Parents cite insurance headaches, knowledge barriers around rare disease management



“There is a complicated balance between reducing health care costs and meeting patient needs,” they said, noting the lack of research on parents’ perspective on their experiences with insurance companies. illness or how policies may interfere with the care of their children.

To fill this gap, the researchers conducted a series of semi-structured interviews with parents of children with metachromatic leukodystrophy (MLD) or spinal muscular atrophy (SMA). These diseases were selected because of their specific characteristics and their orphan drug status.

In particular, “both diseases have an identified genetic mutation and varying degrees of severity within the disease. Both conditions impact motor function, which may require physical therapy or supportive equipment to facilitate mobility. Although the drug nusinersen was approved by the FDA in 2016 for SMA, at the time of the study, no orphan drug for MLD was available.

A total of 15 parents were included in the final sample, of whom 4 had 1 child with MLD, 10 had 1 child with ADS, and 1 parent had 2 children with ADS. All children were insured by a parent’s employer, and 9 were double or triple covered by public insurance.

Throughout the interviews, which lasted around 30 minutes each, several major themes were identified, including the difficulties in obtaining secondary insurance based on the eligibility criteria of the State, the difficulty of accessing health services. health requirements and the need for repeated interactions with insurance representatives.

“The lack of clearly documented or widely recognized clinical guidelines has exacerbated the difficulty in accessing care identified as necessary by their health care team, such as therapy and equipment,” the researchers wrote.

For emotional support and knowledge, parents said they turned to disease-specific organizations, social service agencies or health professionals, among other groups.

After designing an explanatory model based on themes and sub-themes, the researchers concluded that “the cyclical nature of the interaction with insurance for redundant reauthorizations and the external support and financial assistance that are often required to meet their child’s health care needs ”has become evident.

Participants also described the confusion around health insurance documentation, while others described the challenges of gathering information about their child’s condition over time.

Some people reported difficulty obtaining secondary insurance for their children; however, all parents reported that their child had never been uninsured.

“The desire to have immediate coverage for treatments, especially nusinersen, led a mother to refuse anesthesia during childbirth to make sure she could complete social security paperwork and send an e -mail to her insurance company after the birth of her child, ”the authors said.

Difficulties related to insurance emerged as a major obstacle for study participants.

A parent of a child with ADS said, “Almost every state in this country has a waiver that allows medically disabled children to access the state’s Medicaid system, so they can access all of these. services, regardless of parental income, and our state doesn’t have that, so it’s been incredibly difficult for us, and been a major barrier to getting her care, you know, nursing and some equipment that is only covered by Medicaid, it is not covered by Private Insurance. “

Small employers and the substandard plans on offer also posed problems for parents, who cited health insurance as a driving factor in many job decisions and told of fears of losing their jobs or being impacted. due to the costly nature of rare diseases despite the legal ramifications of discrimination.

Each person described the frustrations of communicating with insurance companies.

“I mean, I hate it [interacting with insurance], but it needs to be done, because we can’t afford to not do it right, so we just have to keep keeping this documentation of every call and every time and what they said because I have feel like I’m more organized than them, and I feel like I have to be because my daughter is so worth it, so that’s where we need to be, ”said one parent.

The biggest barriers to coverage among participants were related to equipment, nursing, therapy, and off-grid providers, the authors said. Lack of awareness of the disease has led to a poor understanding and understanding of the patient’s medical situation by insurers. “Many people discussed concerns about the health consequences of delays or refusals by the insurance company, especially the potential for worse health outcomes or lost opportunities, such as participating in clinical trials. “, added the authors.

Fears of losing coverage for pre-existing conditions have also weighed heavily on the minds of many parents.

Overall, “a single health insurance policy was seldom enough to cover the cost of all of their child’s health care needs, leading parents to view access as a web of different policies and social services, ”the researchers wrote.

Compared with parents of children with ADS, parents of those with MLD reported that insurance companies had fewer established guidelines for the disease, increasing the need for interventions from healthcare professionals to facilitate treatment. ‘Health care access.

Parents reported that insurance representatives’ lack of knowledge “leads to greater frustration when insurers ignore the medical benefits and long-term savings of access to equipment, therapy and diagnostics. “.

One potential solution to these challenges is the use of focal points — social workers or health professionals who can help guide parents or caregivers through clinical aspects of the health system.

In addition, investing in programs that could lead to faster diagnosis, including access to genetic testing, could benefit patients, while expanding access to Medicaid and resources to limit the time required for examination. of a claim could lead to additional avenues to obtain insurance, the authors said.

Insurers could also employ specialists trained to handle complex care cases to ease the burden on caregivers.

The small number of parents of children with MLD included in this study is a limitation, and participants may also have been subjected to selection bias. In addition, no participant was uninsured or under public insurance exclusively, which limits the generalization of the results.

“The complexity of the US insurance system forces parents to enroll in multiple plans to maximize coverage, an option that is not available to all families,” the researchers concluded.

“There are policy initiatives that could impact payment and delivery systems that could greatly improve the patient experience and outcomes,” they wrote, but integrating the perspective of caregivers and providers. patients is paramount in reform efforts. “Further studies are needed to understand the full scope of barriers to care and policies that can facilitate better access to care for families living with rare diseases. “


Pasquini TLS, Goff SL, Whitehill JM. Navigating the U.S. Health Insurance Landscape for Children with Rare Disorders: A Qualitative Study of Parental Experiences. Orphanet J Rare Dis. Published online July 15, 2021. doi: 10.1186 / s13023-021-01943-w



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