Impact of mental health in persons living with rare disease: Findings from the AMCP Market Insights Program

Abstract

Within the framework of its Market Insights Program, AMCP convened a panel of experts representing diverse stakeholders to identify alterations to plan design and/or coverage options geared toward improving the diagnosis and treatment of mental health conditions among persons living with rare diseases (PLWRD). PLWRD face unique mental health challenges because of the misunderstood nature of their conditions, potential misdiagnosis, and limited treatment options. Economic burdens arise from increased medical needs, reliance on caregivers, and work disruptions. The interplay of these factors, along with health insurance coverage, creates a distinctive mental health landscape for PLWRD and a need to prioritize mental health support for this patient population. This article aims to (1) summarize expert perspectives on health care system challenges and areas of agreement concerning the management of mental health conditions and (2) advance payers’ understanding of their role in supporting mental health care for patients with rare diseases. Addressing mental health needs of PLWRD presents multifaceted challenges. Managed care organizations play a pivotal role in supporting mental health care for PLWRD through their quality improvement initiatives and policies for coverage and reimbursement, which can impact both the rare disease treatment and mental health services PLWRD receive.

Plain language summary

People with rare diseases often have mental health challenges related to their health care. These vary by disease, but there are common issues that make it difficult for individuals and their families, including delays in getting diagnosed, not knowing what will happen in the future, having limited treatments, and facing financial difficulties. Health care organizations can help by supporting collaborative care models, helping patients see the right doctors to get treatment, and measuring how well programs or treatments work.

Implications for managed care pharmacy

Addressing mental health in rare diseases poses challenges that require a thoughtful and comprehensive approach. Managed care can support improvements in care by recognizing the unique mental health needs of individuals with rare diseases and the impact on patient outcomes and total cost of care. This includes ensuring coverage for mental health services, mental health screening, and use of team-based care. Expanding coverage and reimbursement for rare disease and mental health treatments may also be needed to support appropriate access.

The Orphan Drug Act defines a rare disease as a disease or condition that affects fewer than 200,000 people in the United States.¹ Examples of rare diseases include genetically linked cancers, cystic fibrosis, and debilitating pediatric conditions like Gaucher disease and spinal muscular atrophy. Despite the relatively small patient populations for each individual rare disease, there are an estimated 7,000 known rare diseases, impacting nearly 30 million Americans.² When available, treatments for rare diseases have been shown to provide larger health gains on average than drugs for more common conditions.³ Health gains were defined as incremental quality-adjusted life-years characterized by combining longevity and quality of life and associated with a new drug compared with prevailing treatment options at the time of drug approval. Unfortunately, more than 90% of rare diseases still lack any disease-specific treatment approved by the US Food and Drug Administration (FDA), and the unmet need for patients and families across many rare disease areas remains high, including prolonged diagnostic journeys, limited treatment options, and a huge psychosocial burden due to the lack of coordinated, integrated care.^4-6 Many rare diseases are life-threatening or life-limiting, and patients and caregivers face unique challenges.

Persons living with rare diseases (PLWRD) may have unique experiences with mental health, influenced by the challenges they encounter. Beyond contending with the physical symptoms of their conditions, these individuals also experience the psychological strain of living with a disease that is frequently misunderstood, is prone to misdiagnosis, and may lack viable treatment options. Additionally, a lack of prioritization of mental health well-being during rare disease diagnosis may exacerbate patients’ overall health outcomes. PLWRD often face significant economic burdens due to higher medical needs, often requiring the assistance of a caregiver, and having to miss work. The complex interaction among these factors, along with the constraints of health insurance coverage—such as inadequate access to mental health services—can result in suboptimal outcomes and increased expenses for both the plan and the patient.

To understand the health care system challenges and areas of agreement concerning the management of mental health conditions, AMCP convened an expert panel of stakeholders through its Market Insights Program in December 2023. Panelists included representatives from national and regional health plans (n = 3), integrated delivery systems (n = 1), psychiatrists (n = 2), and patient advocacy representatives (n = 4). The discussion centered on findings from an AMCP and National Organization for Rare Disorders (NORD) survey of NORD members, which explored the need for mental health services in rare diseases, sought insights into mental health care delivery, and identified potential gaps. The panelists also discussed the consequences of untreated mental health on patient outcomes and overall health care costs.

Rare Disease Advocacy Organization Survey

The survey of NORD members was conducted among rare disease advocacy organizations (n = 27) on the impact of mental health in PLWRD (survey available in Supplementary Materials ^{(108KB, pdf)} , available in online article). The results highlighted that the top ranked challenges in order are access to specialists or subspecialists (78%), high out-of-pocket expenses (70%), obtaining prescribed medications (52%), and timely responses to prior authorization requests (41%). A majority (74%) of rare disease advocacy organizations reported a “very prevalent” need for mental health services in the communities they serve, with one-third (30%) responding that “very often” patients report limited or no access to mental health services. A minority of organizations (14%) report having specific language relative to the mental health needs of their communities in current disease treatment guidelines.

Rare disease advocacy groups aim to enhance their understanding of various health insurance issues, including mental health.⁷ The NORD member survey respondents ranked the primary areas they would like to understand better about health insurance: approximately one-third (27%) ranked regulatory impact as the top priority, followed by 23% who ranked opportunities for engagement, 15% who ranked coverage trends, and 12% who ranked the key cost drivers influencing health insurance design and decision-making processes. The panelists addressed the concerns by proposing proactive and collaborative strategies with advocacy groups aimed at enhancing communication and aligning insurance services more effectively with the needs of PLWRD.

Mental Health of PLWRD and Caregivers

PLWRD and their caregivers report significant impacts on mental health (Figure 1).^8-10 Recently, a systematic review and meta-analysis of affective and anxiety disorders in adult patients with rare conditions reported prevalence estimates (pooled prevalence estimates of 13.1% [95% CI = 9.6%-17.7%; P < 0.001] for current major depressive disorder, 21.2% [95% CI = 15.4%-28.6%; P < 0.001] for current affective disorders, and 39.6% [95% CI = 25.5%-55.6%; P < 0.001] for current anxiety disorders), which were found to be higher than those in the general population.¹¹ Similarly, a cross-sectional study found that 42% of patients reported depression scores indicative of moderately or severely elevated symptoms, and 23% reported anxiety.¹² Challenges that are particular to rare conditions and that negatively affect mental health include limited knowledge of the condition among health care professionals, difficulty in accessing medical information, challenges in treatment, lack of psychosocial support, and difficulty coping with stigma and uncertainty.⁹

FIGURE 1.

Emotions Attributed to Living With a Rare Condition⁸

Many rare diseases present with unpredictable symptoms, contributing to heightened uncertainty for patients. The day-to-day unpredictability of their health status often results in elevated frustration, anxiety, depression, and feelings of hopelessness.¹³ Social isolation, coupled with a lack of understanding or support from both social networks and health care providers, can further exacerbate feelings of depression or anxiety, underscoring the significance of social support in alleviating psychological distress.¹⁴

“It’s such a trial and tribulation to get diagnosed with a rare condition itself that the mental health aspects of that condition typically are considered secondary to the quest for the diagnosis itself.” – Patient Advocate, Market Insights panelist

Financial challenges represent a significant aspect of the rare disease landscape, encompassing expenses related to medical care, transportation, and time away from work.¹⁵ These financial burdens not only compound existing mental health issues but also act as barriers to patients seeking necessary care and support. Furthermore, the scarcity of medical professionals possessing expertise in treating rare diseases limits access to care, resulting in prolonged wait times and potential delays in treatment initiation. Even when treatment options are available, the substantial cost of medications or therapies amplifies the financial burden, adding to the stress and anxiety experienced by patients.

“Families with a chronic disease will even stay with a suboptimal employment situation to avoid having to change health insurance and lose access to current providers.” – Health Plan, Market Insights panelist

CASE STUDY: KRABBE DISEASE CAREGIVER JOURNEY

Krabbe disease, also known as globoid cell leukodystrophy, is a rare autosomal recessive disorder resulting from a deficiency of the enzyme galactocerebrosidase.¹⁶ The majority of individuals with Krabbe disease exhibit symptoms during the first 12 months of life, although approximately 10% may present later, even in adulthood. Most patients experience peripheral motor-sensory neuropathy, which can be the initial symptom in cases with later onset. The early-onset forms primarily manifest symptoms associated with dysfunction in the central nervous system. In infantile-onset Krabbe disease, symptoms typically emerge between the ages of 2 and 12 months and include irritability, feeding difficulty, reflux, developmental delay, limb spasticity, axial hypotonia, optic atrophy, and decreased growth.¹⁷ The panelists heard the lived experience of 2 parent caregivers for children with infantile-onset Krabbe disease.

One significant barrier to the timely diagnosis of Krabbe disease is that the neurodiagnostic tests (eg, magnetic resonance imaging, lumbar puncture, and electrodiagnostic examinations) are difficult to obtain in newborns/infants and may not be rapidly available. Newborn screening aims to promptly identify cases, facilitating early treatment with hematopoietic stem cell transplantation, which has been linked to improved outcomes if conducted before symptom onset or in minimally symptomatic patients. Recognizing the importance of timing the initial referral after newborn screening, the speed of diagnostic confirmation of infantile disease, and ensuring the transplantation center’s capacity to promptly address newly diagnosed infantile Krabbe disease, advocates stress the urgency of early identification for timely intervention.¹⁸ Delays in initiating hematopoietic stem cell transplantation can result in diminished clinical outcomes.

Parent caregivers conveyed that emotional distress stemmed from a lack of comprehension about the newborn screening process, resulting in delays in crucial care, and the limited information initially provided by their health care provider. Caregivers described feeling that they did not have adequate training or preparation for the level of care their child needed to thrive. The trauma experienced while seeking care for a loved one with a rare disease can lead to a significant change in perspectives and beliefs about the world, oneself, and one’s partner. The long-term stresses can cause interpersonal relationship challenges that may lead to separation and divorce. This transformation unfolds as challenges are confronted, giving way to emotions of vulnerability and powerlessness. Several panelists, who were also parents or caregivers of a PLWRD, described suffering from posttraumatic stress disorder (PTSD) because of caring for their child with a rare disease or the end of their marriage due to the stresses placed on their relationship.

“Parents should not have to fight so hard to save their child’s life… That is where my PTSD comes from.” – Parent of child with rare disease, Market Insights panelist

Some of the panelists who were parents of PLWRD noted that care delays resulting from insurance issues might have adversely impacted the outcomes for their children. Irrespective of the insurance type, maneuvering through insurance coverage is a time-consuming and exasperating process, involving authorizations, denials, appeals, and repetitive submission of documentation. Concerns about access to care and potential inequities of treatment were also raised. In response, the expert panelists advised that when dealing with a rapidly progressing rare disease and anticipating a high likelihood of treatment approval, payers should proactively assess the evidence and establish policies ahead of time.

However, it was noted by several panelists that some payers institute new-to-market coverage policies that block routine insurance coverage for new drugs for up to 180 days after FDA approval. Although in principle these blocks can be justified to allow an insurer adequate time to review the clinical evidence, discuss with clinical experts, and prepare special delivery or other policies, in practice many insurers now place new-to-market blocks on any new specialty drug. Given the importance of early treatment for many rare diseases, panelists commented that payers will need to recognize their responsibility to ensure that coverage is available when treatment is needed. This preparation is facilitated when manufacturers engage with payers in preapproval information exchange to facilitate the establishment of coverage policies.¹⁹ This proactive approach can prevent any delays in providing insurance coverage for care.

Health Care System Challenges

A significant proportion of PLWRD in need of mental health care encounter limited access to mental health services. Factors including stigma, shortages of mental health professionals, and fragmented service delivery models together contribute to the prevailing gaps in mental health care. The panelists identified and discussed several gaps in mental health care for PLWRD and used a conceptual framework, created for the Market Insights Program, on managed care and behavioral health to aid in the panel discussion (Figure 2).

FIGURE 2.

Conceptual Managed Care Behavioral Health Framework

SHARED DECISION-MAKING

The panelists examined a shared decision-making intervention created at Children’s National, a pediatric health system in Washington, DC, to ensure that treatment plans are in harmony with the preferences and values of patients and their families. The panelists discussed information presented around the primary 5 priorities identified by families participating in the FAmily CEntered pediatric Advance Care Planning (FACE-Rare) program.²⁰ The outcomes indicated the following priorities: having personal time during the day; addressing financial, legal, or work-related concerns; receiving practical assistance at home; understanding what to anticipate in the future while caring for their child; and managing worries and emotions.

Shared decision-making empowers patients and families by providing a degree of control in situations where control is typically limited. The panelists discussed the role of this problem-solving approach in managing treatment decisions to not only reduce anxiety and disease-specific symptoms in patients facing serious illnesses but also improve feelings of peace and meaning for both patients and family caregivers. The panelists also conveyed that families who engage in advance care planning often exhibit improved health outcomes, both physically and emotionally.

MENTAL HEALTH SCREENING

The panelists underscored the significance of early identification and intervention in addressing mental health challenges among caregivers to minimize the impact on family interactions and mental health. As an illustration, the American Academy of Pediatrics advocates for pediatricians to screen mothers for postpartum depression during the infant’s 1-, 2-, 4-, and 6-month well-child visits.²¹ During discussions, the payer panelists highlighted the issue of health care system fragmentation concerning coverage for screening tools when the caregiver and patient are not under the same insurance provider. A proposed solution involved using case managers to investigate whether the caregiver has insurance coverage from a different provider and leveraging that coverage for mental health screening.

“We need better support, not only for patients but for the parents who are enduring the trauma over and over again of watching their child not only suffer, but then pass away. It has been a brutal journey.” – Parent of a child with a rare disease, Market Insights panelist

BEHAVIORAL HEALTH WORKFORCE

The panelists detailed how a large portion of the population in the United States still faces challenges in accessing care and treatment for mental health issues. Even for those with insurance, challenges such as a shortage of available providers, insufficient insurance coverage, high out-of-pocket costs, and fragmented care continue to be prevalent.

Only 28% of the US population resides in regions where there is an adequate number of psychiatrists and other mental health professionals to fulfill the needs of the population.²² The majority of states have less than 40% of the required mental health professionals, and several states have less than 10% of behavioral health needs being met (Figure 3).

FIGURE 3.

Mental Health Care Health Professionals Shortage Areas: Percent of Need Met, as of November 1, 2023²²

“One goal is putting behavioral health in everything we do, and I believe that if we don’t address the emotions of any type of illness, that the outcomes are less than desirable, and the costs are higher and so my work is really to add behavioral health to everything we do.” – Health Plan, Market Insights panelist

HEALTH CARE SYSTEM FRAGMENTATION

Health care system fragmentation can significantly impact mental health services. The panelists discussed the effect that mental health and physical health have on each other. System fragmentation often leads to a lack of coordination among different health care providers and services. Fragmentation was noted to contribute to delays in accessing mental health services. When different components of the health care system operate independently, referrals, appointments, and communication between providers may be slow, causing patients to wait longer for essential mental health interventions. The panelists also explored the impact of vertical integration, the business arrangement in which a company controls different stages along a supply chain, highlighting it as an instance where some health care organizations are working to mitigate fragmentation.

“We started our own chain of primary care clinics because there was a shortage. We learned that there were areas where people waited weeks to months to establish care with a primary care clinic. So, we prioritized it and there are a lot of payers that now have this kind of vertical integration.” – Health Plan, Market Insights panelist

EMPLOYER ENGAGEMENT

Employer-sponsored insurance covers almost 159 million non-senior people.²³ Approximately two-thirds of Americans who obtain coverage through their employer do so in self-insured plans. Self-funded plans are those in which companies choose to pay for some or all the health services of their workers directly rather than purchasing health insurance for them. The panelists discussed the important role employers can play in shaping health care systems through their direct efforts to institute system change, their negotiations with health plans, and their decisions concerning the structure of health benefits for their employees; as such, they play a key role in addressing gaps in mental health care and assuring access to life-changing treatments. Employers and other plan sponsors are encouraged to use reinsurance and financial protection programs provided by payers to effectively handle the expenses associated with rare disease treatments.

“So, there are many stop-loss policies that are running separate riders for a variety of therapies. Gene therapies are a perfect example. They’re not covering it, they exclude it. In the future, I would expect there to be separate riders for gene therapies.” – Health Plan, Market Insights panelist

GENE THERAPY CONSIDERATIONS

The introduction of gene therapy holds significant promise as well as substantial uncertainty for a community marked by the profound impacts of rare diseases. Choosing to undergo a gene therapy treatment necessitates an informed, comprehensive, and collaborative decision-making approach.

“We are changing the course of rare diseases with these gene therapies. And so, I see it as alleviating the mental health burden to some extent; there’s still going to be the trauma of a diagnosis, but the difference is it is trauma with hope instead of just trauma.” – Patient Advocate, Market Insights panelist

A patient-reported tool assessing mental health after gene therapy was presented to the panel as an example of how complementary tools can be used to assist in assessing the mental health outlook associated with receiving gene therapy. The coreHEM Mental Health Outlook questionnaire (coreHEM-MHO) is a patient-reported outcome measurement instrument associated with receiving gene therapy or any durable treatment for hemophilia.²⁴ coreHEM-MHO consists of 26 questions in 2 parts. The first 20 questions are suitable regardless of treatment therapy. The additional 6 questions are specific for those considering or who have been treated with gene therapy. It is recommended that coreHEM-MHO is administered at baseline (before treatment) and then again every 3 months for the first year. Scores from different time points can be compared to demonstrate changes in mental health outlook. The coreHEM project aimed to determine the set of outcome measures required to evaluate efficacy, safety, comparative effectiveness, and value of gene therapy for hemophilia.²⁵ The panelists stated that this kind of information is valuable for assessing the effectiveness of interventions, understanding the impact on patient outcomes, and making informed decisions regarding coverage and reimbursement.

“And I think it’s important, like the early intervention and screening, for mental health to also be considered. To open conversations about emotional well-being is important because it’s a stressful process to go through gene therapy, it could be you will have side effects, or it could be the burden of coordination of treatment—that kind of stuff adds up.” – Psychiatrist, Market Insights panelist

Managed Care Role in Optimizing the Management of Mental Health in Rare Diseases

The experts quantified the current state of addressing mental health requirements for individuals impacted by rare diseases, using a scale ranging from 1 to 5, where 1 signifies the lowest state of addressing mental health needs and 5 was the highest rating. The panelists’ assessment indicated there is currently a low level of attention to addressing mental health needs, with an average rating of 1.7 on where we are today in addressing mental health needs for those affected by rare disease. The conversation regarding managed care’s contribution to optimizing the management of rare diseases and the corresponding mental health needs focused on tackling the recognized challenges within the health care system.

QUALITY IMPROVEMENT

In addition to motivating providers with value-based reimbursement models to enhance mental health screening, payers emphasized the necessity of a national quality measure to significantly influence screening at a broader, national level. The Patient-Reported Outcomes Measurement Information System was proposed as an organizational intervention or plan-level initiative that could serve as a quality improvement effort in the interim. The Patient-Reported Outcomes Measurement Information System comprises a set of highly reliable and precise measures capturing patient-reported health status across physical, mental, and social well-being.²⁶ The panelists expressed interest in being able to also use this kind of data system to identify, and ultimately address, caregivers’ mental health needs.

“If the whole family is insured by the same plan, and payers recognize it in the data, it may stimulate more outreach to see if one can intervene to provide support to improve the care of the caregiver.” – Health Plan, Market Insights panelist

MULTIDISCIPLINARY COLLABORATIVE CARE

Coordinating essential services poses a significant challenge for PLWRD. Typically, managing a rare disease involves engaging multiple specialists, necessitating various appointments that demand coordination and attendance by families. Many of these families may lack experience in navigating complex care settings. The panelists discussed the growing emphasis on treating the whole health of a patient, which emphasizes the importance of addressing all aspects of health in patient care and is founded in interprofessional and team-based care approaches.²⁷

“The point about team-based care is crucial and it ties back to using the transplant model, which leads you down the path of case rates or of value-based payment models. I think it’s clear that all these illnesses are much too complex for one clinician to manage.” – Health Plan, Market Insights panelist

The Ryan White HIV/AIDS Program, Collaborative Care Model (CoCM), and Co-Located Care model were outlined for the panelists to consider reshaping how mental health services are provided.^28,29 The panelists underscored the importance of providing team-based care for PLWRD and pointed to the Ryan White HIV/AIDS Program as a model for delivering such collaborative care. Serving as the nation’s safety net program for individuals with HIV, this program provides outpatient HIV care, treatment, and support services to those lacking health insurance, addressing gaps in coverage and costs for those with insurance limitations. Program recipients gain access to primary medical care, medication, and essential support services including mental health professionals.

The CoCM is a multiprofessional health care model where primary care physicians, specialists, and other health professionals work together to treat complex or chronic illnesses, especially mental health conditions. Several randomized controlled trials have demonstrated that collaborative care increases access to mental health care and is more effective and cost-efficient than the current standard of care for treating common mental illnesses.³⁰ Large health care systems and organizations have begun to adopt CoCM and are seeing improved treatment outcomes and provider and patient satisfaction.³¹ The panelists concurred that the CoCM is both an effective and efficient model to deliver integrated care.

Co-Located Care is a model that involves physically situating behavioral health professionals within primary care/other specialty settings to facilitate more integrated, team-based care. The panelists discussed how integrating behavioral health professionals in the same location can mitigate patient hesitancy to seek mental health care, effectively addressing concerns tied to mental health stigma. Leveraging telehealth to improve access to care was seen as crucial to reducing the fragmentation of mental health care, especially in areas designated as Health Professional Shortage Areas by the Health Resources and Services Administration. The benefits of the Co-Located Care model were seen as improved access to mental health services, greater convenience for patients, enhanced communication between providers, and more holistic care. The significance of collaborating with local children’s hospitals was also emphasized, recognizing the importance of addressing initial mental health challenges faced by patients and their caregivers early in the diagnostic journey.

“You know we’re way behind the Ryan White program model, where we had multidisciplinary teams—we had a psychiatrist and a therapist for every patient and a case manager. So that’s the model I hold out there, but it’s not there for kids with rare diseases.” – Psychiatrist, Market Insights panelist

CENTERS OF EXCELLENCE

The panelists described how an increasing number of commercial and employer-sponsored health plans are adopting Center of Excellence (COE) programs to offer specialized care for specific health services to their enrollees, including those focusing on gene therapies and behavioral health services. COE programs identify providers or facilities based on the quality and cost of care they deliver. Providers within COE programs typically specialize in specific services and may offer additional case management and support services for patients. COE programs have often been used for high-cost specialized treatment, such as transplant and gene therapy. Selection of participating providers may be based on outcomes such as lower readmission rates or additional accreditations earned. The structure of COE programs varies among plans, with some limiting coverage to participating providers and others offering lower cost-sharing for enrollees using providers within the program and providing patient and family travel support to access a COE. COE programs might be a way for payers and employers to direct patients to specific behavioral health providers or specialized treatment centers and manage costs.

“We’ve spent a lot of time thinking about the logistics of people living in outlying areas. And as cell and gene therapies have come along, that, of course, has focused everybody’s attention. So, these centers of excellence and concierge services that will handle a family’s logistics are important. The family already has enough to deal with medically.” – Health Plan, Market Insights panelist

POLICY AND ADVOCACY

National efforts to improve the management of mental health can be seen in new policies introduced by the Centers for Medicare & Medicaid Services as part of the Calendar Year 2024 Physician Fee Schedule Final Rule, Calendar Year 2024 Hospital Outpatient Prospective Payment and Ambulatory Surgical Center Payment Systems Final Rule with Comment Period, and Contract Year 2025 Proposed Medicare Advantage and Part D rules.³² These represent some of the most notable shifts in promoting access to behavioral health in the history of the Medicare program and include bridging coverage gaps in behavioral health access, broadening the behavioral health workforce to incorporate mental health counselors, and ensuring more precise payment for behavioral health services.³² The panelists noted that each of these changes could play a crucial role in significantly enhancing access to behavioral health care for individuals covered by Medicare.

A panelist also shared information about the proposed legislation, H.R.2412—Helping Kids Cope Act, aiming to enhance children’s access to care by offering pediatric behavioral health services at children’s hospitals and through community-based providers. This legislative initiative is seen as a positive step, as it seeks to authorize grants for addressing the substantial numbers of children boarding in emergency departments, supporting the pediatric behavioral health workforce, and addressing other related purposes.³³

“The advocacy and the policy efforts need to be strong. So, if we’re really going to whole health, we need to make that a priority. We need to advocate for comprehensive care models, and we need to also put money into research to look at integrative health approaches.” – Psychiatrist, Market Insights panelist

Finally, given the high cost of cell and gene therapies and the uncertain long-term outcomes, the panelists expressed that it is reasonable for payers to use prior authorization as a component of coverage. However, they further clarified that prior authorization criteria should be based on clinical evidence and that it should include input from clinical experts and patient groups. They emphasized that the authorization process should be transparent, easily accessible, efficient, and prompt for both patients and providers to minimize any undue impact on mental health.

Additionally, numerous panelists advocated for actively involving biopharmaceutical manufacturers in fostering public dialogue on the accessibility and equitable pricing of life-altering treatments, which panelists suggested may lessen the need for prior authorizations. They again emphasized the importance of incorporating input from patients and various stakeholders.

PATIENT AND CAREGIVER SUPPORT

Managed care professionals can play a crucial role in supporting patients and caregivers dealing with rare diseases by staying up to date on research, resources, and information on the behavioral implications of these conditions. One resource highlighted by the panelists is the special issue titled Psychosocial Considerations for Children and Adolescents Living with Rare Diseases, which explores the psychosocial needs of children and adolescents living with a rare condition and interventions that have been developed to address their needs.³⁴

Summary

AMCP organized an expert panel of stakeholders and collaborated on a survey of NORD members through its Market Insights Program to better understand the complexities of the health care system and identify opportunities regarding the mental health needs of PLWRD. The role of managed care in improving the management of mental health in rare diseases involves implementing programs to improve mental health screening, endorsing the use of behavioral health collaborative care models and COE programs for high-investment treatments, and ensuring that utilization management programs do not delay access to recommended treatments. Payers can also support the use of telehealth, which plays a crucial role in addressing mental health needs by providing accessible and convenient mental health services remotely. Payers can contribute to fostering data collection and monitoring outcomes of mental health care and high-investment treatments with the use of standardized assessment tools. These data could prove valuable in evaluating the efficacy of interventions, comprehending their impact on patient outcomes, and making well-informed decisions regarding coverage and reimbursement.

Funding Statement

This supplement was conceptualized by AMCP and Michelle Rice and Associates, LLC. The AMCP Market Insights Program described in this supplement was funded by Takeda and Sanofi. Takeda and Sanofi both manufacturer multiple medications for the treatment of rare diseases. Publication of the supplement was funded by AMCP.