Economic evaluation of ropinirole prolonged release for treatment of Parkinson's disease in the Netherlands.

BACKGROUND: By using the findings obtained from the PREPARED study, we aimed to estimate the cost effectiveness of ropinirole prolonged release (PR) [Requip-Modutab(®)] in Parkinson's disease (PD) versus ropinirole immediate release (IR). In the PREPARED study, ropinirole PR provided a significantly greater improvement in time spent 'off' than ropinirole IR when used as an add-on to levodopa. METHODS: A health state transition model was developed-based on Hoehn and Yahr (HY) stages in PD-to compare the two treatment strategies. The Markov model included the following treatment-related aspects: (i) rate of disease progression; (ii) rates of dyskinesia; and (iii) medication adherence. RESULTS: In our approach, the base-case analysis showed a favourable pharmacoeconomic profile of ropinirole PR versus ropinirole IR. In particular, general cost savings were estimated combined with modest gains in quality of life, due to reduced disease progression and lower dyskinesia rates. Sensitivity analyses showed that this result was rather robust for varying parameters deterministically, although cost savings were lost in some instances. In particular, the treatment benefits of lower dyskinesia rates and improved adherence influenced the cost-effectiveness outcome. Nonetheless, the cost effectiveness remained acceptable within the limits that were investigated. Probabilistic sensitivity analysis revealed that the probability of accepting PR over IR exceeded 95 % for all relevant 'willingness-to-pay' thresholds. CONCLUSION: The results of our study indicate a high likelihood of ropinirole PR being cost saving or at least being considered cost effective for use in the Netherlands. However, claims included in our model regarding dyskinesia and improved medication adherence should be further supported by data from daily practice.

Transferability of model-based economic evaluations: the case of trastuzumab for the adjuvant treatment of HER2-positive early breast cancer in the Netherlands.

INTRODUCTION: Geographic transferability of model-based cost-effectiveness results may facilitate and shorten the reimbursement process of new pharmaceuticals. This study provides a real world example of transferring a cost-effectiveness study of trastuzumab for the adjuvant treatment of HER2-positive early breast cancer from the United Kingdom to The Netherlands. METHODS: Three successive steps were taken. Step 1: Collect available information with regard to the original model, and assess transferability using existing checklists. Step 2: Adapt transferability-limiting factors. Step 3: Obtain a country-specific estimate of cost-effectiveness. RESULTS: The structure of the UK model was transferable, although some of the model inputs needed adaptation. From a health-care perspective, the Dutch estimate amounted to euro5828/quality-adjusted life-year gained. From a societal perspective, the incremental cost-effectiveness ratio was dominant. CONCLUSION: Transferability of a model-based UK-study in three steps proved to be an efficient method to provide an early indication of the cost-effectiveness of trastuzumab and has led to the provisional reimbursement of the treatment.

Direct costs associated with the disease management of patients with unresectable advanced non-small-cell lung cancer in The Netherlands.

INTRODUCTION: Disease management and costs of treatment of patients with unresectable advanced non-small-cell lung cancer (NSCLC) in The Netherlands are not well known. METHODS: A retrospective medical chart review was performed by collecting data from the time of diagnosis until the time of death or the end of the evaluation period. In addition to the demographic data, information was collected on the overall management of the patient. Hospital resource utilisation data collected included number of outpatient specialist visits, number and length of hospitalisation, type and number of diagnostic and laboratory procedures, type and number of radiotherapy cycles and detailed information on chemotherapy. To evaluate the economic impact of second-line treatment, a distinction was made between patients who received only best supportive care (BSC, group A) and those who received chemotherapy as a second-line treatment in addition to BSC (group B). The study was performed from the hospital perspective and reports on 2005 costs. RESULTS: Of 102 patients, 74 belonged to group A and 28 to group B. Patient management included a multidisciplinary approach, the extent of which depended on symptoms of the disease and presence of metastases. The average total treatment cost per patient per year of unresectable advanced NSCLC in The Netherlands was euro32,840 in group A and euro31,187 in group B. In both groups, hospitalisation was the major cost driver. In group B second-line chemotherapy was the second largest contributor of the costs. In spite of the difference in numbers of treatment lines provided to patients in groups A and B the total average costs per patient per year were comparable. Overall, the management of unresectable advanced NSCLC appeared to conform with current guidelines in The Netherlands. CONCLUSION: These patients show high medical resource consumption, with hospitalisation being the main cost driver in both groups. As economic arguments are becoming increasingly important in medical decision making on both national and local levels, this information is relevant for both policy makers and specialists. These data can also be used in future research to evaluate the economic impact of new therapies in NSCLC, especially of those that aim to treat patients in an outpatient setting.

Estimating the implicit value of statistical life based on public interventions implemented in The Netherlands.

OBJECTIVES: In the Netherlands, allocation decisions have not yet been explicitly based on the Value of Statistical Life. However, when policy makers decide whether or not to implement life saving interventions this trade-off is made implicitly. This study aimed to gain insights into this trade-off, hereafter referred to as Implicit Value of Statistical Life (IVSL), by means of a retrospective investment analysis of life saving interventions implemented in the Netherlands. METHODS: A literature search was conducted to find life saving intervention cases meeting the requirements for IVSL calculation. A final sample of ten cases was included in the study concerning interventions implemented in different societal sectors. For each case, an IVSL estimate was calculated according to a uniform method. RESULTS: IVSL estimates derived from the intervention cases differed considerably and ranged from 1 euro to almost 11 million euros. Differences were most extreme when comparing IVSL estimates concerning interventions implemented in different societal sectors. However, IVSL estimates also varied greatly between interventions in the same sector and even within the same interventions when critical assumptions were altered. CONCLUSION: Our findings suggest that there are great imbalances between societal investments for preventing a statistical death. This highlights the need for further deliberation about how to improve transparency of policy decisions. An approach ex ante determining the Value of Statistical Life by means of empirical methods and based on societal preferences might circumvent the problems associated with the IVSL and needs further exploration.

Cost effectiveness of oseltamivir treatment for patients with influenza-like illness who are at increased risk for serious complications of influenza: illustration for the Netherlands.

BACKGROUND: Oseltamivir is effective in the treatment of influenza. Utilisation in The Netherlands is limited, but increasing. OBJECTIVE: To estimate the cost effectiveness of oseltamivir treatment (vs symptom relief only) for patients with influenza-like illness (ILI) who are at increased risk for serious complications of influenza. METHODS: A cost-effectiveness analysis was used, building on a previously developed model (decision tree) that was applied for evaluating influenza vaccination and pandemic preparedness plans. Three patient subgroups were assessed (elderly patients [aged > or = 65 years] without chronic disease, elderly patients with chronic disease, and chronically ill, non-elderly patients). Inputs for the model were taken from various sources including a meta-analysis. A societal perspective was adopted and costs were expressed in euro per life-year gained (year 2003 values). Life-years lost were discounted at 4% in accordance with Dutch guidelines. Deterministic and probabilistic sensitivity analyses were employed to assess the robustness of the results. RESULTS: For chronically ill patients with ILI, visits to the GP for oseltamivir treatment were cost saving. For non-chronically ill elderly patients, incremental cost-effectiveness was estimated at 1759 euros per life-year gained. Cost savings and favourable cost effectiveness were robust in a deterministic and stochastic sensitivity analysis. CONCLUSION: Our model-based analysis suggests that at-risk people presenting with ILI to a GP could be offered oseltamivir at favourable cost effectiveness or even cost savings in the Dutch setting compared with symptom relief with analgesics only.

Economic evaluations of healthcare programmes and decision making: the influence of economic evaluations on different healthcare decision-making levels.

Given the potential role of economic information in healthcare decision making, it is of interest to assess its influence on decisions at a national or regional level (macro level), at a healthcare facility level (meso level) and at the healthcare provider level (micro level). This literature review summarises 36 empirical studies that examined the influence of economic evaluations on these three healthcare decision-making levels. Economic evaluations are considered useful and important; however, their direct influence on decision making (instrumental use) is moderate, especially at the macro and micro levels. A major influence was observed at the meso level, leading to the conclusion that economic evaluations have the most pronounced influence on decision making within healthcare organisations. However, unexpectedly, our literature search did not reveal an empirical study analysing the considerable influence of economic evaluations on decisions by the National Institute of Health and Clinical Excellence in the UK. Our findings indicate that results of economic evaluations cannot be considered the dominant decision criterion for healthcare decision makers at either the macro, meso or micro levels. Enlightenment use (where scientific evidence provides a background of information, ideas and concepts that affect the way policy makers view problems and solutions) of economic evaluations in decision making remains to be proven.

Development and validation of an acceptability and satisfaction questionnaire for a contraceptive vaginal ring, NuvaRing.

OBJECTIVE: To validate an acceptability questionnaire for NuvaRing, a new combined contraceptive vaginal ring. METHODS: A 21-item questionnaire was developed covering: ease of ring use, ease of package use, clarity of instructions, sexual comfort, cycle-related characteristics, compliance and satisfaction. A total of 2145 women completed the questionnaire after 3, 6 or 13 cycles of NuvaRing use. The psychometric properties and predictive value of the questionnaire were assessed using cycle 3 data (n = 1950). The quality of completed questionnaires, item content analysis, construct validity, internal consistency reliability, known groups validity and predictive validity were evaluated. RESULTS: Excluding non-ordinal items, 0.6% of the data were missing. Principal component analysis of 15 ordinal items indicated that two hypothesised dimensions ('ease of package use' and 'clarity of instructions') were consistently linked and so were combined into a single 'ease of comprehension' scale. Item convergent validity (the degree of correlation between an item and its own scale) was 100% for 'ease of ring use' (r = 0.44) and 'satisfaction' (r = 0.58), 83% for 'ease of comprehension' (r = 0.25-0.62) and 67% (r = 0.38-0.54) for 'sexual comfort', but 0% for 'cycle-related characteristics' (r = 0.31). Item discriminant validity (the degree to which an item correlates with its own scale compared with other scales) was >/=96% for all dimensions. Internal consistency reliability was acceptable for all dimensions (adjusted Cronbach's alpha coefficient >0.70). Satisfaction was higher than in the complementary groups for respondents who had no adverse events, chose NuvaRing as the best method of contraception or completed the study; this indicated good known groups validity. Low satisfaction with the method was a good predictor of early discontinuation after cycle 3, indicating that the questionnaire had good predictive validity. CONCLUSIONS: The acceptability questionnaire has good psychometric properties and can predict early discontinuation of the NuvaRing vaginal ring method of contraception.

The Aging Males' Symptoms (AMS) scale: update and compilation of international versions.

BACKGROUND: The interest of clinical research in aging males increased in recent years and thereby the interest to measure health-related quality of life (HRQoL) and symptoms of aging men. The Aging Males' Symptoms scale (AMS) became the most commonly used scale to measure HRQoL and symptoms in aging males in many countries worldwide. The aim of this paper is to review the current state of the instrument particularly concerning versions of the scale in different languages in the light of the quality of the translation process. AMS VERSIONS AVAILABLE: Most of the translations were performed following international methodological recommendations for linguistic & cultural adaptation of HRQoL instruments. Mainly the English version was used as source language for the translation into Dutch, Spanish, Portuguese, Italian, Swedish, and Japanese (attached as additional PDF-files). Preliminary versions that were derived only from forward translations are of secondary quality and available in Finnish, Flemish, and Russian. It is recommended to complete the translation process for the latter languages before using them in international studies. TRANSLATIONS IN PROCESS: The AMS scale is in the process of consensus finding of two existing French versions, and the versions in the Korean, Thai, and Indonesian languages have not yet been completed in the translation process. CONCLUSION: The AMS scale is obviously a valuable tool for assessing health related quality of life in aging men, because it is used worldwide. It is a standardized scale according to psychometric norms. Most of the currently available language versions were translated following international standards for linguistic and cultural translation of quality of life scales. Assistance is offered to help interested parties in the translation process.

Andropause and quality of life: findings from patient focus groups and clinical experts.

OBJECTIVES: To develop a condition-specific quality of life (QoL) questionnaire to assess the symptoms of the decline in testosterone that occurs as men age, otherwise known as the andropause. METHODS: Two focus groups of patients with low testosterone levels and an expert panel of physicians in the treatment of male testosterone deficiency. RESULTS: The patient focus groups confirmed that men are unlikely to realise they have low testosterone levels until this is diagnosed and that they are unlikely to share their experiences with peers. Both patients and physicians considered decreased energy levels and impaired sexual performance had the greatest adverse impact on well-being. Patients generally felt that testosterone replacement therapy led to improved energy levels and, to a lesser extent, improved libido and erectile function. Evaluation of the responses resulted in the identification of seven key domains (energy, emotional, social, social emotional, mental functioning, physical functioning and sexual functioning) that should be considered when assessing the impact of andropause on QoL. CONCLUSIONS: Understanding the impact of low testosterone levels on QoL is critical to diagnosis and effective treatment. The use of an andropause-specific QoL questionnaire will facilitate quantification of patient experiences and may thus improve subsequent therapy.