bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP03 Evidence Synthesis In Spasticity In Children
Authors: Weijers Laure , Guyot Patricia , Kalyvas Chrysostomos , Danchenko Natalya , Freemantle Nicholas , Dinet Jerome , Senturk Oyku 
Published: January 2019

Introduction:Botulinum toxin type A (BoNT-A) is used in the management of lower limb spasticity in children, which affects more than 2.5 million children worldwide. BoNT-A aims to improve active function and to prevent or delay future musculoskeletal complications. The objective was to evaluate the relative efficacy and safety of different BoNT-A to manage spasticity in children, in the absence of head-to-head evidence.Methods:A systematic literature review was conducted in March 2016 to identify all relevant randomized controlled trials. The evidence base was synthesized by means of Bayesian network meta-analyses. Scenario analyses included standardized mean differences (SMD). The endpoints were Modified Ashworth Scale (MAS), Tardieu scale-spasticity grade and Goal Attainment Scale (GAS) (SMD only) at twelve weeks post-injection, and any adverse events.Results:Thirty-eight studies were identified, ten of which met the inclusion criteria for quantitative synthesis. For MAS, abobotulinumtoxinA 15 U/kg/leg was significantly better compared to onabotulinumtoxinA 4 U/kg/leg (−0.99 [−1.49; –0.50]), onabotulinumtoxinA 4 U/kg/leg + casting (−0.81 [−1.30; –0.32]) and numerically (although not statistically significantly) better than onabotulinumtoxinA 8 U/kg (−0.70 [−1.64; 0.22], Pbetter=93%). For GAS, abobotulinumtoxinA 15 U/kg/leg was numerically better than onabotulinumtoxinA 12 U/kg/leg. On Tardieu scale-spasticity grade, abobotulinumtoxinA was comparable to other treatments. AbobotulinumtoxinA 15 U/kg/leg showed the highest SUCRA value on MAS and GAS. On tolerability, abobotulinumtoxinA was found to have comparable or fewer adverse events than onabotulinumtoxinA 4 U/kg/leg.Conclusions:Our analyses suggest that abobotulinumtoxinA offers a comparable or favourable efficacy on tone (measured by MAS), spasticity (Tardieu scale-spasticity grade), functional outcomes (GAS) and tolerability versus onabotulinumtoxinA, in the management of children with lower limb spasticity. The results must be interpreted in the context of the heterogeneity of the evidence base and sparse evidence base.
PP18 An Access Evidence IT Solution Within A Pharmaceutical Company
Authors: Gyldmark Marlene , Rosenthal Rachel , Granser Martin , Escher-Kirch Jasmin 
Published: January 2019

Introduction:During 2014, Roche tested whether the EUnetHTA HTA Core Model© was a useful, exhaustive and relevant value framework to promote efficiencies in scoping, storing and sharing health technology assessment (HTA) evidence within a pharmaceutical company. The conclusion was positive and Roche decided to build a cloud based information technology (IT) platform to store all relevant HTA evidence to support global and regional market access activities, tagged with metadata according to the HTA Core Model©., The platform should be user-friendly and promote efficiencies and knowledge sharing across the organization. Eventually this platform may also be used by external stakeholders to access relevant HTA evidence.Methods:In order to better equip global functions, regions and affiliates in a major pharmaceutical company with user-friendly and fast access to product-relevant HTA and payer evidence as well as access evidence plans, an easy-to-use IT-based platform was needed. The platform, internally called #TAg, is a central repository of information to support market access activities and promote collaboration between Affiliate, Region and Global teams. The platform uses metadata to label all types of evidence and uses the HTA Core Model© domains to categorize the evidence.Results:The platform #TAg was developed throughout 2016/2017 and officially launched on 1 October 2017. Within the first 30 days, the platform has been readily accepted by affiliates, regions and global functions through significant use uptake as measured by user registration and download activities. In addition, #TAg was used successfully in a pilot project for a submission to an external HTA body.Conclusions:A complete knowledge management system for HTA evidence is important for driving efficiency in scoping, storing and disseminating access evidence information within a pharmaceutical company. #TAg has so far proved a good start on such a system with further development expected in the coming years.
PP19 Opioid Poisoning Deaths: A National Picture
Authors: Jones Matthew , Snooks Helen , Evans Bridie , Watkins Alan , Fuller Gordon 
Published: January 2019

Introduction:The factors associated with opioid poisoning death are poorly understood. We performed a retrospective autopsy study of decedents (a term used for people who are deceased) of opioid poisoning in Wales in 2015. Using anonymized linked data, we describe demographic characteristics, patterns of emergency service utilization, and clinical presentation prior to death.Methods:Decedents of opioid poisoning in Wales in 2015 were identified from the Office of National Statistics (ONS) mortality dataset. Records were linked with the Emergency Department Dataset (EDDS) by the National Welsh Informatics Service (NWIS); and held in the Secure Anonymized Information Linkage (SAIL) databank. The data were accessed and analyzed in the SAIL gateway.Results:Age at death ranged from eighteen to seventy-eight years, with a mean age of forty-two years. Average male age was forty-one years and average female age was forty-four and a half years. Seventy-three percent of decedents were men (n = 228/312). Eight-seven percent of decedents (n = 281/312) attended the emergency department in the three years prior to death. In total 2081 attendances were made, forty-one percent of which involved conveyance by ambulance. Attendances per individual ranged from one to 114, with over half of decedents attending more than three times. Diagnostic codes were mostly missing or non-specific, with only seven and a half percent of attendances representing eighty-two decedents, coded as drug related. Treatment codes were also mostly missing or non-specific, with sixteen percent of attendances representing 148 attendees attributed a treatment code. Thirty-nine percent of attendances (n = 822) ended in treatment and discharge, whilst twenty-seven percent (n = 562) led to hospital admission.Conclusions:Matching previously published data, we found that fatal opioid poisoning is preceded by a period of high emergency health service utilization. On average decedents were in their fifth decade and more likely to be male than female. Attendances varied widely, with men less likely to attend than women.
PP26 Facial Palsy Therapy: Can Novel ‘Smart Spectacles’ Help People Smile?
Authors: Szczepura Ala , Khan Amir , Holliday Nikki , Nduka Charles , Neville Catriona , Johnson Karen , Mistry Hema , Oxford Samuel 
Published: January 2019

Introduction:In the United Kingdom (UK), 23,000 people annually are diagnosed with facial palsy (acute onset facial paralysis). For nearly one third this will result in a permanent disability, including in some the inability to smile. In addition to initial pharmacological therapy, guidelines recommend tailored facial exercise (TFE) therapy repeated every day. However, not all patients are currently able to access such specialist physical therapy. ‘Smart specs’ (using miniaturized sensors in the frames to measure facial movement) are currently being developed. Linked to a smartphone, these could allow people to practice TFEs discreetly, provide immediate feedback, and supply data on outcomes to the patient and their clinician.Methods:Modelling of introduction of Facial Remote Activity Monitoring Eyewear (FRAME) into treatment pathways for patients with facial palsy. This included: (i) review on effectiveness of TFE therapy; (ii) national surveys (medical staff, facial therapy specialists and patients) to gather data on access to TFE therapy; (iii) Delphi Exercise to identify consensus on key outcome measures; and, (iv) economic modelling to estimate cost-effectiveness and determine a range of acceptable costs for the technology. In parallel, research to examine target markets to inform product development, and production of integral commercialization plan.Results:Searches short-listed ten studies to add to the three included in the 2011 Cochrane review. Surveys indicate approximately thirteen percent of eligible UK patients access personalized TFE therapy. Estimated annual expenditure on hospital treatments for facial palsy patients is currently >GBP 80 million (>USD 106 million) compared with <GBP 0.5 million (<USD 0.66 million) on TFE therapy. Patients with permanent defects can suffer a loss of up to two quality-adjusted life years (QALYs).Conclusions:Findings from this study, particularly in relation to costs and benefits, will inform the design of a subsequent randomized controlled trial. A novel wearable technology could make a major difference to people's lives, as well as generating potential efficiencies for healthcare.
PP29 Evaluating Supplementary Search Methods: Outcomes To Measure And Why
Authors: Talens-Bou Juan , Cooper Chris , Varley-Campbell Jo 
Published: January 2019

Introduction:In a recently published review of supplementary search methods, we proposed that researchers could usefully record time taken to search and present outcome values in similar way to existing studies, to facilitate generalisability of outcomes, where appropriate. We also discuss the idea of linking literature search effectiveness to study value. In this vignette, we discuss which outcomes we believe are important to measure and why. We discuss this in the context of the review of supplementary search methods and using a recently submitted evaluation of contacting study authors for context.Methods:In a recently completed systematic review, we contacted eighty-two study authors to ask three questions. We aimed to measure the following outcomes when contacting study authors: Effectiveness - determined as number of contacts compared to number of replies; Efficiency - i) time to make contact and ii) time between contact and reply. We determined this in hours, minutes and seconds, in line with other studies; Cost - determined by comparing the efficiency of contacting authors with the effectiveness; and Value - determined by reading and comparing the published studies with the replies received to see if any unique data were identified.Results:Effectiveness: thirty-eight answers were received from eighty-two possible contacts. Efficiency: In total, author contact took six hours, fifty-four minutes and twenty-five seconds across thirty-nine weeks. Replies were received across zero to thirty-nine days (median fourteen days). Cost: Cost for staff time was GBP 80.33 (EUR 91.20) or GBP 2.11 (EUR 2.40) per e-mail reply received. Value: We were able to identify value in author replies for each of the questions asked.Conclusions:In a recently published review of supplementary search methods, and a linked evaluation of the effectiveness of contacting study authors, we suggest outcomes that should be measured to determine effectiveness of literature search methods. We conclude that measuring these outcomes demonstrate both effectiveness and value.
PP50 Microcosting With Time-Driven Activity-Based Costing Applied On Brazilian HTA System: ECMO Case Study
Authors: Etges Ana Paula B S , da Rosa Priscila , Notti Regina K , Cruz Luciane N , Doebber Madeni , Cardoso Liane , Polanczyk Carisi A , Rohde Luis Eduardo 
Published: January 2019

Introduction:Extracorporeal circulatory membrane oxygenation (ECMO) is a technology that allows recovery of adults in cardiorespiratory failure with encouraging results, but is not available in the Brazilian universal public health system (SUS) due to high implementation costs. Time-driven activity based costing (TDABC) is applied to measure processes in an economic perspective by identifying opportunities to make processes more efficient through the reduction of resources used in each activity. The literature has explored the use of TDABC to measure costs related with clinical procedures and technologies in microcosting studies, identifying opportunities to improve the process by making it more efficient. This research measures the real costs to implement ECMO in Brazil to compare with the current public reimbursement system.Methods:This study applied TDABC using data from 6 patients to measure costs of ECMO intervention considering the public perspective in Brazil. In sequence, standard price payed by SUS was used to estimate the current reimbursement amount received by the hospital for ECMO procedure. Cost variable analysis was conducted to understand when and how patients receiving ECMO are using hospital resources. Cost data were collected from an academic public hospital using an average of 18 months (2016–2017) for the department costs.Results:The real average cost was USD 128,923. Most significant resource costs was medical staff, particularly for the three survivor patients, and the ECMO equipment presented the second highest cost. ECMO activities were separated into: before implantation of ECMO, period using ECMO, intensive care post-ECMO and rehabilitation, being the period where ECMO is the most expensive, particularly in nurse and physician costs. The SUS average was USD 31,437, which shows a difference of USD 97,485 between the real ECMO cost and the public reimbursement in Brazil.Conclusions:A critical element of the propagation of ECMO in Brazil and its reimbursement by public health system is the high cost and out-of-date standard payments by the Ministry of Health. Effort to implement a trustworthy method to guide decisions of SUS for the adoption and financing new technologies is essential to contribute to the optimization of public health policies in a country with a universal health system and limited resources dedicated to health sectors.
PP58 Hasty HTA: Delivering Health Technology Assessments Under Severe Time Constraints
Authors: Teljeur Conor , Harrington Patricia , Ryan Máirín 
Published: January 2019

Introduction:Health technology assessment (HTA) is a resource-intensive decision support tool that is typically structured around a set of defined domains. Sometimes urgent requests for HTAs arise that may be subject to very short timeframes, creating unique challenges. This study aimed to describe some of the options for carrying out a “hasty HTA”, and the impact of those options.Methods:We recently completed a HTA for the Department of Health in Ireland with a strict 2-month deadline. We considered the impact of the short timeline using the project management triple constraint framework whereby the quality of a project is constrained by cost, scope, and schedule.Results:When delivering HTAs within short timeframes the schedule is an inflexible constraint. Providing interim advice pending a full assessment may set a precedent, or may not be possible if capital expenditure is already entailed. Additional staff should enable research to be completed faster, although economies of scale may not fully apply. Frequently such resources are not readily available. The reduction of scope through the omission of domains offers the best prospect of facilitating a short timeframe for a HTA. Scope may also be reduced through a less comprehensive analytical approach, but this creates a risk of reduced accuracy. Curtailing data collection and analysis is likely to increase uncertainty in the findings. Risk management is important when comprehensive quality assurance may not be possible.Conclusions:Carrying out HTAs in short timeframes has implications for content, approach, and, potentially, quality. Agencies must consider how they can meet the needs of the decision maker without overly compromising accuracy or relevance. Due to resource constraints, the best approach is likely to be judicious changes to the scope to remove assessment elements that are unlikely to have a substantive impact on the decision.
PP59 Evaluating Reimbursement Applications With Decision-Oriented Evidence
Authors: Vreugdenburg Thomas , Cameron Alun , Maddern Guy , Ma Ning , Scarfe Anje , Tivey David 
Published: January 2019

Introduction:Our research group recently evaluated a minimally invasive surgical procedure in order to inform a reimbursement decision. The application for funding was designed around the study selection criteria from a single pivotal randomized controlled trial (RCT). The aim of this study review was to evaluate the safety and effectiveness of this minimally invasive surgical procedure, and document challenges faced in evaluating a technology based on a highly targeted population.Methods:A systematic literature search of four biomedical databases was conducted (PubMed, Embase, Cochrane library, York CRD) up to 8 August 2017. Specific elements related to the population were patient age, together with level and duration of pain. Primary effectiveness outcomes included pain, patient reported quality of life, mortality and adverse events. The included RCTs were critically appraised against the Cochrane risk of bias tool. Meta-analysis was not possible due to the limited availability of evidence with consistent outcomes.Results:From 4,718 search results, only one pivotal RCT specifically met the inclusion criteria, which demonstrated favorable safety and effectiveness of the procedure; however, the sample population in the trial had limited external validity to the proposed reimbursement population and follow-up was limited to six months. As a result, the selection criteria were broadened to better reflect the manner in which the service may be provided in clinical practice, and capture longer-term safety concerns. Four additional RCTs were included, which provided contradictory results.Conclusions:The results of this review identified two important issues in evaluating a health technology where the assessment has been focused to the results of a single trial. In particular, the generalizability of a trial is defined by the demographic distribution of the sample, not the selection criteria. Designing the review selection criteria around the selection criteria for a single trial can have consequences for a funding decision.
PP63 Factors Influencing Drug Prices Among Philippine Public Hospitals
Authors: Wong John , Modina Cheyenne Ariana Erika , Apostol Geminn Louis , Bagas Joy 
Published: January 2019

Introduction:In the Philippines, medicines are procured at higher rates in government hospitals. The prices of essential medicines have high variability, and a significant portion of out-of-pocket expenditures by Filipinos is for medicines. This study's objective is to determine the factors associated with the variation in drug pricing among public hospitals.Methods:This was a mixed-methods, case-control study of 57 hospitals. Two tools were developed based on: (i) Management Sciences for Health (MSH)’s Rapid Pharmaceutical Management Assessment and (ii) World Health Organization (WHO)’s Good Pharmaceutical Practices. The dependent variable is a drug price reference ratio of a preselected drug basket. Examples of factors studied are: (i) preference for generics, (ii) procurement type, and (iii) time out of stock.Results:Hospitals with proper procurement planning and performance monitoring are expected to decrease the price ratio (R = -0.030). However, interview data showed that forecasting is still not robust enough. Past consumption (91 percent) remained the most frequently used input to procurement planning. Few hospitals took into consideration other factors such as morbidity, mortality, and patient demographics. The expertise of hospital procurement staff increases the hospital's price mark-up. Interview results suggest this is because members and hospital units do not meet eye-to-eye to ensure accountability and coordination across units in planning and implementing the procurement procedures.Conclusions:By having a forward-looking procurement plan, forecasting can be more efficient. Potential improvement lies in finding mechanisms where nearby hospitals could participate in pooled procurement. Pooled procurement could have an impact on reducing prices by capturing economies of scale, provided this is operated efficiently and transparently.
PP68 Urinary And Fecal Collection Devices: A Cornerstone For Autonomy
Authors: Olivier Cyril , Guerin Caroline , Collignon Corinne , Galmiche Hubert , Belorgey Chantal 
Published: January 2019

Introduction:To stay at home, have social interaction, or work, people experiencing urinary retention or uncontrolled urine or feces leakages need specific medical devices (MDs). In France, the MDs used to be covered by the health insurance system if included on a specific list of products and services qualifying for reimbursement. These MDs for urinary and fecal drainage and collection are included under a generic description corresponding to a class of products with the same indications. This coverage modality offered low resistance to unnecessary or wasteful spending. Furthermore, a periodic update of the list is required whereas it has not been done for more than 10 years.Methods:In 2016, Haute Autorité de santé (HAS) assessed the actual clinical benefit of these MDs using a standard health technology assessment method (systematic literature review, opinions of health professionals and patients’ representatives). Manufacturers were asked to provide technical specifications on their MDs.Results:The lack of professional guidelines and well-conducted comparative clinical trials has to be pointed out; among 516 identified publications screened, only seven recommendations, one technological review and one randomized controlled study were selected. Despite this, HAS defined specifications for each generic description, based on users’ experience (patients and caregivers). These included specific indications, minimum technical specifications and, when applicable, conditions of prescribing and use. This assessment took into account individual preferences, the role of the natural carers and the conditions, and opportunities for patients to improve and update their self-care and rehabilitation skills.Conclusions:The HAS assessment of MDs for urinary and fecal drainage and collection provides a cornerstone for the enhancement of the access to the necessary devices for homecare. The expected benefits are an improvement of the quality of life and a reduction of health expenditure due to misuse, complications or hospitalizations.