Siteless Clinical Trials: Thinking Outside the Box by Putting Clinical Research in a Box
Clinical research is a long process that involves a series of studies that gives Food and Drug Administration (FDA) regulators the information needed to determine whether the drug is safe and effective — and ultimately whether it should be approved for use. Patient participation brings immense value to the clinical research process and without participants, there would be no trials, but it can be more work for the patient. Thoughtful, patient-centered study design can result in a study that is not only more pleasant for participants, but that will produce higher-quality data and quicker study completion. While many groups are thinking about ways to make their studies more patient-friendly, Science 37, the company behind this blog, is thinking outside the box by putting clinical research in a box. They have developed a model with “siteless” trials where all the study materials and drugs are boxed up and shipped directly to the patient, and the patient can dial their study team directly through a telemedicine app called NORA. Since the patient receives an iPhone for use during the study, they can use it to reach out via voice calls or email. This model allows a trial to be centered around the patient in the most literal sense — the patient can participate from their own home. Science 37 has worked for several years to contribute to parts of larger studies, but recently, they worked with AOBiome to complete the first start-to-finish siteless clinical trial, and that’s a game changer.
What Was the AOBiome Trial?
The AOBiome trial was a phase 2b trial for patients with mild to moderate acne. In the phase 1 trial, AOBiome determined a dose that was safe to give to patients. This phase 2 trial was designed to not only assess additional safety details, but also determine whether the drug was effective. Patients in the AOBiome trial were randomly assigned to one of two groups: one group received the study drug and the other received a placebo. Since one group of patients received the drug and the other didn’t, comparing the outcome of the two groups would demonstrate how well the drug worked.
How Did Science 37 Design the Trial To Be Different from a Standard Trial?
In a standard setting, the trial site is typically a clinic. Patients go to the site to visit a doctor, get screened, and then enroll if they are an appropriate candidate. Patients also have to visit the trial site intermittently during the course of the study for exams, to discuss how they’re feeling, and also to pick up medications to last until their next visit. On a standard trial, patients have to fill out surveys each time they see the doctor, trying to remember how they’ve been feeling since the past visit. With Science 37’s virtual trials, there are no physical trial sites to visit. All of the initial screening and consent processes take place remotely during a phone call, and the study medication is shipped directly to the patient’s home. An iPhone preloaded with the NORA app is loaned to the patient for the duration of the study. Now, instead of going to a clinic, patients can complete their study visits and have real-time direct contact with the study team through the NORA app.
How Did It Turn Out?
The trial sponsor, AOBiome, reported positive safety and efficacy data — the drug is safe, and it works! But what about the siteless part? Did the Science 37 model make any difference?
- It’s faster: After recruiting and screening 8,000 potential participants, Science 37 was able to enroll 372 patients in just seven months. That’s half the time it would normally take to fully enroll a similar trial. Faster trial completion means drug approval decisions can be made more quickly, and a good drug will be available to all patients sooner.
- More diversity represented: Most clinical research studies are made up predominantly of Caucasian participants — few studies reach even 10 percent minority enrollment, yet this clinical trial boasts 41% of study participants who are non-Caucasian! There is a whole host of potential reasons why minority participation is typically so low, and mistrust in the medical system and the fact that minority communities are often not located near major academic medical centers contribute to the disparity. Implicit bias is another factor, because patients who aren’t offered trials can’t enroll in them. Physicians do the same thing we all do — they use things they know to make generalizations to help them make quick decisions. It’s not meant to be unkind, it’s not even something they do consciously. But failing to offer a trial to a patient due to assumptions about their ability to meet the additional requirements of a clinical trial because of where they live, the kind of job they have, or their family situation, removes the patient from participating in personal health decisions. The Science 37 model overcomes many of these barriers — direct-to-patient outreach eliminates physician bias, the fact that patients are initiating participation reduces the fear that they are being preyed upon, and the fact that patients don’t have to go to a trial site allows them to participate regardless of where they live. Quadrupling minority enrollment is not only impressive from a numbers standpoint, but it improves the quality of the data. More diversity among the trial participants results in data that are a better representation of how a diverse population will respond to a new drug.
- Happy patients: I could talk all day about how this kind of trial is a game-changer and why I think it will accelerate drug development and improve the quality of data available. But really, I think the most poignant words come from those who actually lived this trial: the participants. Here is some of their feedback:
- “I would not have been able to do this study without it being a remote study, as I don’t have the time to schedule weekly or monthly visits to a physical office. I liked that I was able to do the activities outside of normal business hours. I also appreciated that my study coordinator was available to accommodate my odd hours and answer questions quickly, despite being on the other side of the country.”
- “The [best part of the trial was the] conversations with my [coordinator], we laughed a lot and had fun and overall great interaction.”
- On whether this participant would participate in another clinical trial: “I’m looking for another! lol”
The completion of the first start-to-finish trial using a siteless model is huge! This study will pave the way for the pharmaceutical industry to take giant strides in the development of patient-centered research. In the traditional model, participants would rejoice at “patient-centered innovations” like providing parking vouchers. Now, we have a model where making a study patient-centered means that the study literally comes to the patient and centers around their experience. Patient-centered research on this level will not only speed up the process and increase the diversity of clinical research, but it will benefit individual patients. Thinking this far outside of the the traditional box will change the paradigm of how clinical research should be done.