bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP118 Cost-Effectiveness Analysis Of Molecular Profile Selection For Advanced Head And Neck Cancer
Authors: Rognoni Carla , Bossi Paolo , Licitra Lisa , Quaglini Silvana 
Published: January 2018

INTRODUCTION:Relapsed/metastatic head and neck squamous cell cancer patients are offered a combination of platinum-based chemotherapy (PF, cisplatin-fluorouracil) plus cetuximab regimen (PF+C) according to results of the EXTREME trial (1). However, two economic evaluations showed that addition of cetuximab was not cost-effective.This study aimed to evaluate the cost-effectiveness of a putative predictive molecular test (MT) to identify and treat only patients potentially responsive to cetuximab when added to PF.METHODS:A Markov model was developed to compare both health and economic outcomes of PF+C regimen administered to all patients (PF+C ALL) versus the regimen administered only to MT-positive patients (PF+C POS).The model considered the following health states: partial/complete response with/out mild/severe adverse events (AEs), progression and death. Rates of progression and survival, response rates to systemic treatment and adverse events were retrieved from the EXTREME trial (1). According to Mesía et al. (2), we assumed that addition of cetuximab to PF would not negatively affect life quality compared to PF alone, and the baseline utility coefficients for disease control and progression were assumed as .67 and .52, respectively.Only direct costs estimated from the Italian Health Service perspective were included (tariffs and Diagnosis Related Group - DRG - reimbursements).The model was evaluated according to a cut-off of sensitivity at 85 percent and specificity at 70 percent. A 3 years horizon was chosen. Life expectancy, quality-adjusted life years (QALYs) and costs were discounted at 3.5 percent annually.RESULTS:Applying the World Health Organization (WHO) cost-effectiveness threshold of 3 times the gross domestic product for Italy (EUR66,402), PF+C POS resulted a cost-effective choice in comparison to PF+C ALL for a MT cost lower than EUR5,750.CONCLUSIONS:Adding cetuximab to PF only to patients positive to a predictive test may be cost-effective. Efforts should be spent to build such a test upon existing evidences in order to save resources for the health systems and spare unnecessary toxicities to patients.
OP132 How A Shared Management Of Home Infusion Can Control Expenditure
Authors: Olivier Cyril , Collingnon Corinne , Fouteau Emmanuelle , Emery Gregory , Belghiti Jacques 
Published: January 2018

INTRODUCTION:In France, medical devices (MDs) for home-based infusion used to be covered by the health insurance system if included on the list of products and services qualifying for reimbursement under a generic description corresponding to a class of products with the same indications. This coverage modality offered no resistance to unnecessary or wasteful spending. Besides, between 2010 and 2015 the expenditure related to these MDs have increased from EUR192million to EUR289million (+50 percent).METHODS:The French National Authority for Health (HAS) has assessed the actual benefit of these MDs which have the same indications as the drugs infused at home. This work led to standardize the infusion types (gravity, elastomeric pump or active system requiring an energy source) and the quantities of MDs needed to carry out the different cares (installation, connection, withdrawal) according to the infusion route. At this step, considering that the priority was to redefine the MDs required at home for each care type, no economical assessment had been conducted.RESULTS:Based on this medical assessment, the Ministry of Health has distinguished three types of infusion and three types of services (home installation, monitoring and consumables) since 2016. In total, twenty-four packages have been set up for reimbursement with non-cumulative rules. Doctors are in charge to prescribe the appropriate packages; providers and nurses determine together the optimal devices needed for each patient according to his environment.CONCLUSIONS:These HAS recommendations on practice standardization have been the keystone for cost negotiations. The new coverage modalities aim to motivate liberal nurses to choose the best fitted products and providers to deliver the right quantities to patients. The expected benefits are an adjusted evaluation of the necessary equipment and a control of health expenditure due to the fixed costs of each infusion package.
OP135 Confirmatory Versus Explorative Endpoints In Drug Approval Versus Health Technology Assessment
Authors: Niehaus Ines , Dintsios Charalabos-Markos 
Published: January 2018

INTRODUCTION:The early benefit assessment of drugs in Germany and their preceded market authorization pursue different objectives, resulting in divergent decision-making strategies. This is reflected inter alia by the diverse inclusion of confirmatory endpoints within the assessments of oncological drugs. The pharmaceutical manufacturers are facing the challenge of meeting the requirements for both evaluation processes by the available evidence and avoiding hereby negative early benefit assessments. This is mainly due to the concept of mutually relevant clinical trials.METHODS:Identification and gathering of the endpoints is based on a specifically developed guide. The extracted data from the documents of completed assessments up to July 2015 are used to estimate both separately and together the impact of explorative in relation to confirmatory endpoints on the drug approval and early benefit assessment, by contrasting the European Medicines Agency's risk-benefit-ratio and the benefit-harm-balancing of the national Health Technology Assessment (HTA) jurisdiction.RESULTS:Twenty-one of fourty-one studies’ oncological assessments could be included in the endpoint analysis. From a procedural point of view both the drug approval and the early benefit assessment seem to be not confirmatory since they include explorative endpoints as well. Yet, drug approval is in terms of quality of endpoints more confirmatory than early benefit assessment since it contains a higher proportion of primary endpoints. The latter implies only in 67 percent of the assessments a primary endpoint to be relevant for the benefit-harm-balancing. Moreover, explorative mortality endpoints reached the highest agreement and explorative endpoints capturing health-related quality of life no agreement, referring to the mutual relevance of endpoints for the risk-benefit-ratio and the benefit-harm-balancing.CONCLUSIONS:The missing information transparency of the assessment reports compared to the information offered within the early benefit assessment makes an assignment of endpoints with respect to the mutually relevant clinical trial sometimes troublesome. To warrant, in the long run, a broader confirmatory basis for decisions in health care supported by HTA, a closer inter-institutional cooperation of approval authorities and German HTA jurisdictions seems favorable.
PP015 Methodological Quality Of Health Technology Assessment Reports
Authors: Hausner Elke , Knelangen Marco , Sanders Laura , Waffenschmidt Siw 
Published: January 2018

INTRODUCTION:Health Technology Assessment (HTA) reports may have a major impact on the health care provided in a country. Hence, one would assume that these reports have a high methodological quality and thus represent a potentially important source of information, for instance, for identifying primary studies for inclusion in the evidence syntheses (for example, systematic reviews, Cochrane reviews, HTA reports). The aim of the present analysis is to evaluate the methodological quality of HTA reports used as a literature source for HTA reports produced by the German Institute for Quality and Efficiency in Health Care (IQWiG).METHODS:Eligible IQWiG reports were assessments of drug or non-drug interventions considering HTA reports as the literature source for primary studies and published up to October 2016. An HTA report included in the IQWIG report was considered in the analysis if it was a complete report published in English or German and indexed in the Health Technology Assessment Database (Wiley) or MEDLINE. Only the most current HTA report in an IQWiG report was considered; if more than one current HTA report was available, the one for inclusion in the analysis was randomly selected. The methodological quality of the HTA reports identified was evaluated with the AMSTAR (“Assessment of Multiple Systematic Reviews”) tool (1), which comprises 11 items on methodological quality (meaning a maximum achievable score of 11).RESULTS:A total of fifty eligible IQWiG reports using fourty-one eligible HTA reports as literature sources were identified. The mean AMSTAR score of these HTA reports was 5.3 (95 percent Confidence Interval, CI: 4.3, 6.2). None of the HTA reports achieved a score of 11, nineteen (46 percent) had a score between 6 and 10, and twenty-two had a score below 6.CONCLUSIONS:HTA reports included in IQWiG reports only have an average methodological quality.
PP017 Social Cost Benefit Analysis Of Cognitive Behavioral Therapy For Alcohol And Cannabis Addiction
Authors: van Gils Paul , Over Eelco , Suijkerbuijk Anita , Lokkerbol Joran , de Wit Ardine 
Published: January 2018

INTRODUCTION:Due to their chronic nature and high prevalence, alcohol and cannabis addiction leads to a significant (disease) burden and high costs, both for those involved and for society. The latter includes effects on health care, quality of life, employment, criminality, education, social security, violence in the public and private domain, and traffic accidents. In the Netherlands, a considerable number of people with an alcohol or cannabis addiction currently do not receive addiction care. Cognitive Behavioral Therapy (CBT) is effective as a treatment for both alcohol and cannabis addiction and is widely used in specialized addiction care centers. This social cost-benefit analysis (SCBA) models costs and benefits of increasing the uptake of CBT for persons with an alcohol addiction and for adolescents with a cannabis addiction, taking into account a wide range of social costs and effects (1).METHODS:The method follows general Dutch guidance for performing SCBA. A literature search was conducted to evaluate efficacy of CBT for alcohol and cannabis dependence. In addition, the social costs of alcohol and cannabis addiction for society were mapped, and the costs of enhancing the uptake of CBT were explored. Costs and benefits of increased uptake of CBT for different social domains were modeled for a ten year period, and compared with current (unchanged) uptake during this period. Compliance problems (about 50 percent of clients do not finish CBT) and fall-back to addiction behavior (decrease of effects of CBT over time) were taken into account in model estimations.RESULTS:Per client treated with CBT, the estimated benefits to society are EUR10,000-14,000 and EUR9,700-13,000, for alcohol and cannabis addiction, respectively. These benefits result from reduced morbidity and mortality, improved quality of life, higher productivity, fewer traffic accidents, and fewer criminal activities.CONCLUSIONS:This SCBA shows that not only treated clients but also society will benefit from an increase in people treated with CBT in specialized addiction care centers.
PP021 Peer Review Innovations For Grant Applications: Efficient And Effective?
Authors: Frampton Geoff , Shepherd Jonathan , Pickett Karen , Wyatt Jeremy 
Published: January 2018

INTRODUCTION:Peer review of grant applications is employed routinely by health research funding bodies to determine which research proposals should be funded. Peer review faces a number of criticisms, however, especially that it is time consuming, financially expensive, and may not select the best proposals. Various modifications to peer review have been examined in research studies but these have not been systematically reviewed to guide Health Technology Assessment (HTA) funding agencies.METHODS:We developed a systematic map based on a logic model to summarize the characteristics of empirical studies that have investigated peer review of health research grant applications. Consultation with stakeholders from a major health research funder (the United Kingdom National Institute for Health Research, NIHR) helped to identify topic areas within the map of particular interest. Innovations that could improve the efficiency and/or effectiveness of peer review were agreed as being a priority for more detailed analysis. Studies of these innovations were identified using pre-specified eligibility criteria and were subjected to a full systematic review.RESULTS:The systematic map includes eighty-one studies, most published since 2005, indicating an increasing area of investigation. Studies were mostly observational and retrospective in design, and a large proportion have been conducted in the United States, with many conducted by the National Institutes of Health. An example of an innovation is video training to improve reviewer reliability. Although research councils in the United Kingdom have conducted several relevant studies, these have mainly examined existing practices rather than testing peer review innovations. Full results of the systematic review will be provided in the presentation, and we will assess which innovations could improve the efficiency and/or effectiveness of peer review for selecting health research proposals.CONCLUSIONS:Despite considerable interest in, and criticism of, peer review for helping to select health research proposals, there have been few detailed systematic examinations of the primary research evidence in this area. Our evidence synthesis provides the most up-to-date overview of evidence in this important developing area, with recommendations for health research funders in their decision making.
PP022 New Models Are Needed To Optimize The Management Of New Medicines
Authors: Godman Brian , Diogene Eduardo , Fürst Jurij , Garuoliene Kristina , Guerra Augusto , Joppi Roberta , Malmström Rickard , Oortwijn Wija , Selke Gisbert , Timoney Angela , Wettermark Björn 
Published: January 2018

INTRODUCTION:Countries are struggling to fund new premium priced medicines with ever increasing prices. In addition, there are substantial savings as medicines lose their patents. This requires coordinated approaches. Models are being developed centering on three pillars: pre-launch including horizon scanning; peri-launch including pricing and reimbursement (P & R)/ risk sharing; and post-launch including assessing effectiveness (1,2). This will continue to enable access to safe, effective and affordable medicines.METHODS:Desk research of regulatory and other relevant policy documents as well as a thorough and extensive literature search in peer-reviewed databases were conducted.RESULTS:Models to optimize the use of new medicines are being developed. These center on three pillars: pre-launch activities including horizon scanning with a specific focus on unmet needs, drugs expected place in therapy, drugs preliminary budget impact and forecasting (including medicines likely to lose their patents); peri-launch activities including P & R assessment and assessments of risk sharing arrangements; and post-launch activities include assessing the effectiveness and safety of new medicines in routine clinical care (1,2). Pre-launch activities to agree the number of potential patients for new cancer medicines resulted in hospitals staying within budget (3); and health authorities that had instigated activities pre-launch saw limited excess bleeding with dabigatran (3). Risk-sharing arrangements have increased access to new medicines; however, concerns with their confidential nature and administrative burden (2,3). Qualitative and/or quantitative approaches are also being developed to better value (new) medicines. There is also growing use of patient level data post launch, for example, studies highlighted concerns with dabigatran prescribing in Spain and anti-obesity medicines in Sweden. Long-term follow-up studies have shown greater effectiveness of ciclosporin versus tacrolimus for transplants despite the rhetoric.CONCLUSIONS:Stakeholders in the healthcare field are working together and developing methods to increase funding for new valued medicines whilst restricting their use where there are concerns to optimize resource use. This will (need to) continue to enable access to safe, (cost-) effective and affordable medicines.
PP024 Changes In Reporting Characteristics Of Systematic Reviews For The United Kingdom
Authors: Kaltenthaler Eva , Carroll Christopher 
Published: January 2018

INTRODUCTION:A recent publication reported that increasing numbers of systematic reviews are being published and, although standards have improved, many are still poorly conducted and reported, especially non-Cochrane systematic reviews (1). The aim of this study was to assess the quality of the conduct and reporting of systematic reviews undertaken for the United Kingdom (UK) Health Technology Assessment (HTA) programme and published in the International Journal of Technology Assessment in Health Care (2) and compare those undertaken in 2004 and 2014.METHODS:A comparative sample of all systematic reviews published in 2004 and 2014 in the UK HTA monograph series was identified by a structured search of MEDLINE in August 2016. After piloting of the form, two reviewers each extracted relevant data. These data were tabulated and summarized.RESULTS:The search identified twenty-three systematic reviews from 2004 and thirty from 2014. By 2014, compared with 2004, a smaller proportion of treatment (53 percent versus 70 percent) and pharmaceutical (20 percent versus 57 percent) reviews were being published. In 2014, there were much higher percentages of review registrations (70 percent versus 0 percent) and available protocols (90 percent versus 17 percent); increased explicit inclusion of unpublished literature (65 percent versus 39 percent); less frequent use of local checklists (32 percent versus 61 percent) for critical appraisal; more complete reporting of study flow for inclusion (97 percent versus 57 percent) and exclusion (91 percent and 65 percent) of studies; and there were more reviews reporting limitations affecting the review itself (73 percent versus 49 percent). The process had clearly become more reflective and rigorous. However, some previous weaknesses persisted, including the general absence of any assessment of publication bias and the failure to report overall numbers of patients in the review.CONCLUSIONS:Marked improvements can be seen in the conduct and reporting of systematic reviews published by the UK HTA programme as a result of the publication and general acceptance of the PRISMA statement (3) and the increased application of a smaller number of relevant standards.
PP028 Hyperhidrosis Quality Of Life Measures: Review And Patient Perspective
Authors: Jones-Diette Julie , Wade Ros , Wright Kath , Llewellyn Alexis , Rice Stephen , Moloney Eoin , Stoniute Julija , Layton Alison , Levell Nick , Stansby Gerard , Craig Dawn , Woolacott Nerys 
Published: January 2018

INTRODUCTION:Primary hyperhidrosis has no discernible cause and is characterised by uncontrollable excessive and unpredictable sweating, which occurs at rest, regardless of temperature. The symptoms of hyperhidrosis can significantly affect quality of life, and can lead to social embarrassment, loneliness, anxiety and depression.The aim of this literature review was to identify the tools used to measure quality of life in studies of hyperhidrosis. Patient advisors provided insight and their perspective.METHODS:Studies were identified through searches undertaken in January 2016. The search strategies combined topic terms for hyperhidrosis with a recognised search filter for “quality of life”. All studies that reported measuring quality of life or described a quality of life measure/tool in the context of primary hyperhidrosis were included. The information on the tools and their use in hyperhidrosis was summarized in a narrative synthesis. Patient advisors contributed to the interpretation of the findings.RESULTS:The review included 184 studies and many studies used multiple tools. Twenty-two individual tools were identified. The review identified disease specific, dermatology specific, and general health/utility tools. The most commonly identified tools were the Dermatology Life Quality Index (DLQI), the Hyperhidrosis Disease Severity Scale (HDSS), and the Hyperhidrosis Quality of Life Questionnaire (HQLQ). The Hyperhidrosis Quality of Life index (HidroQoL©) is recently designed and validated, and therefore was used only in its validation study.When asked about these four quality of life tools patient advisors agreed that the HidroQoL© tool covered disease-specific quality of life dimensions relevant to them most comprehensively and was easy to complete. The DLQI was considered to be too general and too focussed on the skin. The HDSS was considered to be too basic and not sufficiently discriminating.CONCLUSIONS:Future studies of the effectiveness of interventions for hyperhidrosis on health-related quality of life may benefit from including the HidroQoL© tool.
PP044 Adherence To Enzyme Replacement Therapy In Gaucher Disease
Authors: Quevedo Amanda , Dornelles Alicia , Paskulin Livia , Alegra Taciane , Krug Barbara , Vairo Filippo , Picon Rafael , Shwartz Ida , Picon Paulo 
Published: January 2018

INTRODUCTION:Gaucher disease (GD) is a genetic autosomic disorder for which treatment has been funded by the Brazilian government since the 1990s. In our state most patients are treated with enzyme replacement therapy (ERT) and followed by our Reference Center under the recommendation of the Ministry of Health Brazilian guidelines. There is a lack in the literature about adherence of patients to treatment. The objective was to describe adherence to the treatment in a cohort of all GD patients in the southern state of Brazil.METHODS:This was a cohort study of all GD patients treated with velaglucerase α, taliglucerase α and imiglucerase from January 2010 to January 2015. Adherence was measured as recommended by the Brazilian guidelines as to perform more than 50 percent of the anticipated infusions per year.RESULTS:Our study included thirty-seven patients of both genders. Doses of ERT varied from 15 to 45IU/kg for type 1 patients and from 30 to 60 IU/kg for type 3 patients. A mean of 83 percent of anticipated infusions were performed and from all patients only one did not adhere to the treatment during the 5 years of our study. The majority of the patients performed at least 50 percent of all anticipated infusions.CONCLUSIONS:We noted a very high rate of adherence to treatment with a very few adverse effects. Our data might be showing that the very high rate of adherence in these chronic disease patients may be attributed to the value of treatment by patients and their family, and also due to the existence of a multidisciplinary team at the reference center. These data might be useful for public health policy making in other countries.