Clarity through a complex treatment journey
Several new therapies have recently been FDA approved to treat specific rare diseases, helping many patients better manage their condition. Even so, the treatment journey itself can be difficult for both patients and their caregivers.
In addition to dealing with the burden of the rare disease, these individuals face a variety of challenges that can take a toll on physical and mental health, as well as family relationships.
Challenges include delays in diagnosis, barriers to accessing care (such as travel to specialists and the costs of care) as well as complicated, multi-faceted treatment plans. Patients and caregivers can also have psychosocial issues, including feelings of isolation, loneliness or hopelessness
Because so many rare disease patients are children, these concerns may fall on the shoulders of caregivers, often parents. In these instances, parents are managing their child's treatment while also balancing their own life, work and family needs.
According to the most recent Rare Disease Caregiving in America report, one out of every three rare disease caregivers say they have difficulty in accessing:
- Treatments or therapies for the patient's symptom management (34%)
- Treatments for the underlying disease (31%)
Caregivers themselves may also overlook the importance of addressing their own well-being. The same report notes that just under half of rare disease caregivers have actually had a health care provider ask about their own needs as a caregiver.
According to the authors, the lack of rare disease caregiving support presents a “missed opportunity for outreach.”
A model built around whole-family support
These opportunities and challenges all point to the need for greater clarity, resources and engagement, as well as rare disease caregiver support, in order to make a positive impact on families.
The Optum® Frontier Therapies model was designed to address all these factors. Extensive patient and rare disease caregiving support is incorporated at every stage of the treatment journey, from the welcome call to ongoing outreach that may be needed years down the road.
At Optum Frontier Therapies, the journey starts with giving families a dedicated point of contact. Patients and their caregivers work with a care ambassador who orchestrates complexity behind the scenes to streamline support and solutions.
Highly trained in both empathy and problem-solving, the care ambassador strives to have an “across the kitchen table” relationship with families. Of course, patients and caregivers also have access to clinical expertise provided directly by dedicated pharmacists.
The care ambassador and pharmacist listen to the story of each patient, caregiver and family and then tap into every available resource to help. For example, if medications aren’t refilled by the patient or their caregiver, these team members work to find the root cause.
In some instances, parents or patients have to choose between buying food and medication because of financial concerns. The team researches grant funds and copay assistance programs, among other avenues, to help address cost challenges.
The care ambassador also serves as a valuable bridge to other community resources that can be especially useful in helping families address complexity related to other social determinants of health.
In some cases, individuals may have difficulty accessing transportation to care visits or could have health literacy or language barriers. The care ambassador has extensive knowledge of local resources and assistance programs that may be able to help families with these issues.
Connecting families with shared experiences
Of course, given the challenging nature of rare diseases, families may also be dealing with issues like isolation and loneliness as well as exhaustion, stress, lack of sleep and pervasive worrying.
One of the best ways care ambassadors can help is by connecting them to advocacy groups that include other families of patients managing the same rare disease.
Having a shared experience helps foster lasting and meaningful connections with others. Just as important, it can also help families learn from the stories of people who have overcome similar challenges.
Other common hurdles involve logistics with administering medications. Injections or infusions can be particularly difficult for children, who may be dealing with fear and pain. Or families may have trouble timing medication doses or injections around their busy schedules.
In these cases, the Optum Frontier Therapies pharmacist again emphasizes personalized problem-solving, helping families identify solutions and put them into action.
To simplify treatment, pharmacists will even conduct virtual visits for training on how to administer medication and manage symptoms. Those visits are recorded and can be played back at the caregiver’s or patient’s convenience.
All of the information gathered during the interactions — along with input from patients, caregivers, prescribers, pharmacists and nurses — is then incorporated into a dynamic care plan.
Incorporating coaching, education and resources, this plan is designed to move families along a continuum from collaborative care to empowered, self-directed care.
Real-world example of clarity and support
To understand how these processes benefit both patients and caregivers, it helps to understand the personalized and disease-specific nature of these interventions.
Optum Frontier Therapies provides support to families managing progressive familial intrahepatic cholestasis (PFIC), a genetic condition that affects the liver, with symptoms often starting at just three months of age. The condition can progress to cirrhosis of the liver within the first decade of life.
For new parents, this diagnosis can turn one of the most joyful times of their lives into one filled with worry, concern and fear of the future. In fact, without intervention, few PFIC patients survive into their 20s.
For these parents, ensuring that their child has access to an effective therapy becomes a top concern, and often supplants other responsibilities.
That’s why our clinicians not only coach these caregivers on managing medication to deal with their loved one’s physical symptoms but also support them as they face their own emotional burdens — which are often related.
For example, a newborn with PFIC may not sleep well because of itching, which leads to exhaustion among household members and can even drive depression and anxiety.
Parents of school-age children may be dealing with concerns from educators and school nurses about symptoms and may need information and resources that can empower them to have these conversations.
Optum Frontier Therapies team members can also provide referrals to advocacy groups that can connect these parents with other families who have “been through it” to build camaraderie and address isolation. Some caregivers have never before met another set of parents managing PFIC.
Not only will many parents build lasting connections with these families, but siblings and other household members can also benefit from these relationships.
Every Optum Frontier Therapies team member receives hours of training on each rare disease and works directly with other patients and family members as well as PFIC community organizations. These experts can easily apply this knowledge to help solve problems and provide support.
And in fact, part of this training involves watching live or pre-recorded interviews with families so they can learn about challenges and best practices firsthand.
Making a lasting, positive impact
Throughout all of these processes, care ambassadors always check in to see how everyone in the household is doing since rare diseases have a widespread impact beyond just the patient.
Ambassadors may connect all of these individuals with counseling when needed, along with community resources and ongoing education.
For example, parents of children with PFIC may be referred to the PFIC Network. This organization is dedicated to improving the lives of PFIC patients and families by supporting research programs, providing educational materials, and matching families for peer support.
By combining knowledge of specific rare diseases along with experience working directly with families, the Optum Frontier Therapies team can make a difference in the lives of both patients and the people who care for them.
Their interventions can help patients and caregivers build confidence and better navigate the treatment journey. Given that many rare diseases affect people over a lifetime, the effort put into this type of outreach today can drive meaningful benefits for decades to come.
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