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

Abstract

Objective: Niemann-Pick disease type C (NP-C) is a rare and devastating genetic disorder characterised by a range of progressive neurological symptoms, which imposes a burden on patients, family members, the healthcare system and society overall. The objective of this study was to assess direct and indirect costs associated with NP-C in the UK.

Methods: This was a non-interventional, retrospective, cross-sectional cohort study based on responses from patients and/or their carers/guardians recruited from a UK NP-C database. Resource use and direct medical, direct non-medical and indirect costs were evaluated using data collected via postal survey in October 2007, which included a Medical Resource Use questionnaire. Total annual costs per patient were estimated.

Results: In total, 18 Medical Resource Use questionnaires (29% response rate) were received and analysed. The mean total annual cost (SD) of NP-C per patient was £39,168 (£50,315); 46% were direct medical costs, to which home visits and residential care contributed 68% and 15%, respectively. Direct non-medical costs accounted for 24% of the average annual cost per patient, mainly due to specialist education, and indirect costs 30%. If only direct medical costs were considered, the mean annual cost (SD) per patient was reduced to £18,012 (£46,536).

Conclusions: The direct annual per-patient cost of NP-C illness in 2007 appears moderate when compared with other rare and severely disabling diseases. However, cost estimates may be conservative, since findings are limited by a small sample size, low survey response rate and potential recall bias. As demonstrated by this study, a substantial proportion of the cost is shifted from the healthcare system to the patient, family and non-medical providers. These findings highlight the need for treatments that can slow or stop disease progression in NP-C.

Key words::

• cost
• direct
• indirect
• Niemann-Pick disease type C
• resource use

Acknowledgments

Declaration of interest: This study was funded by Actelion Pharmaceuticals Ltd, Allschwil, Switzerland. J.I. has received research support from Actelion for participation in clinical trial programmes, and speaker fees for participation in sponsored events. C.G. and E.H. are employees of Actelion Pharmaceuticals Ltd. K.G. and K.L. are employees of HERON Evidence Development Ltd, which received consultancy fees from Actelion Pharmaceuticals Ltd. Alpha-Plus Medical Communications Ltd provided writing assistance, paid for by Actelion Pharmaceuticals Ltd.

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