Navigator: providing logistical support to a family and patient with Danon disease
Facilitating remarkable patient experiences and removing the burden of site participation.
A young patient had the opportunity to participate in a clinical study into Danon disease, a rare condition that causes the heart and skeletal muscles to weaken.
Young boys often develop symptoms earlier than girls and are more severely affected. Our team needed to fully understand the nuances of the disease and the challenges the patient faced, so we could provide the best possible care.
As is often the case with rare diseases, there are limited opportunities to participate in clinical trials, which meant the patient, his sister, and their father had to travel from their home in Sydney to the study site in Philadelphia.
As a single father with two young children, navigating a new city would have been challenging enough. In addition, the patient needed to be at the hospital for the entire study duration, so the father requested his brother join the family to provide additional support.
We managed all the logistics, ensuring safe travel to the study site and additional support for their stay. We’re passionate about providing remarkable care for patients and their families, and our team went above and beyond, considering every need during this challenging time.
Transport and accommodation
Our motto is: ‘first, seek to understand’. Before we began planning the travel and logistics, we listened carefully to the needs of the patient and his family. In this instance, the patient’s uncle also needed travel, and we arranged for him to join the family soon after they arrived in Philadelphia.
Due to the nature of Danon disease, which significantly impacts mobility, we arranged accommodation as close to the hospital as possible to reduce unnecessary travel. Old City Philadelphia is a beautiful and historic part of town, close to the hospital and most of the local attractions. A win-win for everyone! We arranged accommodation in a safe part of the neighbourhood, which meant the family could easily explore without requiring taxis or public transport.
At this time, COVID cases were still high, and the father requested support in avoiding large groups and crowded areas. We considered every place the family could come into close contact with people and put in measures to reduce exposure. Our team arranged a perfect temporary home for the family with private entrances and no elevators.
We also provided the family with a new mobile phone with a US SIM card, as the hospital had issues contacting their international number. This meant the father could easily contact the patient and healthcare team during several weeks of in-patient treatment.
A few months after their initial visit, the patient needed to travel back to Philadelphia for a follow-up appointment at the hospital. Fortuitously, the flight from Syndey included a layover in Hawaii and the father requested an extended stay during the layover to have a well-deserved vacation. We were delighted to be able to extend their stay at no additional cost.
The family arranged their accommodation in Hawaii and spent a few days enjoying the warm weather and quality time with each other.
This was a phase one trial with only 12 patients enrolled, which meant engagement and retention were crucial, and the patient needed to complete all treatments to move on to the next phase. He also needed to attend as many assessments as possible while the family was in the US, which would minimise the need for travel in the future.
Thanks to our team’s collaborative efforts, we worked closely with the hospital staff, ensuring the patient completed all visits and assessments with minimal stress for all involved.
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