shower with the participant being allowed to bring in their belongings.
This allows for the trial to be standardized and for qualified clinicians to perform frequent checks and assessments on the participants. Doctors and nurses are equipped to handle issues that may arise and to report these to the pharmaceutical company.
Most medications that are available today, can cause side effects which can differ from person to person. If you have ever wondered where the list of side effects on pamphlets or commercials come from it is from these types of trials. The clinical research sites must report anything abnormal to the pharmaceutical company and disclose it to the FDA for approval.
If any of the criteria are not met, the subject decides to no longer participate or the physician and research site decide the subject is not a good fit. The study participant is able to stop their participation in the trial at any time.
Phase II Clinical Research Trials
If you are interested in participating in clinical research and suffer from a health condition, you could potentially participate in trials that have already passed their first phase.
Phase II trials focus on identifying the efficacy of a new drug. If it passed Phase I that means that it showed to be safe based on the standards set by the FDA or local regulatory agencies.
The next step for a drug or medical device is to test for efficacy or to test if it works. The pharmaceutical company and its scientists have theorized on the mechanism and how it could potentially benefit patients but it has to pass the test.
An example would be a new medication for blood pressure. This would be tested in animals and different biomarkers would be checked. If this passes specified standards it will be approved to proceed to human trials. Once it passes Phase I and there is enough data to show it is also safe in humans, Phase II will include patients who are hypertensive. As opposed to Phase I where the participants must not have health issues. Phase II requires that the participant has hypertension in order to see what results it has. Will it lower blood pressure for enough participants? Does it only work for a specific population?
The pharmaceutical companies will recruit participants who have the issue or “indication” but will also need to meet other requirements in the inclusion and exclusion part of the protocol. If the participant has a health condition that could be aggravated by the drug, they would be disqualified from participating for example. Or the participant might be on a medication that can react to the new drug’s active ingredient which would also disqualify them.
Once the participant has been screened for the study and meets all of the inclusion and exclusion criteria, they will be asked to proceed with the study. The participant will be instructed on what they should do moving forward concerning medication instructions, clinic visits and what to do if they need to report anything abnormal.
These trials can last from weeks to years, and the time commitments can differ depending on the medication. The participant can decide to stop participating at any point or for any reason from any trial as it is always voluntary.
Phase III Clinical Research Trials
Once enough data is collected from Phase II trials the drug will be approved to proceed into Phase III.
This is the last phase before it is brought to market! Phase III is concerned with the difference between the new medication and other treatments that are already available.
Oftentimes, Phase III trials will have participants take either a medication that is already available on the market or the new study medication. This is decided randomly known as randomization, and for the majority of trials will be blinded to the participant and study staff. Meaning that neither the participant or the doctors and nurses know what the subject is taking.
This helps to prevent any type of bias or placebo effect that can interfere with the study. If there is a reason for needing to “unblind” the study staff and participants there is the ability to do so. This is done if there are any type of side effects or interactions that the subject might need treatment for.
Phase III trials can also vary in length and also recruit people who have the disease process for which the medication was designed for. This data is used to compare, as well as provide additional data on the efficacy of the drug and to determine if it might be better or safer than the current standard of care.
Phase IV Clinical Research Trials
Once the medication has passed through Phase I, II and III, the drug is ready to be sold. The medication will be prescribed or sold over the counter. It takes the pharmaceutical companies years, lots of money and the combined efforts of participants and site staff to get these medications out to market. All of this with the hopes that it will improve patient lives and help prevent, cure or treat an existing condition.
Even though drugs at this stage have been proven to be safe and effective. The FDA must still keep a close eye on any reported safety issues. The medication will be widely prescribed and if issues arise it can always be taken off the market or reevaluated.
Who can participate in clinical research trials or clinical research studies?
Anyone who meets the requirements for the specific clinical research trial, is able to participate if they are willing to. Participation in clinical trials must be voluntary and at the discretion of the participant, their physician and study staff. You need to evaluate how your current medical condition is being handled and whether an experimental medication would be worth a shot.
Whether you participate in clinical research or not needs to be evaluated by yourself on a case by case basis alongside your healthcare provider. If your current medication is working there might not be a benefit in attempting an experimental medication. Your physician can help you determine if you should participate in clinical research studies.
Why participate in clinical trials or clinical research studies?
There are many motivations for participating in clinical trials. The biggest motivating factor is helping advance science and healthcare. There are many clinical trials that might or might be of benefit for yourself but can help many others down the line. Other trials might help to treat your current condition and once available it could be a treatment your doctor prescribes for you.
For many it can be a great feeling to know that you are helping in something way bigger. That you are making a difference in volunteering for clinical trials.
Even though the subjects in clinical trials can also be referred to as volunteers, the majority of clinical trials offer monetary compensation. This can be another motivating factor for people who want to participate in clinical trials to make money. Health individuals might want to make money and get paid to participate in clinical studies. Even though this should not be the only motivating factor as people should think hard before participating and you should have other intrinsic motivators of wanting to help.
Conclusion
Clinical research trials take place in order to bring new and improve treatments to market. Healthy individuals participate in the first phase of clinical trials and once it is tested for safety it can proceed to be tested for efficacy in patients. The most simple example of this is a hypertension medication that is first tried in healthy individuals to make sure there are no unwanted effects. Next it can be tried on patients who suffer from hypertension and are willing and able to participate to trial the medication. If it works in patients with the illness or disease process it can then be compared to the current treatments. Once the medication advances through this process it will be brought to market. The FDA still continues to monitor for any adverse events or side effects.
This is however, a very simplistic way to look at the whole process which can take years and millions of dollars! Most of this will go to clinical sites and the subjects who chose to participate are usually compensated as well. This is for their time and for volunteering to take an experimental medication. This is not the only motivating factor when participating as many people enjoy contributing to science and advancing healthcare.
Hopefully this article was helpful in providing a simple breakdown of what clinical trials are and how to participate in clinical research!
This was so informative, thank you so much!!
You’re so welcome, I’m glad to hear!