Treatment of supraglottic squamous cell carcinoma with advanced technologies: observational prospective evaluation of oncological outcomes, functional outcomes, quality of life and cost-effectiveness (SUPRA-QoL).

BACKGROUND: Over the past decade, therapeutic options in head and neck supraglottic squamous cell carcinoma have constantly evolved. The classical total laryngectomy has been partially replaced by alternative organ- and function-sparing techniques with the same prognosis but less morbidity, such as Radiotherapy, Transoral Laser Microsurgery (TLM) and Trans-Oral Robotic Surgery (TORS). Up to now, a prospective comparison of these innovant techniques has not been conducted. METHODS/DESIGN: We will conduct an original international multicentric prospective nonrandomized clinical trial to compare the efficacy between these treatments (Arm 1: Radiotherapy ± chemotherapy; Arm 2: TLM and Arm 3: TORS) with 4 classes of outcomes: quality of life (QoL), oncological outcomes, functional outcomes and economic resources. The population will include cT1-T2 /cN0-N1/M0 supraglottic squamous cell carcinoma. The primary outcome is a Clinical Dysphagia QoL evaluation assessed by the MD Anderson Dysphagia questionnaire. Secondary outcomes include others QoL evaluation, oncological and functional measures and cost parameters. The sample size needs to reach 36 patients per arm (total 108). DISCUSSION: In the current literature, no prospective head-to-head trials are available to compare objectively these different treatments. With the increase of highly efficient treatments and the increase of oncological survival, it is imperative also to develop management strategies that optimize QoL and functional results. We will conduct this innovate prospective trial in order to obtain objective data in these two main issues. TRIAL REGISTRATION: NCT05611515 posted on 10/11/2022 (clinicaltrial.fgov).

Economic impact of antidepressant treatment duration in naturalistic conditions.

OBJECTIVE: To assess the economic impact of the duration of antidepressant drug treatment in a real-life setting. METHOD: A historical fixed cohort study included 27 917 patients aged 18 and over with a new antidepressant treatment registered in the national insurance database. The economic impact concerned healthcare expenditure in the first 3 months after treatment discontinuation. Generalized linear models were used to compare two groups of treatment duration: <6 months and 6 months and over. RESULTS: After adjustment for care costs before and during treatment episode, gender, age, chronic diseases, welfare and prescriber specialty, total healthcare costs (in log) [-0.06 (-0.14;0.01) P = 0.11] and psychiatric care costs (in square root) [-0.08 (-0.41;0.25) P = 0.6] were similar in both groups. Non-psychiatric care costs were significantly lower in the 'long treatment duration' group compared with the 'short treatment duration' group [-11.4 (-15.8; -7.0) P < 0.0001]. The decreases in total and non-psychiatric care costs over the antidepressant treatment episode were larger in the 'long treatment duration' group compared with the 'short treatment duration' group. CONCLUSION: With regard to healthcare costs and global health, antidepressant drug treatments of short duration appear less effective than treatment of recommended duration.

Growth monitoring for short stature: update of a systematic review and economic model.

OBJECTIVES: The aim of the project was to compare different screening rules and/or referral cut-offs for the identification of children with disorders of short stature. We undertook an update of a previous systematic review and economic model that addressed the same question. DATA SOURCES: Sources searched included MEDLINE, EMBASE, Science Citation Index, Social Science Citation Index, Conference Proceedings Citation Index - Science/Social Science & Humanities, Cochrane Library 2009 Issue 4, Office of Health Economics Health Economic Evaluations Database, and the NHS Economic Evaluation Database. REVIEW METHODS: The review was conducted as an update to our previous assessment in 2007. Searching covered January 2005 to November 2009 with no language or publication restrictions. Two reviewers examined full papers for relevance. Data extraction was conducted by one reviewer and independently checked by a second. In addition, searches were conducted to identify quality of life or utility papers to inform the economic evaluation. We developed a probabilistic decision analytic model to estimate the costs and quality-adjusted life-year (QALY) gains from the perspective of the UK NHS and personal social services. The model was a cohort model, assuming a homogeneous population of 5-year-olds at baseline. RESULTS: One study was included in the systematic review. The study was not UK based, but had been identified in the brief as relevant to the UK setting. The study's authors examined the performance of a number of rules to determine sensitivity and specificity of referral for short stature in four patient groups and three reference groups in the Netherlands. They derived an algorithm for referral based on the optimal rules. No new studies were located that provided appropriate quality of life or utilities data for the economic model. The model was based on the previous assessment which was updated to better reflect current UK clinical practice. We compared two alternative monitoring strategies, one of which was based on the study identified in our systematic review (Grote strategy); the other was based on UK consensus (UK strategy). We identified that the UK strategy was the least effective and least costly, with a mean gain of 0.001 QALYs at a mean cost of £21. The Grote strategy was both more expensive and more effective, with a mean cost of £68 and a mean QALY gain of 0.042. The incremental cost-effectiveness ratio was £1144 per QALY gained. CONCLUSIONS: This assessment contributes further knowledge, but does not provide definitive answers on how to deliver growth monitoring. In particular, we were unable to ascertain current practice in the UK for growth screening. Further, we were unable to evaluate through the use of identified studies and modelling an optimal referral cut-off and age at which to screen. We identified a number of research questions that would further inform referral strategies, which in summary would involve further primary and secondary data collection. FUNDING: The National Institute for Health Research Health Technology Assessment programme.

Cost-utility of adjuvant high-dose interferon alpha therapy in stage III cutaneous melanoma in Quebec.

OBJECTIVE: To estimate the cost-utility of adjuvant high-dose interferon in high-risk melanoma patients in Quebec compared to a watchful waiting strategy. METHOD: A Markov model was developed that replicates the findings of the pivotal E1684 trial. It was then used to extrapolate survival over a period of 35 years. Costs of medical resources used during the first year were derived through a detailed analysis of a sample (n = 13) of patients treated in a leading academic hospital. Follow-up costs were assessed through a medical decision algorithm. Utilities were derived from a population-based survey (n = 104) in different locations in Quebec using the time trade-off method. RESULTS: The mean incremental cost per quality-adjusted life-year of adjuvant Interferon therapy is equal to 55,090 CAN dollars over a follow-up of 7 years but drops down to 14,003 CAN dollars when extrapolated over 35 years. CONCLUSIONS: Estimates of the cost-effectiveness of high-dose interferon in melanoma patients show an acceptable cost-effectiveness ratio if long-term survival is taken into account. Estimates are, however, strongly influenced by the observed trial differences in survival, the utility associated to health states, and the discount rate.

Economic evaluation of antibiotic prophylaxis in small-cell lung cancer patients receiving chemotherapy: an EORTC double-blind placebo-controlled phase III study (08923).

BACKGROUND: To determine whether the cost of prophylactic antibiotics during chemotherapy is offset by cost savings due to a decreased incidence of febrile leukopenia (FL). PATIENTS AND METHODS: Small-cell lung cancer (SCLC) patients were randomised to standard or intensified chemotherapy with granulocyte colony-stimulating factor to assess the impact on survival (n = 244). In addition, patients were randomised to prophylactic ciprofloxacin and roxithromycin or placebo to assess the impact on FL (n = 161). The economic evaluation examined the costs and effects of patients taking antibiotics versus placebo. Medical resource utilisation was documented prospectively, including 33 patients from one centre in The Netherlands (NL) and 49 patients from one centre in Germany (GE). The evaluation takes the perspective of the health insurance systems and of the hospitals. Sensitivity analyses were performed. RESULTS: In the main trial, prophylactic antibiotics reduced the incidence of FL, hospitalisation due to FL and use of therapeutic antibiotics by 50%. In GE, the incidence of FL was not reduced by prophylaxis. This resulted in an average cost difference of only 35 Euros [95% confidence interval (CI) (-)1.713-2.263] in favour of prophylaxis (not significant). In NL, prophylaxis reduced the incidence of FL by nearly 50%, comparable with the results of the main trial, resulting in a cost difference of 2706 Euros [95% CI 810-5948], demonstrating savings in favour of prophylactic antibiotics of nearly 45%. Sensitivity analyses indicate that with an efficacy of prophylaxis of 50%, and with expected costs of antibiotic prophylaxis of 500 Euros or less, cost savings will incur over a broad range of baseline risks for FL; that is, a risk >10-20% for FL per cycle. CONCLUSIONS: Giving oral prophylactic antibiotics to SCLC patients undergoing chemotherapy is the dominant strategy in both GE and NL, demonstrating both cost-savings and superior efficacy. The sensitivity analyses demonstrate that, due to the efficacy of prophylactic antibiotics and their low unit cost, cost savings will incur over a broad range of baseline risks for FL. We recommend the use of prophylactic antibiotics in patients at risk for FL during chemotherapy.

Health Economics Unit achievements.

This article describes the activities of the EORTC Health Economics Unit since its inception in early 1994. The aim of the unit is to carry out economic evaluations of competing treatment options in common cancers in order to provide health care decision makers with useful information about the relative benefits and costs of the therapies they have to choose between. These assessments are mainly carried out by integrating collection of economic data in selected phase III randomized controlled clinical trials conducted by the EORTC collaborative groups. Studies with an economic evaluation integrated are currently becoming mature enough for analysis and several publications have resulted or are in press. Some studies with data from other sources than EORTC trials have also been performed and published. In addition, the unit has actively followed and contributed to the continuous methodological development in the field of economic evaluation of health care interventions.

The cost-effectiveness of screening for colorectal cancer.

Screening policies for colorectal cancer are costly, as they are to be applied to a large potential population. Cost-effectiveness analysis of potential screening policies is therefore warranted and depends on local circumstances and healthcare systems. Most studies have used modeling approaches, with a few exceptions on the use of fecal occult blood tests. Current conclusions of economic studies tend to favor either double barium contrast enema or sigmoidoscopy as a mass screening tool, although colonoscopy might prove cost-effective in some circumstances. Further research is needed to assess the cost-benefit of mixed strategies in large populations.

Economic evaluation of NMP22 in the management of bladder cancer.

The purpose of this study was to evaluate, from the perspective of the Quebec health system, the cost and cost-efficacy of using NMP22 compared with the currently recommended monitoring procedure following a transurethral resection of a bladder tumor (TURBT). This evaluation was based on results from the study by Soloway, et al. It was performed using a decision analysis technique which compared a follow-up modality using NMP22 with the conventional follow-up monitoring procedure for the first 6 months after an initial transurethral resection of a bladder tumor. Each routine cystoscopy and cytology costs a total of $155. For the 6 months following initial TURBT, the cost for standard follow-up monitoring totaled $311, while the cost for the NMP22 monitoring modality was $257. On average, using NMP22 would have saved $55 per patient during the first 6 months of follow-up, resulting in a cost saving of approximately 18%. As well, 64.3% of patients would have undergone only one cystoscopy during the 6 month period, instead of the two done using the conventional modality. The trade-off for using NMP22 is that some patients would have a 3 month delay before diagnosis of a recurrence. This would occur in 8.9% of patients. NMP22 is less expensive than conventional monitoring follow-up of bladder cancer, and it can decrease patient discomfort by reducing the need for cystoscopy. Implementing it as routine follow-up monitoring should be considered, particularly for patients with low-grade tumors.

Economic comparisons of the pharmacotherapy of depression: an overview.

As many countries find that their health care expenditure is taking up an increasing proportion of their financial resources, economic aspects of care processes have become more important in the choice of optimal strategies. This review of the economic studies of the comparative treatment of depression shows that nearly every aspect of treatment has important economic consequences. Cost-of-illness studies have documented the high burden on society of this disorder, and the associated loss of productivity and work. Comparative cost-effectiveness/utility studies, the majority of which are based on modelling techniques, have consistently shown a better cost-effectiveness ratio of the newer antidepressants over more traditional tricyclic antidepressants (TCAs), when all therapy-related costs are taken into account.

The difference in mean costs as a pharmacoeconomic outcome variable: power considerations.

The difference in mean costs between a placebo and a drug treated group is used for the pharmacoeconomic evaluation of a new drug. The distribution of costs without treatment (null hypothesis) is determined through hospital records. The sampling properties are established by simulation. The results of the simulation allow determination of a break point of differences corresponding to a given level of significance. For power determination, using this break point, clinical hypotheses are used to generate the distribution of costs under treatment (alternative hypothesis).

Pricing and reimbursement of pharmaceuticals in Belgium.

The Belgian healthcare system has a tradition of access and equity at affordable prices. As in other countries, the system becomes pressured by increasing healthcare costs. This paper describes the actual situation in Belgium with special focus on pharmaceutical products and the potential role of pharmacoeconomics in decision making on price and reimbursement. Nearly all people in Belgium are covered by compulsory health insurance. The system is paid for by social security, the patients and the federal and regional authorities. The part of the consumption of pharmaceuticals that is charged to insurance was about 62.1 billion Belgian francs (BeF), i.e. about 50% of the pharmaceutical market in 1994. Price setting in Belgium has been rather low due to the positive reimbursement list, where the price of a new drug is compared to existing drugs in a comparable therapeutic class (so-called reimbursement criteria). The expenditure on pharmaceuticals is increasing faster than global funding for public health. In order to control drug budgets, different cost-containment measures have been or are being taken, i.e. a mix of price, reimbursement and volume controls. These cost-containment measures are not necessarily in accordance with a health economic approach. This paper suggests the scope for better implementation of pharmacoeconomic evaluation, which can lead to more flexible reimbursement systems in specific indications. Therefore, a formal recognition of the role of objective economic evaluations is needed for both hospital and ambulatory care. This process should be proceeded by improving the understanding and robustness of pharmacoeconomic evaluations.

Drug use in relation to clinical activities as an instrument for prospective drug budgeting. The Belgian experience.

In an effort to control escalating health expenditures, especially in hospitals, many countries are planning or experimenting with prospective budgeting systems. Belgium is no exception and has recently introduced, with some success, limited fixed charges per hospital admission and/or per hospitalisation day for laboratory tests and radiographic investigations. More recently, the focus has shifted to hospital drug expenditures, which have shown high growth rates over the past few years. Until now, such expenditures have been reimbursed on a fee-for-service system, often with limited out-of-pocket charges for hospitalised patients. In order to curb the growth of drug expenditures, it is appropriate to investigate whether the financing of hospital drugs through a prospective budgeting system could be a feasible solution. Therefore, we constructed a database of over 270 000 admissions from a sample of 23 Belgian general and teaching (university) hospitals for the year 1991. Data were obtained from the official Minimum Basic Data Set or Résumé Clinique Minimum, which contains summarised clinical and administrative information, plus detailed expenditures (including medications) for each hospital stay. This information allowed us to categorize each stay into an appropriate diagnosis-related group (DRG). Our first descriptive analysis identified a number of major variables that influenced patients' drug expenditures: all-patient DRG (APDRG), age, disease severity, length of stay in an intensive care unit, emergency admission, death during hospitalisation, and hospital type (teaching or general). A covariance analysis was then performed on all hospital stays combined, and separately on surgical and medical stays. The results indicated that these variables taken together account for between 56.5 and 76.3% of drug expenditures in medical and surgical stays, respectively, with the major variance explained by differences in APDRG category. However, when the data were disaggregated according to major diagnosis category, a large degree of heterogeneity in the explained variance was observed. In patients with drug use- and alcohol-related disorders, 5.2% of drug billings/expenditures were attributable to the APDRG, and the corresponding figure in patients undergoing circulatory system surgery was 84%. This means that, if DRGs are used to define a global prospective drug budget for a hospital, using the hospital's historical case mix as a weighting factor, we should pay particular attention to the hospital profile because the predictive power of such a system could be relatively low in some hospitals. Consequently, we need to construct larger confidence intervals for hospitals in which historical drug expenditures have low predictive power, or search for additional explanatory variables for expenditures in these hospitals.