A common approach to one-way sensitivity analysis is to vary inputs by a constant percentage. An alternative is to derive ranges using evidence-based probability distributions from published sources. Our objective was to compare one-way sensitivity analysis results when using these two approaches for a reference case model, along with two additional case studies.

For the reference case, we replicated a published Human Immunodeficiency Virus/Acquired Immunodeficiency Syndrome (HIV/AIDS) cost-effectiveness Markov model (zidovudine versus zidovudine plus lamivudine in the UK) using TreeAge®. Health states included three HIV/AIDS states and death. We generated one-way sensitivity analyses by varying inputs in two ways: (i) using ±15 percent for all inputs, and (ii) using the 2.5 and 97.5 percentile values of the evidence-based probability distributions for all inputs. Our outcome was the mean difference between lower and upper incremental cost-effectiveness ratios (ICERs) for each variation method for the ten most influential inputs. We assessed the number of inputs with a mean difference between lower and upper ICERs of >10 percent of the deterministic ICER.

The deterministic ICER was GBP7,654/QALY (quality adjusted life year) for combination therapy versus monotherapy. The mean difference in ICER uncertainty for the evidence-based vs. ±15 percent variation method was GBP3,251/QALY (p = .0096). Six inputs had a mean difference in ICER uncertainty of >10 percent of GBP7,654/QALY (that is, mean difference in ICER uncertainty > GBP765) for the evidence-based variation method, compared to only two inputs for the constant percentage variation method.

For the reference case, the magnitude of uncertainty in the outcome was larger for the evidence-based variation method compared to the constant percentage variation method. Evidence-based uncertainty in inputs should be used in all sensitivity analyses to reflect realistic uncertainty in an outcome and aid decision-making about future research strategies. Additional case studies will be presented using validated models in diabetes and asthma.

Emergency admissions to hospital are a major financial burden on health services. In one area of the United Kingdom (UK), we evaluated a predictive risk stratification tool (PRISM) designed to support primary care practitioners to identify and manage patients at high risk of admission. We assessed the costs of implementing PRISM and its impact on health services costs. At the same time as the study, but independent of it, an incentive payment (‘QOF’) was introduced to encourage primary care practitioners to identify high risk patients and manage their care.

We conducted a randomized stepped wedge trial in thirty-two practices, with cluster-defined control and intervention phases, and participant-level anonymized linked outcomes. We analysed routine linked data on patient outcomes for 18 months (February 2013 – September 2014). We assigned standard unit costs in pound sterling to the resources utilized by each patient. Cost differences between the two study phases were used in conjunction with differences in the primary outcome (emergency admissions) to undertake a cost-effectiveness analysis.

We included outcomes for 230,099 registered patients. We estimated a PRISM implementation cost of GBP0.12 per patient per year.

Costs of emergency department attendances, outpatient visits, emergency and elective admissions to hospital, and general practice activity were higher per patient per year in the intervention phase than control phase (adjusted δ = GBP76, 95 percent Confidence Interval, CI GBP46, GBP106), an effect that was consistent and generally increased with risk level.

Despite low reported use of PRISM, it was associated with increased healthcare expenditure. This effect was unexpected and in the opposite direction to that intended. We cannot disentangle the effects of introducing the PRISM tool from those of imposing the QOF targets; however, since across the UK predictive risk stratification tools for emergency admissions have been introduced alongside incentives to focus on patients at risk, we believe that our findings are generalizable.

Gastroenteropancreatic neuroendocrine tumours (GEP-NETs) are rare cancers most often found in the gastrointestinal system or the pancreas. However, patient-reported health state utilities based on clinical trials have not been previously reported in this disease area.

The CLARINET study collected the European Organization for Research and Treatment of Cancer (EORTC) QLQ-C30 data from patients in both stable and progressive disease states, although data for the latter were only available during the early stage of progression due to trial design. Using published algorithms, data were mapped to EQ-5D utility values. Random-effects generalized least squares models were used to investigate the impacts of progression status, tumour site and other patient characteristics on mapped utility values.

In total, 1,053 observations from 204 patients were mapped to EuroQol (EQ-5D) utilities using the McKenzie mapping algorithm. The final random-effects model included age, gender, baseline utility and progression status as covariates; it was not feasible to investigate time-to-death utility due to a limited number of death in the CLARINET study. Tumour location (midgut versus pancreas) does not seem to affect utility. However, the difference in utilities based on progression status is statistically significant (p<.05) in the base case analysis, and the estimated utilities for stable and progressive disease are .776 and .726, respectively. Furthermore, scenario analyses showed that utility for progressive disease is numerically lower than for stable disease, but this may not be statistically significant in some scenarios.

Patients with GEP-NETs experience worse utility values in the progressive disease state compared to the stable disease state, based on patient-reported health-related quality of life (HRQL) data from the CLARINET study. The decline of utility in the progressive disease state may be underestimated because progressive HRQL data were only collected shortly after the progression event in the trial. The estimated trial-based utilities can be used in future economic evaluations for GEP-NET treatments and to provide more insights to physicians on patient-reported quality of life outcomes in GEP-NETs.

Increasing access to surgical care is crucial in improving the general health status of a population. Despite studies indicating the cross-country differences of general health indicators, there is a scarcity of knowledge focusing on the cross-country differences of surgical indicators. This study aims to classify countries according to surgical care indicators and identify risk predictors of catastrophic surgical care expenditures.

For this study, data were used from the World Health Organization and the World Bank on 177 countries. The following variable groups were chosen: total density of medical imaging technologies, surgical workforce distribution, number of surgical procedures, and risk of catastrophic surgical care expenditures. The k-means clustering algorithm was used to classify countries according to the surgical indicators. The optimal number of clusters was determined with a within-cluster sum of squares and a scree plot. A Silhouette index was used to examine clustering performance, and a random forest decision tree approach was used to determine risk predictors of catastrophic surgical care expenditures.

The surgical care indicator results delineated the countries into four groups according to each country's income level. The cluster plot indicated that most high-income countries (for example, United States, United Kingdom, Norway) are in the first cluster. The second cluster consisted of four countries: Japan, San Marino, Marshall Islands, and Monaco. Low-income countries (for example, Ethiopia, Guatemala, Kenya) and middle-income countries (for example, Brazil, Turkey, Hungary) are represented in the third and fourth clusters, respectively. The third cluster had a high Silhouette index value (.75). The densities of both surgeons and medical imaging technology were risk determiners of catastrophic surgical care expenditures (Area Under Curve = .82).

Our results demonstrate a need for more effective health plans if the differences between countries surgical care indicators are to be overcome. We recommend that health policymakers reconsider distribution strategies for the surgical workforce and medical imaging technology in the interest of accessibility and equality.

There are several methods to cost hospital contacts when estimating the cost effectiveness of a new intervention. In England, the National Insitute for Health and Care Excellence (NICE) recommends the use of diagnosis-related group (DRG)-based costs as a valuable way of costing hospital resource use. There are three main sources of unit costs of a DRG: (i) tariffs as used for reimbursement purposes, (ii) benchmarking finished consultant episode (FCE)-level reference costs and (iii) benchmarking spell-level reference costs.

The purpose of this work is to compare the implications of choosing a particular source of DRG-based unit costs when conducting an economic evaluation.

As a case study, we used a cost-utility model developed to compare secondary fracture prevention models of care for hip fracture patients (1). A Markov model was derived from large primary and hospital care administrative datasets in England. Utilities were informed by a meta-regression of thirty-two studies. Hospital resource use (inpatient, outpatient, critical care and emergency care) was valued using the three different 2014–15 DRG-based unit costs and regression-based costing models were derived from 33,000 hip fracture patients to inform the health states of the model (2). For each source of DRG-based costs, we calculated mean life years, Quality-Adjusted Life Years (QALYs) and costs for a representative male and female associated with three models of care: (i) orthogeriatrician (OG)-led, (ii) nurse-led fracture liaison services and (iii) usual care.

Using the benchmarking FCE-level DRG-based costs, the OG-led model was estimated to be the most effective model of care (1.77 QALYs, 95 percent Confidence Interval, CI 1.56-1.98) at a threshold of GBP30,000/QALY. However, it also resulted in the highest costs per patient. We will report the cost-effectiveness results using the two remaining DRG-based costs.

Choosing a particular hospital costing method may have an impact on economic evaluations. We will reflect on the implications for the estimated hospital costs, decision uncertainty and adoption of models of care.

In formulating criteria for Health Technology Assessment (HTA) and priority setting a number of such criteria have been suggested and are used, for example in multi-criteria decision making. Besides taking central aspects like severity of disease, effectiveness, cost-effectiveness and patient safety into account, we also find references to criteria like unmet needs, and lack of alternative treatment. Often these criteria are treated as on par with each other, only given different weights in decision making. However, it seems like there is a conceptual overlap between some of these criteria and if that remains unnoticed, there is a risk of taking the same criteria into account twice. One such example is the relationship between severity of disease and unmet need. The aim of this presentation is to present a tentative analysis of the relationship between severity of disease and unmet need.

The presentation is based on a conceptual and normative analysis.

First it will be argued that we have reason to clarify what is meant by unmet needs, whether it is a need which is not met to any degree or if it is a need for which there is no treatment with curative intent or for which there is only palliative treatment, for example. Second, analyzing unmet needs in relation to severity, a number of different scenarios will be examined, showing that unmet needs can be captured in terms of severity of disease (to some extent dependent upon how we operationalize severity of disease).

The general conclusion of the study is that we have reason to carefully analyze criteria used for decision making in HTA from a conceptual and normative perspective in order to uncover logical relationships and avoid overlapping criteria. In relation to the specific question of unmet needs versus severity of disease, the conclusion is that in most cases unmet need will be redudant in relation to severity and we should be careful using both of them in decision making unless we can provide reasons for why it is an exceptional case.

The objective was to identify the conceptual and methodological issues surrounding integration of ethics in Health Technology Assessment (HTA). We conducted a systematic review examining: (i) social needs, (ii) methodological and procedural barriers, (iii) concepts or processes of ethics assessment used and (iv) results of experimentations for integrating ethics in HTA.

Search criteria included ‘ethic’, ‘technology assessment’ and ‘HTA’. The literature search was done up to 21 November 2016 in Medline/Ovid, SCOPUS, CINAHL, PsycINFO and international HTA Database. Screening of citations, screening of full-text and data extraction were performed by two subgroups of two independent reviewers. The first group was constituted of HTA experts, and the second of ethics and philosophy experts. Data extracted from articles were regrouped in categories for each objective.

A list of 2,420 citations was obtained while 1,646 remained after the removal of duplicates. Of these, 132 were fully reviewed, yielding 67 eligible articles for analysis. Eight categories were identified within the social needs. The mostly evoked were ‘Informed policy decision making’ (n = 16) and 'Informed public/patient decision making’ (n = 12). Ten categories of methodological and procedural barriers were identified. The most mentioned were 'Lack of standardized and recognized proceedings for ethical analysis’ (n = 28) and ‘Lack of shared consensus on the role of ethical theory and ethical expertise’ (n = 17). Within the concepts or processes of ethics assessment, thirteen categories were identified. The most mentioned were ‘Fairness and Equity’ (n = 12), ‘Beneficence and Non-maleficence’ (n = 10) and, ‘Autonomy’ (n = 10). Within results of experimentations, five categories were identified. The most mentioned was ‘Usefulness of ethics for identifying relevant problems’ (n = 3). While few experimentations were identified, no clear operational method was found in our research.

This study confirms the necessity to design an operational method integrating ethics and addressing social needs of HTA. Our results constitute the basis for developing a new theoretical and practical method.

This paper is based on a narrative review to identify and describe approaches to incorporate ethical aspects in Health Technology Assessment (HTA). On the first decade HTA was being established as a new area of research, the social and ethical dimensions seemed to play an essential role. This perspective, centered on the social impact of technology contrasts with the current definition, which focuses on the technical conditions of technology, especially properties and effects. Some authors have discussed the obstacles to include the ethical dimension into this area to a large extent. Those authors were motivated by the perception that there are few sections explicitly dedicated to these dimensions in the evaluation reports.

We searched these scientific databases: Pubmed, Cochrane Library, Centre for Review and Dissemination (CRD), PDQ - Evidence and Virtual Health Library (VHL), and selected studies that presented procedures and methodologies for the inclusion of ethical analysis in HTA.

A total of 308 articles were retrieved, nine of them were included. The identified methods were classified into four groups according to the parameters and procedures: (i) normative-based evaluation, (ii) case comparison-based evaluation; (iii) predefined questionnaire application-based evaluation and iv) debate and deliberation-based evaluation.

The result showed a great diversity of approaches (1 - 3) for the inclusion of the ethical dimension in the evaluation studies of health technologies, which can even be used together. It is suggested that its use considers the characteristics and needs of each different application contexts.

This work presented as methodological base of approaches for the integration of the ethical dimension in the HTA field. Nonetheless, the proposed approaches to the incorporation of philosophical field of ethics into the systematization and objectivity field of the HTA reveal a considerable approach diversity that is applied productively. Since it has been agreed that technology evaluation is contextual, different approaches would help to meet the needs for possible adjustment.

Human enhancement technologies need assessments, but they differ from other health technologies. Therefore we may need other methods for their assessment, also with regard to addressing its ethical issues. The objective of this paper is to describe the elaboration of a method for exposing and elucidating ethical issues with human cognitive enhancement. The approach is elaborated in order to support and facilitate open and transparent deliberation and decision making with an emerging type of technology with great potential and formative implications for individuals and society.

The literature search identified relevant approaches. Conventional content analysis of the identified papers and methods revealed their suitability for assessing human cognitive enhancement. Four selection criteria were applied and followed by method development. Pilot testing on smart-glasses (1) resulted in amendments.

A method for exposing and elucidating ethical issues in the assessment of human cognitive enhancement technologies was developed based on three existing approaches in Health Technology Assessment (HTA) (2). The method consists of six steps and a guiding list of forty-three questions. An overview of the approach will be presented.

A method for exposing and elucidating ethical issues in the assessment of human cognitive enhancement has been developed. The method paves the way for context specific ethical assessment and analysis of a new and emerging type of technology.

While “how to perform” a rigorous Health Technology Assessment (HTA) at the institutional level is well established (1), very little has been experienced for empirically approaching an HTA in hospitals: no scientific evidence is available concerning the correct organizational model, to maximise and to improve the functioning, the performance and the effectiveness of the HTA units (2).

This study aims at crystallizing those design options that may positively contribute to the HTA units’ effectiveness (quality) and/or to the efficiency (timely) (3).

After the collection of qualitative data from ninety-five healthcare professionals by means of ad hoc questionnaires and interviews, a hierarchical sequential linear regression model was conducted to verify the existence of HTA units determinants. Size, multidisciplinary, trust among members, HTA previous skills and organizational support were the variables investigated, determining team performance.

A greater size and the presence of different specialities within the working unit positively influenced effectiveness, even if they spent more time to complete the assessment. Trust, previous HTA skills and organizational support played a key role in team performance. Size and previous HTA skills most explained the variance of team effectiveness (R2 = .317; Adjusted R2 = .249). The five investigated variables presented a higher explanatory nature regarding team efficiency (R2 = .246; Adjusted R2 = .165).

The study suggested the creation of multi-dimensional and multi-disciplinary HTA units to increase their effectiveness. HTA units should be monitored by the hospital management board, because an excessive increase in multi-disciplinary and size could determine inefficiency. Trust within members and the attendance of HTA training course improve performance. According to these results, the study gave solutions both to the scholars of HTA and to hospitals strategic management boards, paving the way to the determination of a more efficient and effective HTA units composition.

The purpose of the study was to gather evidence on safety and overall effectiveness of performing laparoscopic surgery by using 3D versus 2D display systems in a variety of pediatric surgical procedures in order to efficiently support the final investment decision on the video system to be acquired.

A new methodology, that is, Decision-oriented HTA (DoHTA) (1) was applied to assess the technology on clinical, technical, organizational, economic, social, ethical and safety domains. A decision-tree covering all the relevant assessment aspects of 3D systems has been derived and weighted following the Analytic Hierarchy Process. Afterwards, another pairwise comparison list was set up to compare both alternative technologies with respect to every lowest indicator.

DoHTA results of the 3D system has mainly forecast its impact on clinical efficacy and productivity within the specific context of use. The 3D system is particularly suitable in reducing the mean error rate, thanks to the stereoscopic depth cues which are lost in 2D vision (2,3) From the technical perspective, the analyses have indicated the reduction in median instrument path length, an enhancement of median motion smoothness, and the decrease in grasper frequency with the 3D display. However, the comparative cost analysis has pointed out that the 3D procedure cost was higher that its comparator.

The assessment of the 3D visual system seems to reasonably satisfy the criteria of feasibility, clinical effectiveness and safety. However, the adoption of the 3D display system in surgical practice could involve increased hospital costs, mainly because of the initial cost of the technology. Indeed, based on the appreciation of the results of DoHTA, especially taking into account the positive technical and clinical features, we conclude that the 3D system may be a good alternative to the 2D system.

Since the adoption of electronic health record (EHR) systems, which contain large volumes of aggregated longitudinal clinical data, promises a number of substantial benefits including better care, improved safety issues and decreased healthcare costs (1). It is also associated with significant costs and large technical and organizational impacts, therefore it is important to conduct comprehensive evaluations of healthcare delivery outcomes. The purpose of the study is to gather evidence on safety and overall effectiveness of EHR implementation in Bambino Gesù Children's Hospital (OPBG).

Decision-oriented HTA (DoHTA) method (2) was applied to assess the technology on clinical, technical, organizational, economic, legal, ethical and safety domains. It's a new implementation of the European Network for Health Technology Assessment (EUnetHTA) CoreModel integrated with the Analytic Hierarchy Process. It allows defining an evaluation structure represented by a hierarchical decision tree filled by indicators of technology's performances, each of which was given a weight proportional to the impact that this criterion provides to achieve the purpose of the decision problem; finally, the alternatives’ ranking was defined.

The multidisciplinary assessment took into consideration all of the aspects and recommendations about the benefits and disadvantages of EHR (3). The synthesis of scientific evidence integrated with results of the specific context analysis, resulted in the definition of components of the decisional hierarchy structure. In particular, EHR seems to offer many benefits in terms of safety and clinical effectiveness such as improved continuity and quality of care, and increased accessibility of the data. The implementation of EHR resulted in important organizational outcome such as EHR configuration, learning curve and training. For these reasons, the usability was the main technical characteristics of the technology taken into account. Finally, legal aspects on privacy and security of data, covered a key role in the assessment.

A thorough evaluation of the EHR before its implementation has permitted hospital's decision makers to choose knowingly.

Cataract surgery is one of the most frequent ophthalmological surgical procedures performed in children. However, clinical outcomes in younger patients are generally unpredictable. Currently, cataract surgery can be performed through the traditional phacoemulsification ultrasound probe or Femtosecond Laser (1). The aim of this study is to describe the application of Decision-oriented Health Technology Assessment (HTA) (DoHTA) to assess the femtosecond laser-assisted cataract surgery (FLACS) compared to conventional cataract surgery (CCS).

To evaluate safety, costs, organizational aspects, effectiveness and technical characteristics of FLACS compared with CCS, a DoHTA method was applied (2). DoHTA is a new implementation of the European Network For HTA (EUnetHTA) Core Model®, which integrates the Multi-Criteria Decision Analysis (MCDA) using the Analytic Hierarchy Process (AHP). All the relevant assessment aspects of FLACS are summarized in a hierarchical decision tree by means of Key Performance Indicators (KPI), subsequently weighted through pairwise comparisons. Lastly, FLACS and CCS were ranked against lowest indicators of decision tree.

The multidisciplinary assessment took into consideration all the aspects and recommendations about the benefits and disadvantages of FLACS compared to CCS. DoHTA results showed that FLACS surgery is safe and effective for pediatric patients. Furthermore, FLACS seems to overcome CCS with several important developments such as increased precision of anterior capsulotomy, reduced ultrasound power requirement during phacoemulsification, decreased collateral tissue damage, increased accuracy and consistency in surgical results as well as better visual outcomes. Notwithstanding such clinical improvements, FLACS is more expensive than its comparator.

The DoHTA results integrated the evidence from the scientific literature (which is still limited) with experts judgments. Indeed, although FLACS had the highest purchase price, DoHTA results showed that FL improves the quality of cataract surgery. Based on our results and taking into consideration the positive safety and clinical effectiveness features, we conclude that FLACS may be a good alternative to CCS.

This rapid review aimed to determine the effectiveness of Sustained Low Efficiency Dialysis (SLED) when compared to Continuous Renal Replacement Therapy (CRRT) and Continuous Veno-Venous Hemofiltration (CVVH) in the treatment of acute renal failure (ARF) with a view to implementing SLED in a tertiary hospital in 2014.

A rapid review was performed on the effect of SLED for patients with ARF compared with CRRT or CVVH. The outcomes of interest were mortality, hemodynamic stability, reduced utilization of intensive care unit (ICU) and cost-effectiveness. The search terms ("sustained low-efficiency dialysis[MESH]”) were used to search PubMed, the Cochrane Library, UK NHS Centre for Reviews and Dissemination databases and the US National Guidelines Clearinghouse for relevant articles until 2014.

Four observational and two randomized controlled trial (RCT) studies were found. The results showed that 90-day mortality, was similar between groups (SLED: 49.6 percent versus CVVH: 55.6 percent, p = .43). Hemodynamic stability did not differ between SLED and CVVH and between SLED and CRRT. Patients in the SLED group had significantly fewer days of mechanical ventilation (17.7 ± 19.4 versus 20.9 ± 19.8, p = .047) and fewer days in the ICU (19.6 ± 20.1 versus 23.7 ± 21.9, p = .04). Patients treated with SLED needed fewer blood transfusions (1,375 ± 2,573 ml versus 1,976 ± 3,316 ml, p = .02) and had a substantial reduction in nursing time (p < .001). The hospital weekly costs were CAD1,431 for SLED, CAD2,607 for CRRT with heparin, and CAD 3,089 for CRRT with citrate. Dialysis using SLED was associated with higher first post-dialysis mean arterial pressure (p = .003) than those treated with CVVH, which led to lower mortality.

The evidence suggests that SLED can be used as an alternative to CRRT, as the outcomes were similar. SLED provides solute removal equivalent to CRRT at significantly lower cost.

Iron Deficiency Anemia (IDA), a common cause of anemia in the world, is a frequently neglected disease that represents the main extraintestinal manifestation affecting patients with inflammatory bowel disease (IBD) (1). The release of new intravenous (IV) iron compounds represents a great opportunity for both physicians and patients, but the higher costs might hold back their optimal diffusion. A Health Technology Assessment (HTA) approach was used to provide insights on the sustainability of the IV iron formulations in a hospital setting, with a special focus on ferric carboxymaltose.

Epidemiology of IBD, as well as IDA associated with these conditions, was assessed with a systematic appraisal of the published literature. Data on efficacy and safety of IV iron formulations currently used in Italy were retrieved from the available medical electronic databases. A hospital based cost-analysis of the outpatient delivery of IV iron treatments was performed. Organizational and ethical implications were discussed.

The reported prevalence of anemia in patients with IBD varies markedly from 10 to 73 percent for Crohn's Disease and from 9 to 67 percent for Ulcerative Colitis. Although there are no studies on direct comparison of different IV iron preparations, the literature indicates good efficacy and safety profiles of these formulations. However, ferric carboxymaltose seemed to provide a better and faster correction of hemoglobin and serum ferritin levels in iron-deficient patients (2,3). Our analyses indicated that ferric carboxymaltose, in spite of a greater price, would have positive benefits for the hospital, in terms of reduced costs related to individual patient management, and for the patients themselves, by reducing the number of infusions and accesses to health facilities.

This hospital-based HTA reports an overall positive organizational, economic and ethical evaluation for the sustainable introduction of ferric carboxymaltose in the Italian outpatient setting.

Minimizing the delay between regulatory approval and Health Technology Assessment (HTA) recommendation is critical to ensure patients access to medicines of therapeutic value. The aim of this study was to evaluate the level of synchronization between the regulatory decision and HTA recommendation.

Data were collected from the public domain for new active substances that were first appraised by the HTA agency in Scotland (SMC - Scottish Medicines Consortium), France (HAS - Haute Autorité de Santé), Germany (IQWIG - Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen), Australia (PBAC - Pharmaceutical Benefits Advisory Committee) and Canada (CADTH - Canadian Agency for Drugs and Technologies), and that reached an outcome in 2014 and 2015. The year the product was approved by the European Medicines Agency (EMA), Australian Therapeutic Goods Administration (TGA) and Health Canada were also assessed.

In 2014 and 2015, fifty-one products with HTA recommendations were identified for SMC and IQWIG, forty-two for HAS, forty for PBAC and thirty-eight for CADTH.

Of the HTA agencies studied, CADTH had the lowest percentage of HTA recommendations occurring the same year as jurisdictional regulatory approval. Of the products with CADTH recommendations in 2014, only 7 percent were approved by Health Canada in the same year. By comparison, all of the products with PBAC recommendations in 2015 were approved by TGA in the same year.

For 2014 and 2015, comparing the percentage of HTA recommendations with the jurisdictional regulatory agency approval the same year showed 7 percent (2014) versus 29 percent (2015) for CADTH: 35 percent versus 37 percent for SMC: 35 percent versus 44 percent for HAS; 56 percent versus 57 percent for IQWIG; and 91 percent versus 100 percent for PBAC.

This study shows that the parallel submission mechanism to enable synchronizing HTA and regulatory decision making is effective in Australia, whilst there remains a synchronization disconnect in other countries; although this may be improving. The extent of decision timing disconnect, influence of company strategy and type of HTA outcome were also studied. This initial analysis suggests gaps between the timing of regulatory approval and HTA recommendation for HTA agencies outside of Australia.

Immunotherapies are a relatively new innovative class of drug that have garnered excitement in the fight against cancer. In 2011, the immunotherapy drug, ipilimumab, was approved. Since then, four additional drugs have gained approval. This analysis evaluates the initial reception of immunotherapies by Health Technology Assessment (HTA) agencies.

The Context Matters Data Model was used to evaluate the regulatory and HTA agency decisions surrounding the five approved immunotherapies through November 2016: atezolizumab, elotuzumab, ipilimumab, nivolumab, and pembrolizumab. Thirty-three labels from Australia, Canada, Europe, and the United States, and ninety-two assessments from Agenzia Italiana del Farmaco (AIFA), Gemeinsamer Bundesausschuss (Federal Joint Committee; G-BA), Haute Autorité de Santé (French National Authority for Health; HAS), Institute for Clinical and Economic Review (ICER), Institute for Quality and Efficiency in Health Care (IQWiG), National Institute for Health and Care Excellence (NICE), Pharmaceutical Benefits Advisory Committee (PBAC), pan-Canadian Oncology Drug Review (pCODR), and Scottish Medicines Consortium (SMC) were found. Using a sample t-test and a chi-squared test, reimbursement agencies’ decisions were evaluated, and the clinical and economic factors that went into these decisions were examined.

Of the evaluated reviews: sixty-four were for melanoma indications, fourteen were for non-small-cell lung cancer (NSCLC) indications, and seven were for kidney cancer indications. Many of the reviews did not reach any decision, but 75 percent of HTA decisions (n = 72; p = .0000) reached were positive. Elotuzumab, approved for multiple myeloma, received a positive decision from G-BA and a negative one from SMC. There was an association between different disease conditions or drugs and the rate of positive decisions.

For reviews that had clinical reasons for their decisions, 72.9 percent (n = 59; p = .0000) had positive clinical rationales that were associated with positive decisions (p = .000). Economic rationales for decisions were more mixed, with only 48.4 percent (n = 31; p = .0000) receiving positive decisions. Positive economic evaluations were also associated with positive decisions (p = .000). Atezolizumab, approved only in the United States at the time of this writing, has yet to be reviewed by any of the HTA agencies.

Immunotherapies are promising new options for the treatment of cancer. Thus far, reception by HTA agencies has generally been positive.

The Health system in Nigeria is structured into three tiers which include tertiary health care, secondary health care and primary health care (PHC). The latter forms the grassroots system of delivering basic health services to communities in both rural and urban centers. However PHC in Nigeria have been affected by poor service delivery. This has resulted in underuse of PHC due to the acceptance and utilization of health services delivered through this system. This research seek to bridge the gap of inequality, reaffirm that implementing PHC is a human right/duty and fosters patient and consumer involvement for economic, social and environmental sustainability of PHC.

A qualitative method of research was adopted using a participatory research model. The relative data was sourced secondarily from recent findings (July 2015) carried out in seventy-three primary health centers across Anambra State, Benue State, Kaduna State, Plateau State and Federal Capital Territory (FCT) of Nigeria. Issues that were examined included: client perspective and community involvement, status of available services, utilization and service delivery, and infrastructure and human resource capacities. The respondent of 294 client/service user population from interview were recorded and analyzed.

The assessment showed client dissatisfaction to services being provided. In most centers, National Primary Health Care Development Agency (NPHCDA) requirements like availability of basic functional equipment, well trained health workers, patient record system, and access to water and sanitation were not met. Most of the facilities visited reported to be disconnected from the health system due to supportive supervision.

Conclusively, the interest of the underserved Nigerian could be advocated for through local committees of consumer organizations. Their involvement will have an impact in PHC evaluation, policy making, and implementation of action plans aimed at improving PHC services.

Pigmented villonodular synovitis (PVNS) is a very rare, benign proliferative tumor affecting the inner lining of synovial joints and tendon sheets. Information on treatment needs of PVNS patients to inform drug development is currently scarce, hence we conducted qualitative research with patients using an online bulletin board (OBB) methodology to generate insights on objective and emotional aspects related to the medical journey and living with this disease.

OBB is an asynchronous, online qualitative market research tool that allows participants to comprehensively answer pre-defined questions in a comprehensive manner. Patients were recruited via physician referral and underwent screening questions to ensure eligibility for the study and willingness to participate. The discussion was moderated, structured, and allowed open answers and in response to other participants posts. Analysis was conducted using a combination of different qualitative analytical tools.

The patient OBB ran for 4 days with eleven participants (n = 3 Canada, n = 4 United Kingdom, n = 4 United States of America) aged 28–57 years, suffering from PVNS for 2–27 years. The key patient insights were: (i) pain is the primary factor, constituting a significant emotional and psychological burden; (ii) surgery (arthroscopy) does not get rid of PVNS, relapse rate was high in these patients; and (iii) PVNS has a big financial impact on patients, their families, and the healthcare system, due in particular to time off work/lost wages (patient & caretaker), for healthcare system it is repeat costs for surgeries/hospital stays plus other medical expenses. We also identified orthopedic specialists/surgeons are the physicians who predominantly manage PVNS at this point, as surgery is the only option.

This study shows the suitability of the OBB for uncovering qualitative patient insights to inform decision making and strategy in early pharmaceutical drug development. OBB lends itself very well to uncovering patient insights which might not be revealed in focus group or telephone interviews, particularly in a rare disease like this. PVNS patients are in need of a medical drug treatment which can reduce pain, relapses and provide an alternative to surgery, the current standard of care.

Healthcare organizations should assess the relevance of both existing and new practices. Involving patients in decisions regarding which health technologies and interventions should be prioritized could favor a better fit between strategic choices and patients needs.

Following a systematic review of existing multi-criteria decision support tools and a consultation with hospital clinicians and managers, a set of potentially relevant criteria was identified. A three-round modified Delphi study was then conducted among four groups (hospital managers, heads of department, clinicians, and patient representatives) in order to reach consensus on criteria that should be considered in the tool.

In total, seventy-four participants completed the third round of the Delphi study. Consensus was obtained on twelve criteria. There were some significant differences between groups in priority scores given to criteria. Patient representatives differed significantly from other groups on two criteria. Their ranking of the accessibility criteria was higher, and their ranking of the organizational aspect criteria was lower than for the other groups.

Patient representatives can be involved in the development of a multi-criteria decision support tool to identify, evaluate and prioritize high value-added health technologies and interventions in order to enhancing clinical appropriateness The fact that accessibility aspects were more important for patient representatives calls for specific attention to these criteria when prioritizing health technologies or interventions. Furthermore, we need to ensure that the decisions made regarding the relevance of these technologies and interventions also reflect patients’ preferences.