Cost-effectiveness of 4 mg dibotermin alfa/absorbable collagen sponge versus iliac crest bone graft for lumbar degenerative disc disease in the United Kingdom.

AIMS: To develop a model to evaluate the cost-effectiveness of 4 mg dibotermin alfa/absorbable collagen sponge (ACS) versus iliac crest bone graft (ICBG) in patients with lumbar degenerative disc disease in the United Kingdom. MATERIALS & METHODS: A Markov decision-analytic model was constructed to calculate costs and quality-adjusted life-years over a 4-year time horizon in each treatment group, from a United Kingdom National Health Service perspective. An individual patient data meta-analysis was undertaken to synthesize data from four randomized controlled trials and two single-arm studies concerning health-related quality of life and procedural resource use. Current cost data from the United Kingdom were then applied to determine the overall mean cost per patient in each group. One-way and probabilistic sensitivity analyses were undertaken to explore the impact of parameter uncertainty. RESULTS: The model predicted 4-year discounted cost savings of £192 per patient treated with dibotermin alfa/ACS, compared with ICBG, and a gain of 0.0114 QALYs per patient over the same time period. Sensitivity analyses indicated that the results were most sensitive to variability in the differences in health-related quality of life and secondary surgery rate, with dibotermin alfa/ACS having a 60% probability of being cost-effective at a willingness-to-pay threshold of £20,000 per QALY gained. LIMITATIONS: There is uncertainty in the difference in cost and QALYs between the two groups. However, comprehensive sensitivity analyses were undertaken to explore this and present the results in a transparent manner. CONCLUSIONS: Our results provide an economic case for the use of 4 mg dibotermin alfa/ACS versus iliac crest bone graft, with additional health benefits predicted at reduced overall cost.

Resource Use and Cost of Subcutaneous Nerve Stimulation Versus Optimized Medical Management in Patients With Failed Back Surgery Syndrome: An Analysis of the SubQStim Study.

OBJECTIVES: To undertake a detailed healthcare resource use and cost analysis of the SubQStim study, which randomized patients with failed back surgery syndrome (FBSS) with low back pain to receive subcutaneous nerve field stimulation in combination with optimized medical management (treatment) or optimized medical management alone (control). MATERIALS AND METHODS: Patient-level data from the SubQStim study were used to present descriptive analyses of healthcare resource use and estimated costs for pain medication, healthcare visits, adverse events, and device acquisition/implantation. A United Kingdom National Health Service perspective was adopted, using cost data from national tariffs, drug and device prices, and social care cost studies. Results were calculated as the mean cost per patient over the nine-month follow-up period. RESULTS: Mean cost per patient was £18,403 in the treatment group versus £1613 in the control group. Almost 90% of the cost in the treatment group consisted of device acquisition/implantation. Higher adverse event costs were observed for patients in the treatment group, but lower costs were observed for pain medication and healthcare visits. Over nine months, opioid use decreased in the treatment group and increased in the control group. Enrolment and follow-up were terminated early in the clinical study, leading to substantial between-patient variability in each cost category. CONCLUSIONS: Subcutaneous nerve field stimulation has the potential to offset the initial costs of the device by reducing analgesic medication and the number of healthcare visits in FBSS patients, alongside potential gains in health-related quality of life. There remains uncertainty in long-term costs and cost-effectiveness of stimulation and longer-term follow-up analyses are needed.

Parkinson's disease with early motor complications: predicting EQ-5D- 3L utilities from PDQ-39 data in the EARLYSTIM trial.

BACKGROUND: A utility value is a health-related quality of life metric (HRQoL) metric used in a cost-effectiveness analysis. While utilities as outcomes in the treatment of advanced Parkinson's disease (PD) with deep brain stimulation (DBS) are available, they do not currently exist for PD with early motor complications. The objectives of this study were to predict utilities from observed disease-specific HRQoL data using two mapping algorithms, and investigate their performance in terms of longitudinal changes within and between treatment groups, and distribution by PD severity. METHODS: This is a post hoc analysis of data from the EARLYSTIM trial of DBS compared with best medical therapy (BMT) in PD patients with early motor complications We used two published algorithms comprising ordinal and multinomial regression models to map EQ-5D-3L utilities from observed PD-specific 39 item Questionnaire (PDQ-39) scores in EARLYSTIM. Utilities were calculated using the predicted functioning levels of EQ-5D-3L dimensions and the established EQ-5D-3L UK tariffs. Statistical analyses (analysis of variance, two-tailed Student's t-test) were used to test the change from baseline within groups and difference in change from baseline between groups in utilities. Boxplots were developed to investigate the distribution of predicted utilities by PD severity, measured using the Hoehn and Yahr scale. RESULTS: The change from baseline in predicted mean utilities was statistically significant at all visits up to 24 months for the DBS group (p < 0.001) with both algorithms, and statistically significant at 12 months only (p = 0.04) for the BMT group with one algorithm. With both algorithms, the between-groups difference in change from baseline in predicted mean utilities favored DBS at all follow-up visits (p < 0.001). Based on the Hoehn and Yahr scale, predicted utilities deteriorated with increasing disease severity. CONCLUSIONS: Among PD patients with early motor complications, utilities predicted by both mapping algorithms using PDQ-39 data demonstrated a statistically and clinically meaningful improvement with DBS compared with BMT. It was not possible to conclude if one algorithm was more responsive than other. In the absence of utilities collected directly from patients, mapping is an acceptable option permitting economic evaluations to be undertaken.