3

Making ALS Livable in the Near Term

ABSTRACT

As part of its charge, the committee was asked to recommend key actions for the public, private, and nonprofit sectors to undertake to make amyotrophic lateral sclerosis (ALS) a livable disease within a decade. In Chapter 4, the committee makes recommendations that, if implemented, would create an accessible, coordinated, and sustainable ALS care and research system. While these long-term actions are critical to improving ALS research and care for future people with ALS, the committee recognized the acute need and opportunity for immediate, short-term actions that would make ALS a more livable disease today. This chapter provides the evidence and supporting rationale for recommended actions that, if implemented immediately, would achieve that goal.

INSURANCE CHALLENGES

The other insurance companies didn’t do very much. Doctors saw me and wanted to check my muscles and that was it. Anything I needed I had to pay for. When they told me I needed a power chair, the first one I got I had to help pay for that, and you know how much they cost.

—Jim Clingman, person living with ALS

One the most vexing problems individuals living with ALS and their caregivers face is negotiating with insurance companies to obtain the services they need. To start, most insurance coverage policies do not easily accommodate the unique needs of an individual living with ALS, which

can lead to stressful, frustrating interactions with an insurer. As the ALS Association (ALSA) notes in its advice to people living with ALS and their caregivers, insurance is not the easiest subject for anyone to understand because of its complexity, whether it is premiums, deductibles, out-of-pocket maximum payments, and pharmacy coverage based on tier medication systems, just to name a few. According to the ALS Association, “many people do not even know where to start or what questions to ask” when dealing with an insurer due to the complexities of the system, as well as their unique needs (ALSA, 2023a).

Social Security Disability Insurance and Medicare

Persons with ALS experience a variety of insurance situations that depend on a number of factors, including whether a person is still working and if an individual is eligible to receive Social Security Disability Insurance (SSDI). Individuals who have worked for a minimum period in jobs covered by Social Security and have a qualifying condition, such as ALS, are eligible for SSDI. Once a person with ALS receives SSDI, which includes a monthly payment, they also become eligible for Medicare, which provides medical care and equipment.

The federal Medicare program, administered by the Centers for Medicare & Medicaid Services (CMS), is offered to individuals aged 65 and above. However, there are two exceptions to the age 65 requirement: individuals with end-stage renal disease or a disability have access to Medicare coverage. Because ALS causes disability, persons with ALS under age 65 qualify for Medicare. In addition, persons with ALS are exempt from the usual waiting period before Medicare coverage can begin and therefore can receive benefits the first month they are eligible. Many people with ALS receive SSDI and Medicare, but not everyone will be eligible based on their work history. In particular, a younger person diagnosed with ALS with limited work history may not meet the required work history to receive disability benefits and Medicare.

There is uncertainty as to the number of persons living with ALS who do not have access to SSDI and Medicare given their work history. In addition, some people with ALS are not even aware of the benefits they are eligible to receive through SSDI and Medicare. Congress has recognized that ALS is an exceptional disease and corrected eligibility requirements for federal programs to ensure persons with ALS receive access without delay (Korbey, 2023).

For people with ALS who are still working, unique challenges may emerge. SSDI applicants are not allowed to earn more than a certain amount per month, so earning too much through their work could disqualify them from receiving disability benefits. For a person with ALS who is able to

continue working, this may leave considerable gaps in access to disability-related services and financial strain.

For example, a person with ALS, Bruce Rosenblum, who continued working well into his disease course and died in December 2023, described his situation:

Insurance coverage for caregiving is inadequate for ALS patients on disability and virtually nonexistent for patients like myself who, although disabled, are not on disability. Three years ago, my wife retired to become my primary caregiver. But serving as the primary caregiver for a spouse who is progressing through ALS is a physically and emotionally draining job for which additional support is necessary. We supplement my wife’s care, when we are able to find caregivers, which is an entirely separate challenge, with 5 hours of professional caregiving coverage each morning to help with the most challenging activities of daily living. Thirty hours of weekly paid caregiving, which we have to pay for out of pocket, exceeded 50 percent of my take-home pay each of the last 3 years. Add together all of the other uncovered costs of ALS, and that has left us drawing on our retirement savings for most nonmedical living expenses.

The committee finds that even though Congress has recognized the exceptional nature of ALS and made important changes to eligibility requirements for federal programs to make it easier for people with ALS, not everyone with an ALS diagnosis is eligible to receive SSDI and access to Medicare. There is an opportunity to expand the status of ALS as a qualifying condition for Medicare coverage, such that persons with ALS are eligible for Medicare coverage regardless of employment history or other criteria influencing Medicare or SSDI eligibility.

Prior Authorization, Insurance Denials, and Appeals

People living with ALS can face challenges in acquiring medically indicated equipment, technology, and therapeutics and dealing with an often-convoluted system for obtaining prior authorizations. Prior authorization refers to a process in which insurance companies require a clinician or other qualified health professional to seek advance approval before providing certain services, tests, medications, equipment, or procedures. Prior authorization is a tool insurers use frequently as a means of controlling costs and avoiding unnecessary care. The committee was unable to find publicly available information or evidence as to whether prior authorization processes reduce unnecessary costs in ALS care, nor was the committee able to determine whether all prior authorization request denials for ALS are inappropriate. Frustration with prior authorization processes is also not unique to ALS—reforming prior authorization processes across the insurance industry

are the focus of national efforts and are not restricted to just one disease area (AMA, 2022). CMS issued a final rule in January 2024 that requires payers to streamline prior authorization processes electronically and provide more information to patients about prior authorization processes including approvals, denials, or requests for more information beginning in January 2026. The new rule also requires payers to send prior authorization decisions within 72 hours for expedited or urgent requests and 7 calendar days for standard or nonurgent requests and publicly report certain prior authorization metrics annually.¹ Prior authorization challenges are largely associated with Medicare Advantage plans and not traditional Medicare.

Insurance companies and Medicare Advantage plans may deny a prior authorization request and require an individual to “fail first,” which means they will only give authorization if the individual first tries and fails a treatment or is not well served by a piece of equipment. ALSA has reported many examples of Medicare Advantage plans and other private insurers denying access to home mechanical ventilation (ALSA, 2024) despite evidence supporting the use of such equipment in reducing morbidity and mortality. For example, a clinician might order a specific ventilator for an individual living with ALS to use at home, and instead of approving the ventilator requested by the clinician, the insurance company might require that the individual first use and fail on a less expensive ventilator designed for chronic obstructive pulmonary disease patients to use. Importantly, less expensive ventilator devices often have inadequate battery capabilities thereby limiting mobility for people living with ALS and further impairing quality of life. Again, there is an absence of publicly available data from insurance companies showing that prior authorization processes reduce costs while improving outcomes for individuals living with ALS. The committee would find it unacceptable for a person diagnosed with ALS to be forced to use a ventilator that provides less effective breathing support for ALS because it is designed for another type of disease. Cost-controlling measures are only appropriate when two devices are equivalent and deliver similar results and value to a patient.

A 2022 ALSA survey of people living with ALS and caregivers found that one in three people who submitted a prior authorization request or claim were denied at least once (ALSA, 2023b). The top five denials were for prescription medications or medication delivery supplies; wheelchair accessories; power wheelchairs; in-home physical, occupational, or speech therapy; and a wheelchair-accessible vehicle or vehicle modifications.

After receiving an insurance denial, an individual can make multiple appeals to the insurance company, the relevant state agency that regulates insurance companies in their state, and even contact journalists or the

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¹ See CMS Interoperability and Prior Authorization final rule, CMS-0057-F.

media regarding denials of care, services, or equipment the individual deems necessary. According to an ALSA survey, 67 percent of those denied prior authorization and 47 percent who had a claim denied appealed the insurers decisions (ALSA, 2023b). The ALSA survey also found that participants had to appeal up to three times and wait up to 6 months for a resolution to their appeal, with approximately one-third of the appeals denied or only partially granted. Those individuals denied authorization or reimbursement had to either pay out of pocket; forgo the requested drug, service, or device; or discontinue care. The committee finds this length of delay unacceptable given the rapid disease course of ALS.

People responding to the ALSA survey reported frustration with Medicare denials and appeals. Often, individuals must first obtain the services or items in question and assume the financial risk before they can file an appeal (Center for Medicare Advocacy, 2016).

The most common reason for the denial of a claim involves the determination of whether there is medical necessity. Sometimes, a medication or procedure that a care provider deems important is not seen this way by an insurance company. When this occurs, a care provider may need to show proof of the value of a particular treatment over alternatives. Some Medicare contractors issue denials because they mistakenly believe that Medicare only covers skilled services such as physical, speech, and occupational therapy when those services will lead to an improvement in symptoms (Center for Medicare Advocacy, 2018).

The committee finds that ALS is a heterogeneous, multisystem disease that requires a proactive and anticipatory approach to care. Prior authorization processes can be inappropriately blunt tools used by insurers for many services, procedures, tests, and medications that individuals living with ALS need to improve their quality of life. Lengthy prior authorization processes rob an individual living with ALS of their most precious resource—time. Prior authorization processes also add strain and sap energy from clinical staff. There is a need for prior authorization processes to be streamlined, expedited, or removed for ALS care given the time course of the disease.

INADEQUATE HOME SERVICES

I had physical therapy in my home. I have to go through my primary care provider to get the prescription to submit to the company. Everything takes a long time to get approved or how many visits get approved, which to me seems very strange. It’s almost like I have to go back to my primary care provider every once in a while to say, “Hey, I still have ALS.” Why do we have to waste time going over something like this? It’s not like ALS goes away. Now I pay out of pocket for the physical therapy because the insurance is just a pain to get it covered. To me, it is so important for me because during that physical therapy is probably the only time where I feel less pain in my joints and

everywhere. It gives me a physical break but also an emotional support system where afterward emotionally I have the capability to be more interactive with my son, for example, and do things together.

—Julian Rodriguez, person living with ALS, as described to the committee during August 2023 public workshop

I require a home health aide 12 hours a day, 7 days a week. My mother is a huge part of my care team. She makes me healthy food and cooks for my husband and my daughter. She covers when aides are not available for work.

—Desiree Galvez Kessler, person living with ALS, as described to the committee during August 2023 public workshop

People with ALS need significant supports at home to maintain quality of life. The symptoms of ALS impede basic motor function and day-to-day activities. Living with ALS means continually adapting one’s living environment to meet changing needs as the disease progresses. Home health care may be an early need for people living with ALS with rapid progression or co-occurring frontotemporal dementia.

Home health care can include assistance from home health aides with activities of daily living; physical, occupational, and speech therapy; respiratory care and ventilator management; complex nursing care; and hospice. Privately hired caregivers or home health care agencies may provide care in the home; the latter are bound by licensure statutes and state and federal regulations regarding care staff competency. Home health care agencies may also be accredited, such as by the Community Health Accreditation Partner, and certified by CMS.

There are four main challenges persons living with ALS face to receive adequate home health care: choosing a home health care provider, being accepted by a home health care provider, paying for home health care, and coordinating home health care with the medical care team. Several organizations, including ALSA,² AARP,³ I AM ALS,⁴ and CMS,⁵ have prepared guides to help individuals living with ALS and their families choose a home health care provider.

Hiring a home health care agency is expensive, and an individual’s insurance may not cover all services. CMS does not have the authority to pay for concomitant services at home and as an outpatient,⁶ leaving some individuals with ALS challenged to receive needed care in the clinic and also at home.

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² Available at http://www.alsa.org/als-care/resources/fyi/choosing-a-home-health-care.html (accessed May 10, 2024).

³ Available at https://assets.aarp.org/external_sites/caregiving/checklists/checklist_inHomeCare.html (accessed May 10, 2024).

⁴ Available at https://www.iamals.org/wp-content/uploads/2020/10/Questions-to-Ask-a-Potential-Home-Health-Agency.pdf (accessed May 10, 2024).

⁵ Available at https://www.cms.gov/Medicare/Quality-Initiatives-Patient-Assessment-Instruments/HomeHealthQualityInits/Downloads/HHQIHHBenefits.pdf (accessed May 10, 2024).

⁶ 42 USC 1395fff.

Medicare certified home health care is unlimited in duration and coverage continues to be available so long as skilled care is needed and threshold criteria are met. Intermittent care refers to noncoverage of daily skilled care after 21 days.⁷ To be eligible for any home health care support, individuals must have a formal ALS diagnosis and be certified by their doctor as being unable to leave their home without considerable effort and assistance.

For individuals meeting low-income requirements, Medicaid can supplement Medicare. While each state sets its own policies, most state Medicaid plans will pay for personal care services, in addition to skilled care services. Some states also offer “waivers” to keep people out of long-term care facilities and in their homes for as long as possible, since the latter is more cost-efficient.

Some private insurance plans cover private duty nursing and home care expenses for people living with ALS. Long-term care insurance—if the policy covers ALS—will pay for in-home skilled nursing, therapeutic care, and personal care. There are also many organizations and local foundations that help pay for home health care for individuals with ALS.

Good coordination between a home care provider and an individual’s clinical team can benefit patients, family caregivers, providers, and payer organizations by reducing hospital admissions and emergency department visits and lowering health care costs (Friedman et al., 2016; Möckli et al., 2021). Medicare’s Conditions of Participation,⁸ which a home health agency must satisfy to participate in Medicare and Medicaid programs, includes care coordination requirements.

Conclusive, nationwide data on use, access, and experiences with home health services for Medicare beneficiaries living with ALS is limited. There are approximately 66 million Medicare beneficiaries, and even if every one of the approximately 30,000 people living with ALS in the United States were enrolled in Medicare, less than half of 1 percent of Medicare beneficiaries will have ALS at any given time. Medicare use data by disease category is not readily available, particularly for rare diseases such as ALS. However, recent CMS data reveal a worrisome, continuous downward trend in Medicare home health use and declines in skilled nursing, physical therapy, occupational therapy, and speech therapy (AARP, 2023). Because of a lack of CMS analysis of these data, it is unknown why these trends are emerging and whether the appropriate level of home health care is being provided to Medicare beneficiaries (AARP, 2023).

Some home health care agencies wrongly deny that Medicare will pay for their services (Center for Medicare Advocacy, 2013). Medicare policies and

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⁷ Intermittent skilled nursing care is care that is needed or given on fewer than 7 days a week (i.e., not daily) or (daily) less than 8 hours a day over a period of 21 days or less.

⁸ Available at https://www.federalregister.gov/documents/2017/01/13/2017-00283/medicare-and-medicaid-program-conditions-of-participation-for-home-health-agencies (accessed May 10, 2024).

incentives generally favor short-term improvement goals, such as posthospital patients who improve, even though Medicare home health coverage is not limited in time for those who meet threshold criteria, nor is restoration or improvements of one’s condition required.

The limitations in access to high-quality, affordable home health care that exist in the United States have a significant effect on the ability of unpaid caregivers to provide the necessary care for their loved ones with ALS. A 2023 scoping review found persistent unmet needs for caregivers in the following areas: emotional and psychological needs; assistive devices and technology; information and education; and human and professional resources and services (Young et al., 2023). Despite the predictable trajectory of ALS, the biopsychosocial and equipment interventions available for people living with ALS were generally not provided proactively. The authors suggest that home health care for people with ALS and the experience of their caregivers should be addressed by examining these areas of need: interdisciplinary care, including home-health and end-of-life care; caregiver outcomes including burnout; durable medical equipment (DME) and technology; patients’ and caregivers’ diverse racial, cultural, and social backgrounds; and policy implications. Young et al. stated a goal to work with the American Academy of Neuromuscular and Electrodiagnostic Medicine to convene an expert panel to develop consensus-based ALS home health medical standards. The guidelines would exist to promote greater accessibility to needed services and supports.

The committee found that precise data are lacking around home health care use for individuals living with ALS who are Medicare beneficiaries. National trends in home health care use and the experiences of individuals living with ALS shared with this committee indicate there is a serious challenge in accessing high-quality, affordable home health care for the complex and evolving needs of people living with ALS. Many individuals living with ALS are not able to get legally covered care and services.

HIGH OUT-OF-POCKET COSTS

The financial side of it has been very frustrating and difficult but it is also time-consuming, and I feel like time is the biggest, most important currency that we live with. If something takes a couple months to get in, in just a couple of months my body may change, and the reasons why we got those adjustments in the first place may not be suitable now.

—Julian Rodriguez, person living with ALS

My parents spent well over $100,000.00 on costs related to my mom’s ALS even though she only survived 16 months after diagnosis. Compassion Care ALS helped my family a lot with many of these incidental things such as wheelchair-accessible van rentals when needed.

—Jean Swidler, presymptomatic genetic carrier and caregiver, Executive Director of Genetic ALS & FTD: End the Legacy

The looming fear of financial catastrophe can have a negative impact on a person with ALS and their current quality of life. The total cost of ALS care, including costs paid by insurers, is higher than it is for other neurological diseases (Gladman and Zinman, 2015). A systematic review of 12 studies from eight countries published between 2001 and 2015 indicated the total annual cost per person living with ALS in the United States was approximately $70,000; the United States had the highest total costs of those countries in the review (Gladman and Zinman, 2015). One case study of a single person with ALS found that from 2000 to 2010, their total cost of care was more than $1.4 million in 2013 USD (Obermann and Lyon, 2015). Given inflation, recent ALS drug approvals, including Relyvrio,⁹ which costs $158,000 per year for an individual patient, and an overall increase in technology options (e.g., communication devices), the committee surmises that the total annual cost of ALS care is far higher than estimates based on data from 2000 to 2015. While understanding the total economic impact of ALS on individuals, insurers, and governments can be useful, understanding the out-of-pocket costs individuals and families affected by ALS are experiencing, including lost wages, is also critical to understanding the true financial impact of the disease.

ALS was the most common neurological condition that users created campaigns for on GoFundMe, a crowdsourced fundraising platform, between 2011 and 2021 (Galvin et al., 2023). People with ALS and their families often seek financial support for home modifications or services and devices they need to make the disease more livable. Regardless of condition, most individuals seeking financial assistance through crowdfunding had insurance, highlighting the financial burdens faced by individuals living with ALS and their families resulting from inadequate insurance support.

The committee received perspectives from several individuals living with ALS and their family members regarding the amount they spend out of pocket to cover care and services that insurance did not cover or denied. Here is a description of some of those experiences:

• Bruce Rosenblum, an individual with lived ALS experience who participated in the committee’s August 2023 workshop and died in December 2023, spent more than 50 percent of his take-home pay over the last 3 years on 30 hours per week of professional caregiving to help each weekday morning with the more challenging activities of daily living. Bruce’s wife retired 3 years earlier to become his primary caregiver but, as the committee also heard from other individuals living with ALS, additional assistance in

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⁹ As the committee was finishing its work on this report, the company developing AMX0035/Relyvrio announced the latest results of a Phase 3 trial in which the drug performed no better than placebo and the company began the process of removing the drug from the market.

the home is needed. Bruce also paid $5,000 out-of-pocket for a fully electric hospital bed and a low air mattress. Insurance denied coverage for these items, though they were necessary because his wife and primary caregiver lives with a muscle condition and could not reasonably care for him using the less effective items insurance would approve—a semielectric hospital bed and a manual Hoyer lift.

• Committee member Mindy Uhrlaub reported her mother paid $25,000 for a power wheelchair Medicare did not cover.
• Ady Barkan, a person with ALS who died in November 2023, spent $40,000 per month for 24-hour care that was reimbursed primarily through insurance via an ad hoc arrangement (King, 2024).

Of those individuals who responded to an ALSA survey on mobility challenges, 26 percent reported they paid at least some of the out-of-pocket costs for a power wheelchair and 47 percent said insurers refused to pay for their power wheelchair seat elevation (ALSA, 2021) (see Figure 3-1). A recently updated CMS policy now covers seat elevation systems for power wheelchairs (CMS, 2023a).

Some individuals living with ALS also pay for traveling to participate in a clinical trial. A recent cost estimate of clinical trial participation comes from committee members affected by ALS. Joel Shamaskin, committee member and person living with ALS, participated in research at two study sites between 2016 and 2019 and paid $3,500 out of pocket to cover transportation, lodging, and meals associated with his research participation. Mindy Uhrlaub, committee member and at-risk ALS genetic carrier, spent more than $12,000 over the course of 2 years to support travel and accommodations associated with participating in longitudinal, observational research studies involving genetic carriers. These observational studies provided no compensation for participants, nor were participants able to benefit from taking an experimental drug in the context of a clinical trial. Out-of-pocket costs to participate in research are deterrents to participation, and the committee believes the appropriate budget for a clinical trial would anticipate and cover the costs associated with a person with ALS participating (Gelinas et al., 2018).

Drug Costs

Drug pricing policies and insurance decisions regarding whether to pay for a drug are complex and not fully transparent. As more ALS drugs are expected to achieve regulatory approval in the next decade, the pharmaceutical choices to treat this disease will grow, along with the costs to patients. While Chapter 5 discusses some of the trade-offs in drug development and approval decisions, there are also near-term opportunities for reducing the out-of-pocket costs borne by people with ALS for drugs. The committee believes one opportunity to address the high cost of drugs to treat ALS is to negotiate drug prices.

As part of the Inflation Reduction Act of 2022, the Secretary of the U.S. Department of Health and Human Services is required to negotiate prices with drug companies for a small number of single-source, brand-name drugs or biologics without generic or biosimilar competitors covered by Medicare (Cubanski et al., 2023). To qualify for this negotiation, the drug must cost Medicare more than $200 million per year, cannot have a generic competitor, and cannot have a single orphan designation. While it is unlikely ALS drugs will qualify for negotiation under the Inflation Reduction Act criteria in the near future, other opportunities to negotiate the price of ALS drugs could positively affect people with ALS in terms of access to affordable drugs that would make the disease more livable.

ALS Organizations Assisting People with ALS

In addition to high out-of-pocket costs for care, equipment, and therapeutics, there are other direct costs that individuals living with ALS and their families bear. Some persons with ALS will have to move to a more

accessible home, make significant home modifications that can cost tens of thousands of dollars, or purchase a wheelchair-accessible van, which can cost upward of $100,000. They may also have to pay for at-home care out of pocket. In addition, there is the loss of income that comes when the individual living with ALS can no longer work or when other adults in the household have to reduce their hours or forgo their job entirely to care for their loved one.

ALS organizations and patient-serving groups offer many different types of resources and sometimes direct financial assistance with home modifications or procuring a wheelchair-accessible van. However, access to these resources is fragmented and depends on where the person with ALS lives. For example, an ALSA chapter in Minnesota might offer assistance with home modification, but a chapter in another state will not. Each ALS organization, and sometimes each chapter within an organization, will have a different philanthropic model, area of focus, and resources. New organizations spun off of state and local ALSA chapters, such as ALS United, will likely result in more variation across the organizational landscape addressing ALS needs. While this structure means that not every ALS organization will be able to offer the same levels of support, an opportunity exists to coordinate and streamline information and resources so that a person with ALS and their family can receive the support they need. The committee heard from individuals with ALS lived experience—persons with ALS, current and former caregivers, and individuals at risk of developing ALS—that the fragmented nature of information and resources across 50-plus ALS organizations can be confusing to navigate, take significant effort and time, and contribute to overall stress and anxiety.

As all of us know, this is an extremely expensive disease, not just in terms of the medicine but also in terms of all the support that you need, and so it was really important that I maintained a full-time role so that we could take some of that pressure off us as a family unit.

—Siobhan Pandya, former caregiver of a person living with ALS

At 45, my husband and I are supposed to be in the prime earning period of our lives, but he has not been able to work full-time due to having to care for me and our kids. I have had to drastically reduce my hours to 35 percent due to time and energy considerations and my inability to teach anymore. While I receive a disability check from my pension system, it doesn’t add up to what we could be earning as a couple if I were healthy.

—Kristin Rankin, person living with ALS

Recommendation 3-1: Facilitate expedited access to and coverage of essential ALS medical and support services.

The Centers for Medicare and Medicaid Services (CMS) and private insurers should act quickly to enable expedited access to the following essential ALS medical and support services:

1. Provide coverage for home based and outpatient physical and other support services for persons with ALS as necessary, of the type and duration needed by persons with ALS, even if services are occurring concomitantly. Congress should grant CMS the authority to provide concomitant services at home and as an outpatient for progressive, neurodegenerative diseases such as ALS.
2. Commit to expedited (within 72 hours) responses to prior authorization requests for all therapies, durable medical equipment, assistive technologies, and services for persons with ALS.
3. Do not deny services for persons with ALS based on failure to show functional improvement, given the progressive nature of the illness.
4. Establish a call center for persons living with ALS, and possibly other rare diseases, and their caregivers to report challenges in receiving care and services.
5. Work with ALS organizations and persons living with ALS and their families to develop a “Know Your Rights” document that describes Medicare, Medicaid, and private insurance requirements and empowers individuals living with ALS to combat misinformation and improper denial of services.

Recommendation 3-2: Enable all persons with ALS to access and make full use of ALS care.

Congress should act quickly to enable all persons with ALS to access timely, specialty ALS care by doing the following:

1. Expand the status of ALS as a qualifying condition for Medicare coverage, such that persons with ALS are eligible for Medicare coverage regardless of age, employment history, or other criteria influencing Medicare or Social Security Disability Insurance eligibility.
2. Require reimbursement of multidisciplinary ALS care under a bundled payment method commensurate with the services provided.

Recommendation 3-3: Provide centralized resources for people with ALS to receive support for needs not otherwise accessible or covered by insurance. ALS nonprofit organizations and patient-serving associations should collaborate to create and maintain centralized resources

to guide people with ALS and their caregivers to organizations and funding mechanisms that can provide financial support for needs not otherwise accessible to them or covered by Medicare, Medicaid, and private insurance. These might include such things as mental health services, modifications to home environments, securing equipment and assistive technologies, and transportation.

SUPPORTING CAREGIVERS

The term caregiver typically refers to a family member or other individual who supports someone living with ALS. These caregivers are sometimes called informal or unpaid caregivers. This report uses the term unpaid caregiver to encompass the individual—family member or other individual—primarily caring for and supporting the individual living with ALS. While informal caregiver is frequently used in the literature, the committee heard that this terminology conveys something “casual” or “not done to the highest standards” which could diminish their important work for individuals with ALS.

ALS requires a high intensity of care at home, and as a result, caregivers face their own unique challenges (Poppe et al., 2020; Williams et al., 2008) (see Box 3-1). The physical demands of caring for someone with ALS are substantial and range from assistance with activities of daily living such as bathing, using the bathroom, eating, and dressing to managing mobility aids (Young et al., 2023). Eventually, the person living with ALS will need feeding devices and breathing machines. In addition, the spouse or other family member of a person living with ALS often manages the household, including child care, cleaning, doing laundry, paying bills, making health care provider appointments, and communicating with other family members.

The challenge of caring for a person living with ALS can lead to depression, anxiety, and other impairments (de Wit et al., 2018). Caregiver burnout is a serious risk as the patient’s condition deteriorates, the responsibilities of care increase, and the hours spent caring for the individual increase (Shan et al., 2021). Studies have shown that psychological distress, hours of care provided, and lower quality of life were significant predictors of higher caregiver burden (Markella Antoniadi et al., 2020; Galvin et al., 2016).

The quality of life of individuals with ALS is also highly intertwined with the quality of life of their caregivers. The well-being of caregivers, typically providing unpaid care in the patient’s home, is challenged as their role as a caregiver evolves (Poppe et al., 2020; Williams et al., 2008). From the time of diagnosis to the disease’s terminal phase, the caregiver assumes many roles: engaging with professional clinical care providers to develop a care plan; navigating insurance; coordinating medical

equipment acquisition; coordinating in-home paid formal care providers; and often learning to monitor complicated, life-sustaining equipment such as ventilators. The ability of the unpaid caregiver to accomplish these tasks can be severely hampered by a lack of adequate resources and supports. Instructions from clinicians, insurers, or DME manufacturers can be rare, and even when it is available, it can be difficult to access. For example, telephone help lines and servicing for DME may not be available around the clock.

A scoping review of 37 studies examined the supportive care needs of individuals living with ALS and their caregivers and identified many deficits (Oh and Kim, 2017). The most common domain areas of unmet needs were categorized into the seven domains of the Supportive Care Needs Framework:¹⁰ practical, informational, social, psychological, physical, emotional, and spiritual. For example, as described earlier in this chapter, individuals living with ALS who have Medicare or private insurance often face complex, demoralizing insurance processes such as prior authorization requirements for services and assistive technologies that are always medically indicated given the very nature of the progressive disease. In these situations, unmet needs in most, if not all, of these support care areas exacerbate the challenges to a person living with ALS and their caregiver’s quality of life.

The hours devoted to unpaid caregiving are substantial. A 2021 ALSA survey found that 68 percent of those surveyed spent more than 30 hours per week providing care (ALS Focus, 2021). Unpaid caregivers can spend more than 100 hours per week providing care (Kennedy et al., 2022; MNDA, 2016). A large multicenter study at three European sites reported in 2021 that unpaid caregivers in each of three community-based cohorts devoted an average of 35 to 97 hours per week to their duties (Conroy et al., 2021). A 2022 study conducted through ALSA of British Columbia examining the effects of respite care found unpaid caregivers provided on average of 65.3 hours of care per week (Wu et al., 2022). Although advances in other aspects of ALS disease management, including drug therapy and digital technologies, have occurred over the past 20 years, there has been no reduction in the time people with ALS require of their family caregivers. Reports from European and Australian cohorts in 2003 and 2012, respectively, found that just over 12 hours per day was spent tending to the care recipient (Aoun et al., 2012; Kaub-Wittemer et al., 2003).

The time devoted to caregiving, combined with the lack of adequate support, contributes to the high level of distress experienced by the unpaid care provider (Oh and Kim, 2017). Documentation of high caregiver burden is noted in studies from various populations (Conroy et al., 2021; de Wit et al., 2019; Galvin et al., 2018; Larsson et al., 2022; Schischlevskij et al., 2021). The consequences for the unpaid care providers include greater risk of depression and other psychological symptoms (de Wit et al., 2019) and significant impairment of their own physical health (Schischlevskij et al., 2021).

Respite care, a service that provides family caregivers with time away from their caregiving responsibilities, can improve the emotional and physical health of a caregiver. Paying for respite care can be a challenge, as few insurance plans will pay for it. Medicare covers 95 percent of the cost of

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¹⁰ See Fitch, 2008.

respite care in approved facilities as part of its hospice benefit for those individuals with ALS who have elected to enroll in a hospice program. Outside of the hospice benefit, Medicare does not cover respite services, but some Medicare Advantage plans may cover respite services. Some states have a respite voucher program, and some states’ Medicaid policies cover respite care.

For veterans, the U.S. Department of Veterans Affairs (VA) covers up to 30 days of respite care annually through the Program for Comprehensive Assistance to Family Caregivers. Faith-based organizations and charitable organizations may provide financial support to offset the costs of respite services, and funds may be available through the National Family Caregiver Support Program administered by local Area Agencies on Aging. Beyond paying for respite care, caregivers for individuals living with ALS also reported to the committee the added challenge of finding qualified, trustworthy respite care providers they felt comfortable leaving the individual living with ALS in their care for an extended period. Given that caring for an individual living with ALS can require understanding and monitoring complex communication or respiratory devices, only individuals with proper training and experience would be qualified to provide respite care.

Models of Caregiver Support

There are numerous models for supporting caregivers, and federal policy continues to evolve to further meet their needs. For example, CMS has reaffirmed the responsibility of hospitals to engage caregivers in hospital discharge processes and post-acute settings. CMS will also now provide payment when practitioners (physician or nonphysician) train caregivers to support individuals with certain diseases or illnesses in carrying out a treatment plan (CMS, 2023b).

The committee believes the following two models, if adapted for people with ALS and their caregivers, could address the challenges that those who care for family members with ALS face.

U.S. Department of Veterans Affairs Caregiver Support Program

The VA has a Caregiver Support Program (CSP) with a CSP team at every VA facility offering clinical services to caregivers of eligible and covered veterans. Veterans can designate one primary family caregiver and up to two secondary family caregivers for eligibility in the program. Primary caregivers receive:

• Monthly stipend paid directly to the caregiver;
• Access to health insurance through VA if they do not already have health insurance;

• Mental health counseling;
• Certain beneficiary travel benefits when traveling with the veteran to appointments; and
• At least 30 days of respite care per year, for the veteran.

Secondary caregivers do not receive a monthly stipend but are eligible for certain travel benefits when traveling with the veteran to appointments and mental health counseling.

Guiding an Improved Dementia Experience (GUIDE) Model

In response to the Biden administration’s April 2023 Increasing Access to High-Quality Care and Supporting Caregivers Executive Order, CMS initiated a test of a new voluntary, nationwide model program that supports people living with dementia and their unpaid caregivers.¹¹ People living with dementia receive fragmented care and often have multiple chronic conditions, leading to high rates of hospitalization and emergency department visits. In addition, people with dementia often have round-the-clock care needs and behavioral and psychological symptoms. Caregivers for people living with dementia report high levels of stress and depression, which negatively affect their overall health and increase their risk for serious illness, hospitalization, and mortality.

The GUIDE model will test an alternative payment for participants, such as dementia care programs comprising an interdisciplinary team, that provides ongoing, longitudinal care and support to people living with dementia. The model aims to address the key drivers of poor-quality dementia care in five ways (CMS, 2023c):

1. Define a standardized approach to dementia care delivery for model participants. This includes staffing considerations, services for beneficiaries and their unpaid caregivers, and quality standards.
2. Provide an alternative payment methodology to model participants. CMS will provide a monthly per-beneficiary payment to support a team-based collaborative approach.
3. Address unpaid caregiver needs. The model will aim to address the burden experienced by unpaid caregivers by requiring model participants to provide caregiver training and support services, including 24/7 access to a support line and connections to community-based providers.
4. Offer respite services. CMS will pay model participants for respite services provided to a beneficiary in their home, at an adult day

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¹¹ At the time of this report’s writing, the GUIDE model had been announced and applications were under review. The model is scheduled to launch on July 1, 2024, and run for 8 years.

center, or at a facility that can provide 24-hour care for the purpose of giving the unpaid caregiver temporary breaks from their caregiving responsibilities.

5. Screen for health-related social needs. Model participants will be required to screen beneficiaries for psychosocial needs and health-related social needs and help navigate them to local community-based organizations to address these needs.

Since the GUIDE model was developed for people with dementia and their caregivers, not all of the above will apply to people with ALS or their caregivers. For example, caregivers for people with ALS might require training from clinicians, home health agencies, DME manufacturers, and community organizations. These support needs might evolve as the care needs of the person with ALS also progress; they might, for instance, require training for a ventilator, a power wheelchair, or a home or vehicle modification at different times. Because of these complex and developing needs, CMS might extend stipends to pay for caregiver training.

Palliative Care to Improve Quality of Life for Persons with ALS and Caregivers

Palliative care is a field of medicine focused on improving quality of life for patients with serious life-limiting illnesses. Neuropalliative care is a specialty of palliative care for patients with neurological disorders and plays a critical role in improving quality of life for individuals living with ALS and their care partners (Brizzi et al., 2019; Phillips et al., 2020). An American Academy of Neurology Ethics and Humanities Committee position statement highlighted the ethical responsibilities of neurologists to their patients, stating,

It is imperative that neurologists understand and learn to apply the principles of palliative care as . . . many patients with neurologic disease die after long illnesses during which the neurologist acts as the principal or consulting physician. (Taylor et al., 2022)

ALSA has also highlighted the importance of providing palliative care to people living with ALS attending an ALS multidisciplinary clinic. Some of the most troublesome ALS symptoms palliative care manages include pain, cramps, spasticity, breathing difficulties, excessive saliva flow, episodes of sudden uncontrollable and inappropriate laughing or crying, and depression. Integrating a palliative care specialist into a multidisciplinary care system can facilitate care delivery by reducing the travel burden to patients and caregivers.

Financial, time, and resource constraints often hamper comprehensive care that includes palliative care (Kluger et al., 2023). Integrating neuropalliative

care within the multidisciplinary care model—during regular clinical visits through templates and checklists, for example (Kluger et al., 2023)—will require better resource allocation and additional workforce. Other barriers to palliative care include lack of awareness among policy makers, health professionals, and patients about palliative care and its benefits. There also are misconceptions that palliative care is only for patients with cancer or those at the end of life, and is sometimes confused with hospice. Finally, there is no downside to receiving palliative care.

Hospice and Palliative Care

Patients become eligible for hospice care when they are expected to live fewer than 6 months. Most U.S. hospice care is provided by Medicare’s hospice program. Private insurance plans may also provide hospice care for patients who do not qualify for Medicare hospice. Most multidisciplinary hospice care occurs at the patient’s residence and occasionally in a long-term care facility. Persons with ALS may have hospice enrollment extended when there is documented evidence of continued progress of terminal disease. Individuals receiving palliative care may be transitioned to Medicare hospice care when the prognosis is 6 months or less.

Elevating and Responding to the Needs of ALS Caregivers

Caregivers for persons with ALS need significant levels of support from a variety of providers that can span diverse and different venues. VA provides substantial supports to caregivers for veterans with ALS and the committee believes similar supports and financial resources should be available to all caregivers for persons with ALS. To meet these needs, the committee offers the following recommendation.

Recommendation 3-4: Address the needs of unpaid caregivers.

Congress, the Centers for Medicare & Medicaid Services (CMS), private insurers, and ALS organizations should address the needs of unpaid caregivers, including respite care, reimbursement for caregiving, and mental and other health support services, including the following:

1. National and local ALS nonprofits should collaborate to develop a priority list of caregiver needs to inform collective advocacy efforts of national ALS nonprofits. This could be accompanied by a guide for ALS caregivers on what to expect through the course of disease and identify resources, which would be used by all national nonprofits and updated collaboratively.

2. Congress should provide financial support for caregivers by amending the tax code to provide a tax credit that could be used by caregivers for individuals living with ALS, as well as all progressive neurodegenerative diseases to alleviate the financial burden of providing unpaid care. Congress should also provide other types of financial relief for caregivers, including allowing them to apply health savings account or flexible spending account funds to caring for a parent or parent-in-law.
3. CMS should ensure legally covered services for home health aides are accessible.
4. CMS should expand tests of payment and service delivery models, such as the Guiding an Improved Dementia Experience model for people with dementia and their caregivers, to include ALS, or create new programs specifically designed to support persons with ALS and their unpaid caregivers. These tests should include:
   • Stipends paid directly to caregivers on an at least a monthly basis,
   • Reimbursing persons with ALS and their caregivers for accessing mental health counseling and psychotherapy via video telehealth (across state lines), and
   • Access to high-quality respite care services.

RESPIRATORY MANAGEMENT OF ALS

Most deaths from ALS result from respiratory failure, making early recognition of respiratory impairment and longitudinal management of chronic respiratory failure critical to improve both quality and quantity of life for individuals living with ALS (Khan et al., 2023; Miller et al., 2009). ALS leads to weakness of the diaphragm and respiratory muscles resulting in progressive and unrelenting shortness of breath exacerbated by impaired cough mechanisms. Increased risk of aspiration of secretions and pneumonia can lead to increased hospitalizations.

In the United States, clinicians use pulmonary function tests (PFTs) to detect and quantify impaired lung physiology and diaphragm weakness. PFTs allow clinicians to identify those people living with ALS who will benefit most from respiratory support in the form of noninvasive mechanical ventilation (NIV) or home mechanical ventilation (HMV).^12,13 NIV uses

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¹² Many other developed countries rely on a simple symptom-based approach rather than PFTs, allowing for earlier initiation of NIV or HMV.

¹³ Some individuals living with ALS choose to have a tracheostomy to receive home ventilation later in disease progression.

a respiratory device that assists and augments the mechanics of breathing to reduce dyspnea, the sensation of running out of air and not being able to breathe fast or deeply enough. Respiratory support using NIV prolongs survival and improves or maintains quality of life (Bourke et al., 2006; Radunovic et al., 2017). Failing to provide NIV for individuals with ALS is no longer justifiable (Ackrivo, 2023; Radunovic et al., 2017; Rimmer et al., 2019). Research is needed, however, to determine the optimal timing and criteria for initiating NIV, although randomized controlled trials are difficult given it is unethical to withhold NIV from a control group (Radunovic et al., 2017; Rimmer et al., 2019).

Assistive ventilation to support diaphragm and respiratory muscle weakness, cough assist devices to clear airway and respiratory secretions, longitudinal monitoring of PFTs,¹⁴ and attentive adjustment of NIV or HMV to match the progressive decline of respiratory strength are the pillars of optimal respiratory management for individuals with ALS (Ackrivo, 2023; Hansen-Flaschen and Ackrivo, 2023). However, managing comprehensive respiratory care is time and labor intensive, requiring attentive home care to optimize multiple medical equipment devices, coordination with pulmonologists with expertise in management of complex mechanical ventilators, and working with DME companies to maintain high-quality, longitudinal respiratory care (Ackrivo, 2023; Hansen-Flaschen and Ackrivo, 2023). In addition, several other factors complicate providing assistive ventilation for people living with ALS, including:

• Current Medicare policy requires an individual to have a forced vital capacity of less than 50 percent before initiating NIV or HMV therapy. A forced vital capacity of less than 50 percent means someone has significant respiratory impairment and severe shortness of breath. This leads to many people living with ALS not qualifying for NIV until they are severely symptomatic with extremes of shortness of breath. Other international guidelines have expanded criteria for NIV, focusing on an earlier symptom-based management approach that allows people living with ALS to proactively benefit from NIV or HMV support earlier in their disease (Wolfe et al., 2021).
• Commercial insurance companies make it difficult, or impossible, for individuals living with ALS to receive the respiratory devices they need. For example, it has been reported that one United Healthcare plan offered under Medicare Advantage denied 86 percent of prior authorization requests for NIV devices prescribed by physicians for individuals living with ALS (Balas, 2024).

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¹⁴ Clinical practice guidelines suggest closely monitoring PFTs at a minimum of 6-month intervals (Khan et al., 2023).

• Challenges exist because of the limited number of health care providers trained in chronic respiratory failure expertise.
• A lack of home support to assist caregivers with the burden of managing all that goes into providing respiratory care at home, such as cough assist, suctioning secretions, and attending to mechanical ventilators and tracheostomy.
• There is a paucity of research trials relevant to respiratory care for individuals living with ALS. With marked advancements in medical technology, home-based respiratory care therapy creates the opportunity to study and understand models of care that allow people living with ALS to maintain their quality of life at home.

Opportunities to Improve Respiratory Care for Individuals Living with ALS

There are many opportunities to optimize respiratory care for people living with respiratory impairment from ALS. VA has demonstrated exemplary comprehensive care for persons with ALS and care partners, particularly in providing comprehensive respiratory care support. Thanks to technological advancements, home ventilation and home physiology monitoring of ventilation and oxygenation now rivals physiologic monitoring performed in intensive care units, enabling persons with ALS to benefit from advances in respiratory management in the comfort of their home. Despite evidence that proactive, respiratory management prolongs survival and improves quality of life, there are several barriers to delivering optimal clinical respiratory care for persons with ALS, including:

• Inconsistencies and uncertainties exist around policies for HMV and backup ventilators to guarantee 24 hours of ventilatory support in Medicare Advantage and commercial insurance plans.
• Insurance plans can limit access to life-saving and life-supporting NIV or HMV devices for persons with ALS. The lengthy and complex prior authorization adjudication process for services, such as receiving physical therapy outside of the multidisciplinary clinic, and equipment, including power wheelchairs and ventilation at home, unnecessarily increases stress, anxiety, and the overall burden of the disease for persons with ALS. ALS multidisciplinary care teams spend many unreimbursed, unaccounted hours on prior authorization requests for equipment needed to care for a person with ALS.
• There is an insufficient number of providers trained in chronic respiratory failure. Opportunities exist to create a new subspecialty in pulmonary medicine devoted to chronic respiratory failure to meet the evolving needs of persons living with ALS (Cao et al., 2024).

• Sufficient supports for caregivers to persons with ALS are lacking. Caregivers face the challenge of managing everything required for respiratory care at home, such as cough assist, suctioning secretions, and attending to mechanical ventilators and tracheostomy.

The committee believes that a diagnosis of ALS and the very nature of the disease is sufficient justification for an individual to access respiratory devices. There is strong scientific evidence to support the earlier initiation of respiratory intervention for individuals living with ALS than is currently allowed under Medicare policy (i.e., the CMS requirement of a forced vital capacity of less than 50 percent). Individuals living with ALS need earlier access to respiratory devices as well as high-quality respiratory care from qualified providers through a multidisciplinary ALS clinic and at home. A unique opportunity exists to quickly update CMS and other insurer policies to align with the scientific evidence and develop reimbursement models for respiratory therapy to be provided in the home.

The American Academy of Neurology recommends involving respiratory therapists in multidisciplinary ALS care to optimize home respiratory management (Miller et al., 2009). Respiratory therapists have the expertise to educate patients and caregivers regarding optimal management of assisted ventilation and cough assistance strategies to allow patients to remain at home, thereby improving their quality of life and satisfaction and reducing unnecessary hospitalizations (Ackrivo, 2023; Hansen-Flaschen and Ackrivo, 2023; Khan et al., 2023). Home respiratory care is not reimbursed, but if reimbursement were expanded to include home patient care, respiratory therapists could substantially improve the quality of home respiratory care management. In addition, there is a marked lack of pulmonary physicians trained in the management of chronic respiratory failure due to neuromuscular conditions and strategies to increase this area are imperative to optimize longitudinal respiratory care (Cao et al., 2024).

However, as discussed earlier, a common barrier to respiratory care is delayed access to HMV. Current Medicare and insurance guidelines preclude acquisition of home ventilation until a person experiences 50 percent reduction in lung function, which leaves persons with ALS distressed with profound shortness of breath (Wolfe et al., 2021). Therefore, CMS should update payment policies to allow for access to home ventilation, consistent with guidance from pulmonary, respiratory therapy, and sleep medicine professional groups and accepted international standards (Wolfe et al., 2021).

Recommendation 3-5: Enable access to respiratory devices and services for people with ALS.

The Centers for Medicare & Medicaid Services (CMS) and private insurers should immediately align coverage of respiratory devices and services for persons with ALS with the current standard of care. CMS and private insurers should also develop reimbursement models that allow respiratory professionals to provide high-quality, longitudinal respiratory care in the home of a person with ALS.

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