Rare Disease Day 2024: Shining a Light on Caregivers

Contributed by Scott Gray. Scott Gray is the co-founder and CEO of Clincierge, a provider of patient support services for clinical trials. Since 2015, Clincierge patient coordinators have managed logistics and reimbursements in more than 300 clinical trials worldwide. For more information, visit www.clincierge.com.

Established in 2008, Rare Disease Day is an annual observance to raise awareness of the more than 7,000 identified rare diseases and to show support for affected patients and their loved ones. The celebration is held on the rarest day of the year, February 28th or 29th, depending on leap years.

Created by the European Organization for Rare Diseases (EURORDIS), this day serves as a catalyst for rare disease advocacy efforts at local, national, and international levels. Since its inception, Rare Disease Day has been instrumental in fostering an inclusive and diverse global rare disease community. EURORDIS collaborates with numerous international organizations for this annual commemoration, including its counterpart in the United States, the National Organization for Rare Disorders (NORD).

This Rare Disease Day, we shine a light on caregivers, the often overlooked yet indispensable heroes of the rare disease community. These dedicated individuals come in the form of family members, friends, and licensed professionals, serving as pillars of strength and support for patients navigating the complexities of their conditions. From providing emotional reassurance to managing daily tasks and assisting with medical treatment, caregivers play an irreplaceable role in the lives of those affected by rare diseases.

The Weight of Being a Caregiver

Taking on the role of caregiver is often a life-changing experience. One report showed that, on average, caregivers spend:

  • 13 days per month on tasks such as shopping, food preparation, housekeeping, laundry, transportation, and giving medication.
  • 6 days per month on feeding, dressing, grooming, walking, bathing, and toileting assistance.
  • 13 hours per month researching care services or information on the disease, coordinating physician visits, and managing financial matters.

Undoubtedly, these responsibilities create an extremely heavy burden for the caregiver. Handling these many tasks, in addition to providing support to a sick loved one, often takes a severe emotional and physical toll. Compounded by the fact that 1 in 3 caregivers are over the age of 65, many of these individuals also face their own health conditions and physical limitations.

Beyond the weight of these responsibilities lies an even more profound challenge: the role of patient advocate. Far too often, individuals grappling with a rare disease diagnosis find themselves overlooked, misdiagnosed, or neglected by traditional healthcare systems. In these instances, caregivers must become fierce advocates, tirelessly fighting for accurate diagnoses, access to appropriate treatments, and recognition of their loved one’s unique needs.

Within the challenges faced by caregivers lies an opportunity to address broader healthcare issues, particularly in rare disease research. Optimizing clinical trials can speed up the process of bringing much-needed new treatment options to market. By improving research protocols and addressing clinical trial pain points, scientific discoveries can be more quickly translated into tangible therapies, offering hope to individuals living with rare diseases and those who care for them.

Improving Treatment Options Through Patient Support Services

The clinical trial process is foundational in transforming rigorous research into life-changing or potentially lifesaving healthcare advancements. However, nearly 80% of clinical trials fail to finish on schedule, with 20% delayed over six months or more. While the reasons for these delays vary, an estimated two-thirds of trial sites fail to meet initial patient enrollment target numbers. Additionally, 85% of studies cannot retain enough participants through trial completion.

One of the most significant factors preventing enrollment and retention in rare disease clinical trials is the travel associated with participation. One study showed trial patients often travel up to 40 miles from home to participate in clinical research. With the limited availability of trial opportunities for rare and ultra-rare disease patients, travel can often be far more extensive, sometimes crossing state and country borders. The additional stress associated with finding local medical care and lodging, securing passports and visas, and obtaining translation and interpretation services often discourages patients and their caregivers from agreeing to this much-needed treatment.

Given the various obstacles facing rare disease clinical trial participants, pharmaceutical sponsors and contact research organizations (CROs) should consider investing in a patient concierge services provider to improve trial efficiency. Much like a hotel concierge, a patient concierge manages all logistics involved in study participation. These services include, but are not limited to:

  • Itinerary planning (flight and rail ticketing, hotel accommodations, ground transportation)
  • Reimbursements for meals, incidentals, mileage, tolls, and parking
  • Wage reimbursement, where applicable
  • Interpretation and translation services
  • Visa and passport assistance
  • Long-term housing and relocation services

In addition to managing these trial logistics, a patient concierge continuously provides personalized emotional support to rare disease study participants and their caregivers, allowing them to feel more comfortable and secure throughout the duration of a trial. This one-on-one level of support allows the patient to focus on their treatment and the caregiver to focus on assisting the patient, easing the path to trial completion for both.

In Conclusion

Rare Disease Day serves as a poignant reminder of the many unique challenges rare disease patients and their loved ones face. As we shine a light on caregivers this year, we recognize the immense loads they shoulder, both in their daily responsibilities and as advocates for their loved ones within an often complex healthcare system.

By acknowledging the critical role of caregivers and addressing the unique burdens they face, we can pave the way for more significant progress in rare disease care. By investing in a patient support services company for rare disease patients and their caregivers, clinical trial stakeholders show their commitment to alleviating the burdens of participation and facilitating access to vital treatments.

Share this post

Follow us