bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP83 A Conceptual Decision-Making Framework For Pharmaceutical Innovations
Authors: Boersma Cornelis , Geenen Joost , Postma Maarten 
Published: December 2019

IntroductionThe trend of growing healthcare expenditures is unsustainable in many countries. The increasing pressure on healthcare budgets due to, for example, population ageing, increasing numbers of patients with chronic diseases (including multimorbidity), and the introduction of new pharmaceutical innovations, leads to political and societal debate. In particular, the introduction of expensive pharmaceutical innovations causes a lot of discussion and uncovers various paradoxes and dilemmas. There is a societal demand for innovation focused on existing medical needs (e.g., oncological, immune-mediated inflammatory, and orphan diseases), but the price of pharmaceutical innovations is a barrier to patient access. As a consequence, systems try to introduce measures or incentivize market forces to improve access for patients, while also containing budget impact. This does not always lead to better access and affordability. The aim of this study was to develop and test a conceptual decision-making framework for pharmaceutical innovations.MethodsA retrospective study was conducted to identify the successes and challenges of decision-making systems across Europe. A conceptual decision-making framework, including proposed procedures, criteria, and health technology assessment (HTA) requirements (including tools), was developed and tested based on specific case examples (e.g. oncology and hepatitis C).ResultsThe conceptual decision-making framework comprised an algorithm for relevant decision-making criteria (e.g. clinical evidence, medical need, cost-effectiveness, and budget impact). The algorithm was developed hierarchically and ranked the criteria in order to optimally inform various types of investment decisions. This novel approach to conducting budget impact analyses resulted in more realistic predictions of the burden of pharmaceutical innovations on healthcare budgets, and can be used as part of horizon-scanning processes to inform healthcare decision making. Results from selected case examples are presented.ConclusionsThe conceptual decision-making framework and proposed method for budget impact predictions will allow for more balanced future healthcare investment decisions.
PP84 Different Interpretation Of Evidence By A Health Technology Assessment Body And A Decision Maker
Authors: Dintsios Charalabos-Markos , Ruof Jörg , Worm Franziska , Herpers Matthias 
Published: December 2019

IntroductionWithin early benefit assessment of pharmaceuticals in Germany, addenda can be commissioned by the Federal Joint Committee (FJC) to the health technology assessment (HTA) agency, mainly as a result of a hearing. Our aim was to analyze the issues for and impact of commissioned addenda, as well as the agreement between HTA agency recommendations and FJC decisions.MethodsAll available relevant documents on addenda commissioned up to the end of 2017 were screened and their essential content extracted. Differences between the HTA agency and FJC recommendations were tested, and concordance was analyzed using agreement statistics (Cohen's kappa and Fleiss’ kappa).ResultsMost of the 90 addenda commissioned up to the end of 2017 concerned oncological products. In all contingent comparisons, positive changes in added benefit or evidence level on a subpopulation basis (n = 124) were more common than negative changes. Agreement of assessments, addenda, and appraisals reached a moderate strength for added benefit (Fleiss’ kappa 0.47, range 0.41 - 0.54). Overall agreement between addenda and appraisals on a binary nominal basis was poor for added benefit (Cohen's kappa 0.18, range 0.01 - 0.36) and fair for evidence quality (Cohen's kappa 0.35, 0.19–0.52). Cohen's kappa ranged from “less than by chance” (respiratory diseases) to “perfect” (neurological diseases), but was only statistically significant for neurological and other diseases. Three addenda are presented in detail as examples.ConclusionsAddenda have a high impact on decision-makers’ appraisals, offering additional analyses of supplementary evidence submitted by the manufacturers. Nevertheless, the agreement between addenda and appraisals varies, highlighting different methodological approaches and decision-making factors between the HTA agency and the FJC.
PP86 Reimbursement of Combination Oncology Products: Can Two (Companies) Tango?
Authors: Turkstra Erika , Liu Lok Wan , Berardi Andrea , Macaulay Richard 
Published: December 2019

IntroductionA range of innovative, targeted anti-cancer therapies have been developed over the past 20 years. More recently, companies have been developing combinations of these drugs. While this promises substantial efficacy benefits, dual-brand oncology therapy combinations may potentially create substantial economic burden. Obtaining a positive health technology assessment (HTA) recommendation and public reimbursement can be a major challenge, and may be more difficult when each constituent monotherapy is marketed by a different company. We evaluated whether dual-brand oncology therapies developed by a single manufacturer had faster or better outcomes than those developed by two separate manufacturers.MethodsRecent combination oncology drug products were screened in November 2018 to identify whether one or two manufacturers were involved. The websites of various HTA organizations were screened and the relevant data extracted.ResultsA total of 78 recommendations for dual-brand oncology treatments were identified across the HTA agencies screened: 26 of these were for combinations by the same manufacturer and 52 were for combinations with two manufacturers. Dual-brand therapies developed by a single manufacturer were more likely to receive full or optimized/conditional recommendations (58% “recommended” and 12% “optimized/conditional”) than those marketed by two separate manufacturers (42% “recommended” and 8% “optimized/conditional”). Dual-brand therapies with two manufacturers were more likely to receive negative HTA recommendations than those marketed by a single manufacturer (50% versus 31%). However, the median time from marketing authorization to recommendation in European countries was the same (6 months), regardless of whether each constituent monotherapy was marketed by one or two manufacturers.ConclusionsHTA agencies were more likely to issue negative recommendations for dual-brand oncology treatments marketed by two separate companies, compared with those marketed by a single company. A single company may have more flexibility in price setting, which may facilitate more positive HTA recommendations.
PP89 Cost Effectiveness Of Hepatitis A Vaccination In India
Authors: Gurav Yogesh , Bagepally Bhavani Shankara , Thavorncharoensap Montarat , Chaikledkaew Usa 
Published: December 2019

IntroductionDue to epidemiological transition, a rise in hepatitis A outbreaks among adults in the state of Kerala, India has been noted. This has intensified the need for hepatitis A vaccination (HAV), but evidence regarding the cost effectiveness of HAV, which is essential to guide policy decisions, is lacking. This study was undertaken to evaluate the cost effectiveness of HAV among adults in Kerala state.MethodsTo determine the cost effectiveness of HAV from a societal and a payer perspective, a Markov model was constructed with a cycle length of two months. The lifetime costs and outcomes for HAV and no vaccination were compared using a discount rate of 3 percent. Data for the model input parameters of cost, coverage, and effectiveness were derived from the published literatures. One-way and probabilistic sensitivity analyses were applied. A threshold based on the per capita gross domestic product (GDP) was used (1 GDP = INR 127,702.48 [USD 1,886.03]).ResultsThe incremental cost-effectiveness ratios for both societal and payer perspectives were negative, indicating that HAV was dominant, being less costly and more effective than no vaccination. The discount rates and utility values for adults with HAV were the most sensitive parameters.ConclusionsA HAV strategy would be cost-saving, compared with no vaccination, in the Kerala state of India.
PP121 How To Involve Patients In Decisions About Antibiotic Prophylaxis After Tick Bite
Authors: Bouchard Sylvie , Gernigon Gaelle , Karam Fatiha , Daigle Jean-Marc , Morrow Genevieve , Freitas Adriana , Tardif Mélanie 
Published: December 2019

IntroductionAntibiotic prophylaxis with a single dose of doxycycline after a tick bite is one of the tools for preventing Lyme disease, which is becoming increasingly prevalent in Quebec. The aim of this work was to revisit this practice in adults and children younger than 8 years of age.MethodsTo assess the safety and absolute risk reduction (ARR) of doxycycline for preventing Lyme disease in contraindicated populations, two systematic reviews were conducted with a re-analysis of the original efficacy data. A knowledge mobilization framework was used to consider the scientific, contextual, and experiential evidence, taking into account information on patients’ and clinicians’ experiences.ResultsA single dose of doxycycline prescribed within 72 hours of being bitten by a tick (Ixodes scapularis) could prevent cutaneous manifestation of Lyme disease (ARR -2.8%, 95% confidence interval: -11.7–6.1; p = 0.06), without serious side effects, provided that the bite occurred in a geographical region where at least 25 percent of nymph and 50 percent of adult ticks are infected with the disease. However, the level of evidence was low and its generalizability to other contexts was doubtful. The decision to prescribe antibiotic prophylaxis may be based more on the fear of Lyme disease, rather than on effectiveness data and the real risk of contracting Lyme disease.ConclusionsIt may be challenging for clinicians to discuss Lyme disease prophylaxis with patients and their families in contexts where people are fearful of the disease, and the risk of contracting it from a tick bite is uncertain. Decision aids that provide scientific evidence on the real risk of developing Lyme disease after a tick bite, particularly in Quebec, can promote informed decisions based on patient preferences and values by supporting discussion between clinicians and patients.
PP170 Quantifying the Life-Cycle Value of Second Generation Antipsychotics
Authors: Berdud Mikel , Wallin-Bernhardsson Niklas , Zamora Bernarda , Lindgren Peter , Towse Adrian 
Published: December 2019

IntroductionWe estimate the life-cycle value of risperidone – Second-Generation Antipsychotics (SGA) – to balance the view that cost per Quality-Adjusted Life Year (QALY) estimates at launch are enough to guide access decisions. Study results will also drive discussion on access and price to recognize the dynamic nature of pharmaceutical pricing over the long-run.MethodsWe estimated number of patients treated for schizophrenia with risperidone in Sweden and the United Kingdom (UK) between 1994-2017 based on usage data form national statistics and volume sales data from IQVIA. We collected data from literature on the effectiveness (QALYs) and costs (EUR 2017) of risperidone (SGA) and haloperidol – First-Generation antipsychotic (FGA). We estimate the life-cycle value added by risperidone versus haloperidol, and the life-cycle distribution of the social surplus between the payer (consumer surplus) and the innovator (producer surplus).ResultsWe estimated the consumer surplus, the producer surplus, the Net Monetary Benefit (NMB) and Incremental Cost-Effectiveness Ratio (ICER) at each year and in aggregate terms (1993-2017). For the UK the producer surplus was ~28 percent out of the total surplus before patent expiration and five percent after patent expiration. In Sweden, producer surplus was around 6 percent out of the total surplus before patent expiration and one percent thereafter. In both countries, during the life-cycle of risperidone, the NMB per patient increased and the ICER decreased as a response to: (i) the launch of Risperidone Long-Acting Injectable (RLAI); and (ii) the generic entry.ConclusionsThe value added by risperidone increased during the life-cycle due to the launch of RLAI and the generic competition. This suggests that, considering the entire life-cycle, the value added by SGAs to the system is higher than the expected value estimated using cost-effectiveness analysis at launch. Pricing and reimbursement decisions should take into account the dynamic nature of pharmaceutical markets and the value added by innovative medicines over the long-run.
PP179 Health Technology Assessment Of Pediatric Intensive Care Ventilators
Authors: Di Mauro Roxana , Faggiano Francesco , Andellini Martina , Derrico Pietro , Ritrovato Matteo 
Published: December 2019

IntroductionA health technology assessment (HTA) process to evaluate the best intensive care ventilator manufacturers has been carried out in different pediatric intensive care units (ICUs) of Bambino Gesù Children's Hospital (OPBG). The purpose of this study is to determine: (i) the most relevant features of a ventilator to be considered between different manufacturers, and (ii) the methodology to conduct the assessment to support the decision-making process about the choice to adopt the suitable technology for OPBG.MethodsThe decision-oriented HTA method (Do-HTA), developed by the HTA unit of OPBG, was applied to conduct the assessment. Do-HTA involves the integration of the European Network for HTA (EUnetHTA) CoreModel and the Analytic Hierarchy Process with the support of an informatics tool. It provides the definition and numerical evaluation of assessment parameters to evaluate the performance of technologies. A literature review involving ICU professionals was used to define and weight the assessment elements on clinical, technical, organizational, economic, and safety domains. In particular, a subgroup of these domains has been included in a checklist for the comparative evaluation of different ventilator models, each of which was tested in three independent runs performed in three different ICUs.ResultsResults show that safety and clinical effectiveness had highest the impact within the evaluation, followed by organizational, technical and economic aspects. A percentage value per each ventilator has been assigned, representing the global performances regarding the assessment elements.ConclusionsThis study presents and discusses the benefits and drawbacks of innovative features of ventilators, all characteristics to be taken into account during the evaluation process and a methodology to conduct it. The project identified the best performing ventilator model through a collective decision, giving a reliable recommendation to the Hospital Decision Makers.
PP186 Telemonitoring With Pacemakers For Patients With Heart Failure
Authors: Myles Susan , Poole Ruth Louise , Facey Karen 
Published: December 2019

IntroductionEvidence supporting the use of pacemakers is well established. However, evidence about the optimal use of pacemaker telemonitoring for disease management in heart failure is not. Health Technology Wales (HTW) held a national adoption event to encourage implementation and best practice in use of pacemaker telemonitoring in the National Health Service (NHS) Wales to improve patient outcomes in heart failure.MethodsMulti-stakeholder national adoption workshop using a mixture of expert presentations, case studies and interdisciplinary group and panel discussions to agree key actions to understand the value and promote optimal use of pacemakers for remote disease monitoring in patients with heart failure in Wales.ResultsThe workshop was attended by forty-five senior professionals with an interest in improving care of patients with heart failure. Actions to progress included: providing a centralized Welsh system to support technical issues that arise with telemonitoring; considering interoperability with other NHS Wales systems; encouraging value-based procurement with collection of a core outcome set; agreeing implementation issues with both professionals and patients; audit to understand experience, resource use and outcomes; and sharing manufacturer evidence on the accuracy of telemanagement algorithms. It was suggested that these actions be progressed via an All-Wales multi-stakeholder approach, led by the Welsh Cardiac Network.ConclusionsDeveloping a more agile, lifecycle approach to technology appraisal is currently advocated; recalibrating the focus from technology assessment to technology management across the complete technology lifecycle. HTW will endeavour through regular adoption events to facilitate such a paradigm shift that aims to understand value and optimise use of evidence-based technologies.
PP187 Robotic Surgery, Any Updates?
Authors: Andellini Martina , Di Mauro Roxana , Faggiano Francesco , Derrico Pietro , Ritrovato Matteo 
Published: December 2019

IntroductionThis work aims to update the previous robotic surgery health technology assessment (HTA) study conducted in 2013 in Bambino Gesù Children's Hospital. The study, focused on the evaluation of the newest evidence that have emerged over the last three years, aims to identify if there are new perspectives and advantages of introducing this technology in the hospital.MethodsDecision-oriented HTA (DoHTA) method was applied to conduct the assessment. It involved the integration of the European Network for HTA Core Model® (version 3.0) and the analytic hierarchy process providing the definition and the numerical evaluation of assessment parameters through which it is possible to evaluate the performance of the technologies compared. Three years after the first technology's evaluation, an updated literature review was conducted, using the same 2013 key words, to identify changes in the indicators’ performance score. The performance values have been updated through a quantitative and qualitative evaluation of data gathered from the literature review, expert opinion and context analysis. The global weights’ system, developed in 2013, has not been updated because the relative importance of each domain remained unchanged. The performance values of safety, efficacy, costs, and social aspects have been estimated, identifying the differences in terms of percentage values in comparison with the previous study.ResultsResults showed a slight improvement on safety and organizational aspects in robotic surgery; however, clinical effectiveness and economic, social and legal aspects remained unvaried. More specifically, it has been registered a 3 percent reduction of the difference of the distance between robotic and laparoscopic performance values (2013: 14, 15 percent; 2017: 11, 29 percent).ConclusionsResults highlighted a slight improvement in robotic surgery performances even if it confirmed the previous results for which the laparoscopic system outperformed the others and currently is keeping the best performance techniques. Finally, sensitivity analysis and a Monte Carlo simulation were carried out proving the stability and reliability of the solution.
PP193 How Does HTA Address Social Expectations Now? An International Survey.
Authors: Gagnon Hubert , Bellemare Christian , Legault Georges-Auguste , Bédard Suzanne K. , Béland Jean-Pierre , Bernier Louise , Dagenais Pierre , Daniel Charles-Etienne , Tapin Danielle , Parent Monelle , Patenaude Johane 
Published: December 2019

IntroductionAfter surveying its members on ethical issues (2003), the International Network of Agencies for Health Technology Assessment (INAHTA) mandated its Ethics Working Group (2005) to reflect on the role of health technology assessment (HTA) organizations in meeting social expectations. Some aspects of these have since been clarified by two studies addressing either the official position of INAHTA's members or the publication authors. An international survey was carried out on the perception of HTA professionals’ expectations when producing HTA reports: how to fulfil HTA's social role, which value judgments should be made explicit and what should be the status of ethical analysis.MethodsA twenty-two question, web-based, anonymous survey was devised from our recent systematic review on the integration of ethics into HTA and carried from April to July 2018. The information on 328 HTA agencies/contact persons from seventy-five countries was collected from the website of INAHTA, Health Technology Assessment International (HTAi), the European Network for Health Technology Assessment (EUnetHTA), EuroScan International Network, the HTA Network of the Americas (RedETSA) and the HTA Network of Asia (HTAsiaLink), a 2015 World Health Organization survey, HTAi members, and our local HTA network (Québec, Canada).ResultsEighty-nine participants completed and submitted a finalized survey for a 27 percent participation rate representing thirty-three countries. Regarding how the HTA reports should fulfil their social role, our results showed that over 84 percent of the respondents agreed upon the necessity to address it to decision makers, patients and citizens. At a lower and more variable level, the same result was found about the necessity to make value judgements explicit in different sections of the report, including ethical analysis. This contrasts with the variability of responses obtained on the status of ethical analysis although an agreement on the expertise required was observed. Variability in the usefulness of patient, public or stakeholder participation was observed.ConclusionsAt the dawn of this decade, this study reveals high expectations on context-dependent decisions in HTA: the necessity to integrate the ‘explicitation’ of value judgements and systematic ethical analysis to fulfil HTA's social role.
PP194 Intersectoral Costs And Benefits In The Societal Perspective
Authors: Drost Ruben , van der Putten Inge , Ruwaard Dirk , Evers Silvia , Paulus Aggie 
Published: December 2019

IntroductionMany health care interventions have costs and benefits that spill over to sectors outside the healthcare sector. Little is known about these inter-sectoral costs and benefits (ICBs). However, to achieve an efficient allocation of scarce resources, insights on ICBs are indispensable. The main objective of this study was to identify the ICBs related to health care and provide a sector-specific classification scheme for these ICBs. For this sector-specific classification scheme mental disorders were taken as an example, as we expect that this is the sector with the most ICBs.MethodsUsing PubMed, a literature search was conducted for ICBs of mental disorders and related (psycho)social effects. A policy perspective was used to build the scheme's structure, which was adapted to the outcomes of the literature search. In order to validate the scheme's international applicability inside and outside the mental health domain, semi-structured interviews were conducted with (inter)national experts in the broad fields of health care.ResultsThe searched-for items appeared in a total of fifty-two studies. The ICBs found were classified in one of four sectors: “Education”, “Labor and Social Security”, “Household and Leisure” or “Criminal Justice System”. Psycho(social) effects were placed in a separate section under “Individual and Family”. Based on interviews, the scheme remained unadjusted, apart from adding a population-based dimension.ConclusionsThis is the first study which offers a sector-specific classification of ICBs. Given the explorative nature of the study, no guidelines on sector-specific classification of ICBs were available. Nevertheless, the classification scheme was acknowledged by an international audience and could therefore provide added value to researchers and policymakers in the field of health technology assessment. The identification and classification of ICBs offers decision makers supporting information on how to optimally allocate scarce resources. By exploring a new area of research, which has remained largely unexplored until now, the current study has an added value as it may form the basis for the development of a tool which can be used to calculate the ICBs of health care interventions.