A Tale of Two DCTs: Lived Experiences of Both Patient and Caregiver
Jena Daniels

he global virtual clinical trials market is now worth $8 billion, according to Medi-Tech Insights, and is growing at a clip of more than 10% annually. It’s not surprising: Virtual or decentralized clinical trials (DCTs) got their kickstart during the pandemic and have gone mainstream. Are the benefits they promised being realized?

A new study from the Tufts Center for the Study of Drug Development shows that DCTs reduce development cycle times, lower clinical trial screen failure rates, and present fewer protocol amendments. These benefits yield net financial benefits ranging from five to 13 times for phase 2 and phase 3 trials, equating to roughly $10 million ROI and $39 million ROI (return on investment) respectively.

But even with these substantial financial benefits, it is imperative to consider the broader impacts DCTs may have on patients and their caregivers. DCTs allow more people to participate in clinical trials outside of the typical clinical site, enabling faster screening, convenient consent and enrollment, and, in some cases, the benefits of in-home delivery of the intervention and remote measurement of outcomes. DCTs ease participation burden by reducing time and travel costs, which can be especially important for patients who are seriously ill and the 70% of patients who live more than two hours away from a study site.

DCTs also allow recruitment of patients across a wider geographic range, which helps to improve population representation in trials. Since DCTs replace brick-and-mortar sites with digital/virtual technologies, there are no physical boundaries for participants and trials can recruit patients from everywhere for greater population representation. This is important because less than 5% of the US population participates in clinical trials, even though 85% say that they would like to. Statistics also show that racial and ethnic minorities comprise 39% of the US population but only account for 2% to 16% of trial participants. DCTs have the potential to help democratize trial access.

While the numbers are powerful, the real-life patient experiences shine the brightest light on the impact of DCTs. Here is a look at two different journeys: Christine, a patient suffering from a chronic rare disease; and Karen, a long-term caregiver for her disabled daughter. To Christine and Karen, the value of DCTs has less to do with dollars and diversity, and everything to do with living their best lives.

“My World Grew Big Again”: Christine C.

In 2019, Christine spent Thanksgiving weekend alone. Asthmatic and exhausted, she couldn’t find the energy to cook, visit family, or even hold a thought. “That was the end. I decided I was not going to live my life this way as it will only get worse with age,” she said. 

The January before, Christine was diagnosed with Sjögren’s syndrome, an autoimmune disease that primarily attacks essential moisture glands like salivary, sweat, and tear ducts. Common symptoms like chronically dry eyes and mouth can be debilitating, but the exhaustion that some patients experience is nearly incapacitating. This overwhelming fatigue drove Christine to her point of no return.

“It was a relief to be finally diagnosed, but also frightening to learn I had a critical, chronic illness,” Christine said. “At the end of the day, I needed a better way to manage my condition.”

With a professional background in the life sciences industry, Christine studied her disease on her own. She knew that relocating to Boston—a national cradle of breakthrough medical research—would raise her chances of finding a solution. She also found a supportive community through the Sjögren’s Association, which is where she first learned about ongoing clinical trials. However, when she tried to find a clinic, a timeline, or even a phone number, every lead fell short. She called her primary care physician and a rheumatoid arthritis specialist, but neither had even heard of a clinical trial for her disease.

Finally, a web search brought Christine to clinicaltrials.gov, where she found a trial for Sjögren’s hosted by the Tufts Center for the Study of Drug Development, an independent, nonprofit academic research center within Tufts University, located in Boston—right in her own backyard. 

Qualifying for the study was just the beginning. For Christine, finding the strength to get out of bed was a daily battle that she couldn’t predictably control. Travel to the trial site with no vaccine for COVID-19 yet available put her health at serious risk. Having Sjögren’s and contracting COVID-19 while traveling to and from the trial site would certainly have meant hospitalization, during the major shortage of ventilators in 2020 precipitated by the COVID-19 pandemic.

Requiring frequent in-person site visits can pose a serious risk of patient dropouts in many trials. In Christine’s particular case, commuting and traveling often triggered flare-ups of her worst disease symptoms. “I know that, especially for rare diseases, every patient counts in a study. I was worried about being able to follow through,” Christine said. “But I had to be honest about that possibility.”

Christine, considering the circumstances, was offered the opportunity to participate in a DCT. Initially, trial administrators apologized for asking Christine to use a mobile app in lieu of in-person visits. But for her, it made all the difference in the world. Using a leading DCT platform, Christine only needed to visit a physical site once a month and used a mobile app on her phone to capture the rest of the data including medication dosing, patient surveys, and regularly recording how she felt along the way.

“DCTs should be standard for all trials. When people have choices, the barriers to entry are just speed bumps,” said Christine.

 The “hybrid” DCT helped Christine stay consistent with her medication, which she said gave her newfound energy to embrace her life: exercising, socializing, and going to dinner with her son. She steadily lost the 20 pounds that she had gained while bedridden and no longer needs to see a specialist for her dry eyes.

 “I don’t feel like I’m pushing a rock uphill anymore. My world was very small, and then it grew big again.”

“Caregivers Can Make or Break a Trial”: Karen U.

Samantha was born with CDKL5 deficiency disorder, a rare neurodevelopmental condition caused by pathogenic variants in the CDKL5 gene. The disorder manifests in a broad range of clinical symptoms, including such hallmarks as early-onset, intractable epilepsy and neurodevelopmental delay impacting cognitive, motor, speech, and vision. Samantha’s mother, Karen, is Samantha’s primary caregiver, a 24×7 responsibility she has fulfilled since Samantha began having seizures at just 10 weeks old.

Two years ago, at age 13, Samantha started an investigational therapy as a participant in a traditional brick-and-mortar clinical trial. Remarkably, the drug dramatically reduced the frequency and severity of her seizures. In addition, for the first time in more than eight years, Samantha is eating most of her meals orally. None of these remarkable improvements would have been possible without the dedication of her caregivers and a trial protocol that leveraged DCT technologies.

“About halfway into the trial, COVID forced remote participation, which included telehealth site visits and ECGs and blood draws handled at centers close to our home. This dramatically reduced the burden on Samantha but also me as her caregiver and ensured continuation of the trial which ultimately improved my daughter’s quality of life,” explained Karen. “Before the trial went digital, I had to pack us up every three months (more frequently early on) and drive an hour to the airport, fly from Houston to Denver, taxi to a hotel, and spend hours each day in the hospital where the trial was being conducted. It was taxing for us both.”

“I am so grateful for the opportunity that trials have provided to my daughter, but she could not have participated alone. Trial participation can be completely dependent on a caregiver, so trials that reduce burden on caregivers are as important as ones that reduce burden on patients,” explained Karen.

Karen says the caregiver’s role is twofold. The first role is logistical, such as getting the patients to their clinic visits and keeping everything organized. Caregivers often encourage patients to participate in trials in the first place, too, and can increase the likelihood that patients will stay engaged even when feeling the effects of their illness. “Anyone caring for a chronically ill person will play a huge role in determining whether that patient continues to participate,” said Karen.

The second role is observational, e.g., catching warning signs that doctors and even patients may not see or report themselves. Both roles are essential with patients like Samantha and with elderly populations and others whose communication, mental capacity, and mobility may be impacted by their disease. Research is underway to compare how caregiver reports predict patient outcomes as compared to similar reports by patients themselves. Early evidence suggests they can increase the patient’s quality of life, decrease emergency room visits and hospitalizations, and even increase survival.

If a clinical trial depends on the logistical and observational capabilities of caregivers, as in Samantha’s case, success can be bolstered by DCT capabilities. “The more you support that caregiver, the more success you are going to have across the board,” concluded Karen.