Poster Presentations
PP165 Content Instead Of Orders: Experiences Of Launching A Knowledge Base
- Gergő Merész, Bence Takács
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- Published online by Cambridge University Press:
- 31 December 2019, p. 68
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Introduction
In Hungary, the procedure for health technology assessment of innovative pharmaceutical products allows 13 assessors 43 calendar days to evaluate reimbursement submissions. These short timelines have created a need for smart capacity building, namely, streamlining the scientific evaluation process while making sure that the quality of the critical appraisals remain high. The objective of this study was to present and evaluate the implementation of an online knowledge base to distill community knowledge, and also for management purposes.
MethodsThe scope and the content-, functional-, and technical specification was developed, and information technology security requirements were identified during the pre-implementation phase. An existing platform was chosen for adaptation, ensuring that descriptive follow-up data is available on uptake for monitoring purposes. Both the adaptation and maintenance were carried out internally by the Department of Health Technology Assessment at the National Institute of Pharmacy and Nutrition.
ResultsThe key requirements identified when developing the specification were searchability, low maintenance need, low operating costs and attractivity for users. An already existing open-source, flat file content management system was chosen for adaptation. In terms of content, a health technology assessment handbook, process documentation, a news bulletin section was created, and corporate identity elements were added. Since the start of the service in September 2018, the number of total daily page downloads to the knowledge base varied between four and 1,193 (average 205 per day), with the assessment handbook topping the overall page visit statistics.
ConclusionsThe implementation of this knowledge base enables the Department of Technology Assessment to rely more on the formalized community knowledge when carrying out critical appraisal, while enabling better knowledge and quality management. Uptake remains an issue on the long run, indicating a need for continuous content development.
PP166 A Mobile Clinical Decision Support System for Autism Spectrum Disorder
- Noemí Robles, Carme Carrion i Ribas, Montserrat Pàmias, Isabel Parra, Jordi Conesa, Antoni Perez-Navarro, Marc Alabert, Marta Aymerich
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- Published online by Cambridge University Press:
- 31 December 2019, pp. 68-69
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Introduction
eHealth is a new approach for managing several health conditions, but up to now not so many interventions have shown their efficacy/effectiveness. The AUTAPP Project tries to add knowledge in eHealth interventions targeted to Mental Health disorders, specifically Autism Spectrum Disorder (ASD) management that requires complex interventions that integrate different psychosocial interventions. AUTAPP aims to develop an evidence based Clinical Decision Support System (CDSS) using mobile technology for improving the decision process on psychosocial therapies in ASD. This study aimed to identify recommendations on which the algorithm of the CDSS will be developed.
MethodsA systematic review (November 2009-November 2018) was carried out to identify the existing scientific evidence published in relation to the effectiveness of: (i) early detection protocols; (ii) assessment tools; (iii) existing non-pharmacological therapies. Main databases were consulted (PubMed, Cochrane Library, PsychoInfo). Articles were reviewed by two independent reviewers. The quality of included publications and recommendations were assessed according to SIGN criteria.
ResultsA total number of 147 publications were included (477 identified): 96 for non-pharmacological therapies, 33 for assessment tools and eighteen for early detection. Regarding early detection and assessment, 12 recommendations were identified and six obtained the highest level (A), such as the convenience of multidisciplinary diagnosis teams and the usefulness of the Modified Checklist for Autism in Toddlers (M-CHAT) for ASD confirmation. For non-pharmacological therapies, 16 recommendations were collected. Those with higher levels of recommendations were family, environmental and educational (three As and one B). Interventions with lower levels of recommendation (C) were interventions which exercise, computers and neurological approaches.
ConclusionsThis systematic review allows both to identify gaps and opportunities in psychosocial interventions research and be the base for the CDSS algorithm. In the future professionals, careers and people diagnosed with ASD will validate the mobile CDSS.
PP170 Quantifying the Life-Cycle Value of Second Generation Antipsychotics
- Mikel Berdud, Niklas Wallin-Bernhardsson, Bernarda Zamora, Peter Lindgren, Adrian Towse
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- Published online by Cambridge University Press:
- 31 December 2019, p. 69
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Introduction
We 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.
PP171 Cost And Effectiveness Of Chronic Hepatitis C Treatment In Brazil
- Karin Hepp Schwambach, Mareni Rocha Farias, Giacomo Balbinotto Neto, Carine Raquel Blatt
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- 31 December 2019, p. 69
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Introduction
With the discovery of new direct-acting antivirals, the cure for hepatitis C appears to be a reality, but its high price and the availability of new antivirals are a major obstacle. In Brazil, treatments for hepatitis C have been available in the public health system since the 1990s, and in 2015 were made available the antivirals sofosbuvir, daclatasvir and simeprevir. The calculation of the budgetary impact of this merger estimated expenditures between 467 and 666 million Reais (USD 121 and 172 million) per year. This study aims to present and discuss the cost and effectiveness of hepatitis C treatment with direct-acting antivirals with or without alfapeginterferon and ribavirin, based on real-life data, and compare it with the world scenario.
MethodsWe analyzed the treatment data and outcomes of 253 patients from a retrospective cohort performed in a Specialized Care Service, in the city of Porto Alegre. In relation to costs, the direct costs of antiviral drugs, per unit (tablet), were considered according to financial receipts from public purchases. The total cost of the medications used by each individual in each treatment and the cost per cure obtained, expressed in Sustained Viral Response (SVR), were calculated.
ResultsMost patients (66.8 percent) had genotype 1 of the hepatitis virus and 92.9 percent achieved SVR. The mean total cost of treatment of patients with genotype 1 was USD 5,862.31 and USD 6,310.34 per cure; while in patients with genotype 3 the cost was USD 5,144.27 and USD 5,974.76 per cure. The cost with the most commonly used treatment regimen, sofosbuvir, daclatasvir and ribavirin was USD 5,961.25 and USD 6,536.46 per cure. These values were 30 percent lower than the values estimated at the time of drug incorporation.
ConclusionsCost and effectiveness data contextualize a real-life scenario in Brazil. The evaluated treatments presented good effectiveness, but high costs.
PP173 Is Early Modelling Too Late? Preventing Pitfalls And Optimizing Value
- Andrea Berardi, Richard Macaulay, Sukhvinder Johal
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- Published online by Cambridge University Press:
- 31 December 2019, pp. 69-70
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Introduction
Drug development is a risky business. Manufacturers are faced with the dilemma of whether or not to invest at any stage in the development process. Even once marketing authorization has been attained, payers are becoming increasingly demanding of evidence to justify price premiums in the face of increasing budgetary pressures. Cost-effectiveness is a critical decision-making criterion for many payers, and restrictions to sub-populations is common. Early economic modelling at very early phases of the development pathway can inform optimal investment decision-making, including go/no-go decisions and clinical trial design, particularly in population selection. To test the hypothesis of changing payer requirements, we carried out a study on the trends in reimbursement submissions where payers approved but ultimately restricted the population compared to the marketing license or the company's target population.
MethodsA systematic literature review of all single technology appraisals (STAs) by the National Institute for Health and Clinical Excellence (NICE) was carried (01/01/2006- to 16/11/2018). We used a linear regression model to examine the relationship between frequency of optimizations and time.
ResultsIn total, 357 STAs outcomes were identified, 55 percent were recommended and 26 percent were optimized. The proportion of optimized recommendations increased over time vs all other outcomes (p = 0.01), with more technologies being optimized over time (p < 0.01).
ConclusionsThe results indicate an increasing trend by NICE towards maximization of value through approval of drugs in select groups of patients. From a manufacturer's perspective, prediction of such outcomes at an early stage is fundamental for investment purposes and to maximize financial returns. An early stage model provides a framework to examine these issues as well as identifying data gaps, where real world evidence can be planned to support the value argument for products, and to inform clinical trial design through value of information analysis.
PP177 Health Preference Research In Europe: A Review Of Its Use
- Kevin Marsh, Axel Mühlbacher, Janine van Til, Christin Juhnke, Yookyung Christy Choi, Alejandra Duenas, Wolfgang Greiner, Kara Haas, Natalia Hawken, Mickael Hiligsmann, Kimberley Hockley, Ilya Ivlev, Frank Liu, Elizabeth Molsen-David, Elisabeth Oehrlein, Jan Ostermann, Thomas Poder
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- 31 December 2019, p. 70
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Introduction
Health Technology Assessment (HTA) and regulatory decisions involve value judgements. As patient groups, industry, and regulatory agencies conduct more preference studies to quantify these judgements, a better understanding of the methods and practices is needed. Currently, there is no systematic mapping of the use of preference data in Europe. This study aimed to identify (i) the use of quantitative preference data by all relevant HTA bodies and regulatory authorities of the European Union (EU) member states, and (ii) key standards and guidelines.
MethodsThis study used a mixed method approach based on a systematic literature review, survey and subsequent interviews with decision makers and experts.
ResultsA total of 62 survey responses were received. Many respondents reported that their agencies were responsible for supporting more than one type of decision, with 69.0 percent supporting approval decisions, 64.3 percent supporting reimbursement decisions, 61.9 percent supporting pricing decisions, and 64.2 percent supporting guideline development. Respondents reported that their agencies supported these decisions in multiple ways: 78.6 percent by assessing health technologies; 54.8 percent by appraising health technologies; 45.2 percent by compiling an HTA report; 7.1 percent by conducting primary research; 9.5 percent by conducting secondary research. More than 40 percent (42.9 percent) of agencies had the final say on one of the decisions of interest – approval, reimbursement, or pricing. Of the 31 countries studied, 71 percent (n = 22) used quantitative preference data in their reimbursement and pricing decisions. Of those, 86 percent (n = 19) used general population preferences to inform the estimation of quality-adjusted life years (QALY) as part of cost utility analysis.
ConclusionsMuch of this use of preference data can be understood within the standard framework of economic analysis adopted by many HTA agencies; either in in the form of: standard ways to estimate QALYs; ways to broaden the impacts of technologies captured in the QALY; or ways to weigh health gain with other decision-making criteria, such as disease severity or innovativeness.
PP178 Health Technology Assessment Of Laboratory Medicine
- Roxana Di Mauro, Francesco Faggiano, Martina Andellini, Pietro Derrico, Matteo Ritrovato
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- 31 December 2019, p. 70
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Introduction
Recent studies have investigated the re-organization and automation of laboratory medicine as a challenge for the hospital in terms of reduction of costs, turnaround time, workload, optimization of human and technological resources and improvement of safety. The purpose of this study was to conduct a health technology assessment (HTA) evaluation process about the possibility to re-organize and automate laboratory medicine at Bambino Gesù Children's Hospital.
MethodsThe decision-oriented HTA (Do-HTA) method, involving the integration of the European Network for HTA (EUnetHTA) CoreModel and the Analytic Hierarchy Process, was applied to assess the best technology solution. Twenty-one professionals were involved to define tender specifications related to the adaptation works of the new dedicated rooms, and the automatic technologies and organizational solutions for the new laboratory department. Finally, two manufacturer companies were consulted.
ResultsUsing Do-HTA, the study was focused on laboratory technologies while the infrastructure evaluation was conducted by the Engineering and Logistic Units of the hospital. Results showed that the total performance score of the first proposal was slightly higher (2.5 percent) than the second one, proving the comparable high qualitative level of both manufactures technologies. After an accurate analysis, evaluating all aspects (safety, clinical efficacy, cost, organization & technical criteria) and integrating the infrastructure evaluation, the decision has fallen upon the first company offer.
ConclusionsThis HTA project provided an in-depth examination of two proposed technological and organizational solutions. Thanks to the Do-HTA method, which produced and developed data and all needed information, it was possible to guide and assist the decision makers on the choice between the two technical solutions.
PP179 Health Technology Assessment Of Pediatric Intensive Care Ventilators
- Roxana Di Mauro, Francesco Faggiano, Martina Andellini, Pietro Derrico, Matteo Ritrovato
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- Published online by Cambridge University Press:
- 31 December 2019, pp. 70-71
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Introduction
A 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.
PP182 Natalizumab Therapy For Relapsing-Remitting Multiple Sclerosis
- Eva Fuchs
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- Published online by Cambridge University Press:
- 31 December 2019, p. 71
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Introduction
Multiple Sclerosis is a progressive, degenerating disease of the central nervous system (CNS), which affects more than 2.5 million people worldwide. The monoclonal antibody natalizumab (TysabriTM) has been approved by the European Medicines Agency in 2006. Yet, the treatment is associated with an increased risk of developing progressive multifocal encephalopathy (PML). The aim of the systematic review was to investigate whether natalizumab is more effective and safer than alternative pharmacological therapies or placebo over a prolonged period (≥ 36 months) with respect to annualised relapse rate (ARR), disability progression, quality of life and number of serious adverse events (SAEs).
MethodsA systematic literature search was conducted considering randomized controlled trials (no restriction in length) and prospective, non-randomized controlled trials. In terms of safety, prospective single arm studies were additionally included. The risk of bias (RoB) was assessed using the Cochrane RoB tool (RCT), the ROBINS-I tool (NRCT) and the Institute of Health Economics quality appraisal checklist (IHE-20) for case series (single-arm studies). The quality of evidence was determined using the GRADE-method (Grading of Recommendations, Assessment, Development and Evaluation).
ResultsFor the assessment of clinical effectiveness, three studies (one RCT and two NRCTs) met the inclusion criteria. No significant differences regarding the ARR and disability progression were detected, if natalizumab was compared to an alternative treatment with fingolimod. Yet, if compared to placebo or a group of natalizumab interrupters, a 70 percent reduction in the ARR was observed. For the assessment of safety, seven studies met the inclusion criteria. The proportion of patients suffering from SAEs ranged from 2.4 percent to 16.0 percent. In total, 35 cases of PML occurred. The results were supported by a very low quality of evidence.
ConclusionsFuture research should provide more head-to-head RCTs comparing natalizumab with other disease modulating drugs along with a comprehensive documentation of adverse events.
PP185 Clinical Papers: Which Are Ongoing Studies To Assess MHealth In 2020?
- Cyril Olivier, Estelle Piotto, Corinne Collignon, Hubert Galmiche, Chantal Bélorgey
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- Published online by Cambridge University Press:
- 31 December 2019, pp. 71-72
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Introduction
Mobile health systems (MHS) are one of the more spreading technologies in the field of medicine. However, identification of useful MHS is rather challenging. Few of them are, or could be, connected medical devices (cMD). Like other medical devices, cMD must be assessed to validate claimed benefits for reimbursement purposes. Clinical added value demonstration is a major criterion used to satisfy administrative requirements. With the increase of clinical studies that are including MHS, study registries can be used for insight into the type of evidence expected to become available in the near future.
MethodsIn 2018, the French National Authority for Health (HAS) performed a review of registered MHS clinical study designs. The Clinicaltrials.gov database was consulted for all studies indexed with the terms “mHealth” and “mobile health” for the search fields “study title”, “conditions” and “interventions”.
ResultsFour hundred and fifteen clinical studies were registered. Three hundred and eighty studies were interventional with most comprised of a randomized study design (75 percent). Fifteen had a crossover design. Only few observational studies (n = 35) were registered. These mainly concerned (59 percent) patient use of an app on a smartphone without any other device.
ConclusionsPatterns of clinical studies were not found to significantly differ between MHS and other medical devices. Most of the clinical studies were randomized and specific criteria to assess MHS could easily be identified. However, specific methodologies for clinical development are not used in practice for cMD health technology assessment. In the absence of validated and specific methodology for clinical development, current methods that are being used in these ongoing studies will nonetheless be generating evidence for the upcoming years.
PP186 Telemonitoring With Pacemakers For Patients With Heart Failure
- Susan Myles, Ruth Louise Poole, Karen Facey
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- 31 December 2019, p. 72
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Introduction
Evidence 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?
- Martina Andellini, Roxana Di Mauro, Francesco Faggiano, Pietro Derrico, Matteo Ritrovato
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- 31 December 2019, p. 72
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Introduction
This 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.
PP189 Filling In The Blanks: Is RWE From MAAs Used In NICE Decision Making?
- Lok Wan Liu, Adam Hall, Richard Macaulay, Sean Walsh
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- Published online by Cambridge University Press:
- 31 December 2019, pp. 72-73
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Introduction
The National Institute for Health and Care Excellence (NICE) may recommend temporary funding through managed access agreements (MAAs) for oncology drugs (via the Cancer Drugs Fund [CDF]) and highly specialized therapies for rare diseases. MAAs allow for the collection of evidence to address key areas of clinical uncertainty, while providing access of medicines to patients, prior to re-appraisal by NICE. Observational data and other real-world evidence (RWE) are crucial requirements for all MAAs and herein we examine the extent these data are being used to inform HTA decisions at re-appraisal.
MethodsExisting MAAs entered into between the National Health Service (NHS) England and manufacturers as of 30 October 2018 were identified; for drug:indication pairings with NICE re-appraisals, all information was reviewed and the key data extracted.
ResultsOf the twenty-two MAAs identified, only two drug:indication pairings have been subsequently re-appraised by NICE: BV(brentuximab vedotin):non-Hodgkin lymphoma (’recommended’) and pembrolizumab:relapsed or refractory classical Hodgkin lymphoma (’recommended’). Data from a retrospective questionnaire regarding the proportion of patients that received curative stem cell transplant (SCT) post-BV (from patients who received BV in the old CDF) were accepted to provide sufficient evidence on the post-BV SCT rate by NICE. Meanwhile, for pembrolizumab, long-term survival benefit was the key clinical uncertainty; the primary data collection source was updated phase III randomized controlled trial data. At re-appraisal no reference was made to the observational data component; more mature survival data reduced uncertainty over survival benefits and were sufficient to support a positive NICE recommendation.
ConclusionsOf the twenty-two MAAs to date, only two drugs have been re-appraised thus far, with both receiving positive NICE recommendations. Observational data were successfully used to address key clinical uncertainties regarding subsequent real-world treatment patterns for BV, but observational data were not referred to in the NICE recommendation for pembrolizumab. The re-appraisal of more drugs in the future will clarify the importance being placed on observational data collection requested by NICE for existing MAAs.
PP192 An Institutional Ethical Framework For HTA: Stakeholder Participation
- Mireille Goetghebeur, Olivier Demers-Payette, Marie-Pascale Pomey, Isabelle Ganache, Denis Roy
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- 31 December 2019, p. 73
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Introduction
In 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.
PP193 How Does HTA Address Social Expectations Now? An International Survey.
- Hubert Gagnon, Christian Bellemare, Georges-Auguste Legault, Suzanne K. Bédard, Jean-Pierre Béland, Louise Bernier, Pierre Dagenais, Charles-Etienne Daniel, Danielle Tapin, Monelle Parent, Johane Patenaude
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- 31 December 2019, p. 73
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Introduction
After 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
- Ruben Drost, Inge van der Putten, Dirk Ruwaard, Silvia Evers, Aggie Paulus
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- 31 December 2019, p. 74
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Introduction
Many 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.
PP207 Evaluation On Effects Of Antimicrobial Stewardship In Tertiary Comprehensive Public Hospitals in Hainan, China
- Hong Zhou
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- 31 December 2019, p. 74
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Introduction
The National Health Commission issued a special task force on antimicrobial stewardship (AMS). We assess the effects of AMS from 2012 to 2016 in four tertiary comprehensive hospitals in Hainan Province, China, to explore the achievement of AMS to facilitate rational use, to control antimicrobial resistance (AMR), to ensure safety and quality of care.
MethodsData from Hospital Information System (HIS) of hospitals according to the criteria of AMS were analyzed. Microsoft Excel data entry and SAS version 9.3 was used for analysis.
ResultsThe indicators were general compliance to the national criteria from 2012 to 2016. The percentage of following results in hospitals were gradually reduced: the proportion of antibiotic use in outpatient care (11.09 to 3.25 percent); that in emergency departments (8.46 to 1.53 percent); antibiotics use rate of inpatient care (9.13 to 3.12 percent); antibiotics prophylactic use in type I surgical sites (24.19 to 3.38 percent); the proportion of drug cost (5.54 to 0.12 percent) and total cost of antibiotics (3.27 to 0.45 percent); total cost of antibiotics in outpatient care and emergency departments, which was below 10 percent. Pathogenic detection rate of antibiotics from 2013 to 2016 in three hospitals was increased from 38.75 to 59.6 percent. Hainan Provincial Antibiotics Resistance Monitoring Network conscientiously performs duties, several important and special detection rates of AMR close to the average national level, which have been effectively controlled.
ConclusionsIt is needed to continue AMS and to enhance the capacity of rational use of antibiotics by medical professionals. Information systems need to be developed, coordinated and correlated to monitor the consumption of antibiotic use, surveillance of AMR and control of hospital infection.
PP210 Identification Of Frailty to Healthy Ageing In European Population
- Lucinda Paz-Valiñas, Maria José Faraldo Vallés, Rosendo Bugarín-González
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- Published online by Cambridge University Press:
- 31 December 2019, pp. 74-75
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Introduction
The European population is aging rapidly. The number of Europeans aged over sixty-five will double in the next fifty years. Active and healthy aging is a societal challenge shared by all European countries, but also an opportunity. The World Health Organization indicated that frailty has become an indicator of lack of successful aging. Therefore, identification of frail elderly is becoming important. However, there are many different screening tools that are currently used to identify frailty. The optimal test should have the capacity to easily identify from the community-dwelling population, those older people at risk of adverse outcomes. During the past years, gait speed has been repeatedly reported as an appealing instrument as a screening tool to detect frailty.
MethodsSystematic review of literature on gait speed as predictor of frailty was performed.
ResultsA total of 992 articles were retrieved from the literature search and only eleven studies met the inclusion criteria. Frailty is a common geriatric syndrome, characterized by decreased reserve and increased vulnerability to adverse outcomes, including falls, hospitalization, institutionalization and death. Despite frailty is being increasingly recognized in the literature, there is a paucity of direct evidence to guide interventions to reduce frailty. Many single and composite tools to detect the frailty have been proposed but none is consensual, most are time-consuming while evaluating different domains of impairments, and many are not validated. Gait speed seems to be a single, reliable, valid, sensitive, cheap, quick and simple tool that identifies frailty people. However, the way to perform the test parameters vary widely, influencing interpretations of physical performance.
ConclusionsThe evidence recommends to detect frailty in people in order to achieve an active and healthy ageing. Gait speed could be a suitable predictor to identify frailty although this systematic review found many differences between the gait speed protocols used in clinical practice. It is necessary to establish a standard protocol of gait speed agreed by experts in the area on frailty to be implemented with success in clinical practice.
PP238 Budget Impact Of Methionine-Free Amino Acid Formula For Homocystinuria
- Eduardo Mulinari, Nayara Castelano Brito, Lays Pires Marra
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- Published online by Cambridge University Press:
- 31 December 2019, p. 75
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Introduction
The 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. This study estimated the budget impact of the supply of methionine-free amino acid formula (MFAAf) for patients with classical homocystinuria (HCU) in the SUS.
MethodsThe incidence of one case per 250,000 live births in Brazil and the registration of a Brazilian association of patients with HCU was assumed to calculate the population. Mortality and responsiveness to pyridoxine rates were applied. The costs of treatment were estimated according to the recommended dosage in literature and public purchasing prices. For calculating the dose of MFAAf patients, a median age of 19 years and weight of 60 kg were assumed, according to Brazilian study data.
ResultsThe annual cost of treatment was estimated at BRL 77,000 (USD 21,084) per patient. The incorporation of MFAAf for HCU would generate a budget impact in SUS of around BRL 37 million (USD 10.1 million) in 2019 and BRL 188 million (USD 51.5 million) after five years which considers the epidemiological data, and a budget impact of around BRL 6.4 million (USD 1.75 million) in 2019 and BRL 33 million (USD 9 million) after five years which considers the information of a Brazilian association of patients with HCU. The wide range of values in the incremental budgetary impact is due to the lack of information on the epidemiology of the disease in Brazil.
ConclusionsThe incorporation of the MFAAf in the SUS represents an important budgetary impact and covers a small number of patients. CONITEC recommended the incorporation of the MFAAf in the SUS, according to clinical protocol.
Vignette Presentations
VP01 Methods Of Patient Involvement Now And Beyond 2020: A Case Study
- Heidi Livingstone, Gillian Leng, Victoria Thomas
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- Published online by Cambridge University Press:
- 31 December 2019, p. 75
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Introduction
Involving 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.