Navigating the U.S. health insurance landscape for children with rare diseases: a qualitative study of parents’ experiences

Pasquini, Tai L. S.; Goff, Sarah L.; Whitehill, Jennifer M.

doi:10.1186/s13023-021-01943-w

Cited by 29 publications

(66 citation statements)

References 53 publications

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“…In addition, health insurance providers must be willing to authorize patients’ referral to centers of excellence without delay. For children with rare diseases, health insurance status has a notable impact on the continuum of care, and delays or denials of insurance companies have the potential to negatively impact children’s health [ 30 ].…”

Section: Main Textmentioning

confidence: 99%

Congenital hyperinsulinism in infancy and childhood: challenges, unmet needs and the perspective of patients and families

Banerjee

Raskin²,

Arnoux

et al. 2022

Orphanet J Rare Dis

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Background Congenital hyperinsulinism (CHI) is the most common cause of persistent hypoglycemia in infants and children, and carries a considerable risk of neurological damage and developmental delays if diagnosis and treatment are delayed. Despite rapid advances in diagnosis and management, long-term developmental outcomes have not significantly improved in the past years. CHI remains a disease that is associated with significant morbidity, and psychosocial and financial burden for affected families, especially concerning the need for constant blood glucose monitoring throughout patients’ lives. Results In this review, we discuss the key clinical challenges and unmet needs, and present insights on patients’ and families’ perspective on their daily life with CHI. Prevention of neurocognitive impairment and successful management of patients with CHI largely depend on early diagnosis and effective treatment by a multidisciplinary team of specialists with experience in the disease. Conclusions To ensure the best outcomes for patients and their families, improvements in effective screening and treatment, and accelerated referral to specialized centers need to be implemented. There is a need to develop a wider range of centers of excellence and networks of specialized care to optimize the best outcomes both for patients and for clinicians. Awareness of the presentation and the risks of CHI has to be raised across all professions involved in the care of newborns and infants. For many patients, the limited treatment options currently available are insufficient to manage the disease effectively, and they are associated with a range of adverse events. New therapies would benefit all patients, even those that are relatively stable on current treatments, by reducing the need for constant blood glucose monitoring and facilitating a personalized approach to treatment.

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Section: Main Textmentioning

confidence: 99%

Congenital hyperinsulinism in infancy and childhood: challenges, unmet needs and the perspective of patients and families

Banerjee

Raskin²,

Arnoux

et al. 2022

Orphanet J Rare Dis

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“…Health insurance barriers are amplified in rare diseases in which access to the very few experts, affiliated with a small number of centers of excellence, may not be covered by the selected insurance plan [9]. Less than 5% of rare diseases have treatments available [10].…”

Section: Introductionmentioning

confidence: 99%

“…Less than 5% of rare diseases have treatments available [10]. Treatments often are specialty drugs that are expensive [9]. For many rare diseases, there are no disease management guidelines making it difficult for caregivers to demonstrate the need for supportive services and insurance coverage [9].…”

Section: Introductionmentioning

confidence: 99%

Health insurance literacy and health services access barriers in Niemann–Pick disease: the patient and caregiver voice

Diaz

Crowe

Hopkin

2022

Orphanet J Rare Dis

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Background Major challenges to health care access include low health insurance literacy, prohibitive costs, and insurance barriers. Niemann–Pick disease (NPD), comprising acid sphingomyelinase deficiency (ASMD) and Niemann–Pick type C (NPC), is a group of rare, autosomal recessive, highly heterogeneous, neurovisceral, life-threatening, relentlessly progressive lysosomal disorders. Patients experience debilitating systemic and neurological symptoms and substantial emotional and financial stress. Currently, these multifaceted disorders are managed symptomatically as there are no approved therapies. Given the considerable disease burden of NPD, timely access to quality health care is paramount for improving outcomes in these life-threatening disorders. Understanding health insurance literacy and access challenges among patients with NPD and their caregivers is a first step to overcoming treatment barriers. Results Patients from the Niemann–Pick community participated in a health insurance literacy survey and follow-up telephone interviews on perceived access challenges. Of the 79 respondents who completed the survey, 67 participated in interviews. All respondents had stable health insurance coverage. However, 61% of respondents were unaware of Medicaid waivers and did not avail of them. Overall, 50% of respondents with childhood onset NPC selected Medicaid/Medicare and private insurance; 35% utilized Medicaid waivers. Most respondents with ASMD had private insurance only. Although the Niemann–Pick community demonstrated greater health insurance literacy than the general population, knowledge gaps exist in calculating insurance coverage, out-of-pocket maximums, and defining a formulary. The most frequently cited access burden was the process of obtaining medical care and services. Among respondents with ASMD, the greatest access burden was fear of unavailability of or access to medications and treatment. Access challenges adversely impacted patients’ mental health and exacerbated physical symptoms. Delays and denials in obtaining essential medication, equipment, and services contributed to disease progression. Caregivers faced burnout and often questioned the utility of their advocacy. Conclusions This study identified knowledge gaps in health insurance literacy and challenges to access medication and health care services among individuals impacted by NPD. Patients and caregivers need the knowledge and skills to navigate a complicated health care system, understand their rights to medication and services and, ultimately, benefit from improved outcomes, especially in a post–drug approval era.

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“…HIGR Participants are more likely to be from North America and younger than non-participants in the HI community. There is the potential for recall bias; however, rare disease patients and caregivers are very involved in disease management ( 17 – 19 ), which increases the likelihood of accurate responses.…”

Section: Discussionmentioning

confidence: 99%

Global Registries in Congenital Hyperinsulinism

Pasquini¹,

Mesfin²,

Schmitt³

et al. 2022

Front. Endocrinol.

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Congenital hyperinsulinism (HI) is the most frequent cause of severe, persistent hypoglycemia in newborn babies and children. There are many areas of need for HI research. Some of the most critical needs include describing the natural history of the disease, research leading to new and better treatments, and identifying and managing hypoglycemia before it is prolonged and causes brain damage or death. Patient-reported data provides a basis for understanding the day-to-day experience of living with HI. Commonly identified goals of registries include performing natural history studies, establishing a network for future product and treatment studies, and supporting patients and families to offer more successful and coordinated care. Congenital Hyperinsulinism International (CHI) created the HI Global Registry (HIGR) in October 2018 as the first global patient-powered hyperinsulinism registry. The registry consists of thirteen surveys made up of questions about the patient’s experience with HI over their lifetime. An international team of HI experts, including family members of children with HI, advocates, clinicians, and researchers, developed the survey questions. HIGR is managed by CHI and advised by internationally recognized HI patient advocates and experts. This paper aims to characterize HI through the experience of individuals who live with it. This paper includes descriptive statistics on the birthing experience, hospitalizations, medication management, feeding challenges, experiences with glucose monitoring devices, and the overall disease burden to provide insights into the current data in HIGR and demonstrate the potential areas of future research. As of January 2022, 344 respondents from 37 countries consented to participate in HIGR. Parents or guardians of individuals living with HI represented 83.9% of the respondents, 15.3% were individuals living with HI. Data from HIGR has already provided insight into access challenges, patients’ and caregivers’ quality of life, and to inform clinical trial research programs. Data is also available to researchers seeking to study the pathophysiology of HI retrospectively or to design prospective trials related to improving HI patient outcomes. Understanding the natural history of the disease can also guide standards of care. The data generated through HIGR provides an opportunity to improve the lives of all those affected by HI.

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Navigating the U.S. health insurance landscape for children with rare diseases: a qualitative study of parents’ experiences

Cited by 29 publications

References 53 publications

Congenital hyperinsulinism in infancy and childhood: challenges, unmet needs and the perspective of patients and families

Congenital hyperinsulinism in infancy and childhood: challenges, unmet needs and the perspective of patients and families

Health insurance literacy and health services access barriers in Niemann–Pick disease: the patient and caregiver voice

Global Registries in Congenital Hyperinsulinism

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