The Unified Health System (SUS) is based on the principle of health as a citizen's right and the state's duty, which must be guaranteed based on public policies. Although there are several legislations, lists of medicines and clinical guidelines, Brazilians who have been prescribed expensive technologies that are not part of the essential drug lists ask judges to issue court orders obliging public health managers to purchase these drugs or to provide elective medical procedures immediately. Due to the health technical inexperience from judges, prosecutors and public lawyers, a partnership has arisen for the National Committee for Health Technology Incorporation (CONITEC) to provide technical assistance to help their decision-making process. Thus the purpose of this study is to describe CONITEC's experiences in communicating with stakeholders in this process.

A case study method was used and information about the rapid reports developed by CONITEC's Executive Secretariat in response to the applicants in the period of 2012 to 2016, was retrieved from CONITEC database.

Rapid reports (2,773) about health technologies incorporation such as medicines, procedures or medical devices were produced by CONITEC during this period. Most requests covering topics as treatments for diabetes, arterial hypertension, osteoporosis, oncology and epilepsy; diseases for which there are several treatment options in SUS. The data analysis indicated that CONITEC contributed to the evidence based decision-making. On one hand, the Prosecutor's Office has been increasingly requesting information before starting lawsuits and Judiciary Power has increasingly used evidence-based technical information before deciding on the concession of injunctions; on the other hand, from 2012 to 2016 the number of requests decreased for information to State defense in lawsuits that has been already established.

There is a growing interest in technical knowledge for fair decision making that respects the current organization of the evidence-based health system.

The main difficulties encountered in the integration of ethics in Health Technology Assessment (HTA) were identified in our systematic review. In the process of analyzing these difficulties we then addressed the question of the diversity of ethical approaches (1) and the difficulties in their operationalization (2,3).

Nine ethical approaches were identified: principlism, casuistry, coherence analysis, wide reflexive equilibrium, axiology, socratic approach, triangular method, constructive technology assessment and social shaping of technology. Three criteria were used to clarify the nature of each of these approaches:

1. 1. The characteristics of the ethical evaluation

2. 2. The disciplinary foundation of the ethical evaluation

3. 3. The operational process of the ethical evaluation in HTA analysis.

In HTA, both norm-based ethics and value-based ethics are mobilized. This duality is fundamental since it proposes two different ethical evaluations: the first is based on the conformity to a norm, whereas the second rests on the actualization of values. The disciplinary foundation generates diversity as philosophy, sociology and theology propose different justifications for ethical evaluation. At the operational level, ethical evaluation's characteristics are applied to the case at stake by specific practical reasoning. In a norm-based practical reasoning, one must substantiate the facts that will be correlated to a moral norm for clearly identifying conformity or non-conformity. In value-based practical reasoning, one must identify the impacts of the object of assessment that will be subject to ethical evaluation. Two difficulties arise: how to apply values to facts and prioritize amongst conflicting ethical evaluations of the impacts?

Applying these three criteria to ethical approaches in HTA helps understanding their complexity and the difficulty of operationalizing them in HTA tools. The choice of any ethical evaluations is never neutral; it must be justified by a moral point of view. Developing tools for ethics in HTA is operationalizing a specific practical reasoning in ethics.

Randomized controlled trials (RCTs) of complex interventions are conducted in a context-specific environment. Principal trial reports, with a focus on main results, are unable to document adequately the context for an intervention, for example, word limits. Important context may be included in “sibling studies” (that is, studies conducted alongside the main trial, for example, process evaluations, and qualitative studies (1). This presentation explores (i) to what extent is it possible to use a systematic and parsimonious method to identify sibling studies and (ii) what is the potential value of yield from these studies?

The systematic CLUSTER approach (2) to follow up of index studies (Citations, Lead authors, Unpublished materials, Scholar, Theories, Early examples Related projects) has demonstrated the value of retrieved items in qualitative terms. However, the CLUSTER approach is painstaking and laborious and may be prohibitive within a time-tight Health Technology Assessment (HTA). A streamlined CLUSTER approach, using freely available Publish or Perish Software integrated with Google Scholar and Microsoft Excel, offers an economical way of building up “clusters” of study reports. A case study of a UK National Institute for Health Research (NIHR)-funded HTA on the management of medically unexplained symptoms in primary care, utilizing quantitative and qualitative research studies, is used to examine the practical application of the approach.

Systematic comparison of yield from sifting with yield from the Publish or Perish software reveals (i) major trials for which corresponding qualitative studies were not previously identified, (ii) qualitative studies identified independently from, and potentially unlinked to, associated trials, (iii) associated trial reports (for example, protocols, feasibility studies, etc), economic evaluations and systematic reviews, and (iv) commentaries and correspondence; all with the potential to enhance understanding of trial context.

The potential of the Publish or Perish-enabled CLUSTER approach to identify trials or qualitative studies, through “joining up” and mapping of clusters, potentially missed from separate quantitative/qualitative sift processes, means that it should be considered for any HTA that seeks to integrate quantitative and qualitative studies.

Multiple databases are often searched in Health Technology Assessment systematic reviews. However in rapid reviews, time and resources are limited and modifications to the search methodology may be necessary. In this retrospective study, the impact of searching fewer databases for three completed rapid reviews (i) Severe Mental Illness (SMI), (ii) Cannabis Cessation (CC), iii) Premature Ejaculation (PE) for the United Kingdom National Institute for Health Research was investigated.

The database coverage and indexing of the study references from the reviews were initially identified. The impact of fewer databases searched was then tested by (i) the number of studies that might be missed, (ii) the number of records for sifting and (iii) the overall rapid review conclusions.

A total of 178 included study references were found in the reviews (SMI n = 14 for 13 studies, CC n = 34 for 33 studies, PE n = 130 for 102 studies). Searching Medline only for SMI, Medline+Embase for CC, Medline+Embase+Cochrane Library for PE, would result in 1902 (74 percent), 466 (43 percent) and 240 (11 percent) fewer records needed to sift, respectively. There would also be a total of ten ‘would be missed’ references (SMI n = 1, CC n = 5 and PE n = 4). However, nine out of the ten references were found to have no or minimal impact on the overall findings of the reviews. The ten references were secondary reports of an included study, papers that lacked sufficient data for meta-analysis such as a conference abstract or an ongoing trial.

From the three reviews examined, limiting the search to fewer databases had no or minimal impact on the review conclusions despite the variable number of studies that would be missed and records needed to sift. More exploration during the scoping search prior to commencing the review will aid the decision on whether to limit the search to fewer databases.

Evidence about which information resources should be searched to identify economic evaluations (EEs) of healthcare interventions when conducting a systematic review (SR) predates closure of the National Health Service Economic Evaluation Database (NHS EED) and Health Economic Evaluations Database (HEED). We assessed which databases are now the best sources of EEs and identify the most efficient combination of databases, taking into account the order in which databases could be searched.

We gathered a reference set of EEs from published reviews of EEs undertaken to inform Health Technology Assessments (HTA). We calculated yield and relative recall (RR) (number of reference set records identified / total number of records in reference set) for each database, and combination of databases. We assessed the order in which databases should be searched, to identify the most efficient combination of databases to identify the reference set. We report the characteristics of records not included in any database studied and implications for identifying this type of evidence.

To date, a reference set of fifty-five EEs from seven HTAs has been processed. Embase and Scopus each yielded 53/55 records (RR .96). MEDLINE yielded 52/55 (RR .95). Embase or Scopus included all of the journal publications in the reference set; no additional unique records were provided by MEDLINE, CEA Registry, EconLit, or Science and Social Science Citation Indexes. The two records that were not identified were unpublished evidence, one of which was included in the National Institute for Health Research (NIHR) HTA database. Processing will continue until we reach the threshold of a reference set of 350 records.

Preliminary results suggest that searching two or three databases may be most efficient, provided that resources are searched using appropriate strategies. Searchers should concentrate on developing search strategies that work well in those databases to ensure adequate sensitivity, and use freed time to identify grey literature.

Health Technology Assessment (HTA) reports adaptation process is an important tool for emerging HTA agencies. INASanté (National Instance for Accreditation in Healthcare) has chosen to rely on this approach, to develop its first health intervention assessment report: comparative study of computed tomographic colonography versus standard colonoscopy for colorectal cancer screening.

Following consultations with healthcare professionals, the PICO question related to the colorectal cancer screening issue in Tunisia was determined. A literature search strategy covering 10 years (2006-2016) was carried out. Several databases including HTA on the net were explored. Then two independent reviewers conducted literature screening and realized a PRISMA flow diagram. Full text selected reports were submitted to three critical appraisal tools: PRISMA checklist, INAHTA checklist and Critical Appraisal Tools (FLC 2.0). The EUnetHTA adaptation toolkit was used to determine reports adaptability by assessing relevance, reliability and transferability. A structured study of the Tunisian context based on a qualitative data analysis was elaborated. The data synthesis and reporting were finalized with the contribution of a working group. Then an external peer review was conducted before the report dissemination.

Eighty reports were screened to finally retain four eligible. After a critical appraisal performed by two independent reviewers, two reports from the Canadian Agency for Drug and Technolgies in Healthcare and AETSA were selected to be assessed using the EUnetHTA adaptation toolkit. Regarding transferability criteria, the second report was retained. The context study has consisted in a qualitative analysis of seventeen individual interviews with healthcare professionals involved in colorectal cancer screening issues and an up to date Tunisian literature review. The final adapted report was a combination between relevant extracted data from AETSA report and synthesis of the Tunisian context analysis.

This HTA report represents a tool for policy makers to establish the appropriate colorectal cancer screening program for the Tunisian context. HTA reports adaptation process is the best way to give evidence on emerging technologies without wasting time and resources.

Although there is increased awareness of patient and public involvement (PPI) among Health Technology Assessment (HTA) organizations, evaluations of PPI initiatives are relatively scarce. Our objective as members of HTAi's Patient and Citizen Involvement Group (PCIG) was to advance understanding of the range of evaluation strategies adopted by HTA organizations and their potential usefulness.

In March 2016, a survey was sent to HTA organizations through the International Network of Agencies for Health Technology Assessment (INAHTA) and contacts of members of HTAi's PCIG. Respondents were asked about their organizational structure; how patients and members of the public are involved; whether and how PPI initiatives have been evaluated, and, if so, which facilitators and challenges to evaluation were found and how results were used and disseminated.

Fifteen programs from twelve countries responded that involved patient (14/15) and members of the public (10/15) in HTA activities. Seven programs evaluated their PPI activities, including participant satisfaction (5/7), process evaluations (5/7) and impact evaluations (4/7). Evaluation results were used to improve PPI activities, identify education and training needs, and direct strategic priorities. Facilitators and challenges revolved around the need for stakeholder buy-in, sufficient resources, senior leadership, and including patients in evaluations. Participants also provided suggestions based on their experiences for others embarking on this work, for example including patients and members of the public in the process.

We identified a small but diverse set of HTA organizations internationally that are evaluating their PPI activities. Our results add to the limited literature by documenting a range of evaluation strategies that reflect the range of rationales and approaches to PPI in HTA. It will be important for HTA organizations to draw on formal evaluation theories and methods when planning future evaluations, and to also share their approaches and experiences with evaluation.

Multiple drug resistance (MDR) intra-abdominal infections (IAIs) are associated with noteworthy direct and societal costs. Compared to previous studies, the present one takes both resistance rate and total medical costs (TMCs) into consideration, focusing on the impact of MDR on TMCs in IAIs, as well as further estimating the additional costs at a national level.

All inpatients discharged between 1 January 2014, and 31 December 2015 from a teaching hospital were included. Due to limits in budget and the large number of inpatients, the randombetween (bottom, top) function was applied to randomly select 40 percent of patients per year. Subsequently, we manually screened out 254 patients with IAIs, according to the International Classification of Disease (tenth revision) and electronic medical records. Eventually, 101 IAIs patients were included, in which 37 were infected by non-MDR bacteria and 64 by MDR bacteria. The Kruskal-wallis non-parametric test and multiple linear regression were employed to analyze the effect of single and multiple variables on TMCs.

Compared to patients with non-MDR infections, those with MDR were associated with significantly higher TMCs, higher antimicrobial costs, increased insurance, combination antimicrobial therapy, higher usage of antimicrobial agents, greater number of pathogens, longer length of stay, and longer intensive care unit stays. In addition, the average TMCs among patients with MDR were CNY131,801.17 (1USD was equal to CNY 6.227 in 2015), which were CNY 90,200.99 higher than those with non-MDR infections. If our results are generalizable to the whole country, the total attributable TMCs are estimated to be CNY37.06 billion, and the societal costs of CNY111.18 billion in 2015.

This real-world data analysis demonstrated the significant excessive burden MDR infections are posing to the current Chinese healthcare system in terms of both TMCs and healthcare resource utilization. Enhanced antimicrobial stewardship in China is necessary to curb the distribution of MDR bacteria.

Heart failure (HF) is a major public health problem worldwide and in Asia. Sacubitril/valsartan reduces cardiovascular death and hospitalizations for HF. However, decision makers need to determine whether its benefits are worth the additional costs, given the low-cost generic status of current standard of care.

Using a Markov model, we projected lifetime clinical and economic outcomes of sacubitril/valsartan versus enalapril for 66-year-old patients with HF in Singapore. Key health states included New York Heart Association (NYHA) classes; patients in each state incurred a monthly risk of hospitalization for HF and cardiovascular death. Probabilities of events were based on the PARADIGM-HF trial. The uncertain treatment effect of sacubtril/valsartan in Asian patients was modelled using a hazard ratio (HR) of 1 as upper limit in sensitivity analyses. Utilities were obtained from published literature. Local national epidemiological and cost data were applied. Analyses were conducted from the Singapore healthcare payer's perspective. Both one-way and Probabilistic Sensitivity Analyses (PSA) based on 10,000 Monte Carlo simulations were performed.

Compared to enalapril, sacubitril/valsartan was associated with an incremental cost-effectiveness ratio (ICER) of SGD74k (USD52k) per quality-adjusted life year (QALY) gained. The cost-effectiveness of sacubitril/valsartan was highly dependent on its effectiveness in reducing the risk of cardiovascular death. However, this was uncertain, particularly in the Asian subgroup, where results were not statistically significant. In sensitivity analyses using results from Asian patients, the ICERs ranged from SGD41k (USD30k) to SGD1.3 million (USD 0.94 million) per QALY gained. PSA showed the probability of sacubitril/valsartan being cost-effective was below 1 percent, 12 percent and 71 percent at thresholds of SGD20k (USD14k), SGD50k (USD36k) and SGD100k (USD 72k) per QALY gained, respectively.

Given the uncertain ICER, sacubtril/valsartan may not provide good value for money compared to enalapril in reducing cardiovascular morbidity and mortality in patients with HF at the current daily cost. Our study highlights the cost-benefit trade-off that healthcare professionals and patients face when considering HF therapy.

Relapsed/metastatic head and neck squamous cell cancer patients are offered a combination of platinum-based chemotherapy (PF, cisplatin-fluorouracil) plus cetuximab regimen (PF+C) according to results of the EXTREME trial (1). However, two economic evaluations showed that addition of cetuximab was not cost-effective.

This study aimed to evaluate the cost-effectiveness of a putative predictive molecular test (MT) to identify and treat only patients potentially responsive to cetuximab when added to PF.

A Markov model was developed to compare both health and economic outcomes of PF+C regimen administered to all patients (PF+C ALL) versus the regimen administered only to MT-positive patients (PF+C POS).

The model considered the following health states: partial/complete response with/out mild/severe adverse events (AEs), progression and death. Rates of progression and survival, response rates to systemic treatment and adverse events were retrieved from the EXTREME trial (1). According to Mesía et al. (2), we assumed that addition of cetuximab to PF would not negatively affect life quality compared to PF alone, and the baseline utility coefficients for disease control and progression were assumed as .67 and .52, respectively.

Only direct costs estimated from the Italian Health Service perspective were included (tariffs and Diagnosis Related Group - DRG - reimbursements).

The model was evaluated according to a cut-off of sensitivity at 85 percent and specificity at 70 percent. A 3 years horizon was chosen. Life expectancy, quality-adjusted life years (QALYs) and costs were discounted at 3.5 percent annually.

Applying the World Health Organization (WHO) cost-effectiveness threshold of 3 times the gross domestic product for Italy (EUR66,402), PF+C POS resulted a cost-effective choice in comparison to PF+C ALL for a MT cost lower than EUR5,750.

Adding cetuximab to PF only to patients positive to a predictive test may be cost-effective. Efforts should be spent to build such a test upon existing evidences in order to save resources for the health systems and spare unnecessary toxicities to patients.

There is considerable excitement around the development of regenerative medicines (or advanced therapy medicinal products, ATMPs), with the expectation that they may bring substantial clinical gains and offer cures for previous debilitating and fatal diseases. However, high costs mean that Health Technology Assessment (HTA) and reimbursement decisions are challenging for payers and manufacturers, even when the therapies are expected to offer good value for money.

In Europe, seven ATMPs have market authorization, yet only one has achieved national level reimbursement. Statistics such as these put HTA bodies under pressure to review their methods and consider how these can apply to regenerative medicines.

We present a review of one example, from the United Kingdom's National Institute for Health and Care Excellence (NICE), who commissioned an external organization to undertake a mock appraisal of a hypothetical ATMP using standard methods. The therapeutic area chosen for the mock appraisal was chimeric antigen receptor (CAR) T-cell therapy for treating relapsed or refractory B-cell acute lymphoblastic leukaemia.

The role of uncertainty was a key consideration within the report, yet we found that the presentation of uncertainty within the mock appraisal was misleading for decision makers.

We found that the exercise represents a thorough mock HTA of CAR T-cell therapy. However, it focused on testing whether ATMPs could fit into the existing HTA pathway for conventional medicines, rather than seeking to identify the most suitable approach for assessing regenerative medicines. We suggest the latter would have been a more relevant question for the mock appraisal.

Any significant departures from the usual HTA process must be based on solid economic rationale if we are to ensure efficient allocation of resources. Thus, in order for regenerative medicines to be given ‘special treatment,’ it must be demonstrated that societal preferences, or the full extent of health (or non-health) benefits, are not being realised for this group of treatments through existing HTA methods.

Budget impact analysis (BIA) provides short and medium-term estimated effect of new health interventions on budgets and resources. Since January 2016, BIA is required as part of economic dossiers submitted to the French National Authority for Health (HAS) by manufacturers for innovative drugs with an expected 2-year sales revenue above EUR50 million. To this end and in order to promote good practices for conducting BIA, HAS developed a guide for BIA.

Our objectives are:

• – to present the guide development method and the resulting recommendations;

• – to compare the HAS BIA guide with existing BIA guides.

The HAS guide development process rests on findings derived from a systematic literature review on BIA methodology, an HAS retrospective investigation of BIA, public consultation, international expert advice, and approval from the HAS Board and Committee of Health Economic and Public Evaluation (CEESP). Relevant publications were identified through Pubmed and EMBASE and the grey literature (search dates: January 2000 to June 2016).

The search strategy captured 144 publications of which 31 were retained (14 methodological papers, 12 national guides and 5 learned society recommendations). On the basis of this result, an extraction template was designed to synthesize the methodological aspects of BIA. Based on its research findings, HAS developed its first BIA guide which includes recommendations on the following main topics: BIA definition, perspective, populations, time horizon, compared scenarios, BIA models, costing, discounting, choice of clinical data, reporting of BIA and uncertainty exploration.

Compared to existing BIA guides from other Health Technology Assessment (HTA) agencies, the HAS guide specifically described issues relating to off-label use of drugs, disease-related costs and scenario analysis.

It is expected that the HAS BIA guide will improve the quality, transparency and standardization of BIA in France. It should also enhance the usefulness of BIA as an essential part of a comprehensive economic assessment of health care interventions.

The Health Technology Assessment (HTA) of mobile health applications involves significant challenges including rapid product development cycles, sparse evidence and uncertainty over the economic impact. However apps also provide unique opportunities, such as their potential reach and use of real-world data, which will facilitate their contribution to healthcare delivery. The National Institute for Health and Care Excellence (NICE), alongside other agencies, has been piloting the development of a health app assessment programme. This presentation reports the results of a study about the development of the Health App Briefing (HAB) which is designed as the output from a rapid assessment of the effectiveness and cost-saving potential of apps to inform decision makers in the United Kingdom National Health Service.

The HAB is built on the success of the NICE Medtech Innovation Briefings programme because many of the HTA challenges are similar to those found with medical devices. HAB development is grounded in four principles: rapid assessment; transparent process; independence from industry or the health service and input from commentators. The content includes an evidence summary for effectiveness including comments from specialist experts and users; a summary of information relating to the cost saving potential and a summary of other user benefits (including issues of access and usability). Novel features are the presentation of a rating of the potential value of the app to the health system and working with commissioners of the app to obtain real-world information for a case study about the economic impact.

The development of four HABs along with a review of the learning from the piloting process will be presented. The review will include stakeholder feedback from a workshop.

We believe the evaluation of this work presented here will be of interest to other HTA agencies around the world that are deciding how to approach the issues surrounding the assessment of health apps.

Post-approval studies (PAS) constitute an important tool in medical devices (MD) assessment usually supported by registries. However, registries are often poorly designed or incomplete. The French health insurance databases are organized since 2003 into a digital data warehouse, the Système national d'information inter-régime de l'assurance maladie (SNIIR-AM), and is the main source of information on patients, hospital activity and associated expenditure. The aim of the study was to determine if these medico-administrative data can be sufficiently relevant to guide a renewal of MD reimbursement in the context of registry failure.

The initial PAS aimed to assess the impact of the guidelines on practice (characteristics of patients, type of stenosis, indications, use of cerebral protection system, surgical procedure) and to determine the 30-day cumulative morbidity and mortality rate of endovascular procedure associated with stenting. Medico-administrative databases provide information on age, sex, symptomatic or asymptomatic stenosis in-hospital mortality and long-term mortality (with a linkage to epidemiological data) and morbidity estimated by ischemic stroke.

The database allowed selection of a cohort of 2,071 patients in whom carotid stenting was performed in 161 centers (40 percent of stents were implanted in 14 centers) with a follow-up of 1 year. Carotid stents were mainly implanted in asymptomatic patients (81.6 percent). Morbi-mortality in symptomatic patients at 30 days (9.2 percent: 5.7 percent stroke and 3.4 percent mortality) was similar with results observed in a French comparative study EVA-3S (9.6 percent: 2.8 percent stroke and 8.8 percent mortality). These data allow the concerned HAS (French Health Authority) committee to renew the reimbursement proposal of these stents.

Medico-administrative database collecting robust criteria can be used to support reimbursement renewal of high risk implantable medical devices. The implementation of other criteria including the disease etiology and the complications imputability may allow to consider the use of these data for non-invasive MD.

Decision makers worldwide face the challenge of offering the best health care within a context of scarce resources. Technological developments have multiplied in the past decades, with the lifecycle of technologies becoming shorter. As a result, the traditional Health Technology Assessment (HTA) model is often caught in a too early, too late syndrome. In the province of Québec (Canada), there is no standardized process for assessing non-pharmaceutical technologies for reimbursement purposes, and technologies are therefore introduced via multiple sources. There are concerns that the introduction of some of the most promising technologies is delayed, and on the contrary, that others are introduced without providing a real added value to patients and the health system.

INESSS (Institut national d'excellence en santé et services sociaux), collectively with stakeholders of the Québec innovation field, has developed a dynamic process for assessing the added value of innovative technologies. POETIS (Processus optimisé d'évaluation des technologies innovantes en santé) aims to identify the technologies with the highest potential for positive impact on patients and the health system, in order to accelerate their implementation and promote their optimal use.

POETIS comprises four phases aligned with the lifecycle of technologies: research and development, pre-implementation, limited implementation, and diffusion. It allows a continuum of assessment, from the promise of a technology to its real-world benefit. It differs from other approaches because of the sustained involvement of key stakeholders, including patients, and because it assesses technologies iteratively, therefore fostering their adaptation to better suit patients needs. It is hoped for the first technologies to be assessed in 2017.

HTA has to adapt to the challenges of innovation, and this could be done with a lifecycle approach and an enhanced collaboration with end-users. Developed in Canada, the goals behind POETIS are common to many countries and the process could be adapted by other HTA agencies.

Health professionals often advocate and request innovative health technologies, perceiving Health Technology Assessment (HTA) as a delay or counterargument to their requests. To facilitate engagement of professionals and decision makers in the HTA process and endorsement of process outputs, a system for technology requests submission, based on the European Network for HTA (EUnetHTA) Submission Template, was established and subsequently piloted in a cancer research institute.

The “EUnetHTA medical devices evidence submission template” for companies (1) was adapted for use by professionals proposing a health technology for acquisition. Adaptation consisted mainly in: re-arrangement of chapters order with emphasis on the health problem, unmet needs, claimed additional benefits of the technology and potential for research; inclusion of information on costs/financial resources; and inclusion of a summary with a pre-defined set of brief statements to inform appraisal. The headings for the nine one-paragraph statements were: relevance of the health problem; degree of innovativeness of the technology; potential clinical impact; potential research relevance; comparative safety and effectiveness; economic impact; organizational impact; availability/quality of scientific literature; and degree of diffusion. Decision makers discussed the appraisal's statements with the proponents before reaching a conclusion.

From January 2016 technology requests were examined only if presented through the submission template. Results from submissions of three innovative technologies for prostate cancer treatment, endovascular procedures and cataract surgery will be discussed. Acceptability of the submission template was high and professionals — supported by experts available in their institution (clinical engineers, epidemiologists and others) — were successful in completing the dossier. Decision-makers appraisal proved facilitated and transparent. Concerted decisions were taken within a few weeks from submission.

The EUnetHTA tool proved flexible and valuable to initiate an HTA-based decision-making process. Appraisal was cooperative and proponents were involved in the decisions, through a process requiring a mean total time of 6 months. Participants’ misgivings were overcome by transparency and objectivity of the process.

Health Technology Assessment (HTA) is a multidisciplinary activity that systematically examines different dimensions related to the direct and indirect consequences of health technologies when implemented in healthcare systems. HTA is developed by multidisciplinary teams in order to cover all the mentioned dimensions. However, the quality of the processes produced by HTA teams will depend upon the competencies that those teams will acquire and incorporate including knowledge, skills and attitudes (1). The aim of this research was to determine how well these dimensions and competencies are covered in HTA academic Masters degree courses and manuals.

We analyzed what had been done in terms of competencies definition in HTA: how it has been reflected; theoretically and according to the authors, and how competencies can be structured; know-how and values. We explored HTA manuals and HTA academic Masters degree courses. We searched in Google with specific terms: building capacities, HTA, programs, Masters, diplomas. We used the HTAi vortal and the information related to courses (for example Masters degrees) and HTA agencies and network webpages for programs. The inclusion criteria were formal programs that describe HTA capacity building and not partial teaching of certain aspects of HTA and we excluded non-recognized institutions, or where there was no description of the programs or lack of detail regarding objectives and competencies to be achieved.

We found 105 courses or programs and analyzed 8 reports and 3 manuals. The main challenges that we faced were: that information was difficult to retrieve, not similarly structured, difficulties to find key information in webpages, no program description at all in some cases and the need to contact institutions staff or register as a student to receive the information and finally, it was difficult to obtain contact details of key people. We structured the information on competencies in knowledge, skills and attitudes.

The analyzed Masters degree courses and manuals did not cover all of the dimensions of HTA analysis in an equal and standardized way. The ethical, legal, social and organizational aspects were lacking in some of the programs, while, on the contrary, clinical and economic aspects were substantially included. On the basis of the information retrieved it would be good to define core competencies for HTA.

Biological disease-modifying anti-rheumatic drugs (bDMARDs) have become firmly established in the management of patients with rheumatoid arthritis (RA), but some patients do not improve despite therapy. This study evaluated the predictors of effectiveness of the bDMARDs on a cohort of patients with rheumatoid arthritis (RA) in the Brazilian Public Health System.

RA individuals treated with bDMARDs, were included in the open prospective cohort study. The Clinical Disease Activity Index (CDAI) was used to assess the effectiveness comparing results at baseline and after 6 months of follow-up. The association between socio-demographic and clinical characteristics with the disease activity measured by the CDAI was also investigated. The bDMARDs was considered effective when the patient achieved remission or low disease activity and considered not effective when there was still moderate or high disease activity. Pearson's chi-square was applied for the univariate analysis to evaluate the association of effectiveness measured by the CDAI with the socio-demographic (gender, education, marital status and race) and clinical variables (type of drug, EuroQol (EQ)-5D and Health Assessment Questionnaire (HAQ)). Logistic regression was applied in the multivariate analysis of the variables that presented a p< .20 value during the univariate analysis.

All 266 RA patients completed six months of follow-up. The most widely used bDMARDs was adalimumab (57.1 percent), with etanercept used by 22.2 percent, golimumab by 7.5 percent, abatacept by 4.5 percent, tocilizumab by 3.4 percent, infliximab by 2.6 percent, certolizumab by 1.5 percent, and rituximab by 1.1 percent. The bDMARDs reduced disease activity as measured by CDAI at six months of follow-up (p<.001). The percentage of patients achieving remission or low disease activity was 40.6 percent. bDMARDs were more effective in patients with better functionality (Odds Ratio, OR = 2.140 / 95 percent Confidence Interval, CI 1.219 - 3.756) at beginning of treatment and in patients who not had a previous bDMARDs (OR = 2.150 / 95 percent CI 1.144 - 4.042).

In this real-world study, functionality and use of previous bDMARDs are predictors in patients with RA treated with bDMARDs.

Uveitis is inflammation inside the eye whose underlying cause may be infectious or non-infectious. The objective of our study was to assess the cost-effectiveness of the dexamethasone implant and adalimumab compared with current practice (immunosuppressants and systemic corticosteroids) in patients with non-infectious intermediate, posterior or pan-uveitis.

A Markov model was built to estimate costs and benefits of the interventions. Systematic reviews were performed to identify the relevant evidence. Quality of life data collected in three key randomized-controlled trials (1-3) was used to estimate the interventions effectiveness compared with the trials comparator arms, which consisted of placebo plus limited current practice (LCP). An indirect treatment comparison between adalimumab and dexamethasone was considered inappropriate due to lack of necessary evidence. For adalimumab, patients with active and inactive uveitis were considered separately. Due to the short duration of the trials, the rate of blindness, an important complication of uveitis, was highly uncertain. Substantial exploratory analyses were therefore undertaken. The analysis was performed from the perspective of the National Health Service (NHS) and Personal Social Services (PSS). Costs were calculated based on standard United Kingdom sources.

The estimated incremental cost-effectiveness (ICER) of dexamethasone compared with LCP was GBP19,509 per quality-adjusted life year (QALY) gained. The estimated ICER of adalimumab compared with LCP was GBP94,523 and GBP317,547 per QALY in patients with active and inactive uveitis respectively. The factors with the largest impact upon the ICERs were the rate of blindness and the proportion of cases of blindness avoided by interventions.

Dexamethasone and adalimumab resulted in health gains, but at significant extra costs, especially adalimumab which is unlikely to be considered a cost-effective use of NHS resources. The results of the analysis are highly uncertain due to the limited availability of evidence on: the comparative effectiveness of dexamethasone, adalimumab and current practice; the effectiveness of treatments in avoiding blindness; and, the effectiveness of interventions in different subgroups.

In France, medical devices (MDs) for home-based infusion used to be covered by the health insurance system if included on the list of products and services qualifying for reimbursement under a generic description corresponding to a class of products with the same indications. This coverage modality offered no resistance to unnecessary or wasteful spending. Besides, between 2010 and 2015 the expenditure related to these MDs have increased from EUR192million to EUR289million (+50 percent).

The French National Authority for Health (HAS) has assessed the actual benefit of these MDs which have the same indications as the drugs infused at home. This work led to standardize the infusion types (gravity, elastomeric pump or active system requiring an energy source) and the quantities of MDs needed to carry out the different cares (installation, connection, withdrawal) according to the infusion route. At this step, considering that the priority was to redefine the MDs required at home for each care type, no economical assessment had been conducted.

Based on this medical assessment, the Ministry of Health has distinguished three types of infusion and three types of services (home installation, monitoring and consumables) since 2016. In total, twenty-four packages have been set up for reimbursement with non-cumulative rules. Doctors are in charge to prescribe the appropriate packages; providers and nurses determine together the optimal devices needed for each patient according to his environment.

These HAS recommendations on practice standardization have been the keystone for cost negotiations. The new coverage modalities aim to motivate liberal nurses to choose the best fitted products and providers to deliver the right quantities to patients. The expected benefits are an adjusted evaluation of the necessary equipment and a control of health expenditure due to the fixed costs of each infusion package.