Cost of illness associated with Niemann-Pick disease type C in the UK

Figures & data

Figure 1. Percentage of survey respondents (n=18) (a) consulting each type of healthcare professional, (b) accessing each type of other healthcare service, and (c) undergoing each type of test or investigation (in previous 6 months). Total number of respondents reporting any use of each resource category: n=14 for consultations, n=12 for ‘other funded services’, n=11 for tests and investigations.

Figure 2. Patient distribution by total annual cost of NP-C.

Figure 3. Breakdown of (a) mean total annual cost of NP-C per patient and (b) mean annual direct medical costs for NP-C patients. OoP, out-of-pocket; NHS, National Health Service; PSS, Personal Social Services.

Table 1. Recall period and unit cost sources for resources captured in the NP-C Medical Resource Use questionnaire.

Payer	Resource use	Recall period	Source of unit cost data
Direct medical
NHS	Hospital admissionsHospital outpatient visits*Home visitsInvestigations/testsPrescription drugsHospice and respite care	6 months6 months1 month6 months1 day6 months	NHS reference costs; PSSRU 2007PSSRU 2007PSSRU 2007NHS Reference Costs 2006-07BNFPSSRU 2007
Patient (OoP)	Private specialist consultationsNon-prescription drugs	6 months1 month	Collected in questionnaireCollected in questionnaire
Direct non-medical
DWP, DH, DfES	Travel expenditureSocial servicesAids/appliances^†Specialist educational support	1 month6 months6 months6 months	PSSRU, internet and other sourcesPSSRU 2007PSSRU 2006<www.sheffield.gov.uk/?pgid=64269& fs=b> accessed 16.01.2008;<www.dfes.gov.uk/trends/index.cfm? fuseaction=home.showChart&cid=2&iid=6& chid=27> accessed 01.07.2008; PSSRU 2007; NHS Reference Costs 2006–07
Patient (OoP)	Private home helpTravel expenditureAids/appliances^†	6 months1 month6 months	Collected in questionnaireCollected in questionnairePSSRU 2006
Indirect
Society	Productivity losses (absenteeism) due to inability to work	3 months	ASHE 2007, ONS

*GP/nurse/AHP (allied health professional) consultations; ^†adaptations to house/car. Abbreviations: ASHE, Annual Survey of Hours and Earnings; BNF, British National Formulary; DWP, Department of Work and Pensions; DH, Department of Health; DfES, Department for Education and Skills; GP, General Practitioner (primary healthcare physician); NHS, National Health Service; OoP, out-of-pocket (expenses); ONS, Office of National Statistics; PSSRU, Personal Social Services Research Unit.

Table 2. Total mean direct and indirect costs per NP-C patient per month (2007).

Cost category	Range of mean (SD) costs for individual resource items in respective category (£)	Mean (SD) monthly cost* per patient per resource category (£)
Direct medical costs
Tests and investigations	0.02 (0.05)–43.1 (89.5)	74.9 (155.3)
Medication (prescribed)	0.1 (0.4)–10.9 (46.2)	18.3 (60.4)
Medication (non-prescribed; OoP)	—	5.6 (8.8)
Home visits	4.6 (19.3)–1,087.8 (3,762.8)	1,092.3 (3,781.7)
Healthcare consultations	0.08 (0.4)–34.4 (29.2)	47.1 (35.5)
Hospitalisations	3.1 (7.1)–9.4 (27.2)	17.9 (26.8)
Other healthcare services^†	0.2 (0.6)–2.7 (8.2)	14.2 (22.6)
Other healthcare services (OoP)	—	0.9 (3.9)
Residential care	37 (157.1)–298.6 (866.2)	236.1 (718)
Total		1,507.3 (3,884.2)
Direct non-medical costs
Special education	0.3 (1.3)–422.2 (700.6)	764 (1,247.2)
Travel (OoP)	3.9 (12.4)–10.6 (33)	22.8 (46.6)
Total		786.8 (1,241.3)
Indirect costs
Productivity losses (patient)	63.5 (269.4)–770.1 (993.4)	833.6 (977.7)
Productivity losses (carer)	6.8 (267.7)–129.8 (347.2)	136.3 (362.6)
Total		969.9 (1,079.8)
Overall total		3,264 (4,192.9)

*Across entire sample population; ^†including social worker support, occupational therapy, dietician consultation, swallowing therapy, geneticist consultation, speech therapy, physiotherapy, educational psychology and hearing specialist consultation; OoP, out-of-pocket.

Table 3. Sensitivity analysis: total costs in miglustat-treated versus non-miglustat-treated patients*.

Costs	Mean (SD) total monthly cost per patient per resource category (£)
	Patients not taking miglustat (n=14)	Patients taking miglustat (n=4)
Direct medical costs	1,599.8 (4,374.7)	1,183.4 (1,543.8)
Direct non-medical costs	752.8 (1,139.5)	905.8 (1,755.1)
Indirect costs	964.2 (1,105.9)	990.2 (1,143.4)
Overall total	3,316.8 (4,679.4)	3,079.4 (2,163.0)

*Cost of miglustat was excluded from main analysis, as patients taking miglustat received it as part of a clinical trial.