Caring for Rare: Navigating a Complex Journey
A rare disease diagnosis poses more questions than answers for parents learning that their child has a severe and often untreatable condition. According to the National Organization for Rare Disorders (NORD), there are over 7,000 known rare diseases, with 90% of these having no effective treatment. Obtaining a diagnosis is only the first step and leaves many families without direction or resources for finding the medical care their child requires. When caring for a child with a rare disease, a parent will take on many roles, including the responsibilities of a nurse, advocate for their child, chauffeur, researcher, nutritionist, pharmacist, and teacher, to name a few. Rare Parents can feel isolated due to the nature of these uncommon diseases and will seek community and financial resources to alleviate the burdens they face.
Logistical Challenges of Finding Care
In some cases, the family can identify a physician with the expertise to diagnose and treat their child. In these instances, most must seek care from a specialist practicing at a larger medical center. These facilities tend to be in metropolitan areas and present logistical barriers to families that often require frequent appointments. In NORD’s report, Barriers to Rare Disease Diagnosis, Care and Treatment in the U.S.: A 30-Year Comparative Analysis, “[t]hirty-nine percent of respondents needed to travel 60 or more miles in 2019”. A majority of parents admitted the child’s medical treatments affected work schedules; travel time and the time spent meeting with various specialists for multiple tests and assessments adds up quickly for families, and with more than 30 million U.S. workers without paid sick time, many parents face an added financial burden of lost wages, as well as travel costs. Even more burdensome is when the patient is not responding to currently available forms of treatment, or no treatment exists.
Connecting with the Rare Disease Community for Support
The rare disease community and advocacy groups can step in to help with the surmounting costs, but there are limits to the benefits available to families, and research shows that families caring for a child with a rare disease have many unmet medical and financial needs3. A step in the right direction is to join advocacy groups, forums, and online communities to find support and additional resources to assist with the added emotional burdens. Online communities are often a wealth of information for innovative treatments and potentially life-saving clinical trial opportunities.
Rare Disease Advocacy Group Resources for Families
Navigating Barriers to Clinical Trial Participation
A clinical trial can be an option when all other medical alternatives are exhausted. Referral for a clinical trial may mean having to adjust to a new health care team and environment. Unfortunately, few hospitals enroll patients for most rare diseases, again putting the burden on the family to arrange and pay for travel that can take them cross-country. Trial sponsors are sensitive to these obstacles and implement strategies to mitigate the hurdles faced by families. The FDA lays out guidance to regulatory boards that reimbursing participants for reasonable travel expenses incurred due to clinical trial participation does not cause undue influence4. However, even with the benefit of being reimbursed those costs, families must dedicate their time and effort to arrange travel and accommodations and pay for out-of-pocket expenses. The complex logistic and financial planning adds stress to a Rare Parent’s already full plate. Significantly reducing the costs incurred during study participation helps lower the barriers to entry in clinical trial participation.
Patient Support Services Enable Access for Rare Families
Addressing these challenges is no easy task. However, resources are available to families enrolled in trials where the sponsor works with a trusted patient support services provider. Families can benefit greatly from having someone “in their corner” to assist them with their logistical and financial needs, relying on a single-point-of-contact that is GCP-certified and trained on the study protocol, affording insight into the unique support needs of the patients and families. Families of participants enrolled in such studies are eligible for services including pre-booked air, accommodations, and ground transportation during the length of the study. Depending on the participant’s needs, families can expect specialized travel accommodations, such as wheelchair-accessible vehicles for transit between the hospital and their hotel, ADA rooms, and assistance with airplane boarding.
During their time away from home, families will incur costs for meals, travel expenses like parking and tolls, and other incidentals. Families enrolled in trials can be eligible to receive expense reimbursements for these out-of-pocket costs. The personalized support of a patient travel coordinator makes the reimbursement process smooth and stress-free for parents so they can focus on the care of their child. Travel coordinators work with the family to gather all the necessary information to process reimbursements to ensure continuity and continued access to care.
Families caring for a child with a rare disease will face many challenges, but there is some hope for those who can access experimental treatment via a clinical trial. Working with a trusted partner who helps the family navigate the clinical trial experience brings them closer to realizing the hope.