Written patient evidence is submitted to the National Institute for Health and Care Excellence (NICE) by patient organizations and their nominated patient experts. We reviewed the impact that patient evidence had on the committee's decision making. This local learning can help generate global opportunities for Health Technology Assessment (HTA) bodies and patient groups to further develop their understanding and methodology about how patient evidence can support HTA decision making for ultra-orphan conditions.

There were two phases.

Phase one was an online questionnaire about the impact of patient evidence on the committee's decision making for ultra-orphan HTA evaluations. It was sent to the committee chair, lay committee members and selected other committee members.

Phase 2 developed the initial questionnaire findings using in-depth interviews with the committee chair and the lay members. These gained further understanding of the impact of patient evidence and the themes raised.

Key findings showed patient evidence was helpful to understand the:

• • Burden of disease

• • Patient population

• • Likely uptake of new medicines

• • Impact on carers.

For ultra-orphan conditions, where other forms of evidence are scarcer, patient evidence is fundamental to understanding patient needs, the impact of the disease, patient population and preferences.

Patient evidence was useful for the committee in different ways; it provided the committee with new evidence and it helped the committee understand and interpret the evidence submitted by others. Both are key to committee decision making. It was clear that due to the very small patient population, patient groups knew the patient population, their stage of disease, and their preferences in detail.

The findings will be used to inform an updated patient submission template for ultra-orphan HTAs, and supporting guide. These will be available on the NICE website and offered to the HTAi Interest Group on Patient and Citizen Involvement so they can be shared globally.

Value frameworks, analogous to Health Technology Assessment (HTA) internationally, have emerged in the United States to aid stakeholders in assessing the value of new treatments. Since patient perspectives on value may differ significantly from other stakeholders, formalized procedures to involve patients in their work have been created. Despite these efforts, concerns persist that patient involvement is insufficient or “rhetoric.” To assist in this effort, the National Health Council (NHC) created a rubric to aid decision makers in improving the patient centricity of their value assessments.

A convenience sample of twenty-eight organizations was invited to participate in a roundtable discussion. Participants discussed experiences with value frameworks; debated and thematically grouped hallmark patient-centeredness characteristics; and developed illustrative examples of the characteristics. These materials were organized into the rubric, and subsequently vetted via multi-stakeholder peer review.

Participants agreed upon six key domains of patient centeredness: partnership (patients are involved in every step of development/dissemination processes), transparency (assumptions/inputs are disclosed in an understandable, timely way), inclusiveness (perspectives drawn from broad range of stakeholders), diversity (differences in subpopulations, trajectory of disease, and stage of a life should be accounted for), outcomes (includes those that patients have identified as important), and data (variety of credible data sources are used allowing for timely incorporation of new information and account for the diversity of patient populations and patient-centered outcomes). The Rubric describes each domain and includes illustrative examples of how patient engagement/centeredness can be operationalized through direct and indirect pathways.

The NHC Rubric is a first step toward creating patient-centered value assessments that patients and their families can rely on. It is intended to assist all stakeholders, especially the patient community, in assessing the level of patient centeredness and engagement in a given framework or model. It can be a guide to support developers in conceptualizing plans for meaningfully engaging patients.

Beta-interferons are used as first-line therapy for relapsing-remitting multiple sclerosis in Brazil. In order to evaluate the possible inferiority of one of the beta-interferons available and support a guideline update, we conducted an eleven-year (January 2000 to December 2010) nationwide real-world performance assessment using the Unified Health System (SUS) databases.

We assessed whether patients using subcutaneous beta-interferon switched treatment, relapsed or died (composite event) earlier than patients using intramuscular beta-interferons. Patients without a dispensing registry longer than three months were censored. We used the Kaplan-Meier method to estimate the cumulative probability of persistence on initial treatment, and compared groups with the Log-rank test. The influence of the drug on the occurrence of event was assessed with Cox proportional hazards analysis.

The number of patients included was 12,154, and the majority started treatment with subcutaneous beta-interferon-1a (45.7 percent), followed by subcutaneous beta-interferon-1b (27.7 percent) and by intramuscular beta-interferon (26.6 percent). Women represented 73.1 percent and the mean age was 38.93±11.34 years old. The group of patients who used intramuscular beta-interferon switched treatment, relapsed or died earlier (median 47 months; 95 percent Confidence Interval, CI 44–52) than patients using the subcutaneous beta-interferons, (69 months (95 percent CI 64–76) for beta- interferon 1a and 73 (95 percent CI 66–84) months for beta-interferon 1b) (p< .0001 for both comparisons). Accordingly, the use of intramuscular beta-interferon was associated with a higher probability of event (Hazard ratio, HR 1.38; 95 percent CI 1.29-1.48), while the use of the other beta-interferons had a protective effect (1a: HR .86; 95 percent CI .81-.92; 1b: HR .89; 95 percent CI .83-.95).

The inferiority of intramuscular beta-interferon found in the real-world corroborates findings from head-to-head studies and systematic reviews conducted by Cochrane and the National Commission for Technology Incorporation in SUS (CONITEC/Brazil). This result led to disinvestment in intramuscular beta-interferon and was the first case of clinical guideline update using real-world evidence in Brazil.

With the promotion of a tiered medical service system, secondary hospitals will play a more important role in the future. This study aims to explore the cost-benefit of computed tomography (CT) in secondary hospitals in China, with a view to providing information for overall economic management in hospitals as well as for regional planning of medical equipment in different areas.

Fifty-eight secondary hospitals from six provinces located in the eastern, central, and western regions of China were selected as the study sample. Questionnaires were used to collect information on the cost structure, efficiency, and benefits of CT in the secondary hospitals in the past 5 years. Cost analysis was conducted from the perspective of the hospitals, which mainly referred to direct fixed costs and variable costs. We analyzed the investment recovery years a, cost recovery rate b, and benefit-cost ratio to evaluate the economic benefits of CT. We also analyzed the technological benefits of CT based on its effective utilization rate c and positive detection rate.

1. a: Investment recovery years = total original investment / (annual net income + annual depreciation expense)

2. b: Cost recovery rate = average income per check / average cost per check

3. c: Effective utilization rate = single equipment utilization rate * positive detection rate

4. (Single equipment utilization rate = actual working time / rated working time)

Depreciation costs (36.3 percent) were the largest proportion of all costs over the 5-year period, followed by material costs (22.2 percent), maintenance costs (18.2 percent), labor costs (17.1 percent), and electricity consumption (1.2 percent). The investment recovery periods of CT in the eastern, central, and western regions were 2.5, 2.8, and 3.1 years, respectively; the cost recovery rates were 186.5 percent, 172.0 percent, and 174.1 percent, respectively; the benefit-cost ratios were 1.9, 1.7, and 1.7, respectively; the effective utilization rates were 46.1 percent, 58.3 percent, and 71.2 percent, respectively; and the positive detection rates were 52.3 percent, 60.5 percent, and 73.3 percent, respectively.

The current study indicates that the cost-benefit of CT is good in secondary hospitals, especially in terms of economic benefits. But to achieve greater technological benefits in all three regions, more appropriate utilization of CT is needed.

The increase in travelers and refugees combined with global warming may soon lead to the development of tropical diseases such as Schistosoma or Strongyloides infections in some European countries.

Those intestinal parasites may persist for decades with subclinical infections or low-grade disease with nonspecific manifestations. In the presence of immunosuppression, strongyloidiasis can rapidly evolve into life-threatening disseminated disease, whereas chronic schistosomiasis can lead to complications causing future morbidity and death.

Currently in France, an update of diagnostic tests reimbursed for those tropical diseases is ongoing to fully cover diagnostic needs.

Our aim was to assess the clinical relevance of tests used in schistosomiasis’ or strongyloidiasis’ diagnosis and include the most relevant in the national list of reimbursed tests.

The assessment involves a critical analysis of national and international guidelines identified by a systematic literature search, and stakeholders’ views.

This work identifies several autochthonous outbreaks of those diseases in France; such as urogenital schistosomiasis that occurred in Corsica, in summer 2013. Also it enlightens the increase of strongyloides serological tests performed in the past years. Those facts prove the potential development of those infections in Europe.

It underlines that, serology is the first diagnostic test line for most cases and is more sensitive than stool microscopy which represents however the final diagnostic investigation to confirm the intestinal infection.

It confirms the main indications of those two diagnostic tools.

It relies on a tropical infectious disease expert network including the French army health service. They have brought further clarification of diagnostic tests clinical relevance for travelers or autochthonous cases.

This new use of Health Technology Assessment has allowed updating and listing the relevant diagnostic tools which might be crucial to better follow those diseases and it may help the health system to face the increase of tropical infections.

This study explored the cost-effectiveness of expanding Hepatitis C Virus (HCV) screening and subsequent treatment in Swiss custodial settings, given the availability of rapid antibody saliva tests (Oraquick®) and dried blood spot tests (semi-quantitative viremia and viral genotype), and recent therapeutic advances which have higher cure rates and shorter treatment courses (1).

A comprehensive strategy offering screening to all detainees was compared to the current setup of screening high-risk individuals (for example, from endemic countries, active or former injecting drug users). A decision tree simulated the diagnosis pathway, and results from a Markov model were included to predict treatment effects and natural progression over a lifetime time-horizon. Input data were derived from clinical studies, literature reviews, custodial health services and expert opinion (2). The net monetary benefit (NMB) and incremental cost-effectiveness ratio (ICER) of comprehensive compared to current screening were calculated. Deterministic and probabilistic sensitivity analyses were performed to explore parameter uncertainty and whether variations informed by expert opinion changed the cost-effectiveness of comprehensive screening.

At a willingness-to-pay threshold of CHF100,000 (USD99,500) per Quality-Adjusted Life-Year (QALY), comprehensive screening had an 83 percent probability of being cost-effective, with a corresponding NMB of CHF33,451,972 (USD33,284,712) and ICER of CHF7,168/QALY (USD7,132/QALY). Results were most sensitive to the QALYs gained from the treatment model (both treatment and no treatment arms), respective HCV prevalence in the current and comprehensive screening populations, treatment initiation rates, and screening offer acceptance rates. Compared to the current practice of screening high-risk individuals, comprehensive screening is likely to be cost-effective due to the increase in testing rates, which were conservatively estimated in this study. Furthermore, comprehensive HCV screening of prisoners may prove more cost-effective in countries where prisoners are not routinely screened.

Comprehensive screening programs could be considered in prison units with a large proportion of high-risk individuals and where detainees are incarcerated for enough time to complete a treatment course during their sentence.

Drug overuse in healthcare settings is common in China. Clinical pathways are tools that provide the link between the best available evidence and clinical practice. This study aimed to determine if the clinical pathway of community-acquired pneumonia (CAP) had effects on the antibiotic use in patients with CAP.

The study was conducted in Shanghai, Hubei Province, and Gansu Province to represent high, middle, and low levels of socioeconomic status in 2015. In each region, three public tertiary general hospitals and three public secondary general hospitals were selected for chart review of antibiotics’ utilization in the patients with CAP during 2014. A multilevel logistic regression model was used in the study, with a dependent variable of appropriate utilization of antibiotics (right time, right type, and right combined use) and independent variables of hospital adoption of clinical pathway and patient characteristics (sex, age, severity of disease, and number of comorbidities).

Twelve surveyed hospitals (66.67 percent) adopted CAP clinical pathways and 354 cases (66.29 percent) were from these twelve hospitals (CP group). Among the total utilization of antibiotics (796 times) in eighteen types of antibiotics used in patients with CAP, the five recommended types of antibiotics accounted for 82.16 percent.

The percentages of cases that got initial antibiotics in time were 90.60 percent in the CP group and 76.11 percent in the non-CP group. The compliance rate for appropriate types of antibiotic utilization was 88.36 percent in CP group, much higher than that in non-CP group (70.22 percent). For 244 cases that used combined antibiotics, the compliance rate for the recommended combinations of antibiotics was 20.12 percent in the CP group, but 1.25 percent in the non-CP group. After controlling patients’ characteristics, the patients in the CP group got more appropriate antibiotics than those in the non-CP group.

Adoption of the CAP clinical pathway in hospitals can improve antibiotics' utilization.

Medtech innovation briefings (MIBs) are intended to support National Health Service (NHS) decision makers and staff who are considering using new innovative medical devices and in-vitro diagnostics. MIBs are produced in support of the NHS 5-Year Forward View, specifically to accelerate innovation in new treatments and diagnostics. This project aimed to evaluate the extent to which published MIBs address national priorities set by NHS England, including in six clinical areas: cancer, mental health, dementia, diabetes, learning disabilities, and maternity.

Data was extracted from eighty-seven MIBs downloaded from the National Institute for Health and Care Excellence (NICE) website including: study design, amount of evidence, date of CE mark, population, cost, manufacturer, device class, publication date, and category of conditions and disease (as prescribed by NICE). Descriptive analysis was done for each variable and frequency tables were produced for MIBs by disease category.

Cardiovascular disease (n = 19) and cancer (n = 12) were the two most common conditions addressed by MIB-evaluated devices. The four medical conditions with the fewest MIBs (n = 1 each) were: diabetes, liver conditions, neurological conditions, and fertility, pregnancy and childbirth. Of the eighty-five MIBs with stated device classifications, just over half were Class IIa and IIb devices and 18 percent were in-vitro diagnostics. The earliest original CE mark was 1997, and approximately half of the devices obtained or updated their CE mark after 2010.

Chronic conditions such as cancer, cardiovascular disease, and diabetes accounted for 89 percent of total deaths in the UK in 2014, thus, the most commonly published MIBs aptly address these issues. However, MIBs are lacking in five out of six NHS priority areas. There is opportunity for innovative technologies to be reviewed via MIBs and alternative NICE pathways in the areas of diabetes, maternity, mental health, and learning disabilities and dementia.

Existing literature shows evidence on the differences in drug reimbursement decisions across countries. These differences are the reason for this study. The main aim of this research is to model the impact of drug reimbursement decisions on health outcomes (that is, life expectancy, healthy life years and mortality rates). In particular, this study is looking at countries that have different acceptance, restriction and rejection rates for drug reimbursement decisions.

The current study is based on a longitudinal dataset with data from nine European countries from 2002 to 2014. This dataset is formed of primary data on drug reimbursement decisions (that is, cancer drugs) collected in the Advancing and strengthening the methodological tools and practices relating to the application and implementation of Health Technology Assessment (ADVANCE-HTA) project and secondary data on life tables and indicators of health and socioeconomic status (from Eurostat and World Bank). Following the longevity model defined by Lichtenberg (1), a panel data model with country and year fixed-effects is run on this dataset in order to model the impact of the level of access to drugs on health outcomes.

The results show that the rate of adoption of new drugs into a national health system does not have any significant effect on life expectancy. However, more restrictive systems are positively and significantly related with healthy life years. Finally, for mortality rates, higher rejection rates are associated with lower deaths.

To conclude, contrary to the public opinion, results show that a more restrictive drug reimbursement system is not related with a worse health outcome, it is either associated with a positive outcome or it is not related.

European collaboration in Health Technology Assessments (HTAs) has gained increasing recognition in recent years, not only on pharmaceutical products but also on high-risk medical devices. For medical devices, quality assessments of efficacy and safety are particularly important due to the weak market authorization in Europe. Strengthening efforts towards better collaboration thus plays a pivotal role to reduce overlap and save resources. This study explored the level of redundancy in HTA assessments of medical devices in Europe in order to identify areas for better collaboration.

We performed an analysis of European HTA reports of medical devices regarding their timing in relation to market authorization, the respective level of evidence used and the overlaps in topics. The ADVANCE HTA database from 2014 was used to select a cohort group of ten high-risk medical devices. A systematic search was conducted to identify all relevant, European HTA reports investigating the ten devices within a time span of 12 years (2003-2015). We analysed the number of annual assessments per technology and evaluated activity patterns, late and early assessors, and minimum evidence requirements.

The results revealed the amount of redundancies in European HTA production: the number of reports per technology ranged from a minimum of five to a maximum of twenty-two over a time-span of 12 years. Within a single year, one technology was assessed up to six times by different HTA institutes in Europe. Out of fourteen countries included in the evaluation, two countries assessed each technology, and seven countries assessed more than seven out of the ten technologies.

The findings indicate that more efficient collaboration is needed to save scarce resources and time of HTA institutes. Efficient collaboration as such needs to shift the focus beyond the time span of one year, and start building on each others work from previous assessments.

Mechanical thrombectomy (MT) is used in patients with acute ischaemic stroke due to occlusion of a proximal cerebral artery. Over the years endovascular techniques have been used to re-canalise blocked vessels, but are not currently reimbursed by National Health Insurance in France.

The aim was to assess the efficacy and safety of MT in combination with intravenous tissue plasminogen activator (IV t-PA), or as an alternative to it, in adults with an acute ischaemic stroke who are not eligible for thrombolysis or in whom thrombolysis has failed; to support the reimbursement decision by National Health Insurance.

Within the scope of The European Network for Health Technology Assessment (EUnetHTA), a rapid assessment of “Endovascular therapy using devices for acute ischaemic stroke” was jointly produced with Haute Autorité de santé (HAS) as a reviewer.

The EUnetHTA report provided a systematic review based on eight randomized controlled trials (RCT) for effectiveness and all available published data for safety.

To produce its assessment, HAS has adapted the EUnetHTA report by:

1. 1. Updating the systematic literature review including the latest published trials

2. 2. Retaining the subgroup analysis of the five most recent trials considered more relevant in the EUnetHTA report for the assessment of effectiveness

3. 3. Analysing specifically the different endovascular interventions studied in the five RCTs

4. 4. Taking into account contributions from stakeholders.

This horizontal collaboration among European HTA doers has facilitated and shortened the assessment of the clinical benefit of this technology, confirming the relevance of EUnetHTA cooperation.

This clinical assessment of thrombectomy is to be completed by the evaluation of its organizational impact in the management of acute ischemic stroke.

Preference-based measures of health-related quality of life play a key role in the calculation of Quality-Adjusted Life Years (QALYs) for Health Technology Assessment (HTA). The Child Health Utility 9D (CHU9D) is a new preference-based instrument designed specifically for application in children and adolescents (aged 7 to 17 years). This study aimed to compare Chinese and Australian adolescent population preferences for CHU9D health states using profile case best worst scaling (BWS) methods.

Fifty CHU9D health states (blocked into five survey versions) were generated for valuation using a fractional factorial design. Study participants were recruited through an online panel company in Australia, and through primary and secondary schools in China. A latent class modelling framework was adopted for econometric analysis.

A total of 1,982 respondents (51 percent female) in Australia and 902 respondents (43 percent female) in China provided useable survey responses. Latent class analysis indicated the existence of preference heterogeneity for both population groups. In the Australian sample, respondents in Class I placed the most importance on the mental health dimensions of the CHU9D (for example, Worried and Annoyed) and the least importance on daily activities (for example, Activities, Daily routine, Sleep), whilst respondents in Class II placed equal weights on all attributes. In the Chinese sample, respondents in Class I placed the most importance on the Activities dimension of the CHU9D and the least importance on the Annoyed dimension, whist Class II placed the most importance on the Schoolwork dimension and the least importance on Pain.

This study has provided important cross-country insights into the use of profile case BWS methods to elicit health state preferences with young people for application in HTA in children and adolescents. The differential latent classes identified between Australia and China highlights the necessity to derive country-specific adolescent scoring algorithms for the CHU9D instrument for application in HTA.

We test in practice a Multiple Criteria Decision Analysis (MCDA) framework for the value assessment of a set of therapeutic options for the indication of hormone relapsed metastatic prostate cancer (mPC) through a series of simulation exercises with the participation of decision makers from different Health Technology Assessment (HTA)/insurance agencies across Europe, including TLV (Sweden), AETSA (Andalusia-Spain), INAMI-RIZIV (Belgium) and AOTMiT (Poland). The drugs evaluated were abiraterone, cabazitaxel and enzalutamide.

Using a multi-attribute value theory framework, past research outcomes and literature findings, an mPC-specific value tree was constructed incorporating relevant concerns as criteria. By adopting the MACBETH approach the different drugs were scored against the criteria through the development of value functions, relative weights were assigned to the criteria using a swing weighting technique, scores and weights were combined using an additive aggregation technique, and sensitivity analysis of results was conducted. All stages were informed through the participation of a small group of experts from each HTA/insurance agency at a series of decision conferences taking place in each country.

Value parameters considered spanned the dimensions of therapeutic impact, safety profile, innovation level and socioeconomic impact. Overall weighted preference value scores were produced reflecting the performance of the treatments against the criteria while considering their relative importance. Order of treatments’ rankings was identical across all agencies, with enzalutamide scoring highest and cabazitaxel lowest. Therapeutic impact criteria always produced the greatest relative weight. Hypothetical priority setting decisions were made based on “value-for-money” grounds through the use of “cost per unit of value” metrics by incorporating purchasing costs.

The MCDA framework tested possesses a number of characteristics that could facilitate decision making, including the systematic and explicit incorporation of value trade-offs as part of model assessment and the transparency throughout all its stages. Therefore, it has the prospects to act as a practical evaluation tool for value assessment and communication during the HTA process.

Gastric cancer (GC) is one of the most common malignancy and the third leading cause of cancer mortality worldwide. Currently, platinum-based and fluoropyrimidine-based combinations represent the milestone of front-line drug regimens. Unfortunately, there are few treatment options after failure of first-line therapy. Ramucirumab, a human IgG1 monoclonal antibody to VEGFR-2, has been recently approved in the European Union (EU) for use as monotherapy or in combination with paclitaxel as second-line treatment in patients with advanced GC with progressed disease. We performed a cost-effectiveness analysis of the Ramucirumab plus paclitaxel doublet versus paclitaxel alone in patients with previously treated advanced GC, based on results of the RAINBOW trial (1).

A Markov model has been developed in order to estimate the Life Years Gained (LYGs) and the incremental cost-effectiveness ratio (ICER) for both treatments. The model adopted the Italian healthcare system perspective and the time horizon is that of the lifetime of a patient with an advanced GC. The model considered three distinct health states: stable, progression or death. Transition probabilities were extracted from the Kaplan-Meier curves provided in the trial and cubic/spline function was used to approximate the extrapolation of survival curves for each treatment cycle. An internal model validation was performed to validate the Overall Survival (OS) curves generated by our model simulation. We based our economic analysis on clinical data and resource consumption (drugs, drug administration, supportive care medications, disease monitoring and graded 3 or 4 adverse events) on the Italian setting (2,3). All costs were expressed in euros. Sensitivity analysis also have been performed.

This cost-effectiveness study demonstrated that, in 2nd-line therapy, the combination of ramucirumab with paclitaxel provides an incremental benefit (+1.54) at high incremental cost (EUR41,616) per LYGs.

At a threshold of EUR5,000 for LYGs, based on Italian perspective, ramucirumab plus paclitaxel had less probability of being cost-effective. To our knowledge, our study is the first modeling study from an Italian payer perspective and the first worldwide to examine ramucirumab as a 2nd-line treatment.

Although many biologics (Bs) have been approved for the treatment of moderate-to-severe Ulcerative Colitis (UC) in patients who have responded inadequately to conventional therapy, the selection of Bs is controversial due to the lack of head-to-head trials. Indirect economic comparisons of these costly drugs are available from National Healthcare perspectives that are not the Italian ones. Therefore, the objective is to evaluate cost-utility of Bs for the treatment of refractory moderate-to-severe UC both in Italy and in the Lombardy Region.

A Markov model (considering three transition states: remission, clinical response, relapse) was constructed using the software R 3.3.1 markovchain-package to evaluate incremental cost-utility ratios (ICUR) of adalimumab (ADA), infliximab (IFX), infliximab biosimilar (IFX-B), golimumab (GOL) and vedolizumab (VED) treatments of patients over a 10-year time horizon from the perspective of the Italian (N) and Lombardy Region (R) healthcare system. Clinical parameters were derived from clinical trials. Costs (actualized by – 1.5 percent) were obtained from the National database and Regional public tender. Utility was expressed as QALY (Quality-Adjusted Life Years).

Costs per treatment were different from a N and R perspective (ADA -55 percent; IFX -16.7 percent; IFX-B -29.6 percent; GOL -9.6 percent; VED -10 percent). Direct healthcare costs (treatment cost, visits, laboratory tests, hospital admissions) were calculated over 10 years of treatment per patient: ADA (N: EUR114,227, R: EUR68,314, -40.2 percent), IFX (N: EUR130,595, R: EUR103,081, -21 percent), IFX-B (N: EUR110,438, R: EUR78,852, -28.6 percent), GOL (N: EUR118,602, R: EUR96,922, -18.3 percent), VED (N: EUR113,852, R: EUR102,932, -9.6 percent) with associated QALY respectively of 6.68, 6.66, 6.66, 6.70, 7.02. From a N perspective, IFX-B was dominating compared to all other treatments. The ICUR of VED/IFX-B was EUR9,483 for 10 years (willingness to pay EUR948/QALY). From a R perspective, ADA was dominating compared to all other treatments. The ICUR of VED/ADA was EUR101,818 for 10 years (Willingness to Pay, WTP EUR10,182/QALY).

National and Regional cost-utility analyses produced different results. As Regional price discounts can occur, local analysis is needed to estimate the economic impact of therapies to ensure optimal choice.

The reduction of healthcare costs and societal cost due to remote monitoring (RM) of cardiac implantable electronic devices (CIEDs) has been demonstrated in several countries; however, to the best of our knowledge it does not exist for South Korea. This work aims at providing an estimation of the potential benefit of RM versus standard care (SC) of CIEDs in term of healthcare costs in South Korea, in order to provide additional substance to the currently ongoing societal debate about the value of telemedicine.

Healthcare resource consumption was taken from the results of the TARIFF study, a prospective, non-randomized, multicenter clinical trial designed in Italy to assess the economic benefits of RM follow-up in comparison with standard follow-up in 209 patients (107 SC, 102 RM). The main results demonstrated that RM reduced healthcare resource consumption by 54 percent from a healthcare services perspective (SC: EUR1,044.89±1,990.47 versus RM: EUR482.87±2488.10, p<.0001 (1).

In order to perform a cost analysis from the perspective of the South Korean healthcare payer, the following unit costs were assigned to resources collected in TARIFF (hospitalizations, visits, examinations): fee-for-service tariffs, emergency tariffs and outpatient tariffs. Remote follow-up costs were considered as zero.

From the perspective of the South Korean healthcare payer, the overall mean annual cost/patient in the RM group is 53 percent lower than in SC group (SC: EUR405,439±40,135 versus RM: EUR189,96±725,52, p<.0001) (SC: KRW 497,145±49,2137 versus RM: KRW 232,936±890,181, p<.0001). This is mainly due to a significant cost reduction in device-related hospitalizations, examination tests and visits in the follow-up period.

RM of CIED patients is cost-saving from the perspective of the South Korean healthcare system. Introducing appropriate reimbursement for remote monitoring of CIED is not likely to change this result and should make RM sustainable for the provider and encourage widespread adoption of RM.

The cost-effectiveness of Human papillomavirus (HPV)-based primary cervical screening in the Irish healthcare setting is assessed using a decision-analysis approach to inform a decision around changes to the national screening program. Current practices comprises primary screening with liquid-based cytology (LBC) followed by HPV triage, at 3-yearly intervals for ages 25 to 45 years and 5-yearly until age 60 years.

This study assessed changing the primary screening test from LBC to HPV testing, in both an unvaccinated and a vaccinated (against HPV 16/18) cohort. It considered extending the screening interval (to 5-yearly for all), the upper age limit (from 60 to 65 years) and different test sequences (four possible tests were included: HPV, LBC, partial genotyping for HPV16 or HPV 18 and the molecular biomarker p16INK4a/Ki67). A Markov-model for HPV-infection and cervical cancer was developed based on a German cervical screening model (1). The perspective of the healthcare system was adopted and a 5 percent discount rate used.

Strategies using HPV as the primary screening test are more effective than LBC-based strategies. The optimal strategy, at a willingness-to-pay threshold of EUR45,000 per quality-adjusted life year (QALY), for the unvaccinated cohort was HPV-based primary screening with a LBC triage test, at five-yearly intervals from age 25 to 60 years. This strategy is cost saving compared with current practice and cost effective when compared to no screening, with an Incremental cost-effectiveness ratio (ICER) of EUR18,164 per QALY. The optimal strategy for the vaccinated cohort was also HPV primary screening with a LBC triage test, at five-yearly intervals from age 25 to 60 years. While more effective and cost saving compared with current practice, it would not be considered cost effective compared with no screening (ICER of EUR58,745/QALY).

Based on our analyses, HPV-based cervical screening is more effective and cost saving compared with LBC-based screening for both vaccinated and unvaccinated cohorts in an Irish setting.

Misdiagnosis of asymptomatic bacteriuria as catheter-associated urinary tract infection (CAUTI) leads to unnecessary tests and other low-value care. We used this topic as the prototype to develop a clinical pathways program to promote evidence-based decision making in a multi-hospital system.

We convened a task force including hospital and critical care physicians, nurses, laboratory staff, and informatics specialists. Our Health Technology Asessment (HTA) center completed a rapid systematic review on guidelines and algorithms for diagnosing CAUTI. Additional rapid reviews were completed as necessary to address specific follow-up questions. A draft pathway based on the guidelines was developed, and then the task force edited it in an iterative process.

We used the Dorsata platform (Dorsata Inc., Washington, DC) to create, distribute and maintain the pathway. Dorsata has both desktop and mobile interfaces that guide clinicians through decision algorithms. Individual pathways include links to references and a portal for direct user feedback. Pathway owners have access to a real-time pathway utilization dashboard.

A standardized order set with the pathway was added to our electronic health record system. We also held educational meetings for residents and provided “huddle sheets” to nurse educators at each hospital. Posters and computer screen savers were also used to raise awareness of the new pathway.

We now have a total of 111 pathways on Dorsata, developed following the same model as the CAUTI evaluation pathway. Some topics, like breast cancer, have as many as sixteen pathways, addressing different clinical questions like first- and second-line therapy. Over 600 individuals have registered for the mobile app, including attending and resident physicians, nurses, and medical students. The pathway site had 1,619 views in December 2016, the most recent month for which complete records are available. The pathways are proving to have an effect on clinical decision making. For example, the annualized number of unnecessary urine cultures avoided as a result of the pathway is 4,474; resulting in estimated direct cost savings of USD67,110.

Using pathways to present HTA information at the point of care is feasible and can improve the value of care.

Hospital mangers need information for decision making (1). Hospital-based health technology assessment (HTA) methods were tested out to support the budget planning of investments for a new building to be constructed for diagnostic and teaching units at a publicly funded tertiary care university hospital. The hospital board nominated an ad hoc working group to reassess all investment proposals for devices, equipment and furniture for the diagnostic or teaching units that intended to move into the new building. The need for assessment was obliged when the submitted proposals of the units exceeded two-fold the initially allocated investment budget.

Depending on the level of expenditure, all proposals were assessed by one of the following processes: (i) Proposals over EUR250,000 were evaluated by three to five person expert groups using multi-domain assessment adapting Hospital-based HTA-principles; (ii) Proposals between EUR50,000 and EUR250,000 were returned to the units for miniHTA-assessments by clinicians who submitted the initial proposals and (iii) All proposals below EUR50,000 were prioritized by the units to cut the expenditure by at least 25 percent, with a special emphasis on synergistic use of devices and equipment among the units.

The expert groups suggested significant reductions to the proposals, including the withdrawal of a Magnetic Resonance Imaging (MRI)-unit considered to be suboptimally located. Furthermore, the need for a new scanner was declined by promoting adherence to evidence-based diagnostic guidelines and more efficient utilization of existing scanners. Self-assessed MiniHTAs revealed proposals that were unnecessary or the specifications for devices needed re-adjustments. Prioritization revealed excess numbers of devices, for instance the number of cold storage appliances could be reduced. Altogether, the investment proposals were cut by over EUR3.8 million to reach the initial budgetary allocation.

Innovative and flexible usage of hospital-based HTA methodology can be applied to budget planning and evaluation of investment proposals to support decision making. Based on encouraging results, hospital-based HTA was accepted to become a part of hospital strategy as a tool for the annual investment planning.

Inhibitors of Vascular Endothelial Growth Factor (VEGF) have made possible the treatment of the Retinal Vascular Diseases (RVD) for which there were limited therapeutic resources. The aim of this work was to estimate annual direct costs of major macular edema diseases in Italy in particular Choroidal Neovascularization (CNV), Diabetic Macular Edema (DME) and Retinal Vein Occlusions (RVO) from the hospital perspectives.

This descriptive study was aimed at quantifying direct costs incurred by five hospitals in Italy. Administrative and clinical databases of Policlinico Tor Vergata in Rome were analyzed for a 6-year period. In this context, it was possible to stratify patients depending on the disease and number of eyes treated. From these results, a survey with structured questionnaires was developed involving four other hospitals in Italy. Thanks to that, direct costs (drugs and specialist) were estimated from the hospitals perspective in 2016.

Interviews included 7,356 individuals of which 1,860 were treated in both eyes. Within the considered five hospitals, 64 percent of treated patients had CNV, 21 percent DME, and 15 percent RVOs. The average annual administration rate of anti-VEGF treatment resulted in 4.03 (Standard Deviation, SD 3.46) per patient eye: 4.69 (SD 1.75) for cases enrolled for less than one year (naïve) and 3.38 (SD 0.82) per patients treated for more than one year (experienced). Naïve patients had a mean per capita annual cost of EUR2,368 per eye (EUR2,536 for CNV; EUR2,280 RVO; EUR1,986 DME) of which EUR2,952 was related to the administration of on-label drugs mainly Eylea, Lucentis, Macugen, Ozurdex and EUR49 due to off-labels such as Avastin. Experienced patients average annual cost per eye was EUR1,689: EUR2.179 for the on-label drugs, EUR34 due to off-labels (EUR1,839 for CNV; EUR1,327 RVO; EUR1,399 DME). The average rate of the specialist annual visit was four times; the most frequent types were Optical Coherence Tomography (OCT), Angiography, and Fundus Photography (FP).

This is a first attempt to study direct costs incurred from the hospital perspective associated with RVD with overexpression of VEGF in Italy. This might represent a first step for further analysis assessing the burden of RVD diseases from the Italian National Health System perspective globally.