Hepatitis C virus (HCV) infection is one of the main causes of chronic liver disease worldwide. The goal of HCV therapy is to eradicate the infection, which results in eliminating detectable circulating HCV after cessation of treatment, to prevent complications.

A prospective analysis was undertaken in the primary referral center in Turin. Throughout the use of questionnaries submitted to healthcare professionals, clinical and economic data from three different care pathways of HCV treatment were collected and processed. Costs were measured up to 8, 12, and 24-weeks treatment and based on time-driven activity-based costing (ABC) of the two main HCV treatments, Sovaldi and Harvoni. For the ABC analysis, three types of care pathways were considered, based on patient's clinical history resources used: patients treated for 8, 12, and 24 weeks. Gastroenterologists, pharmacists, administrative personel, and storemen were involved in the project. The aim of the analysis was to evaluate the organizational impact of the three different strategies for the treatment of HCV infection with Harvoni or Sovaldi and to estimate the differential cost.

The data indicates that shortening treatment from 24 to 12 weeks and from 24 to 8 weeks leads to a saving of EUR192 and EUR766 for both treatment strategies. When drug costs are also taken into account, the reduction of treatment with shortening treatment from 12 to 8 weeks leads to a saving of EUR15,252.77, a reduction of EUR60,691.07 from 24 to 8 weeks for Harvoni treatment. The reduction of treatment with shortening from 24 to 12 weeks for Sovaldi leads to a saving of EUR37,668.30. The paths of 8 and 12 weeks are those associated with fewer resources in terms of professional's time, costs relating to laboratory tests, and cost of drugs.

The reduction of the amount of time spent by healthcare professionals in the 12 weeks and in the 8 weeks strategies allows a reallocation of the resources employed.

Schizophrenia is a severe mental disease that affects approximately 1 percent of the population with a relevant chronic impact on social and occupational functioning, and daily activities. The aim of this analysis was to evaluate the clinical and economic consequences of long-acting injectable (LAI) treatment in patients with psychotic disorders, with a special focus on schizophrenia, in Italian real world practice.

A retrospective, observational mirror-study was developed to analyze outcomes measure referred to patients with psychotic disorders. Five hospital centers were involved in this study that collected patient level data from clinical databases. Retrospective data for each patient were referred to 6 months before LAI drug administration and 6 months after. A paired-Samples t-test was performed in order to identify statistical differences between pre- and post-LAI administration.

A total number of 308 patients were enrolled in the study (65.6 percent male). Of these 221 were eligible for our analysis (119 with schizophrenia). In the six months after LAI administration period we estimate a 47.3 percent reduction of the antipsychotic drugs (43.8 percent for schizophrenic patients), 94.7 percent reduction of hospitalizations (94.0 percent for schizophrenic patients) and adherent patients increase to 198/221 patients (78/221 in pre-LAI administration period). All differences between pre- and post- LAI administration period were statistically significant with a p< .005. In Italy over 152 thousand schizophrenic treated patients were estimated. Assuming that 20–40 percent of patients are eligible to the Mo.Ma (Model of Management) approach, our model estimates a direct cost reduction during the first year of implementation of around EUR12 million. Additionally, EUR18 million of direct costs in the mid-term and EUR58 million of indirect costs could be saved in the mid-term estimating a total cost reduction, due to the Mo.Ma approach, of about EUR90 million.

This new therapeutic approach could change the cost structure of schizophrenia by decreasing costs with efficient economic resource allocation guaranteed from efficient diagnostic and therapeutic pathways.

The inflammatory marker C-reactive protein (CRP) can be measured by a high-sensitivity assay (hsCRP) specific to vascular inflammation. We aimed to identify published literature on prevalence of elevated hsCRP and associated clinical, economic, and humanistic burden in patients with a history of myocardial infarction (MI).

A comprehensive literature search was performed for publications in English between January 2000 and February 2016 in MEDLINE, EMBASE, and MEDLINE In-Process. Search terms were variations on ‘Post myocardial infarction’, ‘CRP’, ‘epidemiology’ and ‘burden’. Clinical and real-world studies reporting baseline CRP levels in patients with a history of MI were included in the analysis.

Ten studies (prevalence: two; burden: two; both: six) were included. Cut-off points in hsCRP assays varied from >2 mg/L to ≥5.9 mg/L. Prevalence of hsCRP levels >2, >2.3, ≥2.37 and ≥2.9 mg/L were reported in 36 percent, 49 percent, 50 percent and 33 percent of patients, respectively (one publication each). Two publications reported >3 mg/L levels in 27.6 percent and 53.7 percent of patients. Levels of ≥3.3, ≥3.8, ≥4.2 and ≥5.9 mg/L were found in 38.8 percent, 25 percent, 25 percent and 24.7 percent respectively (one publication each). Of six studies reported CV events, four studies found elevated hsCRP levels to be predictive of future risk. Elevated hsCRP levels independently predicted all-cause mortality in four studies and CV mortality in three studies. Three publications included data on comorbidities: Diabetes was associated with elevated hsCRP in two of three analyses; hypertension in one out of two. No consistent associations between elevated hsCRP levels and hyperlipidaemia (one study), stroke or angina pectoris (one study) were found. No study reported economic, resource use or quality-of-life burden.

Due to limited evidence on prevalence of elevated hsCRP and associated burden of illness in patients with a history of MI, further research is warranted. Variations in findings, cut-off points and methods between studies make generalisations difficult.

There is a general consensus on the need to involve patients and the public in Health Technology Assessment (HTA) but questions remain about the best strategies for involving them into HTA structures and activities. The aim of this study was to update a systematic review (published in 2011) on patient and public engagement in HTA.

We searched papers published between January 2009 (end of the initial search) and November 2016 in eight databases and HTA journals using specific search strategies. We identified other publications through citation tracking, Internet search engines, HTA agencies websites, and discussion with experts in the field. Studies in English or French were included if they met the following criteria: (i) qualitative, quantitative or mixed-methods study; (ii) describing patients or public involvement; and (iii) in the HTA field. We extracted information using a pre-established grid including: characteristics of studies, type of activities for involving patients or public, effects on decisions, and factors facilitating or limiting involvement.

We identified a total of 4,762 new publications from the main search strategy. Among them, twenty-eight articles (reporting on twenty-three studies) met the inclusion criteria, whereas seventeen articles were included in the previous systematic review. Research designs are qualitative (18/23), quantitative (3/23) or mixed (2/23). Two main strategies for involving patients and public are generally described. The first is when public representatives participate directly in decision-making processes (participation) and the second is when patient or public input is sought to inform decisions (consultation or indirect participation).

The number of studies on patient and public involvement in HTA has increased in recent years. Findings from this update are mainly consistent with those of the previous systematic review. However, studies are still needed to assess the effectiveness of different strategies for involving patients and the public in HTA.

Most callers to emergency ambulance services are transported to hospital emergency departments (EDs), but ambulance services receive no information on patient outcomes. Pre-Hospital and Emergency Department (PHED) Data is a two-year mixed-methods observational study of the process and potential benefits of linking ambulance and ED data to allow analysis of patient outcomes. We report on our first aim, to examine the potential opportunities and challenges of this data linkage initiative.

We approached six hospital trusts in an English metropolitan area. We used a structured learning log to collect data on the process, time input and reflections. We analyzed these data with descriptive statistics, and qualitatively for themes.

All six trusts agreed to participate. We used an algorithm based on date, time and patient demographics to link data. We achieved a dataset of 775,018 records covering 2012 – 2016, and a linkage rate of 81 percent.

Initial set up tasks within the ambulance service took 30 hours 20 minutes. We then identified five stages of tasks with each hospital trust: negotiating senior approval; exploring data availability; information governance agreement; data transfer; and linking. Mean time spent by the research team on these processes was 30 hours 30 minutes per trust (range: 17 hours 20 minutes to 43 hours 10 minutes), plus additional time from staff of hospital trusts. The most intensive phases were: negotiating senior approval (mean: 8 hours 5 minutes), and data linking (mean: 12 hours 40 minutes). The stage which took the longest was information governance (mean: 19 weeks).

Key themes included the positive attitudes of trusts to participating, the range of decision makers involved, and the need for sustained input from the research team.

We found the process of data linkage was feasible, but requires dedicated time from research and trust staff, over a prolonged period, to achieve set up. Linked data are now being analyzed.

Chagas disease, caused by the parasite Trypanosoma cruzi, affects more than seven million people worldwide and it is considered by the World Health Organization (WHO) a neglected tropical disease (1). About one third of Chagas patients develop gastrointestinal disorders, such as dysphagia and achalasia. Management of the disease focuses on symptom improvement and drugs that relax the lower esophageal sphincter pressure (LESP), such as isosorbide and nifedipine. However, the use of these therapies is doubtful because of their side effects and palliative approach (2). The objective of this systematic review is to assess the effectiveness of isosorbide and nifedipine on gastrointestinal manifestation of Chagas disease.

We searched MEDLINE, EMBASE and LILACS databases to retrieve potentially relevant articles from inception to December 2016. Inclusion criteria: clinical trials, cohorts or cross-sectional design; adults (>18 years old); assessment of effects of isosorbide or nifedipine on gastrointestinal symptoms in Chagas patients. Two reviewers independently screened titles and abstracts, selected eligible studies and extracted data from each study. PROSPERO registration number: CRD42017055143.

Eight studies were included (two case series, two clinical trials and four crossovers). Three studies evaluated the effect of isosorbide in LESP and three in esophageal emptying. All of them found that isosorbide rapidly reduces LESP and increases esophageal emptying rates, improving dysphagia. However, several patients reported collateral effects, such as gastroesophageal reflux, headaches and dizziness. One study evaluated the effect of nifedipine on LESP and one on esophageal emptying. Nifedipine decreased LESP, but there was no effect on esophageal emptying.

The available evidence shows isosorbide is effective in the management of gastrointestinal symptoms. Frequently health care of Chagas disease patients is delivered by primary care physicians. So, information on effectiveness of interventions can be aggregated to clinical guidelines, having an important value to inform general practitioners on the decision-making process regarding treatment of this group of patients, avoiding referencing to a specialized care.

Value added medicines (VAM) are medicines based on known molecules that address healthcare needs and deliver relevant improvements for patients, healthcare professionals and/or payers through drug repositioning, drug reformulation or drug combination (1-3). Recently, the European Commission, through the Safe and Timely Access to Medicines for Patients (STAMP) program, considered the issue of VAM development and regulatory process. Current Health Technology Assessment (HTA) tools may not fully capture the benefits of VAM, which could lead to obstacles for patient access to VAM in several European countries (1). The study objective was to identify how HTA frameworks should evolve to reflect VAM value.

HTA expert interviews were performed as a preparatory step to an advisory board meeting. The following topics were addressed: (i) Eligibility for HTA and early HTA dialogues; (ii) Attributes that should be considered in HTA; (iii) HTA methodology; and (iv) Involvement of stakeholders in HTA.

VAMs bring additional benefit to patients and society. Therefore, the possibility for VAM assessment on a voluntary basis and within the appropriate assessment patterns/tools should be, in principle, included into HTA frameworks, as well as into early HTA dialogues. HTA should be patient-centric, and attributes such as patient preference, adherence, and patient reported outcomes should be considered where relevant. Unmet patient needs and disease burden should be used in a transparent and reproducible deliberative process. All these attributes should be used as explicitly and meaningfully weighted appraisal modifiers. HTA methodology should be comprehensive and should integrate societal perspectives. Patient representatives should take part in the decision-making process.

Current HTA frameworks should evolve to enhance VAM value recognition and encourage industry investment in medicines with high potential value for society.

The infectious Diseases Institute (IDI) is a research institute at the College of Health Sciences, Makerere University. Over the years, the number of research studies has greatly increased with an average of fifty active studies per year. Because of the voluminous study activities, investigators were faced with inadvertences of ethical approval deadlines (1). In 2015, a centralized electronic Regulatory Affairs Information System (RAIS) was developed and piloted to track the regulatory process of the entire research projects. RAIS is a web-based system, developed using a Net framework and runs on any operating system using a web browser such as “Google Chrome” and “Mozilla Firefox”.

A signed approval letter from an accredited Research Ethics Committee, National Drug Authority and Uganda National Council of Science and Technology, the reviewed protocol, consent forms and data collection tools are uploaded electronically into the RAIS with study staff contact information, CVs and Good Clinical Practice (GCP) certificates. RAIS sends automatic “no reply” emails to the investigators and research administration notifying for the need of annual renewal 56, 28 and 14 days before the expiry date of the approvals. The investigator or designated person prepares the application package which is then forwarded to the Research Regulatory Officer for review and submission to the regulatory authority.

From January 2015 to November 2016, fourty-three ongoing studies were uploaded to the RAIS of which eleven were clinical trials, twenty-one observational studies, seven diagnostic and four implementation studies. Studies that obtained their annual approvals before the expiry date was 90.7 percent, compared to 29 percent that had reported early submission for annual renewal between January 2013 and December 2014. RAIS has enabled continuity of study activities with timely annual renewed approvals, supported the tracking of staff GCP certificates and populated timely notifications to investigators, resulting in submission of annual application packages on time.

RAIS has strengthened ethical regulatory compliance and provided an effective platform for tracking regulatory processes, thus enabled continuity of study activities with timely annual renewal approvals and greatly supported the tracking of staff GCP certificates.

The HTAi Vortal is a product of the HTAi Information Retrieval Group (IRG) which has collected resources in the field of Health Technology Assessment (HTA) since 2005.

In 2011, a new technical platform was set up and the legacy Vortal content was split in three sections: HTA producers and networks, Selected references and Career development (including trainings). The same year, a fourth section was created to host a new product of the IRG: SuRe.info.

In 2014, the Vortal added a new service to other Interest sub groups of HTAi: the hosting of “Custom bibliographies”. But while the Vortal was probably quite unique in 2005, other Websites have been developed since then to offer quite similar functionalities.

The present communication aims at evaluating how the Vortal compares with similar tools existing on the Web.

Vortal competitors have been identified using a quick empirical search of the Web.

Functionalities have been identified by testing the website or their archive; maintainers have been sometimes contacted to ask for complementary information. A grid listing all functionalities has been established and filled in with the collected information.

Several competitors have been identified. The Vortal presents functionalities similar to online tools, but detailing level is different. Also, the Vortal provides a better integration resulting in more efficiency. And, the Vortal is the only Web platform to offer a service of publication of custom bibliographies to the different HTAi Interest Sub Groups.

After 12 years of existence, the HTAi Vortal is still a recognized online resource about HTA. While some existing functionalities are to be found in other online tools, some remain unique to the Vortal. Further research is needed to evaluate the preferences of people with interest in HTA.

Solid organ and hematopoietic cell transplantation are some of the more expensive procedures universally paid by the public Brazilian Unified Healthcare System (SUS). Transplanted patients depend on maintenance immunosuppression to prevent death or graft loss. A bottom-up heuristic process proposed new immunosuppression drugs for incorporation into the SUS.

Systematic evidence synthesis and Brazilian transplantation registries base-cases, Kaplan-Myer survival and economic assessments were presented in specialized national congresses with open public Delphi sessions to build professional Clinical and Therapeutic Protocols (PCDT) by consensus. Five consensus transplantation PCDTs with a SUS perspective budget impact and sensitivity analysis were submitted to the Health Ministry SUS Technology Incorporation National Commission (CONITEC) plenary for a decision. PCDTs were publicized in CONITEC Internet and Diário Oficial da União, an, official periodic publication, as well as undergoing widespread dissemination through mailings for Public Consultation. Public contributions were added to PCDTs to support Health Ministry policy making.

The São Paulo State Health Secretariat coordinated the synthesis and economic assessments made by 115 experienced transplantation specialists and health technology evaluators over ten years. Heart, lung, liver, pancreas and hematopoietic cells transplantation PCDTs (with tacrolimus, sirolimus and everolimus alternative immunosuppression) can significantly prevent 27.8 percent, 28.1 percent, 7.2 percent, 11.1 percent and 4.3 percent graft loss or graft versus host disease and death, respectively, for refractory transplantees rescue during the first year post-transplantation, saving healthcare resources. Ten-year follow-up data demonstrated partial benefits were sustained. Analysis demonstrated +USD689,655.17, +USD501,567.40, -USD377,802.51, +USD221.289,42 and +USD50.734,08 budget impact, respectively, resulting in an overall USD1,085,443.55 for 2,146 transplantees. The 5 PCDTs were favorably voted by CONITEC plenary members, 155 public contributions were added by patients and stakeholders, and the Brazilian Health Ministry decided to adopt the SUS reimbursement listing.

Democratic participation gave PCDTs real-world basis adjustments, SUS innovation and improved compliance.

Payment by result agreements have been quite widely used in Italy to provide access for high costs oncologic drugs and minimize uncertainties of real life benefits (1). The aim of this analysis was to overview the Roche experience in terms of Payment by Result (Pbr) in oncology and investigate the relation between timing for the evaluation of treatment failures and observed Time to Off Treatment (TTOT) from Phase III clinical trials (2).

A retrospective analysis of the Roche payment by results schemes in place in Italy was conducted. For each drug included in the analysis it was collected: (i) the negotiated timing to assess the treatment failure for payment by result, (ii) the median time to off treatment curve observed in clinical trials for the experimental drug, (iii) the median time to off treatment observed in clinical trials for the control arm. The mean ratios between timing to assess the treatment failure for payment by result and the time to off treatment observed for the experimental drug or the median time to off treatment observed in the control arm were calculated to identify potential correlations. High level of correlation was expected if ratio was close to 1 (±.2).

Roche products or different indications of the same product were identified as candidates for the analysis from 2008 to 2016. The timing for the evaluation of treatment failures for Pbr varies between 2 and 9 months, depending on the type of tumor and line of therapy. The mean Time to Payment By Result (TTPbr) / Control arm Time To Off Treatment (cTTOT) ratio was 1.16 (±.37) while the mean Time to Payment By Result (TTPbr) / Experimental arm Time To Off Treatment (eTTOT) ratio was .71 (±.13). Data analysis according to different time periods shows that the mean TTPbr/cTTOT and TTPbr/eTTOT for drugs negotiated from 2008 to 2015 were respectively 1.07 and 1.39 whereas for drugs negotiated in 2016 were respectively and .63 and 1.

Good level of correlation between TTPbr and cTTOT was found. This finding is in line with the methodology used by Italian Medicines Agency so far, leveraging the cTTOT as the most appropriate proxy to assess any incremental effect of a new drug compared to the previous Standard of Care. The analysis over time of TTPbr shows that in the first years of payment by result negotiation TTPbr is more correlated to the cTTOT whereas in the last years is moving closer to the experimental one.

Chronic Pain (CP) is the uncontrolled pain that affects patients for a long time. CP can be caused by many conditions, sometimes still poorly understood, and its levels can vary from moderate to intense. The management of resistant CP requires a stepwise approach and spinal cord stimulation (SCS) could be considered an extreme strategy. With the aim of ensuring the economic sustainability, the Veneto Region usually establishes rigorous access criteria to high-cost medical devices through its Regional Technical Committee on Medical Devices (CTRDM) and a Health Technology Assessment (HTA) procedure.

The Regional Health Technology Assessment Unit (CRUF) conducted through Pubmed a literature review of randomized controlled trials, systematic reviews, meta-analysis on SCS published from March 2006 to February 2016. International and national clinical guidelines were included in the analysis as well. The regional multidisciplinary Working Group on CP, which involved local clinicians, pharmacists, clinical engineer and health economist, discussed the collected evidence by consensus. Final recommendations on the appropriate use were submitted to the CTRDM for final approval.

The regional guidance describes the type of pain that can be treated with spinal neurostimulators and the criteria which determine the success of the test procedure. A comparative analysis of spinal neurostimulators available on the market and related patients eligibility criteria have been also included. Moreover, the guidelines stated a list of compulsory requirements in order to become a regional center authorized in performing spinal neurostimulation procedure. Finally, the document describes some indicators for appropriateness monitoring. The CTRDM approved the final version in October 2016.

The regional guidance on SCS aims at ensuring the appropriate use of neurostimulators in patients affected by resistant CP. The strict monitoring of agreed indicators is essential for appropriateness and consequently the sustainability of medical devices expenditure throughout the Regional Health Service.

To outline the methodological issues associated with the assessment of newborn screening for severe combined immunodeficiency, which was conducted to address the policy question of whether this test should be added to an existing newborn screening panel.

We conducted a systematic review of published primary studies and critically appraised the methodological quality of selected studies (1).

Fifteen studies were included; six focused on screening test performance, and seven on treatment effectiveness, and two on the effectiveness of a newborn screening program. The methodological issues identified included: (i) Overall poor methodological quality ratings of included studies using the QUADAS-2 (Quality Assessment of Diagnostic Accuracy Studies-2). This tool was originally developed for assessing diagnostic accuracy studies where subjects usually receive both index test and reference standard so a 2×2 table can be constructed; however it is almost impossible to apply this cross-sectional approach to studies of a screening test for a rare disease like severe combined immunodeficiency. (ii) Case control design using healthy controls could inflate estimates of test accuracy compared to studies using a cohort of consecutive patients, possibly due to spectrum effects and limited-challenge bias. This type of study is useful in the early phase of test development, but estimates of test accuracy based on this type of study should be interpreted with caution. (iii) Some screening programs reported no false negatives, indicating a sensitivity of 100 percent. However, lack of a systematic search for “missed cases” created uncertainty in arriving at a true value for the sensitivity. (iv) Variations in inclusion of pre-term infants, races/ethnicities, and screening protocols made it difficult to compare screening test performance across different studies.

Although severe combined immunodeficiency screening was the first addition to the US Recommended Uniform Screening Panel following an evidence-based review process, caution needs to be exercised when interpreting research findings due to important methodological issues.

Nudging is the application of behavioural sciences aimed at influencing behaviour in a non-prescriptive way. It is a tool of public health decision makers to produce health gain. Just like decisions in the field of Health Technology Assessment (HTA), nudging decisions are inevitably value laden. The current European Network for HTA (EUnetHTA) approach to evaluate ethical aspects encompasses mainly utilitarian and principlistic approaches. The aim of this project is to incorporate the virtue ethics approach in public health decision-making processes based on the example of nudging.

The narrative analysis of nudging is based on a systematic literature search conducted from 28 October to 13 November 2015 in the following databases: Medline via Ovid, Embase, and TRIP Database. A total of sixty-two articles were listed as relevant as a result of searches and, in addition, twenty-five more articles were found through hand searching.

Regardless of the potential issues related to nudging (manipulation or coercion), nudging is considered cost-effective and inevitable because of the malleability of human psychology for example, alcoholic drinks served in smaller glasses nudge people to drink less alcohol.

No policy intervention, nudging or HTA, is value neutral and hence it requires an ethical evaluation. It takes traits of character, virtues, to discern which principle to apply in what circumstances and phronesis, practical wisdom, is the key virtue of a decision maker. Phronesis is not a moral judgement deduced from principles, but it is context specific, bottom-up, action orientated, and framed through dialogues. It focuses on the agent, the decision maker, who, via the use public scrutiny, should be held accountable for phronetic decisions made.

Nudging is a cost-effective tool that can improve the populations health in a non-prescriptive way. Transparent reporting open to public scrutiny is necessary for the sake of evaluating whether the decisions made were phronetic for it takes traits of character, virtues, to decide between competing moral principles.

Hyperhidrosis is characterized by uncontrollable excessive sweating, which occurs at rest, regardless of temperature. Symptoms can significantly affect quality of life. There is substantial variation in the secondary care treatment of hyperhidrosis and uncertainty regarding optimal patient management. The objective of the Health Technology Assessment (HTA) was to review the evidence and establish the expected value of undertaking additional research into effective interventions for the management of primary hyperhidrosis in secondary care. Capturing the perspectives of patients and clinicians treating hyperhidrosis was an important part of the research.

The assessment included a systematic review and economic model, including value of information analysis. Patients, dermatologists, a vascular surgeon and a specialist nurse (who set up the UK Hyperhidrosis Support Group) provided advice at various stages, including at an end-of-project workshop, to help interpret results and prioritize research recommendations.

Patient and clinician advisors were unsurprised by the finding that there is evidence of a large effect of botulinum toxin injections on axillary hyperhidrosis symptoms in the short to medium term; there was consensus amongst patients and clinicians that botulinum toxin injections were very effective. The advisors agreed that a trial of botulinum toxin injections (plus anesthetic) versus iontophoresis for palmar hyperhidrosis would be useful. Patients and clinicians were happy with the sequence of treatments identified as being cost effective for axillary hyperhidrosis: iontophoresis, botulinum toxin injections, anticholinergic medication, curettage, endoscopic thoracic sympathectomy. All patients agreed that the Hyperhidrosis Quality of Life index (HidroQoL©) tool was superior to other commonly used tools for assessing quality of life in hyperhidrosis.

Patients and clinicians considered the key findings of the systematic review and economic analyses to be appropriate. Advisors advocated a trial of botulinum toxin injections (plus anaesthetic) versus iontophoresis for palmar hyperhidrosis. Patients preferred the HydroQoL® tool over other commonly used quality of life tools in hyperhidrosis research.

An appropriate governance of the installed equipment base, by defining replacements strategies and programming introductions of innovative Biomedical Technologies (BT), has direct effects on the efficiency and effectiveness of health systems. An effective health technology management is of paramount importance for providing safe, high quality and innovative care with the constraint of health-care budgets, safeguarding equity, access and choice principles. Data from the regional BT information flow show that, compared to the gold standard (1), the North Region of Italy has about 15 percent less of large medical equipment younger than 5 years and about 15 percent more of equipment older than 10 years.

In order to draw a unified path of BT procurement processes, in 2013 and 2014 regional regulations (2,3) were enacted. Each Public Hospital and Local Health Authorities (ASRs) defined a plan (PLTB) containing, regardless of the form of procurement and type of funding, all BT requests for a value greater than EUR40,000 distinguished in replacement/new acquisition/upgrade, innovative acquisition and donations. Requests of BT have to obtain the authorization by the Regional Healthcare Authority Commission (GTB), in compliance with defined criteria, including financial and sustainability aspects, after the evaluation of the Regional Clinical Engineering Commission (GIC) supported by IRES, Health Technology Assessment and Management research group.

Over the years 2014 and 2015, the ASRs submitted 491 BT requests, of which 87 percent were replacement/new acquisition/upgrade, 9 percent innovative acquisition and 4 percent donations. Altogether 26 percent of these instances were urgent and 2 percent were unique BT on the market. Sixteen percent of requests for replacement/new acquisition/upgrade of BT related to large medical equipment with mean age of 13.3 years, 2 percent regarded innovative BT with average age of 8.4 years and 48 percent widespread technologies with mean age of 15.6 years.

The limitations in investments deriving from being a Region in “Recovery Plan”, have originated an absence of BT programming, as shown in PLTB by the prevalence of requests for the replacement management of obsolete equipment with inadequate performance, high machine downtimes and elevated maintenance costs.

In a budget constrained environment characterized by an increasing number of high-cost medicines, manufacturers need to demonstrate that their drugs can provide value-for-money. In this complex environment Managed Entry Agreements (MEAs) have been developed with the aim of sharing the risk between the National Health Service (NHS) and manufacturers (1). The objective of this analysis was to identify a correlation between Anatomical Therapeutic Chemical Classification (ATC) and different type of agreements assigned taking into consideration the distribution of Italian Medicines Agency registries by ATC and by kind of agreement negotiated (financial or performance based) (2).

This analysis takes into account all drugs under monitoring AIFA registries in place in Italy from 2006. For each registry included in the analysis it was collected the status of the registry (active, closed or incoming), the disease area that the registry covers and the monitored drugs with or without an associated Managed Entry Agreements. Considering the high weight of oncology drugs, a sub-analysis was done to investigate registries distribution for each specific form of cancer.

The majority of drugs monitored are under a registry with no associated risk sharing agreement according to AIFA (60 percent). For what concerned monitored drugs with an associated agreement, performance-based agreement is the most diffused type of MEA. In terms of therapeutic area involved in the monitoring registries activity, oncology was the most common area. Financial based agreements characterize principally medicines used for Leukemia and Hepatitis C, whereas drugs administrated for Melanoma, Breast and Ovarian Cancer and Ophthalmology diseases follow performance based agreements.

MEAs represent a way to guarantee a sustainable access for innovative medicines. It is proven that oncology products are most likely to have a MEA since they represent some of the most expensive drugs launched in recent years. From this study appear a correlation between the therapeutic disease area of the monitored drugs and MEA assigned by AIFA which is influenced also by other factors like budget impact, risk-benefit ratio and the presence of appropriate endpoints to evaluate the treatment response.

One of the barriers of integrating ethics in Health Technology Assessment (HTA) relates to the social role of HTA (1). The aim of this study is to provide a better understanding of the way by which law circumscribes the social role of HTA. Our hypothesis: HTA's social role is embedded within a mixed governance based on hard law and soft law.

Three HTA agencies were conveniently selected for our study: Haute Autorité de santé (HAS) (France), National Institute for Health and Care Excellence (NICE) (United Kingdom) and Institut national d'excellence en santé et en services sociaux (INESSS) (Québec, Canada). Our analysis of the legal, administrative and procedural documents relating to the existence and assessment processes of these three agencies is guided by the following criteria:

1. 1. The normative strength of the documents (categories of hard law or soft law) (2)

2. 2. The definition of the agencies’ social role (1)

3. 3. The integration of ethics in the agencies’ mandate.

Hard law contributes to establish a general mandate and some legal legitimacy for these agencies. Soft law, grounded in the HTA producers' practices, plays a major role in the legal governance of HTA. Our results demonstrate that these agencies existing practices seem to circumscribe their social role further than their constitutive laws. In this context, social actors become responsible to define, structure and operationalize the implementation of HTA.

In addition, the legal framework (hard law) through which HTA unfolds does not clearly support its structural and social role. Despite existing legal frameworks, the normative legitimacy of HTA is not entirely established, as it depends on soft law. Taken altogether, this maintains a persisting conceptual vagueness in HTA governance.

The social role of HTA should be defined either through modifying existing legislations (hard law) or through harmonization of the agencies internal policies and regulations (soft law). Such legal initiatives would help clarify the aims of HTA evaluations: assessments (scientific) or appraisal (value-laden), and therefore give a clearer indication on how best to integrate ethics in HTA.

Our Hospital-based Health Technology Assessment unit (HB-HTA) was founded in 2011 following the nomination of Louis-H. Lafontaine hospital as the Montreal University Mental Health Institute (IUSMM). From the beginning, the HB-HTA has been supporting and advising the Chief Executive Officer of IUSMM in the decision-making process concerning the implementation of new technologies and practices in mental health. Since 2015, the HB-HTA is part of the East of Montreal Regional Integrated Health and Social Services Centre (CIUSSS de l'Est-de-l’Île de Montréal), continuing to support decisions in mental health. Currently, the HB-HTA unit is nested in the Quality, Performance and Ethics department.

Formed by a coordinator, a scientific advisor and a manager, the HB-HTA team plans, organizes and sets up the evaluation activities. The unit benefits from the support of a Steering Committee which consists of representatives of clinical, administrative and research directions, as well as of health users and families. This committee determine the strategic orientation of the HB-HTA unit, prioritize the projects, approves the evaluation products and gives indications on the knowledge transfer process.

To answer the decision questions, our HB-HTA unit employs two types of products: evaluation reports and informative notes. Based on an exhaustive literature search and consultations with stakeholders, the evaluation reports offer recommendations to support the decision-making process. The informative notes are rapid responses based on a partial literature search. The nature of this type of analysis does not allow the formulation of recommendations, however, a conclusion of the consulted literature is offered.

Based on the work of our HB-HTA unit, some important decisions were made by the IUSMM. As an example, the systematic screening of psychiatric patients for drug and alcohol was not favored by our institution; rather than this, priority was given to staff training, in order to better identify and treat psychiatric patients with substance abuse comorbidity.

Anxiety and depression are major drivers of health-related quality of life, adherence to therapy, and motivation to self-manage chronic conditions. A number of studies have shown rates of anxiety and depression to be higher among individuals with hemophilia compared to that of the general population (1). As the primary point of care for persons with hemophilia, hematologists are well-placed to assess the mental health state of their patients (2). The aim of this study is to explore physician- and patient-reported rates of anxiety and depression among a cohort with severe hemophilia.

Data were drawn from the Cost of Haemophilia across Europe – a Socioeconomic Survey (CHESS), a cost-of-illness study in severe hemophilia A and B across five European countries (France, Germany, Italy, Spain, and the UK) (3). Physicians provided clinical and sociodemographic information for 1,285 adult patients, 551 of whom completed corresponding questionnaires, including EuroQol EQ-5D-3L. We compared the self-reported EQ-5D with physician reports of anxiety and depression.

Across the five countries, physicians recorded a diagnosis of anxiety disorder in 189 patients (15 percent; range 4 percent-28 percent) and depression in 178 patients (14 percent; range 10 percent-28 percent). Seventy-three patients (6 percent) recorded comorbid anxiety and depression. Forty-six percent of patients with anxiety and 58 percent of patients with depression were receiving some form of treatment for their condition.

Within the EQ-5D measure, 42 percent of individuals recorded problems with anxiety or depression, with 6 percent of patients reporting “extreme” anxiety or depression. Twenty-two percent of individuals with a self-reported problem were not recorded with a corresponding diagnosis by their hematologist; 39 percent of patients reporting “extreme” anxiety or depression were absent of any physician-reported diagnosis.

Anxiety and depression are notable conditions within the hemophilia community, particularly so among those with severe condition. The mental health of individuals with hemophilia is an important aspect in ensuring therapy adherence and overall wellbeing and should be considered as part of a multidisciplinary approach to management of the condition.