Expert Review of Pharmacoeconomics & Outcomes Research
ISSN: 1473-7167 (Print) 1744-8379 (Online) Journal homepage: http://www.tandfonline.com/loi/ierp20
The economic impact of amyotrophic lateral sclerosis: a systematic review Matthew Gladman & Lorne Zinman To cite this article: Matthew Gladman & Lorne Zinman (2015) The economic impact of amyotrophic lateral sclerosis: a systematic review, Expert Review of Pharmacoeconomics & Outcomes Research, 15:3, 439-450 To link to this article: http://dx.doi.org/10.1586/14737167.2015.1039941
Published online: 30 Apr 2015.
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The economic impact of amyotrophic lateral sclerosis: a systematic review Downloaded by [Deakin University Library] at 06:17 20 October 2015
Expert Rev. Pharmacoecon. Outcomes Res. 15(3), 439–450 (2015)
Matthew Gladman1 and Lorne Zinman*2 1 Department of Medicine, Sunnybrook Health Sciences Centre, Queen’s University, Ontario, Canada 2 Department of Medicine, University of Toronto, Ontario, Canada *Author for correspondence: [email protected]
Amyotrophic lateral sclerosis (ALS) is a devastating neurological disease for which there is no cure, and the associated economic burden is considerable. In this review, the authors summarize the existing body of literature pertaining to the costs associated with ALS to demonstrate the scale and scope of the economic burden of this paralyzing disease. Twelve studies from eight countries published between January 2001 and January 2015 met the inclusion criteria and were included in this review. Direct and indirect costs varied significantly across countries. Standardized to the 2015 US$, the annual total cost per patient ranged from US$ 13,667 in Denmark to as high as US$ 69,475 in the USA, with the national economic burden of ALS estimated at US$ 279–472 million in the USA. Costs associated with ALS were greater than that of other neurological diseases, indicating a continued need for medical advances and financial support for patients and families. Regional cost analyses are necessary to determine how best to spend funds that have been raised globally from the ice bucket phenomenon. KEYWORDS: ALS . Amyotrophic lateral sclerosis . burden of illness . costs . economics . healthcare . ice bucket .
motor neuron disease
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review
Amyotrophic lateral sclerosis (ALS) is a devastating neurologic disorder characterized by the progressive loss of voluntary motor activity, muscle atrophy and ultimately death. The average age at disease onset is in the sixth or seventh decade of life and median survival is 2–4 years from symptom onset [1,2], with the majority of patients succumbing to respiratory failure. ALS results in complete paralysis in most cases, resulting in progressive impairment in activities of daily living, including walking, eating and speaking. As a result, there is a significant negative impact on the quality of life of both patients and their families [3–5]. Currently, there is no cure and the sole drug proven to mildly prolong survival is riluzole [6–8]. Otherwise, the only interventions that have been shown to prolong survival in ALS are the use of bi-level positive airway pressure ventilation [9–14], and the implementation of specialized multidisciplinary ALS clinics [15,16]. Invasive tracheostomy and long-term mechanical ventilation can prolong survival for patients who develop respiratory failure; however, only a minority of patients choose this option [17–19] given the considerable cost, need informahealthcare.com
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for 24-h support, substantial caregiver burden, rate of complications and eventual progression to a completely paralyzed ‘locked-in state’. In the absence of a cure, the primary focus of ALS disease management is on symptomatic treatment and supportive care through multidisciplinary ALS clinics, non-profit ALS Societies, public health resources and private insurance [20]. In addition to the progressive functional disability experienced by patients with ALS, the economic burden on patients, families and society is considerable. Expenses associated with the disease can be divided into direct and indirect costs. Direct costs are those expenses requiring payment, whether by the patient, government or a third-party payer (i.e., health insurance and non-profit-organizations), such as those associated with formal health care, medications, equipment, home or vehicle adaptations, medical devices, mobility aids and services. Direct costs can be further divided into healthcare costs and non-healthcare costs. Indirect costs represent opportunity loss, most notably lost income due to a patient’s illnessrelated absence from work or the voluntary
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caregiver duties taken on by family or friends (otherwise known as ‘informal care’). Both direct and indirect costs can overwhelm the financial resources of patients and their families. The economic burden of any disease can be considered from either the societal perspective, in which no distinction is made between costs to the patient, government or medical insurance companies, or the patient perspective, in which only out-ofpocket costs borne by the patient are considered. Both perspectives are important to consider when evaluating the overall economic burden of ALS. In July 2014, the ‘Ice Bucket Challenge’ emerged as a social media phenomenon in the USA, which involved filming yourself having a bucket of iced water dumped overhead to increase ALS awareness and funding. The challenge spread over social media to become a global activity undertaken by millions of people across over 150 countries and amassing over one billion YouTube views in a few short months. During the peak month of August 2014 alone, the American ALS Association received over US$ 100 million (compared with US$ 2.8 million in August 2013), with millions more donated to ALS Societies around the world. While the majority of funds may be allocated to ALS research, a significant amount will likely be allotted to patient care. In this systematic review, we summarize the existing body of literature pertaining to the costs associated with ALS to demonstrate the scale and scope of the economic burden of this paralyzing disease. This will help to identify the most costly components of the disease and can provide guidance to ALS Societies in how best to assist patients and families with the unprecedented influx of ALS funds recently raised. Methods Search strategy
A PubMed database search was conducted for relevant articles published between January 2001 and January 2015, citing ‘amyotrophic lateral sclerosis’ or ‘motor neuron(e) disease’ and ‘cost’ or ‘economics’. Bibliographies of key references were checked to identify any other relevant articles. Study selection
Two researchers reviewed the titles and abstracts to determine eligibility for full review. Texts were screened for eligibility if there was ambiguity of relevance in the title or abstract of any studies or if there was no abstract available. The following criteria were used to determine whether studies were included for full-text review: . . . . .
study was conducted for ALS; study assessed the direct and/or indirect costs associated with ALS (from either a societal or patient perspective); study was a primary research article based on human subjects; study was published between January 2001 and January 2015; study was published in the English language.
440
Data abstraction
For each publication included in this review, information was collected for the country of investigation, study perspective, study type and methodology, patient characteristics and costs (total, direct healthcare, direct non-healthcare and indirect, when available). Costs reported by each study were standardized to US dollars and inflated to 2015 dollars using the Organization for Economic Cooperation and Development gross domestic purchasing power parities (PPP) conversion rates [21]. Studies reporting monthly costs were extrapolated to per annum costs for ease of comparison. All costs are reported as per person per year (unless otherwise noted). Costs are rounded to the nearest dollar. Results
Twelve relevant studies were identified in the literature that met inclusion criteria. The characteristics, methodology and results reported in the review studied are described here and in TABLE 1. In the Netherlands, Van der Steen et al. [22] conducted a 2009 prospective, longitudinal observational study in 98 ALS patients who maintained a cost-related diary at regular intervals. The study aimed to contrast the direct and indirect costs of ALS patients receiving specialized multidisciplinary care compared with those receiving general care, as well as to identify clinical drivers of cost. The mean monthly cost per patient was e1313, which included all direct and indirect costs, and did not differ significantly in patients receiving multidisciplinary and general care. Total cost increased with disease severity, and, in particular, total direct non-healthcare costs (devices, aids, home adaptations, etc.) was significantly driven by disease severity: e11,669, e4750 and e3855 for patients with severe, moderate-to-severe and mild-to-moderate ALS, respectively. It is important to note that none of the patients in this study underwent invasive or non-invasive ventilation. The study concluded that while specialized multidisciplinary care was associated with improved quality of life and survival in other studies [15,16,23], it was not associated with a higher economic burden on patients or society. In Spain, Lopez-Bastida et al. [24] conducted a 2009 retrospective cost-of-illness study in 63 patients who were surveyed via questionnaire and divided into a high and low severity group, defined based on need for caregiver assistance. Direct healthcare costs, direct non-healthcare costs (primarily, informal care) and indirect costs were estimated based on the data gathered using a variety of public economic resources. The study reported a mean per patient per year (PPPY) total cost e36,194, of which e8289 was attributable to direct healthcare costs, e19,330 to direct non-healthcare costs and e8575 to indirect costs. Patients in the high severity group incurred a total PPPY cost that was more than twice that of low severity patients, at e42,728 and e17,000, respectively. The majority of the expenses can be attributed to a substantial discrepancy in informal care costs, as would be expected given how the two groups were defined. The most expensive categories of direct Expert Rev. Pharmacoecon. Outcomes Res. 15(3), (2015)
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n: 98 patients Mean age: 57.0 years Gender (m/f): 66/32 Mean disease duration from onset: NR (all patients £3 years at time of inclusion) Distribution of severity: 36.7% mild to moderate (ALSFRS 30–40), 42.9% moderate to severe (ALSFRS 20–30), 20.4% severe (ALSFRS 0–20) Distribution of region onset: 80.4% limb onset, 19.6% bulbar onset
Netherlands
Spain
Van der Steen et al. (2009)
Lopez-Bastida et al. (2009)
Retrospective cost-of-illness study using data collected via questionnaire, including both healthcare and non-healthcare resource use in the preceding 6 months, and information pertaining to indirect costs. Direct health care costs were estimated using various public resources. Direct non-healthcare costs included informal care, which along with indirect costs, was estimated using the human
.
– Mild/moderate = e3855 [US$5317] – Moderate/severe = e4750 [US$6552] – Severe = e11,669 [US$16,095]
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– Medications = e4696 [US$7657] – Orthopedic devices = e1767 [US$2881] – Other (hospital admissions, ER visits, clinic visits, medical tests/ exams, home care, transport, rehab) = e1826 [US$2978] High severity vs low severity patients = e8892 vs 6517 [US$14,499 vs 10,623]
– High severity patients = e42,728 [US$69,673] – Low severity patients = e17,000 [US$27,720] Direct healthcare cost . Mean direct healthcare cost = e8289 (US$13,516)
Total (indirect and direct) cost . Mean total cost = e36,194 [US$59,018]
.
– No significance comparing multidisciplinary vs general care or bulbar vs spinal-onset Significantly increased cost with disease severity
– Medical specialist = e984 vs 1056 [US$1357 vs 1457] – Paramedical treatment = e1776 vs 2472 [US$2450 vs 3410] – Medication = e4176 vs 4176 [US$5760 vs 5760] – Hospitalization = e1032 vs 1092 [US$1423 vs 1506] – Household help = e780 vs 972 [US$1076 vs 1341] Direct non-healthcare cost . Mean global (not annual) cost of devices/aids and home adaptations per patient = e5833 [US$8045]
– Mild/moderate = e13,188 [US$18,190] – Moderate/Severe = e16,488 [US$22,741] – Severe = e24,444 [US$33,715] Mean healthcare costs (general care vs multidisciplinary care):
.
Total (direct and indirect) cost . Mean total cost = e15,756 [US$21,732]
Prospective, longitudinal observational study using data from cost-related diary entries of patients over a 6 months period. Based on diary entries and interval questionnaires, direct costs were calculated as per the Dutch guidelines for cost analysis in healthcare research. Indirect costs were estimated using the Health and Labour Questionnaire. Currency–year: 2003
– No significance comparing multidisciplinary vs general care or bulbar vs spinal-onset Trend towards increased total cost with disease severity
Costs reported, per patient per year (PPPY; unless otherwise noted; cost in US$2015)
Study methodology
[24]
[22]
Ref.
ALSFRS-R: ALS functional rating scale (revised); ALSSS: ALS severity scale; NR: Not reported; OOP: Out-of-pocket; OT: Occupational therapy; PPPY: Per person per year; PSW: Personal support worker; PT: Physiotherapy.
n: 63 patients Mean age: 59.1 years Gender (m/f): 33/30 Mean disease duration from diagnosis: 3.7 years Distribution of employment: 27.0% retired, 17.5% housework, 6.3% temporary disability, 42.9% permanent disability, 1.6% active, 4.8% unknown Distribution of severity: 74.6% high severity (need
Study population
Country
Study (year)
Table 1. Characteristics and summary of ALS economics studies.
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n: 46 patients Mean age: NS (17.4% 31–50 years, 67.4% 51–70 years, 15.2% >70 years) Gender (m/f): 26/20 Mean disease duration from onset: 28.9% severe (ALSSS £ 23), 33.3% moderate to severe (ALSSS 24–31), 37.8% mild to moderate (ALSSS 32–40)
Multicenter cost-of-illness study using data obtained both retrospectively via questionnaire for the preceding year and prospectively via diary for a 4-month period. Direct costs were estimated using patientreported co-payments and costs paid by health insurance, estimated using various public resources. Indirect costs consisted of both productivity
Population-based cohort study of all ALS patients diagnosed in Denmark between 1998 and 2009, based on the National Patient Registry (NPR). Direct and indirect cost data was collected utilizing the NPR and various government databases, both retrospectively and prospectively. The same data was gathered for patients’ partners. Randomly selected controls (4:1 ratio), which were age, sex, and location-matched, were used via a Denmark statistics database. Currency year: 2009
capital approach to productivity losses. Currency year: 2004
caregiver assistance), 25.4% low severity
n: 2394 patients Mean age: NR (11.3% 70 years: e11,300 [US$14,627]
Total (direct and indirect) cost . Mean total cost = e$36,380 [US$47,092] . Significant drivers of total cost:
– Outpatient treatment = e955 [US$915] – Inpatient treatment = e9595 [US$9191] – Medication = e2308 [US$2211] – Health insurance = e1409 [US$1350] Indirect cost . Income loss (Patients, pre vs post-diagnosis) = e1956 vs e4651 [US$1874 vs 4455] . Income gain (partners, pre vs post-diagnosis) = e380 vs e3195 [US$364 vs 3060] Cost trends . Annual total health cost significantly increased at time of diagnosis compared to pre-diagnosis, but gradually decreased thereafter. . After diagnosis, employment rate decreased, income from employment decreased, and welfare income increased over time compared to prediagnosis and compared with controls. The opposite was seen in patients’ partners
Direct health cost . Mean direct health cost (pre vs post-diagnosis) = e2508 vs 14,268 [vs control post, e2965; US$2425 vs 13,667 vs 2840]
– 89% early retirement, 9% sick leave, 2% reduced work hours – No association with disease severity
– High severity patients = e25,837 [US$42,130] – Low severity patients = e218 [US$355] . note: 99% of this category is informal care Indirect cost . Mean lost productivity = e8575 [US$13,983]
Direct non-healthcare cost . Mean direct nonhealthcare cost = e19,330 [US$31,520]
Costs reported, per patient per year (PPPY; unless otherwise noted; cost in US$2015)
[26]
[25]
Ref.
ALSFRS-R: ALS functional rating scale (revised); ALSSS: ALS severity scale; NR: Not reported; OOP: Out-of-pocket; OT: Occupational therapy; PPPY: Per person per year; PSW: Personal support worker; PT: Physiotherapy.
Germany
Denmark
Jennum et al. (2012)
Schepelmann et al. (2010)
Country
Study (year)
Table 1. Characteristics and summary of ALS economics studies (cont.).
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Germany
USA
Wasner et al. (2001)
Larkindale et al. (2014)
n: 1528 patients for direct medical cost calculations (945 with commercial plan, 583 with Medicare) n: 124 patients for direct non-medical and indirect cost calculations
n: 171 patients Mean age: 58 years Gender (m/f): 97/72 Mean disease duration: 4.9 years Distribution of region onset: 68% limb-onset, 32% bulbar-onset
Study population
National, retrospective cost-ofillness study in which direct medical costs were derived using commercial and Medicare claims data. Non-medical costs and indirect costs (including potential earning lost from both patients and caregivers) were determined using data from a mailed costof-illness survey. The overall cost to society was estimated based on the total PPPY cost and the estimated 2010 US prevalence of ALS. Currency-year: 2010
Cross-sectional study using a mailed questionnaire to survey patients’ use and the associated costs of complementary and alternative medicine. Currency-year: NR
loss and informal care, and were estimated as per the human capital approach. Currency-year: 2009
Study methodology
54% of patients reported using complementary or alternative medicine Mean global cost per user: e4142 [US$5824]
– – – – –
Inpatient care (acute) = US$9955 [US$10,859] Inpatient care (non-acute or long term) = US$1875 [US$2045] Outpatient care = US$14,957 [US$16,315] Durable medical equipment = US$3087 [US$3367] prescription medication = US$2498 [US$2725]
– Wheelchair use = US$57,903 vs 9932 [US$63,159 vs 10,834] – Ventilator use = invasive: US$84,789 vs NIPPV: US$50,478 vs none: US$24,102 [US$92,486 vs 55,060 vs 26,290] Direct medical cost . Mean direct medical cost = US$31,121 [US$33,946]
– based on prevalence of 1.3–2.2 per 100,000 Total (direct and indirect) cost . Mean total cost = US$63,693 [US$69,475] . Significant drivers of total cost:
Total national cost . US$256–433 million [US$279–472 million]
.
.
– Informal care = e13,510 [US$17,488] – Premature retirement = e7510 [US$9721] – Temporary disability = e380 [US$492]
– Hospitalization = e2990 [US$3870] – Rehabilitation = e320 [US$414] – Outpatient care = e990 [US$1282] – Ancillary therapy = e1450 [US$1877] – Medications = e5370 [US$6951] – Specialized equipment = e1110 [US$1437] – Formal care = e1560 [US$2019] Indirect cost . Mean annual family income loss = e21,400 [US$27,701]
Direct cost . Mean direct cost = e14,980 [US$19,391]
Costs reported, per patient per year (PPPY; unless otherwise noted; cost in US$2015)
[28]
[27]
Ref.
ALSFRS-R: ALS functional rating scale (revised); ALSSS: ALS severity scale; NR: Not reported; OOP: Out-of-pocket; OT: Occupational therapy; PPPY: Per person per year; PSW: Personal support worker; PT: Physiotherapy.
Country
Study (year)
Table 1. Characteristics and summary of ALS economics studies (cont.).
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444
USA
Dubinsky et al. (2006)
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n: 33 patients Mean age: 61.6 years Gender (m/f): 15/18
n: 17,249 Mean age: 65.2 years Gender (m/f): 9263/7986
n: 25 patients (vs 159 lung cancer patients) Gender (m/f): 12/13
Study population
Retrospective cost-of-illness study utilizing a chart review to determine health resource use, and interviews to collect work absenteeism and informal care data used to estimate indirect cost via national minimum wage. Direct costs were determined using the National Health System price lists. Currency-year: 2013
Retrospective cohort comparison of all ALS hospital admissions in USA between 1988 and 2002, using data from the nationwide inpatient sample. Cost (inflationadjusted), reason for admission, length of stay, discharge disposition, and hospice care use were trended over time. Currency year: 2002
Prospective cohort study using patient and financial records to compare the costs associated with hospice care for ALS patients vs lung cancer patients. Currency-year: 2003
Study methodology
– 1988 = US$21,574 [US$28,003] – 2002 = US$24,314 [US$31,559] – Significant increase despite median length of stay decreasing from 6 days to 4 days
Mean hospital-related cost per patient per admission
– Staff services = US$3320 [US$4225] – Medical equipment = US$1252 [US$1593] – Medications = US$866 [US$1102] Mean global cost per lung cancer patient = US$2659 [US$3384] (mean length of stay, 35 days)
Mean global cost per ALS patient = US$5623 [US$7156] (mean length of stay, 87 days)
– Lost productivity = e1321 [US$1995] – Informal care = e1825 [2756]
– Medications = e2437 [US$3680] – Hospitalizations = e686 [US$1036] – Laboratory work = e515 [US$778] Indirect cost . Mean indirect cost = e3145 [US$4750]
Total (direct and indirect) cost . Mean total cost = e7450 [US$11,251] Direct cost . Mean direct cost = e4305 [US$6501]
.
.
.
– Home modifications or moving = US$7106 [US$7751] – Car modifications of purchase = US$2064 [US$2251] – Professional caregiving = US$4570 [US$4985] – Other (i.e., food supplements, travel, etc.) = US$5908 [US$6444] Indirect cost . Mean annual family income loss = US$14,682 [US$16,015] . Significantly increased indirect cost with greater level of care required
No significant difference in cost comparing patients with Medicare or commercial plans Direct non-medical cost . Mean direct non-medical cost = US$17,889 [US$19,513]
.
Costs reported, per patient per year (PPPY; unless otherwise noted; cost in US$2015)
[31]
[30]
[29]
Ref.
ALSFRS-R: ALS functional rating scale (revised); ALSSS: ALS severity scale; NR: Not reported; OOP: Out-of-pocket; OT: Occupational therapy; PPPY: Per person per year; PSW: Personal support worker; PT: Physiotherapy.
Greece
USA
Elman et al. (2006)
Athanasakis et al. (2014)
Country
Study (year)
Table 1. Characteristics and summary of ALS economics studies (cont.).
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Ireland
Canada
Connolly et al. (2014)
Gladman et al. (2014)
n: 49 patients Mean age: 62.6 years Gender (m/f): 23/26 Mean disease duration from diagnosis: 2.8 years Distribution of employment: 61.2% lost job due to ALS, 38.8% retired at diagnosis Distribution of severity: 28.9% moderate (ALSFRS-R 31–38), 46.7% severe (ALSFRS-R 21–30), 24.4% profound (ALSFRS-R £ 20) Distribution of region onset: 89.8% limb-onset, 10.2% bulbar-onset
n: 119 patients Mean age: NS (7.6% 4 hr/week = CDN$20,718 vs 17,768 [US$17,126 vs 14,687] Indirect Cost . Mean annual income loss when patient lost job due to ALS = CDN$ 56,821 [US$46,968]
.
– Renovation costs = CDN$10058 [US$8314] – Mobility costs = CDN$3749 [US$3099] – Medical costs = CDN$2848 [US$2354] – Private PT/OT/PSW = CDN$2920 [US$2414] Significant drivers of OOP direct cost:
Direct cost . Mean OOP direct cost = CDN$19,574 [US$16180]
.
.
.
Direct cost . Median direct cost = e21,540 [US$25,949]
Retrospective cost-of-illness study using hospital-based health resource data from a chart review, as well as community health resource data from a structured questionnaire with a subset of patients (n = 17). Associated costs were estimated using a variety of published resources. Only services paid for by the health service were included, thus excluding out-of-pocket costs, informal care, and productivity losses. Currency year: 2011 – Multidisciplinary clinic = e4440 [US$5349] – Community based care = e15,516 [US$18,692] – Aids and appliances = e1584 [US$1908] Significant drivers of direct cost:
Costs reported, per patient per year (PPPY; unless otherwise noted; cost in US$2015)
Study methodology
[33]
[32]
Ref.
ALSFRS-R: ALS functional rating scale (revised); ALSSS: ALS severity scale; NR: Not reported; OOP: Out-of-pocket; OT: Occupational therapy; PPPY: Per person per year; PSW: Personal support worker; PT: Physiotherapy.
Country
Study (year)
Table 1. Characteristics and summary of ALS economics studies (cont.).
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medical costs were medications and orthopedic devices, which accounted for a mean PPPY cost of e4696 and e1767, respectively. Indirect cost was primarily due to early retirement (89%), whereas sick leaves and reduced work hours only accounted for 9 and 2%, respectively. The study concluded that total expenses increases with disease severity, and the economic and social costs of ALS require increased attention by health authorities and society. In Denmark, Jennum et al. [25] conducted a populationbased cohort study of all ALS patients diagnosed between 1998 and 2009, based on the National Patient Registry. A total of 2394 patients and their partners were matched to controls, and cost data were collected both retrospectively and prospectively. The study reported a mean PPPY direct cost of e14,268, almost sixfold greater than cost prediagnosis and that of the control group. As expected after diagnosis, employment rate and total income from employment decreased, and welfare income increased over time, with PPPY indirect cost being e4651. Interestingly, the opposite trend was seen in patients’ partners. The study concluded that ALS leads to higher healthrelated and social transfer (welfare) costs, lower levels of employment and income, and a seemingly increased workload and economic burden taken on by the spouse to compensate for the increased expenses. In Germany, Schepelmann et al. [26] conducted a 2010 multicenter, retrospective and prospective cost-of-illness study of 46 patients. Data on both direct and indirect costs were obtained using a questionnaire pertaining to the preceding year and 4 months prospective via cost diaries. The reported total PPPY cost was e36,380, of which e14,980 was attributable to direct cost and e21,400 to indirect cost. Several significant cost-driving factors were identified, including disease severity (as per ALS functional scores), artificial ventilation, artificial nutrition, dependency in activities of daily living, presence of dementia, and interestingly, age correlated inversely with total cost. The greatest direct cost was that of medications at e5370 PPPY. Other important direct costs were hospitalization (20%), ancillary therapy (10%), formal care (10%), special equipment (7%), rehabilitation (7%) and outpatient care (2%). Indirect cost consisted primarily of informal care and premature retirement, at e13,510 and e7510 PPPY, respectively. The study concluded that the socioeconomic burden of ALS in Germany has a considerable impact on patients and their families, as the costs of lost productivity is greater than the direct costs paid by health insurance. Another German study conducted by Wasner et al. [27] in 2001 surveyed patients’ use and the associated costs of complementary and alternative medicine (CAM) via questionnaire. They found that 54% of the 171 patients surveyed reported CAM use, at an average lifetime cost of e4142, the majority of which was out-of-pocket. They concluded that patient interest in CAM is significant and future research should explore the use of CAM for management of ALS symptoms. Among the studies conducted in the USA, in 2014 Larkindale et al. [28] used retrospective commercial and 446
Medicare claims data to calculate direct costs and a mail-based questionnaire to estimate direct non-medical and indirect costs using data from 124 respondents. The study reported a total PPPY cost of US$ 63,693, with ventilator use (both invasive and non-invasive ventilation) and wheelchair use being significant drivers of cost. Annual direct medical cost amounted to US$ 31,121 per patient. Outpatient care and acute inpatient care comprised the majority of direct medical costs (48 and 32%, respectively), while non-acute inpatient care, medical equipment and medications accounted for the other 20%. Direct non-medical costs, including home and vehicle modifications and professional caregiving, amounted to US$ 17,889 PPPY. In this study, indirect cost was less than half that of direct cost, estimated at US$ 14,682 PPPY, which included the productivity loss of both the patient and their informal caregivers. Overall, based on a US prevalence estimate of 1.3–2.2 per 100,000, the estimated total national cost of ALS to society was US$ 256–433 million per year. The study concluded that the economic impact of ALS and other neuromuscular diseases is substantial, both for society and affected households, which suggests a possible role for additional policy initiatives to better help affected individuals and their families. In 2006, Elman et al. [29] conducted a prospective cohort study out of Pennsylvania using patient and financial records to compare the costs associated with hospice care for patients with ALS and lung cancer. The mean total cost of hospice care per ALS patient was US$ 5623, more than twice that of lung cancer patients although ALS patients’ mean length of stay was nearly threefold longer at 87 days. The majority of costs were attributable to staff services (59%), medical equipment (22%) and medications (15%). The study concluded that end-of-life care in ALS is costly, and significantly more so than palliative lung cancer patients. Dubinsky et al. [30] conducted a retrospective cohort comparison of all ALS hospital admissions in USA between 1988 and 2002 and found a significant increase in cost per admission from US$ 21,574 in 1988 to US$ 24,314 in 2002, despite decreasing length of stay. In Greece, Athanasakis et al. [31] conducted a 2014 retrospective cost-of-illness study of 33 patients using chart review data to estimate direct costs and structured interviews to evaluate indirect cost. The study reported a mean PPPY direct cost of e4305, with the main factors being medications, hospitalizations and laboratory work at e2437, 686 and 515, respectively. Annual indirect cost was estimated at e3145 per patient, 58% of which was due to loss of income from care provided by family and friends while 42% was patient lost income. The study found that the total annual cost of e7450 was significantly lower than that reported by studies conducted in other countries and concluded that it was attributable to lower medication prices, lower healthcare tariffs and very low minimum wage. In Ireland, Connolly et al. [32] conducted a 2014 retrospective cost-of-illness study using health resource data collected from a chart review of 119 ALS patients. In addition, a subset of 17 patients was further surveyed via a structured questionnaire to determine health resource use in the community, as well as Expert Rev. Pharmacoecon. Outcomes Res. 15(3), (2015)
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Annual cost per person reported (standardized to US$-2015)
US$80.000 use of aids and appliances. Only services paid for by the health service were US$70.000 included, and the study did not deterTotal cost mine out-of-pocket expenses or indirect Direct cost US$60.000 costs. The study reported a median Indirect cost monthly cost per patient of e1795, of US$50.000 which 1293 was attributable to US$40.000 community-based care, 370 to costs associated with the multidisciplinary clinic US$30.000 (including medications), and 132 to aids and appliances. The most significant US$20.000 cost-driving factors were the use of noninvasive ventilation (e513 vs 327), gasUS$10.000 trostomy (e803 vs 378), and they found US$0 an inverse association between monthly costs and length of survival. The study Netherlands† Spain Germany USA Greece‡ Ireland Canada§ concluded that the costs of communityFigure 1. Total, direct and indirect cost of amyotrophic lateral sclerosis per based services are a large contributor to person per year, stratified by country of study. the overall economic burden of ALS on † Netherlands study did not report direct or indirect cost. ‡ health services in Ireland, and the overall Ireland study only reported direct cost. § costs for Ireland seem to be higher than Canada study only reported direct and indirect cost; total cost could not be extrapolated given non-out-of-pocket expenses were not included. other European countries. In Canada, our group [33] conducted a retrospective cost-of-illness study in 2014 using data collected from standardized surveys that reported total (direct and indirect) cost, the standardized total probed out-of-pocket direct costs and indirect costs due to annual cost per patient was found to be US$ 69,475 in the income loss of patients and caregivers. The study reported a USA, 59,018 in Spain, 47,092 in Germany, 21,732 in the mean PPPY out-of-pocket direct cost of CDN$ 19,574, of Netherlands and 11,251 in Greece. The standardized annual which 10,058 was attributable to renovation costs, 3749 to direct cost per patient was found to be US$ 53,459 in the mobility aid costs, 2848 to medical costs, and 2920 to the cost USA, 45,036 in Spain, 19,391 in Germany, 16,180 in Canada of a private physiotherapy, occupational therapy and personal (out-of-pocket proportion), 25,949 in Ireland, 6501 in Greece support work. Several out-of-pocket direct cost-driving factors and 13,667 in Denmark. Annual indirect cost varied greatly were identified, including poor gross motor function (ALSFRS- across studies and was largely dependent on method of calculaR score £6), limb-predominant disease and increased care needs tion and how the costs were defined. Indirect costs were (formal personal support work care >4 h/week). Indirect cost reported to be the highest in Canada at US$ 46,968. The total was stratified by patient employment status. For patients who national cost burden of ALS was only calculated by one lost their job due to disability, mean annual income loss was study [28], which reported a disease-associated cost of US$ CDN$ 36,467, and family members reported an additional 279–472 million per year in the USA. loss of 20,353 due to caregiver responsibilities. Although retired patients had no opportunity loss, their family had a mean Expert commentary annual income loss of CDN$ 16,177. We concluded that out- ALS is a physically and emotionally devastating disease characof-pocket expenses incurred by ALS patients and their families terized by a rapid decline in function and independence and is are substantial, illustrating the need to mobilize additional associated with a considerable increase in care needs over a short period of time. This systematic review verifies that the resources for families affected by ALS. economic burden of ALS is significant, both to society and affected families. In the USA, the 2015 standardized societal Standardized costs To meaningfully interpret the costs reported by the studies cost of ALS was found to be US$ 279–472 million annually. reviewed, all expenses were converted and inflated to a standard Total PPPY expenses varied across countries, but were sizable US$ 2015 currency year, as reported in TABLE 1. An illustration in all reviewed studies, ranging from US$ 11,251 in Greece to of the comparative total, direct and indirect PPPY costs strati- as high as 69,475 in USA (standardized to 2015 US$). Not fied by country of study is shown in FIGURE 1. However, cost surprisingly, the per capita cost associated with ALS is greater comparisons between studies are limited by heterogeneity in than that of any other neurological disease. By comparison, the healthcare systems, payer coverage, study designs, cost defini- annual total cost per person in the USA for dementia, tions, inclusiveness of cost data and study perspectives (societal, Parkinson’s disease, and first-time stroke (weighted by proporpatient-based and/or third-party payer). Of those studies that tion of patients with different stroke types) is estimated at
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US$ 61,400 [34], 22,851 [35] and 10,882 [36], respectively (adjusted to 2015 US$). This disparity is likely, in part, attributable to ongoing interventions in ALS such as artificial ventilation and nutrition, which were significant cost-drivers in multiple studies, as well as profound functional disability, which is invariably accompanied by increased care needs throughout the course of disease, eventual inability to work, durable medical equipment (i.e., wheelchairs) and often home renovations. Like ALS, Parkinson’s disease and dementia are neurodegenerative diseases in which medical and assistive needs invariably increase throughout the course of disease. While annual costs per person are greater in ALS, survival is significantly shorter and thus cumulative costs may be greater in other neurodegenerative diseases. It is important to note that, however, there is a cohort of ALS patients in which survivorship is prolonged [37,38], which would be accompanied by a substantial cumulative cost. We found that across all countries reviewed, ALS is associated with both significant direct and indirect costs. Major contributors to direct healthcare cost included medications, hospitalization, outpatient care, aids and medical equipment. Direct non-healthcare-related expenses were only surveyed in a few studies, but associated costs were consistently large with the most significant contributor being home modifications, which in itself ranged as high as US$ 8314 PPPY in Canada. Indirect cost was driven by productivity loss of patients, in addition to that of voluntary caregivers in some of the reviewed studies. Although indirect costs were substantial, they were likely still underestimated as studies did not consider premature mortality in determining income loss for patients. Direct costs were higher than indirect costs for four of the six studies where they were both measured. This systematic review is timely given the unprecedented influx of tens of millions of dollars in donations to regional ALS associations globally from the ‘Ice Bucket Challenge’. While the majority of funds may be allocated to research, a substantial portion will be earmarked for patient care and each ALS societies will be tasked in determining how these funds are best spent. Identification of the cost-driving factors that have the most significant personal impact to patients and families will help to optimize how funds are spent. This review illustrates the substantial cost associated with ALS. However, most studies focused on a societal rather than patient perspective, such that no distinction is made between costs to the patient, government or medical insurance companies, and thus it is difficult to ascertain where the greatest financial gaps are to individual patients. Determination of out-ofpocket expenses to the individual patient and family likely serves as the truest metric representing the financial toll of the disease. Out-of-pocket expenses would control for the variable contribution made by public healthcare services, non-profit agencies and insurance companies and allow for more meaningful comparisons between different regions and nations. It is also necessary for the determination of cost-driving factors to individual patients and how funds can be optimized. In addition, future 448
research and cost–benefit analyses should focus on how resource allocation impacts quality of life of patients and their families, survival and overall cost to society, to ensure financial assistance is optimized. It is also essential to continue to develop population-based ALS registries to provide the truest determination of incidence and prevalence rates necessary for accurately calculating the economic burden of the disease [39–42]. Healthcare systems, government funding of health care, third-party payer coverage and non-profit organization and government assistance vary greatly between countries. Thus, specific costs that are borne by patients and their families are largely dependent on the regional system in place and it is impossible to generalize to the ALS patient population. In our study of Canadian ALS patients [33], we emphasized the patient perspective and separated out-of-pocket costs from those covered by government or non-profit organizations. Our study identified significant out-of-pocket costs associated with home renovations and the use of private physiotherapy/occupational therapy/personal support work, of which 80 and 100%, respectively, were paid for out-of-pocket. In contrast, government and non-profit organizations covered the majority of mobility aid and medical costs. These results suggest a great need for additional financial assistance toward support of home renovations and private home care for ALS patients in Canada, but these findings may not be generalizable to other regions with different support systems in place. Additional studies, primarily focused on out-of-pocket expenses, are necessary to identify regional funding gaps and individual burden that would benefit from financial aid. Independent of how funds are allocated, the need for increased financial support for ALS patients is evident. During a period of rising disability and healthcare costs, family income concurrently plummets due to lost wages for patients and family members with caregiver responsibilities. Quality of life and financial stability are inversely correlated in ALS [43], a disease consistently associated with increased patient and caregiver stress, depression and anxiety [4,44–47]. Although the mobilization of Ice Bucket funds to provide additional assistance to ALS families may only reduce some of the financial stress, it can have a major impact on improving quality of life. Five-year review
ALS presents a substantial economic burden to both patients and society that is greater than that seen in other neurologic diseases such as dementia, Parkinson’s disease and stroke. Costs associated with ALS were high across all countries, illustrating the devastating nature of the disease and indicating a continued need for medical advances and financial support for those affected. Future research should focus on identifying gaps of financial support for individual patients across different regional healthcare systems, such that aid for those in need may be maximized in the coming years. Ultimately, the discovery of a disease-modifying agent that significantly slows or halts ALS progression will have the greatest impact toward improving quality of life and reducing the financial burden of this devastating disease. Expert Rev. Pharmacoecon. Outcomes Res. 15(3), (2015)
The economic impact of amyotrophic lateral sclerosis
Financial & competing interests disclosure
L Zinman receives research support from the Canadian Institute of Health Research, National Institute of Health, and ALS Canada. The authors have no other relevant affiliations or financial involvement with any
Review
organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. No writing assistance was utilized in the production of this manuscript.
Key issues .
Amyotrophic lateral sclerosis (ALS) is a progressive neurologic disorder with a median survival of 2 to 4 years from symptom onset.
.
ALS is associated with a significant economic burden as it results in progressive impairment in activities of daily living, including walking, eating and speaking.
.
The direct and indirect costs associated with ALS are both substantial.
.
ALS has a significant economic impact on patients, families and societies across the world, with the greatest total cost per patient being
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measured in the USA. .
Total cost per ALS patient is greater than other neurologic diseases, including dementia, Parkinson’s disease and stroke.
.
Major contributors to direct healthcare costs include medications, hospitalization, outpatient care, medical aids and equipment.
.
Direct non-healthcare costs can be quite substantial, with the most important cost being home modifications.
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Indirect costs are substantial and driven by income loss of patients and voluntary informal caregivers (usually, family members)
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Future research should focus on out-of-pocket expenses – which will vary between healthcare systems – to meaningfully determine cost-driving factors to individuals and inform where financial aid would be best directed.
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Expert Rev. Pharmacoecon. Outcomes Res. 15(3), (2015)
ISSN: 1473-7167 (Print) 1744-8379 (Online) Journal homepage: http://www.tandfonline.com/loi/ierp20
The economic impact of amyotrophic lateral sclerosis: a systematic review Matthew Gladman & Lorne Zinman To cite this article: Matthew Gladman & Lorne Zinman (2015) The economic impact of amyotrophic lateral sclerosis: a systematic review, Expert Review of Pharmacoeconomics & Outcomes Research, 15:3, 439-450 To link to this article: http://dx.doi.org/10.1586/14737167.2015.1039941
Published online: 30 Apr 2015.
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The economic impact of amyotrophic lateral sclerosis: a systematic review Downloaded by [Deakin University Library] at 06:17 20 October 2015
Expert Rev. Pharmacoecon. Outcomes Res. 15(3), 439–450 (2015)
Matthew Gladman1 and Lorne Zinman*2 1 Department of Medicine, Sunnybrook Health Sciences Centre, Queen’s University, Ontario, Canada 2 Department of Medicine, University of Toronto, Ontario, Canada *Author for correspondence: [email protected]
Amyotrophic lateral sclerosis (ALS) is a devastating neurological disease for which there is no cure, and the associated economic burden is considerable. In this review, the authors summarize the existing body of literature pertaining to the costs associated with ALS to demonstrate the scale and scope of the economic burden of this paralyzing disease. Twelve studies from eight countries published between January 2001 and January 2015 met the inclusion criteria and were included in this review. Direct and indirect costs varied significantly across countries. Standardized to the 2015 US$, the annual total cost per patient ranged from US$ 13,667 in Denmark to as high as US$ 69,475 in the USA, with the national economic burden of ALS estimated at US$ 279–472 million in the USA. Costs associated with ALS were greater than that of other neurological diseases, indicating a continued need for medical advances and financial support for patients and families. Regional cost analyses are necessary to determine how best to spend funds that have been raised globally from the ice bucket phenomenon. KEYWORDS: ALS . Amyotrophic lateral sclerosis . burden of illness . costs . economics . healthcare . ice bucket .
motor neuron disease
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review
Amyotrophic lateral sclerosis (ALS) is a devastating neurologic disorder characterized by the progressive loss of voluntary motor activity, muscle atrophy and ultimately death. The average age at disease onset is in the sixth or seventh decade of life and median survival is 2–4 years from symptom onset [1,2], with the majority of patients succumbing to respiratory failure. ALS results in complete paralysis in most cases, resulting in progressive impairment in activities of daily living, including walking, eating and speaking. As a result, there is a significant negative impact on the quality of life of both patients and their families [3–5]. Currently, there is no cure and the sole drug proven to mildly prolong survival is riluzole [6–8]. Otherwise, the only interventions that have been shown to prolong survival in ALS are the use of bi-level positive airway pressure ventilation [9–14], and the implementation of specialized multidisciplinary ALS clinics [15,16]. Invasive tracheostomy and long-term mechanical ventilation can prolong survival for patients who develop respiratory failure; however, only a minority of patients choose this option [17–19] given the considerable cost, need informahealthcare.com
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for 24-h support, substantial caregiver burden, rate of complications and eventual progression to a completely paralyzed ‘locked-in state’. In the absence of a cure, the primary focus of ALS disease management is on symptomatic treatment and supportive care through multidisciplinary ALS clinics, non-profit ALS Societies, public health resources and private insurance [20]. In addition to the progressive functional disability experienced by patients with ALS, the economic burden on patients, families and society is considerable. Expenses associated with the disease can be divided into direct and indirect costs. Direct costs are those expenses requiring payment, whether by the patient, government or a third-party payer (i.e., health insurance and non-profit-organizations), such as those associated with formal health care, medications, equipment, home or vehicle adaptations, medical devices, mobility aids and services. Direct costs can be further divided into healthcare costs and non-healthcare costs. Indirect costs represent opportunity loss, most notably lost income due to a patient’s illnessrelated absence from work or the voluntary
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caregiver duties taken on by family or friends (otherwise known as ‘informal care’). Both direct and indirect costs can overwhelm the financial resources of patients and their families. The economic burden of any disease can be considered from either the societal perspective, in which no distinction is made between costs to the patient, government or medical insurance companies, or the patient perspective, in which only out-ofpocket costs borne by the patient are considered. Both perspectives are important to consider when evaluating the overall economic burden of ALS. In July 2014, the ‘Ice Bucket Challenge’ emerged as a social media phenomenon in the USA, which involved filming yourself having a bucket of iced water dumped overhead to increase ALS awareness and funding. The challenge spread over social media to become a global activity undertaken by millions of people across over 150 countries and amassing over one billion YouTube views in a few short months. During the peak month of August 2014 alone, the American ALS Association received over US$ 100 million (compared with US$ 2.8 million in August 2013), with millions more donated to ALS Societies around the world. While the majority of funds may be allocated to ALS research, a significant amount will likely be allotted to patient care. In this systematic review, we summarize the existing body of literature pertaining to the costs associated with ALS to demonstrate the scale and scope of the economic burden of this paralyzing disease. This will help to identify the most costly components of the disease and can provide guidance to ALS Societies in how best to assist patients and families with the unprecedented influx of ALS funds recently raised. Methods Search strategy
A PubMed database search was conducted for relevant articles published between January 2001 and January 2015, citing ‘amyotrophic lateral sclerosis’ or ‘motor neuron(e) disease’ and ‘cost’ or ‘economics’. Bibliographies of key references were checked to identify any other relevant articles. Study selection
Two researchers reviewed the titles and abstracts to determine eligibility for full review. Texts were screened for eligibility if there was ambiguity of relevance in the title or abstract of any studies or if there was no abstract available. The following criteria were used to determine whether studies were included for full-text review: . . . . .
study was conducted for ALS; study assessed the direct and/or indirect costs associated with ALS (from either a societal or patient perspective); study was a primary research article based on human subjects; study was published between January 2001 and January 2015; study was published in the English language.
440
Data abstraction
For each publication included in this review, information was collected for the country of investigation, study perspective, study type and methodology, patient characteristics and costs (total, direct healthcare, direct non-healthcare and indirect, when available). Costs reported by each study were standardized to US dollars and inflated to 2015 dollars using the Organization for Economic Cooperation and Development gross domestic purchasing power parities (PPP) conversion rates [21]. Studies reporting monthly costs were extrapolated to per annum costs for ease of comparison. All costs are reported as per person per year (unless otherwise noted). Costs are rounded to the nearest dollar. Results
Twelve relevant studies were identified in the literature that met inclusion criteria. The characteristics, methodology and results reported in the review studied are described here and in TABLE 1. In the Netherlands, Van der Steen et al. [22] conducted a 2009 prospective, longitudinal observational study in 98 ALS patients who maintained a cost-related diary at regular intervals. The study aimed to contrast the direct and indirect costs of ALS patients receiving specialized multidisciplinary care compared with those receiving general care, as well as to identify clinical drivers of cost. The mean monthly cost per patient was e1313, which included all direct and indirect costs, and did not differ significantly in patients receiving multidisciplinary and general care. Total cost increased with disease severity, and, in particular, total direct non-healthcare costs (devices, aids, home adaptations, etc.) was significantly driven by disease severity: e11,669, e4750 and e3855 for patients with severe, moderate-to-severe and mild-to-moderate ALS, respectively. It is important to note that none of the patients in this study underwent invasive or non-invasive ventilation. The study concluded that while specialized multidisciplinary care was associated with improved quality of life and survival in other studies [15,16,23], it was not associated with a higher economic burden on patients or society. In Spain, Lopez-Bastida et al. [24] conducted a 2009 retrospective cost-of-illness study in 63 patients who were surveyed via questionnaire and divided into a high and low severity group, defined based on need for caregiver assistance. Direct healthcare costs, direct non-healthcare costs (primarily, informal care) and indirect costs were estimated based on the data gathered using a variety of public economic resources. The study reported a mean per patient per year (PPPY) total cost e36,194, of which e8289 was attributable to direct healthcare costs, e19,330 to direct non-healthcare costs and e8575 to indirect costs. Patients in the high severity group incurred a total PPPY cost that was more than twice that of low severity patients, at e42,728 and e17,000, respectively. The majority of the expenses can be attributed to a substantial discrepancy in informal care costs, as would be expected given how the two groups were defined. The most expensive categories of direct Expert Rev. Pharmacoecon. Outcomes Res. 15(3), (2015)
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n: 98 patients Mean age: 57.0 years Gender (m/f): 66/32 Mean disease duration from onset: NR (all patients £3 years at time of inclusion) Distribution of severity: 36.7% mild to moderate (ALSFRS 30–40), 42.9% moderate to severe (ALSFRS 20–30), 20.4% severe (ALSFRS 0–20) Distribution of region onset: 80.4% limb onset, 19.6% bulbar onset
Netherlands
Spain
Van der Steen et al. (2009)
Lopez-Bastida et al. (2009)
Retrospective cost-of-illness study using data collected via questionnaire, including both healthcare and non-healthcare resource use in the preceding 6 months, and information pertaining to indirect costs. Direct health care costs were estimated using various public resources. Direct non-healthcare costs included informal care, which along with indirect costs, was estimated using the human
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– Mild/moderate = e3855 [US$5317] – Moderate/severe = e4750 [US$6552] – Severe = e11,669 [US$16,095]
.
– Medications = e4696 [US$7657] – Orthopedic devices = e1767 [US$2881] – Other (hospital admissions, ER visits, clinic visits, medical tests/ exams, home care, transport, rehab) = e1826 [US$2978] High severity vs low severity patients = e8892 vs 6517 [US$14,499 vs 10,623]
– High severity patients = e42,728 [US$69,673] – Low severity patients = e17,000 [US$27,720] Direct healthcare cost . Mean direct healthcare cost = e8289 (US$13,516)
Total (indirect and direct) cost . Mean total cost = e36,194 [US$59,018]
.
– No significance comparing multidisciplinary vs general care or bulbar vs spinal-onset Significantly increased cost with disease severity
– Medical specialist = e984 vs 1056 [US$1357 vs 1457] – Paramedical treatment = e1776 vs 2472 [US$2450 vs 3410] – Medication = e4176 vs 4176 [US$5760 vs 5760] – Hospitalization = e1032 vs 1092 [US$1423 vs 1506] – Household help = e780 vs 972 [US$1076 vs 1341] Direct non-healthcare cost . Mean global (not annual) cost of devices/aids and home adaptations per patient = e5833 [US$8045]
– Mild/moderate = e13,188 [US$18,190] – Moderate/Severe = e16,488 [US$22,741] – Severe = e24,444 [US$33,715] Mean healthcare costs (general care vs multidisciplinary care):
.
Total (direct and indirect) cost . Mean total cost = e15,756 [US$21,732]
Prospective, longitudinal observational study using data from cost-related diary entries of patients over a 6 months period. Based on diary entries and interval questionnaires, direct costs were calculated as per the Dutch guidelines for cost analysis in healthcare research. Indirect costs were estimated using the Health and Labour Questionnaire. Currency–year: 2003
– No significance comparing multidisciplinary vs general care or bulbar vs spinal-onset Trend towards increased total cost with disease severity
Costs reported, per patient per year (PPPY; unless otherwise noted; cost in US$2015)
Study methodology
[24]
[22]
Ref.
ALSFRS-R: ALS functional rating scale (revised); ALSSS: ALS severity scale; NR: Not reported; OOP: Out-of-pocket; OT: Occupational therapy; PPPY: Per person per year; PSW: Personal support worker; PT: Physiotherapy.
n: 63 patients Mean age: 59.1 years Gender (m/f): 33/30 Mean disease duration from diagnosis: 3.7 years Distribution of employment: 27.0% retired, 17.5% housework, 6.3% temporary disability, 42.9% permanent disability, 1.6% active, 4.8% unknown Distribution of severity: 74.6% high severity (need
Study population
Country
Study (year)
Table 1. Characteristics and summary of ALS economics studies.
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n: 46 patients Mean age: NS (17.4% 31–50 years, 67.4% 51–70 years, 15.2% >70 years) Gender (m/f): 26/20 Mean disease duration from onset: 28.9% severe (ALSSS £ 23), 33.3% moderate to severe (ALSSS 24–31), 37.8% mild to moderate (ALSSS 32–40)
Multicenter cost-of-illness study using data obtained both retrospectively via questionnaire for the preceding year and prospectively via diary for a 4-month period. Direct costs were estimated using patientreported co-payments and costs paid by health insurance, estimated using various public resources. Indirect costs consisted of both productivity
Population-based cohort study of all ALS patients diagnosed in Denmark between 1998 and 2009, based on the National Patient Registry (NPR). Direct and indirect cost data was collected utilizing the NPR and various government databases, both retrospectively and prospectively. The same data was gathered for patients’ partners. Randomly selected controls (4:1 ratio), which were age, sex, and location-matched, were used via a Denmark statistics database. Currency year: 2009
capital approach to productivity losses. Currency year: 2004
caregiver assistance), 25.4% low severity
n: 2394 patients Mean age: NR (11.3% 70 years: e11,300 [US$14,627]
Total (direct and indirect) cost . Mean total cost = e$36,380 [US$47,092] . Significant drivers of total cost:
– Outpatient treatment = e955 [US$915] – Inpatient treatment = e9595 [US$9191] – Medication = e2308 [US$2211] – Health insurance = e1409 [US$1350] Indirect cost . Income loss (Patients, pre vs post-diagnosis) = e1956 vs e4651 [US$1874 vs 4455] . Income gain (partners, pre vs post-diagnosis) = e380 vs e3195 [US$364 vs 3060] Cost trends . Annual total health cost significantly increased at time of diagnosis compared to pre-diagnosis, but gradually decreased thereafter. . After diagnosis, employment rate decreased, income from employment decreased, and welfare income increased over time compared to prediagnosis and compared with controls. The opposite was seen in patients’ partners
Direct health cost . Mean direct health cost (pre vs post-diagnosis) = e2508 vs 14,268 [vs control post, e2965; US$2425 vs 13,667 vs 2840]
– 89% early retirement, 9% sick leave, 2% reduced work hours – No association with disease severity
– High severity patients = e25,837 [US$42,130] – Low severity patients = e218 [US$355] . note: 99% of this category is informal care Indirect cost . Mean lost productivity = e8575 [US$13,983]
Direct non-healthcare cost . Mean direct nonhealthcare cost = e19,330 [US$31,520]
Costs reported, per patient per year (PPPY; unless otherwise noted; cost in US$2015)
[26]
[25]
Ref.
ALSFRS-R: ALS functional rating scale (revised); ALSSS: ALS severity scale; NR: Not reported; OOP: Out-of-pocket; OT: Occupational therapy; PPPY: Per person per year; PSW: Personal support worker; PT: Physiotherapy.
Germany
Denmark
Jennum et al. (2012)
Schepelmann et al. (2010)
Country
Study (year)
Table 1. Characteristics and summary of ALS economics studies (cont.).
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Germany
USA
Wasner et al. (2001)
Larkindale et al. (2014)
n: 1528 patients for direct medical cost calculations (945 with commercial plan, 583 with Medicare) n: 124 patients for direct non-medical and indirect cost calculations
n: 171 patients Mean age: 58 years Gender (m/f): 97/72 Mean disease duration: 4.9 years Distribution of region onset: 68% limb-onset, 32% bulbar-onset
Study population
National, retrospective cost-ofillness study in which direct medical costs were derived using commercial and Medicare claims data. Non-medical costs and indirect costs (including potential earning lost from both patients and caregivers) were determined using data from a mailed costof-illness survey. The overall cost to society was estimated based on the total PPPY cost and the estimated 2010 US prevalence of ALS. Currency-year: 2010
Cross-sectional study using a mailed questionnaire to survey patients’ use and the associated costs of complementary and alternative medicine. Currency-year: NR
loss and informal care, and were estimated as per the human capital approach. Currency-year: 2009
Study methodology
54% of patients reported using complementary or alternative medicine Mean global cost per user: e4142 [US$5824]
– – – – –
Inpatient care (acute) = US$9955 [US$10,859] Inpatient care (non-acute or long term) = US$1875 [US$2045] Outpatient care = US$14,957 [US$16,315] Durable medical equipment = US$3087 [US$3367] prescription medication = US$2498 [US$2725]
– Wheelchair use = US$57,903 vs 9932 [US$63,159 vs 10,834] – Ventilator use = invasive: US$84,789 vs NIPPV: US$50,478 vs none: US$24,102 [US$92,486 vs 55,060 vs 26,290] Direct medical cost . Mean direct medical cost = US$31,121 [US$33,946]
– based on prevalence of 1.3–2.2 per 100,000 Total (direct and indirect) cost . Mean total cost = US$63,693 [US$69,475] . Significant drivers of total cost:
Total national cost . US$256–433 million [US$279–472 million]
.
.
– Informal care = e13,510 [US$17,488] – Premature retirement = e7510 [US$9721] – Temporary disability = e380 [US$492]
– Hospitalization = e2990 [US$3870] – Rehabilitation = e320 [US$414] – Outpatient care = e990 [US$1282] – Ancillary therapy = e1450 [US$1877] – Medications = e5370 [US$6951] – Specialized equipment = e1110 [US$1437] – Formal care = e1560 [US$2019] Indirect cost . Mean annual family income loss = e21,400 [US$27,701]
Direct cost . Mean direct cost = e14,980 [US$19,391]
Costs reported, per patient per year (PPPY; unless otherwise noted; cost in US$2015)
[28]
[27]
Ref.
ALSFRS-R: ALS functional rating scale (revised); ALSSS: ALS severity scale; NR: Not reported; OOP: Out-of-pocket; OT: Occupational therapy; PPPY: Per person per year; PSW: Personal support worker; PT: Physiotherapy.
Country
Study (year)
Table 1. Characteristics and summary of ALS economics studies (cont.).
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USA
Dubinsky et al. (2006)
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n: 33 patients Mean age: 61.6 years Gender (m/f): 15/18
n: 17,249 Mean age: 65.2 years Gender (m/f): 9263/7986
n: 25 patients (vs 159 lung cancer patients) Gender (m/f): 12/13
Study population
Retrospective cost-of-illness study utilizing a chart review to determine health resource use, and interviews to collect work absenteeism and informal care data used to estimate indirect cost via national minimum wage. Direct costs were determined using the National Health System price lists. Currency-year: 2013
Retrospective cohort comparison of all ALS hospital admissions in USA between 1988 and 2002, using data from the nationwide inpatient sample. Cost (inflationadjusted), reason for admission, length of stay, discharge disposition, and hospice care use were trended over time. Currency year: 2002
Prospective cohort study using patient and financial records to compare the costs associated with hospice care for ALS patients vs lung cancer patients. Currency-year: 2003
Study methodology
– 1988 = US$21,574 [US$28,003] – 2002 = US$24,314 [US$31,559] – Significant increase despite median length of stay decreasing from 6 days to 4 days
Mean hospital-related cost per patient per admission
– Staff services = US$3320 [US$4225] – Medical equipment = US$1252 [US$1593] – Medications = US$866 [US$1102] Mean global cost per lung cancer patient = US$2659 [US$3384] (mean length of stay, 35 days)
Mean global cost per ALS patient = US$5623 [US$7156] (mean length of stay, 87 days)
– Lost productivity = e1321 [US$1995] – Informal care = e1825 [2756]
– Medications = e2437 [US$3680] – Hospitalizations = e686 [US$1036] – Laboratory work = e515 [US$778] Indirect cost . Mean indirect cost = e3145 [US$4750]
Total (direct and indirect) cost . Mean total cost = e7450 [US$11,251] Direct cost . Mean direct cost = e4305 [US$6501]
.
.
.
– Home modifications or moving = US$7106 [US$7751] – Car modifications of purchase = US$2064 [US$2251] – Professional caregiving = US$4570 [US$4985] – Other (i.e., food supplements, travel, etc.) = US$5908 [US$6444] Indirect cost . Mean annual family income loss = US$14,682 [US$16,015] . Significantly increased indirect cost with greater level of care required
No significant difference in cost comparing patients with Medicare or commercial plans Direct non-medical cost . Mean direct non-medical cost = US$17,889 [US$19,513]
.
Costs reported, per patient per year (PPPY; unless otherwise noted; cost in US$2015)
[31]
[30]
[29]
Ref.
ALSFRS-R: ALS functional rating scale (revised); ALSSS: ALS severity scale; NR: Not reported; OOP: Out-of-pocket; OT: Occupational therapy; PPPY: Per person per year; PSW: Personal support worker; PT: Physiotherapy.
Greece
USA
Elman et al. (2006)
Athanasakis et al. (2014)
Country
Study (year)
Table 1. Characteristics and summary of ALS economics studies (cont.).
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Ireland
Canada
Connolly et al. (2014)
Gladman et al. (2014)
n: 49 patients Mean age: 62.6 years Gender (m/f): 23/26 Mean disease duration from diagnosis: 2.8 years Distribution of employment: 61.2% lost job due to ALS, 38.8% retired at diagnosis Distribution of severity: 28.9% moderate (ALSFRS-R 31–38), 46.7% severe (ALSFRS-R 21–30), 24.4% profound (ALSFRS-R £ 20) Distribution of region onset: 89.8% limb-onset, 10.2% bulbar-onset
n: 119 patients Mean age: NS (7.6% 4 hr/week = CDN$20,718 vs 17,768 [US$17,126 vs 14,687] Indirect Cost . Mean annual income loss when patient lost job due to ALS = CDN$ 56,821 [US$46,968]
.
– Renovation costs = CDN$10058 [US$8314] – Mobility costs = CDN$3749 [US$3099] – Medical costs = CDN$2848 [US$2354] – Private PT/OT/PSW = CDN$2920 [US$2414] Significant drivers of OOP direct cost:
Direct cost . Mean OOP direct cost = CDN$19,574 [US$16180]
.
.
.
Direct cost . Median direct cost = e21,540 [US$25,949]
Retrospective cost-of-illness study using hospital-based health resource data from a chart review, as well as community health resource data from a structured questionnaire with a subset of patients (n = 17). Associated costs were estimated using a variety of published resources. Only services paid for by the health service were included, thus excluding out-of-pocket costs, informal care, and productivity losses. Currency year: 2011 – Multidisciplinary clinic = e4440 [US$5349] – Community based care = e15,516 [US$18,692] – Aids and appliances = e1584 [US$1908] Significant drivers of direct cost:
Costs reported, per patient per year (PPPY; unless otherwise noted; cost in US$2015)
Study methodology
[33]
[32]
Ref.
ALSFRS-R: ALS functional rating scale (revised); ALSSS: ALS severity scale; NR: Not reported; OOP: Out-of-pocket; OT: Occupational therapy; PPPY: Per person per year; PSW: Personal support worker; PT: Physiotherapy.
Country
Study (year)
Table 1. Characteristics and summary of ALS economics studies (cont.).
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medical costs were medications and orthopedic devices, which accounted for a mean PPPY cost of e4696 and e1767, respectively. Indirect cost was primarily due to early retirement (89%), whereas sick leaves and reduced work hours only accounted for 9 and 2%, respectively. The study concluded that total expenses increases with disease severity, and the economic and social costs of ALS require increased attention by health authorities and society. In Denmark, Jennum et al. [25] conducted a populationbased cohort study of all ALS patients diagnosed between 1998 and 2009, based on the National Patient Registry. A total of 2394 patients and their partners were matched to controls, and cost data were collected both retrospectively and prospectively. The study reported a mean PPPY direct cost of e14,268, almost sixfold greater than cost prediagnosis and that of the control group. As expected after diagnosis, employment rate and total income from employment decreased, and welfare income increased over time, with PPPY indirect cost being e4651. Interestingly, the opposite trend was seen in patients’ partners. The study concluded that ALS leads to higher healthrelated and social transfer (welfare) costs, lower levels of employment and income, and a seemingly increased workload and economic burden taken on by the spouse to compensate for the increased expenses. In Germany, Schepelmann et al. [26] conducted a 2010 multicenter, retrospective and prospective cost-of-illness study of 46 patients. Data on both direct and indirect costs were obtained using a questionnaire pertaining to the preceding year and 4 months prospective via cost diaries. The reported total PPPY cost was e36,380, of which e14,980 was attributable to direct cost and e21,400 to indirect cost. Several significant cost-driving factors were identified, including disease severity (as per ALS functional scores), artificial ventilation, artificial nutrition, dependency in activities of daily living, presence of dementia, and interestingly, age correlated inversely with total cost. The greatest direct cost was that of medications at e5370 PPPY. Other important direct costs were hospitalization (20%), ancillary therapy (10%), formal care (10%), special equipment (7%), rehabilitation (7%) and outpatient care (2%). Indirect cost consisted primarily of informal care and premature retirement, at e13,510 and e7510 PPPY, respectively. The study concluded that the socioeconomic burden of ALS in Germany has a considerable impact on patients and their families, as the costs of lost productivity is greater than the direct costs paid by health insurance. Another German study conducted by Wasner et al. [27] in 2001 surveyed patients’ use and the associated costs of complementary and alternative medicine (CAM) via questionnaire. They found that 54% of the 171 patients surveyed reported CAM use, at an average lifetime cost of e4142, the majority of which was out-of-pocket. They concluded that patient interest in CAM is significant and future research should explore the use of CAM for management of ALS symptoms. Among the studies conducted in the USA, in 2014 Larkindale et al. [28] used retrospective commercial and 446
Medicare claims data to calculate direct costs and a mail-based questionnaire to estimate direct non-medical and indirect costs using data from 124 respondents. The study reported a total PPPY cost of US$ 63,693, with ventilator use (both invasive and non-invasive ventilation) and wheelchair use being significant drivers of cost. Annual direct medical cost amounted to US$ 31,121 per patient. Outpatient care and acute inpatient care comprised the majority of direct medical costs (48 and 32%, respectively), while non-acute inpatient care, medical equipment and medications accounted for the other 20%. Direct non-medical costs, including home and vehicle modifications and professional caregiving, amounted to US$ 17,889 PPPY. In this study, indirect cost was less than half that of direct cost, estimated at US$ 14,682 PPPY, which included the productivity loss of both the patient and their informal caregivers. Overall, based on a US prevalence estimate of 1.3–2.2 per 100,000, the estimated total national cost of ALS to society was US$ 256–433 million per year. The study concluded that the economic impact of ALS and other neuromuscular diseases is substantial, both for society and affected households, which suggests a possible role for additional policy initiatives to better help affected individuals and their families. In 2006, Elman et al. [29] conducted a prospective cohort study out of Pennsylvania using patient and financial records to compare the costs associated with hospice care for patients with ALS and lung cancer. The mean total cost of hospice care per ALS patient was US$ 5623, more than twice that of lung cancer patients although ALS patients’ mean length of stay was nearly threefold longer at 87 days. The majority of costs were attributable to staff services (59%), medical equipment (22%) and medications (15%). The study concluded that end-of-life care in ALS is costly, and significantly more so than palliative lung cancer patients. Dubinsky et al. [30] conducted a retrospective cohort comparison of all ALS hospital admissions in USA between 1988 and 2002 and found a significant increase in cost per admission from US$ 21,574 in 1988 to US$ 24,314 in 2002, despite decreasing length of stay. In Greece, Athanasakis et al. [31] conducted a 2014 retrospective cost-of-illness study of 33 patients using chart review data to estimate direct costs and structured interviews to evaluate indirect cost. The study reported a mean PPPY direct cost of e4305, with the main factors being medications, hospitalizations and laboratory work at e2437, 686 and 515, respectively. Annual indirect cost was estimated at e3145 per patient, 58% of which was due to loss of income from care provided by family and friends while 42% was patient lost income. The study found that the total annual cost of e7450 was significantly lower than that reported by studies conducted in other countries and concluded that it was attributable to lower medication prices, lower healthcare tariffs and very low minimum wage. In Ireland, Connolly et al. [32] conducted a 2014 retrospective cost-of-illness study using health resource data collected from a chart review of 119 ALS patients. In addition, a subset of 17 patients was further surveyed via a structured questionnaire to determine health resource use in the community, as well as Expert Rev. Pharmacoecon. Outcomes Res. 15(3), (2015)
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Annual cost per person reported (standardized to US$-2015)
US$80.000 use of aids and appliances. Only services paid for by the health service were US$70.000 included, and the study did not deterTotal cost mine out-of-pocket expenses or indirect Direct cost US$60.000 costs. The study reported a median Indirect cost monthly cost per patient of e1795, of US$50.000 which 1293 was attributable to US$40.000 community-based care, 370 to costs associated with the multidisciplinary clinic US$30.000 (including medications), and 132 to aids and appliances. The most significant US$20.000 cost-driving factors were the use of noninvasive ventilation (e513 vs 327), gasUS$10.000 trostomy (e803 vs 378), and they found US$0 an inverse association between monthly costs and length of survival. The study Netherlands† Spain Germany USA Greece‡ Ireland Canada§ concluded that the costs of communityFigure 1. Total, direct and indirect cost of amyotrophic lateral sclerosis per based services are a large contributor to person per year, stratified by country of study. the overall economic burden of ALS on † Netherlands study did not report direct or indirect cost. ‡ health services in Ireland, and the overall Ireland study only reported direct cost. § costs for Ireland seem to be higher than Canada study only reported direct and indirect cost; total cost could not be extrapolated given non-out-of-pocket expenses were not included. other European countries. In Canada, our group [33] conducted a retrospective cost-of-illness study in 2014 using data collected from standardized surveys that reported total (direct and indirect) cost, the standardized total probed out-of-pocket direct costs and indirect costs due to annual cost per patient was found to be US$ 69,475 in the income loss of patients and caregivers. The study reported a USA, 59,018 in Spain, 47,092 in Germany, 21,732 in the mean PPPY out-of-pocket direct cost of CDN$ 19,574, of Netherlands and 11,251 in Greece. The standardized annual which 10,058 was attributable to renovation costs, 3749 to direct cost per patient was found to be US$ 53,459 in the mobility aid costs, 2848 to medical costs, and 2920 to the cost USA, 45,036 in Spain, 19,391 in Germany, 16,180 in Canada of a private physiotherapy, occupational therapy and personal (out-of-pocket proportion), 25,949 in Ireland, 6501 in Greece support work. Several out-of-pocket direct cost-driving factors and 13,667 in Denmark. Annual indirect cost varied greatly were identified, including poor gross motor function (ALSFRS- across studies and was largely dependent on method of calculaR score £6), limb-predominant disease and increased care needs tion and how the costs were defined. Indirect costs were (formal personal support work care >4 h/week). Indirect cost reported to be the highest in Canada at US$ 46,968. The total was stratified by patient employment status. For patients who national cost burden of ALS was only calculated by one lost their job due to disability, mean annual income loss was study [28], which reported a disease-associated cost of US$ CDN$ 36,467, and family members reported an additional 279–472 million per year in the USA. loss of 20,353 due to caregiver responsibilities. Although retired patients had no opportunity loss, their family had a mean Expert commentary annual income loss of CDN$ 16,177. We concluded that out- ALS is a physically and emotionally devastating disease characof-pocket expenses incurred by ALS patients and their families terized by a rapid decline in function and independence and is are substantial, illustrating the need to mobilize additional associated with a considerable increase in care needs over a short period of time. This systematic review verifies that the resources for families affected by ALS. economic burden of ALS is significant, both to society and affected families. In the USA, the 2015 standardized societal Standardized costs To meaningfully interpret the costs reported by the studies cost of ALS was found to be US$ 279–472 million annually. reviewed, all expenses were converted and inflated to a standard Total PPPY expenses varied across countries, but were sizable US$ 2015 currency year, as reported in TABLE 1. An illustration in all reviewed studies, ranging from US$ 11,251 in Greece to of the comparative total, direct and indirect PPPY costs strati- as high as 69,475 in USA (standardized to 2015 US$). Not fied by country of study is shown in FIGURE 1. However, cost surprisingly, the per capita cost associated with ALS is greater comparisons between studies are limited by heterogeneity in than that of any other neurological disease. By comparison, the healthcare systems, payer coverage, study designs, cost defini- annual total cost per person in the USA for dementia, tions, inclusiveness of cost data and study perspectives (societal, Parkinson’s disease, and first-time stroke (weighted by proporpatient-based and/or third-party payer). Of those studies that tion of patients with different stroke types) is estimated at
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US$ 61,400 [34], 22,851 [35] and 10,882 [36], respectively (adjusted to 2015 US$). This disparity is likely, in part, attributable to ongoing interventions in ALS such as artificial ventilation and nutrition, which were significant cost-drivers in multiple studies, as well as profound functional disability, which is invariably accompanied by increased care needs throughout the course of disease, eventual inability to work, durable medical equipment (i.e., wheelchairs) and often home renovations. Like ALS, Parkinson’s disease and dementia are neurodegenerative diseases in which medical and assistive needs invariably increase throughout the course of disease. While annual costs per person are greater in ALS, survival is significantly shorter and thus cumulative costs may be greater in other neurodegenerative diseases. It is important to note that, however, there is a cohort of ALS patients in which survivorship is prolonged [37,38], which would be accompanied by a substantial cumulative cost. We found that across all countries reviewed, ALS is associated with both significant direct and indirect costs. Major contributors to direct healthcare cost included medications, hospitalization, outpatient care, aids and medical equipment. Direct non-healthcare-related expenses were only surveyed in a few studies, but associated costs were consistently large with the most significant contributor being home modifications, which in itself ranged as high as US$ 8314 PPPY in Canada. Indirect cost was driven by productivity loss of patients, in addition to that of voluntary caregivers in some of the reviewed studies. Although indirect costs were substantial, they were likely still underestimated as studies did not consider premature mortality in determining income loss for patients. Direct costs were higher than indirect costs for four of the six studies where they were both measured. This systematic review is timely given the unprecedented influx of tens of millions of dollars in donations to regional ALS associations globally from the ‘Ice Bucket Challenge’. While the majority of funds may be allocated to research, a substantial portion will be earmarked for patient care and each ALS societies will be tasked in determining how these funds are best spent. Identification of the cost-driving factors that have the most significant personal impact to patients and families will help to optimize how funds are spent. This review illustrates the substantial cost associated with ALS. However, most studies focused on a societal rather than patient perspective, such that no distinction is made between costs to the patient, government or medical insurance companies, and thus it is difficult to ascertain where the greatest financial gaps are to individual patients. Determination of out-ofpocket expenses to the individual patient and family likely serves as the truest metric representing the financial toll of the disease. Out-of-pocket expenses would control for the variable contribution made by public healthcare services, non-profit agencies and insurance companies and allow for more meaningful comparisons between different regions and nations. It is also necessary for the determination of cost-driving factors to individual patients and how funds can be optimized. In addition, future 448
research and cost–benefit analyses should focus on how resource allocation impacts quality of life of patients and their families, survival and overall cost to society, to ensure financial assistance is optimized. It is also essential to continue to develop population-based ALS registries to provide the truest determination of incidence and prevalence rates necessary for accurately calculating the economic burden of the disease [39–42]. Healthcare systems, government funding of health care, third-party payer coverage and non-profit organization and government assistance vary greatly between countries. Thus, specific costs that are borne by patients and their families are largely dependent on the regional system in place and it is impossible to generalize to the ALS patient population. In our study of Canadian ALS patients [33], we emphasized the patient perspective and separated out-of-pocket costs from those covered by government or non-profit organizations. Our study identified significant out-of-pocket costs associated with home renovations and the use of private physiotherapy/occupational therapy/personal support work, of which 80 and 100%, respectively, were paid for out-of-pocket. In contrast, government and non-profit organizations covered the majority of mobility aid and medical costs. These results suggest a great need for additional financial assistance toward support of home renovations and private home care for ALS patients in Canada, but these findings may not be generalizable to other regions with different support systems in place. Additional studies, primarily focused on out-of-pocket expenses, are necessary to identify regional funding gaps and individual burden that would benefit from financial aid. Independent of how funds are allocated, the need for increased financial support for ALS patients is evident. During a period of rising disability and healthcare costs, family income concurrently plummets due to lost wages for patients and family members with caregiver responsibilities. Quality of life and financial stability are inversely correlated in ALS [43], a disease consistently associated with increased patient and caregiver stress, depression and anxiety [4,44–47]. Although the mobilization of Ice Bucket funds to provide additional assistance to ALS families may only reduce some of the financial stress, it can have a major impact on improving quality of life. Five-year review
ALS presents a substantial economic burden to both patients and society that is greater than that seen in other neurologic diseases such as dementia, Parkinson’s disease and stroke. Costs associated with ALS were high across all countries, illustrating the devastating nature of the disease and indicating a continued need for medical advances and financial support for those affected. Future research should focus on identifying gaps of financial support for individual patients across different regional healthcare systems, such that aid for those in need may be maximized in the coming years. Ultimately, the discovery of a disease-modifying agent that significantly slows or halts ALS progression will have the greatest impact toward improving quality of life and reducing the financial burden of this devastating disease. Expert Rev. Pharmacoecon. Outcomes Res. 15(3), (2015)
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Financial & competing interests disclosure
L Zinman receives research support from the Canadian Institute of Health Research, National Institute of Health, and ALS Canada. The authors have no other relevant affiliations or financial involvement with any
Review
organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. No writing assistance was utilized in the production of this manuscript.
Key issues .
Amyotrophic lateral sclerosis (ALS) is a progressive neurologic disorder with a median survival of 2 to 4 years from symptom onset.
.
ALS is associated with a significant economic burden as it results in progressive impairment in activities of daily living, including walking, eating and speaking.
.
The direct and indirect costs associated with ALS are both substantial.
.
ALS has a significant economic impact on patients, families and societies across the world, with the greatest total cost per patient being
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measured in the USA. .
Total cost per ALS patient is greater than other neurologic diseases, including dementia, Parkinson’s disease and stroke.
.
Major contributors to direct healthcare costs include medications, hospitalization, outpatient care, medical aids and equipment.
.
Direct non-healthcare costs can be quite substantial, with the most important cost being home modifications.
.
Indirect costs are substantial and driven by income loss of patients and voluntary informal caregivers (usually, family members)
.
Future research should focus on out-of-pocket expenses – which will vary between healthcare systems – to meaningfully determine cost-driving factors to individuals and inform where financial aid would be best directed.
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Expert Rev. Pharmacoecon. Outcomes Res. 15(3), (2015)
