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.

Since the creation of the National Committee for Health Technology Incorporation in the Brazilian Health System (CONITEC), a new phase started in the public Brazilian Health System (SUS): a continuous updating of the system based on Health Technology Assessment (HTA). CONITEC was created by federal law in 2012 and is responsible for advising the Ministry of Health regarding the incorporation or disinvestment of health technologies. The whole process involves a strong interaction with society, including the composition of the committee, which has the participation of the National Health Council. The objective of this study was to describe the results of CONITEC in five years of operation.

This is a retrospective descriptive study, based on information from the database (period 2012–2016) and CONITEC's website.

Since 2012, CONITEC assessed 541 technologies, including drugs (360), health products (71) and procedures (110); 303 assessment requests came from SUS agencies and institutions and the other 238 requests from pharmaceutical companies, medical societies, patient associations and the judiciary bodies. In this period, there were 190 public consultations, during which more than 24,000 feedback from society were received. The average time for evaluation was 146 days. The committee recommended the incorporation of 186 technologies into SUS, the disinvestment of 43 and was unfavorable to the incorporation of 88, generating a budgetary impact of approximately BRL2.5 billion (USD764 million).

From 2012–2016, CONITEC tripled the average annual incorporation of new technologies compared to the period 2006–2011. In this process, it was necessary to assess efficacy, safety and cost-effectiveness of technologies, generating positive results for the expansion of access, health gains for patients and sustainability for the system. It should be considered that the use of evidence for decision making strengthens transparency in public management and the development of active processes of information, communication and social participation.

In Turkey, there is a scarcity of knowledge about the predictors of health outcomes at a national level, and it is well known that there is a gap between rural and urban parts of developing countries in terms of the level of health outcomes. This study aims to find out predictor factors of the public health outcomes at a province level in Turkey.

Life expectancy at birth and mortality are used as public health outcome indicators. Logistic regression and Random Forest classification generated by using 50, 100, and 150 trees were used to compare prediction performance of health outcomes. The results of different prediction methods were recorded changing the “k” parameter from 3 to 20 in k-fold cross validation. The Area Under the ROC Curve (AUC) was used as a measure of prediction accuracy. Prediction performance differences were tested using Kruskall-Wallis analysis and visualized on a heatmap. Finally, predictor variables of public health outcomes were shown on a decision tree.

Study results revealed that Logistic regression outperformed Random Forest classification. The difference between all prediction methods to predict public health outcome indicators was statistically significant (p<.000). The heatmap shows that AUC values to predict mortality have superior performance when compared with life expectancy at birth. Decision tree graphs present that the most important predictor variables were total number of beds for mortality and percentage of higher education graduates for life expectancy at birth.

The results of this study represent a preliminary attempt to determine public health outcome indicators. It is hoped that the results of this study serve as a basis to understand the determinants of health care outcomes at province level with focus on a developing country. This study illustrates that there is a need to spend extra effort for future studies to analyze public health outcomes to improve social welfare functions in health systems.

The early benefit assessment of drugs in Germany and their preceded market authorization pursue different objectives, resulting in divergent decision-making strategies. This is reflected inter alia by the diverse inclusion of confirmatory endpoints within the assessments of oncological drugs. The pharmaceutical manufacturers are facing the challenge of meeting the requirements for both evaluation processes by the available evidence and avoiding hereby negative early benefit assessments. This is mainly due to the concept of mutually relevant clinical trials.

Identification and gathering of the endpoints is based on a specifically developed guide. The extracted data from the documents of completed assessments up to July 2015 are used to estimate both separately and together the impact of explorative in relation to confirmatory endpoints on the drug approval and early benefit assessment, by contrasting the European Medicines Agency's risk-benefit-ratio and the benefit-harm-balancing of the national Health Technology Assessment (HTA) jurisdiction.

Twenty-one of fourty-one studies’ oncological assessments could be included in the endpoint analysis. From a procedural point of view both the drug approval and the early benefit assessment seem to be not confirmatory since they include explorative endpoints as well. Yet, drug approval is in terms of quality of endpoints more confirmatory than early benefit assessment since it contains a higher proportion of primary endpoints. The latter implies only in 67 percent of the assessments a primary endpoint to be relevant for the benefit-harm-balancing. Moreover, explorative mortality endpoints reached the highest agreement and explorative endpoints capturing health-related quality of life no agreement, referring to the mutual relevance of endpoints for the risk-benefit-ratio and the benefit-harm-balancing.

The missing information transparency of the assessment reports compared to the information offered within the early benefit assessment makes an assignment of endpoints with respect to the mutually relevant clinical trial sometimes troublesome. To warrant, in the long run, a broader confirmatory basis for decisions in health care supported by HTA, a closer inter-institutional cooperation of approval authorities and German HTA jurisdictions seems favorable.

In the last decade an increasing number of high-priced, new cancer treatments received marketing authorization in Europe. What is actually known about the clinical benefit of those therapies at the time of approval needs to be elucidated in order to inform decisions about the use and reimbursement of these novel treatment options. Thus, the aim of the current analysis was to systematically investigate oncological therapies approved between January 2009 and April 2016. We extracted, as well as quantified the level of knowledge of the clinical benefit at the time of marketing authorization.

To assess the benefit of new interventions as well as expanded indications, we extracted the median gain of the two study endpoints: progression-free survival (PFS) and overall survival (OS). Information is based on approval documents provided by the European Medicines Agency (EMA) and assessments from the Austrian Horizon Scanning programme (HSO). We included all cancer therapies approved in Europe between 1 January 2009 and 15 April 2016.

Cancer drugs for 134 new indications approved since 2009 were identified. In the case of thirty-seven indications (27 percent), no data was available for PFS or for OS. A positive difference in median overall survival was reached by seventy-six licensed indications (55.5 percent); twenty-two (16 percent) of them showed a difference of more than three months. Regarding the study endpoint progression-free survival, an improvement was shown in ninety indications (65.2 percent).

Scarce knowledge regarding the clinical benefit of anti-cancer therapies is available at the time of approval. In addition, the survival benefit of the approved indications is less than three months in the majority of approved therapies.

Under the Orphan Regulation, the European Medicines Agency (EMA) intended to incentivize the research and development of new treatments for rare and life-threatening conditions. Marketing authorisation of orphan medicinal products (OMPs) by the EMA is only the first step, as medicines are made available to patients when reimbursement or Health Technology Assessment (HTA) decisions are implemented by national health systems. We analyzed the availability and access to OMPs in the United Kingdom (UK), France, Germany, Italy and Spain. We compared the availability, which is the possibility to prescribe a given OMP, to the access, which refers to the full or partial reimbursement by the public health service.

We collected data on launches, HTA decisions, any centralized commissioning and/or reimbursement decision for all the OMPs authorised since 2000 in the UK countries (England, Scotland and Wales), France, Germany, Italy and Spain.

Since the Orphan Regulation inception, the EMA granted marketing authorization to 143 OMPs. These OMPs are most widely accessible in Germany and France. Reimbursement in Germany is immediate after authorization. France and Italy present a delay of 19 months from authorization to reimbursement, which is shorter than in other countries. In England, less than 50 percent of centrally authorised OMPs are routinely funded by the National Health Service (NHS), including one-third of these recommended by the National Institute for Health and Care Excellence (NICE), and those reimbursed via commissioning policies and the Cancer Drugs Fund.

The assessment of degree of access to OMPs across Europe is limited by differences in the national HTA and reimbursement systems and the heterogeneous information made publicly available on their decisions. Nonetheless, our study suggests that the primary purpose to grant equal availability to OMPs to the patients in the Eropean Union via the implementation of the orphan regulation was partially achieved with important variations of access observed across the countries included in our study.

Multiple-sclerosis (MS) is a highly disabling chronic disorder affecting young adults with long term economic consequences on society that escalate as MS disability increases (1,2). In the long-term, progression of MS results in increased level of disability and most patients will eventually experience some degree of functional impairment of the nervous system that impacts on mobility as well as sensory and coordination issues, bladder and sexual functioning, and mood and cognitionon (2). This is usually accompanied by a deterioration of their quality of life. Patient relevant outcome measures (PROMS) are largely used to measure individual disability, and quality of life in MS (2). International evidence from the International Multiple Sclerosis Study (IMPrESS) (2) was used to quantify the relationship between healthcare resources utilisation and disability, quality of life in individuals with MS.

Multivariable logistic regression was performed in order to identify patient-related variables reporting disability (Barthel) and utility (EQ-5D) that predict use of healthcare services (visits to GP, specialists, nurses, hospitalisation and treatment) and work limitation within the participants of the IMPrESS.

Reponses were collected from 1,152 individuals across 21 countries of which 74.3 percent (856) were useful for analysis. Preliminary findings indicated that for the pooled data sets both EQ-5D and Barthel scores were predictors of healthcare resource use, across different categories (p<.05), except for nurse visits (Barthel only; p<.09). Overall the association between PROM data and use of healthcare resources appeared to be stronger with EQ-5D compared to Barthel. EQ-5D appeared to also predict the impact of MS on loss of productivity (in terms of work limitation; p<.05).

PROMs can be used to predict the economic consequences of MS on healthcare providers and society, but more research is needed to confirm the robustness of the evidence and its validity across individual healthcare system settings.

Ultrasound (US) detects synovitis more accurately than clinical examination (CE) in people with rheumatoid arthritis (RA). This review aimed to investigate the use of US, compared to CE alone, in treatment strategies for RA, and to estimate its potential to be cost-effective in making treatment decisions.

A systematic review was conducted of studies: investigating RA treatment response or strategies that compared US with CE-assessed synovitis; and of tapering RA treatment (1). A model was constructed to investigate the potential cost-effectiveness of US in (i) selecting patients suitable for treatment tapering; and (ii) avoiding treatment escalation (2).

Seven prospective cohort studies suggested US-detected synovitis was significantly associated with a treatment response or tapering failure, whereas in most cases clinical examination alone was not. Two randomized controlled trials (RCTs) identified suggested that US added to the Disease Activity Index (DAS)-based treatment strategies but did not significantly improve primary outcomes, but was associated with improved rate of DAS remission. The evidence showed that some patients (proportions varied widely) who had achieved low disease activity could have treatment tapered, with no, or little, short-term harm to the patient.

The model estimated that an average reduction of 2.5 percent in the costs of biological disease-modifying anti-rheumatic drug (bDMARDs) was sufficient to cover the costs of performing US every three months. This value increased to 4 percent and 13 percent for the costs of conventional disease-modifying anti-rheumatic drug (cDMARDs) depending on the assumed regimen.

Use of US to monitor synovitis could potentially be a cost-effective approach, given that low proportions of patients for whom clinicians consider amending treatment, would need to taper treatment, or remain on therapy without escalation. US could provide clinicians with more confidence in reducing the drug burden. However, there is considerable uncertainty in this conclusion due to lack of robust data relating to key parameters.

Sudden cardiac arrest (SCA) is the most common cause of death in patients with coronary artery disease. Mostly, ventricular tachycardia (VT) and ventricular fibrillation (VF) are the underlying aetiology of SCA, which is claimed to be successfully treated by a novel therapy, a wearable cardioverter defibrillator (WCD, LifeVest®).

The assessment, performed within the European Network for HTA (EUnetHTA), aimed to provide valid data on clinical effectiveness and safety of the WCD. Furthermore, the project intended to elicit patients views on aspects regarding their cardiac disease and the WCD therapy as well as to identify neglected outcomes.

A synthesis of evidence on the basis of a systematic literature search in Medline via Ovid, Embase, the Cochrane Library, and the Centre for Reviews and Dissemination (CRD) databases was performed. The search was complemented by citation tracking and handsearch.

A face-to-face semi-structured focus group interview was performed with five cardiac disease patients in the scoping phase.

Since no prospective controlled trials were found, no assessment of effectiveness could be performed. With regard to safety, five prospective studies were included, but the quality of the body of evidence was very low. Adverse events (AEs) reported were skin rash/itching (6 percent), false alarms (14 percent), palpitations/lightheadedness/fainting (9 percent) and discontinuation due to comfort/lifestyle issues (16-22 percent). Serious adverse events (SAEs) were inappropriate shocks (0-2 percent) and unsuccessful shocks (0-.7 percent). Frequency of SAEs leading to death was 0-.3 percent.

Patients of the focus group reported that experiencing a sense of security was crucial to them. The WCD therapy was not considered an option for weeks or months, due to expected restrictions in living a ‘normal’ and secure life.

No statement can be made about the device effectiveness – further research is needed. More data and more adequate reporting of AEs and SAEs are needed in order to establish the device safety. In particular, more data is needed for risk stratification of high risk patients in order to further narrow down the wide range of indications for the WCD use.

Between December 2013 and April 2016, an unprecedented epidemic of Ebola Virus Disease (EVD) took place. This epidemic urged countries all over the world to be prepared for the possibility of having an EVD patient (1). Besides morbidity and mortality of the disease, containment efforts also have economic consequences for society. In this study, costs of preparedness for and response to EVD made by the Dutch health system were estimated.

We used an activity-based costing method in which cost of personnel time targeted at preparedness, and response activities was based on a time recording system and interviews with key professionals of the organizations involved. In addition, patient days of hospitalizations, laboratory tests, personal protective equipment (PPE), as well as costs for additional cleaning and disinfection were acquired via the organizations. All costs are expressed at the 2015-euro price level.

The estimated total costs of EVD preparedness and response in the Netherlands were averaged at EUR14.1 million, ranging from EUR7.6 to EUR24.9 million. There were thirteen possible cases clinically evaluated and one confirmed case, admitted through an international evacuation request, corresponding to approximately EUR1 million per case (2). Preparedness activities of personnel, especially of all ambulance care services and hospitals that could possibly receive a case, and expenditures on PPE, were the main cost drivers.

The estimated total cost of EVD preparedness and response in the Netherlands was substantial. Costs made by healthcare organizations were higher than among public health organizations (3). Designating one ambulance care service and fewer hospitals for the assessment of possible patients with viral hemorrhagic fever or other highly infectious disease of high consequence might improve efficiency and reduce future costs. The experiences and collaboration of healthcare organizations that managed patients with possible EVD can serve as a valuable resource for future outbreaks of other highly infectious diseases.