Rare Disease Study Requires International Patient Travel Services

Wednesday, Mar 17. 2021
Organizing patient air travel, short-term relocation, and expense reimbursement for a family with an infant, clinical trial participant.

 

Challenge

Roughly 300 million people, globally, are affected by one of 7,000 individual rare diseases. For those with Type 1 Spinal Muscular Atrophy (SMA), the diagnosis is life-limiting, and treatment options are limited. Type 1 SMA or Werdnig-Hoffman disease is diagnosed in infants prior to 6 months of age. The average life expectancy for 95% of infants diagnosed with SMA-1 is 18 months.

We supported one long-term, extensive study of SMA-1 where the sponsor required support for international, short-term relocation for families who received a recent diagnosis of SMA-1. Families of infants who receive a diagnosis, and were invited to participate in the study, had only a few days to determine their enrollment. The study, for a single gene therapy treatment, required intensive monitoring and physiotherapy. Infants and families who enrolled would travel to Milan from their home to one of several sites in Europe and remain until the patient is 18 months old. For families, this is a highly emotional time as they are receiving a life-altering diagnosis for newborns and are then faced with a time-limited decision for study participation.

Given the intricacies of the recruitment timeline, from the point of enrollment, each family would have three days to solidify arrangements and arrive for a first site visit. The time to make arrangements for international travel and relocation can require weeks of preparation for the average person. The financial and logistical constraint of a move for a family with a new rare disease diagnosis was often unfathomable to new parents and would impact their decision of whether or not they enrolled in the study.

 

Solution: Managed International Patient Travel

We worked with the sponsor to identify the three site locations in Rome, Milan, and Genoa and tailor a travel process for the newly enrolled participant families. From the time a new patient is enrolled, Paragon Travel Support Specialists would arrange the travel, relocation, expenses, and transit lodging within a three-day window. Once a new patient was submitted for enrollment within our web-based patient portal, Paragon would provide international patient travel services and arrange:

  • Travel documents and interpreters
  • Ground travel from point of origin to the nearest major airport
  • Air travel, tickets
  • Overnight lodging and hotel stays
  • Local, furnished living arrangements for 15 months in the vicinity of the treating hospital
  • Car service for the first and future site visits

Our Travel Support Specialists are a designated point of contact for study participants. Patients and caregivers work with a trusted name to provide 24/7 service for any challenges that they might face. International travel can be overwhelming under the best circumstances, so for these families, our goal was to ensure they were able to focus on study participation.

For the duration of the 18-month study, patient families would require living expense support and ongoing travel assistance. Private car service and taxi options were provided for site visit travel. Paragon Travel Support scheduled and arranged the non-clinical services for these families to ensure their continued study participation. For families, particularly those from Eastern European countries who often only speak their native language, we helped them overcome the challenge of waking up in a different country, different culture, and with a different language. The Paragon specialists were readily available for translation support in almost any situation.

To address cost, Paragon provided expense management and reimbursement for participating families. Expense allowances, banking needs, residency permits, and more were completed with the support of a travel specialist. Our team organized documentation and processed rapid reimbursements for a wide variety of living expenses, emergency management, and more.

 

Result

Our team relocated families from Poland, Ukraine, Germany, England, Russia, the US, and the Netherlands and supported their short-term stay through site visit and expense management. In one instance, we successfully relocated a family to Milan during Fashion Week when travel into and out of the city is at its peak, seasonally. international patient travel

Paragon’s Travel Support Specialists prioritized the patient and caregiver experience in each interaction throughout the study. Our goal was to make participation in the study as worry-free as possible, given the already challenging circumstances. The sponsor was extremely satisfied with Paragon’s ability to provide concierge international patient travel support in this study. Feedback from patient families was overwhelmingly positive.

“We are safe at home. Journey was long but our children did well traveling. Thank you for your support during last year, we very appreciate of your help!”

 

Our specialists support the patient families through the very end of the study. We help to support patient engagement through timely, informed customer service and excellence in logistics management.

 

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We’re interested in sharing our experience in a variety of clinical research support areas and therapeutic classes, including rare disease and pediatric studies. Send a message to info@paragonglobalcrs.com for more information.

Paragon Global CRS

Paragon Global Clinical Research Support (CRS) provides sites, sponsors and CROs with patient logistics, patient travel solutions, event management and meeting planning.

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