The Irish, Scottish and Welsh national Health Technology Assessment (HTA) bodies (Health Information and Quality Authority, Health Technology Assessment Group, Scottish Health Technologies Group, Health Technology Wales) have recently (2018) established a ‘Celtic connections’ regional HTA alliance on non-medicine technologies. The primary purpose is to add value by realizing potential economies of scale and scope in non-medicine HTA efforts.

A Memorandum of Understanding (MoU) was agreed to: formalize collaboration and partnership working; improve shared understanding of work programs and processes; collaborate on and co-produce evidence reviews of mutual interest; increase both the volume and range of technology topics for which advice is developed in each nation; promote knowledge exchange; and enhance professional and personal development for each agency's staff.

Early benefits include: collaboration on one technology topic resulting in the production of bespoke guidance in three countries; an update of a partner's rapid review; identification of a further potential topic collaboration (sacral nerve stimulation); a six month senior staff secondment; and reciprocal observer membership on each country's national committees. Other general benefits have included: reduced duplication of effort; improved quality assurance through ‘critical friend’ peer review; enhanced access to methodological advice and a broader range of stakeholders; and development of a forum for discussion and peer support.

The alliance offers real potential to optimize use of the scarce resources for non-medicine technologies across the three countries and increase evidence review and guidance volume through adapting or co-producing outputs. Longer term benefits are anticipated to include: improved knowledge exchange; advancing skills of staff; building and broadening capacity through shared learning and access to a wider professional peer group; improved staff recruitment and retention; production of joint publications and other modes of dissemination; and increased profile for each country's work.

The Irish Health Service (HSE) Health Technology Assessment Group (HTAG) aims to maximise the impact of its work by collaborating with HSE Procurement, formalised through an evidence-based Memorandum of Understanding (MOU). This study aims to inform the MOU.

A sequential mixed-methods study design was used. A rapid review of the literature identified no substantive body of evidence on collaboration between independent national health technology assessment (HTA) and procurement bodies. Personnel involved in HTA or procurement were invited by email to complete a survey, take part in an interview, or both. The quantitative and qualitative data were analysed using descriptive statistics and thematic analysis, respectively. Findings were integrated using a conceptual framework that examined the complementarity of HTA and procurement processes relevant to an MOU.

Thirteen surveys were completed (response rate was 13 percent). Eleven interviews (five Ireland, two Canada, three UK, one New Zealand) were conducted between August and November, 2017. No formalised collaboration between independent national HTA and procurement bodies was identified. However in New Zealand, HTA and procurement are an integrated function of the Pharmaceutical Management Agency (PHARMAC). In other jurisdictions, successful ad hoc collaborations occurred where there was a clear need expressed by Procurement for additional evidence required for decision-making, and where HTA personnel tailored their research approaches accordingly. Key themes to successful collaboration were relationships, communication, clear roles, rigorous research and ‘system support’. Good individual relationships and ready access/communication promoted successful outcomes. Successful outcomes included improved clinical practice, and major cost savings. Collaboration may be focussed on: innovative or established devices; specific types of HTA/research products; specific categories/specialties; or specific procurement departments.

All participants considered collaboration to be beneficial but requiring good relationships and ‘system support’. Furthermore, successful collaboration requires clarity regarding the purpose, parties involved, their roles, responsibilities, modes of communication, information to be shared, and the expected outcomes.

Citation analysis is a standard tool for measuring the impact and influence of scientific work. One purpose behind controlled trials is to answer clinical and policy questions and to contribute directly or indirectly (contributing to systematic review and meta-analyses) to the production of practice guidance. The citation of trials within systematic reviews and policy or guidance documents therefore represents an authentic and meaningful measure of impact.

All 136 randomized controlled trials published by the United Kingdom (UK) Health Technology Assessment (HTA) programme in a 10-year period (2006-2015) were identified. Web of Science citation index was used to collect citation data relating to each trial. Altmetrics were used to identify additional policy and guidance documents. Citation data were collected and tabulated, and descriptive statistics produced. Additional data were collected for principal ‘spin-off’ publications.

Eighty-eight percent of trials were cited by at least one Cochrane or non-Cochrane systematic review or meta-analysis; 37 percent by at least one Cochrane review (90 Cochrane reviews in total); 85 percent by at least one non-Cochrane systematic review or meta-analysis (365 in total). Forty-four percent of trials were cited by at least one unique piece of published policy or guidance. Mean number of review citations per published trial: 25.30; mean number of systematic reviews/meta-analyses per trial: 3.34; mean number of guidance documents per trial: 0.85. Trial investigators published the primary clinical outcome data in 27 additional peer-reviewed journal articles, generating citations in a further 66 unique reviews and 22 unique guidance documents.

Based on the payback model, this sample of 136 UK HTA trials represent meaningful impact: 88 percent of trials were cited in systematic reviews and 44 percent in guidance documents. Chronological data indicate that there might be a sizeable time-lag between publication and impact, especially for policy documents and Cochrane reviews.

The National Centre for Pharmacoeconomics (NCPE) assesses the cost-effectiveness of new drugs for which reimbursement by the healthcare payer, the Health Service Executive (HSE), is sought in Ireland. This research aims to create a systematic approach for the NCPE review group (RG) to assess each of the cost-effectiveness models submitted by the applicant by creating cost-effectiveness model appraisal guidelines.

The RG consists of clinical, statistical and health economic expertise. In order to systematically appraise the HTA submission, which includes a cost-effectiveness model, clear guidelines on how each of the members of the RG can work together are required. The current members of the RG in the NCPE were given a draft of the guidelines created by the primary author, and additional feedback and testing was performed using the expert experience of the team. A version of the guidelines was tested for its usefulness.

Three checklists were created. The purpose of the first checklist is to evaluate if the cost-effectiveness model works correctly. The second checklist ensures that each of the assumptions included in the HTA dossier are the same as those included in the cost-effectiveness model. The final checklist validates the assumptions used in the cost-effectiveness model to ensure they are reasonable and appropriate for decision making. The final version of the checklists were validated by choosing cost-effectiveness models with known errors and/or discrepancies and testing that the issues were captured by the checklists.

These guidelines are not an exhaustive list of checks that should be performed, but are presented as the minimum requirements for consideration to be included with each RG assessment of the corresponding HTA submission. The guidelines will be constantly updated as the process evolves over time. The cost-effectiveness models should follow the National Health Information and Quality Authority (HIQA) Guidelines for the Economic Evaluation of Health Technologies in Ireland.

Health technology assessment (HTA) includes consideration of health and economic factors, playing a key role in optimizing healthcare provision in Europe. Medical devices are an important contributor to both health outcomes and the cost of healthcare provision, yet they are rarely addressed in current guidance for health-economic evaluation. Our aim is to help improve assessment of medical devices via review of European health-economic guidelines and recent research.

Searches for European HTA guidelines were performed and where available were reviewed by two researchers working independently. Additionally, a systematic review of published literature focused on assessment of medical devices was conducted. English, German, or French literature published between 2000 and 2017 was analyzed. The status of HTA guidance to date was subsequently reviewed in light of current research findings and suggestions made to help improve standardization.

Of the 41 investigated European countries, 22 had official HTA guidance. Only four of 22 (18 percent) dedicated documentation to guidance specific to medical devices. Where differences between pharmaceuticals and medical devices were highlighted, specifics for health-economic assessment of medical devices were generally absent. The systematic review yielded 472 unique articles, 28 of which underwent full-text review. Issues surrounding medical device value assessment that commonly emerged were: limited evidence base, learning curve effects, organizational impact, incremental innovation, diversity of devices, dynamic pricing, and transferability. While identification of issues was ubiquitous, actionable suggestions on how to overcome them were less common. The most frequent recommendations were use of Bayesian methods, inclusion of real-world data, and modelling the learning curve. Key to implementation is determination of the medical device type and its impact duration.

Current guidelines rarely address the needs of medical devices. Practical recommendations for improvements exist and provide opportunity to start discussion on how best to serve the medical devices field and improve the HTA process.

Clinical care pathways (CPWs) provide a step-wise multidisciplinary care plan for patients with a particular health condition. Their aim is to optimize patient outcomes and organization of care by supporting evidence-based practice. It therefore seems inevitable that health technology assessment (HTA) should be incorporated within the development process of a CPW. As CPWs become increasingly utilized, there is a need to understand the added value and strategies to integrating HTA in the development of a CPW.

Through a case study of an HTA on treatments for chronic low back pain requested as part of the development of a CPW for chronic musculoskeletal pain, we demonstrated the three key strategies to include HTA in CPWs described by Rehaluk 2016 and added a fourth one. We then showed how these strategies contribute to the development of a CPW which answers the quality criteria outlined by the Cochrane Effective Practice of Care group through a strength, weaknesses, opportunities, and threats analysis.

We confirmed four key strategies to including HTA in CPWs (organizational positioning of the HTA unit, partnership and communication with stakeholders, tailoring the integration of contextual data with evidence from the literature, explore tools to facilitate the use of HTA findings). The inclusion of HTA through these strategies contributes to the development of a CPW which meets the ten criteria to evaluate the quality of a CPW outlined by the Cochrane Effective Practice of Care group. Through a strength, weaknesses, opportunities, and threats analysis, we describe how each of the criteria were met and how this led to recommendations influencing our regional organization of care.

The inclusion of HTA in CPW development increases its capacity to directly influence organization of care. HTA can represent a pivotal vehicle to ensure good quality CPWs.

The strategic MedTech investment for the expansion of a central London paediatric hospital must sustain its ambitions to remain a state-of-the-art hospital, whilst implementing recent and future MedTech innovations and taking into account spatial and financial limitations. Horizon scanning (HS) is an important health technology assessment (HTA) tool to achieve these goals. To this end, we developed a methodology to help decide the suitability of investing in the following imaging-based MedTech: a hybrid theatre incorporating a biplane, intra-operative MRI (iMRI), multi-detector computed tomography (CT) scanners, and an EOS imaging system and predict the complementary technologies required for the decade to come. These technologies not only require adequate spatial resources but a significant upfront capital investment.

Three sources of information were used: i) a literature search, selected journals and other horizon scanning resources that examined current efficiency, safety, and cost-effectiveness for the proposed technologies, ii) expert elicitation in the form of user-group meetings and one-to-one discussions with clinical and service management teams and iii) hospital data consisting of audit and information from capital equipment bids.

With the exception of limited comparative data on iMRI (mainly including adults), little evidence exists to support investment in the proposed technologies. However, the decision of whether to adopt these technologies was influenced not only by existing evidence on the proposed technologies and associated cost but other factors such as local disease burdens, hospital staff requirements (training, expertise), space requirements for the new MedTech, and its impact on organizing healthcare services and hospital workflows. Complementary technologies associated with radiation monitoring image visualization and control were identified.

Strategic MedTech investment requires a holistic approach that assigns equal weight to information arising by expert elicitation and hospital audit data with existing literature evidence. The decision for adoption is heavily influenced by the clinical expertise and hospital workflows.

The Xpert™ Clostridium difficile kit is a nucleic acid amplification test indicated after discrepant results from an enzymatic test; was submitted for incorporation in a teaching hospital in Brazil. In order to evaluate the potential for improvement with Xpert™ incorporation, the performance of the available technology (enzymatic test) was assessed using a real word evidence approach. Additionally, the association between enzymatic test results and the agreement to the Infectious Diseases Society of America (IDSA) recommendations for stool test submission (≥ 3 unformed stools in 24 hours without laxatives) for Clostridium difficile were evaluated.

This is a retrospective cohort study conducted at a tertiary teaching hospital. We included all consecutive tested patients that were submitted for enzyme immunoassay – glutamate dehydrogenase (GDH) plus toxin detection from 15 March to 8 May 2018. Data referent to episodes of unformed stools in 24 hours and use of laxatives were recorded. Statistical significance was tested by Fisher Exact test (α = 0.05).

One hundred and thirty-eight consecutive patients were tested: 4 (2.9 percent) were positive for GDH and toxin (group III); 114 (82.6 percent) were negative for both (group I). Twenty (14.5 percent) cases were discrepant, all being positive to GDH and negative for toxin (group II). There were not negative GDH and positive toxin cases. The IDSA guidelines were followed in 33 (28.9%), 3 (15%) and 3(75%) test orders in groups I, II and III, respectively (p = 0.03).

Only a minority of patients had discrepant results in enzymatic tests and would be candidates for the Xpert™ test. The low adherence to IDSA guidelines could explain the low positivity rate of enzymatic tests at the hospital. Considering the uncertainty about the potential of the new test for changing infection control practices, Xpert™ was not recommended for incorporation. Using real world evidence data is important for contextualized health technology studies in hospitals.

The role of Health technology assessment (HTA) as a systematic approach in the evaluation of health interventions and technologies is becoming increasingly important as the quest for attaining universal health coverage globally continues to increase. Some developed countries in Europe and the Americas now apply HTA extensively in healthcare policy decisions, however, developing regions and countries like sub-Saharan Africa and Nigeria respectively, seem not to be making significant progress in this area. Given that evidence suggests that Nigeria and indeed several countries in sub-Saharan Africa are performing poorly on most healthcare indices as the region continues to be ravaged by predictable and avoidable epidemics and disease outbreaks, the need to build HTA capacity has never been more paramount.

A review of HTA capability in Nigeria was done. Pharmacists in Nigeria's Capital were randomly sampled. Semi-structured questionnaires were administered. Descriptive statistics were used in data analysis. P values less than 0.05 were considered to be significant.

In Nigeria, there is no institution tasked with undertaking HTA and there seems to be limited knowledge, capacity and awareness on the issue. Pharmacists, being the most accessible healthcare professionals according to evidence, are a key group that could play an active role in HTA and its implementation in developing countries like Nigeria. However, out of 322 pharmacists randomly sampled, 93 percent were not aware of HTA and its application in healthcare decision-making.

There is no paucity of healthcare programs and plans in Nigeria but they seem to fail due to lack of evidence-based assessment, decision-making and implementation. Hence, there is an increasing need to raise awareness on the importance of HTA in healthcare decision-making; strengthen HTA capacity by developing and sustaining institutional capacity and adequate human resource for HTA; and creating regional annexes of HTA organizations in Africa.

The European Network for Health Technology Assessment (EUnetHTA) facilitates and produces Health Technology Assessments (HTA) across Europe. Project Management (PM) provides the coordination and strategic overview of assessment production and enables the flow of scientific knowledge and assessment publications through collaboration and standardized processes, procedures and documentation.

EUnetHTA established a central PM function at the Ludwig Boltzmann Institute for HTA (LBI-HTA) for central coordination and assessment production of non-pharmaceutical technologies. LBI-HTA subsequently pursued capability and capacity through a decentralized hub-and-spoke-PM model with six activity centers (AC) providing decentralized coordination and PM of assessments. LBI-HTA provided central oversight and supervision with training days, e-meetings and ad hoc e-mail and telephone support as required. This was complemented by standardized operating procedures (SOPs) in the online Companion Guide (CG). A qualitative data collection via electronic questionnaires collected feedback from AC-PM, LBI-HTA-PM and assessment authors. Specific questions with free-text responses assessed current experiences, challenges, recommendations, communication and task distributions of the centralized and decentralized PM processes from these different perspectives.

The feedback concluded that PM is a separate, well-defined and important role for assessment coordination and production. The AC-PM received adequate training from the central PM and authors experienced no difference between projects managed centrally or decentrally. The CG and SOPs are important for guiding standard practice and allowing AC-PM to operate independently. Challenges were around extended timelines due to complex topics, external stakeholder involvement, insufficient team communication and not yet published SOPs resulting in additional central support.

Decentralized coordination of assessments, knowledge management and governance achieve scale, capacity and capability through a designated pool of agencies with established roles and growing experience in sustainable collaboration of HTA production. Valuable insight into the PM model's operational efficiency, avoidance of duplication and resource savings potentially provides a sustainable post 2020 European network policy and efficiency model for high quality HTA assessment production.

Environmental scan reports, usually consisting of literature reviews and/or key informant consultations (such as online surveys or personal interviews), broadly describe the current local, national, and international landscape surrounding health care practices, programs, or the use of technologies. Funding agencies and health organizations recognize environmental scans as a valuable way to inform decision-makers about the context, practice variations, and knowledge gaps surrounding a topic. Despite their increasing popularity in health technology assessment (HTA), there is limited guidance available for conducting environmental scans, variation in methods used across and within HTA agencies, and lack of consensus on an appropriate definition, purpose, and process.

We conducted an informal literature review and consulted experienced researchers from other HTA agencies to identify existing methods guidance for conducting environmental scans. We then adapted these methods to conduct an environmental scan of initiatives to accelerate cancer diagnosis.

There was limited and vague guidance on the definition, purpose, and process of conducting environmental scans in the context of HTA. This introduced challenges but provided the flexibility to modify our approach to meet requestor needs. Our environmental scan included: (i) a literature review, to identify and describe relevant initiatives and to locate data on effectiveness (which is often out-of-scope for environmental scans but was of priority to the requestor); (ii) stakeholder surveys, which helped “fill in the gaps” of the literature review and helped locate additional initiatives; and (iii) targeted key informant interviews, which provided rich follow-up data on the initiatives most important to the requestor.

By describing our experiences adapting limited methods guidance to meet requestor needs, we hope to contribute to the evolving discussion about the definition, purpose, and process of environmental scans to inform health policy decision-making. We will reflect on challenges encountered, potential solutions, and lessons learned, and will discuss ongoing areas of methodological uncertainty.

Resource use measurement is known to be a challenging and time-consuming, but essential step in economic evaluations of health care interventions. Measuring true quantities of resources utilized is of major importance for generating valid costing estimates. As consequence of the absence of a gold standard and of acknowledged guidelines, the choice of a measurement method is often based on practicality instead of methodological evidence. An overview of resource use measurement issues is currently lacking. Such overview could enhance clearance in the quality of resource use measurement methods in economic evaluations and may facilitate to opt for evidence based measurement methods in the future. This study aims to provide an overview of methodological evidence regarding resource use measurement issues in economic evaluations.

Literature was searched by three different methods. First, a search strategy was used in six different databases. Second, the Database of Instruments for Resource Use Measurement (DIRUM) was hand-searched. Third, experts from six different European Union countries within the field of health economics were asked to provide relevant studies. Data was analyzed according to the Resource Use Measurement Issues (RUMI-) framework, which was developed for this study.

Of the 3,478 articles provided in the initial search, 77 were fully analyzed. An overview with evidence is provided for every resource use measurement issue. Most research focused around the issue ‘how to measure’, in particular the effect of self-reported data versus administrative data. In contrast, little to no research has been done on issues ‘what to measure’ and ‘for which purpose to measure’.

Results of this study provide insight in the effect of a chosen measurement method. The results stress the importance of measuring the true quantities of resources utilized for generating valid costing estimates. Furthermore, this article highlights the lack of evidence in appropriate resource use measurement methods.

Decision-makers in low- and middle-income countries (LMICs) often must prioritize health spending without quantitative benchmarks for the value of their purchases. The Tufts Global Health Cost-Effectiveness Analysis (GH CEA) Registry (healtheconomicevaluation.org/GHCEARegistry/) is a freely-available, curated and standardized dataset designed to address this need.

All indexed English-language articles published between 1995 and 2017 are currently included in the GH CEA Registry. Studies are limited to those reporting cost-effectiveness in terms of cost per disability-adjusted life years (DALYs) averted, a commonly-employed metric in global health. Abstracted data include intervention type, comparator(s), country, funding source, study characteristics (e.g., perspective, time horizon), primary study findings, sensitivity analyses, and disaggregated data on costs and DALYs. Study quality is assessed using a numerical scoring system (from 1-7, higher scores indicating better quality) based on accuracy of findings and comprehensive reporting of methods and results.

To date, 620 articles have been included in the GH CEA Registry. Among LMICs, studies have been conducted primarily in Sub-Saharan Africa (41 percent) or South Asia (34 percent), have focused on communicable diseases (67 percent), and have involved immunization, educational, or pharmaceutical interventions (67 percent). As a priority-setting example, seven percent of interventions from higher-quality studies (ratings of 5 or higher) were reported to be cost-saving (i.e., lower costs and greater DALYs than standard care), two-thirds of which involved primary disease prevention (e.g., immunization, educational or behavioral interventions).

The GH CEA Registry is a new tool for decision-makers in LMICs, particularly those without a formal health technology assessment infrastructure but with a remit for providing access to essential, cost-effective health interventions. New functions are under development, including league tables for priority ranking, a repository for shared models, and tools for enhancing transferability between settings.

Australia has had some success at utilizing Health Technology Assessment (HTA) to disinvest and reassess medical services. This has been achieved through a range of methods including identifying services through initiatives such as ‘Choosing Wisely’, examining real world service data and seeking expert clinical opinion. This presentation will discuss how better international collaboration in disinvestment and reassessment methods using HTA could lead to more efficient health care systems.

Both the Australian and South Korean governments have a particular interest in disinvestment and reassessment in their health care systems. These countries have been sharing information over the past two years with a common goal of improving their health systems through a rigorous reassessment process. The Australian Government is in the process of reviewing all publicly funded services utilizing expert clinical committee advice, often referring the reassessment of services to a HTA process. A similar process is also being undertaken in South Korea.

Australia has disinvested in a wide range of services using HTA, including hip arthroscopy, lipectomy and hyperbaric oxygen therapy. It is also undertaking an extensive reassessment of 5,700 services. Reassessment may not lead to HTA, but it often includes an examination of whether a service should be subjected to HTA to remain publicly funded. Australia and South Korea have similar approaches in undertaking disinvestment and reassessment. HTA disinvestment and reassessment strategies have generated good outcomes for consumers, health care providers and funders in both countries.

Disinvestment and reassessment of medical services require funders that support the continual improvement of health care systems. Disinvestment and reassessment HTA can be difficult, mainly due to external interests - an issue experienced by many countries. Further international collaboration in this area may provide a more supportive environment to undertake HTA for disinvestment.

The National Institute for Health and Care Excellence (NICE) has increasingly agreed to reimburse innovative products with high levels of uncertainty as part of managed access agreements (MAAs) while additional data are collected, through the new Cancer Drugs Fund (CDF) or highly specialized technology (HST) pathways. This research aimed to review the data collection stipulations of current MAAs.

We reviewed all current MAAs entered into between NHS England and manufacturers as of 29 October 2018 and key data were extracted.

Twenty-two MAAs were identified (19 through the CDF; three through HST). All MAAs involved an observational data collection component. The source of observational data collection was existing NHS databases (19/22 MAAs: 86.5 percent), existing independent registries (one MAA: 4.5 percent [ataluren]); bespoke MAA registry maintained by manufacturer (1/22 MAA: 4.5 percent [asfotase alfa]), and registries developed as a part of regulatory approval and maintained by the manufacturer (1/22 MAA: 4.5 percent [elosulfase alfa]). Only eight MAAs (asfotase alfa, ataluren, elosulfase alfa, brentuximab vedotin, venetoclax, ibrutinib, daratumumab, and pembrolizumab) had observational data collection as the primary method of data collection. Additionally, 17/22 MAAs (77 percent; all from the CDF) also required ongoing data collection from clinical trials as a key component of the data collection arrangement.

This research identified observational data collection as a requirement in all MAAs, which is primarily through existing registries (except ataluren, which required development of a bespoke registry), while ongoing trial data collection was limited to the CDF. The relatively low cost of using existing registries to fulfil data requirements, with the ability to achieve reimbursement whilst still collecting data from ongoing RCTs, make MAAs an attractive proposition for manufacturers. NICE reportedly plan to increase use of MAAs, with ongoing NICE consultation for changes in the appraisal process potentially allowing expansion to include all indications, which would mean increased opportunities to explore innovative MAAs to support access in the future.

Bariatric surgery has become one of the fastest growing operative procedures due to its sustained results and the increasing prevalence of obesity worldwide. Despite this fact, bariatric surgery carries the usual risks and threats of surgical interventions and therefore its benefits might be undermined by its mid and long-term complications.

This retrospective study included obese patients requiring bariatric surgery from January 2004 to December 2017 provided by a private healthcare organization in Belo Horizonte, Brazil. Data regarding healthcare utilization were extracted from an administrative database (software Oracle Business Intelligence). Continuous variables were expressed as mean and standard deviation. Log-Rank test was used to adjust the survival curve (software STATA 13.1, Stata Corp, USA). This historical cohort resulted in no interventions, neither during the instituted treatment nor after the observed outcome. Privacy of subjects and the confidentiality of their personal information were handled in accordance with the ethical principles of the Declaration of Helsinki.

In total, 16,786 patients were included in the study (mean age 37.2 ± 10.2 years; female 79.2 percent; mean body mass index 42.4 ± 5.5 kg/m2). Patients were followed for up to seven years before and after surgery (total of 78,113 patients/year). For this group, the hospitalization rate was 0.099 / patients-year before versus 0.151 / patients-year after the bariatric surgery (p < 0.001). There were 224 deaths (1.33 percent) identified during the follow-up period, 0.4 percent in the first 30 postoperative days. The average costs for hospitalization were USD 3,339.36 and USD 4,305.04 for open and laparoscopic surgery, respectively.

Bariatric surgery has been an increasingly popular choice in the management of obesity. In our sample, it did not reduce the overall mid-term healthcare utilization rate.

The Department of Sciences and Technology (Decit) of the Brazilian Government has played a vital role in drafting of the National Policy for Biologic Medicines. Decit has provided methodological support to the working group, conducting a rapid review and a rapid evidence synthesis to subsidize decisions and recommendations.

We used the Methodological Guidelines for the Elaboration of Evidence Synthesis for Health Policies, which is a product of our own team, based on the SUPporting POlicy relevant Reviews and Trials (SUPPORT) Tools for evidence-informed health Policymaking.

The Decit team participated in the key steps to develop an evidence-informed policy. Our product, “Barriers to Access to Biologic Products: a Rapid Review” was used for the prioritization of health problems and the description of the problem. We then proceeded to the evidence synthesis planning and definition of the research question from an acronym. Together with the coordination of the working group, we decided to tackle the problem of interchangeability of biologic products motivated solely by economic factors in a synthesis of policy evidence. Our evidence synthesis went so far as to describe policy options. The working group used this product to inform a Policy Dialog.

This was the first time that the Decit team provided hands-on methodological assistance the development of a health policy. Not all steps recommended in the SUPPORT Tools were feasible due to time restraints. We observed that rapid evidence synthesis products were helpful to inform decision-making.

Due to the high judicialization rates which pressure the financing of biologic medicines by the Brazilian Unified Health System (Sistema Único de Saúde - SUS), it has been decided to formulate the National Policy for Biologic Medicines. After identification of problems and prioritization, interchangeability based only on economic criteria was the main problem to be confronted. The primary objective of this study was to identify political options to approach the problem of interchangeability in systematic reviews.

We conducted a rapid evidence synthesis for policy based on an adaptation of the SUPPORT tools, and searched in six literature databases. The selection of studies was performed in a systematic, transparent and independent manner. The International Network of Agencies in Health Technology Assessment (INAHTA) members were consulted to learn how this practice occurs worldwide.

We included seven systematic reviews and one policy brief, whose options to approach the problem were: production of robust scientific evidence on interchangeability; implementation of a pharmacovigilance system; appreciation of the clinical efficacy in the practice of interchangeability; and educational strategies for healthcare professionals in Brazil. Nine countries responded to our query.

Evidence-informed policy has a central role for the Brazilian Ministry of Health. The present rapid evidence synthesis for policy will subsidize decision making regarding the interchangeability of biologic medicines within the Brazilian Unified Health System.

The French health technology assessment (HTA) body, Haute Autorité de la Santé (HAS), started to provide early advice on evidence generation plans to pharmaceutical manufacturers in 2010. It became an official mission in 2016. Requests are eligible when the product has a new mechanism of action, if there is an unmet or partially met medical need in the claimed indication and when the pivotal study has not yet started. This analysis aims to provide a first overview of clinical developments for which pharmaceutical companies sought an early dialogue with HAS.

For each product that went through an early dialogue procedure with HAS, information regarding the clinical development was collected on pharmaceuticals companies’ pipelines, clinicaltrials.gov, the website of the European Medicine Agency (EMA) and HAS's internal database.

By the end of 2018, HAS has performed 84 early dialogues of which 53 were conducted in collaboration with the EMA and/or others European HTA bodies. They were mainly focused on phase III trials. Following early dialogue, the clinical study for which the company sought advice was not yet implemented in 25 cases. When the clinical trial was effectively launched, results were negative in 10 cases, positive in 11 cases and the study was still ongoing for 29 products. In nine cases, the clinical development was officially withdrawn or suspended before the initiation of the trial. Overall, only eight medicinal products were appraised by HAS, they all obtained a clinical added value score.

The success rate of clinical development for products that underwent an early dialogue procedure tends to be higher than data from literature, although it is likely to decrease in follow-up analysis. This could be partially explained by HAS's eligibility criteria that restrict early dialogues to promising products and by the scientific recommendations provided to pharmaceuticals companies.

Chimeric antigen receptor (CAR) T-cell therapy is offered as a once-only treatment for patients with certain cancers that are not responsive to standard treatment. While clinicians, patients and their families increasingly seek access to CAR T-cell therapy, there is no revenue stream to support access through public or private health systems.

The New South Wales (NSW) Ministry of Health and Victorian Department of Health and Human Services oversighted a health technology assessment (HTA) to explore the status and geography of regulatory frameworks supporting delivery of CAR T-cell therapy, evidence for the safety, efficacy and cost, clinical trials conducted or underway and manufacturing aspects.

CAR T-cell therapies are approved in the European Union and United States of America, and being considered in Australia, Canada, China and Japan. Efficacy, safety and cost-effectiveness is limited by the size and single-arm design of early stage trials and variation between them. While overall response ranges from 36–93 percent, early results for some cancers are less favorable. Durability of treatment effect is unknown, adverse events are common and can be life-threatening and risk of delayed onset toxicity remains unknown. Treatment requires access to approved manufacturing facilities (none in Australia) and specialist clinical staff.

CAR T-cell therapy is promising and demand is increasing, but the limited safety profile and evidence base should mitigate policy and investment decisions. Broader consideration should be given to developing, or identifying access to, manufacturing and clinical workforce capability and capacity to meet national demand. Australia is likely to encounter similar issues in other jurisdictions, such as limited evidence base and complex safety issues. Factors to be considered on a local and national basis for assessment and implementation include: (i) Regulatory support for industry; (ii) Strategies to manage uncertainties in long-term risks, benefits and costs; (iii) Access to accredited manufacturing facilities; (iv) Developing clinical and manufacturing workforce capability and capacity.