A personal approach to the development of new treatments in rare disease
New ways to help clinical study participation and much-needed treatments that fit with people’s lives
By Gina D. Wagner
Finding new treatments for rare disease can change lives. Researchers, who are usually the most knowledgeable about the disease they are studying, are trying to bring these treatments to benefit as many people as possible. But getting access to those treatments isn’t easy for many, especially those who don’t live near the academic centers where the specialists usually practice. Studies can require travel to a distant study center over the course weeks or months, which can be a challenge for those who are working or have family responsibilities. Many patients may have to decline joining a study they are interested in, due to physical limitations to make a journey or concerns about exposure to infections.
Some researchers, including leading myositis specialist Dr. Rohit Aggarwal from the University of Pittsburgh Medical Center, are on a mission to train local researchers and specialists and collaborate with them to establish more centers of excellence in rare disease.
Thanks to technology, people today may be able to participate in a study for a new treatment, but not be limited by travel. Forward-thinking researchers are now designing clinical studies that allow participants to determine the frequency of their visits to a study center based on their daily schedules and lives. Here are some of the ways they’re accomplishing this:
Decentralized Care
Increasingly, researchers are now able to partner with local laboratories, pharmacies, hospitals, and medical offices to provide the services and care that participants would be getting on their visits to the study sites.
Telehealth and Home Visits
Before COVID, not everyone had reliable access to Wi-Fi and video conferencing. But a few years into the pandemic, a lot has changed. Our perceptions and openness to remote meetings and telehealth has shifted. We’re more comfortable doing things at home. As a result, some studies now offer the option for patients to participate via telehealth or home healthcare visits.
“I love that we’re doing hybrid models and we’re learning safe and effective ways to offer this without losing any quality in the research,” says Dr. Prince Adotama, a board-certified dermatologist and faculty member at NYU who specializes in bullous disorders. It’s safer to some degree to allow patients to stay home, especially those who are at higher risk for COVID and other viruses or who may not be as mobile. “They’re not alone. With home nurse visits, someone is still seeing them, interfacing with them, and administering the treatment,” adds Dr. Adotama.
Dr. Aggarwal is taking the lead on putting patients first in the design of his new study, the Myositis Interstitial Lung Disease Nintedanib Trial (MINT Study). “Some patients want to interact with the researchers on a more regular basis, while others want to avoid coming in as much as possible. Our study was designed so that participants can participate remotely, from the comfort of their own home anywhere in the US,” Dr. Aggarwal explains. This novel clinical study requires no visit to a study site at all, if that’s what patients choose. Alternatively, if patient prefers they can get study visits at one of the many clinical trial sites associated with the study. For people with myositis-associated lung disease who may have limited mobility and a fear of infection, not having to travel to a study site for multiple visits may be a relief.
Reaching Underserved Populations
Dr. Aggarwal may get his wish – since flexible clinical trials, like his, lower the barriers to participation from more diverse populations, accommodating job concerns, caregiving responsibilities, and transportation needs.
Some studies may also offer reimbursement for any travel that is required, and, increasingly, flexibility in scheduling, so that patients can work around job and family responsibilities. Also, studies often include free testing and diagnostics as well as free or reduced cost treatments.
Mobile Technology
As we become more used to using apps to schedule appointments, track health records, and fill prescriptions, we can become more comfortable participating in clinical studies using those technologies. Some trials use wearables, like smart watches, to provide real-time, continuous data and monitoring, so that patients don’t have the added work of self-reporting or going into a clinic just to monitor vitals. Mobile technology has become so popular in recent years that government agencies and health industry leaders are developing the best practices for using technology in research. Though experts caution that there’s still work to be done in this arena to make sure the wearables perform perfectly and seamlessly with the research.
Helping Researchers Help Us
Dr. Aggarwal observes that patients in trials receive more attention and flexibility than clinical patients. As a result, their satisfaction with the process is very high. In a survey conducted by Know Rare and Advanced Clinical, a research organization, 13 out of 14 people who participated in a clinical study said they would do it again.
At the end of the day, this is the biggest goal—to help patients receive the highest level of care and advancements for their conditions. And it’s working, notes Dr. Aggarwal. “Once patients join a trial,” he says, “they never want to leave.”
For more information about Dr. Aggarwal’s MINT Study, click here.
If you or a loved one is affected by myositis and would like to learn more about the MINT Study, you can also connect with one of our patient supporters by filling out this form:
To learn more about clinical trials and see if there’s a trial for your condition, visit Know Rare’s Study Finder.
