bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP90 The Value Of Multi-Criteria Decision Analysis Use On Health Technology Decision Making Process
Authors: Etges Ana , Zanotto Bruna , da Rosa Prisciane , Polanczyk Carisi 
Published: January 2019

Introduction:The use of multi-criteria decision analysis (MCDA) in health technology assessment (HTA) studies has become more common due to the fact that MCDA offers a comprehensive technique for decisions that involve multiple criteria and stakeholders. How MCDA contributes to the HTA decision making process is an issue to be investigated. A systematic review was carried out in order to provide an overview of the benefits identified in MCDA applications for the strategic HTA decision making process.Methods:A systematic review developed by Philip Wahlster et al. (2014) was updated. The papers were analyzed in order to determine how MCDA is connected with traditional HTA, and to identify opportunities through the application of MCDA. In total 965 papers were found, and 43 articles were included in the review. The included articles detailed MCDA applications oriented to tactical and strategic decision making processes. The review was conducted by two researchers.Results:Of the available studies published on MCDA, 76 percent were published between 2014 and 2017, and 24 percent were published prior to 2014. Regarding the MCDA methodology defined in the included studies, 10 used the analytical hierarchy process, four used multi-attribute theory, and others refer the methodology only as “MCDA”. Seventeen studies also included health technology economic analysis, in special cost-effectiveness, safety and technological innovation. The studies suggest MCDA adds value since it allows different stakeholders to be engaged in the decision making process.Conclusions:The increase in studies on MCDA and healthcare point to the possibility to add different criteria, engage people with different knowledge levels, and make the decision-making process more transparent. In comparison with other technical areas, the use of MCDA in healthcare is more focused on achieving the decision about adding the new technology, and to show how to engage stakeholders than to explain how to develop the algorithms and methodologies.
PP105 Applying Horizon Scanning To Decision Making: The Case of Tafamidis
Authors: Gomes Pollyanna Teresa Cirilo , de Souza Andrea Brígida , de Brito Artur Felipe 
Published: January 2019

Introduction:Horizon scanning (HS) is an important tool for guiding health policy formulation and the decision-making process in Brazil. In 2016, the Ministry of Health started to draft Brazilian clinical practice guidelines for transthyretin familial amyloid polyneuropathy (TTR-FAP), which is a rare disease caused by a mutation of the transthyretin gene. An initial HS report was conducted that provided information about new and emerging technologies for TTR-FAP. The HS identified five drugs that were based on two mechanisms of action: transthyretin stabilization (diflunisal, tafamidis, and tolcapone) and gene silencing (ALN-TTR02 and ISIS-TTR-Rx). At that time in Brazil there were no drugs registered for the treatment of TTR-FAP. However, a few months later tafamidis was licensed in Brazil. In early 2017 the manufacturer submitted an application to the National Committee for Health Technology Incorporation (CONITEC), with the aim of incorporating tafamidis into the Brazilian health system. As a result the HS report was updated to support the assessment by CONITEC. This study aims to show how HS is being used to support CONITEC in this issue.Methods:As per the EuroScan toolkit, we performed a reassessment of the technologies included in the initial HS report. We searched clinical trial registers, the websites of pharmaceutical companies, conference proceedings, scientific journals, HS databases, and regulatory websites for further information. The data were synthesized and a reformulated landscape of the technological environment for TTR-FAP therapy was presented to the CONITEC Plenary.Results:The main difference between the initial and final HS output was that tafamidis was approved for use in Brazil, making it the only registered drug for TTR-FAP. Another difference was related to the start of a new clinical trial with diflunisal for TTR-FAP, indicating that this drug could be a potential competitor for tafamidis. It was also possible to add published positive results from a clinical trial with ISIS-TTR-Rx, which were unavailable when the first report was written. Beyond that, it appears that there are two promising gene silencers on the horizon that could represent potential competitors for tafamidis.Conclusions:The analysis of tafamidis for incorporation into the Brazilian health system is ongoing, but HS was able to deliver strategic information that could affect the final recommendation of CONITEC.
PP110 The Prophylactic Removal Of Impacted Third Molars: A Systematic Review
Authors: Pilkington Gerlinde , Hounsome Juliet , Renton Tara , Dickson Rumona 
Published: January 2019

Introduction:Impacted third molars (I3Ms) are blocked from fully erupting; many I3Ms are asymptomatic, however there could be pain and pathological changes. Historically, I3Ms were removed prophylactically. Current options in the United Kingdom include either retention with standard care (watchful waiting), or removal due to pathological changes. We conducted a systematic review of the prophylactic removal of asymptomatic impacted mandibular third molars (IM3Ms) compared with standard care.Methods:We searched five electronic databases from 1999 onwards. Inclusion criteria: randomized and non-randomized trials, observational studies, and systematic reviews (SRs) comparing the prophylactic removal of IM3Ms with standard care or studies assessing the outcomes of either approach; outcomes included pathology associated with retention, postoperative complications, adverse effects of treatment and health-related quality of life. Two reviewers independently screened all titles and/or abstracts, applied inclusion criteria to potentially relevant publications, and quality assessed and data extracted the included studies. No meta-analysis or network meta-analyses were undertaken.Results:Following screening of 11,373 references, 13 studies (four cohort studies and nine SRs) were included. One cohort study investigated the prophylactic removal of asymptomatic IM3Ms in comparison with standard care and retention, two investigated the prophylactic removal of asymptomatic IM3Ms, and one studied the retention and standard care of asymptomatic IM3Ms. Two studies reporting surgical complications found no serious complications; however, one study reported intense pain and postoperative infection. Pathological changes due to retention of asymptomatic IM3Ms were reported by three studies. Nine SRs of the management of third molars were included in this review, however none focused solely on IM3Ms.Conclusions:Consistent with previous systematic reviews, we found no RCT data to support or refute the prophylactic removal of asymptomatic IM3Ms, despite extensive searching of the literature. The review however did identify evidence from two longitudinal studies demonstrating the outcomes when asymptomatic IM3Ms are left in situ.
PP118 Cardiac Safety Of Trastuzumab For Metastatatic Breast Cancer
Authors: de Lemos Livia Lovato Pires , Almeida Paulo Henrique Ribeiro Fernandes , Alvares Juliana , Rabelo Roberta , Santos Vania Canuto , Acurcio Francisco , Guerra Augusto 
Published: January 2019

Introduction:Metastatic breast cancer (MBC) is considered incurable. Trastuzumab (T), a monoclonal antibody that blocks HER-2 is used in combination with other chemotherapies or as monotherapy to treat various stages of breast cancer, including MBC. The aim of this study was to evaluate the safety of T as first line treatment or after progression in women with MBC.Methods:We conducted a systematic review of randomized controlled trials. We searched the databases: MEDLINE (Pubmed), LILACS, Cochrane Library and EMBASE (accessed November 2016) and performed manual search. The methodological quality assessment was performed using the Cochrane Collaboration risk of bias tool. We adopted the random effects model for meta-analysis. The results were presented as relative risk (RR) with 95% confidence intervals.Results:The search retrieved 2,238 publications. After eligibility criteria assessment we included five studies on T in the first line treatment (T n = 493; no-T n = 492) and two studies on T after progression (T n = 226; no-T n = 226). In general, studies presented moderate quality. Five were funded by the pharmaceutical industry. Regarding first line treatment, the group of patients that used T had three times higher risk of developing cardiac adverse event compared to the group that did not use T (RR = 3.3; 95% CI: 1.52 − 7.29; I2 = 0%, p = 0.39). The continuity of T after progression revealed no difference between the groups regarding the risk of developing cardiac adverse event (RR = 5.31; 95% CI: 0.62 − 45.49; I2 = 0%, p = 0.62).Conclusions:The evidence regarding the higher risk of cardiac adverse event with T as first line treatment for MBC is robust and this should be taken into account when balancing risks and benefits of treatment. The evidence for continuation of T after MCB progression is week and more studies are needed to confirm the findings.
PP129 The Need For Building Pharmacists HTA Capacity; The Nigerian Scenario
Authors: Chukwu Otuto Amarauche 
Published: January 2019

Introduction:The role of Health technology assessment (HTA) as a systematic approach in the evaluation of health interventions and technologies is becoming increasingly important as the quest for attaining universal health coverage globally continues to increase. Some developed countries in Europe and the Americas now apply HTA extensively in healthcare policy decisions, however, developing regions and countries like sub-Saharan Africa and Nigeria respectively, seem not to be making significant progress in this area. Given that evidence suggests that Nigeria and indeed several countries in sub-Saharan Africa are performing poorly on most healthcare indices as the region continues to be ravaged by predictable and avoidable epidemics and disease outbreaks, the need to build HTA capacity has never been more paramount.Methods:A review of HTA capability in Nigeria was done. Pharmacists in Nigeria's Capital were randomly sampled. Semi-structured questionnaires were administered. Descriptive statistics was used in data analysis. P values less than 0.05 were considered to be significant.Results:In Nigeria, there is no institution tasked with undertaking HTA and there seems to be limited knowledge, capacity and awareness on the issue. Pharmacists, being the most accessible healthcare professionals according to evidence, are a key group that could play an active role in HTA and its implementation in developing countries like Nigeria. However, out of 322 pharmacists randomly sampled, ninty-three percent were not aware of HTA and its application in healthcare decision-making.Conclusions:There is no paucity of healthcare programs and plans in Nigeria but they seem to fail due to lack of evidence-based assessment, decision-making and implementation. Hence, there is increasing need to raise awareness on the importance of HTA in healthcare decision-making; strengthen HTA capacity by developing and sustaining institutional capacity and adequate human resource for HTA; and creating regional annexes of HTA organizations in Africa.
PP130 Economic Burden Of Nine Human Papillomavirus Strains (HPV9)-Related Diseases: A Real World Cost Analysis From Italy
Authors: Mennini Francesco Saverio , Fabiano Gianluca , Favato Giampiero , Sciattella Paolo , Bonanni Paolo , Pinto Carmine , Marcellusi Andrea 
Published: January 2019

Introduction:The objectives of this study were to estimate the economic burden of human papillomavirus (HPV) in Italy, accounting for total direct medical costs associated with nine major HPV-related diseases, and to provide a measure of the burden attributable to HPV 6, 11, 16, 18, 31, 33, 45, 52, 58 infections.Methods:A cost-of-illness incidence-based model was developed to estimate the incidences and costs of invasive cervical cancer, cervical dysplasia, cancer of the vulva, vagina, anus, penis, oropharyngeal, anogenital warts, and recurrent respiratory papillomatosis (RRP) in the context of the Italian National Health System (NHS). We used data from hospital discharge records (HDRs) of an Italian region and conducted a systematic literature review to estimate the lifetime cost per case, the number of incident cases, the prevalence of HPV9 types. Costs of therapeutic options not included in the diagnosis-related group (DRG) tariffs were estimated through a scenario analysis.Results:The total annual direct costs were EUR 540.7 million, with a range of EUR 338.3 – EUR 789.7 million. These costs could increase considering innovative therapies for cancers treatment (range EUR 16.2 – EUR 37.6 million). The fraction attributable to the HPV9 genotypes without innovative cancers treatment was EUR 329.2 million (range EUR 150.1 – EUR 576.7 million), accounting for sixty-one percent of the total annual burden of HPV-related diseases in Italy. Of this amount, EUR 136.7 million (forty-two percent) was related to men, accounting for sixty-four percent of the costs associated with non-cervical conditions.Conclusions:The infections by HPV9 strains and the economic burden of non-cervical HPV-related diseases in men were found to be the main drivers of direct costs. The fraction of the total direct lifetime costs attributable to infections by HPV9 strains and the economic burden of non-cervical HPV-related diseases in men were found to be the main drivers of direct costs.
PP146 Cost-Effectiveness Of Nivolumab Plus Ipilimumab In Advanced Melanoma
Authors: Tibet Birol 
Published: January 2019

Introduction:This study was done to assess the cost effectiveness of nivolumab plus ipilimumab (NIV+IPI) versus nivolumab alone (NIV) for previously untreated patients with advanced melanoma (AM) from the Dutch health system perspective.Methods:A Markov model was constructed with a lifetime horizon. Future effects and costs were discounted at 1.5 and four percent, respectively. Risks of progression and death were based on progression-free survival rates obtained from a phase III clinical trial (NIV+IPI and NIV versus ipilimumab). Conjectural overall survival rates were calculated indirectly by using progression-free survival and overall survival rates from another trial (NIV versus dacarbazine), and were extrapolated later using the Weibull distribution. Utility values of health states and disutility values of adverse events were derived from the literature. Unit costs were derived from the Dutch Diagnosis Treatment Combination Care Products Tariff, Erasmus University Medical Center prices, and Dutch pharmacy purchase prices. Chronic management costs of AM and treatment costs of adverse events were calculated based on the results of a survey of clinicians that determined the necessary healthcare services and their utilization rates.Results:On average, over a lifetime an AM patient treated with NIV+IPI was estimated to live 4.2 years and 2.6 quality-adjusted life-years (QALYs) at a discounted net cost of EUR 262,824 per patient, while a patient treated with NIV was estimated to live 3.3 years and 2.0 QALYs at a discounted net cost of EUR 195,341 per patient. The incremental cost-effectiveness ratio was EUR 70,770 per life-year saved, and the incremental cost-utility ratio was EUR 115,533 per QALY gained.Conclusions:At a willingness-to-pay threshold of EUR 80,000 per QALY gained, NIV+IPI may not be a cost-effective tool, compared with NIV, for preventing the high mortality and morbidity associated with AM from the Dutch health system perspective.
PP148 Your Money Or Your Life? Are Price Negotiations Health Technology Assessment Best Practice?
Authors: Macaulay Richard , Turkstra Erika 
Published: January 2019

Introduction:Many countries use Health Technology Assessment (HTA) organizations to evaluate the clinical and economic impact of new therapeutic interventions. In some markets, HTA outcomes directly link to reimbursement decision-making based on the manufacturer's submitted price (e.g. NICE and SMC [UK]). In others, the HTA outcome leads to price negotiations with manufacturers by a separate body (e.g. HAS/CEPS [France] and G-BA/GKV [Germany]). This research compares major examples of each approach to inform a discussion on whether such price negotiations align with HTA best practice.Methods:Publically-available technology assessment outcomes for G-BA/GKV, NICE and SMC (01/01/2011-31/12/2015) were extracted and compared.Results:Of 112 G-BA benefit assessments, 45 percent offered no additional benefit with automatic reference pricing; 55 percent offered additional benefit, qualifying for price negotiations; 77 percent had prices negotiated, 14 percent had price fixed by court, and eight percent withdrew from market. Of 156 NICE Single Technology Appraisals, 51 percent were recommended, 17 percent restricted, 20 percent not recommended, and 12 percent non-submissions. Of 497 SMC appraisals, 35 percent were accepted, 28 percent restricted, 17 percent not recommended and 19 percent non-submissions. Forty-eight percent and 24 percent of NICE and SMC positive appraisals were associated with a Patient Access Scheme (PAS), with 86 percent and 88 percent being simple discounts schemes, respectively.Conclusions:Making reimbursement decisions for new medicines based on a clear set of criteria may be the most objectively fair and transparent method of HTA; however, as the NICE and SMC examples show, although strong downward price pressure is exerted (high frequency of PASs), this may come at the cost of many therapies (~33 percent) being denied access. By contrast, the flexibility enabled by a distinct price negotiation phase may enable more therapies access, as shown by the G-BA/GKV example (<10% medicines withdrawn). Nevertheless, the relative effectiveness of the downward price pressures, a key determinant of HTA process effectiveness, cannot be compared due to the confidential nature of UK PAS discounts.
PP149 Features Of Accountable And Reasonable Processes For Coverage Decision-Making
Authors: Wagner Monika , Samaha Dima , Casciano Roman , Brougham Matthew , Petrie Charles , Abrishami Payam , Avouac Bernard , Kind Paul , Mantovani Lorenzo Giovanni , Sarria-Santamera Antonio , Schlander Michael , Tringali Michele 
Published: January 2019

Introduction:The Accountability for Reasonableness (A4R) framework addresses the legitimacy of coverage decision processes by defining four conditions for accountable and reasonable processes: Relevance, Publicity, Appeals, Implementation. Cost-per-quality-adjusted life year (QALY) and multicriteria-centered processes may have distinct implications for meeting A4R conditions. The aim of this study was to reflect on how the diverse features of decision-making processes can be aligned with A4R conditions to guide legitimized decision-making. Rare disease and regenerative therapies (RDRTs) pose special decision-making challenges and offer a useful case study.Methods:To support reflection on how different approaches address the A4R conditions, thirty-four features operationalizing each condition were defined and organized into a matrix. Seven experts from six countries explored and discussed these features during a panel (Chatham House Rule) and provided general and RDRT-specific recommendations for each feature. Responses were analyzed to identify converging and diverging recommendations.Results:Regarding Relevance, panelists highlighted the importance of supporting deliberation, stakeholder participation and grounding coverage decision criteria in the legal framework, goals of sustainable healthcare and population values. Among seventeen criteria, thirteen were recommended by more than half of panelists. Although the cost-effectiveness ratio was deemed sometimes useful, the validity of universal thresholds to inform allocative efficiency was challenged. Regarding Publicity, panelists recommended communicating the values underlying a decision in reference to broader societal objectives, and being transparent about value judgements in selecting evidence. For Appeals, recommendations included clear definition of new evidence and revision rules. For Implementation, one recommendation was to perform external quality reviews of decisions. While RDRTs raise issues that may warrant special consideration, rarity should be considered in interaction with other aspects (e.g. disease severity, age, budget impact).Conclusions:Improving coverage decision-making towards accountability and reasonableness involves supporting participation and deliberation, enhancing transparency, and more explicit consideration of multiple decision criteria that reflect normative and societal objectives.
PP156 Risk Assessment Of Equipment Used In Intensive Care Units
Authors: Jyh Juang Horng , Diamente Loraine Martins , Spadella César Tadeu 
Published: January 2019

Introduction:Knowledge and proper use of hospital equipment are essential for preventing adverse events associated with their use. The risks controls for medical devices and equipment are of major importance in ensuring patient safety and the quality of care delivered by healthcare professionals. Monitoring equipment (ME), infusion pumps (IP), and mechanical ventilators (MV) are frequently used in intensive care units, but they are subject to technical, human, and process failures that may pose harm to and even cause the death of patients. The aim of this study was to evaluate the risks related to the use of ME, IP, and MV in the adult intensive care unit (AICU) of a public hospital in Brazil, and to investigate the causes of technical complaints and the adverse events associated with them. We hope the outcomes may serve as a basis for the facility to create mechanisms to diminish the risk and increase the safety and quality of care delivered to critical patientsMethods:A 12-month prospective, observational descriptive study was conducted using an active and passive search of processes related to: hospital medical equipment use; available human and material resources; training programs and continuing professional education; equipment disinfection, sterilization, and assembly processes; and the hospital risk management measures regarding the reports and actions for technical, human, and process failures and the adverse events and incidents related to them. All the data collected were checked against current Brazilian legislation and the equipment technical manuals. The root cause of every failure and adverse event was investigated.Results:The active search identified seventy-five reports on technical complaints in the study period: sixty-five were related to IP, six to ME, and four to MV. The reasons for the complaints included: deficiencies in the quantity, qualification, training, and capacity of professionals handling the devices; inadequate disinfection of MV accessories; absence of or difficulty in accessing the equipment technical manuals; and a lack of preventive and corrective maintenance programs. One single adverse event caused by an IP medication error was attributed to a programing error.Conclusions:Failures and deficiencies in the knowledge and management of hospital equipment can potentially increase risks to patients and healthcare professionals. Increasing compliance with Brazil's current legislation related to the technical and operational norms of hospital equipment might create safer practices and improve care quality for critical patients.