bluuebunny/crossref_metadata_2025 huggingface dataset:
PP36 Early Diagnosis And Treatment Of Psoriatic Arthritis
Authors: Iragorri Nicolas , Spackman Eldon
Published: January 2019

Introduction:

Screening for psoriatic arthritis (PsA) is expected to identify patients at earlier stages of the disease. Early treatment is expected to slow disease progression and delay the need for biologic therapy. This study estimated the cost-effectiveness of screening tools for PsA in Canada.

Methods:

A Markov model was built to estimate the associated costs and quality-adjusted life-years (QALYs) of screening tools for PsA in patients using topical treatment for psoriasis. The screening tools included: the Toronto Psoriatic Arthritis Screening (ToPAS) questionnaire; the Psoriasis Epidemiology Screening Tool (PEST); the Psoriatic Arthritis Screening and Evaluation (PASE) questionnaire; and the Early ARthritis for Psoriatic patients (EARP) questionnaire. Health states were defined by disability levels, as measured by the Health Assessment Questionnaire (HAQ), and state transition was modeled according to annual disease progression. Screening was assumed to be effective during a 2-year sojourn period. Incremental cost-effectiveness ratios (ICERs) were estimated based on health-state specific costs and utilities. A probabilistic analysis was undertaken to account for parameter uncertainty. All results were compared with the commonly cited cost-effectiveness threshold of CAD 50,000 (USD 37, 600) per additional QALY.

Results:

Screening with the ToPAS questionnaire resulted in cost savings compared with no screening or the EARP questionnaire, with a total cost of CAD 30,706 (USD 23,090) and 17.29 QALYs. The PEST dominated the PASE questionnaire and was more costly and more effective than the ToPAS questionnaire, with an ICER of CAD 312,398 (USD 234,909). The results were most sensitive to test sensitivity and specificity, HAQ progression, and average HAQ score at diagnosis and the start of biologic therapy. A scenario analysis tested screening efficacy for a 1-year period before diagnosis, with the ToPAS questionnaire remaining the most cost-effective option.

Conclusions:

Screening was cost-effective compared with no screening at the commonly used cost-effectiveness threshold of CAD 50,000 (USD 37, 600). Value of information analyses will be useful for determining the need to collect further information around test accuracy parameters.
PP51 Updating Canadian Pharmaceutical Budget Impact Analysis Guidelines
Authors: Foroutan Naghmeh , Levine Mitchell , Tarride Jean-Eric , Xie Feng
Published: January 2019

Introduction:

The Canadian BIA guideline was published by the Patented Medicine Prices Review Board (PMPRB) in 2007. Our initial systematic literature review of national and international BIA guidelines showed that a number of new recommendations relating to BIA model structure, input data and reporting format have been adopted in other jurisdictions such as UK, Australia, Poland, Ireland, Belgium, France and the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). The main objective of the present study was to conduct a comparative review of national, international and Canadian Federal, provincial and territorial BIA guidelines and provide a list of new recommendations related to the BIA key elements which have not been discussed or included in the Canadian PMPRB BIA guidelines.

Methods:

BIAs guidelines were searched in databases such as MEDLINE, EMBASE, Cochrane, and the gray literature including regulatory agency websites. An Excel-based data abstraction form was designed in order to highlight differences between recommendations related to the BIA key elements provided by PMPRB, provincial, and other national and international BIA guidelines.

Results:

Twelve guidelines were reviewed in detail. Sixty percent of the recommendations were new or were different from recommendations in the Canadian PMPRB BIA guidelines. They related to BIA key elements such as perspective, target population, costing, presenting results, data sources and handling the uncertainty.

Conclusions:

The present literature review is the initial step towards updating the Canadian BIA guidelines. This study presents a comparative review of key elements in BIA among different guidelines and provides a list of relevant practical recommendations for the improvement of the Canadian BIA guidelines. The new methodologic advancements and recommendations that were identified are being presented to Canadian stakeholders for their opinion and feedback prior to the development of a proposed new set of Canadian guidelines.
PP65 Coordinated Implementation And Evaluation Of Promising Stroke Therapy
Authors: Lambert Laurie , Azzi Leila , Désy François , Brière Anabèle , Boothroyd Lucy , Vutcovici Maria , de Guise Michèle
Published: January 2019

Introduction:

One important objective at the Institut d'excellence en santé et en services sociaux (INESSS) is to guide the implementation of promising new technologies into Québec's healthcare system. A comprehensive evaluation framework was recently developed that takes into account the dynamic and iterative nature of the life cycle of such technologies. This framework is presently being used to inform the decision-making process concerning use of thrombectomy for ischemic stroke.

Methods:

A field evaluation has been undertaken since April 2016 in all four of Québec's specialized tertiary stroke centers. This real-world evidence is communicated regularly to the clinical teams as well as decision-makers. A systematic literature surveillance is also ongoing, with results being shared amongst clinical experts on our interdisciplinary advisory committee. On the basis of the generated evidence from these sources, recommendations to optimize structures, processes of care and clinical outcomes will be developed, in collaboration with the interdisciplinary committee.

Results:

Thrombectomy has been shown to be safe and effective for treating ischemic stroke in the randomized trial setting in high-volume, expert centers. Real-world evidence from Québec indicates increasing use of this new technology but with wide variation across health regions. Observed times to treatment appear favorable for patients admitted directly to tertiary centers, but inter-hospital transfer is associated with important increases in delays from first door to thrombectomy. The documentation of 90-day outcomes is problematic, especially for patients transferred out of tertiary stroke centers prior to discharge. Uncertainties raised in the literature include patient selection criteria and optimal processes of care during prehospital and inter-hospital phases of the patient's trajectory.

Conclusions:

The ongoing comprehensive evaluation of thrombectomy for ischemic stroke in Québec is a concrete example of how the use of an innovative, disruptive technology can be optimized. We acknowledge the contribution of the members of the clinical expert committee.
PP69 Prostatic Artery Embolisation For Benign Prostatic Hyperplasia
Authors: Pwee Keng Ho
Published: January 2019

Introduction:

Prostatic artery embolization (PAE) was introduced in the 1970s to control major bleeding after prostate surgery. The procedure was noticed to improve the lower urinary tract symptoms of benign prostatic hyperplasia (BPH) and in 2010, PAE was first investigated as an alternative treatment for BPH. A rapid health technology assessment (HTA) was carried out to inform our hospital's decision on introducing this procedure.

Methods:

The rapid HTA investigated the safety and clinical effectiveness of PAE for patients with BPH. The PICO elements were: Population- Patients with symptomatic BPH; Intervention- PAE; Comparator- Conventional management; Outcomes- Adverse effects, clinical outcomes. The NHS Centre for Reviews & Dissemination databases, Cochrane Database of Systematic Reviews, and PubMed (MEDLINE) were searched for systematic reviews and HTA reports.

Results:

Eight systematic reviews from the most recent two years were found. The primary evidence base consists of two randomized controlled studies of PAE versus transurethral resection of the prostate (TURP), one matched pair analysis of PAE versus open prostatectomy in patients with large prostates, and several non-comparative studies. The comparative studies showed patients had better International Prostate Symptom score, quality of life and reduced prostate volume with TURP and open prostatectomy from 1 to 24 months. With respect to adverse events, embolized patients had more adverse events than controls, particularly acute urinary retention and post-embolization syndrome. However, controls had more abnormal ejaculation; and adverse effects from surgery naturally only occurred in controls.

Conclusions:

PAE appears to be a promising technology lacking long term outcomes. It has potential for patients who are not fit or not keen on surgery, or who may have large prostates, but who are still vascularly suitable for embolization. It would be suitable to carry out under clinical research conditions to clarify the incremental benefits of the technology and which patient groups are best served by the procedure.
PP76 Providing Information About Rheumatoid Arthritis Guideline In Brazil
Authors: Dias Carolina , Borin Marcus , Alvares Juliana , Guerra Augusto , Acurcio Francisco
Published: January 2019

Introduction:

Specialized Component of Pharmaceutical Service (SCPS) is a strategy to access high cost medicines in the National Health System (NHS) of Brazil, ensuring the completeness of medical treatment in which lines of care are defined in the Clinical Protocols and Therapeutic Guidelines (CPTG). To access the SCPS, the physician has to give to the patient a filled form, following some requirements and the CPTGs. In order to improve rational prescription and to facilitate patients’ access to medicines, we visited physicians and presented key information regarding the CPTGs of rheumatoid arthritis (RA) and the SCPS medicines request process; then, we sought to know their perceptions.

Methods:

From August to September 2017, 43 specialists and general practitioners that prescribed medicines of SCPS in Minas Gerais were visited by researchers about the CPTG of RA and how to prescribe to provide easier access to these medicines to their patients. After the visits, a researcher contacted the physicians by phone to evaluate their satisfaction with the visits and about the program through a brief questionnaire.

Results:

Twenty-eight physicians answered to our phone call, providing a response to the questionnaire. Sixty-eight percent indicated they were very satisfied with the visit. Fifty percent stated that the content of the visit was relevant to their practice, and 60.7 percent said that the distributed material was going to be useful for their professional practice. Regarding the guidelines, 43 percent affirmed that the visit really helped them to improve their understanding of medicine requests in the NHS and 42.9 percent said that the visit increased their understanding of which patients are eligible for RA treatment in the SCPS; 57.1 percent of those affirmed that the visits increased their knowledge.

Conclusions:

The physicians, who were mostly specialists, already had knowledge about CPTG and prescription practices of SCPS’s medicines, nevertheless, they showed interest in the visits to review and improve their knowledge and clinical practice.
PP81 Engagement Of Healthcare Professionals In Health Technology Assessment With Negative Results
Authors: Gallo Luciana , Elias Flavia , Pereira Ana Carolina , Araujo Wildo
Published: January 2019

Introduction:

We conducted health technology assessments (HTAs) of the interventions used between 2012 and 2014 to improve the treatment of homeless people with pulmonary tuberculosis in the Federal District of Brazil. The HTA, which was not ordered by policymakers, was based on the evidence-based national theoretical model compared with local interventions indicated in focus groups, semi-structured interviews, and secondary data produced by the Health Secretariat. The results demonstrated that the implementation of the interventions was unsatisfactory. Our objective was to present the feedback process for policymakers and the Health Secretariat, particularly its challenges.

Methods:

The feedback was categorized as: (i) an executive abstract with key messages (i.e. underreporting of cases in the surveillance system, lack of primary care, and underestimation of the health problem) reported to policymakers involved in the surveillance and healthcare systems; and (ii) oral presentations in eight meetings organized by the research group and local policymakers.

Results:

Between 2016 and 2017 we conducted eight feedback meetings. All of the professionals (n = 8) involved in the Tuberculosis Surveillance and Control Program were present in at least one of the meetings, but healthcare professionals and the Secretary of Health did not participate. The barriers presented by the professionals were: (i) lack of material resources (i.e. cars and gas, phones, diagnostic tests, medications); (ii) lack of human resources (i.e. suboptimal professional staff); and (iii) feeling insecure when performing extramural activities due to the potentially unsafe work environment.

Conclusions:

Gathering feedback on a HTA that was not ordered by policy makers can be a challenge. Mainly we demonstrated a negative result on research done in a vulnerable population with a neglected disease, in this case tuberculosis. However, this provided an opportunity for professionals in the surveillance system to discuss the challenges of implementing tuberculosis control among the homeless population.
PP83 Early Assessment Of Proof-Of-Problem To Guide Health Innovation
Authors: Kluytmans Anouck , Tummers Marcia , Vanderwilt Gert , Grutters Janneke
Published: January 2019

Introduction:

In the fast-paced world of health technology innovation, early health technology assessment (HTA) gained recognition as a tool to help prioritize and steer the development of those innovations that potentially add value. Much of early HTA seems technology-driven; a certain novel technology is introduced and the focus is on assessing its expected cost-effectiveness. We argue that a first step in assessing innovation would be to derive proof-of-problem through combining evidence from literature and stakeholder engagement. We applied this approach to a novel surgical instrument aimed to facilitate meniscus surgery.

Methods:

First, we identified a broad scope of stakeholders in meniscus surgery (i.e. meniscectomy). Through interviewing them we derived key problems in meniscectomy as-is, and determined which outcomes matter most. We used stakeholder and literature input to quantify the room for improvement in current meniscectomy. Together with stakeholders we interpreted the problem quantification and conducted an early assessment of the proposed surgical innovation. Finally, we made use of this early stakeholder engagement to uncover possible barriers and facilitators to the innovation's implementation.

Results:

While all stakeholders were enthusiastic about the innovation, there was a shared perception that there is little room for improvement in meniscectomy at present. Put differently; the innovation poses a great solution to problems that may not exist. In addition, by involving a broad range of stakeholders we were able to identify barriers and facilitators to future implementation early on, such as surgeons’ preferences.

Conclusions:

We conclude that the innovation's value may lie with applications outside of meniscus surgery. Regarding methodology, we showed how a shift of focus from solution to problem definition provides a different perspective on an innovation's potential value, borne out of needs not currently met. In doing so, early HTA is in a unique position to help navigate the stream of health technology innovation before actual development of the innovation.
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 patients

Methods:

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.
PP162 Bridging Brazil's Know-Do Gap On Social Engagement In Health Technology Assessment
Authors: Sousa Maria-Sharmila , Figueiró Mabel , Silva Aline , Silva Everton , Silva Marcus , Pereira Viviane , Barreto Jorge
Published: January 2019

Introduction:

Social engagement in health encompasses the idea of involving (parts of) society as full partners in the decision-making regarding both development and implementation of health technologies. Evidence shows that patient engagement is linked with fewer adverse events, better patient self-management, fewer diagnostic tests, decreased use of healthcare services and shorter lengths of stay in hospitals. Matching the escalating healthcare requirements to face the ongoing societal and economic challenges regarding access and coverage to (new) health technologies is not an easy task for health providers.

Methods:

We conducted a systematic review (CRD42017068714) designed to address the institutional implementation of social engagement by the Brazilian Ministry of Health. All systematic reviews were evaluated using the new version of AMSTAR and, once all findings are synthesized, we will use the GRADE-CERQual approach to assess for confidence.

Results:

From 399 publications that met the inclusion criteria, 80 described the implementation of social engagement during the development and implementation of (new) health technologies at various levels (local, regional, national, supranational), countries and for different health technologies and social actors. The remaining 319 publications constitute case studies describing barriers and enablers to implementing social engagement in HTA and coverage decision-making processes. By mapping barriers and facilitators, we explored effectiveness and sustainability, further observing how citizen science-based strategies can ultimately reform health service delivery by innovating the social engagement in health technology development and implementation.

Conclusions:

This systematic review addresses the know-do gap on social engagement in health technology development and implementation, from a global perspective, as a way of improving the Brazilian Ministry of Health's HTA activities and enabling a Brazilian strategy to reform health service delivery. Enabling social engagement as early as possible, during all the stages of the development cycle, grants a more effective and sustainable health care system.
PP164 Identifying Complications Of Partial Nephrectomy Using Physician Claims
Authors: Sun Jian , Stafinski Tania , Nagase Fernanda Inagaki , Menon Devidas
Published: January 2019

Introduction:

Many population-based studies identify surgical complications using hospital discharge abstract databases (DAD). With DAD, however, complications occurring after the discharge date cannot be followed up. This study used physician claims data to identify the complications of partial nephrectomy, and to compare the rates of complications of open, laparoscopic, and robot-assisted nephrectomies.

Methods:

Physician claims, DAD, and ambulatory care data from April 2003 to March 2016 were provided by Alberta Health. DAD and ambulatory care data were used to extract information on patients with kidney cancer who underwent partial nephrectomy. All physician claims within 30 days before and after surgery for the cohort were extracted. The numbers of the same International Classification of Diseases, Ninth Revision (ICD-9), codes before and after surgery were compared. If a number increased after surgery, this diagnosis was initially identified as a complication. All diagnoses with neoplasms were excluded. The incidence rates of complications for the three surgery groups were calculated. Chi-squared tests were conducted for the following nephrectomy comparisons: laparoscopic versus open; robot-assisted versus open; and robot-assisted versus laparoscopic.

Results:

A total of 1,890 kidney cancer patients had partial nephrectomies. Among them, 1,080, 411, and 399 had open, laparoscopic, and robot-assisted nephrectomies, respectively. One patient who had two different nephrectomies on the same day was excluded from analysis. The robot-assisted group had lower rates of digestive complications (ICD-9: 537–578, 787, 789, 998.6) and infections (ICD-9: 004–041, 998.5) than the open group, and higher rates of genitourinary complications (ICD-9: 584–599, 788, 997.5) than the laparoscopy group. The robot-assisted group had lower rates than the open group for most of the complication categories, but the differences were not statistically significant.

Conclusions:

Robot-assisted surgery appears to be superior to open surgery, but no better than laparoscopic surgery, in terms of minimizing the risk of complications following partial nephrectomy.
PD04 Cost-Utility Of Quetiapine For Schizophrenia: A Systematic Review
Authors: Santos André , Ruas Cristina
Published: January 2019

Introduction:

Schizophrenia is a chronic debilitating condition characterized by disorders in thought, affect and behavior. The worldwide prevalence is around 0.3 to 1 percent. The pharmacological treatment is based on antipsychotic drugs, but their efficacy is limited, culminating in discontinuation of treatment, relapses, and readmissions to health services. Quetiapine was initially approved for use in the United States of America in 1997. The drug has moderate affinity for D2 and 5-HT2A receptors and high affinity for H1 receptors. This study aimed to conduct an assessment of the cost-utility of quetiapine for schizophrenia around the world.

Methods:

Cost-utility studies of head-to-head comparisons of quetiapine against other antipsychotic drugs for the treatment of patients with schizophrenia and related disorders were included, irrespective of the diagnostic criteria used. An electronic search on Medline, Lilacs, Center for Reviews and Dissemination, The Cochrane Library and PsycINFO was conducted and complemented by references of included studies, Google Scholar and conference abstracts. Monetary values were converted to PPP-USD for the same base-year of the study.

Results:

Six economic evaluations were included, representing four countries and a multicentric analysis. Comparisons between quetiapine and twelve other antipsychotic drugs were identified. Three studies found quetiapine to be dominated by risperidone and the remaining three found it to be more expensive and more effective with incremental cost-effectiveness ratio (ICER) values of USD 36,535, 8,786 and USD 127,600 per quality-adjusted life-year (QALY). Three studies found quetiapine, in comparison to olanzapine, to be inferior, one found it to be superior and two studies found it to be more expensive and more effective with ICER values of USD 139,699 and USD 224,000 per QALY. The reports were considered to be of reasonable quality. Yet the mixture of contexts might influence the results.

Conclusions:

In general, there seems to be a trend favoring olanzapine and risperidone over quetiapine. None of the studies favored quetiapine over all the other drugs.