Telehealth ‘Helpful’ Alternative to In-person Care, Rare Disease Patients Say

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found.

The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800 rare disease patients and caregivers across the U.S. Results were made available in the report, “Ensuring Access to Telehealth for Rare Diseases.”

Telehealth refers broadly to the use of communication technology — phones and computers — to facilitate healthcare appointments without a patient and doctor, or other professional, needing to be in the same physical space.

Telehealth may offer advantages for people with rare diseases, as the number of expert health providers for a given rare disease is often limited, meaning patients can travel long distances for appointments. Indeed, 39% of the surveys’s respondents reported traveling at least 60 miles to receive medical care, and 17% had moved (or were considering relocation) to be closer to care.

Rare disease patients also often take medications that compromise or weaken their immune systems, raising a risk of infection with in-person visits to hospitals or clinics, and of serious coronavirus complications.

During the COVID-19 pandemic, the use of telemedicine increased dramatically as a preferred option to face-to-face appointments for care.

“Throughout the coronavirus pandemic, nearly 8 in 10 rare disease patients experienced canceled medical appointments,” the report states. “Fortunately, during this time period, telehealth emerged as an increasingly common way for people to safely access medical care without risking exposure to the virus.”

According to NORD’s report, prior to the start of the pandemic, about 0.01% of healthcare appointments across the U.S. were done via telehealth. By mid-April, that number had risen to two-thirds (69%) of total appointments.

Consistently, rare disease patients reported accessing telehealth during the ongoing pandemic. Over 83% of these patients were offered a telehealth visit and of these, 88% accepted one.

Of those using telemedicine, 92% described it as a positive experience.

“We didn’t have to drive 6.5 hours in one day and were able to do it in our living room. It was awesome!” one patient is quoted in the report. “We have been able to get more doctors’ appointments in because they were doing telehealth.”

Another reported feeling safer using telehealth, because they “didn’t have to go into an office” and be in contact with people who were ill.

Some patients and caregivers reported more frequent healthcare appointments via telemedicine, because it removed the constraints of in-person visits.

“It was great seeing my doctors without needing to go in. I have had more doctors’ appointments since COVID due to the accessibility of telemedicine,” one person responded.

In addition to making routine care easier, more widespread use of telehealth could allow for greater participation in clinical trials. Survey results suggest that interest in trials is high, with 88% of respondents open to considering an investigational treatment. This is substantially higher than the 62% who voiced a similar willingness in a survey given three decades ago.

“My daughter’s appointments at Boston Children’s were all canceled. Telehealth was very helpful as it allowed us to move forward with a trial drug therapy that would have been delayed another year despite her progressive decline in health,” one survey respondent said.

Most (70%) taking part to the NORD’s surveys also said they would like the option of telehealth for future medical appointments.

NORD has been working with other organizations to craft policy statements meant to guide policymakers on how best to aid rare disease patients with regard to telehealth, both during and after the pandemic.

The group encourages people in the U.S. to send a note to their senators and representatives, asking them to extend policy changes made earlier this year that allowed greater use of telemedicine, and to protect rare disease patients in a next coronavirus relief package.

“Currently, the Trump Administration, Congress and state governments are trying to decide which changes to telehealth should be made permanent beyond the COVID-19 public health emergency,” the report states. “Our continued advocacy can help on this issue so that the positive changes that we have seen during the pandemic will endure after it ends.”