Living with ALS (2024)

4

Creating a Sustainable and Accessible ALS Clinical Care and Research System

ABSTRACT

This chapter discusses the importance of establishing an integrated, nationwide system of care and research for individuals living with amyotrophic lateral sclerosis (ALS) as well as at-risk genetic carriers. The two goals for this system will be (1) to ensure all individuals with ALS and at-risk genetic carriers, regardless of where they live and the resources at their disposal, have equal access to high-quality, evidence-based care, and (2) for such care to be integrated with research, including health services, care delivery research, and clinical research to develop biomarkers and new therapeutics. This is important, as the number of individuals diagnosed and living with ALS in the United States continues to rise. The care and research system should build on existing facilities and ultimately constitute an integrated network linking comprehensive, regional, and community or local ALS centers. Each network level should have defined clinical care services and research capabilities and be held accountable for reducing access delays in their area, reaching underserved populations, and ensuring continuity of care throughout an individual’s ALS disease course. Creating this network will require urgently expanding the number of clinics providing multidisciplinary care, growing and supporting the ALS multidisciplinary workforce, increasing capacity for early referrals and consultations for timely ALS diagnosis and access to personalized, standard-of-care therapies, and establishing new payment models to support the system of ALS clinics.

The committee believes that every individual living with ALS deserves early and continuous access to multidisciplinary,¹ state-of-the-art care to help them lead longer lives, remain functionally independent, and optimize overall quality of life. Multidisciplinary clinics provide coordinated, team-based management across multiple medical and allied health specialties and serve as a one-stop shop for complex multisystem diseases, such as ALS. Today, there are only around 200 multidisciplinary ALS clinics in the United States that provide high-quality care. That is too few to serve all individuals with ALS. The clinics are also insufficiently connected to coordinate the availability of specialized ALS expertise and research efforts to every person with ALS. There is also no definitive count of the number of people living with ALS today who receive evidence-based standard of care at a multidisciplinary clinic but one estimate from the ALS Association (ALSA) suggests that it is at best about half of the population.²

Multidisciplinary care visits require multiple hours and involve several health professionals, but this type of care is reimbursed at the same rate as a single specialist’s 30- to 60-minute office visit (Paganoni et al., 2017). This places a financial strain on ALS multidisciplinary clinics, hindering their ability to hire, retain, and expand the clinic staff to meet the needs of people living with ALS and their families. Today, larger ALS clinics rely heavily on philanthropy and institutional resources, which is unsustainable, exacerbates the significant variations in resources that exist across U.S. ALS clinics, and limits access to state-of-the-art care.

People living with ALS also need easy access to participation in cutting-edge clinical research trials without prohibitive cost or travel burdens. Over the past 5 years, discoveries about ALS genetics, disease targets, and disease pathways have increased clinical trial activity and biomarker and therapeutic development. However, the ALS clinical research system is disjointed, underresourced, and not sufficiently connected to the ALS clinical care team. Furthermore, there are substantial geographic, racial and ethnic, and socioeconomic disparities in access to, and participation in, clinical

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¹ As stated in Chapter 2, the committee notes that interdisciplinary, rather than multidisciplinary, is the more accurate term because interdisciplinary denotes that the various disciplines are coordinated toward a common and coherent approach, while multidisciplinary refers to the addition of the competencies of multiple professionals who stay within the boundaries of their fields (Choi and Pak, 2006). The Veterans Health Administration refers to the ALS interdisciplinary team in its directive on providing ALS care to veterans. The committee has chosen to use multidisciplinary in the report because it is the more widely used term.

² In an October 18, 2023, letter to the committee from the ALS Association, it is stated: “Although multidisciplinary ALS care can add nine months of life, it is woefully underfunded and often difficult to deliver. Only about half the people served at ALS Certified Treatment Centers of Excellence receive this well-established, evidence-based standard of care.”

trials, expanded access programs, and clinical research overall. In addition to serving more individuals with ALS, expanding access to clinical research will be critical for accelerating therapeutic discoveries for this disease and informing timely, evidence-based health policy changes.

In this chapter, the committee lays out the current obstacles to accessing high-quality ALS care and proposes a reenvisioned, newly coordinated system of care and research accessible to every individual with ALS. The committee recommends establishing a system of care built on a hub- and-spoke model that integrates clinical research as a key pillar of care across ALS clinics, similar to the system developed for cancer care. This nationwide system of care and research will leverage existing clinical and research expertise and infrastructure, along with the latest technological resources, to provide all individuals living with ALS, regardless of geography and resources, access to high-quality, standardized, evidence-based multidisciplinary care. This integrated system of care and research will also enable all individuals living with ALS, if interested, to participate in clinical research aimed at making ALS a livable disease within the next decade. A more complete discussion on accelerating ALS therapeutic development can be found in Chapter 5; however, this chapter explains how an expanded and integrated multidisciplinary care network could also serve as the foundation for building a robust infrastructure for the natural history studies and clinical trials needed to advance therapeutic development.

The chapter also recommends innovative strategies for addressing four major challenges to expanding access to ALS care. Meeting these challenges is necessary for advancing research for ALS as well as other neurodegenerative diseases. The four challenges are:

1. Achieving equity across racial, ethnic, and geographically diverse groups by reaching more people, particularly underserved populations, and helping them gain access to early and continuous care at an ALS multidisciplinary clinic;
2. Workforce training and education to expand the ALS workforce and enhance professional education and awareness of ALS among health professionals;
3. Financing multidisciplinary care and research by implementing adequate payment and reimbursement models for multidisciplinary care and research and studying multidisciplinary care outcomes in terms of their value to people with ALS, families, clinicians, and insurers; and
4. Connecting the U.S. Department of Veterans Affairs (VA) health care system and veterans living with ALS to the broader care and research system.

ALS MULTIDISCIPLINARY CARE

A multidisciplinary clinic serves as a single site for people living with ALS, confirming diagnoses; initiating and monitoring therapies, medications, and assistive technology devices; and managing multisystem symptoms. A multidisciplinary clinic also provides specialized health and supportive services to track disease progression, improve quality of life, and prolong functional independence. These services include occupational, physical, speech and language, and respiratory therapy; nutritional support; mental and behavioral health services; and durable medical equipment (DME). A multidisciplinary clinic also provides neuropalliative care services for individualized advanced care planning based on ALS disease progression.

Numerous studies have demonstrated that multidisciplinary care decreases 1-year mortality by as much as 30 percent, reduces hospitalizations and cost of care, improves quality-of-life outcomes, and increases patient satisfaction (Boylan et al., 2015; Chiò et al., 2006; Cordesse et al., 2015; Corr et al., 1998; de Almeida et al., 2021; Driskell et al., 2019; Galvin et al., 2017; Hogden and Crook, 2017; Traynor et al., 2003). As a result, there is consensus in the field that multidisciplinary care should be the standard of care for all individuals living with ALS (Andersen et al., 2005; Hogden and Crook, 2017; Miller et al., 2009).

Variation in Access to ALS Centers

In contrast to the evidence supporting the usefulness and effectiveness of multidisciplinary care, evidence regarding the availability and use of multidisciplinary care in ALS is limited. ALSA estimates that approximately 12,000 people living with ALS receive care at a multidisciplinary clinic every year, representing about 40 percent of the overall U.S. ALS patient population, as estimated by the Centers for Disease Control and Prevention (CDC) (ALSA, 2023; Mehta et al., 2023). This 40 percent is a rough estimate that does not consider whether the care received was at a single visit or continuous, nor does it include people with ALS who receive care at a Muscular Dystrophy Association (MDA) specialty center or a VA clinic, both of which also provide multidisciplinary care for people with ALS. For comparison, the Cystic Fibrosis Foundation Patient Registry (see Chapter 5) estimates that 77 to 84 percent of people with cystic fibrosis in the United States attend a Cystic Fibrosis Foundation–certified clinic (Cromwell et al., 2023; Knapp et al., 2016).

ALSA lists 226 multidisciplinary clinics in the United States, certifying 89 of these as ALS Treatment Centers of Excellence and 18 as Recognized Treatment Centers.³ Both ALS Treatment Centers of Excellence and Rec-

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³ The complete list of multidisciplinary ALS clinics is available at https://www.als.org/support/certified-centers-clinics.

ognized Treatment Centers must meet certain standards regarding the care they provide (see Box 4-1). However, Recognized Treatment Centers may not offer opportunities to participate in ALS research. The other 119 are categorized as affiliated clinics, which deliver models of care other than those officially part of the ALSA Certified Center Program. These include both group and solo private practices. Alaska, Delaware, and Wyoming currently have no ALSA-affiliated treatment centers.

MDA also provides care to individuals living with ALS and their families because they usually have a care team with the skills needed to treat ALS as well as other neuromuscular diseases. MDA supports a care center network of more than 150 clinics nationwide. Some of these specifically serve people with ALS, whereas others are multidisciplinary neuromuscular clinics that care for individuals with ALS as well as other neuromuscular conditions. As of 2023, the VA system has nine ALSA certified Centers of Excellence (with three pending applications), two recognized treatment centers (with two pending applications), and 54 ALS multidisciplinary clinics.

While the literature is sparse, evidence suggests that many people living with ALS do not have access to multidisciplinary care. Specialty ALS clinics delivering multidisciplinary care are not uniformly distributed across the United States (see Figure 4-1), and travel distance to a multidisciplinary clinic is a commonly reported barrier for many people living with ALS (Schellenberg and Hansen, 2018; Stephens et al., 2015). A spatial analysis of 2013 data from the National ALS Registry found that 44.9 percent of people living with ALS in the United States lived more than 50 miles from a multidisciplinary clinic and nearly 25 percent were living more than 100 miles from a multidisciplinary clinic (Horton et al., 2018). However, in 2013,

there were only 72 multidisciplinary clinics across the country, including those operated by ALSA, MDA, and the Les Turner ALS Foundation (Horton et al., 2018). Today, ALSA alone has 89 Certified Treatment Centers of Excellence, which is its highest designation. Nevertheless, the literature reveals significant use of telehealth by people living with ALS, suggesting substantial demand for multidisciplinary care outside their range of travel (Haulman et al., 2020; Helleman et al., 2020). One result of a lack of universal access to a multidisciplinary clinic is that the average time from symptom onset to a patient’s first appointment at a multidisciplinary clinic in the United States is 19 months (Galvin et al., 2015).

Cost is another significant barrier to accessing multidisciplinary care, estimated at $70,000 to $180,000 annually for each person living with ALS in the United States (Gladman and Zinman, 2015; Obermann and Lyon, 2015). As discussed in Chapter 3, people living with ALS bear much of this cost out of pocket. Other cost-related issues include the varying acceptability of insurance at each clinic, as well as systems of referral and prior authorization. Barriers related to distance and cost are further compounded by marginalization across racial, ethnic, and income-related lines.

The limited data regarding the use and access to multidisciplinary care in the United States suggest that many people with ALS cannot access multidisciplinary clinics or face significant barriers such as travel and cost. Thus, it is critical to improve access to multidisciplinary care so every person with ALS can receive state-of-the-art, evidence-based care without an undue burden from travel or cost.

Variation in Multidisciplinary ALS Care Teams

The structure of multidisciplinary ALS clinics varies widely in terms of their size and the scope of care they provide. In addition, ALS clinic team requirements and expectations vary across VA, ALSA, and MDA clinics (see Table 4-1). The ALS multidisciplinary care team can vary depending on whether team members are based in the multidisciplinary clinic or whether they are part of the external network of medical specialists, in-patient or nursing care institutions, home health agencies, not-for-profit ALS groups, and other DME experts from whom ALS individuals and families may receive care.

TABLE 4-1 ALS Clinic Team Requirements and Expectations Across VA, ALSA, and MDA Clinics

SOURCES: MDA, 2023; Miller et al., 2009; VHA, 2021.

Each ALS clinic in the United States today operates almost entirely on its own, with little connection to other ALS clinics. Clinic staff also vary in size and specialty. The committee was unable to find peer-reviewed literature discussing the staffing models at different clinics. However, the following breakdown reflects the committee’s experience and information-gathering discussions:

• Smaller ALS clinics serving fewer than 100 people with ALS annually may have a core clinical team comprising an ALS specialist physician, a part-time physical therapist, and a part-time ALS nurse who staffs a half-day ALS clinic one or two times a month while performing other hospital and clinical duties during the remainder of the month.
• Larger ALS clinics serving from 100 to 1,000 people or more with ALS annually typically have a core clinic team comprising:
  • One to seven ALS specialists;
  • Up to six full-time or part-time ALS nurses;
  • Up to six full-time ALS nurse practitioners or other advanced practice providers;
  • Allied health clinicians, including one to four full-time or part-time ALS physical therapists, as many as two part-time ALS speech therapists and two part-time ALS occupational therapists; and
  • One to three full-time ALS patient service coordinators, up to six part-time ALS clinic coordinators for clinical outcomes collection, and one case manager or social worker.

If the resources are available, many larger clinics may have additional clinical staff such as electrophysiologists, geneticists and genetic counselors, respiratory therapists, palliative care specialists, pulmonologists, mental health professionals, interventional radiologists, care coordinators, house call nurses, and community health workers. These team members may be a direct part of the multidisciplinary ALS clinic infrastructure. Others may primarily provide care at another clinic within the same institution or system, while being connected to the multidisciplinary ALS clinic. The ALS specialist physicians staffing these clinics are generally neurologists with subspecialty training in neuromuscular disorders or neuropalliative care, or they may be physical medicine and rehabilitation specialists with training or experience in ALS.

The VA ALS Care System as a Potential Model for Removing Barriers and Care Delays

The VA ALS system of care developed organically following the granting of service connection for ALS with clinics following the American Academy of Neurology clinical practice guidelines. In 2021, VA partnered

with ALSA to increase the number of VA ALS clinics certified as Treatment Centers of Excellence.

VA’s ALS specialty clinics provide multidisciplinary care to veterans with ALS, who account for more than 16 percent of individuals living with ALS (Valor Healthcare, n.d.). Based in part on a 2006 Institute of Medicine report, VA decided in 2008 that any veteran who develops ALS and served more than 90 days in any military branch with an honorable discharge is eligible for VA care as a 100 percent service-connected condition (IOM, 2006).⁴ As a result, veterans with ALS and their families receive these benefits:

• All health care services, whether related to ALS or not;
• No copays for medical care, prescriptions, and DME;
• Power mobility, assistive technology, adaptive recreation, and respiratory equipment, with no limitations on when or how often equipment can be ordered;
• Monthly special disability compensation that ranges from approximately $4,000 to $9,000 per month;
• Paid home caregiver support, up to 65 percent of monthly skilled nursing facility costs;
• Special Adaptive Housing grant of more than $100,000 to cover modifications for home accessibility or to reduce mortgage and mortgage insurance payments;
• Adult day health and custodial care in the veteran’s home or by a contracted skilled nursing facility or ventilator-capable skilled nursing facility;
• A grant of more than $24,000 toward purchasing a wheelchair-capable van or other adaptive vehicle;
• Transportation to and from medical appointments and lodging when necessary; and
• Enrollment in VA’s Civilian Health and Medical Program, the federal health benefits program for family members of totally and permanently disabled veterans with a service-related disability.

A unique and important feature of the VA model versus non-VA ALS care (see Table 4-2) is that veterans with ALS are often spared the debilitating financial burdens related to receiving ongoing care, accessing pharmacological and nonpharmacological therapies, and maintaining DME. As discussed in Chapters 2 and 3, many people living with ALS face difficulties

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⁴ Presumptive Service Connection for Amyotrophic Lateral Sclerosis, CFR 38.3.318. https://www.ecfr.gov/current/title-38/chapter-I/part-3/subpart-A/subject-group-ECFR39056aee4e9ff13/section-3.318 (accessed May 10, 2024).

TABLE 4-2 Differences in VA Versus Non-VA ALS Care

^a 38 CFR § 17.417

^b Many centers varyingly offer clinical trials, expanded access programs, registry enrollment, natural history studies, and clinical biomarker research. There is a growing number of centers that are connected to central or smart Institutional Review Boards, have reliance agreements in place facilitating participation in multisite research operations, and have established clinical research infrastructure and processes. One example is the 74-site Healey ALS platform trial.

obtaining prior authorization and financial burdens from early and continued access to specialist consultations, medications, therapies, and care services.

INTEGRATING CLINICAL RESEARCH INTO THE ALS CARE DELIVERY NETWORK

Over the past decade, ALS drug development in the United States and globally has expanded rapidly, indicating industry interest in this disease. As of 2020, there were at least 120 experimental therapeutics and gene therapies in active ALS clinical trials with a global market estimated to reach $1.02 billion by 2032 (GlobalData, 2021; Market.US, 2023). Despite this, in the committee’s discussions with ALS clinical trialists, they heard that the difficulty of recruiting enough interested and willing people with ALS to a clinical trial slows the drug development process. The committee also heard that there are individuals with ALS interested in participating in clinical trials, but the restrictive inclusion criteria intended to enhance the scientific validity of the study also preclude some people with ALS from participating. Input from individuals with ALS and at-risk genetic carriers revealed multiple reasons they would want to participate in clinical trials. These include:

• Potentially receiving a new drug or intervention that is not available outside of a clinical trial;
• Contributing to drug development and science to help others who have or will develop ALS;
• Doing one’s part as a research participant when it is estimated that only 10 percent of people with ALS participate in clinical trials;
• Providing hope and purpose during a difficult disease course;
• Providing a sense of control by taking a more active role in one’s health care;
• Demonstrating to worried family that you are doing everything possible to improve your chance of survival;
• Setting an example for others of how to react to a life-altering illness;
• Creating more opportunities to learn about one’s own illness status and progress, and potentially receive more observations and data points to follow;
• Learning about potential new treatments one may have an opportunity to take in the future; and
• Because it feels good to be part of research to find new treatments.

Overall, studies have shown that less than 10 percent of people with ALS participate in clinical trials and research, in contrast to other rare diseases such as pediatric cancer, which has a 60 percent participation rate (Bedlack

et al., 2008; Gelijns and Gabriel, 2012; Mehta et al., 2021). A 2019 survey found that while 78 percent of individuals living with ALS would participate in clinical trials, only 20 percent knew a great deal about ALS clinical trials. Of those who had not participated in a clinical trial, 17 percent were unaware of any trials and 13 percent did not know how to find information about trials (Ipsos, 2019). The same survey reported that nearly 40 percent knew someone who could not afford to participate in a clinical trial because of travel costs, 25 percent feared participating, and nearly 75 percent were more likely to consider participating if they had a patient navigator or advocate at their primary hospital. In addition, there are racial and ethnic disparities in trial access and participation; less than 10 percent of ALS research participants are from underrepresented, minoritized populations (Raymond et al., 2019). Given these statistics, it would behoove the ALS community to make all individuals with ALS and at-risk genetic carriers aware of clinical trial opportunities early and universally and to encourage them to take advantage of the opportunity to participate in clinical trials.

One proven approach to increase clinical trial enrollment of a more diverse patient group is to create a coordinated, integrated network of sites, which would both provide clinical care for individuals with ALS and serve as a clinical trial and research network. Pediatric oncology, cancer, and cystic fibrosis models have shown that integrating research with clinical care and allowing every patient to participate in clinical trials—without increasing their travel or cost burden—accelerates progress, largely by increasing enrollment (Gelijns and Gabriel, 2012; Woodcock et al., 2021). The committee proposes a similar framework of integrating clinical trials into a clinical care network to address the current knowledge gap in ALS patients about clinical trials. While scientific breakthroughs and innovative trial designs are important, increasing trial participation and equity in access are equally important to accelerate ALS therapeutic development.

Another major obstacle to ALS trial and research participation is a narrow window of eligibility; many patients are diagnosed and placed on treatment late, at which point they fail to meet inclusion criteria, leading to missed opportunities. Many recent and ongoing clinical trials prioritize enrollment of early ALS patients within 18 months from symptom onset and that have been on standard-of-care medications for a few months before participation (Albanese et al., 2022; Paganoni et al., 2021).

Aside from the lack of patient knowledge of trials and diagnostic delay, a few other reasons might account for low enrollment in ALS clinical trials, including:

• As the number of FDA-approved ALS treatments expands, the existing therapeutic regimen can become more demanding for patients, making it more difficult to manage existing treatments and participate in clinical trials for new drugs.

• Clinical trials may be unattractive to individuals living with ALS because of the possibility of receiving a placebo instead of the study intervention, travel time to the trial site, financial burdens, and painful procedures or drug administration.
• Patients may distrust the medical system because of historic mistreatment and ongoing discrimination and inequities.

BUILDING THE IDEAL ALS CARE DELIVERY SYSTEM

Making ALS a livable disease requires diagnosing individuals earlier, in order to initiate evidence-based multidisciplinary care by ALS specialists immediately and continuously. At the same time, the progressive nature of the disease often makes travel difficult. Therefore, to facilitate continuous multidisciplinary care and support throughout their illness, individuals living with ALS will need access to centers at geographically accessible locations within their communities. However, it is not feasible to establish new stand-alone and dedicated multidisciplinary ALS centers to serve every person with ALS immediately near their home given resource and workforce restraints. However, existing resources may be leveraged and expanded to deliver care to more people with ALS closer to their homes. Thus, to increase access to state-of-the-art care and clinical research for every person with ALS, the committee recommends that a new ALS care system be established:

Recommendation 4-1: Build an inclusive and integrated ALS multidisciplinary care and research system.

The Centers for Medicare & Medicaid Services and the National Institute of Neurological Disorders and Stroke, in partnership with current ALS multidisciplinary care clinic system leaders (e.g., U.S. Department of Veterans Affairs, ALS Association, Muscular Dystrophy Association), and community-based providers should build an inclusive and integrated multidisciplinary care and research system for people living with ALS. This network should consist of:

1. Community-Based ALS Centers,
2. Regional ALS Centers, and
3. Comprehensive ALS Care and Research Centers.

This new system, modeled after “hub-and-spoke” systems of care and research for cancer and stroke, is designed to fill gaps in access to ALS care and research across the United States. The Comprehensive ALS Care and Research Centers will serve as “hubs,” centralizing oversight, while the Community-Based ALS Care Centers and Regional ALS Centers will serve as “spokes” (see Figure 4-2).

Each care setting in the network should provide defined clinical care services, enrollment in the National ALS Registry, and access to clinical research. They will also be accountable for achieving quality metrics. Their accreditation would be based on these capabilities and metrics, pursuant to nationally recognized quality metrics like those set and revised by the American Academy of Neurologists (AAN). This reimagined ALS care system will build on and strengthen the preexisting ALSA- and MDA-certified multidisciplinary clinic systems, centralizing oversight to ensure care quality, provide additional infrastructures to collect population health data (see Chapter 6), and coordinate care across levels.

Building on current ALS clinic accreditation programs, this system will bring in new clinics to increase the number of clinics providing this high-quality care. This includes community neurologists, private and group practices, as well as clinics that provide multidisciplinary care for neurological diseases other than ALS. Innovative approaches to bringing care to people with ALS will also be encouraged; examples may include expanded telehealth services, house call visits, and travel or satellite clinics. By bringing in this wide variety of care settings, areas without a large number of people with ALS will still have clinics available.

This system will also enhance participation in clinical research. As “hubs,” Comprehensive ALS Care and Research Centers will be expected to have substantial breadth and depth of ALS research, including preclinical, clinical, preventive, and population-based research. Regional and even community-based centers without the capacity to lead major clinical research will be able to provide people with ALS access to research through their connections to hubs. In this way, people with ALS will have better access to clinical trials, natural history studies, and other research even if they are not receiving care at a Comprehensive ALS Center. All levels of care will facilitate enrollment in a more comprehensive National ALS Registry, as described in Chapter 6.

Hubs will also support spoke centers in several additional ways, increasing the quality of ALS diagnosis, care, and research across wide geographic areas. Most directly, hub centers will complement services provided by regional and community-based ALS centers as necessary. This will include consultation for complex care or diagnostic needs, as well as connecting people with ALS to comprehensive centers via telehealth. Hub centers will provide a full spectrum of diagnostic and referral services for primary care providers, community neurologists, and other specialists. Hubs will also be responsible for developing, promoting, and sharing professional education resources, as well as spearheading community outreach initiatives.

Establishing this reimagined system will require partnerships across the government (i.e., National Institutes of Health [NIH]/National Institute of

Neurological Diseases and Stroke [NINDS], Centers for Medicare & Medicaid Services [CMS]), current ALS multidisciplinary clinic system leaders (i.e., ALSA, MDA), home health agencies, community-based providers, and local community support organizations to (1) create new public–private partnerships and community-based connections, and (2) provide financial incentives to bring more community-based practitioners under the umbrella of this integrated hub-and-spoke model of ALS care. The roles of each type of clinic are described below.

Community-Based ALS Care Centers

Establishing a larger number of local, certified community-based multidisciplinary ALS care centers will facilitate access to high-quality routine care without requiring people with ALS to travel great distances. These centers may not have the same resources or organization as a dedicated ALS multidisciplinary clinic. They may instead be independent ALS clinics, other multidisciplinary neurological clinics, integrated care networks of various specialists, solo or group neurology practices, or even certain VA clinics. However, they will still be accountable for providing multidisciplinary ALS care adhering to the latest quality metrics and practice standards. These clinics will primarily provide post-diagnosis, routine follow-up care following a care plan formulated by a Comprehensive ALS Care and Research Center or Regional ALS Center (discussed below). Importantly, all clinical staff at these credentialed community-based centers would be trained in standard ALS clinical outcomes and monitoring. This training is already available and can be expanded; examples include trainings developed by the Northeast ALS Consortium (NEALS) Outcome Center, educational webinars, and continuing medical education (CME) programs.

Community-Based ALS Care Centers may lack the infrastructure locally to lead clinical research efforts. However, persons living with ALS receiving care would still be able to participate in clinical research through their community clinic, since community clinics will be integrated with regional and comprehensive clinics. In that way, community-based clinics would be able to recruit a more diverse population, increasing representation in research and reducing inequities. Furthermore, community-based clinic staff would coordinate with home health providers to meet the out-of-clinic needs of people with ALS and their families. These clinics would also have the capacity to engage at-risk ALS genetic carriers and facilitate their access to ALS registries.

Therefore, in this new, integrated model, people with ALS who live farther from a Comprehensive or Regional ALS Center may visit Community-Based ALS Care Centers for services such as:

• Initial diagnosis, with referral or consultation to other ALS centers available for second opinions;
• Day-to-day, routine follow-up care post-diagnosis, adhering to AAN quality metrics and practice standards;
• Connection to community-based primary care and support agencies, telehealth, home health, palliative and hospice agencies, and DME companies;
• Participation in research, such as clinical trials and natural history studies; and
• Referral to other local specialists or community-based organizations for further support.

Certified community-based ALS care clinics could include current ALSA- or MDA-affiliated clinics, along with new entities brought on board to increase access to care throughout the country. For example, existing multidisciplinary centers catering to other neuromuscular or neurological disorders, such as multiple sclerosis, muscular dystrophy, Charcot-Marie-Tooth disease, spinal muscular atrophy, Huntington’s disease, Parkinson’s disease, or dementia, that may not be dedicated ALS clinics as part of current ALSA, MDA, or VA systems but could enter the new integrated care and research system and be accredited to treat ALS. Using an existing multidisciplinary clinic staffing infrastructure will be especially critical in geographic areas where there are fewer people living with ALS—or clinicians with knowledge about ALS—to develop and sustain a dedicated ALS clinic.

New community-based ALS clinics could be incentivized to care for people living with ALS via new reimbursement models. For instance, a group of ALS clinicians, specialists, and allied health professionals, including home-based palliative and home health care professionals within a community, may organize into an integrated care network and receive accreditation to deliver ALS multidisciplinary care. By incentivizing specialists who already practice in an area to create an integrated care network, commit to delivering high-quality ALS multidisciplinary care, and meet all other accreditation requirements, multidisciplinary care can still be delivered in less-resourced settings or in those with fewer people with ALS. In summary, certified community-based ALS care clinics would provide localized multidisciplinary care currently missing in the ALS care system. Staff at these clinics would be trained in ALS-specific clinical outcomes, quality metrics, other practice standards, and the latest updates in care by CME courses. Certified community-based ALS care clinics would also offer ALSA- or MDA-led education on ALS awareness.

Regional ALS Centers

Larger multidisciplinary clinics dedicated to ALS would be designated as Regional ALS Centers. While Community-Based ALS Care Centers would be responsible for ongoing management of routine care, they may not have the capacity for more complex, surgical, or specialized care needs. Regional ALS Centers would have the staffing and facilities necessary to provide such care, and as a result, many would be at larger hospitals. Like the other two levels, such centers would provide high-quality, post-ALS diagnosis multidisciplinary care according to AAN quality metrics; they would also provide a wider range of diagnostic and care services than community-based centers.

Nevertheless, Regional ALS Centers may not have the capacity to provide all ALS diagnosis or complex multispecialty care and services,

such as those related to complex imaging, genetic testing, or counseling. They would, however, be able to provide specialty care and support services, while referring to or consulting Comprehensive ALS Care and Research Centers as needed. This integration would allow for telehealth consults with Comprehensive ALS Care and Research Centers for second opinions or complex care needs. This is similar to the American Heart Association and American Stroke Association’s comprehensive stroke centers, which provide telehealth-based second opinions to smaller stroke centers.

Unlike the Comprehensive ALS Care and Research Centers discussed next, Regional ALS Centers may not have the capacity to coordinate or lead cutting-edge ALS clinical research, or to secure federal funding for preclinical research. This builds on the preexisting ALSA model of multidisciplinary care, which primarily differentiates between levels based on whether research is offered. Like all clinics in this reimagined integrated care and research system, Regional ALS Centers would offer the opportunity to participate in clinical trials or expanded access programs, enroll people with ALS in a more comprehensive National ALS Registry or natural history studies, and report on quality and population health outcomes.

Specialty services delivered by Regional ALS Centers may include:

• Diagnostic assessment and confirmation
• Specialized follow-up care needs such as:
  • Pulmonary care,
  • Tracheostomy or ventilation support,
  • Evaluation for specialized DME such as eye-tracking communication devices,
  • Medically complex cases requiring surgical procedures or post-procedural intensive care, and
  • Augmented communication and technological care services
• Support for insurance approval of standard-of-care therapies

Comprehensive ALS Care and Research Centers

Comprehensive ALS Care and Research Centers, like all clinics in this model, would provide high-quality multidisciplinary care based on the latest practice standards, facilitate access to research, and enroll people with ALS into the National ALS Registry. Beyond that, however, they would also serve as hubs that would:

• Actively conduct clinical trials and clinical research projects.
• Provide the research community with centralized imaging, biofluid and tissue biorepositories, and registry services.

• Engage genetic carriers and facilitate access to ALS research opportunities for genetic carriers.
• Train and develop the ALS workforce.

To serve as a hub and facilitate access to cutting-edge services for every ALS patient, clinicians at Comprehensive ALS Care and Research Centers should be allowed to provide consultations, second opinions, and other care services via telehealth without state line restrictions to patients receiving care at regional and community centers and their physicians. This would align with the current VA ALS system of care, which currently universally permits interstate telehealth within the Veterans Health Administration.⁵ The committee recognizes that telehealth policies are complex, vary across states, and are constantly evolving. Telehealth capabilities that cross states lines to allow ALS centers to coordinate and provide a high level of care to all persons with ALS would improve the success of the newly integrated system. VA is already allowed to use telehealth to serve veterans with ALS, and the committee believes persons with ALS who receive care outside the VA system also deserve access to the benefits of telehealth.

Care Quality Metrics

Like all accredited clinics, Comprehensive ALS Care and Research Centers will provide care following AAN quality metrics,⁶ along with routinely offered ALS diagnostic, therapeutic, and, when approved for future clinical practice, preventive services. These services would include standard-of-care treatments and procedures, electromyogram and nerve conduction studies, genetic testing and counseling, biofluid or neuroimaging biomarker testing if approved for future clinical practice, functional rating scales, and respiratory tests. As the literature develops and the standard of care continues to advance, such as with ongoing revisions to the AAN guidelines, quality metrics may continue to change as well. Certified raters would regularly assess these and other clinical outcome measures to monitor disease progression and other specialty services at their institution, such as pulmonology, physical medicine and rehabilitation, urology, psychology or psychiatry, and interventional radiology.

Clinical Research Capabilities and Expectations

Since clinical research drives innovation and therapeutic discoveries for ALS, Comprehensive ALS Care and Research Centers will need to integrate clinical research into clinical care, contributing to population

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⁵ 38 CFR § 17.417.

⁶ Available at https://www.aan.com/practice/neuromuscular-quality-measures (accessed June 10, 2024).

health outcomes research and care delivery research. These centers will need to engage in high-volume natural history, registry, or other biomarker research; participate in presymptomatic gene carrier research; participate in a clinical research consortium or care delivery network; and offer clinical trials or expanded access protocols. To facilitate care coordination and research recruitment, these centers must have at least one full-time patient research and care navigator on staff.

Like the National Cancer Institute model of Comprehensive Cancer Centers, Comprehensive ALS Care and Research Centers would be incentivized to lead community outreach and engagement efforts and coordinate with Regional ALS Centers and Community-Based ALS Care Clinics to increase access among individuals from underserved populations and to ensure continuity of care, even in later disease stages or when a patient can no longer travel. As discussed in greater detail later in this chapter, such efforts may include setting up community engagement and advisory committees comprising community-based ALS and caregiver members, nonprofit organizations and foundations, primary care physicians, and home health agencies to understand the changing care needs and barriers to health care use and participation in the local community.

Comprehensive ALS Care and Research Centers could also pilot or expand creative care delivery programs such as ALS house call nursing programs, ALS multidisciplinary mobile clinics, travel satellite clinics, and medical home programs, which are active in some U.S. regions. Such programs could bring care closer to the homes of persons living with ALS to reduce travel burden, particularly during the later disease stages. Box 4-2 describes the specialty care medical home as a potential model for coordinating community-based multidisciplinary care for ALS.

Integration Across Levels

People with ALS may enter this new integrated care and research system in a variety of ways. Some people with ALS see an ALS specialist relatively early, or they may see clinicians at hospitals that have Comprehensive ALS Care and Research Centers or Regional ALS Centers. Others may initially see their primary care physician or non-ALS specialists. A person suspected to have ALS will be referred to the nearest comprehensive or regional center for diagnostic confirmation, formulation of a multidisciplinary care plan, and a discussion of opportunities to participate in research. For complex scenarios, they may also use telehealth for remote second opinions with Comprehensive ALS Care and Research Centers. Telehealth will also be available for second opinions and referrals throughout all levels of the system.

The comprehensive or regional ALS center would continue to provide follow-up specialist appointments for continuity of care or complication management as necessary. Day-to-day, routine follow-up care would be managed by the nearest available multidisciplinary center (whether comprehensive, regional, or community-based). All levels will have the capacity to deliver this care; by integrating more ALS centers, especially Community-Based ALS Care Centers, into the system, more people with ALS will have standard of care treatments available to them.

ALS research is also integrated into this reimagined system. Regardless of what type of clinic a person with ALS attends for their routine care, they will always have access to clinical research due to the integrated nature of the system. All center types in this new integrated care and research system will be incentivized to enroll and retain all people with ALS in the National ALS Registry, as proposed in Chapter 6, for population health monitoring.

How the Integrated ALS Care and Research Model Should Work

The above model will create a highly integrated system of care that should more easily reach underrepresented and underserved ALS patients and those living in remote areas of the United States and all U.S. territories and improve the current system of care for individuals living with ALS (see Table 4-3). Today, many non-ALS-trained neurologists provide care to ALS patients in general neurology or general neuromuscular clinics, which, under the new recommended model, could be integrated into the new ALS care system under the guidance of the comprehensive ALS centers. This would expand the number of general neurology and neuromuscular clinics within the ALS care and research system, enhancing access to care and reducing the time to diagnosis.

The newly networked community-based or regional clinic would be responsible for measuring and improving diagnostic timelines within its geographic area. A designated person or team would coordinate with the hub and take action to reduce delays in diagnosis. The designated diagnostic person or team could work proactively with local Federally Qualified Health Centers (FQHCs) and local practices such as sports rehabilitation or general neurology clinics to educate non-ALS specialists on how to identify possible ALS symptoms and instruct them on the referral process to the local ALS specialist. The Comprehensive ALS Care and Research Centers coordinating the local diagnostic teams would be adequately reimbursed to dedicate time and attention identifying people who may have ALS and educating local providers, but this expense is necessary to improve the diagnosis process.

The NINDS Access for All in ALS (ALL ALS) research consortium and network of sites, initiated in 2023, could serve as a foundation on which to build the ideal ALS care network. Currently, the ALL ALS consortium, funded under the Accelerating Access to Critical Therapies for ALS Act,⁷ primarily collects natural history, clinical, biomarker, and imaging data, along with autopsy tissue, from several thousand people with ALS and deposits them in central data banks and biological specimen banks for future research. However, the committee envisions this consortium’s network could be developed or expanded to meet the goals of the proposed care delivery network, as successfully achieved by several National Cancer Institute–funded care delivery networks.

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⁷ Accelerating Access to Critical Therapies for ALS Act, Public Law 117-79.

TABLE 4-3 Comparing the Current ALS Clinical Care and Research System to the Committee’s Proposal for an Integrated ALS Care and Research System

^a In an October 18, 2023, letter to the committee from the ALS Association, it is stated: “Although multidisciplinary ALS care can add nine months of life, it is woefully underfunded and often difficult to deliver. Only about half the people served at ALSA Certified Treatment Centers of Excellence receive this well-established, evidence-based standard of care.”

SOURCES: ALSA, 2024; Chen et al., 2023; Falcão de Campos et al., 2022; Galvin et al., 2015; Gwathmey et al., 2023a; Matharan et al., 2020; Richards et al., 2021.

ALS CARE SYSTEM CHALLENGES

The current ALS care system faces several challenges in providing timely and equitable access to high-quality multidisciplinary care for all people living with ALS. Among the most significant is the common delay in diagnosis (see Chapter 2), which averages 7 to 10 months for bulbar-onset ALS and 10 to 22 months for limb-onset ALS (Gwathmey et al., 2023b). While ALS remains invariably fatal, advances in therapeutic development and clinical research will likely prolong the lifespan and improve quality of life for people living with ALS. Given that prompt and early initiation of multidisciplinary care and medications improve outcomes, delays in diagnosis and treatment can significantly affect the course of the disease. Unacceptable delays result largely from delays in referral to an ALS specialist because the primary care physician did not recognize the ALS symptoms, referrals to one or more specialists other than a neurologist, and misdiagnosis, including by a general neurologist (Goyal et al., 2023; Matharan et al., 2020; Morren et al., 2023; Richards et al., 2021). Other factors delaying prompt diagnosis include age of onset—people with ALS older than 60 years of age are more likely to be initially misdiagnosed than are younger people with ALS (Belsh and Schiffman, 1996)—and neurological comorbidities (Mitchell et al., 2010; Palese et al., 2019). Moreover, coordination is lacking between general neurologists, who may only see a few people living with ALS each year, and ALS specialists.

Once a person with ALS is diagnosed, there is also variation across clinics in the quality and consistency of care they receive, including prescribing of standard-of-care therapeutics, use of noninvasive ventilation, use of off-label supplements and medications, clinical trial participation, and access to clinical trials (Chiò et al., 2011; Hogden and Crook, 2017; Katyal and Govindarajan, 2017; Skulstad Johanson et al., 2022; Thakore et al., 2019). Some ALS multidisciplinary clinics may not prescribe certain medications approved by the U.S. Food and Drug Administration because of perceived insufficient benefits, or offer genetic testing or counseling, evaluate voice or message banking, provide speech-generating devices, or offer the palliative care or hospice services as recommended (Fahrner-Scott et al., 2022).

Moreover, challenges exist from low clinic capacity and lack of training and growth opportunities for ALS specialists and the health care workforce. In addition to ALS specialists, pulmonologists, who provide critical life-extending respiratory care to people with ALS, are in limited supply, creating few opportunities for new consults (Ackrivo, 2023; Hansen-Flaschen, 2021). Access to essential home health aides and home care services are limited resulting from gross underpayment for hourly paid workers.

Administrative burden and insurance conflicts also affect many ALS multidisciplinary clinics and the individuals they serve. Clinic staff spend

hours filling out paperwork, seeking prior authorization, calling insurers and pharmacies, appealing denials, and fielding phone calls from people living with ALS and their families, which can delay or prevent using potentially effective therapies and engender patient dissatisfaction. This current inefficient system hinders ALS clinics from meeting the needs of people they are treating for ALS, slows their ability to serve new people with ALS, and results in redundancies that are a waste of time and money.

If the status quo of the current care system continues, increased demand from a growing ALS prevalence, both from more diagnosed individuals and people living longer with ALS, will worsen the existing inequities in access to multidisciplinary care. Therefore, as earlier diagnosis and therapeutic regimens improve, new ALS care delivery systems will need to be sufficiently coordinated and nimble to meet the evolving needs of individuals living with ALS. Furthermore, new ALS care standardization will reduce redundancies, facilitate care from physicians with experience treating individuals with ALS, and provide the latest evidence-based care. By integrating the new ALS care delivery and research systems as this chapter recommends, more people living with ALS will have access to high-quality care.

Meeting the Equity Challenge

Many people with ALS in historically marginalized and underserved communities do not have early or consistent access to multidisciplinary ALS centers and care. For example, the time it takes a Black individual with ALS to receive a diagnosis can be more than 50 percent longer than a White individual (Chen et al., 2023; Gwathmey et al., 2023a). This is despite the fact that Black people living with ALS were, in one study, found to live closer to a multidisciplinary center than White people living with ALS (Horton et al., 2018). Black people living with ALS are also often more advanced in their disease and disability at the time of diagnosis (Richards, 2020). Providers’ implicit biases, structural racism, and patient distrust, disinterest, or fear of seeking medical attention are also sources of this racial disparity (Carter, 2021, 2022; Casey, 2023; Chen et al., 2023). The disparity in diagnosis time is also affected by the type of clinician people with ALS may visit initially to seek care. As discussed earlier, community-based clinicians without specific training or ALS experience may be less aware of the range of early-stage ALS signs. Efforts to improve health outcomes and reduce disparities for people with ALS will ideally move away from “cultural competency” education to “structural competency” and an understanding of the way social and economic forces influence health outcomes at levels above individual physician–patient interactions (Metzl and Hansen, 2014).

Significant racial inequities exist in the U.S. health care system overall—Black people and American Indians and Alaska Natives, for example, fared

significantly worse across the majority of 30 measures of health status and health outcomes between 2019 and 2021 (Hill et al., 2023)—which the ALS care and research system exists within. However, it is worthwhile to pursue targeted approaches to improve racial equity in ALS, and the committee offers the following recommendation:

Recommendation 4-2: Improve racial and ethnic equity in the ALS care and research system.

ALS multidisciplinary clinics should partner with community members and community-serving organizations to pursue targeted approaches to understanding and improving racial and ethnic equity in ALS care and outcomes in their geographic area.

The committee believes there are several opportunities that ALS clinics, in partnership with entities that serve the local community such as FQHCs, should pursue, including the following:

• Create community-focused steering committees. Each Community-Based ALS Care Center, as recommended in this report, in the newly integrated ALS care and research system should include a steering committee that would include multiple community members—individuals living with ALS, former ALS caregivers, at-risk genetic carriers, and other ALS experts from diverse racial and ethnic backgrounds, including non-English speaking individuals, among others—to build a bridge to the community and help develop programs to bring people who might otherwise go unnoticed into the ALS system.
• Collect and analyze data on racial equity. ALS centers should be expected to measure and address racial equity. Local clinics should report on the unique factors contributing to diagnostic delays in their geographic area. This responsibility to their population could be tied to funding and be a condition of qualifying as an ALS center.
• Adopt antiracism and implicit bias training as an expected and regular part of training. Each Comprehensive ALS Care and Research Center should lead antiracism and implicit bias training for center staff and clinicians that is not a one-time exercise but a regular part of training. Training would include developing an understanding of disparities in ALS, the social and structural determinants of health, and what it looks like to provide good care to diverse and historically underrepresented populations.

Meeting the Challenge of Workforce Development and Education

Since ALS is a rare disease, a non-neuromuscular or non-ALS specialist neurologist may see only one or two people living with ALS in their clinical practice per year. It is even rarer for primary care physicians and other physicians, who may not recognize or consider ALS early in their diagnostic workup and differential diagnosis, but instead “wait and watch” until symptoms have progressed before they refer the person to specialized multidisciplinary clinic.

Training and education are needed to ensure that nonspecialist clinicians suspect and refer people who may have ALS, even if not fully meeting the traditional diagnostic criteria of ALS. Education and training should also include information on diseases that mimic ALS to reduce false positive referrals. The ALSA thinkALS tool is one approach to aid the non-ALS specialist clinician suspect ALS and reduce referral delays. Artificial intelligence tools could be developed that parse out pertinent clinical features from electronic health records during clinical consultation with a non-ALS physician, providing an alert about the possibility of ALS.

The projected increase in ALS prevalence will exacerbate the demand for the already limited number of ALS specialists and other clinician staff (Arthur et al., 2016; Miller et al., 2021). Optimal care for individuals living with ALS requires collaboration across multiple medical specialties, including neuromuscular neurology and physiatrists (McDonald and Fowler, 2012), but there are significant workforce limitations in neuromuscular specialists, affecting patient access to subspecialized care. For example, the American Board of Psychiatry and Neurology only issued 98 certifications for physicians specializing in neuromuscular medicine in 2022.⁸

There are gaps in clinician education about ALS diagnosis and the benefits of multidisciplinary care. For example, not all U.S. neurology residencies include ALS clinic rotations. In addition, there are only two to three dedicated ALS fellowships yearly to train the next generation of ALS clinicians, researchers, and clinical trialists. For example, the American Academy of Neurology offers one 2-year or 3-year clinical ALS research training grant awarded competitively to one clinician per year. Academic institutions may offer fellowships in neurodegenerative disorders that are limited and not linked to accreditation. Current NINDS career development, or K, awards focus mainly on helping postdoctoral fellows transition to independent investigators but are not set aside for a particular subspecialty.⁹

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⁸ Additional information is available at http://www.abpn.com/about/facts-and-statistics (accessed June 10, 2024).

⁹ Additional information is available at https://www.ninds.nih.gov/funding/training-career-development/career-development-awards (accessed June 10, 2024).

CDC offers an ALS continuing education module for primary health providers and other health professionals.¹⁰ It would be appropriate for every graduate medical, nursing, or physician assistant professional school to include an ALS module such as this in their curricula. Since most people with ALS die from respiratory failure, pulmonologists need training in the treatment and supportive care of people with ALS. Physiatrists, physical medicine, and rehabilitation specialists, palliative care clinicians, critical care specialists, emergency physicians, neurosurgeons, spine surgeons, and otolaryngologists would also benefit from ALS-specific training. Persons with ALS will need to receive care from other health professionals not specific to their ALS diagnosis, such as dentists, audiologists, and others, who would need to understand what ALS is and how the disease affects the person they are treating.

There is also a severe shortage of home health care workers. According to the Home Care Association of America, home health care providers turn away 25 percent of referred patients because of staff shortages (HCAA, 2023). Factors behind the shortage include low pay compared to other sectors, lack of opportunities for career advancement, dramatic slowdown in the flow of immigrants who usually account for most of this workforce, preference for part-time work to remain below income limits that qualify for public assistance benefits, and a first-year turnover rate of 64 percent (HCAA, 2023). As described in Chapters 2 and 3, persons with ALS have significant home health needs requiring a home health aide with knowledge of complex life-sustaining machinery, such as ventilators. It will be necessary to adequately compensate a trained home health workforce for their experience if ALS is to become a livable disease in the next 10 years.

The comprehensive ALS centers in the proposed network could serve as training facilities, tied to their certification, to develop yearlong clinician/clinical translational research fellowships and other short-term training programs. Doing so would create opportunities to train future ALS clinicians and clinical researchers, including physicians, advanced care practitioners, nurses, and allied health professionals. The newly proposed network would also be responsible for ongoing ALS education and training of the clinician and lay community. Funding for the new NINDS-funded research financing model recommended in this report could be earmarked for this training.

NINDS recently published a set of strategic priorities for the ALS community, including mechanisms to encourage pursual of ALS-related research and training (Koroshetz, 2023). NINDS, with the American Academy of Neurology and the Accreditation Council for Graduate Medical Education, could take steps to make the ALS specialty clinician, hybrid clinician,

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¹⁰ Additional information is available at https://www.cdc.gov/als/ce/Index.html (accessed June 10, 2024).

clinical trialist, or clinical researcher track a sustainable, adequately compensated, and more attractive career pathway for junior faculty entering the workforce to address the pending crisis regarding the shrinking ALS specialist workforce.

The proposed integrated ALS care and research model will only succeed if the workforce exists to meet the needs of persons living with ALS and their families. There is an urgent need for the public, private, and nonprofit sectors working in ALS to provide incentives and opportunities to recruit and retain the ALS workforce needed. This includes ALS specialists, clinical trialists, health service researchers, and the critically needed home health workforce with the skills to care for the complex needs of a person with ALS.

Meeting the Challenge of Payment and Reimbursement for ALS Multidisciplinary Care and Research

As discussed in Chapters 2 and 3, many persons with ALS and their families experience significant financial challenges. As noted above, multidisciplinary clinics also have significant financial challenges given the longer visits needed to diagnose and establish care for persons with ALS (Boylan et al., 2015; Paganoni et al., 2017). The traditional fee-for-service payment model fails to reimburse for more than 50 percent of the costs of an ALS multidisciplinary clinic visit (Boylan, 2015; Paganoni et al., 2017), so centers must seek philanthropic, foundational, and institutional resources to meet costs.

The current payer limitations of a fee-for-service payment model for evaluation and management encounters in ALS means that multidisciplinary ALS clinics are not reimbursed for many of the staff providing care during the ALS clinic visit, including physical therapists, occupational therapists, speech therapists, social workers, case managers, genetic counselors, ALS clinic nurses, certified ALS clinical outcomes monitoring services, and clinical research access coordinators. Under the fee-for-service model, institutions must absorb the indirect costs of the 4- to 5-hour long multidisciplinary visits since insurance reimbursements account for only 30- to 60-minute face-to-face time spent with the physician. There is also the financial burden arising from uncompensated time clinical staff spend coordinating the evaluation, insurance authorizations, coordinating care with home health and home hospice agencies, and communicating with people with ALS and their caregivers.

A 2015 report by the Massachusetts General Hospital ALS clinic, one of the largest U.S. ALS centers, showed that 409 unique people with ALS and 1,285 ambulatory office encounters averaged $580 in actual cost per patient per clinic visit. However, the insurance-billable encounter recouped only $263 per patient per clinic visit, leaving a $317 shortfall

per patient per clinic visit (Paganoni et al., 2017). Nonprofit organizations, such as ALSA and MDA, and local nonprofits supporting single ALS centers, such as the Les Turner ALS Foundation in Chicago or the ALS Hope Foundation in Philadelphia, can offset some of these costs through semiregular, nonguaranteed funds. Moreover, donated funds may be insufficient to cover the shortfall; for example, ALSA contributes only $25,000 to each multidisciplinary clinic annually. Thus, for the most part, ALS clinics must self-support multidisciplinary clinic operational costs via the ALS clinic sundry funds, departmental, or institutional support, and philanthropic support.

Major health care institutions may support salary for one to two dedicated ALS registered nurses per ALS clinic based on insurance reimbursements. However, many major ALS centers see and provide care to 600 to more than 900 people with ALS annually and require as many as nine dedicated ALS registered nurses to adequately deliver high-quality nursing care. Because of staffing shortages at ALS clinics, the same nurses often also fulfill case management or social worker roles to coordinate care with local Visiting Nurse Association operations, palliative care teams, nursing homes, hospice, and insurance prior authorization and disability paperwork. Smaller ALS centers with limited financial resources to hire and support multiple ALS nurses refer their people with ALS to other larger centers, which may be as far as 100 miles away or across state lines.

Because of the continually changing care needs stemming from the progressive nature of ALS, care provided during clinic visits may not suffice. To provide optimal continuity of care, ALS nurses often spend multiple hours per week per patient answering patient or family phone calls or online portal messages related to clinical care, medication changes, or coordinating care with local primary care or other non-ALS care teams. Since there are no payment models or pathways for nurses to bill insurance for their services, they are not compensated for the vital services they provide to individuals living with ALS.

The current funding structure for ALS multidisciplinary clinics is not a sustainable model to provide high-quality care. A new model is necessary to make that care accessible to all people with ALS. Reimbursement policies need to promote the seamless delivery of clinical care and home-based services and equipment, including the use of telehealth to provide services across state lines. This is similar to what VA offers veterans with ALS.

The committee is aware of legislative efforts in Congress to create a supplemental, facility-based Medicare payment for ALS-related services at multidisciplinary clinics. Condition-specific reimbursement mechanisms have been implemented by CMS for end-stage renal disease, cancer, and other diseases. Such a model can be piloted by public actors such as the CMS Innovation Center or by private insurers.

The committee’s broader vision for an integrated ALS care and research system includes a companion approach to reimbursement. Reimbursement policy can both increase the number of clinics caring for people with ALS and expand the geographic areas served by the ALS care and research system.

For example, large academic health systems could be incentivized to establish Comprehensive ALS Care and Research Centers, caring for more people with ALS and serving as research hubs. Increasing the number of these comprehensive ALS centers would establish a common infrastructure of trained and experienced health care personnel, which could also serve as centers for other neurological disorders that would benefit from similar multidisciplinary care, such as muscular dystrophies, inherited neuropathies, multiple sclerosis, Parkinson’s disease, and dementia.

Similarly, clinics that do not serve substantial populations of people with ALS should be incentivized to become a Regional Multidisciplinary ALS Center or Community-Based Multidisciplinary ALS Care Clinic. For example, smaller clinics across multiple specialties could be incentivized to organize themselves into a disease-specific “pod” to provide multidisciplinary ALS care as an integrated care network. As discussed earlier, such a pod would obviate the need for creating an entirely new ALS clinic in an area where resource limitations would make that challenging. While they would serve other patients, they would be part of this new clinical care and research system as the equivalent to a single multidisciplinary clinic, with all the same accreditation standards.

This model would allow outreach, scalability, and the expansion of network sites, even to geographic areas with low ALS volume, while assuring access and connectivity for all persons with ALS to the specialized services they need and opportunities to participate in research more easily. Research center designation and reimbursement should track with the level of leadership and participation in the new integrated clinical care and research model.

Recommendation 4-3: Align reimbursement to achieve the goals of the ALS clinical care and research system.

The Centers for Medicare & Medicaid Services, private insurers, the National Institutes of Health, and the National Institute of Neurological Disorders and Stroke should align reimbursement and the goals of the new, inclusive, and integrated ALS clinical care and research system.

Value-Based Payments

In addition to greater overall reimbursement to accredited clinical programs that participate in the ALS research and clinical care system,

value-based payments may help promote effective and high-quality care.¹¹ There are two options for creating value-based financing models to support the ALS care and research under the recommended integrated model.

The first option is for clinical programs to receive standard value-based payments for ALS multidisciplinary clinic encounters. In this model, clinics would bill for each evaluation and management encounter on a fee-for-service basis. Payments would then be adjusted for meeting key quality metrics, including measures of access and quality for care delivered in rural, low socioeconomic, and underrepresented and marginalized communities. In this model, reimbursements for surgical or medical procedures, such as per cutaneous gastrostomy, intrathecal or intravenous infusions, lumbar punctures, and electromyographies; inpatient hospitalization; emergency department costs; other outpatient specialist consultation referrals; and home health care costs provided within or outside the institution would be subject to standard fee-for-service or bundled payment models.

A second option is capitated payment, under which clinical programs would receive a global, value-based payment for all the care a person with ALS needs. For this payment, the clinical program would be responsible for providing individualized ALS care, regardless of how many evaluation and management encounters or home visit encounters that a person with ALS may need throughout the year.

Unlike adjustments to fee-for-service payments, global value-based payments can reflect the heterogeneity of ALS presentation and care. People with ALS would be able to receive care for their unique needs with fewer restrictions on timing, setting, or services available. As long as services are specified within the network beforehand, preapproval for specialty services (e.g., pulmonology, physiatry) would no longer be necessary, and delivery of support services (e.g., psychology, care navigation) can be supported. Under this model, insurance-based restrictions on the number of clinic visits a person with ALS may have within a certain length of time may also be removed. Finally, care across all three settings in the committee’s proposed network would be coordinated, and home-based care could be further incentivized to bring care closer to people with ALS instead of relying on clinic visits alone.

Challenges to establishing capitated payments include setting the right level for people with different types of ALS and disease trajectories.

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¹¹ Value-based capitation adds performance metrics to the capitation formula to ensure patients do not receive suboptimal care through under-utilization of health care services. Meeting or exceeding value-based performance metrics can be linked to financial rewards such a bonuses (Alguire, 2023).

Any definition of “value” in value-based care and associated reimbursement structures would need to be crafted carefully so as not to require patient improvement as part of the criteria. The heterogeneity in how ALS presents and progresses also may make it difficult to estimate per-person costs. For these reasons, caution in proceeding with fully capitated models too quickly is warranted.

Meeting the Challenge of Paying for Integrated ALS Research

The system of ALS care must be coordinated with and integrated into the ALS research system. Today, ALS care and research often exist separately, and opportunities are missed to study care delivery and outcomes because the research infrastructure is lacking. The committee envisions the following research role and funding model for the ALS care and research setting in the new network. The concepts are based on the NCI model of centers that provide clinical care and conduct research. There could be clear capabilities and expectations with NINDS core funding at each of the three proposed care and research settings.

Comprehensive ALS Care and Research Centers would lead laboratory, clinical, and population-based research, including biology and genomics, treatment, prevention, behavioral health, health equity, and other social determinants. This would include initiating early-phase clinical trials and leading cooperative clinical trials that include an array of clinical sites, including the regional and community-based clinics, as well as sites outside of the proposed care network to ensure broad patient access to research studies. These comprehensive ALS centers, as coordinating centers, would receive funding from research grants and via foundation- and industry-sponsored research, that it would then distribute to support participating clinical sites, including regional and community-based clinics. This has been a successful approach in several NIH-sponsored multicenter studies.

The Community-Based ALS Care Centers and Regional ALS Centers would be expected to participate in research led by Comprehensive ALS Care and Research Centers, but they could also participate in other research studies, including industry-sponsored studies. Research funding and NINDS designation for the Regional ALS Centers might be modeled after NCI cancer centers, which have the capabilities to lead or participate in federally funded population health research, outcomes research, care delivery research, implementation and dissemination research, and satisfying registry and natural history study enrollment goals. Such funding would also cover personnel support for research navigators to ensure cross-collaboration across care settings in the network.

Connecting VA to the Integrated ALS Care and Research System

As discussed earlier in this chapter, VA is known for strong comprehensive rehabilitative programs for veterans with complex disabilities, much of which were established through congressionally directed funding. The VA ALS system of care follows an interdisciplinary, proactive patient-centric approach, providing early and continued access to specialist consultations, medications, therapies, equipment, and care services, and represents a successful model for non-VA ALS care systems.

Nonetheless, the VA ALS care model does not address several critical issues in the ALS landscape, such as ensuring equitable access to high-quality multidisciplinary ALS care across the country or addressing the projected extreme shortage in the ALS health care and clinical research workforce needed to provide multidisciplinary care and advance therapeutic development (Harp, 2023; Majersik et al., 2021). Moreover, although it does support advanced fellowship training programs in amputation, spinal cord injury, and brain injury, VA does not offer opportunities specific to ALS clinical care. It also does not address the geographic, racial, ethnic, and socioeconomic disparities in access to clinical trials or expanded access programs. While the literature clearly suggests an increased risk of ALS in persons with military service, as concluded in the 2006 Institute of Medicine report Amyotrophic Lateral Sclerosis in Veterans: Review of the Scientific Literature (IOM, 2006) no specific work has been undertaken within the U.S. Department of Defense/VA to further understand and propose means to mitigate this increased risk. Furthermore, veterans receiving ALS care within VA clinics have limited access to clinical research, although it does operate a brain bank for postmortem studies of veterans with ALS.

The committee believes that, given the known higher prevalence of ALS in veterans, it is critical to include the VA ALS system of care in the new integrated ALS network of care and research proposed in this report. However, because of rules separating the VA system from other care systems, VA cannot be completely fit into the care and research model proposed earlier in this chapter. Notably, nonveterans are not allowed to receive care at VA clinics, and veterans going outside of the VA system for care will continue to rely on VA for critical benefits, such as DME and medications.

Still, integration would ensure equitable and streamlined access to the highest standard of clinical care for veterans, as well as the ability to participate in clinical trials. With such integration, veterans newly diagnosed with ALS in private care settings would be able to more rapidly connect to comprehensive VA care and resources, and veterans with ALS receiving care in the VA system to access research and additional resources when needed. Clinical trials based at VA facilities would be logistically easier for veterans

with ALS to access, removing the barrier of traveling to an outside facility. Finally, given the unique differences in clinical practice between VA ALS clinics and the civilian sector (see Table 4-1), there is ample opportunity for health services research to determine whether these differences improve outcomes for persons with ALS to inform future medical and health policy.

Investment in the VA to achieve these goals would require separate, congressionally mandated funding to build a research infrastructure and organized network of care that would integrate into the proposed model of care under Recommendation 4-1. Without this specific funding source, the VA ALS Centers of Excellence cannot address the gaps outlined earlier in this chapter related to access to clinical research trials and expanded access programs.

Recommendation 4-4: Enhance access to ALS clinical care and research and education opportunities within the U.S. Department of Veterans Affairs (VA).

Congress should allocate specific funding to create a VA network for ALS clinical care, research, education, and innovation to align with the new system of care outlined in this report. VA should use these funds to resolve ALS workforce shortages, ensure access to comprehensive ALS care for veterans regardless of geographic location, increase the number of health professional training opportunities to support ALS care for veterans, and invest in clinical and informatics resources at VA to enhance existing collaboration with the Centers for Disease Control and Prevention ALS registry.

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