The National Oral Health Policy (NOHP) “Smiling Brazil” was launched in 2004, with the goal of reorienting the model of oral health care in the Unified Health System. Up to then, this area was impaired by limited access and curative procedures. The NOHP aims to reorganize Primary Health Care in Oral Health, expand and qualify Specialized Care and add fluoride in the public water supply. This review will bring a reflective view of NOHP evaluation.

This review work searched for evidence on the Bireme and Google Academic databases, with the keywords “Evaluation” and “National Oral Health Policy” in October 2017. The search was limited to full texts in Portuguese, English and Spanish. After reading the titles, the abstracts and finally the complete texts, the articles that did not correspond to the evaluation objective of the NOHP were excluded.

Of the 381 initial articles found, fifteen were selected for inclusion in this study. The majority reported advances in the quality and scope of oral health care with expanded access and provision of services, such as preventive actions, health education, fluoridation of the public water supply and an increase in population coverage. There was also an improvement in the main indicators, in resolution, financial investments and epidemiological surveys. Moreover, few studies showed improvement in user satisfaction. Conversely, difficulties were identified in overcoming the traditional care model, in training and professional appreciation. Challenges included the need to expand access to fluoridated water, increase coverage, build a more comprehensive care network and reduce regional disparities.

After 13 years, advances and challenges can be observed in the quality and comprehensiveness of oral health care in Brazil. There is evident improvement in indicators; however there remains a lack of access and resolution in the actions, with a large number of regional discrepancies.

Screening for psoriatic arthritis (PsA) is expected to identify patients at earlier stages of the disease. Early treatment is expected to slow disease progression and delay the need for biologic therapy. This study estimated the cost-effectiveness of screening tools for PsA in Canada.

A Markov model was built to estimate the associated costs and quality-adjusted life-years (QALYs) of screening tools for PsA in patients using topical treatment for psoriasis. The screening tools included: the Toronto Psoriatic Arthritis Screening (ToPAS) questionnaire; the Psoriasis Epidemiology Screening Tool (PEST); the Psoriatic Arthritis Screening and Evaluation (PASE) questionnaire; and the Early ARthritis for Psoriatic patients (EARP) questionnaire. Health states were defined by disability levels, as measured by the Health Assessment Questionnaire (HAQ), and state transition was modeled according to annual disease progression. Screening was assumed to be effective during a 2-year sojourn period. Incremental cost-effectiveness ratios (ICERs) were estimated based on health-state specific costs and utilities. A probabilistic analysis was undertaken to account for parameter uncertainty. All results were compared with the commonly cited cost-effectiveness threshold of CAD 50,000 (USD 37, 600) per additional QALY.

Screening with the ToPAS questionnaire resulted in cost savings compared with no screening or the EARP questionnaire, with a total cost of CAD 30,706 (USD 23,090) and 17.29 QALYs. The PEST dominated the PASE questionnaire and was more costly and more effective than the ToPAS questionnaire, with an ICER of CAD 312,398 (USD 234,909). The results were most sensitive to test sensitivity and specificity, HAQ progression, and average HAQ score at diagnosis and the start of biologic therapy. A scenario analysis tested screening efficacy for a 1-year period before diagnosis, with the ToPAS questionnaire remaining the most cost-effective option.

Screening was cost-effective compared with no screening at the commonly used cost-effectiveness threshold of CAD 50,000 (USD 37, 600). Value of information analyses will be useful for determining the need to collect further information around test accuracy parameters.

With the rapid increase in technologies and innovations to support a growing aging population in many countries, health technology assessment (HTA) of technologies for the aging populace warrants special consideration. Building on our efforts at Health Technology Assessment international (HTAi) conferences in 2016 and 2017, this presentation will highlight themes generated from two previous HTAi collaborations, with an aim of continuing to build interest and capacity in HTA for aging-related technologies in an international ecosystem that is responsive to local needs and global opportunities.

Researchers from Canada's technology and aging network (AGE-WELL) collaborated with international panelists at HTAi conferences in 2016 and 2017 to explore interest in HTA focused on aging. International panelists shared the current state of aging and HTA in their respective countries. At both sessions, opportunities were provided for participants to rate the importance of themes identified by the panelists.

At the 2016 session, the two most highly ranked themes were: (i) how HTA can help identify the unmet needs of older adults in society that could be met by technology; and (ii) engagement of older adults and caregivers. These two themes became the starting point for the panel discussion in 2017. At this session, the highest ranked themes were: (i) identification of challenges in HTA and aging; (ii) approaches to advancing the effectiveness of HTA in addressing technology and aging; and (iii) development of an aging-related interest group in HTAi.

International collaborations have identified a number of recommendations to consider for HTA and aging-related work including: developing a good mutual awareness and understanding of barriers and opportunities; the importance of co-creating solutions with patients, healthcare providers, researchers, innovators, and funders; and the identification of a suite of methods and tools that can help accelerate technological innovation in care delivery.

Health technology assessments (HTA) for combination drug therapies in oncology are increasingly common. Companies face multiple challenges when determining their economic value due to their complexity and high cost, while payers must balance the need for these vital innovations with sensitivity to rising costs. The study objective was to evaluate the current HTA frameworks in Europe and identify the potential barriers/solutions to reimbursement of brand-on-brand (BoB) combination therapy.

A targeted literature review of HTA agency websites was undertaken to identify any literature/guidance relating to HTA decision-making for combination oncology therapies in France, Germany, Sweden, and the UK.

In France and the UK, BoB HTA decisions reflect clinical- and cost-effectiveness. Combination therapies have been accepted for use in France and the UK, for example, dabrafenib plus trametinib, are assessed through standard HTA processes, exemplifying that positive reimbursement is not unattainable where there is an unmet need and high clinical value. Despite this flexibility, many therapies will fail to prove their cost-effectiveness, resulting in delays or arbitrary pricing decisions. Potential solutions are the use of the ‘efficiency frontier’, as typified by the German HTA system, giving more ‘scope‘ to expensive innovations; or the Swedish HTA approach, which applies variable cost-effectiveness thresholds according to therapeutic area, disease severity, and social criteria. Other possibilities include indication-specific pricing, multiple-criteria decision analysis, and net monetary benefit with willingness-to-trade weights. One likely issue to arise is when different companies are involved, necessitating co-operation. In this scenario, a simplistic solution would be arbitration of the division of the combined price, circumventing the need for HTA agencies to make changes to decision-making criteria.

Constructive debates and collaboration between industry and decision-makers are vital to achieve a harmonized HTA process for high-cost combination therapies which offer advanced benefits and improved safety outcomes, whilst satisfying HTA bodies and providing better access for patients.

Health services users must participate in health technology assessment (HTA) activities. Users, caregivers, and citizens have the practical experience of healthcare and social services. HTA outputs are more useful when values and preferences of patients, caregivers, and citizens are taken into account. Despite this, the best methods of stakeholders’ involvement, timing for doing so, selection of participants, and the type of users to recruit depending of methods and contexts remain unspecified. Herein, an involvement policy has been developed to formalize the participation of users, caregivers and citizens in the services offering of a regional HTA unit.

A steering committee composed of stakeholders (i.e. user, caregiver, citizen, User Experience Service representative, manager, provincial HTA body representative, HTA unit members) was constituted to discuss user involvement in a regional HTA unit. A preliminary vision statement emerged from this committee, and included objectives and principles for users, caregivers, and citizens participation. This statement was deliberated using a Delphi consensus method. Three rounds of deliberations were needed to reach a strong consensus.

Four objectives and four principles that should underlie the development of an involvement policy reached consensus. Participants agreed that users, caregivers, and citizens should: i) propose principles of involvement for each HTA projects; ii) co-realize evaluations with HTA professionals; iii) contribute to evaluation processes; and, iv) be involved in some management decisions of regional HTA units. Four principles to formalize users, caregivers and citizens’ involvement in regional HTA units also emerged. These principles were about utility and feasibility of involvement as well as ethical and methodological considerations.

Users, caregivers, and citizens must participate in the activities of regional HTA units. Each of them have different roles and can contribute to evaluation processes. Their involvement in HTA activities is warranted for co-producing better evaluation more adapted to users’ needs in healthcare and social services.

Instituto Nacional de Traumato-Ortopedia (INTO) administrates the Enoxaparin drug to prevent deep vein thrombosis (DVT) after extensive orthopedic surgeries. Nevertheless, new oral anticoagulants that offer more comfort and efficacy, but present higher risk of bleeding, have been putting in question the use of Enoxaparin. Making use of the MACBETH method, this study develops a Multicriteria Value Measurement model to evaluate such drugs.

MACBETH was applied in helping INTO to evaluate two drugs (Rivaroxaban and Enoxaparin), taking into account drug benefits and risks, through a series of interviews and decision conferences attended by INTO stakeholders that acted as evaluators in the model-building process, supported by M-MACBETH DSS (www.m-macbeth.com). Following MACBETH preference elicitation process, the evaluators were asked to make qualitative pairwise comparison judgements of difference in value between stimuli for constructing quantitative value and weighting scales. These scales allow measuring the relative value of the drugs on each evaluation criterion, separately and globally. The value measurement process was informed by a literature review and meta-analysis of randomized clinical trials with a critical appraisal of the evidence.

We report a model-structure with eight criteria, hereafter presented by decreasing order of their weighting: Death from any cause, Clinically significant bleeding, Proximal DVT, Distal DVT, Existence of antidote, Thrombocytopenia, Costs, and Comfort. From the value model developed and after performing sensitivity and robustness analyses, Rivaroxaban was considered a robust option for thrombosis prophylaxis, under the MACBETH value framework and at the light of a simple additive aggregation of those eight criteria.

This study shows how a value measurement socio-technical framework, combining MACBETH with scientific evidence within a participatory group evaluation process, can support health technology assessment in a user-friendly and effective way. MACBETH facilitates transparent and robust decision-making in the face of complex evaluation problems that the hospital often faces.

The use of long-acting insulin analogues have been reported in patients with type 1 diabetes mellitus who exhibit important oscillations of their daily blood glucose, although the therapeutic benefits are lacking. The aim of this study was to evaluate the effectiveness and safety of the insulin analogue glargine compared detemir to support health decision-making.

We performed a systematic review with meta-analysis of observational studies (cohort and registry), available in the MEDLINE (Pubmed), Latin American and Caribbean Health Sciences (LILACS), EMBASE and Cochrane Library databases (accessed August 2017), including research in the electronic journal Diabetes Care and gray literature. Several combinations of terms were used, including disease terms, interventions and type of study. The results evaluated were: glycated hemoglobin; weight gain; occurrence of severe hypoglycemia; total insulin dose; and, fasting capillary glycemia. Methodological quality was assessed using the Newcastle scale. The meta-analyses were performed in Review Manager® 5.2 software using a random effects model. Protocol number CRD42017054925 (International Prospective Register of Ongoing Systematic Reviews).

A total of 705 publications, eight cohort studies were included. The quality of included studies was classified as high. In the meta-analysis, the results for episodes of severe hypoglycemia (p = 0.002), measurements of fasting capillary glycemia (p = 0.01), and weight gain (p = 0.001) were favorable for detemir. The glycated hemoglobin endpoint (p = 0.49, heterogeneity = 89 percent) revealed high heterogeneity and no statistically significant difference between groups, showing no difference between the interventions for glycemic control.

Although some results are favorable to detemir, it was not possible to identify significant differences in effectiveness and safety between the two analogues evaluated, requiring new long term studies and better quality of methodological studies.

Index mining is a new discipline that aims to search for the composite measures or indices most relevant to the contexts or outcomes. After reviewing three frailty indices and principal component (PC)-based indices, we hereby show certain occasions that can lead to ineffective indices, which consist of bias or fail to represent the theories.

We reproduced and reviewed the three frailty indices and the 134,689 PC (principal component) -based indices from previous publications. The impact of aggregating the input variables on the final indices was analyzed using forward stepwise regression.

Several methods to combine the input variables were related to ineffective projection of information onto the indices. The most common causes leading to ineffective summation of input variables were shown in three examples involving different types of input variables, which were positively or negatively correlated or uncorrelated to the outcome. Ineffective indices were created often because of the summation of redundant information or uncorrelated variables.

The creation of ineffective indices can be avoided if the relationships between input variables and outcomes are properly scrutinized. The creation of composite measures and indices is still a discipline under active development. The three examples we identified are the mistakes that may be repeated unintentionally and need to be addressed with explicit rules. A reporting guide for the creation of composite measures has been proposed. A proper review of index objectives, data characteristics, and data limitations before creating composite measures or indices is recommended.

Dry eye disease (DED) is a common condition that significantly impacts patients’ quality of life. Previous studies have explored the impact of DED on patients’ lives qualitatively; however, patients’ preference structures have not been thoroughly explored quantitatively.

A targeted literature review and social media listening project guided design of a discussion guide for in-depth patient interviews (n=12). These, in turn, guided construction of a quantitative questionnaire administered to moderate to severe DED patients, 40 per country in Australia, Germany, United States and United Kingdom (total n=160). Patients’ preference structures were explored through an online survey using a self-explicated conjoint methodology, because of its high respondent-friendliness. Additionally, we administered the EQ5D-5L instrument to determine the health states/utilities of patients. Reaction to a hypothetical novel treatment was further obtained to check for convergent validity with the self-explicated conjoint. Finally, we asked respondents to rate the ease and relevance of the questionnaire to them.

Qualitative research uncovered important patient perspectives that were built into the quantitative survey. For example, patients seek medical advice when their symptoms are not improving. Patients’ lives are most affected by sensitivity to light, itchy and tired eyes and an inability to perform computer/screen work; however, of most concern/worrying to them is that their DED will get worse and they go blind. Results from the quantitative preference research will also be shared and its implications for future clinical trials in DED outlined. The results of the patient research and preference study are to be shared with health technology assessment (HTA) bodies and regulators through the early dialogue scientific advice process.

A process of using qualitative research to determine what matters to patients and then quantification through respondent-friendly preference research can identify outcomes that are most patient-relevant, to inform future drug development strategies.

The objective of this qualitative analysis was to explore caregiver perceptions and experiences of receiving or not receiving a genetic diagnosis following genome-wide sequencing (GWS) in children with suspected genetic disorders.

One caregiver for each child completed an online survey two weeks after enrolling in the Clinical Assessment of the Utility of Sequencing and Evaluation as a Service (CAUSES) study, beginning in January 2016, and again about six months after receiving the GWS results. The survey covered the caregivers' experiences and quality of life and children's healthcare resource utilization, and provided open-ended questions for comments. The follow-up survey was completed by twenty families who had received a diagnosis with their GWS results and by twenty-two families who had not received a diagnosis. A thematic analysis of the free-text comments from both groups was performed using NVivo 11.4.2.

Caregivers from both groups expressed similar experiences of negative socioeconomic effects of caregiving, particularly related to employment and time burden. Caregivers who did not receive a diagnosis with the GWS results were generally hopeful of receiving a diagnosis in the future and reported expectations of a positive benefit from receiving a diagnosis, both in terms of access to additional resources and of positive psychological effects. The absence of a diagnosis was a source of anxiety for many caregivers. By contrast, caregivers who had received a diagnosis reported positive, neutral, and negative psychological effects from the knowledge gained; no participants commented on the consequences for access to additional services or other socioeconomic effects.

Our findings suggest that caregivers may have high expectations for what a diagnosis can provide to them and their families, which may not be fully met once a diagnosis is obtained. The study underpins the importance of patient-centered communication of genomic testing results so that families can set realistic expectations of what having a diagnosis will achieve.

Globally, countries are investing substantially in e-health. Failures of programs to achieve valuable economic, clinical and societal outcome are increasingly reported. Unsuitable governance models may be one explanation. Research on governance models’ usefulness for realization of valuable outcomes is incomplete and scattered. Our goal is to fill this gap by producing knowledge on e-governance in Norway. Our hypotheses are: i) Co-governance and Relational Coordination will positively impact the realization of valuable outcome; and, ii) Multilateral stakeholder dialogue and collaboration, including health service delivery perspective, have been proposed to innovate health technology assessment (HTA). This will improve the relevance of HTA e-governance research.

We undertook the following: i) Systematic Review of e-governance in healthcare ii) Participatory observations, in depth interviews/focus groups iii) Document retrieval and analyses iv) Creation and support of arenas for dialogue between stakeholders on values and governance v) Analyses of co-produced value adjustments vi) Analyses of the usefulness of the Scientific Dialogue Approach for changing HTA paradigms. The study populations were: i) Governmental bodies responsible for innovation of the electronic health record (EHR) in Norway; ii) Regional and municipal authorities and management responsible for implementation of her; and, iii) The leaders of different levels at a municipal “Health House” established as a hybrid between primary and specialist health services

The project runs between January 2018–2022. Expected findings are: i) Diverging and common values; ii) Diverging governance models; iii) Diverging attitudes towards “best governance practices”; iv) Diverging levels of trust; v) Different world views, belief-systems and individual values; vi) Attitudes towards consensus building or conflict; and, vii) Experiences to feed into the discussion of stakeholder dialogue as an HTA approach.

We expect: i) To present results from the systematic review and preliminary findings from the first phases of participatory observations; ii) That results from the overall project will have high impact on the Norwegian governance models of e-health; and, iii) Publications in high impact scientific journals.

Extracorporeal circulatory membrane oxygenation (ECMO) is a technology that allows recovery of adults in cardiorespiratory failure with encouraging results, but is not available in the Brazilian universal public health system (SUS) due to high implementation costs. Time-driven activity based costing (TDABC) is applied to measure processes in an economic perspective by identifying opportunities to make processes more efficient through the reduction of resources used in each activity. The literature has explored the use of TDABC to measure costs related with clinical procedures and technologies in microcosting studies, identifying opportunities to improve the process by making it more efficient. This research measures the real costs to implement ECMO in Brazil to compare with the current public reimbursement system.

This study applied TDABC using data from 6 patients to measure costs of ECMO intervention considering the public perspective in Brazil. In sequence, standard price payed by SUS was used to estimate the current reimbursement amount received by the hospital for ECMO procedure. Cost variable analysis was conducted to understand when and how patients receiving ECMO are using hospital resources. Cost data were collected from an academic public hospital using an average of 18 months (2016–2017) for the department costs.

The real average cost was USD 128,923. Most significant resource costs was medical staff, particularly for the three survivor patients, and the ECMO equipment presented the second highest cost. ECMO activities were separated into: before implantation of ECMO, period using ECMO, intensive care post-ECMO and rehabilitation, being the period where ECMO is the most expensive, particularly in nurse and physician costs. The SUS average was USD 31,437, which shows a difference of USD 97,485 between the real ECMO cost and the public reimbursement in Brazil.

A critical element of the propagation of ECMO in Brazil and its reimbursement by public health system is the high cost and out-of-date standard payments by the Ministry of Health. Effort to implement a trustworthy method to guide decisions of SUS for the adoption and financing new technologies is essential to contribute to the optimization of public health policies in a country with a universal health system and limited resources dedicated to health sectors.

The Canadian BIA guideline was published by the Patented Medicine Prices Review Board (PMPRB) in 2007. Our initial systematic literature review of national and international BIA guidelines showed that a number of new recommendations relating to BIA model structure, input data and reporting format have been adopted in other jurisdictions such as UK, Australia, Poland, Ireland, Belgium, France and the International Society for Pharmacoeconomics and Outcomes Research (ISPOR). The main objective of the present study was to conduct a comparative review of national, international and Canadian Federal, provincial and territorial BIA guidelines and provide a list of new recommendations related to the BIA key elements which have not been discussed or included in the Canadian PMPRB BIA guidelines.

BIAs guidelines were searched in databases such as MEDLINE, EMBASE, Cochrane, and the gray literature including regulatory agency websites. An Excel-based data abstraction form was designed in order to highlight differences between recommendations related to the BIA key elements provided by PMPRB, provincial, and other national and international BIA guidelines.

Twelve guidelines were reviewed in detail. Sixty percent of the recommendations were new or were different from recommendations in the Canadian PMPRB BIA guidelines. They related to BIA key elements such as perspective, target population, costing, presenting results, data sources and handling the uncertainty.

The present literature review is the initial step towards updating the Canadian BIA guidelines. This study presents a comparative review of key elements in BIA among different guidelines and provides a list of relevant practical recommendations for the improvement of the Canadian BIA guidelines. The new methodologic advancements and recommendations that were identified are being presented to Canadian stakeholders for their opinion and feedback prior to the development of a proposed new set of Canadian guidelines.

Since 2005, new hospital examination and treatment methods (NUB) were reimbursed by hospital individual supplementary fees as long as they were not sufficiently covered by a DRG. In 2016, the NUB procedure was decisively changed by legal norm §137 h SGB.V to evaluate medical devices (MD) of high risk classes, particularly invasive, or new theoretical-scientific concepts versus treatment alternatives by the Federal Joint Committee (G-BA). Hospitals and manufacturers have to submit detailed information on the application of the MD and the scientific evidence to G-BA along with a NUB application. This assessment may lead to exclusion of the method from reimbursement by the statutory health insurance (SHI).

The published MD consultation submissions, assessments and G-BA resolutions (to date) were analyzed regarding evaluation criteria, treatment potential and study obligation.

In 2017, nineteen procedures were reviewed by G-BA with respect to §137 h. Two ultra-controlled high-intensity focused ultrasound (HIFU) indications were regarded as having potential benefit but not sufficient evidence yet, thus respective studies have to be initiated. Three procedures were regarded as eligible according to §137 h but not yet evaluated. Six procedures (ultra-controlled HIFU in five indications, targeted lung denervation in chronic obstructive pulmonary disease) were rated as having no potential benefit, while eight procedures were regarded as not eligible according to §137 h.

Initially put into place for high risk class and primarily invasive devices, consultations and assessments under §137 h show that there is some uncertainty around applicability criteria. The majority of those procedures which fell under the assessment law failed to be granted potential benefit as treatment alternative. Currently consultations are ongoing which could possibly lead to the exclusion of these methods from the performance spectrum of the SHI. Manufacturers should revise their study concepts in order to fulfill the specific demand for robust evidence.

Smartphones have been one of the success stories of the last decade. Recently apps have been used to promote, manage, and provide medical and healthcare education. Since smartphones are used to support healthcare and public health interventions, they can also provide a useful and easy method for collecting data for healthcare research. In addition, smartphone apps have been successfully used to support telemedicine and remote healthcare in developing nations. This study aimed to assess the effectiveness and analyze costs before and after the inclusion of type 2 diabetic patients in a smartphone app healthcare program.

The smartphone app healthcare program is available for Android and IOS systems, and is used to manage behavior changes and to improve patient adherence to pharmacotherapy. Patient follow up is done through a specialized telephone monitoring center made up of a physician, nurses, nutritionists, and psychologists who provide constant monitoring and guidance to patients. A retrospective study was conducted of twenty-nine patients before (year 2016) and 12 months after they were included in the smartphone app healthcare program. Data on physician visits, hospitalizations, and medical and laboratory exams were collected from medical records. The cost analysis was conducted from the private healthcare group perspective and was performed using the micro-costing method.

Ninety-eight percent of patients had reduction or maintenance of glycosylated hemoglobin levels, reaching the therapeutic goal (glycosylated hemoglobin of less than 7%). The cost analysis showed a twenty-five percent total cost saving due to a twenty-three percent reduction in the number of physician visits, a thirty-three percent decrease in hospitalizations, and a thirty-five percent cutback in medical and laboratory exams.

The smartphone app healthcare program can facilitate and improve diabetes care, especially with respect to controlling and managing the use of health resources.

High-cost innovative therapies are considered as high-risk investments in the reality of limited health care budgets. Health technology assessment (HTA) facilitates evidence-based decision making that relies on robust assessment of the clinical cost-effectiveness of the technology that is often not available for expensive cancer therapies (CT).

The objective of the study is to undertake a systematic comparison between HTA agencies worldwide in order to identify similarities and differences in the methods and processes in HTA of CT. A cross-sectional web-based survey was conducted between September 2013 and May 2015. We received responses from 161 HTA organizations based in thirty-nine countries.

HTA of CT is mainly performed by agencies in South America (38.46 percent), Australia (37.05 percent) and Europe (36.07 percent), followed by agencies in North America (20.00 percent) and Asia (16.67 percent). Logically, the agencies in high income countries produce more assessments of CT (40.23 percent), which in 34.43 percent they determine as innovative technologies compared with 10.00 percent of the units based in middle income countries and active in CT assessment (11.11 percent). We prove association (p < 0.05) between (i) the type of HTA and income per capita; the level at which the organization operates; its main activity; and the level of recommendation dissemination; (ii) the main target group and consumers of the final HTA product; the stage of evolution of the technology, on which it is likely to be assessed; and approaches to identify innovative technologies. The most active in the preparation of HTA reports are biomedical companies (50.00 percent), government agencies (42.11 percent) and professional organizations (40.00 percent). HTA bodies that assess CT distribute recommendations (37.50 percent) nationally and they are mainly addressed to private health care providers (66.67 percent).

Making coverage decisions based on HTA recommendations control the technologies introduction into the healthcare system that is why it's very important this tool to be properly adjusted to the specific needs of CT assessment.

In decision-making processes, health technology assessment (HTA) plays an important role ensuring the adoption of effective technologies and translating scientific evidence into decisions. Bambino Gesù Children's Hospital developed a new method which integrates EUnetHTA Core Model with multi-criteria decision analysis (MCDA) enabling decision makers to make a more informed decision between different alternatives. This approach quantifies assessment parameters, which are defined by literature evidence, or by expert opinion when lacking such evidence. MCDA results (i.e. decision tree of assessment elements, weighting systems and numerical values of technology’ performance) are derived from expert judgement. This means that indicators are weighed by the same weight system; either they are supported by strong literature evidence or otherwise based on expert opinion. The objective of this work is to use the GRADE approach to weight the relevance of each indicator starting from its source of information because different level of evidence should result in different weights.

A GRADE level was associated with each judgement value of performance indicators and a normal probability function was built with the standard deviation inversely proportional to GRADE level to describe the possible dispersion of the judgement due to the different levels of evidence that support each indicator. The higher the GRADE value, the lower the associated standard deviation. A Monte Carlo simulation was carried out to evaluate the expected value of technology’ performance modulated by GRADE level.

Four Gaussian distributions were built and associated to four GRADE levels. When an indicator has a low GRADE level, its performance value will vary in a broader way according to the linked Gaussian distribution.

This study showed the importance of applying the GRADE system to indicators’ sources of information because this can modify the overall computation of parameter weights and performance, proportionally to their robustness.

Health technology assessment (HTA) is a resource-intensive decision support tool that is typically structured around a set of defined domains. Sometimes urgent requests for HTAs arise that may be subject to very short timeframes, creating unique challenges. This study aimed to describe some of the options for carrying out a “hasty HTA”, and the impact of those options.

We recently completed a HTA for the Department of Health in Ireland with a strict 2-month deadline. We considered the impact of the short timeline using the project management triple constraint framework whereby the quality of a project is constrained by cost, scope, and schedule.

When delivering HTAs within short timeframes the schedule is an inflexible constraint. Providing interim advice pending a full assessment may set a precedent, or may not be possible if capital expenditure is already entailed. Additional staff should enable research to be completed faster, although economies of scale may not fully apply. Frequently such resources are not readily available. The reduction of scope through the omission of domains offers the best prospect of facilitating a short timeframe for a HTA. Scope may also be reduced through a less comprehensive analytical approach, but this creates a risk of reduced accuracy. Curtailing data collection and analysis is likely to increase uncertainty in the findings. Risk management is important when comprehensive quality assurance may not be possible.

Carrying out HTAs in short timeframes has implications for content, approach, and, potentially, quality. Agencies must consider how they can meet the needs of the decision maker without overly compromising accuracy or relevance. Due to resource constraints, the best approach is likely to be judicious changes to the scope to remove assessment elements that are unlikely to have a substantive impact on the decision.

Our research group recently evaluated a minimally invasive surgical procedure in order to inform a reimbursement decision. The application for funding was designed around the study selection criteria from a single pivotal randomized controlled trial (RCT). The aim of this study review was to evaluate the safety and effectiveness of this minimally invasive surgical procedure, and document challenges faced in evaluating a technology based on a highly targeted population.

A systematic literature search of four biomedical databases was conducted (PubMed, Embase, Cochrane library, York CRD) up to 8 August 2017. Specific elements related to the population were patient age, together with level and duration of pain. Primary effectiveness outcomes included pain, patient reported quality of life, mortality and adverse events. The included RCTs were critically appraised against the Cochrane risk of bias tool. Meta-analysis was not possible due to the limited availability of evidence with consistent outcomes.

From 4,718 search results, only one pivotal RCT specifically met the inclusion criteria, which demonstrated favorable safety and effectiveness of the procedure; however, the sample population in the trial had limited external validity to the proposed reimbursement population and follow-up was limited to six months. As a result, the selection criteria were broadened to better reflect the manner in which the service may be provided in clinical practice, and capture longer-term safety concerns. Four additional RCTs were included, which provided contradictory results.

The results of this review identified two important issues in evaluating a health technology where the assessment has been focused to the results of a single trial. In particular, the generalizability of a trial is defined by the demographic distribution of the sample, not the selection criteria. Designing the review selection criteria around the selection criteria for a single trial can have consequences for a funding decision.

With heightened awareness of the value of patient and provider perspectives to decision making, Qualitative Evidence Synthesis (QES) is increasingly used within a health technology assessment (HTA) context. Acceptability, feasibility and implementation can all be addressed by synthesis of qualitative research. Concerns have been raised about the quality of the synthesis product, especially when conducted within a constrained time window. How can we test the validity of qualitative studies and assess confidence in synthesized qualitative findings, particularly when time is tight?

A brief examination of issues relating to production and use of QES identified from within the Canary Islands HTA agency will identify practical and methodological challenges. How can existing approaches address wider patient, social, organizational and ethical considerations that inform HTA? The potential for use of Evidence To Decision frameworks and approaches such as GRADE CERQual (a transparent method for assessing the confidence of evidence from reviews of qualitative research) will be briefly examined.

This presentation will identify potential gaps between the needs of a small HTA agency and the methodological support and tools required to address these gaps, based on experience of conducting QES to date. Issues identified are particularly relevant to other small HTA agencies but are also generalizable to larger agencies and guideline producers worldwide. Pragmatic solutions are suggested. A future research agenda for potential methodological and applied research is outlined and current GRADE-CERQual development initiatives briefly shared.

Despite significant progress in developing methodologies for integrating QES within HTA decision making, substantive challenges remain. Observations derived from this small HTA agency can inform further developments across all HTA organizations. Research is required to examine the impact of potential dissemination bias, application of tools across a wider HTA decision making framework and use of rigorous approaches within a time-limited evaluation window.