bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP61 Advanced Therapy Medicinal Products Germany: Drugs Or Methods Review?
Authors: Müller Elvira , Neeser Kurt , Oelze Ilse-Barbara 
Published: December 2019

IntroductionAdvanced Therapy Medicinal Products (ATMPs) comprise medicines for human use based on gene therapy, somatic cell therapy or bioprocessed tissue products. ATMPs are pharmaceutically manufactured drugs and mostly subject to central authorization requirements. In terms of social law, it is an ambiguous situation and more heterogeneously dealt with. ATMPs are assigned to method evaluation as well as to the Arzneimittelmarkt-Neuordnungsgesetz (AMNOG) procedure designated for drugs.MethodsGuidelines from Gemeinsame Bundesausschuss (G-BA), Institute for Quality and Efficiency in Health Care (IQWiG) and respective legislation, consultation results and methods/medical devices (MDs) evaluations according to §137h and for drugs according to AMNOG were reviewed and analyzed. Decision criteria and reasoning, assessment outcomes and potential impact on price negotiations were the main aspects for comparison.ResultsATMPs are subject to benefit assessment, with a decision at first on whether to be evaluated as a drug (e.g., Alofisel) or a method/device (e.g., Holoclar). By definition, an ATMP is classified as a treatment method, if the correct administration has at least the same significance for a successful therapy outcome as its mode of action. Depending on the respective decision, an evaluation as method follows or it must undergo the AMNOG process. According to G-BA's and IQWiG's point of view, randomized controlled trials (RCTs) are the “gold standard” for a benefit assessment of new therapies, including ATMPs. However, conduction of RCTs is not always possible for ATMPs which creates a disadvantage in the assessment right from the beginning. Otherwise no distinction is made between drugs and ATMPs in terms of reimbursement modalities. Outcomes based agreements could help overcoming inequalities and lead to quality-oriented reimbursement.ConclusionsATMPs represent a grey zone causing difficulties in classifying them either as method or drug. For individualized therapies evidence beyond RCTs and new reimbursement possibilities should be considered. Until new regulations are in place it is advisable to enter early into respective discussions with authorities.
PP107 Harpoon™: A Novel Device For Transapical Mitral Valve Repair
Authors: Hijas-Gómez Ana Isabel , Polo-DeSantos Mar , Luengo-Matos Setefilla , Sánchez-Gómez Luis María 
Published: December 2019

IntroductionMitral regurgitation (MR) is the most prevalent heart valve condition in Western countries. Open-heart mitral valve reconstruction is the conventional surgical treatment for MR, whereby the valve's cords are replaced with expanded polytetrafluoroethylene cords. Novel devices have introduced minimally invasive alternatives, such as transapical beating-heart valve repair. Among these alternatives, the Harpoon™ Mitral Valve Repair System (Edwards Lifesciences LLC) may have potential advantages (a smaller diameter valve introducer to minimize bleeding and a different anchoring mechanism). This study aimed to assess the efficacy and safety of Harpoon in minimally invasive mitral valve surgery.MethodsAn early assessment of the technology was conducted by reviewing relevant literature from the following databases: PubMed, EMBASE, Web of Science, the Trip Database, the International Clinical Trials Registry Platform, ClinicalTrials.gov, the Cochrane Library, and the Centre for Reviews and Dissemination. Relevant clinical studies published up to 30 January 2018 were included.ResultsOnly two publications, by the same research group, were included: an observational study of 11 patients and the prospective, nonrandomized TRACER trial (n = 30). During the procedure, MR was reduced from severe to none in 73 to 86 percent of patients and severe to mild in 14 to 27 percent. At one month, MR was rated as mild or lower in 82 to 89 percent of patients. At six months, MR had worsened to moderate or severe in 16 percent of patients from the TRACER trial. Safety issues within 30 days (18% to 27% of patients) included intraoperative conversion to open surgery, reoperation, pleural effusion, hemopericardium, and atrial fibrillation. There were no intra- or postoperative deaths.ConclusionsCurrent evidence on the Harpoon device is scarce. Although published studies showed improvement in MR in most patients, there are still issues regarding safety, lack of long-term results, comparability with other procedures, and costs. While promising, further research is required before recommending routine use of this technology.
PP113 A Framework To Enhance Eurasian Economic Union Cooperation On Health Technology Assessment: Lessons From The European Network for Health Technology Assessment
Authors: Sultanov Marat , Kostyuk Alexandr , Almadiyeva Alima , Absattarova Karlygash 
Published: December 2019

IntroductionThe Eurasian Economic Union (EAEU), which currently includes Armenia, Belarus, Kazakhstan, Kyrgyzstan, and Russia, was established in 2015. Pursuing economic integration, and modeled in part after the structure of European Union, the EAEU launched a common medicines market in 2017. There have been various developments regarding cooperation in health technology assessment (HTA) across the EAEU countries, exemplified by a conference held in Kazakhstan in 2017. Here we discuss some considerations for developing cooperation in HTA throughout EAEU based on the experiences of implementing the European Network for Health Technology Assessment (EUnetHTA).MethodsLegal and review documents regarding the implementation of EUnetHTA were obtained from the European Commission website and research databases to inform this narrative review.ResultsAchieving recognition of the role of HTA at an inter-governmental level, akin to the actions of the European Commission prior to establishing EUnetHTA, appears pivotal at the current stage of HTA development among EAEU members. Similar to the EUnetHTA project stage of 2006-2008, the existing HTA structures and national standards will need to be accurately and systematically assessed by a working-group appointed specifically for that purpose. Besides the importance of accepting a unifying framework similar to the EUnetHTA core model, implementation features that are specific to the context of EAEU countries could include the development of common adaptation toolkits and glossaries. Capacity building efforts may also prove crucial to ensure the sustainability of HTA-related cooperation.ConclusionsOptimization of resources by streamlining HTA processes, whether in research, policy, or results dissemination, and avoiding duplication of effort by HTA agencies, is relevant in the context of limited healthcare resources in developing countries. This overview is an attempt at facilitating discussion to inform policy and research efforts to streamline HTA processes.
PP130 Oral Supplements For Protein-Energy Wasting In Chronic Kidney Disease
Authors: Brito-Garcia Noe , Toledo-Chávarri Ana , Trujillo-Martin Maria , Leon-Salas Beatriz , Serrano Pedro , González-Hernández Nerea 
Published: December 2019

IntroductionMalnutrition, specifically protein-energy wasting (PEW), is common in patients with chronic kidney disease (CKD), and its prevalence increases as CKD progresses. Oral nutritional supplementation (ONS) with complete formulas specific to this pathology, is a strategy aimed at meeting energy and protein requirements that are not possible with dietary recommendations and advice alone. This study systematically reviewed the available scientific literature on the safety and effectiveness of nutritional therapy with complete formulas specially designed for adults with CKD who have PEW that is not reversible through ordinary food consumption.MethodsWe systematically searched for articles published up to May 2018 in several electronic databases. We included comparative studies that evaluated the safety and effectiveness of complete nutritional formulas for malnourished adults with CKD. Relevant outcomes included rates of death, hospitalization, and adverse effects, and changes in nutritional status, anthropometrics, and health-related quality of life (HRQoL).ResultsThree systematic reviews and 22 primary studies were identified. The primary studies comprised nine randomized controlled trials, nine non-randomized comparative studies, and four before-after studies (the latter were only included in the safety review). The majority of studies were conducted in patients on hemodialysis. The studies exhibited methodological heterogeneity in terms of the methods used to measure nutritional status and the interventions and comparators evaluated. There was also inconsistency among the results. Adherence to ONS, especially in the long term, can be affected by taste fatigue produced by repeatedly taking the same formula. Some studies recommend supplementation during hemodialysis sessions.ConclusionsThe studies with less risk of bias indicated a trend toward improvements in rates of death and hospitalization, HRQoL and, to a lesser extent, some anthropometric variables and serum markers, such as albumin, when ONS was given to patients with CKD. High quality comparative studies are needed to make conclusive statements about the effectiveness of this intervention.
PP137 Toric Intraocular Lenses and Spectacle Independence: A Systematic Review
Authors: O'Boyle Derek , Vives Caridad Perez , de Haan Jan , Ender Frank , Busutil Rafael 
Published: December 2019

IntroductionAstigmatism is a common ocular condition that causes reduced visual acuity. The condition is highly prevalent in cataract patients, with preoperative astigmatism of at least 0.5 diopters being present in 78 percent of cataractous eyes. Residual uncorrected astigmatism after cataract surgery is associated with significant costs, primarily driven by the lifetime cost of spectacles (estimated at EUR 1,608 to EUR 3,608 in Europe). Toric intraocular lenses (IOLs) are a safe and effective way of correcting astigmatism, while also reducing the need for spectacles after cataract surgery. The objective of this review was to assess the published evidence relating to spectacle independence in patients implanted with toric IOLs, compared with those receiving non-toric IOLs with or without astigmatism reducing surgical interventions (SI).MethodsA systematic literature search was conducted of the EMBASE, MEDLINE, and Cochrane Library databases. Articles were selected if they included adult patients undergoing phacoemulsification who had age-related cataracts and preoperative regular corneal astigmatism of at least 0.5 diopters, and assessed spectacle independence as an outcome.ResultsSeven studies met the inclusion criteria: four randomized controlled trials and one non-randomized comparative study comparing toric IOLs with non-toric IOLs, and two randomized controlled trials comparing toric IOLs with non-toric IOLs plus SI. Spectacle independence was evaluated as the number of patients who reported not requiring spectacles for distance viewing at 3 or 6 months. Figures for spectacle independence ranged from 60 to 100 percent for toric IOLs, 31 to 50 percent for non-toric IOLs, and 36 to 65 percent for non-toric IOLs plus SI. In each study, toric IOLs demonstrated superior spectacle independence compared with the control group.ConclusionsThe benefits of toric IOL implantation for astigmatic cataract patients included a higher rate of spectacle independence, compared with non-toric IOLs with or without SI. For this group of patients, the lifetime economic burden of spectacle acquisition costs can be reduced with the implantation of toric IOLs during cataract surgery.
PP150 Bevan Health Technology Exemplars: Early Dialogue To Systematize HTA
Authors: Myles Susan , Charles Sion , Davies Claire , Elston Lauren , Howson Helen , Poole Ruth Louise 
Published: December 2019

IntroductionWales has ambitious health, wealth, and innovation policies and a clear goal to use the economic muscle of the Welsh National Health Service (NHS) to support its strong life sciences sector. Health Technology Wales (HTW) has a clear remit to appraise technologies over the span of their lifecycle from innovation to obsolescence. HTW is collaborating with the Bevan Commission through their national Health Technology Exemplars (HTEs), which partners NHS and industry stakeholders to strengthen innovation within the Welsh health system.MethodsHealth technology assessment (HTA) methods were used to produce topic exploration reports for assessing the evidence underpinning applicant innovations. A “Dragons’ Den” expert panel was convened to select the successful HTEs.ResultsFourteen Bevan HTEs were awarded funds, which were matched by industry partners. Application of HTA methods resulted in more critical consideration of technology value propositions, including: developing pull models of innovation focused on delivering health technology solutions for current problems facing NHS Wales; supporting early dialogue between the NHS and industry partners around demonstrating evidence of improved patient outcomes; and focusing on transformative rather than incremental innovation. The most promising innovations will progress to rapid HTA, where the evidence generated will be used to develop guidance for NHS Wales.ConclusionsHTA methods were productively deployed at the innovation phase of the technology lifecycle to support evidence-informed allocation of scarce innovation resources. In this way, HTW is working with key stakeholders to identify and offer early support to the most promising innovations, with the aim of expediting their adoption and realizing health benefits for patients as quickly as possible. The Bevan Commission has partnered with HTW to routinely build in HTA and evidence considerations in its future innovation calls and competitions. Thus, HTW has established a “feeder” pipeline for assessing bottom-up service-led innovations and encouraging evidence consideration throughout the lifecycle of innovative technologies.
PP156 Reimbursement Of New Treatment Methods In Hospitals: Status In Germany
Authors: Oelze Ilse-Barbara , Müller Elvira , Neeser Kurt 
Published: December 2019

IntroductionSince 2005, new treatment and diagnostic methods (NUBs) were reimbursed by individual supplementary fees. The assessment procedure for NUBs is induced by hospitals submitting a request for additional compensation of healthcare treatment to the Institute for the Fee System in the Hospital (InEK). In 2016, the legal norm §137h SGB V was introduced to evaluate medical devices (MD) of high risk classes by the Federal Joint Committee (G-BA). InEK grants a status that is valid for twelve months and impacts additional compensation as well as assessment required by G-BA. The effects of this rating seem to differ between hospitals and Statutory Health Insurance (SHI).MethodsThe published InEK decisions on NUBs were analyzed according the decision criteria and possible impact on price negotiations with SHI.ResultsIn 2018, 705 NUB requests were assessed by InEK. NUB Status 1, granting negotiation of additional coverage, was assigned to 171 procedures. Status 2 – no additional reimbursement possible - was given in 472 cases, the remaining had not sufficient information. Most NUBs (n = 368) requests did not fall under §137h; however, those with sub-Status “B” (allocated to 12) led to controversies; no participant had requested an evaluation according to §137h for the NUB. Two consultation requests receiving Status 1 B were regarded as not eligible according to § 137h by the G-BA. To avoid price negotiation delays, early consultations according to § 137h are recommended by G-BA during the NUB application.ConclusionsThe NUB process enables hospitals to receive a supplemental payment when using innovative technologies not listed in the existing German healthcare system. The question of which requirements must be fulfilled to guarantee the reimbursement should be asked at an early stage. Consultation requests to the G-BA in due time are strongly recommended. Contact between manufacturers and hospitals are advisable to support the NUB application.
PP192 An Institutional Ethical Framework For HTA: Stakeholder Participation
Authors: Goetghebeur Mireille , Demers-Payette Olivier , Pomey Marie-Pascale , Ganache Isabelle , Roy Denis 
Published: December 2019

IntroductionIn a context of rapidly evolving technologies and growing evaluative challenges, the National Institute of Excellence in Health and Social Services (INESSS) is developing an institutional ethical framework making explicit and transparent the guiding principles and new modalities of process for health technology assessment for public coverage.MethodsThis framework is co-built by the INESSS experts - drugs, social services, technology and health services and cross-cutting methodologies - through literature and practice reviews as well as a consultative process on key topics with external collaborators.ResultsThe development process aims to: (i) identify the principles applicable to all the objects evaluated, (ii) define the evaluation strategies used to appropriately address evaluation challenges in the clinical, organizational, economic and societal dimensions, (iii) equip the scientific teams to successfully integrate diversified knowledge from the literature, stakeholders participation and medico-administrative data banks, and (iv) facilitate deliberation leading to evidence-informed recommendations. It is envisioned as a fully integrated process rooted in a reflexive multi-criteria approach supporting fair and reasonable decision. The presentation will focus on one of the key aspects of this framework, i.e., the development of principles for stakeholder participation based on a recent INESSS methodological forum on the topic, and the agile deployment of innovative processes and tools in various projects, including the patient partnership developed with a pioneering academic centre.ConclusionsThis framework provides explicit, transparent and cross-cutting processes and a framework for continuous improvement. The goal is to promote stakeholder engagement and enable increasingly complex arbitration aimed at equity and social justice, in a context of rising costs and uncertainty, and focused on the creation of value for our fellow citizens.
VP01 Methods Of Patient Involvement Now And Beyond 2020: A Case Study
Authors: Livingstone Heidi , Leng Gillian , Thomas Victoria 
Published: December 2019

IntroductionInvolving patients and the public in the health technology assessment (HTA) has always been fundamental to NICE. To ensure the appropriate method of patient involvement remains relevant to the evolving types of HTA, NICE uses varying methods of involvement. These methods have been reviewed to ensure they remain current and relevant for HTA now and beyond 2020, and also to give guidance on the approaches that should form a standard baseline and those that could be optional.MethodsWe identified and mapped the different methods of patient involvement used at NICE across five types of HTAs: diagnostics; medical technologies; medicines; ultra-orphan conditions; and surgical procedures. We looked at the varying methods of early engagement identifying similarities and differences, and considered the benefits and challenges of each.ResultsThe different methods of patient and public involvement include: lay members (generalist and topic expert) involved in decision making, individual patient input (written and oral), and patient group (organisation) input (written). The types of involvement fell into the following categories: written group submissions, written individual statements, surveys of individuals, pre-meeting events/workshops, oral testimonies at committees, and written consultation responses. The common methods across all HTA types were generalist lay members and consultations.ConclusionsThis review highlighted the varying methods of involvement at NICE and highlighted additional methods that could be standardised across the different types of HTAs as a baseline. These included patient organisation submissions and a method for additionally including individual patients in each type of HTA. We identified that where patient involvement started early and continued at each stage of the process including a pre-meeting event, it was particularly helpful to the stakeholders’ ability to contribute.
VP02 Involving Patients In HTA Beyond 2020: A Thematic Review
Authors: Livingstone Heidi , Li Xia , Leng Gillian , Thomas Victoria 
Published: December 2019

IntroductionInvolving patients and the public in the health technology assessment (HTA) is crucial and a key part of the NICE patient and public involvement (PPI) policy. To advance the development of our PPI policy in HTA and build capacity for 2020 and beyond, we took stock of knowledge on stakeholders’ views of involving this cohort in HTA.MethodsWe carried out a thematic review of the existing evidence on the involvement of patients and the public in HTA, including: technology appraisals consultation 2017 (110 comments): technology appraisals consultation 2018 (205 comments); and PIP review consultation 2017 with a CHTE focus (162 comments). We used Thomas and Harden's (2008) thematic synthesis to code the data ‘line-by-line’, to develop ‘descriptive themes’, and then to generate ‘analytical themes’. This was followed by using Patton's (1999) triangulation of qualitative data sources to further challenge and refine the emergent themes.ResultsWe identified three themes, namely (i) earlier and full engagement, (ii) simpler and easier engagement, and (iii) patient evidence. Respondents emphasised the significance of involving patients earlier and throughout the process of developing every appraisal to enable them to gain a greater sense of participation and ownership. Respondents also expressed a strong view of making it simpler and easier for patients to engage in the process through various methods, e.g., standardising the approaches, and support and training. Finally, respondents expressed their positive attitudes toward using patient evidence in HTA, clarifying how patient evidence is captured and used, and offering a clear feedback mechanism to the impact of patient evidence on decision-making.ConclusionsThis review highlighted the significance of earlier and full engagement with people, making it simpler and easier for people to work with us, and being clearer about how we use patient evidence with a clearer feedback mechanism as to the impact of their input on the final decisions.