Public preferences for the allocation of societal resources over different healthcare purposes.

OBJECTIVE: Increasing healthcare expenditures require governments to make difficult prioritization decisions. Considering public preferences can help raise citizens' support. Previous research has predominantly elicited preferences for the allocation of public resources towards specific treatments or patient groups and principles for resource allocation. This study contributes by examining public preferences for budget allocation over various healthcare purposes in the Netherlands. METHODS: We conducted a Participatory Value Evaluation (PVE) choice experiment in which 1408 respondents were asked to allocate a hypothetical budget over eight healthcare purposes: general practice and other easily accessible healthcare, hospital care, elderly care, disability care, mental healthcare, preventive care by encouragement, preventive care by discouragement, and new and better medicines. A default expenditure was set for each healthcare purpose, based on current expenditures. Respondents could adjust these default expenditures using sliders and were presented with the implications of their adjustments on health and well-being outcomes, the economy, and the healthcare premium. As a constraint, the maximum increase in the mandatory healthcare premium for adult citizens was €600 per year. The data were analysed using descriptive statistics and a Latent Class Cluster Analysis (LCCA). RESULTS: On average, respondents preferred to increase total expenditures on all healthcare purposes, but especially on elderly care, new and better medicines, and mental healthcare. Three preference clusters were identified. The largest cluster preferred modest increases in expenditures, the second a much higher increase of expenditures, and the smallest favouring a substantial reduction of the healthcare premium by decreasing the expenditure on all healthcare purposes. The analyses also demonstrated substantial preference heterogeneity between clusters for budget allocation over different healthcare purposes. CONCLUSIONS: The results of this choice experiment show that most citizens in the Netherlands support increasing healthcare expenditures. However, substantial heterogeneity was identified in preferences for healthcare purposes to prioritize. Considering these preferences may increase public support for prioritization decisions.

Rethinking the eidos, genos, and diaphora of the health utility concept: a psychological perspective.

The notion of utility gained a strong foothold in health economics over the last decades. However, the concept of health utility has not yet been decisively or irrefutably defined and the definitions that exist often do not take into account the current state of psychological literature. This perspective paper shows that the current definition of health utility emphasizes decision-making processes, deploys personal preferences, assumes psychological egoism, and attempts to objectively and cardinally measure utility. However, these foundational axioms that underly the current definition of health utility are not necessarily in concurrence with the current state of psychological literature. Due to these perceived shortcomings of the current health utility definition, it may be beneficial to redefine the concept of health utility in accordance with the current state of psychological literature. In order to develop such a revised definition of health utility the commonly deployed formula (Eidos = Genos + Diaphora) originating from Aristotle's metaphysics is applied. The revised definition of health utility proposed in this perspective paper alludes to health utility as 'the subjective value, expressed in terms of perceived pain or pleasure, that is attributed to the cognitive, affective and conative experience of one's own physical, mental and social health state, which is determined through self-reflection and interaction with significant others'. Although this revised definition does neither replace nor supersede other conceptualizations of health utility, it may serve as a refreshing avenue for further discussion and could, eventually, support policymakers and health economists in operationalizing and measuring health utility in an even more accurate and veracious manner.

Cancer treatments touch a wide range of values that count for patients and other stakeholders: What are the implications for decision-making?

BACKGROUND: Cancer rates and expenditures are increasing, resulting in debates on the exact value of this care. Perspectives on what exactly constitutes worthwhile values differ. This study aims to explore all values-elements regarding new oncological treatments for patients with cancer and all stakeholders involved and to assess their implications in different decision-making procedures. METHOD: Thirty-one individual in-depth interviews were conducted with different stakeholders to identify values within oncology. A focus group with seven experts was performed to explore its possible implications in decision-making procedures. RESULTS: The overarching themes of values identified were impact on daily life and future, costs for patients and loved ones, quality of life, impact on loved ones, societal impact and quality of treatments. The expert panel revealed that the extended exploration of values that matter to patients is deemed useful in patient-level decision-making, information provision, patient empowerment and support during and after treatment. For national reimbursement decisions, implications for the broad range of values seems less clear. CONCLUSION: Clinical values are not the only ones that matter to oncological patients and the stakeholders in the field. We found a much broader range of values. Proper recognition of values that count might add to patient-level decision-making, but implications for reimbursement decisions are less clear. The results could be useful to guide clinicians and policymakers when it comes to decision-making in oncology. Making more explicit which values counts for whom guarantees a more systematic approach to decision-making on all levels.

Differences between expert reported and patient reported burden of disease rankings.

Many attempted to develop burden of disease rankings for the purpose of resource allocation, priority setting, cost-effectiveness evaluation, and service development in healthcare. As this proved difficult the World Health Organization commissioned expert panels to develop internally consistent burden of disease rankings. Although these rankings provide valuable insight in the biomedical burden of different diseases, they do not yet provide insight in the psychological burden of different diseases experienced and reported by patients on a daily basis. Since expert reported and patient reported burden of disease could differ, deviations between expert reported and patient reported burden of disease rankings are likely. To explore how these rankings differ, it is important to develop patient reported burden of disease rankings and compare these to expert reported burden of disease rankings. In this study patient reported burden of disease rankings were developed by ranking the subjective health experience of patients. To measure subjective health experience an online questionnaire was administered to a large panel of Dutch citizens. The final sample consisted of 58,490 panel members. This final sample contained 36 diseases and was largely representative of the Dutch population. The data were analysed by using reliability tests, descriptive statistics and Spearman rank-order correlation coefficients. This study shows that expert reported and patient reported burden of disease rankings could differ. Burden of cardiovascular diseases ranks low on patient reported burden of disease rankings, while it ranks higher on expert reported burden of disease rankings. Burden of psychiatric diseases and gastrointestinal diseases ranks high on patient reported burden of disease rankings, while it ranks lower on expert reported burden of disease rankings. Burden of pain diseases ranks high on patient reported burden of disease rankings, while it is still overlooked in expert reported burden of disease rankings. This study suggests that it can be beneficial to develop and utilize patient reported burden of disease rankings in addition to the already existing expert reported burden of disease rankings in order to establish a more comprehensive perspective on burden of disease. This could improve decision-making on resource allocation, priority setting, cost-effectiveness evaluation, and service development in healthcare.

Association between societal costs and treatment response in children and adolescents with ADHD and their parents. A cross-sectional study in the Netherlands.

Attention Deficit Hyperactivity Disorder (ADHD) is associated with considerable burden of illness at a patient, family and societal level. Although pharmacological treatment is recommended by authoritative guidelines, evidence on its influence on the broader burden of illness is limited. As treatment induces costs, proper healthcare decision making requires evidence on the associated societal costs or benefits and particularly the difference that response to treatment can make. Data on ADHD related resource use of patients 8-18 years and parents were collected by means of a cross-sectional, online survey amongst members of the Dutch parent association. Children were stratified to responders and non-responders to treatment according to pre-defined expert definitions. Analyses were performed on 618 questionnaires (428 responders; 190 non-responders to treatment). Children were 11.8 years on average and mainly boys (82 %). Total monthly costs for children were €578 and €839 for responders and non-responders, respectively (p = 0.021), with a breakdown to direct medical costs (€322 vs. €512; p = 0.068), direct non-medical costs (€222 vs. €296; p = 0.090), and indirect non-medical costs (€34 vs. €57; p < 0.001). For parents, total costs were €246 vs. €399 for the responding and non-responding children, respectively (p = 0.006), with a breakdown to direct medical costs (€130 vs. €211; p = 0.010) and indirect non-medical costs (€116 vs. €181; p = 0.092). Total monthly costs of children and their parents together were €824 and €1228 for responders and non-responders to treatment, respectively (p = 0.002). These results stress the importance of a focus on response to treatment, not only beneficial for patients and their family, but also resulting in considerable societal benefits.

Probabilistic Markov Model Estimating Cost Effectiveness of Methylphenidate Osmotic-Release Oral System Versus Immediate-Release Methylphenidate in Children and Adolescents: Which Information is Needed?

BACKGROUND: Incidence of attention deficit hyperactivity disorder (ADHD) in children and adolescents has been increasing. The disorder results in high societal costs. Policymakers increasingly use health economic evaluations to inform decisions on competing treatments of ADHD. Yet, health economic evaluations of first-choice medication of ADHD in children and adolescents are scarce and generally do not include broader societal effects. OBJECTIVES: This study presents a probabilistic model and analysis of methylphenidate osmotic-release oral system (OROS) versus methylphenidate immediate-release (IR). We investigate and include relevant societal aspects in the analysis so as to provide cost-effectiveness estimates based on a broad societal perspective. METHODS: We enhanced an existing Markov model and determined the cost effectiveness of OROS versus IR for children and adolescents responding suboptimally to treatment with IR. Enhancements included screening of a broad literature base, updated utility values, inclusion of costs and effects on caregivers and a change of the model type from deterministic to probabilistic. RESULTS: The base case scenario resulted in lower incremental costs (€-5815) of OROS compared with IR and higher incremental quality-adjusted life-year (QALY) gains (0.22). Scenario analyses were performed to determine sensitivity to changes in transition rates, utility of caregivers, medical costs of caregivers and daily medication dose. CONCLUSIONS: The results indicate that, for children responding suboptimally to treatment with IR, the beneficial effect of OROS on compliance may be worth the additional costs of medication. The presented model adds to the health economic information available for policymakers and to considerations on a broader perspective in cost-effectiveness analyses.

Association between quality of life and treatment response in children with attention Deficit Hyperactivity Disorder and their parents.

BACKGROUND: It has been shown that Attention Deficit Hyperactivity Disorder (ADHD) lowers the Quality of Life (QoL) of patients and their families. Medication as part of the treatment has a favourable effect on symptoms as well as functioning. Evidence on the impact of pharmacological treatment on symptoms of ADHD and the QoL of the patient and their family is still limited. There is a need for further research on QoL in ADHD as well as the relationship between ADHD and the impact on families rather than solely on patients. AIMS OF THE STUDY: Measure QoL of children with ADHD and their parents and explore the association of QoL with treatment response. METHODS: A cross-sectional survey was performed using an online questionnaire to collect QoL data of children with ADHD (based on proxy reporting of parents) and their parents in a sample of members of an ADHD parent association. QoL was measured by EQ-5D and KIDSCREEN-10. Treatment response was based on descriptions by experts, based on compliance and functioning. RESULTS: Analyses were based on 618 questionnaires (treatment responder n=428, treatment non-responder n=190). Mean age of the children was 11.8 years (82.4% male). QoL according to EQ-5D utility was 0.83 and 0.74 for responders and non-responders, respectively (p<0.001). For KIDSCREEN-10 the index was 42.24 and 40.33 for responders and non-responders, respectively (p<0.001). EQ-5D utility scores of the parents were 0.83 on average; no association with their child's treatment response could be established. A significant positive correlation between EQ-5D utility of the children and EQ-5D utility of the parents (R2=0.207, p<0.001) was found. The association between treatment response and children's QoL was significantly influenced by age category, having a sibling with ADHD, and presence of comorbidity. DISCUSSION: Strengths of this study are its sample size and the inclusion of QoL of parents, which has not been reported simultaneously before. The facts that data were derived from members of the ADHD parent association, the data for children were based on parents' report, and lack of possibility for confirmation of the clinical diagnosis are the main study limitations. IMPLICATIONS FOR HEALTH CARE PROVISION AND USE: QoL of children with ADHD was shown to be significantly positively associated with response to treatment and negatively affected by comorbidity. In determining the treatment effects in ADHD, QoL and family overall well-being should be a standard consideration as well as an integrated part of health policy discussions on ADHD. IMPLICATIONS FOR HEALTH POLICIES: Policymakers in the field of ADHD should focus on QoL of the patient, but also on the broader effects of effective treatment on the well-being of the parents. IMPLICATIONS FOR FURTHER RESEARCH: Suggestions for further research include the repetition of this study including a control group and obtaining children's self-report on QoL and clinicians report on diagnosis.

Cost utility of telaprevir-PR (peginterferon-ribavirin) versus boceprevir-PR and versus PR alone in chronic hepatitis C in The Netherlands.

BACKGROUND: The hepatitis C virus may lead to cirrhosis, liver cancer, liver transplant, and increased mortality. With standard treatment peginterferon-alpha and ribavirin (PR), sustained viral response (SVR) was less than 50 %. SVR rates improve greatly when PR is combined with telaprevir or boceprevir. OBJECTIVES: The aim of this study was to assess the cost utility of telaprevir-peginterferon-ribavirin (TPR) versus PR and boceprevir-peginterferon-ribavirin (BPR) in treatment-naïve (TN) and treatment-experienced (TE) adults with chronic hepatitis C in the Netherlands. METHODS: A Markov model with a lifelong time horizon and annual cycles was developed. Clinical data stemmed from phase III trials (TPR vs PR, BPR vs PR). A mixed treatment comparison (MTC) was developed to compare TPR and BPR indirectly. Unit costs and utilities based on EQ-5D were established in a Dutch cross-sectional study. Cost per quality-adjusted life-years (QALYs) was calculated according to the societal perspective. RESULTS: Treating TN patients with TPR generates 1.12 additional QALYs with €333 additional cost compared with PR, resulting in an incremental cost-utility ratio of €299/QALY. In TE patients, TPR dominates PR with cost savings (-€7,819) and 1.63 additional QALYs. TPR dominates BPR yielding additional QALYs (0.26 in TN; 0.71 in TE) and cost savings (-€7,296, -€18,144, respectively). CONCLUSIONS: TPR seems a cost-effective alternative to PR in TN patients and dominant in TE patients. TPR was a dominant, more effective and less costly alternative to BPR in both patient types. The cost effectiveness of both TPR and BPR is well below generally accepted willingness-to-pay thresholds and may be considered cost effective.

Validity and responsiveness of the EQ-5D and the KIDSCREEN-10 in children with ADHD.

BACKGROUND: The aim of our study is to compare the validity of a generic preference-based Quality of Life (QoL) instrument for adults to that of a generic child-specific QoL instrument in children and adolescents with attention deficit hyperactivity disorder (ADHD). METHODS: EQ-5D and KIDSCREEN-10 data were collected using a questionnaire survey performed among parents with a child or adolescent diagnosed with ADHD. The measurements were compared to assess (dis)similarities of the instruments' constructs and responsiveness to different health states. Principal component analysis (PCA) with varimax rotation was used to identify factors underlying the constructs of both instruments. Instruments' index scores of respondents with different treatment and comorbidity profiles were compared using Student's t tests. Cohen's effect sizes were calculated for an indirect comparison of the instruments' responsiveness and discriminating ability. Separate analyses were performed in children aged 8-12 and 13-18 years. RESULTS: A strong relation was found between the EQ-5D and KIDSCREEN-10 index scores. However correlations between EQ-5D and KIDSCREEN-10 items were moderate or low. The PCA identified five separate factors of quality of life. A physical and a mental factor included a combination of three EQ-5D dimensions and six KIDSCREEN-10 items; the remaining EQ-5D and KIDSCREEN-10 items constituted complementary factors without any overlap between the separate instruments. Scores of both instruments differed significantly according to respondents' response to treatment and comorbidity profile. Cohen's effect sizes indicated comparable results of the instruments' responsiveness and discriminative ability. CONCLUSIONS: The results highlight that the instruments measure different constructs of QoL in children with ADHD. Despite this, the analyses showed comparable responsiveness and discriminative ability of the instruments. These results suggest that for economic evaluations, the EQ-5D is an appropriate and valid instrument for measuring QoL in children.

Predictors and impact of non-adherence in adults with attention-deficit/hyperactivity disorder receiving OROS methylphenidate: results from a randomized, placebo-controlled trial.

BACKGROUND: Medication non-adherence has an important impact on treatment efficacy and healthcare burden across a range of conditions and therapeutic areas. The aim of this analysis was to determine predictors of non-adherence and impact of non-adherence on treatment response in adults with attention-deficit/hyperactivity disorder (ADHD). METHODS: Post-hoc analysis of a 13-week randomized, double-blind placebo-controlled study of OROS methylphenidate (MPH) 54 and 72 mg/day. Primary efficacy variable was the Conners' Adult ADHD Rating Scale - Screening Version (CAARS:O-SV). Daily adherence was calculated as average daily adherence (100 × capsules taken/2), with overall adherence calculated as the average daily adherence. Predictors of adherence were assessed using mixed-effects logistic regression. Descriptive statistics were generated for change in CAARS:O-SV score for adherent (> 95% adherence) and non-adherent subjects. Predictors of change were analyzed using a mixed model. RESULTS: Subjects were allocated to OROS MPH (54 mg, n = 87; 72 mg, n = 92) or placebo (n = 97). Mean adherence was 92.6% and 93.3% (OROS MPH 54 and 72 mg/day, respectively), versus 97.5% (placebo). Adherence was higher and less variable in completers. Factors significantly associated with non-adherence included female sex, shorter time since ADHD diagnosis, higher education level (completion of university) and score on the Drug Use Screening Inventory psychiatric disorders subscale. Improvements from baseline in CAARS:O-SV score were numerically greater in subjects defined as adherent than in those who were non-adherent. Significant predictors of CAARS:O-SV change in patients who completed the study included percentage adherence up to the point of assessment (p < 0.0001), baseline score (p < 0.0001) and family history of ADHD (p = 0.0003). CONCLUSION: The results of this analysis suggest that newly diagnosed patients, those with a high score on the DUSI-R psychiatric disorder scale, women, and subjects with high educational degrees may be at increased risk of non-adherence. Clinicians and policymakers should therefore pay special attention to these individuals, as non-adherence is a significant predictor of reduced response to treatment. TRIAL REGISTRATION: EudraCT #: 2007-002111-82.

Diffusion of a new drug: a comparative analysis of adoption, treatment complexity, and persistence of risperidone long-acting injectable therapy in the Netherlands.

OBJECTIVE: The study's objective was to analyze the adoption and persistence of risperidone long-acting injectable (RLAI) therapy after its introduction in the Netherlands in 2003 compared with the adoption and persistence of existing first-generation antipsychotic (FGA) depot drugs as an example of the diffusion of a new drug in the Netherlands. METHODS: Data on antipsychotic use were obtained from the InterAction DataBase (IADB.nl), a database containing pharmacy dispensing records of patients in the northern Netherlands, from May 20, 2003, to December 31, 2006. Treatment complexity for patients prescribed RLAI was analyzed on the basis of psychotropic comedication at baseline and during treatment, as well as on the number of previous antipsychotic therapies. Differences in treatment complexity between patients using RLAI and those using FGA depot drugs were estimated using parametric regressions. To evaluate persistence, survival analysis techniques were applied to estimate the probability of patients continuing the use of RLAI or FGA depot drugs over time. RESULTS: Data on 435 patients who were treated with depot antipsychotics were extracted from the IADB.nl. Patients had a mean (SD) age of 40.7 (13.8) years, and 65% of them were male. The results of this analysis indicated that persistence for patients prescribed RLAI was significantly lower compared with other depot antipsychotics (RLAI vs zuclopenthixol, P = 0.002; RLAI vs all other depot antipsychotics, P = 0.009). At the initiation of treatment, patients prescribed RLAI had more previous psychotropic comedication and had, on average, approximately 5 and approximately 1.5 times more prior depot drug therapies compared with zuclopenthixol and any other FGA depot drug, respectively. CONCLUSIONS: The findings of this study suggest that RLAI has been prescribed more often for difficult-totreat patients than have other available depot antipsychotics. This may explain the low adoption and poor persistence observed in the first few years after the introduction of RLAI. Further research with more extensive data should be pursued to obtain better understanding of the current diffusion of RLAI in daily clinical practice.

Long-acting methylphenidate-OROS in youths with attention-deficit hyperactivity disorder suboptimally controlled with immediate-release methylphenidate: a study of cost effectiveness in The Netherlands.

BACKGROUND: Attention-deficit hyperactivity disorder (ADHD) is the most common mental health disorder in youths. Stimulants are the drugs of first choice in the treatment of ADHD. It has been suggested that full costs associated with the treatment of ADHD may be reduced by once-daily administration regimens of stimulants. OBJECTIVES: To estimate the cost effectiveness of treatment with long-acting methylphenidate osmotic release oral system (OROS) [Concerta] for youths with ADHD for whom treatment with immediate-release (IR) methylphenidate is suboptimal. STUDY DESIGN: We developed a Markov model to obtain an incremental cost-effectiveness ratio (ICER). The analysis covered 10 years, with a Markov cycle of 1 day. Costs (in 2005 euros ) included medication, consultations and treatment interventions, and additional costs for attending special education. Quality-adjusted life-years (QALYs) were used as the effectiveness measure. Outcome probabilities were taken from the medical literature and an expert panel of five child psychiatrists and paediatricians. Univariate sensitivity analyses were performed to assess the robustness of the base-case estimate. Multivariate sensitivity analysis was used to estimate a worst- and best-case ICER. RESULTS: The ICER of methylphenidate-OROS treatment in youths with ADHD for whom treatment with IR methylphenidate is suboptimal was euro 2004 per QALY. Total costs after 10 years were euro 15,739 for the IR methylphenidate pathway and euro 16,015 for the methylphenidate-OROS pathway. In the univariate sensitivity analysis, the ICER was sensitive to changes in resource use and the probability of stopping stimulant treatment in favour of IR methylphenidate. An ICER of 0 was reached with a 6.2% price reduction of methylphenidate-OROS. CONCLUSION: Methylphenidate-OROS is a cost-effective treatment for youths with ADHD for whom treatment with IR methylphenidate is suboptimal. Higher medication costs of methylphenidate-OROS were compensated for by savings on resource use, yielding similar 10-year costs compared with treatment with IR methylphenidate. Our analysis is sensitive to both clinical parameters and (differences in) resource utilization and costs between the groups modelled, warranting further research within clinical trials and observational databases, and into the full scope of costs.

Cordectomy by CO2 laser or radiotherapy for small T1a glottic carcinomas: costs, local control, survival, quality of life, and voice quality.

BACKGROUND: The clinical results of radiotherapy and endoscopic cordectomy for T1a glottic carcinoma are reported to be similar, but costs of both treatments may differ. Therefore, we retrospectively evaluated the costs, voice quality, quality of life, and clinical results of both treatments. METHODS: Costs and effects from the first visit up to 2 years of follow-up were calculated, based on chart data of 35 and 54 patients who were treated with curative intent for T1a glottic carcinoma from 1995 to 1999 with radiotherapy and endoscopic cordectomy, respectively. Voice quality and quality of life were evaluated by means of patient questionnaires. RESULTS: Total costs (in Euros) of radiotherapy and CO(2)-laser cordectomy were 8322 euro and 4434 euro, respectively, including the costs of treating possible recurrences. The effects of both treatments were equal, including voice quality and quality of life. CONCLUSIONS: We conclude that endoscopic cordectomy is an efficient alternative for radiotherapy for these patients.

Compliance and efficiency before and after implementation of a clinical practice guideline for laryngeal carcinomas.

We evaluated whether the implementation of a nationwide clinical practice guideline for diagnosis, treatment and follow-up of laryngeal carcinomas led to changes in hospital costs, balanced against clinical changes observed following the guideline's implementation. Charts of 822 patients with larynx carcinoma (459 treated before the introduction of the guideline and 363 thereafter) in five hospitals were retrospectively investigated. In all phases, no differences in total hospital costs were observed after the guideline's implementation. Total mean costs were Euro 3,207 (95%CI 3,091-3,395) for diagnosis, Euro 3,169 (2,153-4,182), Euro 5,026 (3,996-6,057), Euro 6,458 (5,579-7,337), Euro 8,037 (7,469-8,606), Euro 12,765 (10,763-14,769), Euro 19,227 (16,848-21,605) for treatment of dysplasia, carcinoma in situ, T1, T2, T3 and T4 carcinoma, respectively, and Euro 1,856 (1,491-2,220) for 1 year disease-free follow-up. In an earlier study, we observed several positive changes after the guideline's implementation. Balanced against the equal costs before and after the guideline's implementation, we conclude that the efficiency of the care process improved.

Cost analysis of common treatment options for indolent follicular non-Hodgkin's lymphoma.

BACKGROUND AND OBJECTIVES: We assessed direct health care costs associated with the most commonly prescribed treatments for indolent follicular non-Hodgkin's lymphoma (FL). DESIGN AND METHODS: New and previously diagnosed FL patients (>or=18 years) known during 1997-1998 to 15 Dutch hospitals were selected for inclusion. Each patient was followed for 3 years, and resource use associated with each of the treatments, including watchful waiting, was recorded. The hospital perspective was adopted. Unit costs were based on 2003 price levels. RESULTS: Two hundred patients were included of whom 75% underwent one or more treatments during the 3-year data collection period [25% were not treated because of a watchful waiting strategy (10%) or complete remission (15%)]. Allogeneic and autologous stem cell transplantations were the most expensive treatments, with a mean (median) per patient cost of 45,326 euro(44,237; n=7) and 18,866 euro (16,532; n=9), respectively (up to discharge only). Intravenous fludarabine cost 10,651 euro (9,995; n=33), rituximab (10,628 euro; 10,124; n=7), and CHOP 7,547 euro (5,833; n=42). Classical FL treatments were found to be the least expensive treatments used with an estimated cost for cylophosphamide, vincristine and prednisone of 5,268 euro (2,644; n=58), for radiotherapy of 4,218 euro (4,313; n=52), and for chlorambucil of 2,476 euro (1,098; n=53). INTERPRETATION AND CONCLUSIONS: This study presents information on resource use and costs associated with the most commonly prescribed FL treatments. In addition to differences in effectiveness, commonly used treatments vary considerably in terms of resource use and overall cost. This information is of value for resource planning, given the high costs of new treatment modalities.

Costs of treatment intensification for head and neck cancer: concomitant chemoradiation randomised for radioprotection with amifostine.

This study presents an overview of costs of a chemoradiation protocol in head and neck cancer patients and an analysis of whether prevention of acute toxicity with amifostine results in a reduction to costs. Fifty-four patients treated with weekly paclitaxel concomitant with radiation were randomised for treatment with subcutaneously administered amifostine (500 mg) and analysed with respect to costs of treatment. Total costs for work-up, treatment and toxicity were calculated per treatment arm. No significant differences were found between treatment arms in preliminary results regarding response (98%), toxicity and 2-year survival (77%). Average costs for toxicity were Euro 3.789, largely influenced by hospital admissions (Euro 3.013). Total costs for amifostine administration amounted to Euro 6.495 per patient. The average total costs of treatment were Euro 19.647 versus Euro 13.592 with or without amifostine, respectively. The applied (subcutaneous) dose of amifostine appeared to be insufficient for radioprotection and reduction of related costs in the concomitant chemoradiation scheme, whereas total costs increased remarkably. Although it would be accompanied by a further cost raise, applying a higher amifostine dose might reduce (mucosal) toxicity and therefore in the long run lower related costs for hospital admission and tube feeding.

Cost determinants in aggressive non-Hodgkin's lymphoma.

BACKGROUND AND OBJECTIVES: The 5 factors of the International Prognostic Index (IPI) for aggressive non Hodgkin's lymphoma (NHL) age, disease stage, serum lactate dehydrogenase (LDH), performance status, number of extranodal sites) are validated predictors of a patient's survival. Given the need for economic evaluations, we analyzed whether the IPI and the presence of B-symptoms (night sweats, fever, weight loss) can identify subgroups of patients with favorable or unfavorable cost profiles. DESIGN AND METHODS: Chart data for 374 patients with newly diagnosed stage II-IV aggressive NHL treated between 1993-2001 with CHOP chemotherapy were used. Costs were calculated up to two years from the start of treatment. The cost of granulocyte colony-stimulating factor (G-CSF) was not included, as some patients received this due to trial participation. Regression analyses and non-parametric bootstrap tests were performed to determine the significance of prognostic factors. RESULTS: Mean first-line treatment costs (excluding G-CSF) were pound sterling 10047 (<60 years) and pound sterling 12232 (>60 years). Two-year follow-up costs averaged pound sterling 14039 and pound sterling 9026 for the two age groups, respectively. The 5 IPI variables, the 2 IPI risk group variables (resulting from the 5 factors) and B-symptoms all showed significant univariate associations with first-line treatment costs. They were also associated with higher 2-year costs, except for age, LDH, and standard risk group index. Lower predictability of total 2-year costs was due to wide variations in second-line treatments. INTERPRETATION AND CONCLUSIONS: The IPI factors and B-symptoms are predictive of treatment costs. The detailed information presented in this paper is of value for those who need to make cost-effectiveness estimations in NHL, which is a relevant topic, given new treatment modalities that are emerging.

Economic assessment on the management of chronic lymphocytic leukaemia.

In the last decade, several new promising treatments for chronic lymphocytic leukaemia (CLL) have been developed. Healthcare costs are increasing and new treatments tend to be very expensive; therefore, information about the cost effectiveness in treatments for CLL is urgently needed. The authors performed a literature review on the currently available economic evaluations on CLL treatments. A total of 65 articles were found, of which 11 could be included. These articles were evaluated on the basis of six methodological requirements for economic evaluations, enabling readers to judge the value of the studies. Only a small amount of information was available on the costs of CLL treatments. Future economic evaluations should be performed according to the methodological requirements for these studies, which should also be properly documented.

Evaluating adherence to the Dutch guideline for diagnosis, treatment and follow-up of laryngeal carcinomas.

BACKGROUND AND PURPOSE: An evidence-based clinical practice guideline for laryngeal carcinomas was introduced in the Netherlands late 1999. The objective of this guideline was to ensure uniformity in the diagnosis, treatment, and follow-up. We retrospectively evaluated whether clinical practice changed according to the recommendations of this guideline and whether it succeeded in its aim. MATERIAL AND METHODS: In five out of eight Dutch university hospitals, chart data of 459 patients treated before the guideline introduction were compared to data of 363 patients treated after the guideline introduction. RESULTS: Patient and tumour characteristics were comparable among both groups. In general, the guideline recommendations were properly complied with. The patients treated before the guideline introduction were actually also for a large part already treated according to the guideline's recommendations. After its introduction, several changes according to the guideline were observed: increased rates of reassessment of biopsy samples taken in local hospitals, psychological screening (although still only performed in 10.5% of patients), application of accelerated radiotherapy schedules, clinical trial treatments, function-preserving treatments, and decreased rates of total laryngectomy, and annual chest X-rays during follow-up. CONCLUSIONS: Although a causal relationship cannot be established in this kind of observational studies, several positive changes were observed after the introduction of the guideline, and therefore the guideline seems to have contributed to more uniformity. The largest changes were seen for the guideline recommendations based on the highest levels of evidence.