Changing lives – creating remarkable long-term trial experiences
Overcoming cultural, geographical, and financial barriers to facilitate a life-changing trial for one of the world’s rarest diseases
A very young patient had the opportunity to participate in a clinical study into Leukocyte adhesion deficiency (type 1) (LAD), a rare blood disorder affecting the immune system estimated to occur in only one in a million people worldwide.
Although the severity of LAD syndromes and their symptoms vary from one person to another, all affected individuals develop an increased susceptibility to developing severe and often life-threatening recurrent bacterial and fungal infections.
The one-year-old patient was based 400 miles outside of Delhi in India. The trial site was in California, U.S.A, so ensuring safe and comfortable travel to the site was a huge consideration. This required the family to take a train and two airplanes to arrive at the clinical site, with over 24 hours of travel.
As well as the geographical challenges, the family had already lost one child to the same condition, so the emotional load on the parents was immense. Culturally, although the mother spoke some English, the father didn’t speak the language at all, so ensuring effective and clear communication was another aspect that needed to be planned well in advance.
Emotional and cultural support
“Putting patients first” is our mantra at mdgroup. Although travel and accommodation are often the first considerations when getting patients to a trial site, our priority was ensuring the family felt secure and supported before their journey began.
In our first introduction to the family, we learned that the parents had lost their first child to the same syndrome, so we were instantly mindful of the fear they were undoubtedly feeling on top of the stress of being involved in a clinical trial.
We always go above and beyond for our patients and took extra time and care to reassure them, keep lines of communication open and manage every expectation and question.
We had just started organising their involvement in the trial when the coronavirus pandemic hit in March 2020. Just two days before the family was due to fly to the States, India went into an extremely strict lockdown.
For the next nine months, we were in constant communication with the family, reassuring them that the trial would be ready and waiting for them. In January 2021, the patient and her parents could finally leave India and join the trial.
We were aware that being in a different continent to participate in a nine-month-long trial would be a huge cultural change for the family. They would be living in the U.S. for the whole of the trial duration, so the sponsor organised a chaperone for them to assist with day-to-day life. Our team kept in close touch with the sponsor, the chaperone, and the clinical site to ensure a smooth transition to US ways of living.
The chaperone helped them to negotiate travel to and from their temporary home, go to the supermarket, and organise meals out — anything they needed support with to try to relax and enjoy spending time together as a family while living through this extraordinary experience.
We also ensured they had financial support and gave the family a reloadable MasterCard, which we regularly topped up with funds. This meant the family didn’t have to spend any of their own money while participating in the trial.
Being a part of a trial that required the family to spend nine months away from home inevitably leads to daily living costs, and we strived to ensure they never felt any financial burden.
Transport and accommodation
Although there were two other trial sites the family could have attended (The U.K. and Spain), after the delays and trauma of having to pause their involvement for nine months on the cusp of leaving India due to the lockdown, the sponsor decided that getting the family into a trial as quickly as possible was a priority. As such, the sponsor decided to bring the family to the States, where their trial involvement could start sooner.
This involved a 10-hour train ride from their home to Delhi, a 16-hour flight from Delhi to Newark, New Jersey, and a six-hour plane journey to California. All of these had to be arranged with safety, ease, and comfort in mind for a poorly one-year-old.
Upon arrival in California, we arranged a fully-furnished apartment for the family to live in, so they had somewhere to call ‘home’ for their time participating in the trial.
Long-term commitment to care
We’ve been working with this family since late 2019, and in February 2023, we supported them with their 18-month follow-up visits. The one-year-old we met in 2020 is now four years old and a year and a half out from treatment!
From birth, this little girl had been in and out of the hospital with serious infections resulting from her LAD syndrome, but she is currently thriving. She hasn’t suffered from any complication, infection, or hospitalisation for the past six months — a huge milestone for this treatment and the family.
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