Anticoagulants d'action directe: une revue de la littérature des études coût/efficacité en Europe

RésuméContexteLes résultats des études coût/efficacité des innovations thérapeutiques sont devenus un critère de référence dans la plupart des pays développés. Lorsque de nouveaux traitements proposent des alternatives à des traitements de référence éprouvés mais très peu coûteux, les payeurs anticipent un impact budgétaire important et cherchent à connaître la valeur supplémentaire apportée par ceux-ci.ObjectifL'objectif de cette étude a été de présenter les différents modèles élaborés autour des trois premiers anticoagulants oraux directs (AOD), le dabigatran, le rivaroxaban et l'apixaban, ainsi que leurs résultats dans l'indication de prévention des accidents vasculaires cérébraux (AVC) pour les patients souffrant de fibrillation atriale (FA). Le périmètre de l'étude a été limité aux pays européens et au Canada, tous pays disposant d'un système de couverture universelle des dépenses de soins.MéthodeÀ partir d'une revue de la littérature, les caractéristiques générales des études et leurs principaux résultats ont été présentés et comparés.RésultatsDix-neuf études ont été sélectionnées, couvrant onze pays européens et le Canada, comparant chacune des molécules aux antivitamines K (AVK) et entre elles. Toutes les études ont calculé un ratio de coût par QALY. La majorité des résultats (34/46) se situe au-dessous de 20 000 € par Quality-Adjusted Life Year (QALY). L'apixaban et le dabigatran à la dose de 150 mg bid présentaient les ratios les plus favorables.ConclusionLes nouveaux traitements présentent des ratios coût/efficacité acceptables en Europe et au Canada, au regard des standards habituels. Au-delà des différences intrinsèques entre les différents systèmes de santé, la diversité des résultats témoigne cependant d'une nécessaire standardisation des études à des fins de comparabilité.AbstractBackgroundThe cost-effectiveness analysis of therapeutic innovations has become a reference for decision makers in developed countries. When new treatments are available as alternatives to existing well established and cheap treatments, payers anticipate a major budget impact and will assess the extra benefit for society for extra Euros spent.AimsThis study aimed at presenting the different published results of cost-effectiveness analyses performed for the three first new oral anticoagulants, dabigatran, rivaroxaban and apixaban, for the prevention of strokes for patients with atrial fibrillation. The study covered European countries and Canada, which all propose universal coverage for healthcare expenditures.MethodsA literature review was performed. The general characteristics and main results of selected studies were presented and compared.ResultsNineteen studies were selected, covering 11 European countries and Canada. All studies have performed the estimation of a cost per QALY. The majority of the results (34/46) were under €20,000 per QALY. Apixaban and dabigatran 150mg bid presented with the most favourable results.ConclusionNew oral anticoagulants appear to have an acceptable cost-effectiveness ratio for European countries and Canada considering usual standards. Nevertheless, beyond intrinsic differences between healthcare systems, the observed variability of results strongly suggest a need for a standardisation of models used across countries

Faciliating More Efficient Negotiations for Innovative Therapies: A Value-Based Negotiation Framework

OBJECTIVES: An increasing number of innovative therapies (e.g., gene- and cell-based treatments) have been developed in the past 20 years. Despite the significant clinical potential of these therapies, access delays may arise because of differing perspectives of manufacturers and payers regarding issues such as the value of the product, clinical and financial uncertainties, and sustainability.Managed entry agreements (MEAs) can enable access to treatments that would not be reimbursed by conventional methods because of such concerns. However, although MEA typologies exist, there is currently no structured process to come to agreements on MEAs, which can be difficult to decide upon and implement.To facilitate more structured MEA negotiations, we propose a conceptual "value-based negotiation framework" with corresponding application tools. METHODS: The framework was developed based on an iterative process of scientific literature review and expert input. RESULTS: The framework aims to (i) systematically identify and prioritize manufacturer and payer concerns about a new treatment, and (ii) select a mutually acceptable combination of MEA terms that can best address priority concerns, with the lowest possible implementation burden. CONCLUSIONS: The proposed framework will be tested in practice, and is a step toward supporting payers and manufacturers to engage in more structured, transparent negotiations to balance the needs of both sides, and enabling quicker, more transparent MEA negotiations and patient access to innovative products

Socioeconomic and geographic determinants of survival of patients with digestive cancer in France

Using a multilevel Cox model, the association between socioeconomic and geographical aggregate variables and survival was investigated in 81 268 patients with digestive tract cancer diagnosed in the years 1980–1997 and registered in 12 registries in the French Network of Cancer Registries. This association differed according to cancer site: it was clear for colon (relative risk (RR)=1.10 (1.04–1.16), 1.10 (1.04–1.16) and 1.14 (1.05–1.23), respectively, for distances to nearest reference cancer care centre between 10 and 30, 30 and 50 and more than 90 km, in comparison with distance of less than 10 km; P-trend=0.003) and rectal cancer (RR=1.09 (1.03–1.15), RR=1.08 (1.02–1.14) and RR=1.12 (1.05–1.19), respectively, for distances between 10 and 30 km, 30 and 50 km and 50 and 70 km, P-trend=0.024) (n=28 010 and n=18 080, respectively) but was not significant for gall bladder and biliary tract cancer (n=2893) or small intestine cancer (n=1038). Even though the influence of socioeconomic status on prognosis is modest compared to clinical prognostic factors such as histology or stage at diagnosis, socioeconomic deprivation and distance to nearest cancer centre need to be considered as potential survival predictors in digestive tract cancer

Market access agreements for pharmaceuticals in Europe: diversity of approaches and underlying concepts

<p>Abstract</p> <p>Background</p> <p>Market Access Agreements (MAA) between pharmaceutical industry and health care payers have been proliferating in Europe in the last years. MAA can be simple discounts from the list price or very sophisticated schemes with inarguably high administrative burden.</p> <p>Discussion</p> <p>We distinguished and defined from the health care payer perspective three kinds of MAA: Commercial Agreements (CA), Payment for Performance Agreements (P4P) and Coverage with Evidence Development (CED). Apart from CA, the agreements assumed collection and analysis of real-life health outcomes data, either from a cohort of patients (CED) or on per patient basis (P4P). We argue that while P4P aim at reducing drug cost to payers without a systematic approach to addressing uncertainty about drugs' value, CED were implemented provisionally to reduce payer's uncertainty about value of a medicine within a defined time period.</p> <p>Summary</p> <p>We are of opinion that while CA and P4P have a potential to reduce payers' expenditure on costly drugs while maintaining a high list price, CED address initial uncertainty related to assessing the real-life value of new drugs and enable a final HTA recommendation or reimbursement and pricing decisions. Further, we suggest that real cost to health care payers of drugs in CA and P4P should be made publicly available in a systematic manner, to avoid a perverse impact of these MAA types on the international reference pricing system.</p

Measuring and explaining mortality in Dutch hospitals; The Hospital Standardized Mortality Rate between 2003 and 2005

Background. Indicators of hospital quality, such as hospital standardized mortality ratios (HSMR), have been used increasingly to assess and improve hospital quality. Our aim has been to describe and explain variation in new HSMRs for the Netherlands. Methods. HSMRs were estimated using data from the complete population of discharged patients during 2003 to 2005. We used binary logistic regression to indirectly standardize for differences in case-mix. Out of a total of 101 hospitals 89 hospitals remained in our explanatory analysis. In this analysis we explored the association between HSMRs and determinants that can and cannot be influenced by hospitals. For this analysis we used a two-level hierarchical linear regression model to explain variation in yearly HSMRs. Results. The average HSMR decreased yearly with more than eight

Challenging the holy grail of hospital accreditation: A cross sectional study of inpatient satisfaction in the field of cardiology

Extent: 7p.Background: Subjective parameters such as quality of life or patient satisfaction gain importance as outcome parameters and benchmarks in health care. In many countries hospitals are now undergoing accreditation as mandatory or voluntary measures. It is believed but unproven that accreditations positively influence quality of care and patient satisfaction. The present study aims to assess in a defined specialty (cardiology) the relationship between patient satisfaction (as measured by the recommendation rate) and accreditation status. Methods: Consecutive patients discharged from 25 cardiology units received a validated patient satisfaction questionnaire. Data from 3,037 patients (response rate > 55%) became available for analysis. Recommendation rate was used as primary endpoint. Different control variables such as staffing level were considered. Results: The 15 accredited units did not differ significantly from the 10 non-accredited units regarding main hospital (i.e. staffing levels, no. of beds) and patient (age, gender) characteristics. The primary endpoint "recommendation rate of a given hospital" for accredited hospitals (65.6%, 95% Confidence Interval (CI) 63.4 - 67.8%) and hospitals without accreditation (65.8%, 95% CI 63.1 - 68.5%) was not significantly different. Conclusion: Our results support the notion that - at least in the field of cardiology - successful accreditation is not linked with measurable better quality of care as perceived by the patient and reflected by the recommendation rate of a given institution. Hospital accreditation may represent a step towards quality management, but does not seem to improve overall patient satisfaction.Cornelia Sack, Peter Lütkes, Wolfram Günther, Raimund Erbel, Karl-Heinz Jöckel and Gerald J Holtman

Sustainable Financing of Innovative Therapies: A Review of Approaches

The process of innovation is inherently complex, and it occurs within an even more complex institutional environment characterized by incomplete information, market power, and externalities. There are therefore different competing approaches to supporting and financing innovation in medical technologies, which bring their own advantages and disadvantages. This article reviews value- and cost-based pricing, as well direct government funding, and cross-cutting institutional structures. It argues that performance-based risk-sharing agreements are likely to have little effect on the sustainability of financing; that there is a role for cost-based pricing models in some situations; and that the push towards longer exclusivity periods is likely contrary to the interests of industry