bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP110 Survey Of Health Technology Assessment Evaluation Strategies For Patient And Public Involvement
Authors: Weeks Laura , Polisena Julie , Scott Anna , Holtorf Anke-Peggy , Staniszewska Sophie , Facey Karen 
Published: January 2018

INTRODUCTION:Although there is increased awareness of patient and public involvement (PPI) among Health Technology Assessment (HTA) organizations, evaluations of PPI initiatives are relatively scarce. Our objective as members of HTAi's Patient and Citizen Involvement Group (PCIG) was to advance understanding of the range of evaluation strategies adopted by HTA organizations and their potential usefulness.METHODS:In March 2016, a survey was sent to HTA organizations through the International Network of Agencies for Health Technology Assessment (INAHTA) and contacts of members of HTAi's PCIG. Respondents were asked about their organizational structure; how patients and members of the public are involved; whether and how PPI initiatives have been evaluated, and, if so, which facilitators and challenges to evaluation were found and how results were used and disseminated.RESULTS:Fifteen programs from twelve countries responded that involved patient (14/15) and members of the public (10/15) in HTA activities. Seven programs evaluated their PPI activities, including participant satisfaction (5/7), process evaluations (5/7) and impact evaluations (4/7). Evaluation results were used to improve PPI activities, identify education and training needs, and direct strategic priorities. Facilitators and challenges revolved around the need for stakeholder buy-in, sufficient resources, senior leadership, and including patients in evaluations. Participants also provided suggestions based on their experiences for others embarking on this work, for example including patients and members of the public in the process.CONCLUSIONS:We identified a small but diverse set of HTA organizations internationally that are evaluating their PPI activities. Our results add to the limited literature by documenting a range of evaluation strategies that reflect the range of rationales and approaches to PPI in HTA. It will be important for HTA organizations to draw on formal evaluation theories and methods when planning future evaluations, and to also share their approaches and experiences with evaluation.
OP116 Cost-Effectiveness Of Sacubitril/Valsartan In Heart Failure
Authors: Lin Liang , Aziz Mohamed Ismail Abdul , Wu David Bin-Chia , Ng Kwong 
Published: January 2018

INTRODUCTION:Heart failure (HF) is a major public health problem worldwide and in Asia. Sacubitril/valsartan reduces cardiovascular death and hospitalizations for HF. However, decision makers need to determine whether its benefits are worth the additional costs, given the low-cost generic status of current standard of care.METHODS:Using a Markov model, we projected lifetime clinical and economic outcomes of sacubitril/valsartan versus enalapril for 66-year-old patients with HF in Singapore. Key health states included New York Heart Association (NYHA) classes; patients in each state incurred a monthly risk of hospitalization for HF and cardiovascular death. Probabilities of events were based on the PARADIGM-HF trial. The uncertain treatment effect of sacubtril/valsartan in Asian patients was modelled using a hazard ratio (HR) of 1 as upper limit in sensitivity analyses. Utilities were obtained from published literature. Local national epidemiological and cost data were applied. Analyses were conducted from the Singapore healthcare payer's perspective. Both one-way and Probabilistic Sensitivity Analyses (PSA) based on 10,000 Monte Carlo simulations were performed.RESULTS:Compared to enalapril, sacubitril/valsartan was associated with an incremental cost-effectiveness ratio (ICER) of SGD74k (USD52k) per quality-adjusted life year (QALY) gained. The cost-effectiveness of sacubitril/valsartan was highly dependent on its effectiveness in reducing the risk of cardiovascular death. However, this was uncertain, particularly in the Asian subgroup, where results were not statistically significant. In sensitivity analyses using results from Asian patients, the ICERs ranged from SGD41k (USD30k) to SGD1.3 million (USD 0.94 million) per QALY gained. PSA showed the probability of sacubitril/valsartan being cost-effective was below 1 percent, 12 percent and 71 percent at thresholds of SGD20k (USD14k), SGD50k (USD36k) and SGD100k (USD 72k) per QALY gained, respectively.CONCLUSIONS:Given the uncertain ICER, sacubtril/valsartan may not provide good value for money compared to enalapril in reducing cardiovascular morbidity and mortality in patients with HF at the current daily cost. Our study highlights the cost-benefit trade-off that healthcare professionals and patients face when considering HF therapy.
OP138 Access To Orphan Drugs In The United Kingdom And Other European Countries
Authors: Zamora Bernarda , Garau Martina , Maignen François , O'Neill Phill , Mestre-Ferrandiz Jorge 
Published: January 2018

INTRODUCTION:Under the Orphan Regulation, the European Medicines Agency (EMA) intended to incentivize the research and development of new treatments for rare and life-threatening conditions. Marketing authorisation of orphan medicinal products (OMPs) by the EMA is only the first step, as medicines are made available to patients when reimbursement or Health Technology Assessment (HTA) decisions are implemented by national health systems. We analyzed the availability and access to OMPs in the United Kingdom (UK), France, Germany, Italy and Spain. We compared the availability, which is the possibility to prescribe a given OMP, to the access, which refers to the full or partial reimbursement by the public health service.METHODS:We collected data on launches, HTA decisions, any centralized commissioning and/or reimbursement decision for all the OMPs authorised since 2000 in the UK countries (England, Scotland and Wales), France, Germany, Italy and Spain.RESULTS:Since the Orphan Regulation inception, the EMA granted marketing authorization to 143 OMPs. These OMPs are most widely accessible in Germany and France. Reimbursement in Germany is immediate after authorization. France and Italy present a delay of 19 months from authorization to reimbursement, which is shorter than in other countries. In England, less than 50 percent of centrally authorised OMPs are routinely funded by the National Health Service (NHS), including one-third of these recommended by the National Institute for Health and Care Excellence (NICE), and those reimbursed via commissioning policies and the Cancer Drugs Fund.CONCLUSIONS:The assessment of degree of access to OMPs across Europe is limited by differences in the national HTA and reimbursement systems and the heterogeneous information made publicly available on their decisions. Nonetheless, our study suggests that the primary purpose to grant equal availability to OMPs to the patients in the Eropean Union via the implementation of the orphan regulation was partially achieved with important variations of access observed across the countries included in our study.
PP023 Applying Oncology Patient Registries As A Health Technology Assessment Tool
Authors: Kostyuk Alexander , Kostyuk Alexandr , Akanov Amangali 
Published: January 2018

INTRODUCTION:The limited healthcare resources have to be invested efficiently; Health Technology Assessment (HTA) is applied ever more often in many health care systems for “rational decision-making”. The oncology patient registries (OPR) track the eligibility of patients and the complete flow of treatments, guaranteeing appropriateness in use of pharmaceutical products, according to approved indications.METHODS:Normative legal acts and other regulatory documents in the field of oncology medical and pharmaceutical activity, include content and maintenance oncology registries. The system, process and information analysis, direct observation, comparative analysis, logical modelling, sociological methods (surveys and expert opinions) are applied.RESULTS:A temporary coverage/funding of oncology drugs often requires additional collection of data on safety, effectiveness, cost-effectiveness, and the appropriate use of the drug. Many of the oncology drugs show little or marginal effectiveness at time of approval and reimbursement agencies demand further data before deciding whether to cover the new drug. Pragmatic clinical trials, patient access schemes and standard data requirements on patient relevant outcomes in OPR are some of the approaches to generate further evidence and to fill the gap between knowledge on efficacy at time of approval and demanded knowledge on effectiveness for coverage decisions. For each monitored drug, patients eligible for treatment are registered in the specific therapeutic indication dynamic monitoring database to collect epidemiologic and clinical data, including data on the safety profile, and ex-post information missing at first evaluation stage.CONCLUSIONS:OPR provide a detailed view of the morbidity, mortality and resource utilization associated with an oncologies diseases entity. This data is of prime importance in coming to decisions on coverage of a drug or treatment. The collation of information is also quick and efficient owing to better methods of data management. OPR of Kazakhstan are equipped with sophisticated data processing software and technologies.
PP030 Socioeconomics Of Cardiac Rehabilitation: A Meta-Analysis
Authors: Ge Yin , Takura Tomoyuki , Nozomi Ebata 
Published: January 2018

INTRODUCTION:The Guidelines for Rehabilitation in Patients with Cardiovascular Disease recommends convalescent cardiac rehabilitation (CR) as the standard treatment for patients with ST elevation myocardial infarction (STEMI) (class I, evidence level B) (1). However, health economic evaluation of cardiac rehabilitation (CR) is limited.METHODS:This systematic review, meta-analysis study elucidated the cost-effectiveness of CR in the short term. The target population in this study included convalescent and comprehensive CR patients with coronary artery disease (CAD), most with myocardial infarction (MI). We used mortality, life years (LY, expected life years), medical costs, and cost-effectiveness as the evaluation parameters in this analysis. We set medical costs in the analysis associated with testing, diagnosis, and treatment during the observation period related to CR. For cost-effectiveness analysis, we analyzed medical cost per LY. We examined the differences in effects for two comparisons (CR versus Usual Care, UC) using the Risk Ratio (RR) and Standardized Mean Difference (SMD). We assumed the standard deviation (SD) of cost effectiveness in this study by applying the error propagation.RESULTS:We reviewed fifty-nine studies and identified three that matched our selection criteria. The studies had the following characteristics: two randomized clinical trials and one systematic review/meta-analysis; a control that does not include exercise in patients with CAD; an observation period longer than 1 year; adapting medical costs, LY, cost/LY, and mortality as the evaluation index. In total, 129,272 patients were included. Meta-analysis results revealed that the CR arm significantly improved LY (SMD: -.78, 95 percent Confidence Interval (CI): -1.37, -.19) compared with UC. Similar to LY, the CR arm significantly improved the mortality (SMD: .57, 95 percent CI: .22, 1.47) compared with UC arm. Since medical costs showed a high tendency (SMD:.02, 95 percent CI: -.08, .13), cost/LY demonstrated no improvement (SMD: .00; 95 percent CI: -.17, .18). Substantial statistical heterogeneity was observed between the studies with respect to LY and cost/LY.CONCLUSIONS:While sufficient evidence to conclude health economic efficiency is not available at present, these results suggest that CR is not potentially cost-effective in the short term.
PP086 Horizon Scanning In Multiple Sclerosis Decisions In Brazil
Authors: de Souza Andrea Brígida , Vidal Avila , Gomes Pollyanna , Canuto Vania , Petramale Clarice 
Published: January 2018

INTRODUCTION:In Brazil, the pharmaceutical sector has requested an individual incorporation in the Brazilian public health system (SUS) for each new drug for multiple sclerosis that receives sanitary authorization for marketing. Horizon Scanning within Brazilian Ministry of Health has played a key role in the recommendations made by the National Committee for Health Technology Incorporation (CONITEC). Horizon Scanning seeks to predict which technologies have potential to impact health care in SUS, before their formal request. This study aims to present the impact of horizon scanning in two assessments made by CONITEC on drugs to treat Multiple Sclerosis.METHODS:Grey literature was searched to find new and emerging drugs for multiple sclerosis treatment. Regulatory agencies were also searched: European Medicines Agency (EMA), Food and Drug Administration (FDA) and Brazilian Regulation and Health Surveillance Agency (Anvisa). A pre-defined standardized form was used. Information extracted about each drug was identified as: drugs name, mechanism of action, indication, administration route, finished phases of clinical trial and registration in other countries.RESULTS:In 2014, horizon scanning identified seven drugs while CONITEC was assessing Fingolimod for multiple sclerosis. In this case, the drug's administration route was a differential, as only three new drugs identified were also orally administrated. Thus, Fingolimod received a positive recommendation for incorporation. In 2016, horizon scanning identified fourteen drugs while Teriflunomide was under assessment. At this moment, the orally administrated Fingolimod was already available and it was identified other eight new drugs with the same route. Therefore, the initial recommendation was against its incorporation.CONCLUSIONS:Horizon scanning has proved to be of major importance for assisting recommendation-making process of the committee. In the two cases presented, horizon scanning information could predict which technologies were being developed and could be registered in Brazil. These new technologies had influenced the recommendations made by CONITEC's members. As a result, a horizon scanning section in all CONITEC's reports became mandatory.
PP094 Autologous Stem Cell Transplantation For Multiple Sclerosis
Authors: Giske Liv , Frønsdal Katrine , Lauvrak Vigdis , Stoinska-Schneider Anna , Fure Brynjar 
Published: January 2018

INTRODUCTION:Multiple Sclerosis (MS) is an inflammatory neurological disease. The standard treatment is disease modifying drugs which may alleviate symptoms and slow the progress of disability, but not lead to remission. Autologous Hematopoetic Stem Cell Transplantation (AHSCT) is a new technology for treatment of MS in Norway. Disease remission in some patients treated with AHSCT has been postulated, in particular in patients in the early course of disease with high disease activity classified as having Relapsing-remitting multiple sclerosis (RRMS), but severe complications have also been reported.METHODS:We conducted a systematic literature search. No restrictions were set in terms of study design, although case series should include at least ten patients with a majority having RRMS. We performed a cost analysis from a healthcare perspective with a time frame of one year.RESULTS:One randomized controlled trial (RCT) (n = 21, RRMS = 7), one registry study (n = 345), and seven case series (n = 442, RRMS = 277) without control groups were included. Estimated transplant-related mortality was 2 percent or lower. Serious adverse events, such as infections, were common during follow-up. Stabilization or improvement in neurological status was reported in 63 percent to 89 percent of the patients after three years, and the number of Gd+ and T2 lesion volume assessed by Magnetic Resonance Imaging was reduced. The quality of evidence assessed using the “Grading of Recommendation Assessment, Development, and Evaluation” tool was low for transplant-related mortality and very low for the other outcomes. HSCT costs were approximately between 480,000 and 605,000 Norwegian kroner per patient in the intervention year. A heterogeneous disease progression, significant risks associated with the method, lack of effective treatment options, and uncertainty about the benefit versus risk, implies that any decision to offer HSCT in the treatment of MS is ethically challenging.CONCLUSIONS:The main limitation in this HTA is the absence of controlled studies, which introduces a high risk of bias. Studies without control groups, including mainly patients with RRMS, reported that disease activity could be delayed or stopped for a period of up to three years in several patients, whereas adverse events were common. Ethical considerations are associated with significant uncertainty of benefit versus harm. The low level of evidence implies the need for controlled trials.
PP102 Perceived Quality By Patients Hospitalized At Home Undergoing Domicillary Radiography
Authors: Davini Ottavio , Giacometti Marika , Scardino Matteo , Cerutti Stefano , Guida Antonio , Tibaldi Stefano , Romano Valeria , Rivoiro Chiara , Grosso Marco 
Published: January 2018

INTRODUCTION:Europe currently has the highest proportion of older people in the world and is expected to maintain this leading position for the next 50 years. A teleradiology service for frail patients living at home or in nursing-homes (R@dhome), funded by the Italian Ministry of Health, has been operating from June 2013 to May 2015 (1). The goal was to offer, within the path of home care patients, radiologic care for fragile elderly patients (2). In this report of the study a secondary outcome was evaluated; the perceived quality for patients and caregivers undergoing home radiology.METHODS:The study was a randomized controlled trial (RCT) with 136 patients; 71 cases and 65 controls. The perceived quality assessment was only performed among home inpatient group cases, at home or in nursing homes and not in the control group patients (who had treatment in a hospital). The assessment instrument was a semi-structured interview based on the benchmark system SERQUAL®. The identified dimensions of quality were: tangible aspects, reliability, responsiveness and empathy in a subpopulation of non-dementia patients. Eligible subjects were interviewed between May 2014 and May 2015.RESULTS:The percentage of satisfied patients, evaluated on the dimensions of perceived quality, was 97.7 percent. The main reasons given were: short waiting time, best comfort and safety, efficiency, operator's kindness and less need to travel for the treatment.CONCLUSIONS:R@dhome benefits are not limited to clinical or financial aspects but ethical, social and relational advantages have also been shown in this study (3). Given the fragility of the patients, positive results were mostly achieved by reducing the risk of trauma in transport between their home and the hospital.
PP111 The Use Of Long-acting Injectable Antipsychotics In Schizophrenia
Authors: Bini Chiara , Mennini Francesco , Marcellusi Andrea , Verzura Claudio , Trovini Giada , Rapinesi Chiara , Kotzalidis Georgios D. , De Filippis Sergio , Carrus Dario , Ballerini Andrea , Francomano Antonio , Ducci Giuseppe , Del Casale Antonio , Brugnoli Roberto , Girardi Paolo 
Published: January 2018

INTRODUCTION:Schizophrenia is a severe mental disease that affects approximately 1 percent of the population with a relevant chronic impact on social and occupational functioning, and daily activities. The aim of this analysis was to evaluate the clinical and economic consequences of long-acting injectable (LAI) treatment in patients with psychotic disorders, with a special focus on schizophrenia, in Italian real world practice.METHODS:A retrospective, observational mirror-study was developed to analyze outcomes measure referred to patients with psychotic disorders. Five hospital centers were involved in this study that collected patient level data from clinical databases. Retrospective data for each patient were referred to 6 months before LAI drug administration and 6 months after. A paired-Samples t-test was performed in order to identify statistical differences between pre- and post-LAI administration.RESULTS:A total number of 308 patients were enrolled in the study (65.6 percent male). Of these 221 were eligible for our analysis (119 with schizophrenia). In the six months after LAI administration period we estimate a 47.3 percent reduction of the antipsychotic drugs (43.8 percent for schizophrenic patients), 94.7 percent reduction of hospitalizations (94.0 percent for schizophrenic patients) and adherent patients increase to 198/221 patients (78/221 in pre-LAI administration period). All differences between pre- and post- LAI administration period were statistically significant with a p< .005. In Italy over 152 thousand schizophrenic treated patients were estimated. Assuming that 20–40 percent of patients are eligible to the Mo.Ma (Model of Management) approach, our model estimates a direct cost reduction during the first year of implementation of around EUR12 million. Additionally, EUR18 million of direct costs in the mid-term and EUR58 million of indirect costs could be saved in the mid-term estimating a total cost reduction, due to the Mo.Ma approach, of about EUR90 million.CONCLUSIONS:This new therapeutic approach could change the cost structure of schizophrenia by decreasing costs with efficient economic resource allocation guaranteed from efficient diagnostic and therapeutic pathways.
PP137 Regional Process For Planning Medical Equipment Procurement In Italy
Authors: Medici Mario Fregonara , Bellelli Stefania , Villarboito Luciano , Pepe Michela 
Published: January 2018

INTRODUCTION:An appropriate governance of the installed equipment base, by defining replacements strategies and programming introductions of innovative Biomedical Technologies (BT), has direct effects on the efficiency and effectiveness of health systems. An effective health technology management is of paramount importance for providing safe, high quality and innovative care with the constraint of health-care budgets, safeguarding equity, access and choice principles. Data from the regional BT information flow show that, compared to the gold standard (1), the North Region of Italy has about 15 percent less of large medical equipment younger than 5 years and about 15 percent more of equipment older than 10 years.METHODS:In order to draw a unified path of BT procurement processes, in 2013 and 2014 regional regulations (2,3) were enacted. Each Public Hospital and Local Health Authorities (ASRs) defined a plan (PLTB) containing, regardless of the form of procurement and type of funding, all BT requests for a value greater than EUR40,000 distinguished in replacement/new acquisition/upgrade, innovative acquisition and donations. Requests of BT have to obtain the authorization by the Regional Healthcare Authority Commission (GTB), in compliance with defined criteria, including financial and sustainability aspects, after the evaluation of the Regional Clinical Engineering Commission (GIC) supported by IRES, Health Technology Assessment and Management research group.RESULTS:Over the years 2014 and 2015, the ASRs submitted 491 BT requests, of which 87 percent were replacement/new acquisition/upgrade, 9 percent innovative acquisition and 4 percent donations. Altogether 26 percent of these instances were urgent and 2 percent were unique BT on the market. Sixteen percent of requests for replacement/new acquisition/upgrade of BT related to large medical equipment with mean age of 13.3 years, 2 percent regarded innovative BT with average age of 8.4 years and 48 percent widespread technologies with mean age of 15.6 years.CONCLUSIONS:The limitations in investments deriving from being a Region in “Recovery Plan”, have originated an absence of BT programming, as shown in PLTB by the prevalence of requests for the replacement management of obsolete equipment with inadequate performance, high machine downtimes and elevated maintenance costs.