PharmacoEconomics
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The dystonias are a group of movement disorders arising from CNS dysfunction and characterised by involuntary and prolonged spasms of muscle contraction. Recently there has been increasing demand for treatment with botulinum toxin (BT), a relatively expensive neurological paralytic agent. As there has been no systematic assessment of patient benefit from BT, this study was undertaken to develop and test a methodology for assessing the cost utility of BT therapy for patients with dystonias. ⋯ The cost-per-QALY estimates ranged considerably, depending on the type of dystonia, the duration of BT treatment, type of health-related quality-of-life (HR-QOL) tariff used and baseline characteristics of participants. The study findings reflect the general clinical impression of BT: that it can benefit patients with dystonia, but the benefit may be small compared with many treatments for other diseases. The nature of the disease and its cyclical treatment caused practical difficulties in recruiting participants, administering questionnaires and in estimating QALY gains.