bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP53 Health Technology Assessment Acceptability Of Innovative Survival Metrics In Oncology
Authors: Macaulay Richard 
Published: December 2019

IntroductionMost new oncology therapies are studied in the advanced/metastatic setting. However, there is an increasing focus on earlier stage disease. Nevertheless, measuring Overall Survival (OS) in neo-/adjuvant therapy trials can be very challenging due to the increased life expectancy and the confounding effects of subsequent treatments. Thus, their primary endpoints tend to be surrogate survival metrics (e.g. metastases-free survival). This research aims evaluates the health technology assessment (HTA) acceptability of such endpoints through recent neo-/adjuvant HTA assessments.MethodsThe European Medicines Agency (EMA) website was screened for any neo-/adjuvant oncology therapies approved (1 January 2013-22 October 2018) and any corresponding publicly-available assessments by HTA bodies (NICE, SMC, IQWiG, G-BA, CADTH, PBAC, HAS) were identified and key data extracted.ResultsSix neo-/adjuvant therapies have received marketing authorization by the European Commission (EC). These six have been on the market for an average of 8.9 months (range: 0.9-39.3 months, median: 3.3 months). In four of the six, the pivotal trial primary endpoints were measures of relapse-/disease-free survival, (others: pathological complete response and PFS/OS co-primary). Only one had mature OS data available at EC-approval. Four of the six therapies had received at least draft guidance by an HTA body, encompassing 11 HTA assessments in total (4: NICE, 2: IQWiG, HAS; 1: SMC, CADTH, G-BA). Only two of 11 (18%) were positive outcomes (both NICE), the remaining nine were negative.ConclusionsOncology therapies are increasingly receiving regulatory approval in the neo-/adjuvant setting. However, their pivotal trials are frequently powered to show benefits in disease-/metastases-free survival. Whilst sufficient for regulatory approval, translating this to favorable HTA decisions has been more challenging. Clearly establishing linkages between surrogate survival metrics and OS alongside measuring metrics that clearly portray patient benefits (e.g. time to symptomatic progression) could improve HTA-acceptability. Further, some payers allow for temporary reimbursement whilst additional evidence is generated (e.g. Cancer Drugs Fund in England).
OP60 Challenges In Evaluating Smart Medical Devices
Authors: Mähs Mareike 
Published: December 2019

IntroductionSmart medical devices can empower elderly to live independently in their familiar surroundings. To enhance their dissemination, they have to be shown to be cost-effective. Economic studies evaluating such technologies are missing or are criticized for their low quality. There are several challenges in the evaluation of smart medical devices, including their complex nature and innovative character. The question arises: how can evaluations elicit the benefits and cost-effectiveness of smart medical devices. This research has the aim of outlining challenges and demands on the evaluation of smart medical devices.MethodsThe embedding of the technology in existing structures can influence the effectiveness of the technology. By comparing such a technology with a regular intervention, learning effects have to be considered. Regular modifications and further developments of these technologies can complicate the traceability of the effects. Complex cause-effect relationships with possible interactions arise that are difficult to quantify and express in standardized endpoints, utilities or monetary values. Demands on the evaluation of smart medical devices have been explored with literature reviews and scenario techniques using the example of intelligent rollators.ResultsIt is important to apply mixed-method approaches not only in the clinical but also practical setting and conduct observational as well as qualitative studies. Potential users, their relatives and care personnel should be involved in the evaluation of intelligent rollators and attention should be payed to subjects with disabilities. Prospective studies should be conducted at different stages along the lifecycle of the technology. A conceptual model should be developed and evaluated as well as adapted on a regular basis.ConclusionsThe research shows the need to adapt common methods used in economic evaluation to the characteristics of smart medical devices. As a next step, a framework for the economic evaluation of such technologies within the scope of Health Technology Assessment is developed based on these demands.
OP80 Impact Of Patient Group Participation At Scottish Medicines Consortium Committee Meetings
Authors: Dickson Jennifer , Lockhart Lindsay , Taylor Louise , McCormack Jackie , Walker Laura 
Published: December 2019

IntroductionThe Scottish Medicines Consortium (SMC) encourages patient group (PG) representatives to participate in the decision-making committee meetings, answering questions from committee members and providing points of clarity throughout discussions if required. In a continuous improvement approach the process and the participant experience is continually evaluated to monitor impact and emerging themes.MethodsThe interactions between committee members and PG representatives are recorded in writing by the public involvement team to monitor the questions or points of clarity raised. These interactions were analyzed using thematic analysis to look for emerging themes. Following the meeting, PG representatives are invited to complete an online survey on their experience of working with SMC.ResultsFrom July 2017 to October 2018, 36 PG representatives have attended committee meetings for the discussion of their submission. Committee members asked 17 PG representatives to contribute. Key themes that have emerged to date include insight into the impact of living with the condition on quality of life and how a new medicine may affect this. Survey feedback has been positive with participants reporting that patient engagement has been strengthened, and that the patient voice is heard, valued and supports committee members in making fully informed decisions. PG representatives expressed a willingness to participate again. Feedback also highlighted that the preparatory support offered to PG representatives by the public involvement team is highly valued.ConclusionsPatient group participation in committee meetings has been received positively by PG representatives. They report that discussions relating to quality of life impact of medicines on patients and carers better reflect the lived experience, enriching committee's deliberations. This demonstrates SMCs commitment to openness and transparency and has strengthened patient engagement in our processes.
OP84 Collaborative Program To Improve Early Management Rheumatoid Arthritis
Authors: Dartnell Jonathan , London Jane , Elliott Jeffrey , Hill Catherine , Buchbinder Rachelle 
Published: December 2019

IntroductionOptimal rheumatoid arthritis (RA) management requires coordinated management and consistent communication by health practitioners with patients. Suboptimal methotrexate use is a factor leading to increased use of biological disease modifying antirheumatic drugs (bDMARDs), which account for significant government drug expenditure. A multidisciplinary co-design approach was used to develop and implement a program aiming to improve early management and quality use of medicines (QUM) for people with RA in Australia.MethodsLiterature review and key informant interviews identified broad potential QUM issues in RA management. An initial exploratory multidisciplinary meeting prioritized QUM issues, identified audiences and perspectives, and scoped focus areas to address with education. Iteratively through co-design meetings and activities, program objectives were agreed, barriers and enablers for change explored, characteristics of intervention activities considered and rated, and program products developed and reviewed. Program evaluation included participation and distribution data, surveys and interviews, and analyses of general practice and Pharmaceutical Benefits Scheme (PBS) data.ResultsQUM issues addressed include: (i) timely initiation of conventional synthetic (cs) DMARDs; (ii) appropriate use and persistence with csDMARD therapy, especially methotrexate; and (iii) clarity around professional roles and best practice for prescribing, dispensing, and monitoring DMARDs, and managing lifestyle factors and other risks associated with RA. The educational program (October 2017 to June 2018) included: an article promoting key messages (email to ~115,000 health practitioners), prescriber feedback report based on PBS data (to all Australian rheumatologists), an RA action plan (completed by health practitioners for consumers), an interactive case study (553 participants), visits to 1200 pharmacies promoting key messages, a multidisciplinary webinar (431 live and 366 on-demand), fact sheets for consumers available through MedicineWise app (medicine management app for consumers), and social media activity.ConclusionsA multidisciplinary co-design process has provided a model for developing a multifaceted QUM program incorporating and addressing multiple perspectives.
OP93 Collaboration Between Health Technology Assessment And Procurement: A Rapid Mixed-Methods Study
Authors: Harkin Kathleen 
Published: December 2019

IntroductionThe Irish Health Service (HSE) Health Technology Assessment Group (HTAG) aims to maximise the impact of its work by collaborating with HSE Procurement, formalised through an evidence-based Memorandum of Understanding (MOU). This study aims to inform the MOU.MethodsA sequential mixed-methods study design was used. A rapid review of the literature identified no substantive body of evidence on collaboration between independent national health technology assessment (HTA) and procurement bodies. Personnel involved in HTA or procurement were invited by email to complete a survey, take part in an interview, or both. The quantitative and qualitative data were analysed using descriptive statistics and thematic analysis, respectively. Findings were integrated using a conceptual framework that examined the complementarity of HTA and procurement processes relevant to an MOU.ResultsThirteen surveys were completed (response rate was 13 percent). Eleven interviews (five Ireland, two Canada, three UK, one New Zealand) were conducted between August and November, 2017. No formalised collaboration between independent national HTA and procurement bodies was identified. However in New Zealand, HTA and procurement are an integrated function of the Pharmaceutical Management Agency (PHARMAC). In other jurisdictions, successful ad hoc collaborations occurred where there was a clear need expressed by Procurement for additional evidence required for decision-making, and where HTA personnel tailored their research approaches accordingly. Key themes to successful collaboration were relationships, communication, clear roles, rigorous research and ‘system support’. Good individual relationships and ready access/communication promoted successful outcomes. Successful outcomes included improved clinical practice, and major cost savings. Collaboration may be focussed on: innovative or established devices; specific types of HTA/research products; specific categories/specialties; or specific procurement departments.ConclusionsAll participants considered collaboration to be beneficial but requiring good relationships and ‘system support’. Furthermore, successful collaboration requires clarity regarding the purpose, parties involved, their roles, responsibilities, modes of communication, information to be shared, and the expected outcomes.
OP98 Limitations In Health-Economic Guidance For Medical Devices
Authors: Blüher Maximilian , Mittard Virginie , Torres Rafael , Saunders Rhodri 
Published: December 2019

IntroductionHealth technology assessment (HTA) includes consideration of health and economic factors, playing a key role in optimizing healthcare provision in Europe. Medical devices are an important contributor to both health outcomes and the cost of healthcare provision, yet they are rarely addressed in current guidance for health-economic evaluation. Our aim is to help improve assessment of medical devices via review of European health-economic guidelines and recent research.MethodsSearches for European HTA guidelines were performed and where available were reviewed by two researchers working independently. Additionally, a systematic review of published literature focused on assessment of medical devices was conducted. English, German, or French literature published between 2000 and 2017 was analyzed. The status of HTA guidance to date was subsequently reviewed in light of current research findings and suggestions made to help improve standardization.ResultsOf the 41 investigated European countries, 22 had official HTA guidance. Only four of 22 (18 percent) dedicated documentation to guidance specific to medical devices. Where differences between pharmaceuticals and medical devices were highlighted, specifics for health-economic assessment of medical devices were generally absent. The systematic review yielded 472 unique articles, 28 of which underwent full-text review. Issues surrounding medical device value assessment that commonly emerged were: limited evidence base, learning curve effects, organizational impact, incremental innovation, diversity of devices, dynamic pricing, and transferability. While identification of issues was ubiquitous, actionable suggestions on how to overcome them were less common. The most frequent recommendations were use of Bayesian methods, inclusion of real-world data, and modelling the learning curve. Key to implementation is determination of the medical device type and its impact duration.ConclusionsCurrent guidelines rarely address the needs of medical devices. Practical recommendations for improvements exist and provide opportunity to start discussion on how best to serve the medical devices field and improve the HTA process.
OP137 Translating Results From Clinical Audit Studies To Local Context
Authors: Moga Carmen , Chojecki Dagmara , Guo Bing 
Published: December 2019

IntroductionDespite widespread use of oxygen (O2) therapy, there is relatively little available information on routine O2 administration and monitoring; this is an issue particularly when considering the potential risks associated with inappropriate O2 utilization. A rapid health technology assessment (HTA) was conducted to inform the Respiratory Health Strategic Clinical Network Oxygen Summit in Alberta on aspects related to current practice in the use of O2 therapy in acute care, including administration, safety and quality, and inappropriate practice. Clinical audit is a tool used to determine deviations in practice and to identify opportunities for improvement. The objective of this presentation is to describe the experience and lessons learned from including clinical audit studies in the rapid HTA.MethodsA standardized rapid review approach was used to identify, select, and synthesize evidence from studies published in English from 2005 to 2016. A supplementary literature search conducted in 2018 provided additional background information on the value, applicability, and limitation of using results from clinical audit studies to inform questions of good practice.ResultsTwenty-four clinical audit studies on O2 therapy were identified; the majority were conducted in the United Kingdom. The studies varied in design, methodology, and data and outcomes reporting. Ten studies investigated the appropriateness of O2 therapy prescription pre- and post-implementation of local initiatives and interventions, which helped pinpoint major gaps in current practice, and identified general recommendations for improvement of practice. A list of reporting criteria is proposed for improving the reporting of clinical audit studies results.ConclusionsConducting clinical audit studies is resource-intensive. In the absence of other research evidence and local practice data, translating results from clinical audit studies conducted in other jurisdictions, while challenging, can help address appropriateness questions. However, inferences from these studies may be suitable only for certain topics or an operating context.
OP138 Stakeholders’ Involvement When Developing A mHealth Assessment Tool
Authors: Puig Elisa Puigdomenech , Ferrer Elisa Poses , Masana Lina , Espallargues Mireia 
Published: December 2019

IntroductionDue to the specific characteristics and challenges of mobile health (mHealth) technologies there is a need to have assessment tools based on their particularities to be used by health technology assessment (HTA) agencies and evaluation experts. In the development of a comprehensive and practical evaluation tool for the evaluation of mHealth solutions we aimed to include the views and opinions of key stakeholders: health professionals, developers, hospital managers, HTA agencies, patients and general public.MethodsFocus groups and an online modification of the Delphi technique are being used to discuss and agree on domains and criteria to be included in the mHealth assessment tool. Domains and criteria used for health apps evaluation were drawn from a literature review on the topic. The initial list includes 95 criteria grouped into the following domains: purpose of the app, privacy and security, clinical effectiveness, content of the intervention, user experience and usability, interoperability, expenses, impact on the organization, and legal and ethical aspects. Data coming from focus groups is currently being analyzed from a thematic and content analysis perspective.ResultsFocus groups with professionals have showed that the most important domains to be considered when evaluating health apps are those related with security, user experience, and clinical effectiveness. Some criteria were considered to be mandatory (mainly regarding safety issues), on which a first step assessment should indicate whether the app ‘pass or fails’ for the subsequent throughout assessment. Focus groups with patients will provide insight on critical aspects related to the choice, use and adherence to a health app.ConclusionsInsights from main stakeholders on the design of the tool for mHealth assessment are relevant and complementary between them. Next steps include (i) the agreement of criteria by using an online modification of the Delphi Technique and (ii) piloting of the tool.
OP157 Carbon Ion Radiotherapy: A Systematic Review
Authors: Goetz Gregor , Mitić Marija , Mittermayr Tarquin , Wild Claudia 
Published: December 2019

IntroductionDue to the promising physical dose distribution of carbon ion radiation therapy (CIRT), CIRT can be regarded as a novel tumor irradiation technique and is sometimes considered as a breakthrough therapy for various tumor types. However, it is unclear whether superiority or inferiority can be claimed when compared to standard irradiation. This study aimed to assess the scientific evidence regarding the effectiveness and safety of CIRT.MethodsA systematic literature review was conducted using the European Network for Health Technology Assessment (EUnetHTA) Core Model® for rapid relative effectiveness assessment. The literature search for clinical outcome studies on CIRT was performed in four databases [Cochrane (Central), Centre for Research and Dissemination (CRD), Embase and OVID MEDLINE]. The risk of bias was assessed using the Cochrane Risk of Bias Tool (for randomized controlled trials) and the Institute of Health Economics (IHE) Checklist (for observational studies). The evidence synthesis was restricted to 12 tumor regions (and 54 indications) and studies with a low or moderate risk of bias, published between 2005 and 2017.ResultsIn total 27 studies were eligible for the qualitative synthesis of the evidence regarding the effectiveness and safety of CIRT; one randomized controlled trial that primarily focused on the feasibility of CIRT, three case-control studies, three before-after studies focusing on quality of life, and 20 further case series studies. Overall, insufficient scientific evidence was found for 13 (out of 54) indications in seven tumor regions and no scientific evidence was found for 41 (out of 54) indications.ConclusionsTheoretically, CIRT is undoubtedly a promising cancer treatment. To date, however, 54 oncologic indications in 12 tumor regions under investigation lack randomized controlled trials assessing the long-term effectiveness and harms associated with its use. CIRT must be considered as an experimental treatment due to the lack of high-quality clinical research.
PP14 Budget Impact Of Sapropterin Dihydrochloride For Phenylketonuria
Authors: Mulinari Eduardo , Brito Nayara Castelano , Marra Lays Pires 
Published: December 2019

IntroductionThe National Committee for Health Technology Incorporation (CONITEC) evaluates health technologies to recommend their inclusion or exclusion within the Brazilian Public Health System (SUS), and uses the budget impact assessment to estimate costs to the system. The Ministry of Health (MS) guideline recommends treatment of phenylketonuria (PKU) with restricted phenylalanine diet and phenylalanine-free amino acid formula (PFAAf) supplementation. CONITEC evaluated the inclusion of sapropterin dihydrochloride for PKU in the SUS.MethodsThe population eligible for treatment was evaluated by the number of patients receiving PFAAf between 2014 and 2017 registered in the SUS. Patients were stratified by age/weight and a simple linear regression was performed to estimate the future population. The costs of treatment and testing the responsiveness of sapropterin dihydrochloride were estimated according to the recommended dosage guideline of the MS, leaflet and public purchasing prices. A univariate deterministic sensitivity analysis was performed to evaluate different prices, responsiveness test methods and variations in the reduction of formula use.ResultsThe incorporation of sapropterin dihydrochloride would generate an incremental budget impact in the SUS of around BRL 79 million (USD 21.7 million) in 2019 and BRL 300 million (USD 82.1 million) in five years (2019-2023). The univariate sensitivity analysis estimated that the incremental budget impact could be between BRL 66 and BRL 103 million (USD 18 and USD 28 million) in the first year and between BRL 251 and BRL 388 million (USD 69 and USD 106 million) in five years. Sensitivity analysis showed that the price of sapropterin dihydrochloride was the most sensitive variable in the model.ConclusionsThe incorporation of sapropterin dihydrochloride in the SUS represents a significant budgetary impact and covers a small number of patients. Sapropterin dihydrochloride was recommended by CONITEC for the treatment of women with PKU, with a positive drug responsiveness test, and who are in the preconception period or in the gestational period.