Approximately 360 million people globally are living with a rare disease. Over half of these are children. Many of the diseases are life-threatening and only 5% have an approved treatment. Trials in rare diseases are typically conducted at fewer sites, are longer in duration and are more frequently terminated than trials with more common indications.1 While there is continued growth in the development of drugs to treat rare diseases, clinical trial accessibility for patients remains challenging due to the requirements inherent in these studies.
Participation in a paediatric rare disease trial involves not just the child, but the whole family and may be the only potential treatment available. It is critical to consider the experience of the child and their caregivers not only at the beginning but also throughout the study, which can be many years in length.
There are physical and logistical, financial, and psychological and emotional pressures that come into play when participating in a clinical trial.
Physical and Logistical
Most patients with a rare disease already visit specialists on a regular basis. Participation in a clinical trial requires additional travel, time away from school and work and the need to arrange other family care while away from home. Depending on the trial site location, families may travel significant distances by car, train, or air. Air travel can be particularly challenging due to the length and discomfort of the journey and the difficulty accommodating wheelchair-bound travellers.
Rare disease trial sponsors frequently employ organisations that specialise in the provision of patient travel support during trials. These organisations can coordinate travel to meet the specific requirements of each family taking into consideration work and school commitments for parents and children, respectively.
These organisations can also work with trial sites to ensure that specific accommodations meet the needs of patients during site attendance. This may include the selection of residential-style lodging with suitable patient and family accessibility located near the study site.
Study site accommodations should include a child-friendly environment for entertainment for the patient and additional space for siblings if they are attending. For those sites without sufficient space, these travel support services could arrange alternative facilities for waiting and caring for patient siblings.
Families incur additional costs and not all expenses are reimbursed, such as loss of a parent’s wages to attend study visits. Often parents are expected to pay upfront for trial-related expenses and submit receipts which can be inconvenient and burdensome.