Ariel Beresniak

Limitations of the methods used for calculating quality-adjusted life-year values.

AbstractObjective: To test the validity of the techniques used to calculate quality-adjusted life-year (QALY) values based on utility functions, using a real population dataset. Design: Using the standard gamble technique, we gathered the preferences of a population sample of 189 individuals on a combination of probabilities concerning four simple health states (no physical disability, limp, walk with crutches and need a wheelchair), and three life-year spans (5, 10 and 15 years). Each of the four assumptions of the multiplicative model was tested based on the results of the experiment. Results: The utility of the health state ‘limp’ was high at 0.89 and that of ‘walk with crutches’ only slightly lower at 0.85. However, of the 189 individuals, only 57 are not in contradiction with the assumption of mutual utility independence since they strictly preferred (15 years, ‘need a wheelchair’) over (10 years, ‘need a wheelchair’) and (15 years, ‘need a wheelchair’) over (5 years, ‘need a wheelchair’). For these 57 individuals, the results of this study do not fit the assumptions underpinning the multiplicative model. Conclusion: This work suggests that the techniques used as a basis from which to calculate QALY values are flawed. In particular, the underlying assumptions of the multiattribute utility model do not correspond to behaviour patterns observed in a real population. It therefore appears that use of the QALY technique should be questioned in healthcare decision-making settings.

Relationships between black tea consumption and key health indicators in the world: an ecological study

Objectives The aim of this study was to investigate potential statistical relationships between black tea consumption and key health indicators in the world. The research question is: Does tea consumption is correlated with one or more epidemiological indicators? Design Ecological study using a systematic data-mining approach in which the unit of the analysis is a population of one country. Setting Six variables, black tea consumption data and prevalence data of respiratory diseases, infectious diseases, cancer, cardiovascular diseases and diabetes, have been studied at a global level. Participants Data from 50 participating countries in the World Health Survey were investigated. Primary and secondary outcomes measures Level of statistical relationships between variables. Results Principal component analysis established a very high contribution of the black tea consumption parameter on the third axis (81%). The correlation circle confirmed that the ‘black tea’ vector was negatively correlated with the diabetes vector and was not correlated with any of the other four health indicators. A linear correlation model then confirmed a significant statistical correlation between high black tea consumption and low diabetes prevalence. Conclusions This innovative study establishes a linear statistical correlation between high black tea consumption and low diabetes prevalence in the world. These results are consistent with biological and physiological studies conducted on the effect of black tea on diabetes and confirm the results of a previous ecological study in Europe. Further epidemiological research and randomised studies are necessary to investigate the causality.

Direct Cost-Modeling of Rheumatoid Arthritis According to Disease Activity Categories in France

Objective. The objective of this cost-of-illness study was to assess the use of direct medical resources, excluding drug costs, by patients with rheumatoid arthritis (RA) in France, and to construct cost estimates according to level of disease activity. Methods. Three categories of RA disease activity were defined according to Disease Activity Score 28-joint count (DAS28) thresholds: remission (DAS28 < 2.6), low disease activity state (LDAS; i.e., DAS28 ≤ 3.2), and moderate to high disease activity (MHDAS; i.e., DAS28 > 3.2). Eight resource utilization items were defined: medical visits, laboratory tests, hospitalization, imaging, physiotherapy, nursing, adaptive aids, and transportation. Resource utilization and unit costs from the national-payer perspective were estimated through expert opinion and simulated using distribution ranges for each item. Cost distributions were computed by Monte-Carlo simulations estimating overall costs per 6 months over a 2-year period. Results. For patients achieving remission, costs were estimated at a mean of €771 (SD 199) for the first 6 months and at €511 (SD 162) for each subsequent 6-month period. For patients achieving LDAS, costs were estimated at €905 (SD 263) for the first 6 months and €696 (SD 240) for each subsequent 6-month period. For patients in MHDAS, costs were estimated at €1215 per 6 months (SD 405). Conclusion. This cost-of-illness assessment provided current estimates of direct medical costs for RA according to disease activity in France. The findings suggest that achieving remission or LDAS is associated with substantially lower medical costs for RA versus being in MHDAS.

Cost-effectiveness of recombinant versus urinary follicle-stimulating hormone in assisted reproduction techniques in the Spanish Public Health Care System

AbstractPurpose: To evaluate the relative cost-effectiveness of recombinant and urinary follicle-stimulating hormone (FSH) in assisted reproduction techniques in the Spanish National Health Service. Methods: Markov modelling was used to compare costs and outcomes of three complete treatment cycles using recombinant or urinary FSH for controlled ovarian stimulation. Cost and effectiveness estimates were obtained from the literature and from Spanish clinicians. A Monte Carlo technique was used to randomise the distribution of outcomes at each stage. The analysis was performed by passing a virtual population of 100,000 patients through the computer simulation in each of 5000 Monte Carlo simulations. Results: The cost per pregnancy was €12,791 ± 1202 (

Equity, Access and Economic Evaluation in Rare Diseases. The Impact of Orphan Drug Legislation on Health Policy and Patient Care

11,346 ± 1066) with recombinant and €13,007 ± 1319 (

Assessing the Financial Impact of Reusing Electronic Health Records Data for Clinical Research: Results from the EHR4CR European Project

11,537 ± 1170) with urinary FSH (p < 0.0001). The mean number of cycles per pregnancy was 4.69 and 5.21, respectively. Conclusions: Recombinant FSH is more cost-effective than urinary FSH in the Spanish public health care system.

Is there an alternative to quality-adjusted life years for supporting healthcare decision making?

Currently, there are in excess of 5000 rare diseases, that is, diseases that affect only a small proportion of a given population. The majority of these conditions lack appropriate treatments for a variety of reasons, including limited markets for the development of new pharmaceutical products coupled with prohibitively high costs associated with research, manufacturing and marketing. This paper discusses the issues associated with the equity of and access to orphan drugs used in rare diseases. Options for healthcare policy across Europe are presented. The interest in using robust evaluation parameters is emphasized, despite this not being in agreement with the use of artificial indicators such as quality-adjusted life years (QALYs), which appear to lead to divergent and erroneous results. New decision-making methods must govern the policy, statistics, and economics relevant to the use of orphan drugs in rare diseases.

Cost effectiveness modeling comparing recombinant FSH with urinary FSH for ovarian stimulation: a multinational evaluation

Background: The new technological platform developed by the Electronic Health Records for Clinical Research (EHR4CR) European research project (2011-2016) has been specially designed to enable the trustworthy reuse of health data contained in hospital-based electronic health records (EHR) for enhancing and speeding up clinical research scenarios. In particular, protocol feasibility assessments, patient identification for recruitment, and clinical data exchange for study conduct, in accordance with data privacy, ethical and legal requirements. The objective of our study was to assess the financial impact of adopting these advanced solutions compared to current practices, from the perspective of the primary sponsors of clinical trials in Europe. Methods: Considering a scalable implementation of EHR4CR solutions in up to 5-10% of Phase II, III and IV clinical trials to be commercially sponsored in Europe over 5 years, two potential market sizes were defined. The first has a European initial scope (i.e., for European clinical trials only), and the second has a European subsequent broader scope (also including European arms of global studies). Based on expert opinions, the EHR4CR initial scope target market was estimated to be 30% of the broader scope. Direct costs to clinical research sponsors were estimated under current practices, and with the EHR4CR platform. Uncertainty was managed using 100,000 Monte Carlo simulations. Results: Compared to current practices, the potential average 5-year savings with EHR4CR solutions for Phase II, III and IV commercially sponsored clinical trials in Europe were estimated at €175.5 m for the European initial scope market, and at €585.3 m for the European broader scope market. These results were confirmed by robust probabilistic sensitivity analyses. Conclusions: Compared to current practices, EHR4CR solutions appear cost-saving for primary sponsors of clinical trials. These results suggest that the potential for savings would increase with a broader adoption of EHR4CR solutions in Europe, and beyond.

Cost-effectiveness modelling of recombinant FSH versus urinary FSH in assisted reproduction techniques in the UK

ABSTRACT Introduction: Over the years, a number of criticisms have been raised about the robustness of the Quality-Adjusted Life Years (QALY) indicator and its use in cost-utility analyses; however, costs/QALY are still nowadays recommended as reference case by several Health Technology Assessment (HTA) agencies from some Commonwealth countries, such as the National Institute for Health and Care Excellence (NICE) in the UK, claiming that no alternatives exist to allocate health care resources. Areas covered: This review presents a selection of robust alternative methodologies that could be used to support HTA decisions more accurately and more fairly than using the QALYs, including for determining the level of patient access and reimbursement coverage to healthcare interventions. Expert commentary: Because of the scientific complexity of the situations raised by existing and innovative health technologies and interventions, there is currently no single alternative paradigm to propose at this time, but a spectrum of additional analytical techniques which could handle various outcomes including costs and health consequences, and which are not based on a simple multiplicative formula.

Analysis of the cost effectiveness of recombinant versus urinary follicle-stimulating hormone in in vitro fertilization/intracytoplasmic sperm injection programs in the United States.

Introduction: The focus on cost-containment imposed by shrinking national health care budgets is increasingly putting pressure on investigators to demonstrate that newer interventions are not only effective but also cost-effective. In the area of assisted reproductive technology (ART) a shift in usage is occurring from urinary derived to biotechnology – derived (recombinant) gonadotropins for ovarian stimulation. Provision of treatment with ART often involves several cycles of treatment, each associated with multiple steps with varying outcomes at each step. To take into account all the situations that are possible during the repeated cycles of treatment when trying to evaluate different ovarian stimulation regimens is a complex task that requires large numbers of subjects to adequately address the cost-effectiveness of these therapies. A more efficient approach is to employ modeling techniques that can easily be applied to the different national health care systems. The objective of this study was to compare the cost-effectiveness of rec ombinant(r) FSH with urinary(u) FSH in the UK, USA, Germany and Spain.