The Brazilian public health system (SUS) provides technologies based on the best available scientific evidence. However, there is a large number of lawsuits against the government for access to non-standard technologies, a phenomenon called “judicialization of the right to health”, which disrupts the system's operating logic. The aspect of judicialization that most impacts the health system involves unregistered technologies without scientific evidence of superiority being comparing to the alternatives already offered in the country. The aim of this study is to report experience of the National Committee for Health Technology Incorporation in the Brazilian Health System (CONITEC) to mitigate the effects of the judiciary, with the elaboration of informative documents about technologies directed towards policy-makers, patient, users of system health, professional health and other stakeholders.

The main judicialized technologies in the country were identified and then a meeting with experts was realized to discuss a more appropriate format for these documents. After defining the format, a review of the literature was carried out to identify the best available evidence of those health technologies.

A question-and-answer (QA) format document was drawn. The QA addressed information on the use of the technology for a specific clinical condition. Health registry and price in Brazil, if it has already been evaluated by CONITEC and its respective recommendation, as well as strategies of care and therapeutic alternatives available in the SUS were included. Their content has been adapted to a lay language and all of the documents were made available on the CONITEC website in the “Law and Health Section”.

The availability of QA represents a strong link between evidence and actions in health. For, they enable broad access to quality information by the lay public and stakeholders who seek information to support evidence-based decision-making.

Public consultation is one of the phases provided by the law that rules the health technology incorporation process in Brazilian Public Health System (SUS). In the Brazilian model, anyone can participate as long as he/she identifies her/himself. During the decision-making process these suggestions are analyzed by the National Committee for Health Technology Incorporation (CONITEC) and, sometimes, they are responsible for changing a preliminary recommendation for a technology. This study aims to identify the health technologies for which CONITEC revised the initial negative recommendations due to the contributions received during the public consultation.

A descriptive study using as input data the information on coverage decisions available on the CONITEC website.

Since CONITEC's creation until October 2017, CONITEC enacted 241 public consultations. Fifteen cases of change to the preliminary negative recommendation were found and among these eight (53 percent) had the economic studies or proposed technology price reconsidered by the companies. In the other seven decisions, the Board also regarded as important the reasons for changing the initial recommendation: new evidence on efficacy and safety as well as the analysis of different outcomes previously unconsidered in the preliminary assessment.

During the public consultations, besides technical-scientific information, personal experiences and opinion reports on each health technology analyzed, CONITEC received new price offers and economic studies from the applicants. This new material has allowed, in some cases, these technologies to become competitive and to be included as alternatives to those already available, provided there is no clinical impairment. This study reinforces the importance of the public consultation and social participation in the process of health technologies incorporation in Brazil, considering its capacity to add new information to the decision-making process.

The Federal District in Brazil has about 2.9 million inhabitants and the public health system is focused on medical specialties, with one university hospital and twenty regional hospitals. This ecosystem is favorable for fostering health technology assessment (HTA) to improve the efficiency and effectiveness of health care. The objective was to identify institutions that could form a HTA network to support decision-oriented evidence in the public health system.

Stakeholders from the hospitals and training/research institutions in the Federal District were surveyed. An online questionnaire (Google Docs) was developed to identify the potential and capacity of institutions to analyze or produce clinical and economic evidence. Two HTA seminars were held to spread knowledge about HTA and to encourage stakeholders to complete the survey.

The questionnaire response rate was thirty-five percent (25/70). Fifteen institutions were cited by the respondents as having the potential to build a HTA network. Twelve of the institutions produced rapid reviews and clinical guidelines, but only three of these had an organized priority setting process or produced assessments at the request of the hospital manager. The challenges identified were training and willingness of decision makers to organize HTA units in the hospitals.

An executive group was created which defined a strategy to support the implementation of HTA units as part of the HTA National Network (REBRATS). A regulation proposal was also created to encourage decision makers to activate a HTA network in the Federal District.

The use of drugs for clinical conditions that differ from those approved in prescribing information (product labels) is known as off-label use. In Brazil, the Brazilian Health Regulatory Agency (ANVISA) is the official organization that regulates the use of health technologies. For technologies to be incorporated into the Brazilian public health system, registration with ANVISA is mandatory. However, occasionally, it is necessary to evaluate technologies for off-label use in the interests of public health. This study aimed to identify the health technologies recommended by the National Committee for Health Technology Incorporation (CONITEC) with an off-label indication between January 2012 and October 2017.

A descriptive study was undertaken using data available on the CONITEC website.

The study identified seven drugs with a favorable recommendation for off-label use: everolimus, sirolimus, and tacrolimus as immunosuppressants in transplant recipients; clozapine for bipolar affective disorder; pentoxifylline for cutaneous leishmaniosis mucosa; risperidone for adults with autism spectrum disorder, and bevacizumab for age-related macular degeneration and diabetic macular edema. For these decisions the Committee considered the scientific evidence available for the indication proposed, the severity of the disease, and the existence or absence of alternative treatments. This was possible because Brazilian legislation allows ANVISA to authorize the off-label use of health technologies provided that the analysis is supported by scientific evidence regarding effectiveness, accuracy, and safety for the intended purpose.

The off-label use of health technologies is a worldwide practice that can favor vulnerable populations and neglected diseases. This practice should be seen as positive when there is evidence supporting off-label use, and such decisions should not be influenced by political, economic, or marketing considerations.

The conventional drug sensitivity test is traditionally used in Brazil to diagnose drug-resistant tuberculosis. However, the test can take up to 60 days to return a diagnosis, which is considered too long for certain vulnerable populations. Therefore, this study analyzed the available scientific evidence on the accuracy and time to diagnosis of the nitrate reductase assay for diagnosing resistant tuberculosis, compared with the conventional drug sensitivity test.

We searched MEDLINE, Embase, and The Cochrane Library for systematic reviews with meta-analyses. The articles were screened by title and abstract. The full-texts of potentially relevant articles were then screened according to the inclusion criteria.

Three systematic reviews with meta-analyses were selected that compared the nitrate reductase assay with the conventional drug sensitivity test. The accuracy of the nitrate reductase assay was satisfactory in most of the results when compared with the sensitivity test, except for one study that showed low sensitivity for the detection of streptomycin resistance. In addition, the nitrate reductase assay had a shorter time to diagnosis than the drug sensitivity test.

The results of this study reinforce the idea that the nitrate reductase assay may diagnose drug-resistant tuberculosis earlier than the conventional drug sensitivity test and be a helpful strategy for controlling the disease, especially in vulnerable populations that are more likely to be affected by tuberculosis. For a broader analysis of the benefit of the assay, it is suggested that studies investigate the impact of the shorter time to diagnosis on morbidity and mortality in patients with drug-resistant tuberculosis. In addition, economic analyses comparing the nitrate reductase assay with the sensitivity test are recommended to evaluate the cost-benefit ratio.

Eculizumab is a monoclonal antibody indicated for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) or with atypical hemolytic uremic syndrome (aHUS). In Brazil in recent years eculizumab was the most expensive drug requested through court orders, obliging public health managers to import it from the USA. From 2012 to 2016, approximately BRL 424 million (USD 112 million) was spent on eculizumab. The purpose of this study was to assess the regulatory situation and the scientific evidence on the safety and efficacy of eculizumab.

A literature search was conducted in PubMed, The Cochrane Library, and the Centre for Reviews and Dissemination databases on September 2017. The websites of regulatory agencies were also searched.

In 2007, the use of eculizumab was approved by the United States Food and Drug Administration and the European Medicines Agency. In Brazil, despite the provision of eculizumab through judicial proceedings since 2009, the manufacturer of eculizumab only requested a licence for it in 2017, after several meetings with the government when the company agreed to provide the drug at approximately half the price of the imported product. The efficacy of eculizumab in PNH patients was assessed in one randomized, placebo controlled study, one single arm study, and one long-term extension study. The drug reduced hemolysis and the need for transfusion, although the studies had methodological problems. The efficacy of eculizumab in the treatment of aHUS was assessed in four prospective, controlled open-label studies, two long-term extension studies, and one retrospective study. Eculizumab normalized platelet counts and reduced the need for plasmapheresis, although the studies had no control group. Eculizumab was well tolerated, with no meningococcal infections occurring after patients were immunized.

Some companies have no interest in licensing their products in Brazil because their provision by judicial proceedings is more lucrative. This situation promotes litigation and irrational prescription of drugs, and also obligates the Brazilian government to import expensive health products.

Vemurafenib plus cobimetinib (VC) for the treatment of metastatic melanoma was requested to be included in the National Formulary in Uruguay. The standard of care for metastatic melanoma in Uruguay is dacarbazine. There is no published head-to-head trial assessing the effects of VC versus dacarbazine. The objective of this study was to perform an indirect comparison of the effects of dacarbazine, compared with VC, based on the results of trials that included both treatments versus the same comparator (vemurafenib alone).

We searched Pubmed and The Cochrane Library for trials comparing either VC or dacarbazine with vemurafenib. Trials were assessed in terms of risk of bias, similarity of interventions and inclusion and exclusion criteria, and comparability of characteristics of patients in the vemurafenib arm. We performed an indirect comparison using the Bucher method.

From the literature search we retrieved two studies that met the inclusion criteria: a randomized clinical trial that assessed VC versus vemurafenib or placebo and another assessing dacarbazine versus vemurafenib. Both studies were similar in terms of methodological quality, inclusion and exclusion criteria, and comparability of the vemurafenib arms. However, the comparison of overall survival and progression-free survival curves for the vemurafenib arms were quite different between the two trials. At 9 months, overall survival was eighty-one percent and fifty-five percent and progression-free survival was thirty percent and fifteen percent, respectively. The indirect comparison provided the following hazard ratios: 0.24 (95% confidence interval [CI]: 0.14–0.48) for overall survival; 0.13 (95% CI: 0.09–0.19) for progression-free survival; and 0.15 (95% CI: 0.02–1.29) for grade 4 adverse events.

Treatment with VC increased overall survival and progression-free survival, compared with dacarbazine. Severe adverse events were less frequent with the combined therapy. However, the differences in the vemurafenib survival curves increases doubts about the accuracy of the indirect estimators of overall survival and progression-free survival.

Rising costs and the rapidly increasing volume of findings from research in health care are driving the demand for comprehensive information to inform the allocation of resources. Health technology assessment (HTA) applies rigorous processes to provide high-quality synthesized information to policymakers and healthcare payers. HTA involves combining large amounts of research publications to systematically evaluate the properties, effects, and impacts on a topic of interest.

The time and resources required to complete a full HTA are often demanding. There is an opportunity to apply high-performance computing (inclusive of artificial intelligence and machine learning disciplines) to HTA. This project applied high-computing technology to create a research synthesis tool to support HTA and then developed a service that integrates as much relevant data as possible to strengthen HTA. This was a joint project that combined expertise from the areas of health technology, machine learning, information technology, and innovation.

The information gathered for this phased project from HTA subject matter experts and other stakeholders was collated to inform a research synthesis tool and a broader concept of the project.

The results of this study will inform the design of a research synthesis tool that covers the entire HTA process (literature search, screening titles and abstracts, data extraction, quality assessment, and analysis). The collaborators included Alberta Innovates, the Alberta Machine Intelligence Institute, the University of Alberta, Cybera, and PolicyWise. Alberta Innovates, which is an accelerator and innovator of research in the province of Alberta, Canada, was the primary source of funding for this project.

Deep brain stimulation (DBS), which uses an implantable device to modulate brain activity, is an adjunctive treatment for partial-onset seizures in patients with medically refractory epilepsy. Our objective was to perform an exploratory cost-utility analysis of DBS in conjunction with medical therapy, compared with medical therapy alone, using the latest clinical data and costs for the Australian healthcare system.

A deterministic five-state Markov model was used to project treatment response and outcomes over the patients’ lifetimes, based on 5-year data from the recent Stimulation of the Anterior Nucleus of the Thalamus for Epilepsy (SANTE) DBS trial and drug outcome data identified through a literature search. Costs were based on 2017 data for the Australian healthcare system, and response-specific utilities were derived from published literature. We estimated the lifetime discounted incremental cost-effectiveness ratio (ICER) in Australian dollars per quality-adjusted life-year (QALY) for patients 36 years of age, fifty-five percent of whom were men. Costs and effects were discounted at five percent per annum. The robustness of projections was evaluated through scenario and sensitivity analyses.

Under assumed continued treatment benefit, DBS was projected to add 3.48 QALYs over the patients’ lifetimes, at an increased cost of AUD 142,304 (USD 105,960), resulting in an ICER of AUD 40,951 (USD 30,492) per QALY gained. Reducing the analysis horizon to 20 years increased the ICER to AUD 49,803 (USD 37,083). Increasing the DBS generator life from 3 to 6 years decreased the ICER to AUD 23,956 (USD 17,838) per QALY. Longer follow-up periods and younger age at treatment were associated with greater cost effectiveness. Results were sensitive to assumptions about health state-specific utility estimates and long-term treatment effects.

Our exploratory findings suggest that DBS is a cost-effective treatment strategy in the Australian healthcare system for patients with medically refractory epilepsy. DBS therapy might meaningfully improve patient outcome at a health economic profile that compares favorably to other well accepted therapies. Consideration of indirect costs would further add to this value proposition.

Mechanical ventilation in the intensive care unit (ICU) increases the risk of hospital-acquired conditions (HACs) such as ventilator-associated pneumonia (VAP) and pressure injuries (PrI). Continuous lateral rotation therapy (CLRT) has been shown to reduce VAP and PrI incidence, but the value of switching to CLRT over standard care is presently unknown. We evaluate the cost-effectiveness of CLRT beds compared to standard care in ICUs and determine the return on investment (ROI) associated with its implementation.

A Markov model was constructed to predict health state transitions from the time of ventilation through 28 days using the healthcare sector perspective. Daily transition probabilities were extrapolated from prospective clinical studies comparing CLRT with standard care. Costs were estimated in 2014 USD. Utility scores were extracted from the published literature. Cost per quality-adjusted life-years (QALYs) was calculated and sensitivity analyses were conducted. A secondary analysis from a societal perspective with a one-year time horizon included the costs of patient and caregiver lost productivity. ROI analysis was performed to estimate the net benefit and breakeven point of the investment. Value of Information analysis was performed to determine whether further research is warranted.

From both perspectives, CLRT was dominant. From the healthcare sector perspective, the expected cost for CLRT per patient was USD 47,165 compared to standard care at USD 49,258 per patient, showing that CLRT saves cost per patient. The expected effectiveness of CLRT per patient was 0.0418 QALYs compared to 0.0416 QALYs for standard care. CLRT was dominant in 99.94 percent of Monte Carlo simulations. CLRT also reached the break-even point after 5 months. Expected Value of Perfect Information was equal to 0.019, indicating little value of additional evidence at the current level of parameter uncertainty.

CLRT is highly cost-effective compared to standard care by preventing ventilator-associated infections and PrIs in an ICU setting.

Minimally invasive capsulorhexis is an incision in the anterior capsule in the peripheral zone for cataract extraction. It allows reduction of the size of the lesion, ensuring a better transparency of the visual axis, preserving the capsule almost intact and a layer of lenticular epithelial cells. The procedure could have a potential regenerative effect of the lens in a natural way. The objective of this study is to assess the efficacy and safety of minimally invasive capsulorhexis to promote lens regeneration in children's cataracts.

This technology was identified by the early Awareness and Alert System, “SINTESIS-new technologies” of Agencia de Evaluación de Tecnologías Sanitarias (AETS) Instituto de Salud Carlos III (ISCIII). An early assessment was conducted. The searched databases were: PubMed, Centre for Reviews and Dissemination (CRD), and Cochrane Library. Clinical studies using the procedure published in any language until 29 September 2017 were reviewed.

An open-label, randomized trial in pediatric cataract patients (age: 0–2 years) was retrieved. Twelve patients underwent minimally invasive capsulorhexis, while twenty-five patients received the standard treatment. Regarding efficacy, a transparent regenerated biconvex lens was found in 100 percent of eyes three months after surgery, but wasn't found in the control group. 100 percent of capsular openings healed within one month after surgery in the experimental group, but not in the control group. Both groups increased their visual acuity parameters without significant differences. Regarding safety, children receiving the standard technique had a higher incidence of corneal edema (eight percent in the intervention vs thirty percent in the control group), anterior chamber inflammation (seventeen percent vs seventy-four percent), additional laser capsulotomy (zero percent vs eighty-four percent) and increased visual axis opacification (four percent vs eighty-four percent).

Minimally invasive capsulorhexis in children's cataracts seems to be a promising new procedure. Preliminary efficacy results were good and safety profile was better than standard treatment. However, it would be necessary to continue further studies to confirm these results.

Systematic reviews are useful for identifying gaps in research, setting priorities for future research, and informing clinical practice and public policy decisions. However, appropriate methods are needed to ensure that systematic reviews are of suitable quality in order to maximize their potential to achieve impact. The aim of this study was to evaluate the quality and transparency of systematic reviews conducted on prostate artery embolization (PAE), a topic of considerable interest in urology.

We conducted a cross-case analysis. Existing reviews were identified through a systematic search of four biomedical databases (Cochrane Library, York Centre for Reviews and Dissemination, Embase, Medline) from inception up to 8 December 2016. Systematic reviews that evaluated the safety and effectiveness of PAE to treat benign prostatic hyperplasia were included. Included reviews were critically appraised using the AMSTAR (A MeaSurement Tool to Assess systematic Reviews) tool, and were scored against the PRISMA (Preferred Reporting Items for Systematic reviews and Meta-Analyses) criteria.

From 536 search results, nine relevant systematic reviews were identified, of which eight were published in 2016. None of the included reviews were prospectively registered on the PROSPERO database. The median AMSTAR score was four of 11 (range 0–7). The most common methodological concerns were related to comprehensive searches (33 percent), inclusion of grey literature (0 percent), and evaluation of publication bias (0 percent). Reviews adequately reported a median of 17 of 21 items (range 6–19) against the PRISMA checklist.

Despite the availability of robust guidelines for conducting systematic reviews, methodological limitations in reviews of PAE are prolific, leading to considerable heterogeneity. There is also a significant duplication of effort, which can be prevented by prospectively registering systematic reviews on PROSPERO. Reducing duplication and increasing methodological quality are imperative to reducing waste in urological research.

Failure at the proximal neck for endovascular aortic repair (EVAR) in abdominal aortic aneurysm (AAA) is more common in the presence of unfavorable proximal neck anatomy. In patients with hostile neck, EndoAnchors provide proximal fixation and reduces potential type I endoleak or endograft migration. However, the population size for AAA patients with hostile anatomic neck among Korean is unknown and cost-analysis with regard to EndoAnchors has not been established.

To figure out the population size of AAA patients with hostile neck anatomy, retrospective medical chart review was conducted from four major medical centers. Hostile proximal aortic neck was defined as any or all of neck length 28 mm, infrarenal neck angulation >60°, ≥50 percent of circumferential thrombus, ≥50 percent of calcified neck, and conical neck. Cost-analysis on EndoAnchor use for treatment purpose was conducted based on Korean National Health Insurance Claims dataset (HIRA-NIS 2015).

Two-hundred and ten patients’ anatomic data treated with EVAR were included; 130 (61.9 percent) patients met the criteria for a hostile aortic neck and 32 (15.2 percent) patients had multiple hostile anatomy parameters. Endograft migration was reported in four (1.9 percent) patients and intra or post-op type I endoleak was reported in 21 (10.0 percent) patients. Based on 1-year claims data, 1,607 patients were treated with EVAR in 2015 and the annual average medical costs for open repair were USD 16,151. Given the patients with type I endoleak or endograft migration needs open repair if not treated with EndoAnchors, the estimated annual costs for patients treated with EndoAnchor were USD 2,234,321 and those for patients without EndoAnchor were USD 2,595,508, therefore USD 361,187 can be saved annually.

The population size with hostile aortic neck in Korea was comparable with those in western countries. Economically, EndoAnchor is a cost-saving treatment for type I endoleak and migration after EVAR from Korean payer.

Health technology assessments (HTAs) are increasingly used by Norwegian health authorities as the evidence base when prioritizing which health care services to offer. HTAs typically consist of a systematic review of the effects and safety of two or more health care interventions, and an economic evaluation of the interventions, based on systematic literature searches in bibliographic databases. Objective: To identify the best performing of seven search filters to retrieve health economic evaluations used to inform HTAs, by comparing the cost-effectiveness analysis (CEA) filter to six published filters in Ovid Embase, and achieve a sensitivity of at least 0.90 with a precision of 0.10, and specificity of at least 0.95.

In this filter validation study, the included filters’ performances were compared against a gold standard of economic evaluations published in 2008–2013 (n = 2,248) from the National Health Service Economic Evaluation Database (NHS EED), and the corresponding records (n = 2,198) in the current version of Ovid Embase.

The CEA filter had a sensitivity of 0.899 and precision of 0.029. One filter had a sensitivity of 0.880 and a precision of 0.075, which was closest to the objective. The filter with lowest sensitivity (0.702) had a precision of 0.141.

Developing search filters for identifying health economic evaluations, with a good balance between sensitivity and precision, is possible but challenging. Researchers should agree on acceptable levels of performance before concluding on which search filter to use.

Candidate health technologies identified for disinvestment will require prioritization depending on the system's capacity for dealing with the assessments or for further considerations. Compilations of low value lists, such as the National Institutes for Health and Clinical Excellence's, “Do not do recommendations”, can serve as databases for prioritization topics. Prioritization processes can also be triggered by experience or event-based regional requests and decisions; new evidence on safety, effectiveness and cost-effectiveness, variations in clinical practice, patient or consumer voicing, discrepancies between practice and guidelines; and or time-based mechanisms, such as approval of new health technologies and reassessment five years after introduction.

A search of the published and grey literature was conducted to identify the current methods or tools used to prioritize potential health technologies and services for disinvestment. The description of the methods and tools identified, the prioritization criteria, and the stakeholders involved in the process were reviewed and summarized.

The methods and tools used for prioritization that were identified in the literature include the PriTec Prioritization tool, nominal group technique, Program Budgeting and Marginal Analysis, consensus building, and online surveys. Further, common criteria for prioritization centered on the disease burden, possible risks and benefits, costs and cost-effectiveness, utilization, and time-based criteria. Prioritization can be conducted by health care professionals, decision makers, patients or patient groups and representative community members.

The prioritization process for disinvestment candidates should be transparent and guided largely by evidence. It is highly recommended that the list of predefined criteria be developed with input from all relevant stakeholders to meet the objectives of the specific health care setting. The commonly cited basic requirements include clinical parameters, economic measures, and social, ethical or legal considerations.

The pan-Canadian Oncology Drug Review (pCODR) program was established by Canada's provincial and territorial Ministries of Health (except Quebec) to assess cancer drug therapies and make recommendations to guide drug reimbursement decisions. The pCODR Expert Review Committee (pERC) makes reimbursement recommendations, providing a rationale for the recommendation and next steps for stakeholders. The objective of this analysis was to identify reviews and reasons pERC has requested real-world evidence (RWE) data collection.

A retrospective analysis of pERC Final Recommendations (January 2012 – May 2017) was conducted. pERC Final Recommendations include drug information, reimbursement recommendation, rationale for recommendation following pERC's Deliberative Framework (clinical benefit, patient-based values, economic evaluation, and adoption feasibility), next steps for jurisdictions to consider to support their funding decisions, summary of deliberations, and evidence in brief. Reviews were included if there was a next step advising the collection of RWE to reduce uncertainty in the drug under review.

Out of eighty-four reviews, forty-one (forty-eight percent) included a next step to collect RWE to address a gap in the available evidence. Reasons for RWE data collection, in descending order of frequency, were to inform: sequencing of available therapies; magnitude of clinical benefit and cost-effectiveness or the true cost-effectiveness; duration of treatment and cost-effectiveness; defining the population or disease progression; quality of life; and dosage.

In almost half of pERC's recommendation there is an indication that there is a gap in the existing evidence that could potentially be addressed through the collection of RWE. This reflects the rising number of new cancer drugs, limited evidence supporting submissions (for example non-comparative studies), and newer drugs such as immunotherapies which may not have a fixed treatment duration. Further research includes development of mechanisms for RWE data collection to help inform pERC recommendations and assist stakeholders with adoption feasibility of reviewed drugs.

Cystic fibrosis (CF) is the most common autosomal recessive disorder in Caucasians, occurring in one out of every 2,500–2,800 births worldwide, and is associated with a high burden of disease. In Australia, prenatal testing for CF is indicated for pregnant couples identified as carriers or when a fetus is found to have an ‘echogenic bowel’ (FEB). We aimed to determine the effectiveness of prenatal CF testing and to assess ethical dimensions. A key challenge in assessing a prenatal test is selecting appropriate endpoints to indicate clinical effectiveness.

A systematic review was conducted and a linked evidence approach was used to answer the effectiveness question. The literature on ethical considerations relating to prenatal testing was also reviewed.

No studies were identified on the direct effectiveness of prenatal CF testing or downstream consequences. Linked evidence showed good diagnostic performance with a test failure rate of 4.5 percent. Termination of pregnancy occurred in the majority of cases where two mutations were identified in a fetus of carrier parents (155/163; 95 percent), indicating testing impacts clinical management. In FEB cases with CF, termination occurred in around sixty-five percent of pregnancies. Both terminating a pregnancy and having a child with CF were associated with poor short term parental psychological outcomes. Evidence indicates prenatal testing leads to a decreased number of CF-affected births. However, ethical analyses indicated that ‘informed decisions’ should have been the primary outcome of interest.

Proper counselling prior to testing ensures that the aim of prenatal testing is informing reproductive choices in a non-directive way, rather than decreasing the number of CF-affected births (which is ethically problematic). These results suggest that for health technology assessments undertaken on contentious topics, ethical analysis should be undertaken first so appropriate endpoints are selected for the subsequent systematic review of clinical evidence and for the economic model.

The Ministry of Health in Uruguay has a health technology assessment division that provides decision makers with evidence-based information on the efficacy, safety, and costs of health technologies to be included in the Comprehensive Plan of Health Care. Since 2010, patients have begun to demand access to unfunded, high-cost technologies through writs of protection. Judicialization of the right to health increased rapidly from 2010 to 2014. In this context, a Technical Advisory Commission was created in 2015 to assess patient requests on a case-by-case basis. The purpose of this study was to evaluate the results obtained with a new strategy developed to face the judicialization of access to high-cost technologies.

The methodology used to evaluate the implementation of the strategy consisted of reviewing a database of access requests from October 2016 to October 2017. The demographic characteristics, technologies requested, prescriptions, and results of the process were analyzed.

In the study period 654 technologies were requested for funding through the process. The included population had a mean age of 60 years; sixty-two percent were men. Of the technologies requested, eighty-five percent were drugs and fifteen percent were devices. The requested technologies included cancer treatments (thirty-five percent) or drugs and devices for the treatment of rheumatologic, ophthalmologic, infectious, neurologic, and cardiovascular conditions. The six most requested technologies (forty-five percent of all requests) were: abiraterone for prostate cancer; aortic endoprosthesis for vascular aneurysm; lenalidomide, rituximab, and azacitidine for oncohematologic diseases; and cetuximab for colorectal cancer. The Ministry of Health funded thirty-six percent of the requests.

The new strategy was successful in reducing the judicialization of access to unfunded, high-cost technologies in Uruguay, and it helped to prioritize the inclusion of new drugs in the national formulary.

Trastuzumab is the most recent biological therapy incorporated by the Brazilian Health Ministry for HER-2 overexpressed metastatic breast cancer treatment (2012). The aim of this study was to investigate if access to this technology is appropriate.

We performed a web-based questionnaire, which received answers from October 2016 to April 2017. Oncologists that work in the care of patients with overexpressed HER-2 metastatic breast cancer were the focus of the survey. Forty-three professionals informed work location, sector (public, private or both) and trastuzumab access. This research was approved by Brazilian Ethics Committee (CAE 59076316300005260).

Among 43 valid answers, nine informed they work in the public sector, 10 in the private sector and 24 in both sectors. In total, 33 reported to work in public and 34 in private sector. We observed that 17 (51.5 percent) participants who work in the public sector do not have access to trastuzumab, while in private sector only one participant (2.9 percent) reported the lack of access to this technology for HER-2 overexpressed metastatic breast cancer treatment. Regarding the respondents who informed the lack of access, six (33.3 percent) work in Northeast Brazilian region, six (33.3 percent) in Southeast, two (11.1 percent) in South, one (5.6 percent) in Central-West and three (16.7 percent) did not give this information. Eleven respondents reported they do not have another treatment option for these patients, while seven informed access only to chemotherapy without biological therapy.

Trastuzumab is a biological therapy that can increase HER-2 overexpressed metastatic breast cancer patients overall survival by nine months on average. The questionnaire results indicate that its access in Brazil is still irregular, mainly in public sector, even five years after its incorporation by the Brazilian Health Ministry. Although universal access is one of Brazilian Public Health System main directives, there is evidence of a serious issue regarding its services equity.

Since established in 2009, the National Evidence-based healthcare Collaborating Agency (NECA) has been the sole government-funded Health Technology Assessment (HTA) institution in Korea, yet little effort has been made to systematically evaluate the influence of its products. In this study, we aimed to measure the impact of the HTA products of NECA on clinical and policy decisions by introducing a systematic framework.

We included HTA reports published from 2009 to 2015. Among the 141 research reports published during this period, there were 67 HTA reports. We gathered data on the influence by literature and news article search, review of administrative documents and directly listening to the decision makers. The influence was categorized into three decision types: changes in clinical guidelines, administrative decision on investment/disinvestment and healthcare policy making. Whether a research report was used directly in decision making, or followed by subsequent researches or round-table conference, was recorded to examine the knowledge transfer process.

In total, 67.2 percent of the included HTA reports were used to support clinical and policy decisions. Twenty-seven reports had influenced administrative decisions on investment/disinvestment. Ten provided evidence for new health policies or legislation. Eight were reflected in clinical guidelines. The impact of HTA reports published by NECA was more evident when the research was directly requested by decision-making bodies such as government institutions. Although most HTA reports were conducted in collaboration with clinicians, the use of results by clinicians was limited. Definitive results were more likely to be used, but reports with competing interests had fewer impacts.

HTA by NECA had impacts on the rational use of healthcare resources in Korea, and NECA has established its role as an intermediary between governmental decision-making bodies and clinicians. However, more continuous approaches rather than one-time HTA research are needed for HTA on controversial topics to have impacts on decision making.