In 2017 the Brazilian Ministry of Health (BMH), through the Department of Science and Technology (DECIT) and in partnership with the Hospital Alemão Oswaldo Cruz (HAOC), financially supported research activities focused on health technology assessment (HTA) on topics deemed important by the BMH. The aim was to help resolve the priority health problems of the Brazilian population and to strengthen the management of the Unified Health System, within the scope of HTA.

A survey of HTA research needs was carried out in all BMH sectors through internal meetings conducted by representatives from each of the sectors. The problems and needs were then discussed, prioritized, and transformed into research lines in a workshop sponsored jointly by DECIT and the HAOC. Following this, a specific public call was made to the HTA community to comment on the prioritized research lines. The submitted research projects were then judged and selected by a committee of experts in the field. The approved projects were contracted, and when the projects were completed the results were presented and discussed by the researchers in a final seminar for representatives of the BMH technical areas.

A total of 135 research gaps were identified, of which forty-two lines of research were included in the research call after the prioritization workshop and the search for evidence in the literature. The call involved an amount of BRL one million (USD 280,442), and seventeen research projects were financed, including two systematic reviews, seven rapid reviews, and eight economic evaluations.

The promotion of research by the BMH has enabled the search for scientific evidence to support public policies and decision making in health services.

Increased capacity for utilization of Health Technology Assessment (HTA) is key to ensuring high-impact; affordable health technologies reach the people who need them most. It also enables an environment for research in access to lifesaving technologies and innovations. The number of people suffering from one or more chronic diseases is rapidly increasing and the existing systems of care are not adequately addressing this increase. Increased HTA visibility provides an opportunity for a formal connection of Health Technologies (HTs), building a new working relationship among patients and healthcare professionals, health system strengthening, engagement of patients /consumers and health care professionals for follow up on existing HTs, and assessing new Health Technology innovations.

We reviewed existing HTA literature, and hosted a meeting of fifty participants at the first HTA meeting representing different health stakeholders including patients. The meeting explored innovative synergies that addressed best practices in health policy and technological decision making, and opportunities for integrating HTA in Uganda's Health system.

Induced practical skills in coordinating a number of uncoordinated health technologies to over forty participants, an HTA strategy developed to complement national HTA initiatives and to generate information and tools to facilitate the start of hospital-based HTA initiatives in hospitals countrywide and regionally, to improve quality and efficiency of current initiatives. An HTA association, “Uganda Association of Health Technology Assessment - UAHTA” was formed, and a work plan for piloting hospital based HTA was developed.

A Health Technology Assessment (HTA) system will enable comprehensive patient-centered, integrated (as opposed to simultaneous but independent) assessment of complex health technologies. There is a need for increased capacity and partnerships towards evidence generation, and accountability measures.

Shared decision-making (SDM) is an essential component of patient-centered care, involving communication and discussions between physicians and patients on various options to meet their health needs. This study examines the current situation of patients’ participation in decision-making in relation to the clinical application of drug-eluting stents (DES). Further, the impact of patients’ involvement in decision-making on patients' adoption of DES was analyzed, with a view to providing research outcomes to guide clinical practice.

A cross-sectional study was conducted from July to December 2016 in selected hospitals in Fujian Province, Sichuan Province, and Shanghai in China. Patients with coronary heart disease completed a survey, which contained the 9-item Shared Decision-Making Questionnaire (SDM-Q-9) about satisfaction with decision-making processes, and questions on DES. Data were analyzed with cluster analysis, correlation analysis, multivariate logistic regression, and multivariate linear regression.

One hundred and seventy-nine patients with coronary heart disease from 15 hospitals in the three regions completed the questionnaire. There were good validity and reliability for SDM-Q-9, with Cronbach's alpha as 0.96 and intra-class correlations 0.59–0.79 (all P < 0.01). Among these respondents, 42.1 percent adopted DES, 83.4 percent were supportive of SDM and 61.33 percent thought they had better communication with physicians regarding decision-making. Patients’ level of SDM involvement was found to be positively associated with their satisfaction with the decision-making process (P < 0.001) and their adoption of DES (P < 0.05). Also, satisfaction with shared decision-making regarding treatment was positively associated with adoption of DES (P < 0.001).

Most of the patients with coronary heart disease preferred SDM, and SDM was found to be an important predictor of patients’ satisfaction with decision-making processes and adoption of DES. Better communication between physicians and patients is needed in order to improve patients’ satisfaction and promote the appropriate use of DES technology in China.

The NPS MedicineWise pharmacist-delivered phone service, Medicines Line, aims to provide evidence-based medicines information to consumers. We evaluated outcomes of the Medicines Line, including common consumer inquiries and resultant decision-making, and explored consumer motivations for seeking medicines information.

The evaluation involved conducting paper-based and telephone surveys of a sample of 200 Medicines Line callers, and semi-structured telephone interviews of a subset of twenty callers. Quantitative data were analyzed using SPSS software. Qualitative data were analyzed using content analysis.

Preliminary analysis found that the majority of callers thought the Medicines Line had improved their knowledge (ninety-six percent), confidence (eighty-two percent) and decision-making (eighty-nine percent). The most common reasons for calling the Medicines Line were inquiries about side effects or medicine compatibility. The medicines most commonly asked about were antidepressants (twenty percent), analgesics (thirteen percent) and antibiotics (nine percent). Questions about sertraline accounted for thirty-six percent of antidepressant inquiries. Interview themes regarding motivations for using the service included: trust; efficiency and convenience; specialized drug knowledge; and reporting adverse drug reactions to protect others from medicine-related harm. Medicines Line was perceived to be especially useful as an alternative to family physician or specialist consultations when consumers had a non-urgent inquiry about a medicine, and as a service to provide medicines information in remote communities.

These results indicate that pharmacist-delivered medicines information telephone services are an effective and efficient way of handling medicines inquiries. Medicines information telephone services are effective in improving health literacy, by increasing callers’ knowledge and confidence to source evidence-based medicines information and improving their ability to make informed decisions about medicine use. This evaluation identified knowledge gaps in medicine side effects and antidepressant use. Identifying such knowledge gaps may be useful in informing future health professional education programs, community campaigns, and shared decision-making resources.

The Canadian Agency for Drugs and Technologies in Health (CADTH) pan-Canadian Oncology Drug Review (pCODR) plays an important role in public reimbursement decision-making for oncology drugs in Canada. This research studies the relation of positive pCODR decisions to new cancer treatment and their subsequent inclusion in Canada's public drug plans.

We studied all oncology drugs that received an approval from Health Canada and were reviewed by the pCODR from inception till 26th Sep, 2017. The data was obtained from CADTH and Health Canada. Data such as indication, submission type and date, recommendation date, final recommendation, and subsequent provincial funding status was extracted and analyzed. Impact was evaluated by analyzing the percentage of drug submissions with assessment outcome (positive recommendation rate and conditional recommendation rate) and time taken for the final decision (recommendation gap). The percentage of drugs included in public formulary after positive recommendation by pCODR (coverage rate) and the gap in days from positive recommendation to subsequent coverage in provinces (coverage gap) was also assessed.

Among 119 drugs reviewed by pCODR, the positive recommendation rate was eight percent. Nine applications comprising seven drugs for six indications received positive recommendations, and genitourinary treatments received maximum positive recommendations. The conditional recommendation rate was 52 percent; 62 applications of 45 drugs for 46 indications received conditional recommendation. Lymphoma and myeloma treatments received maximum conditional recommendations. The average recommendation gap for positive and conditional recommendations was 180 and 172 days, respectively. The coverage rate for drugs with positive recommendation was 100 percent for all provinces except 89 percent for Newfoundland and Labrador, and 67 percent for Prince Edward Island. Among the provinces, British Columbia had a maximum of 433 days and Saskatchewan has the minimum of 165 days coverage gap.

Despite Health Canada's approval, only a fraction of oncology drugs receive positive pCODR recommendation; furthermore, provincial drug plans take time to include these in the reimbursement formularies. While health technology assessment is crucial for appropriate allocation of limited resources, efforts should also be made to reduce access barriers, particularly to positively recommended oncology drugs inclusion in provincial formularies.

High quality evidence for test accuracy can be scarce. We assessed the test accuracy of two tests (Actim Partus and PartoSure) for the prediction of preterm birth. Twenty published full-text papers were included whilst conference abstracts were excluded. Since systematic reviews of diagnostic tests on other topics may need to rely on data from conference abstracts, we test whether the findings of our review would change with conference abstracts included.

Conference citations previously excluded (n=108) were re-screened for inclusion using the following criteria: i) the diagnostic test was Actim Partus or PartoSure ii) test accuracy data of preterm delivery within seven days was reported iii) the population was women with signs/symptoms of preterm labor with intact membranes. Relevant test accuracy data were extracted and used to calculate sensitivity and specificity. Pooled sensitivity and specificity for each test were run using data from full-text papers and conference abstracts combined. These values were compared with the pooled sensitivities and specificities produced for the systematic review using full-text papers only.

Preliminary pooled sensitivities of the sixteen full-text Actim Partus studies and sixteen full-texts and two abstracts were 0.77 (95% confidence interval (CI) 0.68, 0.83) and 0.76 (95% CI 0.69, 0.83) respectively whilst pooled specificities were 0.81 (95% CI 0.76, 0.85).and 0.80 (95% CI 0.75, 0.84) respectively. Preliminary, pooled sensitivities of the four full-text PartoSure studies and four full-texts and three abstracts were 0.83 (95% CI 0.61, 0.94) and 0.82 (95% CI 0.65, 0.92), respectively, whilst pooled specificities were 0.95 (95% CI 0.89, 0.98) and 0.96 (95% CI 0.94, 0.97), respectively.

Our findings suggest that the test accuracy results would not alter substantially with the inclusion of conference abstracts. However, work is ongoing to investigate how the assessment of heterogeneity and risk of bias across studies would alter given the difficulties associated with limited methodological reporting from conference abstracts.

The reliability of health technology assessment (HTA) is built on accessing evidence systematically to inform conclusions and recommendations; however, the availability of primary evidence is a source of bias which can undermine an HTA. This omission is often because attempts to generate primary evidence have not been completely successful. Where partial evidence exists, ignoring it constitutes avoidable bias. Taking the Hip Op trial as an example (a study of developmental dysplasia of the hip (DDH)) we consider how despite lack of quantitative outcomes data, rich information was obtained that should inform HTA in this area.

The Hip Op trial was an open label trial comparing early against late surgery in the management of DDH. In parallel, a qualitative study attempted to explore the experience of parents of children with DDH.

The trial protocol called for recruitment of 636 children, but due to changes in clinician equipoise and service configuration only 29 could be recruited. The trial was stopped early. While baseline data for the 29 children was available, no estimate of effect was attempted due to a lack of outcome data; however, the qualitative data was rich, representing the biggest qualitative sample worldwide on this topic. It reflected the patient experience, and shows a clear preference towards early intervention, despite the absence of quantitative evidence.

The qualitative work here gives a clear indication that parents have a strong preference. This is data which would not be captured in traditional HTA reports, which tend to focus on quantitative data and meta-analysis. This is, however, information that is important to patients, and should inform clinicians and payers. We discuss how HTA do-ers should make efforts to find this data from ‘failed’ primary research and incorporate it into their reports, and how HTA do-ers could be alert to this situation.

Treatment options for hemorrhoidal disease (HD) include conservative treatment (e.g. laxatives), rubber band ligation, and more invasive surgical treatment options. Outcomes reported in clinical trials evaluating treatment effectiveness are heterogeneous, making comparisons difficult. Moreover, clinical outcomes, such as recurrence, complications and symptoms, do not fully represent the relevant benefits and harms of treatment to the patient. We therefore developed (i) a core outcome set (COS) for HD treatment, and (ii) a patient-reported outcome measure (PROM) evaluating symptoms and impact on daily life.

Literature review established outcomes most commonly used in studies evaluating HD treatment. A Delphi study with health professionals and patients was conducted to rank and discuss the outcomes in terms of importance and completeness, and reach consensus on a COS. In addition, individual patient interviews (n=15) were held to gain insight into patient experiences with HD and treatment. A panel of experts subsequently developed a PROM that focused on the core outcomes. Face and content validity were assessed (n=10) using a retrospective verbal probing technique.

Recurrent symptoms, complications and treatment satisfaction were the primary focus for health professionals, while patients were more concerned with overall impact on daily life. Patients ranked blood loss, pain and itching as the most bothersome symptoms. A PROM was developed, consisting of seven items covering three domains: severity of symptoms, impact on daily life, and treatment satisfaction (if applicable). The questions and response options were clear to patients and content validity was good. The questionnaire took approximately three minutes to complete.

We developed a COS and a PROM for HD treatment. The PROM can be used in clinical trials as the primary outcome measure evaluating treatment effectiveness from the patient's perspective. It can also support shared decision-making regarding individual treatment pathways in clinical practice. A psychometric validation study is currently underway.

Conceptual models (CMs) are useful tools for researchers and health technology assessment bodies to understand the interplay among environmental characteristics (e.g., health care system), patient characteristics, health behaviors, and patient outcomes. The objective of this pilot study was to elicit perspectives of patients with atrial fibrillation (AF) and health care providers (HCPs) to develop a patient-centered CM of the AF patient experience in a US-based sample.

We developed two preliminary versions of the Andersen model of healthcare utilization (standard and patient-friendly versions) based on the published literature and the help of a patient advisor. For example, instead of describing “predisposing characteristics,” the patient-friendly CM describes, “what is it about me, or other afib patients that could impact disease or outcomes;” “enabling resources” is swapped for “helpful resources,” and “perceived need” is changed to “what impacts whether I believe I need to be treated”. Five patients from an online patient community and 10 HCPs from the University of Maryland Medical System provided feedback on the preliminary models. Audio recordings of interviews were transcribed verbatim, analyzed, and findings incorporated into a revised CM.

Interviewee additions under “what impacts whether I believe I need to be treated” included: absence of symptoms and fear of experiencing an AF episode; under “helpful resources” suggested additions include resources for navigating insurer formulary/benefits. Suggested additional outcomes of interest include anxiety, bruising, and shortness-of-breath. While patients found the patient-friendly version easy to understand, HCPs required explanation of standard-version headers, for example ‘predisposing characteristics’ and ‘enabling resources’, which had been adapted in the patient-friendly version.

Soliciting input from stakeholders ensures CMs are pragmatic, reflect the real-world experiences of patients and HCPs, and incorporate variables or other considerations not currently described in published literature. Researchers can utilize CMs to aid in selection of variables for observational studies.

Clinical practice guidelines (CPGs) are an ideal implementation mechanism for promoting effective clinical practice, but without due consideration of costs they may do more harm than good and become a source of inefficiency. The Alberta Guideline Adaptation Program sought current best practice for incorporating economic information into CPGs to better leverage health technology assessment (HTA) and health economic expertise in its guideline development program.

A comprehensive, systematic review of published and grey literature was undertaken to: (i) catalogue theoretical frameworks and practical methods for incorporating economic information into CPGs and forecasting the post-implementation economic impact of CPGs; (ii) summarize current methods for evaluating the economic impact of CPGs; and, (iii) identify barriers and facilitators to incorporating economic information into CPGs.

Rigorous economic analyses were infrequently incorporated in CPG development. While a selection of guidance documents and CPG manuals published between 2001 and 2017 by leading CPG developers emphasized the health economist's role and the importance of incorporating economic evidence into CPGs, few provided adequate guidance on the best way to do this. There is no agreement on how best to monitor the economic impact of CPGs. Analysis of a sample of over 100 studies published between 2005 and 2013 identified the three main methods currently used to assess the post-implementation economic impact of CPGs: pre-test/post-test cost analyses, mapping studies, and modelled cost-effectiveness studies. The key elements of each study type were summarized and compared.

The review highlighted the under-recognized know-do gap among developers with respect to using health economics information and expertise in CPG development. It identified the advantages and potential limitations of applying health economics to CPG development, as well as areas where developers can better utilize HTA researchers and health economists to improve the quality of guidelines and better document the resource implications and feasibility of the interventions they recommend.

In March 2017, CADTH released the fourth edition of the Guidelines for the Economic Evaluations of Health Technologies. As part of the update a few notable changes were made to topics such as discount rate, target population, modeling, effectiveness, analysis, and the theoretical foundations for the Guidelines. In this presentation we will describe: the implementation of the Guidelines; approaches taken to facilitate the adoption of the Guideline statements; and the tools provided to assist users and doers in using cost-effectiveness information in healthcare decision making.

Given some of the changes made to the Guidelines, CADTH identified the need to engage stakeholders early in preparation for the release of the fourth edition. Feedback on topics was sought from various stakeholders (researchers in the field, industry, patient groups, and decision makers) throughout the process. Also, suggestions for tools to support the understanding and implementation of the Guidelines were noted by CADTH. To further support use of the Guidelines, CADTH committed to undertake a number of activities including: workshops for decision makers and researchers; worked examples to illustrate the approaches; and development of tools to assist in the use of recommended methods. Updates to align drug submission guidance with the Guidelines are ongoing.

The final version of the Guidelines was greatly influenced by the stakeholder feedback received, with a focus on greater clarity. Whilst efforts to increase acceptance and adoption of the guidelines are ongoing, we present preliminary findings with respect to engagement with stakeholders and adoption of new guidance in drug submissions.

The plan to engage stakeholders continues to be effective. As such, there has been general acceptance of the changes and an interest in education and tools to assist with implementation of the Guidelines.

Early detection of primary hepatocellular carcinoma (PHC) patients with cirrhosis is critical to enhance PHC patients’ survival rates and to save medical costs. The study aimed to generate real world evidence to support the importance for early detection of PHC patients, and this evidence will contribute to a cost effectiveness analysis of the national liver cancer surveillance program.

A retrospective analysis was performed on 98,275 PHC patients with cirrhosis in the National Center Cancer Registry from 2005 to 2014, linked to the Korea National Health Insurance claims database. The hazard ratio (HR) of mortality within five years and medical costs for the patients were compared by surveillance, epidemiology, and end results (SEER) stage.

There were differences in survival rates and medical costs depending on their characteristics including sex, age at diagnosis, SEER stage and types of initial treatment of cancer. The HR of mortality within five years of the PHC patients with distant stage versus local stage was 3.36 with 95% Confidence Interval (95% CI: 3.33–3.38) which is higher than those of the patients with regional stage (HR 1.93, 95% CI: 1.92–1.95). The estimated annual medical cost was USD 38,208 with standard deviation (SD) 54,399 for localized stage but USD 16,345 (SD 42,377) for distant stage.

If PHC patients with cirrhosis were detected at early stage, their survival rates would be clinically better with a big saving for medical costs than if they were detected at distant stage. This result itself highlights that importance of the national liver cancer surveillance program. Future studies are indicated to apply these quantitative results into the cost-effectiveness analysis of the Korean national liver cancer surveillance program.

The Philippines has an increasing number of newly diagnosed cases of human immunodeficiency virus and acquired immune deficiency syndrome (HIV/AIDS). Most Filipinos rely on out-of-pocket (OOP) expenditure to finance their healthcare needs. In 2010, the Philippine National Health Insurance Corporation (PhilHealth) introduced an Outpatient HIV/AIDS Treatment (OHAT) package to cover the necessary basic healthcare expenses of patients. The objective of this study was to review the OHAT package in terms of patients’ financial risk protection, specifically the amount of OOP expenses incurred and the package's support value.

The study was divided into two phases: (i) patient surveys (PS); and (ii) facility costing surveys (FCS). PS focused on information from enrolled and non-enrolled patients, specifically their current financial needs and expenses. The FCS reviewed actual cost breakdown for each treatment hub of package inclusions.

The calculated maximum support value of the package in 2015 was 267 percent. The median annual patient OOP expenditure was PHP 4,700 (USD 91). Maximum expenditure reached as high as PHP 392,000 (USD 7,551) per year mostly due to treatment for opportunistic infections (OIs), which are currently not included in the package. High OOP expenditure was also due to non-uniform coverage of services across different hubs; there was no consensus among providers on what specifically should be included in the package. This reflected a variety of package support values, with some hubs falling below patient expenditure.

The current OHAT package, if properly implemented, is sufficient to cover the basic yearly healthcare needs of patients. However, non-uniform implementation and variation in prices of services per treatment hub means that coverage is not always sufficient in all areas, which can cause continued high OOP expenses for patients even with insurance coverage. Furthermore, coverage of OI's as the main driver of increased OOP expenses should be explored.

Getting technologies adopted in the UK healthcare system can be time-consuming and complex. The National Institute for Health and Care Excellence Office for Market Access (NICE OMA) has developed a novel approach to enable greater and more coordinated dialogue between life sciences companies and healthcare system stakeholders on market access issues.

When establishing NICE OMA, interactions were carried out with life sciences trade associations and key healthcare system stakeholders to explore challenges in market access landscape. Feedback highlighted that dialogue with NICE and other stakeholders is often limited and occurs in high-risk situations; indicating a need for greater and more coordinated dialogue between industry and multiple healthcare system stakeholders outside of formal processes.

The approach developed is a safe harbor engagement framework which enables NICE OMA to facilitate interaction between life sciences companies and key healthcare system stakeholders; this collaborative approach promotes shared understanding of aspects that will allow innovative technologies to reach patients faster. It brings together multiple organizations in a safe environment where ideas can be exchanged between participants, allowing organizations to think beyond their own area of interest and to work collaboratively. Companies have used the engagement framework flexibly to engage at different stages along the development to adoption journey. Feedback indicates that companies have benefitted from channeling discussions through NICE to bring together key leaders from different organizations, as well as the neutral facilitation of discussions. Healthcare system partners have gained insights/knowledge that hadn't been apparent beforehand. Patient and clinical representatives have appreciated the opportunity to provide views to a broad range of stakeholders often early in the development of the technology.

The NICE OMA safe harbor engagement framework has been well-received to date. Further feedback will be sought to understand the impact in helping to optimize the market access journey.

A recent article reported a high level of commonality across European Health Technology Assessment bodies’ (HTABs) positions in former parallel scientific advice procedure. Since 2017, the EUnetHTA joint action 3 (JA3) offers a new early dialogue process involving a higher number of European HTABs. The present analysis aims to describe if the JA3 process modified the level of agreement across HTABs.

A descriptive analysis of the written recommendations provided during every JA3 early dialogues coordinated by the French National Authority for Health (HAS) until November 2017 was conducted. The level of commonality for each HTAB position identified was assessed globally and by domain (population, comparator, outcomes, study design and health economics) and classified as follows: “full agreement” if all HTABs had the same position, “partial agreement” if more than half HTABs had the same position and “disagreement” in all other cases.

Four JA3 early dialogues were performed until November 2017: two in oncology, one in neurology and one in metabolic disorders. Between five and nine HTABs from eleven European countries participated. A total of forty-six positions were identified in these four early dialogues: ten on population, five on comparator, fifteen on outcomes, four on study design and twelve on health economics. Of the forty-six positions, full agreement was reached for twenty-eight positions, partial agreement for seventeen positions and only one disagreement was observed. The level of full agreements was highest for questions on comparators (five out of five) and population (nine out of ten) and lower for questions on health economics (six out of 12).

Although the JA3 process substantially increased the number of HTABs participating in the early dialogues, this descriptive analysis suggests that the level of agreement remains very high. This may be facilitated by the high level of dialogue and coordination between HTAB ensured by the EUnetHTA process.

The National Institute for Health and Care Excellence Medtech Innovation Briefings (MIBs) are commissioned by the National Health Service (NHS) England and designed to support NHS and social care commissioners and staff who are considering using new medical devices and other medical, digital or diagnostic technologies. MIBs are fast flexible summaries of single technologies that are intended to be responsive to commissioners needs for information about innovative technologies. MIBs include a description of the technology, how it is used and its potential role in the treatment pathway. They also include a review of relevant published evidence and likely costs. As a relatively new product, the format of MIBs continues to evolve and in 2016 a more streamlined evaluation template was introduced. To ensure MIBs continue to meet users’ needs, a study was conducted to understand the opinions and requirements of core stakeholders and to identify key areas for future development.

An initial cross-sectional online survey with NHS staff who were potential users of MIBs was carried out in December 2015. A second round of online and mail-out surveys were circulated between November 2016 and May 2017 to medical technology manufactures and an additional group of NHS staff. Descriptive analysis was used for all quantitative data and qualitative data was summarized using thematic analysis.

Thirty-nine medical professionals and fourty-two manufacturer representatives participated in the surveys. More than half of clinicians were aware of MIBs and thought that raising awareness and visibility should be a future priority. Manufactures regarded MIBs as having a positive or mixed impact on innovation, access, or uptake by the healthcare system.

Stakeholders are using MIBs in a variety of ways and there was and a range of suggestions for their future development particularly regarding moving from single technology evaluation to simultaneous assessment of similar technologies.

The Romanian healthcare system has been struggling to use a more transparent approach in evaluating health care technologies for more than 10 years. No systemic and satisfactory approach to evaluate health technologies was implemented until the present. The objective of the presentation is to describe the characteristics of the HTA system used by the Romanian healthcare authority as well as the consequences of the drug assessments by using the actual Romanian health technology assessment (HTA) evaluation framework, from the initiation in May 2014 to the end of year 2017.

The drug reimbursement context and the healthcare legislation regarding HTA evaluation were studied. A critical appraisal of the scorecard was conducted, taking into consideration general principles of the health technology assessment. A descriptive analysis covering the assessment drug reports issued by the National Agency for Drug and Medical Devices (NADMD) issued between May 2014 and December 2017 was presented, together with the decision made by the Ministry of Health and the Romanian government.

During the analyzed period of time, more than 10 updates of the reimbursement list were implemented by the Ministry of Health. By November 2017, more than 180 drugs (new INN, new indications or fix dose combinations) were included in the reimbursement system with conditional or unconditional reimbursement; more than 230 reports were assessed by the NADMD. While the new drugs reimbursed between May 2014 and November 2017, in the most part demonstrated cost savings, a lot of new innovative drugs proposed to be evaluated were rejected since the drugs had no comparators on the Romanian market and their costs were considered to have a negative impact on the healthcare budget.

The rapid HTA assessment has many strengths, by using a proper scorecard. Limitations and weakness of the actual scorecard were identified, mainly regarding the lack of a basic budget impact analysis which must include at least the direct healthcare cost, as well as the imported results of different healthcare environments that are not matching the Romanian context. Opportunities to implement a more rapid and accurate HTA evaluation are identified since the scorecard could be updated in order to address the HTA general principles.

The National Committee for Health Technology Incorporation (CONITEC) has a structured process for the incorporation, disinvestment, or alteration of different health technologies in the Brazilian public health system and provides technical support for the decision-making process. Since its creation, CONITEC has received several submissions for the incorporation of medicines and the update of clinical practice guidelines for multiple sclerosis (MS). Nowadays, more than twelve different therapies are currently available to treat MS and the Brazilian clinical practice guideline, which was last updated in 2015, offers six medicines to treat MS that are divided into first, second, and third line treatments. The purpose of this study was to describe CONITEC's assessments of applications for incorporation, disinvestment, or alteration of medicines for MS.

A case study method was used to evaluate information, retrieved from CONITEC's database, about the health technology reports developed by CONITEC's Executive Secretariat in response to applications received in the period from 2012 to 2017.

Ten technical reports on health technologies for MS were produced by CONITEC during the study period. This number represented four percent of the external submissions for incorporation of technologies for several clinical conditions in the public health system. Six medicines were evaluated. The highest number of submissions were for incorporation (n = 6), followed by alteration of treatment lines (n = 3), and disinvestment (n = 1); fifty percent of the submissions were not recommended. The main reasons for rejection were low or unproven efficacy, high budget impact, and inadequacy of the proposal based on the evidence presented. CONITEC's favorable recommendations caused a profound change in the current clinical practice guideline and had a significant impact on the health system.

MS is considered a rare disease in Brazil, but there is significant pressure from society to provide better treatment options that will impact the MS scenario in the health system. The recent CONITEC assessments have led to a revolution in the treatment of MS in Brazil, which is now in the process of being updated.

A statewide health technology assessment (HTA) program was implemented to increase equity of access and support robust assessment of technologies, with a focus on those that are high-cost, high-risk, or have state-wide impact.

Local hospital networks and clinicians refer technologies to the South Australia Policy Advisory Committee on Technology (SAPACT) for assessment. Independently produced, comprehensive HTA reports are developed using internationally recognized evidence and critical appraisal methodologies. Clinical and economic systematic analyses are utilized, with extensive clinical consultation, to develop recommendations for new technologies and their role in models of care. Feasibility of adoption and local implementation are considered, including existing service delivery and appropriate training and credentialing. For approved technologies, SAPACT may also develop audit criteria and seek implementation reports on clinical outcomes.

The HTA framework has been successfully adopted across South Australia Health, increasing the incorporation of evidence-based decision making in the use of high-cost and high-risk health technologies. Over 35 evidence evaluations for high-risk and high-cost health technologies have been conducted for a broad range of treatment interventions. SAPACT develops and utilizes HTA decision-making criteria for transparency of Committee considerations. The program recommends adoption or rejection of technologies, or it may request a re-submission due to safety concerns or a lack of proven effectiveness. SAPACT has also granted temporary approval through adoption under clinical evaluation to inform investment decisions. A key component is working with clinicians to define specific treatment criteria and patient selection. SAPACT continues to strengthen relationships with all stakeholders, increase patient input through the development of public summary documents for technologies, and improve monitoring and reporting of clinical outcomes.

The HTA program has been very productive and positively received. The success of the program is underpinned by its engagement with clinicians, hospital networks, and consumers. The completion of SAPACT HTA reviews and the publication of the SAPACT decision-making criteria have increased the credibility of decisions, supporting enhancements in patient care and cost efficiency for the state government.

We examined relationships between measures of total knee arthroplasty (TKA) “appropriateness” constructs and surgeon TKA recommendations in people with knee osteoarthritis (OA). Although TKA is highly effective, fifteen to thirty percent of recipients report dissatisfaction and/or little or no symptom improvement. More appropriate selection of surgical candidates may improve both patient outcomes and healthcare resource use, but no validated appropriateness criteria exist currently in Canada.

Patients 30 years of age or older with knee OA referred for surgical consultation at two large joint arthroplasty centres in Alberta, Canada were invited to participate. Participants completed a standardized pre-consult questionnaire, which included the following sociodemographics and validated measures of appropriateness constructs for TKA: knee symptoms; non-surgical management; patient readiness for and expectations of TKA; and net patient benefit. Post-consultation, surgeons were asked to confirm knee OA and their recommendation. We used multivariable logistic regression to examine the relationship between measures of appropriateness constructs and receipt of surgeon TKA recommendation.

Of 3,009 patients approached, 2,360 completed the questionnaire and 2,064 (sixty-nine percent) were eligible at surgical consultation (mean age 65.7 years, standard deviation 9.1; fifty-nine percent were women); 1,495 (seventy-two percent) were recommended for TKA. The likelihood of receiving a TKA recommendation was independently associated with: knee symptoms (odds ratio [OR] per unit increase in pain intensity, 1.19 (95% confidence interval [CI]: 1.11–1.27)); prior non-surgical OA management (OR for prior knee injection, 1.53 (95% CI: 1.21–1.94)); readiness for surgery (OR if definitely/probably willing to undergo TKA, 3.03 (95% CI: 1.99–4.59)); and TKA expectations (OR outcome “very important”: ability to perform daily activities, 1.40 (95% CI: 1.04–1.88); straighten the knee/leg 1.42 (95% CI: 1.13–1.80); participate in exercise/sports 0.75 (95% CI: 0.58–0.98)).

In our cohort of patients with confirmed knee OA who consulted a surgeon for TKA, appropriateness constructs were significantly associated with receipt of a TKA recommendation. Research is ongoing to evaluate the predictive validity of these measures for patient-reported outcomes associated with TKA.