Behind the Scenes of a Clinical Trial

We know that clinical trials are an essential part of the drug development process, but what do they actually entail?

We spoke to two patients who have volunteered in several clinical trials for atopic dermatitis (AD) treatment as well as two full-time researchers, one of whom is also a parent of a child who participates in a clinical trial. Together, they paint a full picture of what to expect of a clinical trial – before, during and after.

How do you find a clinical trial (or how do they find you?)

Some patients look for trials on their own, while others hear about them from their doctor – or through advertisements.

Tristen K., age 21, lives in Texas and found out about a clinical trial after doing some research online about his eczema. “A local dermatologist had a sponsored ad on Facebook for eczema patients interested in a clinical trial,” he said. “From there, I looked up the dermatologist’s office, the doctor, as well as the clinical study.”

Prior to finding this study, Tristen said he hadn’t known anything about clinical trials, but he was interested in learning more about how to treat and manage his moderate-severe eczema. “I knew nothing going in. However, the staff walked me through every step.”

Because many clinical trials take place at a hospital or doctor’s office, it’s often convenient for researchers to look within their own medical network for patients that might benefit from a trial.

Lindsay Chandler works as a clinical trial administrator at Oregon Health and Science University (OHSU) in Portland, Oregon. Chandler explained that trials can be especially valuable for patients who have not had success with existing treatments. In some cases, she said, “We may be able to recruit patients who were denied treatment for a variety of reasons.” Within a hospital network, Chandler and other researchers might identify “a patient who may be suffering, or a person who might not have been qualified with insurance to obtain treatment.”

Traciee T., age 54, lives in California and heard about an open clinical trial from a nurse at her primary healthcare clinic. She signed up to be notified if any trials opened up that might work for her. “They called as soon as a trial came up that fit my issues,” Traciee said. “Atopic dermatitis is such a head scratcher, and most treatments can be frustrating and costly. To be able to get free meds, great hands-on care, and a little compensation for your time can be beneficial in the long run.”

What happens when you get there?

The first step is filling out all the paperwork. Patients are screened based on the information they provide about whether the trial is right for them and they are right for the trial.

Silena Gaines works as a senior clinical research associate at PRA Health Sciences in Columbia, South Carolina. “We want to be sure patients have read through everything carefully,” said Gaines. Before anyone officially joins a clinical trial, they have to fully understand the purpose of the research. “We want to be sure that the benefits outweigh any risks. We review what will happen and ensure that their primary physician or other doctors are informed about their participation in the trial.”

For a long-term study about a complex condition like eczema, a prospective patient fills out their entire medical history to ensure they are a good fit for the trial’s objectives. There are inclusion and exclusion criteria for the study, meaning who might be qualified for the study versus who might not be the right fit.

The inclusion and exclusion criteria could involve many different details, such as:

• the age of the patient
• the severity of their eczema
• the age when they were first diagnosed
• the type of flares they experience
• whether they have any other medical conditions
• details on any medicine they’ve taken in the past
• details on which treatments have or haven’t worked for them previously
• and any other criteria the investigators are studying.

The study doctor will also review a patient’s current medications to ensure that nothing will conflict with the study. The net goal is to find the right patients who will potentially benefit from the right trial. And once the patients have been enrolled, the next step is getting started – either with the treatment or a placebo.

You’re in the trial – now what?

Once you’re in, you get all the information you could possibly need. Gaines calls this “the gold standard of care.” The study team, including the trial recruiter, nurses, research clinicians and primary investigators will meet with the patient. Together, they discuss all the key points of the trial the patient will need to understand. This includes where the patient will receive the medicine, how often they come in, any notes they need to take at home, any risks or possible side effects, and a dedicated phone number for the patient to call with any questions.

The researcher will also counsel the patient on starting a “wash out” period, a period of time when the patient stops taking any medications that might invalidate or interfere with the treatment. The wash out period allows the provider to establish a baseline for treatment.

Establishing baseline lab tests is also critical before the trial can proceed. The researchers need an accurate picture of the patient’s vital signs, heart rate, blood chemistry, weight, height, severity of symptoms in the skin and anything else that may change over the course of the treatment.

Gaines and Chandler both noted that patients receive thorough training about what they should watch and observe in their own bodies as a part of the trial, along with contact information for the trial site coordinator if a patient has any concerns during the trial. Chandler pointed out that patients enrolled in trials she has coordinated are provided with a dedicated phone line they can call any time.

Gaines, as a researcher, explained what a typical workday looks like for her. “I’ll go to the site where the trial is being conducted and meet with the principal investigator. I want to be sure the trial is following the protocols, see if they need help recruiting, are they ready for any audits, do they have the necessary supplies?”

Gaines has the unique experience of not only being a research associate but also the mother of a young child who is in a clinical trial for severe eczema.

She was thrilled to have the opportunity to enroll her child in the study. “As an eczema parent, it’s traumatic: you’re willing to do anything to help your child get relief.” Gaines’s son was able to enroll in an ongoing study for atopic dermatitis in preschool-aged children. “You get to see in real-time if your child is healthy,” she said. “You know you don’t want your child to suffer, so this has been a blessing for us. We would have gone across the country. He’s happy, not scratching, interacting with his peers and he enjoys going to school again.”

For adults and children too, the experience of participating in a trial may feel like just another visit to the doctor’s office. “I tell my son we’re just going to the doctor,” she said, about bringing him to his trial appointments. “He doesn’t mind getting the labs done, but he doesn’t like having the injection. Showing him pictures helps and taking the time to explain. I remind him that he always feels good after. Sometimes they put lidocaine on the injection area, so the shot doesn’t hurt so much.”

Of course, not everyone will experience improvements in their condition and some will get the placebo.

What the experts wish more people knew

Still, the experts agreed that too many people focus on the possible side effects and don’t realize how safe clinical trials really are. “I know the idea of side effects can be intimidating, especially for a parent,” said Chandler. “We’re on high alert for any adverse event as soon as possible. And our principal investigators, the doctors, are very hands on, very involved.”

Traciee said that a common misconception about clinical trials is that the “participants are like guinea pigs.” Tristen, too, pointed out “the heavy vetting process” he went through before joining the trial and that he could have dropped out any time.

Chandler expressed her consistent surprise at how many people think they have to pay in order to participate. “People are often under the wrong impression about trials,” she said. “Research, to some people, is code word for expensive. It’s the opposite. The medication and care in the trial is covered by the trial sponsor.  And when we have a treatment that’s extremely effective, the patients are over the moon grateful. It makes such a difference in so many patients’ lives.”

Tristen echoed the same sentiment about the financial element of trials. “Any time I had to make an office visit, I received $50.” Traciee was compensated for her participation, too, though not for transportation. She had to drive under 20 miles, whereas Tristen had to drive further and received compensation for his mileage.

What happens when the trial is over?

At the end of a successful trial, patients may be given the opportunity to participate in what is called an “open label” study. At that point, all participants would know they were getting the drug rather than placebo. There are also opportunities at this stage to continue on the drug for a set period of years, and potentially to have it paid for by the pharmaceutical company.

“It would have been hard if it was the placebo,” said Gaines, about her son’s trial. “But we have the open label option. If he qualifies, he’ll get the roll over for the study drug after five years. This happens sometimes if the drug is already approved in that therapy area.”

Some parents find it helpful to share their experience with other parents whose children might benefit from a trial. “I let the other parents know,” said Gaines. “People want to understand what a clinical trial is. They want to be a part of it. They want to know the data that’s been released on these particular drugs. You make short term sacrifices for long term benefits.”

Chandler called the benefits of clinical trials “a wonderful gift to patients.” She also pointed out that “one of the positives of Covid-19 is that people are finding out about clinical trials and realizing how important they really are.”

Gaines emphasized the importance of asking questions. “If I could tell prospective participants anything it would be that trials are not scary. Don’t be afraid to ask your doctor if there are other treatment options for you. Be proactive about your health – it’s an exciting time for medicine!”

Chandler also noted that “subject participation is a precious commodity. It means so much to our team at OHSU and the scientific community in general, as it aids in the global mission to find cures and improve health outcomes.” She continued: “we’ve found that patients derive satisfaction not only from their own treatment journeys, but also from the knowledge that they, as individuals, are contributing to the greater good. Because of the data that they are generating, researchers and scientists are able to advance major developments in treatment.  Clinical research is an ever-changing landscape of new ideas, and we strive to bring that feeling of excitement and inclusion to the public.”

There are many eczema drugs in development. If you are considering participating in a trial, speak to your dermatologist or primary care physician. You can check here for a trial near you.