As the pandemic shifted cocktail hour onto zoom and professors posted their full course material online, many rare patients reveled. Not because we weren’t suffering through the pandemic too, but the safety precautions are even more pertinent for rare patients, who may be at higher risk or just eager to avoid an unnecessary bout of illness. But while the rest of the world adapted to a daily schedule based from home, many patients with chronic needs were already well acquainted with participation from a distance. Disabilities advocates saw decade-long pursuits achieved as the world settled into life online and made the rest of life increasingly accessible online. As researchers scrambled to maintain gathering uninterrupted research, drug trials were reimagined and out emerged decentralized clinical trials (DCTs): trials that made research remote, providing patients with technology to engage with the lab from home.
Rare disease scientists and patients saw how this reconfiguring of research is more necessary for rare patients than anyone, whose low and dispersed patient groups make conducting any research enormously difficult. Participants are often expected to fund themselves to travel great distances, in order to interrupt their lives with potentially invasive medical procedures. DCT’s revolutionize what taking part in a clinical trial can look like, equipping patients with their own monitor technology and communication lines which collect and channel data back to researchers in real time.
While bringing data collection home is a small silver lining after a tough pandemic, the scientists contend—this shift is not a subpar corona substitute. Bringing research home is the way of the future.
As reported in Clinical Trials Arena; at the virtual OCT West Coast Conference, medical experts unpacked how DCT’s gather more precise and accurate data by greatly increasing the eligible patient pool by eliminating financial and accessibility barriers, and by gathering consistent information from the patient’s day to day routine.
“DCTs are the future of all research,” explained Haley Arellano, associate director of Precision for Medicine. “Specifically, rare disease research and [it] is something all companies should consider embracing and working on building into their trials.”
Why It’s Hard to Research Rare Disease
Many rare patient groups know the costs their sparse numbers have on their pursuit to understand the disease. Big Pharma rarely go down the path of rare drug creation, all too mindful that the customer base will not cover costs. Pursuing rare drugs takes the force of patients and loved ones to push the science the research, and even then, the small communities face layers of unique barriers that make it difficult to gather a diverse enough group of patients to capture a full picture of the disease.
Often rare research requires participants to travel to doctors across the world on their own pay check. The burden of such a trip is often too much to take on, deterring some, and making participation inaccessible for others. These time and financial costs change the nature of who partakes in studies. However, any barriers create biases in the data since it likely effects some demographic groups more than others, such as women or people of color or the most disabled, who might have a harder time making the trip. Ensuring data collection includes everyone is pertinent to creating solutions for everyone.
Nagaraja ’Sri’ Srivatsan, senior vice president and chief digital officer of IQVIA, said,
“The decentralized model works very well for a smaller patient population that might be spread across the world and cannot come to the sites, which is often the case for rare disease patients.”
To create inclusivity for people of all abilities requires making participation as easy as possible, in order to reach the greatest number of patients possible. Real data comes from information on the whole array of patients, not only those able to stomach international travel.
“All in all, accessible research means a more comprehensive study population which means better data,” explained Arellano.
Better Data
In order to move trials home, researchers began to gather data using ever-cheaper monitoring technology, often through apps and other telemedicine. By providing a continuous story with information gathered from a patient’s day-to-day, a more representative and accurate picture develops of their symptoms and lived experience. Any tools are shipped directly to patients and made easy to use, eliminating the need for travel to a nurse or healthcare worker. This means monitoring is integrated into daily routines of whoever is interested in partaking, meaning data is gathered on the wide array of routines.
“Decentralization gives a better insight into the patient experience by collecting data in their real-life environment – when they are running errands, taking care of children or exercising,” said Arellano. “You are capturing data that is actually applicable to their day-to-day life.”
The physicians collecting data from lived experiences and the monitoring technology collects much more information than before. Wearable technology like watches and patches track vitals, sleep patterns, posture, or whatever symptoms most relevant. Trips to the doctors are eliminated by eDiaries, in which patients fill out questionnaires from home. Srivatsan explained,
“[Decentralized trials] also really help with the data acquisition component, which includes diaries, biometric data and connected devices. These are very important in rare disease situations as there are a lot of things that must be managed and monitored and you can start to take a lot of that diagnostic data from home, which enables you to see trends and patterns.”
These changes simultaneously collect a much greater amount of data, and are vital for reducing the burden of travel on the individuals. They also provide an extra incentive for patients, who are often interested themselves in their own data and vitals providing them a window into their own health. Luckily, bringing trials homes doesn’t mean compromising what type of data can be collected. Arellano said,
“Typically, when we think of home health we think blood draws and vital signs. But home healthcare providers can actually do a lot more specialized testing in patient homes including spirometry, ECG, EEG, telemedicine-guided physical exams and in-home infusions.”
Better Compliance
By making participation more accessible, patient are shown to increasingly comply with the demands of the study. At times, patients might not be able to visit the lab or might forget to do their part, invalidating their results. This is particularly common for rare disease trials and can be particularly damaging since small patient groups mean every patient is a valuable data point. However, DCT’s reduce the risk of damaged or incomplete data with app reminders and automatic data collection. The technology and doctors can keep track of patients in real time and correct any issues before the patient’s data becomes invalidated. Since partaking is that much easier, a simple reminder can make a difference.
Better Communication
This information is not only gathered more easily, but it then can be accessed immediately by the healthcare practitioners. This means any changes or fluctuations in a person’s vitals are noticed in real time, and physicians can contact patients to inquire about what might be at play. Having real time access to one another allows doctors to catch any hiccups.
“Once you put the infrastructure to engage the patient, there are lots of mechanisms you can put into the patients’ homes to start getting more and more rich data and information to engage them better and detect changes earlier.” – Nagaraja ’Sri’ Srivatsan
Making more trials remote means greater inclusion of the disabled community at large in data collection. Rare patients should be part of research for more than just rare drugs. Decentralized trials open up accessibility, in time, allowing rare voices be heard.
