Cost-effectiveness analysis of degarelix for advanced hormone-dependent prostate cancer.

OBJECTIVE: To evaluate the cost-effectiveness of degarelix vs luteinizing hormone-releasing hormone analogue (triptorelin) plus short-term antiandrogen treatment for advanced prostate cancer. METHODS: We developed a decision analytic model based on a clinical trial and literature review. The two interventions evaluated were: (i) monthly injection of degarelix and (ii) 3-monthly triptorelin therapy plus short-term flutamide, cyproterone or bicalutamide treatment. The model consisted of a decision tree monitoring a hypothetical cohort of patients aged 70 years from the start of hormonal treatment to the end of the first month, and a Markov model monitoring patients from the end of month 1 for a time horizon of 10 years (i.e. when 96% of patients are assumed to have died). The base-case analysis assumed patients present with asymptomatic metastatic prostate cancer. Costs and outcomes were collected over the model time horizon. Outcome measures were quality-adjusted life years (QALYs), lifetime costs and incremental cost-effectiveness ratios. Sensitivity analyses (one-way and multi-way) and probabilistic sensitivity analyses were conducted to explore the uncertainties around the assumptions. RESULTS: In the base-case analysis, the incremental cost-effectiveness ratio (ICER) for degarelix vs triptorelin plus antiandrogen was £59,000 per QALY gained. The model was most sensitive to the rate of significant adverse events in the triptorelin plus antiandrogen group. The model was also sensitive to the assumed survival of patients with metastatic prostate cancer and the price of degarelix. The results of the probabilistic sensitivity analyses suggested that there was a low probability (9.6%) of degarelix being the most cost-effective treatment option when a willingness-to-pay threshold of £30,000 per QALY gained is assumed. CONCLUSION: Degarelix is unlikely to be cost-effective compared to triptorelin plus short-term antiandrogen in the management of advanced prostate cancer with respect to the usual thresholds of cost-effectiveness used in the UK: £20,000-30,000 per QALY gained (used by the National Institute for Health and Clinical Excellence).

Cost-effectiveness of alemtuzumab for T-cell prolymphocytic leukemia.

OBJECTIVES: The aim of this study was to evaluate the cost-effectiveness of alemtuzumab (CAMPATH-1H) compared with conventional chemotherapy in people with T-cell prolymphocytic leukemia (T-PLL). METHODS: We developed a decision-analytic model to assess the costs and benefits of alemtuzumab or conventional therapy based on their effects on quality of life of patients. The main outcome was the incremental cost-effectiveness ratio incorporating costs per additional quality-adjusted life-year (QALY) gained over lifetime. Due to the limited data available, a large number of assumptions had to be made to construct the cost-utility model. One-way, multi-way, and probabilistic sensitivity analyses (PSA) were conducted to explore the impact of these uncertainties. Expected values of perfect information were also calculated for four specific scenarios. RESULTS: Depending on different key assumptions made, the PSA suggested distinct conclusions using a willingness-to-pay threshold of 30,000 GBP per QALY gained. Using this threshold, the probability that alemtuzumab would be cost-effective varies from 0 percent to 53 percent for the four modeled scenarios. Population expected value of perfect information analysis suggests that resolving the parameter uncertainty in the analysis for people with T-PLL in the United Kingdom would have considerable value--up to 5.3 million euro. CONCLUSIONS: Alemtuzumab appears more likely to be cost-effective if used earlier in the course of T-PLL and where it replaces the use of multiple alternative therapies. However, cost-effectiveness is highly uncertain and future research is clearly justified. Nevertheless, our analysis demonstrates the feasibility of considering the cost-effectiveness of an agent despite the presence of significant uncertainty to provide appropriate assessment information to policy makers.

Public participation in decision-making on the coverage of new antivirals for hepatitis C.

Purpose - New hepatitis C medicines such as sofosbuvir underline the need to balance considerations of innovation, clinical evidence, budget impact and equity in health priority-setting. The purpose of this paper is to examine the role of public participation in addressing these considerations. Design/methodology/approach - The paper employs a comparative case study approach. It explores the experience of four countries - Brazil, England, South Korea and the USA - in making coverage decisions about the antiviral sofosbuvir and involving the public and patients in these decision-making processes. Findings - Issues emerging from public participation ac tivities include the role of the universal right to health in Brazil, the balance between innovation and budget impact in England, the effect of unethical medical practices on public perception in South Korea and the legitimacy of priority-setting processes in the USA. Providing policymakers are receptive to these issues, public participation activities may be re-conceptualized as processes that illuminate policy problems relevant to a particular context, thereby promoting an agenda-setting role for the public. Originality/value - The paper offers an empirical analysis of public involvement in the case of sofosbuvir, where the relevant considerations that bear on priority-setting decisions have been particularly stark. The perspectives that emerge suggest that public participation contributes to raising attention to issues that need to be addressed by policymakers. Public participation activities can thus contribute to setting policy agendas, even if that is not their explicit purpose. However, the actualization of this contribution is contingent on the receptiveness of policymakers.

Patterns of public participation.

Purpose - The paper summarizes data from 12 countries, chosen to exhibit wide variation, on the role and place of public participation in the setting of priorities. The purpose of this paper is to exhibit cross-national patterns in respect of public participation, linking those differences to institutional features of the countries concerned. Design/methodology/approach - The approach is an example of case-orientated qualitative assessment of participation practices. It derives its data from the presentation of country case studies by experts on each system. The country cases are located within the historical development of democracy in each country. Findings - Patterns of participation are widely variable. Participation that is effective through routinized institutional processes appears to be inversely related to contestatory participation that uses political mobilization to challenge the legitimacy of the priority setting process. No system has resolved the conceptual ambiguities that are implicit in the idea of public participation. Originality/value - The paper draws on a unique collection of country case studies in participatory practice in prioritization, supplementing existing published sources. In showing that contestatory participation plays an important role in a sub-set of these countries it makes an important contribution to the field because it broadens the debate about public participation in priority setting beyond the use of minipublics and the observation of public representatives on decision-making bodies.

Cost effectiveness of oromucosal cannabis-based medicine (Sativex®) for spasticity in multiple sclerosis.

BACKGROUND: Spasticity is common in patients with multiple sclerosis (MS) and is a major contributor to disability. Sativex®, an oromucosal spray containing cannabis-based medicinal products, has been found to be effective in reducing spasticity symptoms. OBJECTIVE: Our objective was to estimate the cost effectiveness of Sativex® plus oral anti-spasticity medicines compared with the current standard treatment for moderate or severe spasticity in MS in the UK. METHODS: A Markov model was used to assess the costs and benefits of Sativex® plus oral anti-spasticity medicines or current standard treatment based on their effects on the quality of life of patients. The main outcome was the incremental cost-effectiveness ratio (ICER) in terms of costs per additional QALY gained over 5 years of treatment. One-way, multi-way and probabilistic sensitivity analyses were conducted to explore the impact of uncertainties on the findings. RESULTS: In the base case, Sativex® plus oral anti-spasticity medicines resulted in incremental costs of £7600 and a QALY gain of 0.15 per person over 5 years (ICER = £49 300 per QALY).[year 2009 data for costs]. Findings were sensitive to the costs of Sativex® (price and dose) and differences in utilities between responders and non-responders. CONCLUSIONS: Using a willingness-to-pay threshold of £30 000 per QALY, Sativex® appears unlikely to be considered cost effective by UK funders of healthcare for spasticity in MS. This is unfortunate, since it appears that Sativex® use is likely to benefit some patients in the management of this common consequence of MS.