Health technology assessment (HTA) must adapt to support the changing health system landscapes and improve access to valuable innovation under budgetary constraints. This is exemplified by the pricing and reimbursement of high-cost combination therapies increasingly used in oncology. Variability exists in current HTA practices across different countries, resulting in discrepancies in reimbursement outcomes and patient access. Using Italy as a case study, the objective was to assess the challenges faced by HTA agencies in the negotiation of pricing and reimbursement of combination therapies.

A targeted literature review of Italian HTA agency websites was undertaken to identify any literature/guidance relating to HTA decision-making for combination oncology therapies.

In Italy, there is no fixed cost-effectiveness threshold and decisions are based on multiple criteria. Managed market entry agreements are extensively used; price-volume agreements and drug registries are common. While this framework allows flexibility and avoids the rigidity of incremental cost-effectiveness ratio thresholds, it has raised concerns about transparency and budget impact. Combination therapies are not given specific concessions; however, market access for a combination of a new high-cost drug with an existing one is complex, particularly if the drugs are manufactured by different companies. The added value provided by the new drug in the combination should be rewarded while the older product benefits from the increased volume of use. The price of the older drug cannot be lowered unless the pricing and reimbursement contract is expiring or a new indication/formulation is pending, presenting a challenge to both pharmaceutical companies and HTA agencies.

Combination therapies pose a challenge for HTA agencies. In the Italian system this is partially mitigated by the use of multiple criteria for decision-making and managed access agreements. However, these approaches have also led to concerns about a lack of transparency in decision-making.

Interventions and services for people with mental health problems can have broad remits: they are often designed to treat people with a variety of diagnoses. Furthermore, addressing mental health problems can have long-term implications for economic, social, and health outcomes. This represents a challenge for health technology assessment, for which long-term trial data can be lacking. In this review, we sought to identify how analysts have tackled this problem. We reviewed the methods used to extrapolate costs and outcomes for the purpose of economic evaluation where long-term trial data are not available.

We conducted a systematic review of the medical and economic literature evaluating long-term costs and outcomes for mental health interventions and services designed to treat or prevent more than two mental health conditions. We searched key databases including MEDLINE, Embase, PsycINFO, CINAHL, and EconLit. Two authors independently screened citations. Articles were excluded if they reported within-trial analyses or employed a time horizon of less than 5 years.

The search identified 829 unique records. No papers could be included in the review.

This review highlights the lack of research and understanding available to inform the appraisal of broad mental health interventions. In light of our findings, we consider the reasons for this lack of information and review relevant literature on the subject. Potential barriers to research in this context include: (i) challenges in understanding the value of broad mental health services, such as the mental and physical health nexus, intersectoral costs and benefits, and interpersonal impacts, (ii) methodological difficulties, such as data availability, patient heterogeneity, and the challenge of extrapolation, and (iii) parity of esteem. We make recommendations for resolving this problem with regard to funding, data collection, modelling methods, and outcome measurement.

Recently, voluminous research and commentary have touted genetic and molecular testing to improve the management of urologic cancer. The purposes of such testing include screening, risk assessment, diagnosis, prognosis, pharmacogenetics, and monitoring (for example, recurrence, predicting treatment response). An interactive graphical tool (“evidence map”) would help policy makers examine the current state of research, identify prevailing trends, and prioritize research efforts.

A professional information specialist searched MEDLINE/EMBASE for articles published in 2010 or later that primarily focused on genetic/molecular testing and either kidney or bladder/urothelial cancer. Two research analysts classified all relevant abstracts regarding to cancer type, genetic marker(s), clinical purpose(s), assay methods, publication type, and author country/region. We created an interactive map using HTML5 and JavaScript.

We identified 4,731 articles, 828 (18 percent) of which met our inclusion criteria. Our map has interactive filters which allow flexible selection of articles and automatic updating of the counts. For example, one can quickly redraw the map to focus only on U.S./European systematic reviews and meta-analyses. Research on bladder/urothelial cancer focuses on both diagnosis and prognosis, with some interest in monitoring. In kidney cancer, research on prognosis outweighs research on diagnosis. Overall, research on genetic/molecular markers is in an exploratory phase, e.g. for kidney cancer prognosis alone, 173 empirical studies considered hundreds of different markers.

Assessing prognosis is a common purpose of genetic tests for both bladder/urothelial and kidney cancer. Increased research on the monitoring of bladder/urothelial cancer may be due to its high recurrence rates, whereas lower interest in genetic tests to diagnose kidney cancer may be due to effective imaging tests. For policy makers, evidence maps can inform decisions about the scope of commissioned systematic reviews as well as the targets for recommendation statements. Interactive features allow maps to be redrawn to align with users’ specific interests.

Specialized Component of Pharmaceutical Service (SCPS) is a strategy to access high cost medicines in the National Health System (NHS) of Brazil, ensuring the completeness of medical treatment in which lines of care are defined in the Clinical Protocols and Therapeutic Guidelines (CPTG). To access the SCPS, the physician has to give to the patient a filled form, following some requirements and the CPTGs. In order to improve rational prescription and to facilitate patients’ access to medicines, we visited physicians and presented key information regarding the CPTGs of rheumatoid arthritis (RA) and the SCPS medicines request process; then, we sought to know their perceptions.

From August to September 2017, 43 specialists and general practitioners that prescribed medicines of SCPS in Minas Gerais were visited by researchers about the CPTG of RA and how to prescribe to provide easier access to these medicines to their patients. After the visits, a researcher contacted the physicians by phone to evaluate their satisfaction with the visits and about the program through a brief questionnaire.

Twenty-eight physicians answered to our phone call, providing a response to the questionnaire. Sixty-eight percent indicated they were very satisfied with the visit. Fifty percent stated that the content of the visit was relevant to their practice, and 60.7 percent said that the distributed material was going to be useful for their professional practice. Regarding the guidelines, 43 percent affirmed that the visit really helped them to improve their understanding of medicine requests in the NHS and 42.9 percent said that the visit increased their understanding of which patients are eligible for RA treatment in the SCPS; 57.1 percent of those affirmed that the visits increased their knowledge.

The physicians, who were mostly specialists, already had knowledge about CPTG and prescription practices of SCPS’s medicines, nevertheless, they showed interest in the visits to review and improve their knowledge and clinical practice.

We conducted health technology assessments (HTAs) of the interventions used between 2012 and 2014 to improve the treatment of homeless people with pulmonary tuberculosis in the Federal District of Brazil. The HTA, which was not ordered by policymakers, was based on the evidence-based national theoretical model compared with local interventions indicated in focus groups, semi-structured interviews, and secondary data produced by the Health Secretariat. The results demonstrated that the implementation of the interventions was unsatisfactory. Our objective was to present the feedback process for policymakers and the Health Secretariat, particularly its challenges.

The feedback was categorized as: (i) an executive abstract with key messages (i.e. underreporting of cases in the surveillance system, lack of primary care, and underestimation of the health problem) reported to policymakers involved in the surveillance and healthcare systems; and (ii) oral presentations in eight meetings organized by the research group and local policymakers.

Between 2016 and 2017 we conducted eight feedback meetings. All of the professionals (n = 8) involved in the Tuberculosis Surveillance and Control Program were present in at least one of the meetings, but healthcare professionals and the Secretary of Health did not participate. The barriers presented by the professionals were: (i) lack of material resources (i.e. cars and gas, phones, diagnostic tests, medications); (ii) lack of human resources (i.e. suboptimal professional staff); and (iii) feeling insecure when performing extramural activities due to the potentially unsafe work environment.

Gathering feedback on a HTA that was not ordered by policy makers can be a challenge. Mainly we demonstrated a negative result on research done in a vulnerable population with a neglected disease, in this case tuberculosis. However, this provided an opportunity for professionals in the surveillance system to discuss the challenges of implementing tuberculosis control among the homeless population.

In the fast-paced world of health technology innovation, early health technology assessment (HTA) gained recognition as a tool to help prioritize and steer the development of those innovations that potentially add value. Much of early HTA seems technology-driven; a certain novel technology is introduced and the focus is on assessing its expected cost-effectiveness. We argue that a first step in assessing innovation would be to derive proof-of-problem through combining evidence from literature and stakeholder engagement. We applied this approach to a novel surgical instrument aimed to facilitate meniscus surgery.

First, we identified a broad scope of stakeholders in meniscus surgery (i.e. meniscectomy). Through interviewing them we derived key problems in meniscectomy as-is, and determined which outcomes matter most. We used stakeholder and literature input to quantify the room for improvement in current meniscectomy. Together with stakeholders we interpreted the problem quantification and conducted an early assessment of the proposed surgical innovation. Finally, we made use of this early stakeholder engagement to uncover possible barriers and facilitators to the innovation's implementation.

While all stakeholders were enthusiastic about the innovation, there was a shared perception that there is little room for improvement in meniscectomy at present. Put differently; the innovation poses a great solution to problems that may not exist. In addition, by involving a broad range of stakeholders we were able to identify barriers and facilitators to future implementation early on, such as surgeons’ preferences.

We conclude that the innovation's value may lie with applications outside of meniscus surgery. Regarding methodology, we showed how a shift of focus from solution to problem definition provides a different perspective on an innovation's potential value, borne out of needs not currently met. In doing so, early HTA is in a unique position to help navigate the stream of health technology innovation before actual development of the innovation.

Subsequent to review by Canada's two central health technology assessment (HTA) agencies, confidential drug prices are negotiated by the pan-Canadian Pharmaceutical Alliance (pCPA) on behalf of public drug plans. This analysis is the first to examine characteristics of drugs considered for negotiation, and the duration of negotiations, from inception in 2011 to August 2017. The objectives were to identify how HTA recommendations impacted price negotiations, and in particular the role of health economics in the process.

The dataset contained 208 drug indications from the pCPA archives: those with a decision to negotiate (n=155) or a decision not to negotiate (n=53). Data were abstracted from the publicly-maintained websites of the respective agencies; descriptive statistics were conducted.

There was close but imperfect alignment between the HTA agency listing recommendation and the pCPA's decision to negotiate. The incremental cost-effectiveness ratio (ICER) of negotiated drugs (as estimated by HTA agencies) approached CAD 200,000/QALY (i.e. USD 157,000) for oncology drugs, but was closer to CAD 100,000/QALY (i.e. USD 78,000) for non-oncology drugs, revealing that negotiations would require a substantial discount to achieve conventionally ‘acceptable’ value-for-money. ICERs were influential to non-oncology drug recommendations (and were increasingly used to set pCPA negotiation targets) but did not appear to influence oncology drug HTA recommendations. The time period required to initiate negotiations was dramatically shorter for oncology versus non-oncology drugs (53 versus 263 days), and also differed markedly between therapeutic areas. The time period for pCPA activities was surprisingly similar for drugs recommended without a price condition and for those conditional on a price reduction.

These findings revealed an implicit prioritization pattern at the pCPA, as well as the evolving role of health economics in Canada's two-stage reimbursement process.

Medication delivery is one of the most common interventions in clinical practice. It requires the direct involvement of nurses and high precision infusion pumps in order to increase the infusion accuracy. Any mistake in the medication delivery process can lead to a medication error, resulting in adverse events with considerable increases in hospital length of stay and cost. Research studies should analyze this area more in emerging countries, as their realities differ from the realities of developed countries, where most of the literature of this area has been developed. This research study analyses this area in Brazil, a leading emerging country. The incorporation of these technologies in health services have caused two major problems: uncertainty around its effectiveness in reducing adverse drug event rates related to infusion dose errors, and the high cost of their inputs. The objective of this study was to analyze the cost-effectiveness of intelligent drug library infusion pumps to reduce adverse drug events during intravenous medication delivery in pediatric and neonatal patients.

Cost-effectiveness was evaluated using a decision-tree framework, considering two scenarios as the base case: the reference one, which uses conventional infusion pumps for intravenous medication delivery with a volume greater than 60 mL, and an alternative one, which uses the drug library infusion pumps. The analysis is with the Unified Health System (Brazil's publicly funded health care system) perspective. The Monte Carlo simulations addressed the uncertainties of the framework. The effectiveness measure was avoidance of adverse drug events.

The probabilistic analysis showed the drug library infusion pumps to be more cost-effective than conventional pumps. This ratified what had already been revealed by acceptance curve, which demonstrated that the drug library infusion pumps are more likely to be cost-effective compared to the conventional infusion pumps (with a minimum of the incremental cost-effectiveness of USD 1,501.28).

The study demonstrated that the use of the drug library infusion pumps in the pediatric and the neonatal intensive care unit can improve the results of the adverse drug event reduction strategy.

Many countries face the challenge of an aging population. Development of suitable technologies to support frail elderly living in care homes, sheltered housing or at home remains a concern. Technology evaluation in real-life conditions is often lacking, and randomized controlled trials of ‘pre-designed’ technologies are expensive and fail to deliver. A novel alternative would be ‘living labs’-real-life test and experimentation environments where users and producers co-create innovations and large-scale data can be collected.

The goal of the living labs and Data Driven Research and Innovation (DDRI) Programme is to use data driven analytics and insights to support technology development for independent living, healthy aging and more cost-effective care. This involves a cluster of long-term residential care facilities providing 24/7 living lab settings, linked to an embedded innovation hub. DDRI also encompasses private vehicles (e.g. sensors in cars) to enable elderly to drive safely for longer. Collaborations have been established with Universities in England, Scotland and Ireland and with international industry partners.

Several projects are underway: (i) develop machine learning algorithm from non-intrusive sensor data to build a well-being representation for individual residents/citizens; (ii) evaluate innovative interventions for good sleep environment and nutritional support; and (iii) establish ethics framework to ensure that needs of residents, families and staff are embedded in design, communication, and evaluation of future DDRI projects. In addition, fifteen interdisciplinary doctoral fellowships are in place, six universities are working closely with individual living lab settings, and an innovation hub has been established in one care home for horizon-scanning and strategic technology selection and implementation.

Over the next five years, a national network of 20 residential living labs with over 1,500 participants will be established. Generation of new user-led technologies, blueprints for capture of individual data at significant scale, and ethical and organizational guidelines will be developed. Intelligent mobility via data capture/feedback in vehicles will be established.

The use of multi-criteria decision analysis (MCDA) in health technology assessment (HTA) studies has become more common due to the fact that MCDA offers a comprehensive technique for decisions that involve multiple criteria and stakeholders. How MCDA contributes to the HTA decision making process is an issue to be investigated. A systematic review was carried out in order to provide an overview of the benefits identified in MCDA applications for the strategic HTA decision making process.

A systematic review developed by Philip Wahlster et al. (2014) was updated. The papers were analyzed in order to determine how MCDA is connected with traditional HTA, and to identify opportunities through the application of MCDA. In total 965 papers were found, and 43 articles were included in the review. The included articles detailed MCDA applications oriented to tactical and strategic decision making processes. The review was conducted by two researchers.

Of the available studies published on MCDA, 76 percent were published between 2014 and 2017, and 24 percent were published prior to 2014. Regarding the MCDA methodology defined in the included studies, 10 used the analytical hierarchy process, four used multi-attribute theory, and others refer the methodology only as “MCDA”. Seventeen studies also included health technology economic analysis, in special cost-effectiveness, safety and technological innovation. The studies suggest MCDA adds value since it allows different stakeholders to be engaged in the decision making process.

The increase in studies on MCDA and healthcare point to the possibility to add different criteria, engage people with different knowledge levels, and make the decision-making process more transparent. In comparison with other technical areas, the use of MCDA in healthcare is more focused on achieving the decision about adding the new technology, and to show how to engage stakeholders than to explain how to develop the algorithms and methodologies.

Hemophilia gene therapy trials demonstrate a “cure” could be achievable, thereby changing disease management. CoreHEM aims to develop multi-stakeholder consensus around a clearly defined, core outcome set (COS) - a minimum set of outcomes that should be measured and reported in all clinical trials of a specific condition - that will demonstrate and allow differentiation of the effectiveness and value of gene therapy relative to the current standard of care. Health technology assessment (HTA) frequently suffers from a lack of relevant, consistently reported outcomes. When uniformly implemented, COS increase the predictability and consistency of appraisals, coverage, and reimbursement decisions by payers and HTA agencies.

A COS was developed using a modified Delphi process, including online surveys and an in-person consensus meeting. A literature review and key informant interviews were used to create an initial list of outcomes for voting. Participants (patients, including representatives from the National Hemophilia Foundation and the World Federation of Hemophilia, healthcare providers, payers, HTA agencies, regulators and industry representatives) condensed and prioritized the list by rating each outcome on a scale of 1 (not important to include) to 9 (essential). Participants could also suggest outcomes for voting. Outcomes were eliminated from consideration if <70 percent rated the outcome from 7–9, unless the patient stakeholder group average score was ≥7.

After two Delphi rounds, there was consensus on three outcomes: frequency of bleeds, factor activity level, and duration of expression. Additional outcomes included after an in-person consensus meeting were chronic pain, mental health status, and utilization of the healthcare system (direct costs). Adverse events of interest were evaluated and separately reported.

Including the coreHEM COS in clinical development programs will ensure that relevant, consistent outcomes are available for decisions by HTA agencies, clinicians, and patients. This should result in faster access to novel, high-value therapies for appropriate patients.

São Paulo city in Brazil has implemented social and health care for homeless people with pulmonary tuberculosis since 2007. We conducted a health technology assessment (HTA) of the interventions provided based on a national theoretical model using 2015 data and an overview of systematic reviews. The HTA was requested by national policymakers. The results demonstrated that the interventions for pulmonary tuberculosis were satisfactory. The municipal secretariat implemented actions to improve the national treatment recommendations and adopted incentives to increase adherence to treatments. Our objective was to describe the feedback process for the Health Secretariat.

The feedback was categorized as: (i) an executive abstract with key messages (i.e. ninety-seven percent of notified cases underwent sputum smears, nineteen percent were hospitalized, and fifty-nine percent were cured) reported to policymakers involved in the surveillance program; and (ii) three meetings were organized jointly by the research group and local policymakers.

In 2016 we conducted a meeting to present the results. Thirty-nine professionals involved in the primary care team working on the streets (thirty-five percent) and the Tuberculosis Surveillance and Control Program (five percent) were present. The main barriers presented by the professionals were issues of human resources (i.e. suboptimal professional staff and having two different social organizations responsible for health care). The main facilitators presented by professionals were: (i) using homeless-peers as healthcare workers; (ii) having a network linking the primary care and surveillance programs; and (iii) periodic training.

In addition to the positive results, the HTA presented an opportunity to discuss the sustainability of incentives for adhering to treatments adopted by the policymakers, such as meal allowances and housing support, to improve social conditions among the homeless.

The dual mobility concept was invented to prevent postoperative hip dislocation. It has been used for 40 years, but demonstrative data are limited and many designs are available. In France, many implants for hip arthroplasty are reimbursed by the national health insurance system through a generic description that corresponds to a class of products having the same indications and technical features (designs, coatings, types and systems of fixation). Dual mobility cups were one of them until 2017. The ministry decided to set up the assessment of each design of dual mobility cup, marketed in France for their reimbursement.

Manufacturers of these devices submitted medico-technical application dossiers, with technical characteristics and clinical data, to the French National Authority for Health (HAS). HAS has assessed prospectively the actual benefit of these implants.

Sixteen companies marketing 42 kinds of dual mobility cups associated with 22, 28 or 32 mm femoral heads and cementless or cemented fixations have submitted dossiers. Their demands were argued on non-specific and specific clinical data. For few implants with non-specific clinical data, arguments to demonstrate the equivalence towards other implants were not accepted for reimbursement. For other implants, the specific data available were only low quality studies. No randomized controlled trials were identified. When the risk/benefit balance was acceptable, the implants were approved for reimbursement and HAS required post-launch data to assess the real-world safety of these implants (i.e. dislocation and long-term survival).

All the dual mobility cups marketed in France have been assessed by the French health technology assessment authority. Most of them have been approved for reimbursement, despite of low level of evidence. The brand name inscription enables a specific follow up and the analysis of a post-approval study results in five years.

Population growth, epidemiological and demographic transition, and a shortage of healthcare workers are affecting health care systems in Australia, Canada, the United Kingdom (UK), and the United States (US). Community paramedicine (CP) programs provide a bridge between primary care and emergency care to address the needs of patients with low acuity but lack of access to primary care. However, how to capture the key characteristics of these programs and present them in a meaningful way is still a challenge. The objective of this presentation is to identify and describe the characteristics of currently existing CP programs in the four countries to inform policy-making on CP program development in Alberta.

Information was obtained from systematic reviews, health technology assessments, general reviews, and government documents identified through a comprehensive literature search. The characteristics of the CP programs are described using a framework originally developed in Australia with three categories: (i) the primary health care model, (ii) the health integration model (in Australia, called the substitution model), and (iii) the community coordination model.

In general, Australia emphasizes rural/remote paramedics, whereas Canada, the UK, and the US implement expanded paramedic practice within different environments including rural, remote, regional, and metropolitan settings. Extended care provider programs have been intensively investigated and widely implemented in the UK. While the identified CP programs vary in terms of program components, designation of providers, skill mix, target population, and funding model, the majority of these CP programs fall under the primary health care category of the Australian framework.

Transitioning from hospital-based to community-based health care requires careful consideration of all key factors that could contribute to future program success. Delineating key components of CP programs using the Australian framework will help Alberta decision-makers design, develop, and implement appropriate CP programs that adequately address local needs.

Health state utilities measured by the generic multi-attribute utility instruments (MAUIs) differ. Empirical evidence suggests that some MAUIs are more sensitive than others in reflecting the quality of life (QoL) of patients in particular disease areas. Additionally, in order to estimate utilities based on cancer-specific health-related quality of life instruments (CSQoLs), a number of mapping functions have emerged. Although it is common practice to apply a CSQoL instead of a MAUI in clinical trials, CSQoL cannot be used to estimate utility values for economic evaluations. Mappings based on MAUIs that are not sensitive to changes in cancer patients’ QoL may result in misleading approximations of utilities that could affect allocation of resources. The study objective is to explore the validity and sensitivity of the major MAUIs to variation in the QoL measured by cancer-specific instruments. We aimed to investigate (i) the sensitivity of the general MAUIs scores to changes in the CSQoL, and (ii) whether particular dimensions of the general instrument are more sensitive.

A two stage systematic literature review is conducted. First, an update of the review done by McTaggart-Cowan et al. (2013) on the mapping methods used to determine utilities from cancer-specific instrument. Second, an analysis of studies that measure the relationship between CSQoLs and general MAUIs.

The literature suggests that differences exist between MAUIs in their capacity to capture the QoL dimensions of the CSQoLs. Additionally, the main challenge to build an appropriate mapping function for deriving utilities values from CSQoL is the definition of an appropriate methodology that (i) responds to the distribution of the selected sample and (ii) can successfully be validated in additional samples.

In the context of health technology assessment and cost effectiveness analysis, it is crucial to carefully select and report the CSQoL and MAUI involved in the estimation of the additional benefits. Policy makers need to be awarded of the sensitivity of the instruments to changes in QoL in relation to the CSQoL dimensions QoL.

Systematic reviews (SRs) are the most valid and reliable scientific evidence to evaluate the effectiveness of healthcare interventions. However, substantial resources and months are required to conduct such a review. Most hospital-based health technology assessment (HB-HTA) units don't have the time and the academic team to produce SRs. Rapid evidence assessment (REA) may represent, in this local context, an interesting avenue. The aim was to evaluate characteristics of REA and their impacts on healthcare decision making.

A SR was performed in several databases and grey literature to search data on REA including Mini-HTA and rapid reviews methodologies through March 2017. Data selection, extraction and quality assessment were performed by two independent researchers. Outcomes were about REA's methodology including question, search strategy, inclusion criteria, study selection, data extraction, quality assessment, critical appraisal and impacts on decision making.

Twelve publications on REA have been included. More similarities were found in the methodology between rapid review and SR than with Mini-HTA. Shortcuts in performing rapid reviews included evaluation scope, number of databases, gray literature websites, studies design mainly SR, reviewers number, critical appraisal and production time (3 to 6 months). Study selection and data extraction by two independent reviewers in rapid reviews were seen in thirty-four percent to thirty-eight percent and ten percent to twenty-two percent, respectively. Furthermore, assessment quality was optional. Although it is performed within a short timeframe (2 months), methodology to conduct Mini-HTA is not well defined in the literature. The scope is mainly to support decision making in the introduction of new medical devices. Impacts of REA on local health decision making process are not well documented.

Methodology to conduct REA is quite diverse. According to the data available, rapid review is a more robust methodology for HB-HTA producers than Mini-HTA. Although impacts were not well reported, rapid reviews could be more useful to support health decision making in local context.

Deprescribing – a process for reducing or stopping drugs when the balance of benefits and harms may no longer be in a person's interests – is a key aspect of managing multimorbidity and polypharmcacy in older people. Several deprescribing interventions have been developed (e.g. in Australia and Canada), although significant challenges for successful implementation remain. Through key stakeholder consultation in the care home setting in South West England, we take the initial steps to develop a context-informed deprescribing approach. Engaging stakeholders from the outset gains insight into acceptability, feasibility, and relevance of deprescribing interventions developed elsewhere informing co-production of an effective, implementable approach.

Consultation workshops were held with two groups of stakeholders: (i) care home residents and their families; (ii) care home staff and health care professionals (general practitioners, medical specialists, pharmacists, nurses, allied health professionals). Focus groups were held with each group separately to understand perspectives on: deprescribing in general; contextual considerations; and, perspectives on deprescribing interventions developed in other countries. A combined focus group then considered components of a deprescribing intervention for care homes. Qualitative data were audio recorded, transcribed, and thematically coded.

Participants described the nature of local relationships, dynamics, structures, and resources, as important considerations in the development of a deprescribing approach in care homes. Perspectives and concerns around deprescribing among the stakeholder groups varied, although the importance of eliciting local stakeholder feedback in the early stages of developing a deprescribing intervention was a common thread.

Early engagement and co-production are crucial in developing an approach to deprescribing in care homes. The combination of stakeholder involvement and qualitative research is important for developing an effective, contextually relevant intervention as the balance between interests can be incorporated into the approach. Leveraging the experience in other countries is a novel and valuable step.

Procurement is one tool for the public sector to acquire need-based, innovative and effective solutions. To succeed in purchasing services that succeed in improving patients’ outcomes and optimize cost of care, the process must be accompanied with tools for early decisional support. Documenting the effects of healthcare innovation is therefore fundamental when dealing with prioritizing adequate technology. The aim of the present study was to review the literature to identify early assessment methodology applicable to innovative procurement processes.

A scoping review was performed in January and February 2017 with the objective of selecting literature reporting on early assessment of health innovation. Methods for early assessment of health innovation were identified with the aim of investigating whether the methods change depending on where in the innovation process (development, introduction, and early diffusion) they are applied, and if the literature pointed to dominant methods. Next, critical elements of the innovative procurement process were identified, and methods relevant to the need-based phase of procurement were assessed.

In total 1064 articles met the search strategy. Based on predefined inclusion and exclusion criteria, thirty-nine articles were included in the study. When viewed in the light of innovative procurement, stakeholder insight was an important source of data in early assessment of potential benefits of health innovation. Such data can be applied in scenario analyses to provide necessary outcome overviews and to direct and accelerate the procurement process. Further, various simulation and analysis methods may be used in new ways to increase the impact of the scarce availability of data in early innovation phase.

The present review identified tools for early decisional support that address risks and step-wise healthcare management support. Information based on the present review will also be addressed in Panel 26 “Accelerating Value Based Health Care with Innovative Procurement and Early Decisional Support”

Horizon scanning (HS) is an important tool for guiding health policy formulation and the decision-making process in Brazil. In 2016, the Ministry of Health started to draft Brazilian clinical practice guidelines for transthyretin familial amyloid polyneuropathy (TTR-FAP), which is a rare disease caused by a mutation of the transthyretin gene. An initial HS report was conducted that provided information about new and emerging technologies for TTR-FAP. The HS identified five drugs that were based on two mechanisms of action: transthyretin stabilization (diflunisal, tafamidis, and tolcapone) and gene silencing (ALN-TTR02 and ISIS-TTR-Rx). At that time in Brazil there were no drugs registered for the treatment of TTR-FAP. However, a few months later tafamidis was licensed in Brazil. In early 2017 the manufacturer submitted an application to the National Committee for Health Technology Incorporation (CONITEC), with the aim of incorporating tafamidis into the Brazilian health system. As a result the HS report was updated to support the assessment by CONITEC. This study aims to show how HS is being used to support CONITEC in this issue.

As per the EuroScan toolkit, we performed a reassessment of the technologies included in the initial HS report. We searched clinical trial registers, the websites of pharmaceutical companies, conference proceedings, scientific journals, HS databases, and regulatory websites for further information. The data were synthesized and a reformulated landscape of the technological environment for TTR-FAP therapy was presented to the CONITEC Plenary.

The main difference between the initial and final HS output was that tafamidis was approved for use in Brazil, making it the only registered drug for TTR-FAP. Another difference was related to the start of a new clinical trial with diflunisal for TTR-FAP, indicating that this drug could be a potential competitor for tafamidis. It was also possible to add published positive results from a clinical trial with ISIS-TTR-Rx, which were unavailable when the first report was written. Beyond that, it appears that there are two promising gene silencers on the horizon that could represent potential competitors for tafamidis.

The analysis of tafamidis for incorporation into the Brazilian health system is ongoing, but HS was able to deliver strategic information that could affect the final recommendation of CONITEC.

Stakeholders from the innovation field in Québec (Canada) have collectively stressed the need to formalize the process for evaluating innovative technologies in the province. In the context of innovation, and more so for non-pharmaceutical technologies where the pace of development is rapid and the lifecycle short, evidence supporting the added value can be limited and uncertainties are common. Therefore, pragmatic approaches are needed to guide recommendations and to assure that the process is rigorous, transparent and fair.

Inspired by international experiences, the Institut national d'excellence en santé et services sociaux (INESSS) has developed a novel framework, where four types of recommendations are possible (introduction, refusal, limited or conditional introduction). The starting point is an evaluation of the technology's added value, for the patient, the population and the healthcare system, and the identification of uncertainties. The value of addressing uncertainty with further research is assessed, based on the value-of-information theory, and the distinct characteristics of medical devices are taken into account (e.g. learning curve effect, irrecoverable costs and incremental innovation). Those elements interact to support the formulation of recommendations by INESSS’ advisory committee.

The development of the framework was an iterative process supported by the use of the preliminary framework for the assessment of several innovative technologies. Challenges with its use were identified, and led to methodological and operational improvements. So far, the experience with the framework is positive and stakeholders confirm its relevance to support fair and reasonable recommendations for innovations.

In the rapidly changing landscape of innovation, HTA has to adapt to the challenges of assessing technologies in a context of promise and uncertainties. The framework developed by INESSS is a tool for supporting timely and fair value-based decision-making, which will benefit the healthcare system, and the patients and population it serves.