3 ways decentralized clinical trials could help advance research on rare diseases

Bloomington, MN – Pandemic-era changes may have permanently altered the clinical trial landscape as decentralized clinical trials gain broader acceptance. During COVID-19, more than three-quarters (76%) of pharmaceutical companies, device manufacturers and contract research organizations conducted off-site clinical trials. These types of trials specifically provide increased opportunity for individuals with rare diseases to participate as they reduce numerous barriers of traditional trials.

According to the National Organization for Rare Disorders, rare diseases affect 1 in every 10 people in the United States. And yet, only around 5% of 7,000 rare diseases identified have an FDA-approved treatment. To develop therapeutics that target rare diseases, called orphan drugs, clinical trials are essential. Historically, conducting trials in this area has been challenging, given the complex biology of some of these uncommon conditions, coupled with the small patient populations, which may be spread out geographically.

But the new momentum of decentralized clinical trials continues to drive the market, which is projected to have a compound average growth rate of 14.8% through 2027. Lynsey Psimas, Ph.D., director of sales and pharma services at Pearson Assessments US, said that this acceleration could bring new hope to countless patients and families affected by rare diseases.

“A silver lining to the pandemic is the way healthcare organizations everywhere are now democratizing their approach to patients, as is the case with decentralized clinical trials,” Psimas said. “With the help of technology, clinical trials are reaching broader patient populations everywhere. That could be a game-changer for researchers focused on rare diseases, who, in the past, have had to contend with barriers that often felt insurmountable.”

In recent years, digital health technologies have surged, with wearable devices, mobile health apps, telemedicine and an array of information technologies becoming a part of people’s daily routines. This evolution ushers in a new era for health-centered businesses working to put the patient at the center of all they do by meeting them where they are, said Psimas — including sponsors of clinical trials. Psimas shared three reasons the growing acceptance of decentralized clinical trials could help advance research on rare diseases and help patients who live with these conditions.

  1. Easier access for patients. Participating in a clinical trial can be burdensome even for healthy volunteers. For patients with rare diseases, it may be even more so if the trial involves extensive travel, if the patient requires caregiver assistance, if mobility is limited or if the patient is more susceptible to other contagious diseases. A decentralized clinical trial can ease all those burdens and allow a patient to participate in a study that is more accommodating and convenient. “Through the use of technology, a decentralized clinical trial truly puts the patient at the center of the research by allowing them to communicate information from the comfort of their own home,” Psimas said. “For patients with rare diseases, this could allow them to participate in trials that are hundreds or even thousands of miles from where they live. It enables them to contribute to research and hopefully, one day, benefit from breakthroughs that they helped make possible.”
  2. Improved diversity, equity and inclusion in research. For years, the biopharmaceutical industry has set goals for increasing diversity in clinical trials, but progress is still needed. According to the Food and Drug Administration’s Center for Drug Evaluation and Research’s (CDER’s) Drug Trials Snapshots Summary Report published in 2023, the demographic data of patients participating in trials for novel drugs approved in 2022 skewed white (80%), with just 4% Black or African American, 7% Asian, 8% American Indian or Alaska Native, and less than 1% Native Hawaiian or Pacific Islander. Further, women made up 45% of participants, and seniors 65 and older accounted for 33%. Easier access to trials could enable people from more diverse backgrounds and incomes to participate, Psimas said. “For people who have rare diseases, the importance of participating in clinical trials cannot be overstated,” she said. “For many of these patients, there are no therapies on the market and an investigational drug is the only hope. By removing common barriers to participation, decentralized trials can open up possibilities to participants, while also improving the diversity of demographic data for researchers.”
  3. Advances in data collection and analysis. Digital platforms allow patients to share real-time data with researchers, from biomarker insights gleaned from digital sensors to ongoing self-reporting via communications platforms. All this can happen without disrupting their usual daily routine, Psimas said. “When patients participate in traditional clinical trials, they’ve had to travel to a particular place at a specific time to give samples and share feedback, which can be quite burdensome,” she said. “However, in a decentralized clinical trial, when a patient shares information on a day-to-day and even hour-by-hour basis, researchers benefit from more data, which allows even more insights.” And because the method of communication is continual and convenient, Psimas added, it may also help improve patient engagement and boost patient retention.

Still, more data does not always equate to better data. To ensure quality data, the proper clinical outcome assessments (COAs) are needed, Psimas said. And in decentralized trials, electronic clinical outcome assessments (eCOAs) are key. Pearson Assessments, which has been in the assessment business for more than 80 years, creates assessments in a vast array of therapeutic areas that can be adapted and used in virtual administration on eCOA platforms. That, Psimas said, can save time and improve efficiency while also helping with patient compliance, virtual data collection and reduced errors in administration. “Pearson’s team of clinical experts offer scientific consultation to pharmaceutical companies to assist in eCOA selection, evaluating psychometric and normative data and more, to make sure that they are utilizing valid assessments and generating outcomes that are meaningful to patients with rare diseases who are counting on these breakthroughs to be safe and effective,” Psimas said.

Psimas said she believed that decentralized clinical trials offered new possibilities for people living with rare diseases. When designing the trial, she said, the proper protocols are essential to get medicines to market in a timely manner so they can benefit those who need them most. “Incorporating valid and reliable COAs into trials and enabling access to patients across the globe are critical steps toward ensuring the successful development of potentially life-saving drugs,” Psimas said.