Pharma Intelligence recently published a white paper titled “Rare Disease Trial Strategies” that piqued our team’s interest in how we may be able to help sponsors, CROs, and sites address some of the top challenges in this space. Some of the statistics shared in this resource were eye-opening such as how approved drugs are available for just 5% of the approximately 7,000 rare diseases identified so far. While the United States leads the global development landscape in rare disease trials with just over half of all rare disease trials involving a clinical trial site in the US, there is still room for further improvement.
How might on-site mobile care with 20/20 Onsite help expedite approvals and improve the patient experience for those trying to receive potentially life-saving treatment? We’ve outlined a few scenarios below.
The World Health Organization defines rare diseases as having a prevalence of one or fewer in 1,000 people — so it’s no surprise the patient pool for rare disease clinical trials is smaller than non-rare disease trials, impacting enrollment speed.
In the chart above, you can see the most pronounced difference in enrollment is for non-oncology rare diseases where a clinical trial site enrolls an average of .68 patients per month compared to the average of all non-oncology diseases at 2.02 patients enrolled per month.
Additionally, it may be challenging to adequately screen patients where it isn’t feasible to narrow qualifying criteria based on their disease stage or other conditions.
As your clinical research partner on wheels, we can travel to any community throughout the United States regardless of access to a major medical center or established site. In fact, in 2021 alone we drove over 75,300 miles through all 48 continental US states to visit hundreds of patients directly.
In addition to reaching more patients, we can help pre-screen the patients we do reach to help qualify them for the next phase of the trial. That means once they reach the clinical site, they will have already gone through one round of screening, positively impacting statistics such as enrollment speed and screen failures.
The Pharma Intelligence white paper revealed the most prevalent obstacles to participation in trials are being able to locate ones that are geographically convenient and appropriate for patients’ condition/disease.
Another study found the longest distance traveled by a patient in a phase 1 clinical trial was 41.2 miles. The burden of travel is highest among patients enrolled in NIH-sponsored trials, phase 1 studies, or those living in low-income areas. The study also found that white patients enrolled in cancer clinical trials tended to travel longer distances than non-white patients, indicating non-minority patients may have the means to travel for care compared to other ethnicities.
Mobile healthcare services are a proven way to reach more diverse communities and reduce travel burdens, especially rare disease patients who may not be able to travel alone to appointments. Our clinic can travel directly to patients’ homes, places of work, or physicians' offices, making it easier for patients to participate in follow-up appointments. Last year, our clinical patients traveled an average of 12.36 miles to receive care onboard our Mobile Vision Clinic compared to the over 40 miles mentioned above.
Driving our Mobile Vision Clinic directly to patients isn’t the only way we can create more accessible care — we can also pre-screen patients virtually. In one instance, our optometrists used virtual methods to interview a patient and determine if he was qualified to participate, eliminating the need for an in-person evaluation.
"The patient was effusive about his gratitude for this trial existing, and his ability to partake. He was especially thankful that we were able to see them just across the street from his job. Transportation, even within a short distance, is difficult for this patient given that they can't drive. Being met where they were and reducing any additional travel or logistics coordination made them feel particularly well cared for." - A 20/20 Onsite clinical trial patient account
Recommended reading: How Sponsors Can Reduce Travel Barriers to Improve Patient Recruitment and Retention
In a first-hand account published in Orphanet Journal of Rare Diseases, a patient describes her struggles living with a rare disease. Her condition took 17 years for a correct diagnosis after seeing a variety of doctors (the average rare disease patient waits 4 years and sees 5 different doctors). She says, “one way in which I view my life is as a series of precarious balances. Balancing the need for a clear diagnosis with the need to get on with life. Balancing the time spent in hospitals as a patient with the time spent studying there as a medical student. Balancing the wish to have completely honest conversations with doctors with the worry that they will no longer take you seriously if you do so.”
When trying to find treatment, rare disease patients face mounting stressors and questions that are only complicated by complex trial procedures or inconvenient study locations. They may have to coordinate schedules with loved ones in order to get transportation and take time off of work to check in with a doctor. All of this, in addition to the disease itself, puts a toll on their mental health.
38% of clinical trial patients who dropped out of a clinical trial found their site visits to be stressful. As a patient with a rare disease, the experience with a doctor should be the least stressful part of finding care! When you work with 20/20 Onsite, you get access to a pre-screened, licensed optometrist and an experienced customer success team member who have contributed to our consistently above-average patient satisfaction score.
Take a look at some of the feedback we have received from patients and clinics in our current clinical programs:
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