Many health care interventions have costs and benefits that spill over to sectors outside the healthcare sector. Little is known about these inter-sectoral costs and benefits (ICBs). However, to achieve an efficient allocation of scarce resources, insights on ICBs are indispensable. The main objective of this study was to identify the ICBs related to health care and provide a sector-specific classification scheme for these ICBs. For this sector-specific classification scheme mental disorders were taken as an example, as we expect that this is the sector with the most ICBs.

Using PubMed, a literature search was conducted for ICBs of mental disorders and related (psycho)social effects. A policy perspective was used to build the scheme's structure, which was adapted to the outcomes of the literature search. In order to validate the scheme's international applicability inside and outside the mental health domain, semi-structured interviews were conducted with (inter)national experts in the broad fields of health care.

The searched-for items appeared in a total of fifty-two studies. The ICBs found were classified in one of four sectors: “Education”, “Labor and Social Security”, “Household and Leisure” or “Criminal Justice System”. Psycho(social) effects were placed in a separate section under “Individual and Family”. Based on interviews, the scheme remained unadjusted, apart from adding a population-based dimension.

This is the first study which offers a sector-specific classification of ICBs. Given the explorative nature of the study, no guidelines on sector-specific classification of ICBs were available. Nevertheless, the classification scheme was acknowledged by an international audience and could therefore provide added value to researchers and policymakers in the field of health technology assessment. The identification and classification of ICBs offers decision makers supporting information on how to optimally allocate scarce resources. By exploring a new area of research, which has remained largely unexplored until now, the current study has an added value as it may form the basis for the development of a tool which can be used to calculate the ICBs of health care interventions.

The National Health Commission issued a special task force on antimicrobial stewardship (AMS). We assess the effects of AMS from 2012 to 2016 in four tertiary comprehensive hospitals in Hainan Province, China, to explore the achievement of AMS to facilitate rational use, to control antimicrobial resistance (AMR), to ensure safety and quality of care.

Data from Hospital Information System (HIS) of hospitals according to the criteria of AMS were analyzed. Microsoft Excel data entry and SAS version 9.3 was used for analysis.

The indicators were general compliance to the national criteria from 2012 to 2016. The percentage of following results in hospitals were gradually reduced: the proportion of antibiotic use in outpatient care (11.09 to 3.25 percent); that in emergency departments (8.46 to 1.53 percent); antibiotics use rate of inpatient care (9.13 to 3.12 percent); antibiotics prophylactic use in type I surgical sites (24.19 to 3.38 percent); the proportion of drug cost (5.54 to 0.12 percent) and total cost of antibiotics (3.27 to 0.45 percent); total cost of antibiotics in outpatient care and emergency departments, which was below 10 percent. Pathogenic detection rate of antibiotics from 2013 to 2016 in three hospitals was increased from 38.75 to 59.6 percent. Hainan Provincial Antibiotics Resistance Monitoring Network conscientiously performs duties, several important and special detection rates of AMR close to the average national level, which have been effectively controlled.

It is needed to continue AMS and to enhance the capacity of rational use of antibiotics by medical professionals. Information systems need to be developed, coordinated and correlated to monitor the consumption of antibiotic use, surveillance of AMR and control of hospital infection.

The European population is aging rapidly. The number of Europeans aged over sixty-five will double in the next fifty years. Active and healthy aging is a societal challenge shared by all European countries, but also an opportunity. The World Health Organization indicated that frailty has become an indicator of lack of successful aging. Therefore, identification of frail elderly is becoming important. However, there are many different screening tools that are currently used to identify frailty. The optimal test should have the capacity to easily identify from the community-dwelling population, those older people at risk of adverse outcomes. During the past years, gait speed has been repeatedly reported as an appealing instrument as a screening tool to detect frailty.

Systematic review of literature on gait speed as predictor of frailty was performed.

A total of 992 articles were retrieved from the literature search and only eleven studies met the inclusion criteria. Frailty is a common geriatric syndrome, characterized by decreased reserve and increased vulnerability to adverse outcomes, including falls, hospitalization, institutionalization and death. Despite frailty is being increasingly recognized in the literature, there is a paucity of direct evidence to guide interventions to reduce frailty. Many single and composite tools to detect the frailty have been proposed but none is consensual, most are time-consuming while evaluating different domains of impairments, and many are not validated. Gait speed seems to be a single, reliable, valid, sensitive, cheap, quick and simple tool that identifies frailty people. However, the way to perform the test parameters vary widely, influencing interpretations of physical performance.

The evidence recommends to detect frailty in people in order to achieve an active and healthy ageing. Gait speed could be a suitable predictor to identify frailty although this systematic review found many differences between the gait speed protocols used in clinical practice. It is necessary to establish a standard protocol of gait speed agreed by experts in the area on frailty to be implemented with success in clinical practice.

The National Committee for Health Technology Incorporation (CONITEC) evaluates health technologies to recommend their inclusion or exclusion within the Brazilian Public Health System (SUS), and uses the budget impact assessment to estimate costs to the system. This study estimated the budget impact of the supply of methionine-free amino acid formula (MFAAf) for patients with classical homocystinuria (HCU) in the SUS.

The incidence of one case per 250,000 live births in Brazil and the registration of a Brazilian association of patients with HCU was assumed to calculate the population. Mortality and responsiveness to pyridoxine rates were applied. The costs of treatment were estimated according to the recommended dosage in literature and public purchasing prices. For calculating the dose of MFAAf patients, a median age of 19 years and weight of 60 kg were assumed, according to Brazilian study data.

The annual cost of treatment was estimated at BRL 77,000 (USD 21,084) per patient. The incorporation of MFAAf for HCU would generate a budget impact in SUS of around BRL 37 million (USD 10.1 million) in 2019 and BRL 188 million (USD 51.5 million) after five years which considers the epidemiological data, and a budget impact of around BRL 6.4 million (USD 1.75 million) in 2019 and BRL 33 million (USD 9 million) after five years which considers the information of a Brazilian association of patients with HCU. The wide range of values in the incremental budgetary impact is due to the lack of information on the epidemiology of the disease in Brazil.

The incorporation of the MFAAf in the SUS represents an important budgetary impact and covers a small number of patients. CONITEC recommended the incorporation of the MFAAf in the SUS, according to clinical protocol.

Involving patients and the public in the health technology assessment (HTA) has always been fundamental to NICE. To ensure the appropriate method of patient involvement remains relevant to the evolving types of HTA, NICE uses varying methods of involvement. These methods have been reviewed to ensure they remain current and relevant for HTA now and beyond 2020, and also to give guidance on the approaches that should form a standard baseline and those that could be optional.

We identified and mapped the different methods of patient involvement used at NICE across five types of HTAs: diagnostics; medical technologies; medicines; ultra-orphan conditions; and surgical procedures. We looked at the varying methods of early engagement identifying similarities and differences, and considered the benefits and challenges of each.

The different methods of patient and public involvement include: lay members (generalist and topic expert) involved in decision making, individual patient input (written and oral), and patient group (organisation) input (written). The types of involvement fell into the following categories: written group submissions, written individual statements, surveys of individuals, pre-meeting events/workshops, oral testimonies at committees, and written consultation responses. The common methods across all HTA types were generalist lay members and consultations.

This review highlighted the varying methods of involvement at NICE and highlighted additional methods that could be standardised across the different types of HTAs as a baseline. These included patient organisation submissions and a method for additionally including individual patients in each type of HTA. We identified that where patient involvement started early and continued at each stage of the process including a pre-meeting event, it was particularly helpful to the stakeholders’ ability to contribute.

Involving patients and the public in the health technology assessment (HTA) is crucial and a key part of the NICE patient and public involvement (PPI) policy. To advance the development of our PPI policy in HTA and build capacity for 2020 and beyond, we took stock of knowledge on stakeholders’ views of involving this cohort in HTA.

We carried out a thematic review of the existing evidence on the involvement of patients and the public in HTA, including: technology appraisals consultation 2017 (110 comments): technology appraisals consultation 2018 (205 comments); and PIP review consultation 2017 with a CHTE focus (162 comments). We used Thomas and Harden's (2008) thematic synthesis to code the data ‘line-by-line’, to develop ‘descriptive themes’, and then to generate ‘analytical themes’. This was followed by using Patton's (1999) triangulation of qualitative data sources to further challenge and refine the emergent themes.

We identified three themes, namely (i) earlier and full engagement, (ii) simpler and easier engagement, and (iii) patient evidence. Respondents emphasised the significance of involving patients earlier and throughout the process of developing every appraisal to enable them to gain a greater sense of participation and ownership. Respondents also expressed a strong view of making it simpler and easier for patients to engage in the process through various methods, e.g., standardising the approaches, and support and training. Finally, respondents expressed their positive attitudes toward using patient evidence in HTA, clarifying how patient evidence is captured and used, and offering a clear feedback mechanism to the impact of patient evidence on decision-making.

This review highlighted the significance of earlier and full engagement with people, making it simpler and easier for people to work with us, and being clearer about how we use patient evidence with a clearer feedback mechanism as to the impact of their input on the final decisions.

Limited public money is available for funding research and the majority of clinical research undertaken is funded by industry. Mechanisms to regulate conflicts of interest within the research process have been implemented. However, these policies by themselves do not protect against potential sponsorship bias that would affect research results to inform decision makers when using the results of these trials. Therefore, the main aim of this study was to evaluate the influence of sponsorship bias on the treatment effects of RCTs.

This was a meta-epidemiological study. A random sample of RCTs included in meta-analyses of physical therapy (PT) area were identified. Data extraction including assessments of appropriate influence of funders was conducted independently by two reviewers. To determine the association between biases related to sponsorship biases and effect sizes, a two-level analysis was conducted using a meta-meta-analytic approach.

We analysed 393 trials included in forty-three meta-analyses. The most common sources of sponsorship for this sample of PT trials were government (n = 205, 52.16 percent) followed by academic (n = 44, 11.2 percent), and industry (n = 39, 10 percent). The funding was not declared in a high percentage of the trials (n = 85, 22 percent). The influence of the trial sponsor was assessed as being appropriate in 246 trials (63 percent) and considered inappropriate/unclear in 147 (37 percent) of them. There was a significant difference in effects estimates between trials with appropriate and inappropriate influence of funders (ES= 0.15; 95% CI -0.03, 0.33;). Trials with inappropriate/unclear influence of funders tended to have on average a larger effect size than those with appropriate influence of funding

Treatment effect size estimates were 0.15 larger in trials with lack of appropriate influence of funders. Systematic reviewers should perform sensitivity analyses based on appropriateness of influence of sponsorship in included trials.

Conclusions and recommendations of health technology assessment (HTA) reports have an impact on all relevant actors involved in the health system (health authorities, administrators, health professionals, patients, citizens and industry). The involvement of all those relevant stakeholders in the HTA process facilitates making valid and informed decisions and an efficient allocation of resources. Improving communication, participation and transparency among all agents will lead to more efficient evaluation and decision-making processes.

To review key aspects of the relations between HTA agencies and health industries, two process were carried out: a narrative review of literature searched in Medline, PubMed, Embase, CINAHL and WOS (2007-2017) and a review of websites of international HTA agencies. References and webs with information on the framework, objectives, methodologies, impact or results of the relationships were included.

A total of 1961 references were located and forty-five were selected. From the synthesis of the selected references the following key aspects of the relationships between HTA and industry were identified: (i) the importance of early dialogues with industry to align HTA objectives with the generation of evidence; (ii) challenges of the bias in the evidence produced by industry; (iii) difficulties in industry engagement in HTA processes; and (iv) industry interest in HTA. The review of six agency websites provided information on industry involvement in strategic activities, early dialogues, provision of documentation, management of industry clarifications, review of the report/allegations and other forms of relationship.

Both the review of the literature and the contents of the web pages of international agencies with experience in relations with industry show that the interest is in the creation of collaborative frameworks between regulatory authorities that decide on authorization and price and reimbursement and HTA agencies, while both try to maintain an early, transparent and systematic interaction with the healthcare industry.

Health technology assessment (HTA) bodies evaluate the clinical and/or economic impact of new therapies to inform public reimbursement decision-making. This research evaluates the value for money of current or proposed fees for HTA in countries with mandatory cost-effectiveness HTA bodies relative to their respective public drug expenditure.

HTA appraisal fees were identified from publicly-available websites: National Institute for Health and Care Excellence (NICE), Canadian Agency for Drugs and Technologies in Health (CADTH), Institut National d'Excellence en Santé et Services Sociaux (INESSS), and Pharmaceutical Benefits Advisory Committee (PBAC). Annual national public drug expenditure (ANPDE) were sourced from the National Health Service England, Canadian Institute for Health Information, and the Pharmaceutical Benefit Scheme.

NICE is proposing to charge GBP 126,000 (EUR 142,582) for a single technology or highly specialized technology appraisal, CADTH charges CAD 72,480 (EUR 48,576) for a Schedule A submission, INESSS charges CAD 38,921 (EUR 26,089) for the first evaluation of a new drug or new indication, and PBAC charges AUD 136,716 (EUR 87,576) for a Major Lodgment. The ANPDE in England: GBP 16 billion (EUR 18.1 billion), Canada: CAD 14.5 billion (EUR 9.7 billion), Quebec: CAD 4 billion (EUR 2.7 billion) and Australia: AUD 8.7 billion (EUR 5.6 billion). The appraisal cost to drug expenditure ratio for these countries/regions were: 126,984, 200,055, 102,772, and 63,636, respectively.

HTA submissions in the United Kingdom, Canada and Australia require financial contributions from manufacturers. These contributions bear little relation to the market size and cumulatively exceed EUR 300,000 (assuming no resubmissions). By adopting charging/cost recovery models, HTA bodies are aiming to reinvest the proceeds to increase the efficiency and capacity of appraisals, expediting patient access. However, these fees may be burdensome, especially for SMEs with promising therapies for orphan/rare diseases, and they may thus have the potential to deter/delay their submissions.

Health technology assessment (HTA) processes typically combine both evidence and values in order to inform decisions about relative value. Health-economic evaluation and other economic evidence are thought by many to be important for such processes, but there is typically tension between the information offered by health-economic assessment, and the context-specific interpretation of such information. This study reviews the meaning, and interpretation, of ‘health-economic evaluation’ aimed at informing HTA processes. One central aim is to answer the question: “Can health-economic evaluation provide a representation of ‘value for money’ for HTA?”

A seminal article was used as a starting point and then a variety of search techniques, including bi-directional citation searching, were used to obtain evidence relating to the study objective. A critical review is undertaken spanning the last fifty years of health-economic evaluation, which provides perspective on the balance between more context-independent assessments and the context-specific interpretation of those assessments.

Although health-economic evaluation can legitimately be undertaken in a variety of ways, we find that processes of ‘valuation’ are fundamental to all approaches to economic evaluation in practice. Values influence how these economic value frameworks tend to be operationalized, promoted and understood. Our critical review provides those interested in prioritization with a timely reminder that health-economic evaluation should be thought of as largely context- and content -specific.

Health-economic evaluation can typically only offer a truncated representation of ‘value for money’ to HTA processes. In answer to the question posed above, this study finds that health-economic evaluation will typically not provide a full assessment of ‘value for money’. Therefore, it should always be accompanied by an assessment of its qualities: what is covered in the analysis, how well what is covered is measured or analysed, and what is left out. Humility about what health-economic evaluation can offer would seem useful, especially given that other elements of value exist, such as the potential harms and benefits of medical-industry profits and environmental sustainability.

The development of health technology assessment (HTA) reports is a time-consuming process that requires highly trained human resources. In many Latin American countries this type of personnel is scarce. The adaptation of HTA could be a time-saving process to get inputs for decision. The objective of this study is to determine the frequency of use of HTA adaptation process and to describe type of tools used in this process in Latin American countries.

The Health Technology Assessment Network of the Americas (REDETSA) is a non-profit network formed by ministries of health, regulatory authorities and health technology assessment agencies (PAHO/WHO). During the last meeting of REDETSA in November 2018, we performed an exploration survey to gather information related to the topic in order to promote the creation of an adaptation working group. The question was whether HTA agencies did adaptation of HTA reports and, if so, what methods and tools were used and what sections of the report were adapted.

Thirty-three institutions from fourteen Latin American countries answered to the consultation. Seven countries do adaptation of HTA (50 percent) and one country does adoption. Of those countries that adapt HTA, three do only economic transferability. Methods and tools are usually developed locally or there is not a systematic approach. In two countries, the economic study transferability tool developed by Hutter and Antoñaza is used.

Adaptation of HTA is not well developed among Latin American agencies, although it seems to be an efficient strategy when assessing efficacy and safety. Adaptation of economic studies is still controversial; nevertheless, it is used in some of the countries of the region. It is necessary to advance in the development of HTA adaptation tools, developed and adapted to local contexts in the region.

Any technology submission for the high-cost treatment fund in Chile requires an economic evaluation; however, this is time consuming and given its high number, it is not possible to inform decisions within the established period of time. This presentation proposes a guide for the transferability of international economic evaluation results to our national context, with the intention to inform decision makers in a brief period of time.

A literature review on transferability analysis, tools and instruments to perform transferability analysis and on how to assess quality of economic evaluations was conducted. In addition, a workshop was held to discuss the proposal with other relevant researchers, in order to receive feedback.

The proposed instrument is based on Welte and consists of: (i) a research question is formulated and a systematic review of economic evaluations is conducted, (ii) the three Welte knock-out criteria are applied to these results and, if these are met, the articles pass to the next stage, (iii) a scored comparison based on twelve criteria is conducted on the articles and each article is compared against the Chilean (economic) reference case, (iv) high-scored economic evaluations will be grouped according of their incremental cost-effectiveness ratio (ICER). If all ICERs do not converge, to the same conclusion, the intervention would not be transferable. If the ICERs of these studies converge, then the results will be compared against the national threshold. If the ICERs are greater than the threshold, the intervention would not be cost-effective. If the ICERs are lower than the threshold, then the intervention would be cost-effective in Chile.

Despite a de novo analysis still being the gold standard to inform decision makers, the proposed instrument could be used as an alternative, given the short time limit and the scarcity of qualified human resources.

Hemodialysis (HD) and peritoneal dialysis (PD) are commonly used to treat patients with end-stage renal disease (ESRD). However, their costs have grown considerably in recent years as the rates of non-communicable diseases including diabetes and hypertension have grown. This will adversely impact on national health budgets especially in low- and middle-income countries (LMICs). Currently, there is limited knowledge about the costs of ESRD and the different components within the public healthcare system in South Africa. Consequently, our objective was to examine the direct medical costs of both approaches from a public provider perspective to provide future guidance.

A prospective observational study undertaken at a leading public hospital in South Africa based principally on patients’ notes and costs from nationally procured lists. A micro-costing approach was used to estimate health care costs among adult patients with ESRD who had received either HD and PD for at least 3 months.

The majority of patients (35 percent) were aged 40 to 50 years. Patients aged 29-39 years were mostly on HD (28 percent) while those between 51-59 years mostly on PD (29 percent), with HD typically managed in the private sector with limited facilities in the public sector. The average age of patients on HD and PD was 41 and 42 years respectively. Variable costs (USD 20, 488.79) were the highest cost component for PD patients with fixed costs the highest component for HD patients ((USD 16,231.45). The annual cost of HD (USD 31,993.12) was higher than PD (USD 25,282 per patient) but not statistically significant (p = 0.816). The overall burden if appreciably more patients with ESRD are managed appropriately within the public system (covering 80 percent of the population) would be considerable and become unaffordable.

HD costs more than PD. These cost estimates are useful for carrying out future health economic analyses and for allocating greater resources to prevent progress to ESRD.

The Zika virus is a newly emerging infection associated with increasingly large outbreaks especially in countries such as Brazil where an estimated 326,224 cases were confirmed between 2015 and 2018. Common symptoms associated with Zika include headache, conjunctivitis, fever, erythema, myalgia, vomiting, diarrhea, and abdominal pain. However, the symptoms are usually self-limiting and last on average for 4 to 7 days, with patients typically not accessing the public healthcare system (SUS). In severe cases, symptoms include neurological disorders and neonatal malformations. A future Zika vaccine can contribute to decreasing the number of cases and associated complications. However, this has to be balanced against continuing costs to control this and other vector borne diseases. Consequently, information about consumers’ willingness to pay (WTP) for a hypothetical Zika vaccine can help with price setting discussions in Brazil starting with the private market before being considered within SUS.

A cross-sectional study was conducted among residents in one of the main provinces of Brazil (Minas Gerais) regarding their WTP for a hypothetical Zika vaccine with agreed characteristics. This included a mean effective protection of 80 percent, with the possibility of some local and systemic side-effects. The discussed price was USD 56.41 (BRL 180.00) per vaccination as this figure was utilized in a previous WTP study for a dengue vaccine.

Five hundred and seventeen people were interviewed. However, thirty would not be vaccinated even if the vaccine was free. Most of the resultant interviewees (489) were female (58.2 percent), were employed (71.2 percent), had private health insurance (52.7 percent), had household incomes above twice the minimum wage (69.8 percent) and did not have Zika (96.9 percent). The median individual maximum WTP for this hypothetical Zika vaccine was USD 31.34 (BRL 100.00).

WTP research can contribute to decision-making about possible prices alongside other economic criteria once a Zika vaccine becomes available in Brazil alongside other programmes to control the virus.

Managed access arrangements (MAAs) represent a way of enabling patient access to promising treatments while collecting real world data to inform future health technology evaluations (HTE) and commissioning decisions. In July 2016, the National Institute for Health and Care Excellence (NICE) recommended Ataluren for treating Duchenne Muscular Dystrophy within a MAA. NICE is uniquely placed to oversee the implementation and monitoring of this MAA in collaboration with multiple stakeholders to ensure the final outputs meet the needs of a future HTE.

NICE assembled an Ataluren Managed Access Oversight Committee (MAOC) consisting of representatives from the manufacturer, patient organisations, commissioning body and treatment centres. This group were to meet every six months under the chairmanship of NICE with the primary function of reviewing the progress of data collection and identifying operational challenges in implementing the terms of the arrangement.

The Ataluren MAOC has convened four times since the MAA commenced and these discussions identified a number of important actions. Data completeness was a concern and prompted stakeholders to collaborate on implementing measures to circumvent this, to ensure data quality for future HTE. Lack of awareness and understanding of the MAA in the patient community was highlighted and resulted in the production of lay information. A review of the statistical analysis plan resulted in the need for an agreement amendment. To ensure an audit trail and appropriate critique, NICE produced an amendment process to define and justify amendments made during the agreement term.

MAOC meetings play an important role in monitoring the progress of MAAs and have ensured that implementation issues are identified promptly and resolved with input from key stakeholders. This process allows NICE to coordinate the work of stakeholders to facilitate the success of the MAA, and will be adopted in future NICE MAAs in ultra-rare diseases.

Randomized trials and similarly robust research methods generate evidence in carefully controlled settings, often with strict inclusion criteria. But patients in the ‘real world’ often have multiple comorbidities, and treatments are delivered within diverse environments. Trials are also difficult to fund, and rarely collect longitudinal data. Because of these, and other limitations, researchers are increasingly recognizing the inherent value of real world evidence (RWE). This is not only true for pharmaceutical products, and may have even more relevance in the evaluation of complex interventional procedures and non-medicines healthcare technologies. The Idea, Development, Exploration, Assessment, Learning (IDEAL) Framework guides the developmental ‘pipeline’ of surgical (and other) procedures, as well as medical device research (IDEAL-D). IDEAL informs the production of high-quality evidence of safety and effectiveness, but there is potential to further expand its applications.

Our aim is to investigate the feasibility of using of RWE alongside the IDEAL Framework in the assessment of procedures and devices. Methodological experts from the IDEAL Collaboration, HTA agencies and other healthcare research organisations are contributing their unique perspectives and experiences to explore these methods. As part of this work, Cedar Healthcare Technology Research Centre (Cedar) has attempted to retrospectively apply the IDEAL criteria to a series of RWE projects conducted on behalf of the National Institute for Health and Care Excellence (NICE) Interventional Procedures and Medical Technologies Evaluation Programmes.

Cedar's experience indicates that there may be options for using retrospective routinely-collected linked data and other existing sources to address some of the requirements of IDEAL. Likewise, the IDEAL Framework is expected to be a helpful reference when designing new databases and clinical registries for prospective collection of relevant and informative evidence. Examples from several projects will be shared at the Health Technology Assessment International (HTAi) conference.

Initial signs are that there are likely to be a number of ways in which IDEAL and RWE could complement one another.

To inform the development of a national clinical guideline for Chronic Obstructive Pulmonary Disease (COPD), prioritised by the National Clinical Effectiveness Committee (NCEC) in Ireland, a systematic review was conducted to examine the cost-effectiveness of long-acting beta2-agonists (LABAs) in combination with long-acting muscarinic antagonists (LAMAs) compared with LAMA or LABA monotherapy.

Medline, Embase, the Cochrane Library and grey literature sources were searched up to 19 June 2018. Studies evaluating cost-effectiveness published post-2008 in English were included. Screening, data extraction, and quality assessment using the Consensus Health Economic Criteria (CHEC-list) and International Society for Pharmacoeconomics (ISPOR) questionnaires were conducted independently by two reviewers. Costs were adjusted to 2017 Irish Euro using consumer price indices and purchasing power parity as per national guidelines.

From a total of 8,661 articles identified, nine studies (all cost-utility analyses) were included in the review. Studies ranged from low to high quality and compared LAMA/LABA combination therapy with LAMA monotherapy. The results reported were mixed, ranging from combination therapy being dominated by (that is, more costly and less effective than) LAMA monotherapy to being dominant (that is, less costly and more effective). However, when excluding low quality, less applicable studies, the remaining six studies reported incremental cost-effectiveness ratios (ICERs) of between EUR 2,770 and EUR 26,462 per quality-adjusted life year (QALY) gained. Only one study additionally compared LABA monotherapy as a comparator, reporting combination therapy to be even more cost-effective than in the LAMA monotherapy comparison.

Applying a cost-effectiveness willingness-to-pay threshold of EUR 45,000 per QALY gained, this systematic review found that LAMA/LABA combination therapy is cost-effective compared with LAMA or LABA monotherapy in COPD patients.

Despite availability of a cheap, widely accessible vaccine, pertussis remains an important cause of morbidity and mortality in children worldwide. A resurgence of pertussis in Brazil peaked at 8,815 cases in 2014. We estimate the economic burden of pertussis hospitalizations and outpatient cases in Brazil in 2014.

Taking the Brazilian public health system (SUS) perspective we obtained numbers of hospitalizations from the National Hospitalization Information System (SIH) for discharge diagnosis ICD10:A37 and numbers of confirmed outpatient cases from the surveillance information system (SINAN). We estimated costs per case for seven age groups (<1, 1-4, 5-9, 10-19, 20-39, 40-64, and 65+ years). Hospitalization costs were obtained from SIH, which reimburses direct medical (hospital stay, healthcare professional services, and physical therapy) and non-medical costs (parent/caregiver stay accompanying a hospitalized child). Cost of outpatient management was estimated from national guidelines (diagnostic exams, medical visits, and medications) and national pricing lists. Total economic burden was derived by multiplying costs/case by numbers of hospitalized and outpatient cases, respectively, and converted to US Dollars (USD) (December 2014: 1 BRL = USD 0.39).

A total of 8,815 pertussis cases occurred in Brazil in 2014; 55.9 percent were hospitalized. Total cost to the public health care system was USD 2.6 million, 95 percent for hospitalizations. Cost/case was highest at the extremes of age for both hospitalized <1y, BRL 1,378.54 (USD 537); 65y+, BRL 1,875.00 (USD 731) and outpatient cases BRL 41 (USD 16) for <4y and 20y + . Children <4 years accounted for 95.4 percent of hospitalizations, 51.2 percent of outpatient cases, and 95.4 percent of total costs. Children <1 year accounted for 88.1 percent of hospitalizations, 29.1 percent of outpatient cases, and 89.3 percent of total costs.

Pertussis economic burden in an outbreak year was largely due to hospitalizations in children <1y. Additional prevention strategies are required targeting this population.

The IDEAL (Idea, Development, Exploration, Assessment, Learning) Framework measures the maturity of evidence base behind surgical innovation. The NICE Interventional Procedures (IP) programme issues guidance for the United Kingdom National Health Service (NHS) on use of surgical innovation. One of four recommendations can be made: (a) standard arrangements, (b) special arrangements, (c) research only, and (d) do not use. This study aimed to investigate whether the recommendation of NICE IP guidance corresponded with the stage of innovation as determined by IDEAL, thus IDEAL's role in informing future guidance production.

A retrospective sample of 103 pieces of guidance issued between 2015 and 2018 was analysed. One researcher examined the evidence base and determined the corresponding stage of the IDEAL framework, numbered 1, 2, 2a, 3 and 4. The primary outcome measure was the association between stage of evidence on IDEAL framework and the recommendation of published NICE IP guidance.

There were twenty-one (20 percent), thirty-three (32 percent), three (3 percent), forty (39 percent) and six (6 percent) procedures at IDEAL stages 1, 2, 2a, 3 and 4, respectively. Of those at stage 1 (idea), 48 percent were given research only arrangements, 43 percent special arrangements, and 10 percent standard. Many of the procedures at stages 2 (development) and 2a (exploration) were given standard arrangements (39 percent and 67 percent respectively). Forty-three percent of stage 3 (assessment) and 67 percent of stage 4 (learning) guidance were identified standard. At stage 4 none were given a ‘research only’ recommendation.

Procedures given ‘standard’ arrangements guidance are more likely have a mature and robust evidence base as determined by IDEAL. Those with limited evidence are more likely to be given a more cautious ‘research only’ guidance. Routine use of this framework could help inform future guidance production however cannot replace the decision-making function of the NICE committee which also involves patient experiences, population characteristics, risk of serious safety events, and equity issues.

SecurAcath (Interrad Medical), a catheter securement device designed for central venous catheters, was assessed by the National Institute for Health and Care Excellence (NICE) in 2017 resulting in Medical Technology Guidance 34 (MTG34). Due to the limited number and quality of published evidence, novel methods were used to deliver a report that allowed a recommendation on adoption to be made.

KiTEC, an external assessment centre for NICE, independently evaluated the manufacturers submission of clinical and economic evidence. The submission was characterised by a lack of strong clinical evidence, comprising just one randomized clinical trial (RCT) and a small number of non-comparative observational studies, some of which were available as conference abstracts or poster presentations. KiTEC ran a meta-analysis of these studies along with data on the comparators, securement with sutures and securement with StatLock (Bard Access Systems). Due to the lack of comparative studies, KiTEC pooled data on five outcomes (migration, dislodgement, catheter-related infection, CRBSI, unplanned removals/reinsertions) and calculated relative risks for each. KiTEC revised the manufacturer's cost model, changing a number of parameters and assumptions. The decision to recommend SecurAcath for use in the National Health Service (NHS) was also supported by qualitative evidence from expert clinicians who had used the SecurAcath in practice.

KiTEC's meta-analysis showed non-inferiority for SecurAcath over the comparators. The limited information in the studies made it impossible to ascertain study heterogeneity in the meta-analysis. KiTEC's economic analyses showed that SecurAcath could be cost saving in some scenarios, but not for short indwell times (≤5 days). However, clinical expert opinion was overwhelmingly positive and this qualitative evidence was viewed alongside the less conclusive clinical and cost-effectiveness evidence. SecurAcath was recommended to be used in the NHS, with annual savings estimated to be a minimum of GBP 4.2m.

In cases where there is a lack of published evidence, unpublished material and expert clinical opinion can be used to bolster the case for the adoption of medical devices.