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SKY-0515

Huntington's disease

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211 sources

Product information and disease descriptionPlace of product in therapyDisease descriptionBurden of Huntington's disease

Economic burden and healthcare resource utilization

28 evidence topics · 30 sources

U.S. commercial and Medicaid claims (2002 to 2009): direct medical costs by stage

“Among 1272 HD patients, the mean age was similar in commercial (752 patients) and Medicaid (520 patients) populations” (opens the source at this quote in a new tab)

“The mean total annualized cost per patient increased by stage (commercial: $4947 (SD = $6040)-$22,582 (SD = $39,028); Medicaid: $3257 (SD = $5670)-$37,495 (SD = $27,111).” (opens the source at this quote in a new tab)

“The vast majority (73.8%) of Medicaid Late stage patients received nursing home care and the majority (54.6%) of Medicaid Late stage costs were associated with nursing home care.” (opens the source at this quote in a new tab)

“Key limitations include the classification of patients into a single stage, as well as a lack of visibility into full long-term care/nursing home-related costs for commercial patients.” (opens the source at this quote in a new tab)

U.S. Medicare fee-for-service claims (2013 to 2017): costs versus matched beneficiaries and by stage

“We identified 3,688 matched pairs of beneficiaries with and without HD.” (opens the source at this quote in a new tab)

“Mean [SD] annual total healthcare costs were higher for HD beneficiaries than beneficiaries without HD ($41,631 [57,393] vs. $17,222 [31,218], p < .001) and were primarily driven by outpatient pharmacy costs ($19,182 [45,469] vs. $4,318 [11,553], p < .001).” (opens the source at this quote in a new tab)

“In the stratified analysis, total healthcare costs were highest among beneficiaries with late-stage HD (mean [SD] cost: $20,475 [$41,122] for early-stage vs. $29,733 [$44,977] for middle-stage vs. $56,657 [$64,185] for late-stage; p < .001).” (opens the source at this quote in a new tab)

U.S. Medicaid Analytic eXtract (2010 to 2014): costs versus matched beneficiaries and by stage

“A total of 1,785 beneficiaries without HD were matched to 595 beneficiaries with HD (139 early-, 78 middle-, and 378 late-stage).” (opens the source at this quote in a new tab)

“The mean (SD) annual total costs were higher for beneficiaries with HD than beneficiaries without HD ($73,087 [$75,140] vs. $26,834 [$47,659], p <.001) and driven by inpatient costs ($45,190 [$48,185] vs. $13,808 [$39,596], p <.001).” (opens the source at this quote in a new tab)

“Total healthcare costs were highest among beneficiaries with late-stage HD (mean [SD] cost: $22,797 [$31,683] for early-stage HD vs. $55,294 [$129,290] for middle-stage HD vs. $95,251 [$60,197] for late-stage HD; p <.001).” (opens the source at this quote in a new tab)

U.S. MarketScan commercial, Medicaid, and Medicare Supplemental claims (2009 to 2018): HRU and costs per patient per month by stage

“A total of 2,669 individuals with HD were identified. Of these, 1,432 (53.7%), 689 (25.8%), and 548 (20.5%) had early-, middle-, and late-stage HD at baseline, respectively.” (opens the source at this quote in a new tab)

“Mean total all-cause healthcare cost PPPM for individuals with HD was $2,889, and it was significantly higher in middle-stage individuals, at $7,988, compared with early- and late-stage individuals, at $3,726 and $5,125, respectively; p <0.0001.” (opens the source at this quote in a new tab)

“Our evaluations didn't include the indirect costs of HD, which may be substantial as HD typically affects people at their peak earning potential.” (opens the source at this quote in a new tab)

U.S. MarketScan claims: costs versus propensity-matched controls without HD

“A total of 2,473 individuals with HD and 9,522 matched non-HD controls were identified.” (opens the source at this quote in a new tab)

“Mean total costs (2018 USD PPPM) for the HD cohort ($2,260 [SD = $4,682]) were twice the total costs in the non-HD cohort ($1,056 [SD = $3,078]) (P < 0.0001) and were highest across all disease stages.” (opens the source at this quote in a new tab)

U.S. MarketScan claims: late-onset versus adult-onset HD

“In total, 763 individuals with LoHD and 1,073 individuals with AoHD were matched with 3,762 non-HD controls.” (opens the source at this quote in a new tab)

“In the 12 months post-index, mean total costs for the LoHD cohort ($29,055) were significantly higher than for non-HD controls (≥60 years old: $17,286; 21-59 years old: $12,688; p <.001) and similar to total costs in the AoHD cohort ($31,701; p =.47).” (opens the source at this quote in a new tab)

U.S. MarketScan claims (2017 to 2021): HD-related costs by diagnosing provider type

“Three hundred and forty eligible patients had a mean age at diagnosis of 49 years.” (opens the source at this quote in a new tab)

“HD-related mean costs totaled $2,489 ($1,179 inpatient and $1,310 outpatient).” (opens the source at this quote in a new tab)

“Patients diagnosed by neurologists had significantly lower HD-related total non-medication costs vs. those diagnosed by PCPs (-$2,256; p < 0.05).” (opens the source at this quote in a new tab)

Systematic review of cost-of-illness studies: annual costs by stage and cost type (2023 U.S. dollars)
Cost type and study“Early”“Middle”“Late”
Inpatient, Divino“1150”“5045”“9216”
Inpatient, Exuzides“1225”“4250”“22,228”
Inpatient, Patel“791”“3,070”“65,465”
Outpatient, Divino“2956”“10,508”“30,104”
Outpatient, Exuzides“4736”“10,361”“13,665”
Outpatient, Patel“5676”“11639”“6226”
Drug, Divino“2483”“4518”“4838”
Drug, Exuzides“15,663”“16,790”“23,944”
Drug, Patel“6330”“14149”“13,108”
Caregiving, Jones“6073”“44,662”“133,200”
Caregiving, Silva-Pardes“6041”“16,502”“21,438”
Systematic review of cost-of-illness studies: synthesis

“Only five studies were included for extraction and synthesis including three from the USA, one from the United Kingdom (UK), and one from Peru.” (opens the source at this quote in a new tab)

“Outpatient costs were approximately 2.5 times greater than inpatient costs for early and middle stages of HD.” (opens the source at this quote in a new tab)

“Among all the costs associated with HD, annual caregiver cost emerges as the most significant costs in the economic burden of HD, ranging from a minimum of $6041 for early stage to a maximum of $133,200 for late-stage HD.” (opens the source at this quote in a new tab)

Global systematic review: annual costs by region (2024 U.S. dollars)

“Out of the initial 608 de-duplicated articles, 19 full-text articles were included.” (opens the source at this quote in a new tab)

“Annual costs in 2024 USD ranged significantly by region: Americas ($2542-$90,515), Europe ($40,000-$215,020), Asia ($1915-$7132), and Oceania ($3678-$8721).” (opens the source at this quote in a new tab)

HDBOI study (France, Germany, Italy, Spain, United Kingdom, United States): direct, nondirect, and indirect costs

“HDBOI cost estimates were €12,663 (n = 2094) for direct medical costs, €2984 (n = 359) for nondirect medical costs, and €47,576 (n = 436) for indirect costs.” (opens the source at this quote in a new tab)

“Costs are higher in patients who are at later stages of disease; for example, direct medical costs estimates were €9220 (n = 846), €11,885 (n = 701), and €18,985 (n = 547) for ES, MS, and AS, respectively.” (opens the source at this quote in a new tab)

“Costs show large variations between patients and countries.” (opens the source at this quote in a new tab)

HDBOI study: design, participants, and costing methods

“The HDBOI study is a noninterventional, retrospective, cross-sectional, international burden of illness study of people with diagnosed HD in France, Germany, Italy, Spain, the UK, and the USA.” (opens the source at this quote in a new tab)

“A total of 2094 PwHD were enrolled in the HDBOI study, 40% of whom (n = 846) were classified as ES, 34% as MS (n = 701), and 26% as AS (n = 547; Table 1).” (opens the source at this quote in a new tab)

“Information regarding HD-related health care resource use used to compute direct medical costs was available for all 2094 PwHD. Information on direct nonmedical costs was available for 359 PwHD and indirect costs for 436 PwHD.” (opens the source at this quote in a new tab)

“Costs are reported in 2020 euros for all outcomes” (opens the source at this quote in a new tab)

“To minimize underestimation of costs due to potentially reduced care interactions during the coronavirus disease 2019 (COVID-19) pandemic, health care resource use (used to compute direct medical costs) was extracted for a set time period before the pandemic (March 2019 through March 2020).” (opens the source at this quote in a new tab)

“For actively employed PwHD and caregivers, productivity loss was quantified as the number of days missed from work due to HD in the past 3 months multiplied by the country average salary per day.” (opens the source at this quote in a new tab)

“For those unable to work due to HD, an opportunity cost was assigned based on 1 year of average salary per country.” (opens the source at this quote in a new tab)

HDBOI study: mean annual costs by disease stage (2020 euros)
Cost categoryTotal, mean (SD)Early stage, mean (SD)Mid stage, mean (SD)Advanced stage, mean (SD)
Direct medical costs“€12,663 (€34,012)”“€9220 (€31,855)”“€11,885 (€31,827)”“€18,985 (€38,811)”
Direct nonmedical costs“€2984 (€3627)”“€2275 (€3530)”“€2956 (€3632)”“€4180 (€3500)”
Indirect costs“€47,576 (€47,985)”“€35,859 (€44,874)”“€51,996 (€45,549)”“€56,947 (€51,723)”
Total costs“€62,372 (€51,197)”“€42,477 (€45,651)”“€64,688 (€53,698)”“€86,177 (€44,966)”
HDBOI study: cost drivers and mean annual cost components by stage (2020 euros)

“Inpatient hospitalizations and residential care were the primary drivers of direct medical costs” (opens the source at this quote in a new tab)

“Total costs (sum of direct medical, nonmedical, and indirect costs), available for a subsample of 307 PwHD (patients with a completed eCRF, PPIE-P, and PPIE-C), were driven primarily by indirect costs, which accounted for 84%, 80%, and 66% of ES, MS, and AS total costs, respectively (Appendix Table A2).” (opens the source at this quote in a new tab)

Cost componentEarly stage, mean (SD) and nMid stage, mean (SD) and nAdvanced stage, mean (SD) and n
Consultations“€727 (€1216) n = 829”“€767 (€975) n = 693”“€1051 (€1335) n = 545”
Hospital inpatient“€35,803 (€102,653) n = 28”“€30,698 (€39,581) n = 23”“€12,658 (€42,550) n = 60”
Treatment“€6333 (€25,525) n = 667”“€6539 (€28,257) n = 571”“€7922 (€26,248) n = 454”
Residential care“€41,879 (€46,946) n = 16”“€56,673 (€40,806) n = 26”“€45,977 (€31,915) n = 98”
Professional caregiver“€1286 (€4355) n = 204”“€2308 (€9831) n = 164”“€2582 (€8632) n = 114”
State support, patient“€841 (€2123) n = 204”“€1199 (€2497) n = 164”“€2677 (€3437) n = 114”
Lost productivity, PwHD“€23,216 (€16,848) n = 75”“€31,824 (€13,934) n = 109”“€34,343 (€10,295) n = 98”
HDBOI study: mean annual costs by country (2020 euros)

“Direct nonmedical costs were highest in Italy (€3977) and the UK, and indirect costs were highest in the USA (€74,228 [$90,187]), the UK, and Italy” (opens the source at this quote in a new tab)

“The HDBOI study reported direct medical costs of €27,743 in the USA and a substantial indirect cost burden of €74,228 from lost work productivity.” (opens the source at this quote in a new tab)

CountryDirect medical costsIndirect costs
France“4167”“39471”
Germany“11560”“39451”
Italy“6934”“47048”
Spain“5814”“43547”
United Kingdom“12606”“52472”
United States“27743”“74228”
HDBOI study: stated limitations of the cost estimates

“Because recruitment was driven through clinician office visits, AS patients may have been underrepresented if they were admitted to long-term residential or nursing care homes.” (opens the source at this quote in a new tab)

“Cost estimates for AS PwHD may have been underestimated due to the small sample of patients with advanced disease and subsequent subgroup analyses.” (opens the source at this quote in a new tab)

“For Germany, direct medical costs may have been underestimated, as only copayments were publicly available, which do not reflect the true cost of the service delivered.” (opens the source at this quote in a new tab)

United Kingdom EHDN REGISTRY: annual societal costs by TFC stage (2013 pounds)
Category“Stage I: TFC 11–13 (n = 43)”“Stage II: TFC 7–10 (n = 37)”“Stage III: TFC 3–6 (n = 38)”“Stage IV: TFC 1–2 (n = 13)”“All stages (n = 131)”
Informal care, mean“£1073.86”“£4941.41”“£21 051.79”“£62 784.00”“£14 085.24”
Total, mean“£2250.06”“£11 867.76”“£29 671.91”“£89 759.62”“£21 605.08”
United Kingdom EHDN REGISTRY: cost drivers

“The mean annual cost per person with HD was £21 605. The largest proportion of this cost (65%) was due to informal care (£14 085).” (opens the source at this quote in a new tab)

“This equates to £195 million per year in the UK.” (opens the source at this quote in a new tab)

Norway: six-month healthcare and societal costs

“Total six-month healthcare costs and total societal costs were € 18,538 and € 66,789 respectively.” (opens the source at this quote in a new tab)

“Main six-month cost components for the three disease phases were informal care costs (€ 30,605) accounting for approximately half the total societal costs, and costs due to production loss (€ 18,907) being slightly higher than the total healthcare costs.” (opens the source at this quote in a new tab)

Long-term care in advanced HD: North American systematic literature review

“Most patients with advanced HD receive LTC in a nursing home (35%) or at home (32%), with fewer patients receiving LTC from hospitals, hospices, and multi-setting facilities” (opens the source at this quote in a new tab)

“A long period of time is spent in both nursing home and hospice care, leading to sizeable annual costs for this care provision.” (opens the source at this quote in a new tab)

Hospice and palliative care utilization in the United States

“Of the 164,032 patients admitted to these hospices, 101 (0.06%) had a primary diagnosis of HD.” (opens the source at this quote in a new tab)

“Patients had a median length of stay in hospice of 42 days, which was significantly longer than that of other hospice patients in the sample (17 days), P < 0.001.” (opens the source at this quote in a new tab)

“8521 patients with HD were admitted to the hospital. Of those, 321 (3.8%) received specialty PC.” (opens the source at this quote in a new tab)

Predictors of health care utilization: Enroll-HD Northern America

“Visits from 1,631 participants in the Northern America region from the Enroll-HD study were included in this study.” (opens the source at this quote in a new tab)

“Clinical factors, such as psychiatric symptoms, functional ability, and comorbidities, were associated with use of multiple types of health care services.” (opens the source at this quote in a new tab)

Employment loss by disease stage: Enroll-HD

“At baseline visit, 47.5% of patients with TFC score 11–13 (n=2,512) were unemployed; 19.9% of them due to illness and 15.4% were on sick leave.” (opens the source at this quote in a new tab)

“89.8% of patients with TFC score 7–10 (n=2,794) were unemployed; 37.8% of them due to illness and 19.7% were on sick leave.” (opens the source at this quote in a new tab)

“By 4 years post-clinical diagnosis, <20% patients stayed employed.” (opens the source at this quote in a new tab)

Employment and work productivity: HDBOI participants

“Approximately 27% of the PPIE-P sample reported stopping work early (i.e., retiring) due to HD.” (opens the source at this quote in a new tab)

“Of those who reported being employed for pay overall work productivity loss increased with disease severity: 43% in ES, 55% in MS, and 57% in AS.” (opens the source at this quote in a new tab)

Employment: chorea and cognitive decline

“Patients with chorea were less likely to be employed full-time (16.7% vs 25.7%; P<0.04) and more likely to be on long-term sick leave (17.4% vs 5.0%; P<0.01).” (opens the source at this quote in a new tab)

“For every year delay in cognitive symptom onset, there was a 0.806 year increase in age at leaving work (SE = 0.030, p < 0.001, adj-R2 = 0.628).” (opens the source at this quote in a new tab)

“Twenty-three studies were identified and showed that while employment and working capacity in HD are negatively influenced by cognitive decline and motor impairments, apathy already plays a role in the prodromal stage.” (opens the source at this quote in a new tab)

Canada (Alberta): direct healthcare costs under a universal public system

“The mean total all-cause direct healthcare costs were $23,211 [$38,599] per person-year, with hospitalizations accounting for 57.8% of all-cause costs.” (opens the source at this quote in a new tab)

“Costs were higher among individuals with a long-term care stay, a proxy for disease severity.” (opens the source at this quote in a new tab)

Other countries: Israel, China, Peru, Brazil, and Germany

“Total healthcare and medication costs per patient (US dollars) were significantly higher for PwHD than controls ($7,343 vs. $3,625; P < 0.001).” (opens the source at this quote in a new tab)

“The average annual direct medical cost, direct non-medical cost, and indirect cost were 3,265.65, 805.82, and 801.97 Euros, respectively.” (opens the source at this quote in a new tab)

“The average annual cost of HD reached USD 8120 per patient in 2015.” (opens the source at this quote in a new tab)

“The mean cost of HD increased with the degree of patient dependency: from USD 6572 for Barthel 4 & 5 (slight dependency and total independency, respectively) to USD 23,251 for Barthel 1 (total dependency).” (opens the source at this quote in a new tab)

“Significant economic impacts were observed, with average annual direct medical costs of $4686.82 per HD patient.” (opens the source at this quote in a new tab)

“Thirty-three out of 34 HD patients were unemployed or retired, and 16 relied on government benefits, reflecting broader socioeconomic implications.” (opens the source at this quote in a new tab)

“Physical therapy was the most often used medical aid in HD patients (46.4%). Nursing services and speech therapy were used by 27.9 and 22.7% of HD patients, respectively, whereas use of psychotherapy was rare (3.2%).” (opens the source at this quote in a new tab)

U.S. indirect and out-of-pocket costs: HD-Charge survey

“The indirect and out-of-pocket costs of HD in the US have not been quantified.” (opens the source at this quote in a new tab)

“Quantify out-of-pocket expenses for HDGECs and companions” (opens the source at this quote in a new tab)