Challenges Working with Terminally-Ill Patients in an International Rare Disease Study

Allucent is a clinical research organization (CRO) that has vast experience in rare diseases. To date, we have completed over 120 studies and worked with more than 11,000 patients who suffer from rare diseases. Our CRO services have supported 14 drug approvals. Roughly nine out of 10 project managers and clinical research associates on our team have worked on a rare disease study.

Understanding Rare Disease

Rare disease is a challenging area of research. Keep in mind that rare disease impacts fewer than 1 in 2,000 people (EU definition). While somewhere between 3.5% and 6% of the world’s population have a rare disease, there have been more than 6,000 rare conditions identified and even more that do not have names yet. In fact, many of these conditions are so rare that it can be difficult to find patient-participants.

Allucent surmounts the challenges encountered in rare disease by being flexible, patient-centric and bringing the research to where the patients are or when needed, by bringing the patients to where the research can be facilitated.

Rare Disease Research in Practice

In reality, researching rare diseases is even more difficult than the numbers would suggest at first glance. Amongst people with rare diseases, 35% do not see their first birthday and 30% will pass away before the age of 5. This startling fact shrinks the number of people available and/or willing to participate in rare disease research. As such, rare disease studies often involve terminally ill participants as well as multiple sites.

Crossing borders is one way to increase the number of potential patients. For example, Allucent recently managed an Ultra-Rare Disease Natural History study. The condition occurs in only one in 50,000 newborns. We developed four sites in the United States, but we also looked to Canada, France, Germany, and the United Kingdom. While some clinical studies take place within a single country, the sheer rarity of some indications necessitates a flexible model where recruiting patients in country or across borders may be necessary. This approach helps improve recruitment and may even shorten timelines, but conducting international research trials with terminally ill patients has its own challenges.

In this article, we are going to look at some of the primary concerns that CROs have when working with terminally-ill patients in an international rare disease study.

Supporting Patients Through Travel Arrangements

Most Sponsors today, especially in rare disease, will pay or reimburse patients to travel to research sites. The distance and duration of travel can have a broad range. The duration of their stay is always protocol/study driven, and we have seen durations of up to 5 day stays in some studies. This is a large burden on the patient as well as the caregivers who support them and their participation in clinical trials. To ease the burden, supplying study participants and caregivers with travel concierge services and support is critical. After all, these individuals may travel to an unfamiliar city, state or even country to participate. Working with severely ill persons and navigating borders adds additional elements to the mix.

When a terminally ill patient travels to a research site, the Sponsor organization / CRO should handle the coordination of those arrangements. In many cases, the study participants will need additional care upon arrival or even during their trips to and from the facility. In the case of our natural history study in an Ultra-Rare disease with subjects up to three years old, this meant arranging airfare and hotels as well as nursing staff to accompany the participants during their travel. In this case, the Sponsor used a travel agent to manage travel for the patients and their families. Incidental expenses were covered through travel reimbursements.

Managing Language Barriers

Crossing borders also adds a potential language barrier. In our recent Ultra-Rare, we had to ensure that a translator would be available on-site so that the participants and their families could understand every detail of what was happening. Accepting patients from other countries is generally up to the site. The decision is made with input from the staff. In some cases, you may be fortunate and the facility/site may have a fluent speaker on-staff or you (Sponsor or CRO) may need to hire a translator to support the site.

Moving Forward in Rare Disease Research

Working with terminally-ill patients transforms the landscape of any study. Patient safety, dignity, and privacy are paramount, but when that research takes place internationally, the challenges are compounded. Sponsor organizations need to have an experienced CRO with strong rare expertise in place to handle these issues and still deliver successful studies. Look no further than Allucent. We go the distance to create a healthier world.


Co-authored by Barbara Bennett, Senior Clinical Trial Leader

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