Health Technology Assessment Guidance Research Articles

PCR295 Patient Experience in Health Technology Assessment (HTA): Landscape Assessment and Comparison of HTA Guidance on Patient Reported Outcome Measures (PROMs), With a Review of HTA Appraisals in Multiple Myeloma (MM)

PCR295 Patient Experience in Health Technology Assessment (HTA): Landscape Assessment and Comparison of HTA Guidance on Patient Reported Outcome Measures (PROMs), With a Review of HTA Appraisals in Multiple Myeloma (MM)

Effectively Leveraging RWD for External Controls: A Systematic Literature Review of Regulatory and HTA Decisions.

Real-world data (RWD)-derived external controls can be used to contextualize efficacy findings for investigational therapies evaluated in uncontrolled trials. As the number of submissions to regulatory and health technology assessment (HTA) bodies using external controls rises, and in light of recent regulatory and HTA guidance on the appropriate use of RWD, there is a need to address the operational and methodological challenges impeding the quality of real-world evidence (RWE) generation and the consistency in evaluation of RWE across agencies. This systematic review summarizes publicly available information on the use of external controls to contextualize outcomes from uncontrolled trials for all indications from January 1, 2015, through August 20, 2021, that were submitted to the European Medicines Agency, the US Food and Drug Administration, and/or select major HTA bodies (National Institute for Health and Care Excellence (NICE), Haute Autorité de Santé (HAS), Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG), and Gemeinsamer Bundesausschuss (G-BA)). By systematically reviewing submissions to regulatory and HTA bodies in the context of recent guidance, this study provides quantitative and qualitative insights into how external control design and analytic choices may be viewed by different agencies in practice. The primary operational and methodological aspects identified for discussion include, but are not limited to, engagement of regulators and HTA bodies, approaches to handling missing data (a component of data quality), and selection of real-world endpoints. Continued collaboration and guidance to address these and other aspects will inform and assist stakeholders attempting to generate evidence using external controls.

PCR96 Benefits of ePRO Measurements (EPROM) in Clinical Trials and Implications from HTA Perspective

Evidence of electronic patient-reported outcomes (ePROs) has been a significant innovation in PRO endpoint assessment in clinical trials. Yet, the health technology assessment (HTA) agencies’ perceptions of the use of ePRO measurement during clinical trials and implications for the benefit assessments are unknown. We aim to synthesize the advantages of using ePRO in clinical trials and to identify key HTA guidance that may inform future clinical trial design.

Health technology assessment of diagnostic tests: a state of the art review of methods guidance from international organizations.

To identify which international health technology assessment (HTA) agencies are undertaking evaluations of medical tests, summarize commonalities and differences in methodological approach, and highlight examples of good practice. A methodological review incorporating: systematic identification of HTA guidance documents mentioning evaluation of tests; identification of key contributing organizations and abstraction of approaches to all essential HTA steps; summary of similarities and differences between organizations; and identification of important emergent themes which define the current state of the art and frontiers where further development is needed. Seven key organizations were identified from 216 screened. The main themes were: elucidation of claims of test benefits; attitude to direct and indirect evidence of clinical effectiveness (including evidence linkage); searching; quality assessment; and health economic evaluation. With the exception of dealing with test accuracy data, approaches were largely based on general approaches to HTA with few test-specific modifications. Elucidation of test claims and attitude to direct and indirect evidence are where we identified the biggest dissimilarities in approach. There is consensus on some aspects of HTA of tests, such as dealing with test accuracy, and examples of good practice which HTA organizations new to test evaluation can emulate. The focus on test accuracy contrasts with universal acknowledgment that it is not a sufficient evidence base for test evaluation. There are frontiers where methodological development is urgently required, notably integrating direct and indirect evidence and standardizing approaches to evidence linkage.

HTA56 Is Evidence on Environmental Impact Included in Health Technology Assessment and Does It Influence Decision-Making?

The global environmental impact of healthcare is high. National governments have implemented measures to reduce emissions in the healthcare sector, including the NHS in the UK, as outlined in its Long-Term Plan, 2019. When assessing new technologies, health care decision makers have the opportunity to examine their environmental impact during Health Technology Assessment (HTA). This study assessed if environmental impacts were included in HTA frameworks or guidance from a selection of HTA agencies and whether it influenced decision-making.

PP206 Health Technology Assessment Guidance In The United Kingdom: Addressing Issues Specific To Medical Devices

IntroductionThe United Kingdom spends approximately GBP4.2 billion (USD5.6 billion; EUR4.7 billion) each year on medical devices, but healthcare providers receive little health technology assessment (HTA) guidance on cost-effective device procurement. Our objective was to assess the availability of HTA guidance for medical technologies and to identify key challenges related to the economic assessment of these technologies.MethodsNational Institute for Health and Care Excellence technology appraisal (TA) and Medical Technologies Evaluation Programme (MTEP) appraisals published online between November 2009 and October 2020 were identified. The “case for adoption” recommendation, type of devices, and critiques of economic analyses for each MTEP appraisal were extracted and categorized.ResultsIn comparison to 415 publicly available TAs for pharmaceuticals, only 45 medical technologies have been appraised through the MTEP. MTEP-submitted technologies can be categorized into diagnostic (7), monitoring (3), prophylaxis (5), therapeutic (28), and other (2). Furthermore, 11 were implants, seven were used by patients, and 27 had provider interaction. Major points of MTEP criticism were a failure to model cost consequences, training costs, and organizational impact. There was also the barrier of transferring costs across budgeting divisions.ConclusionsIn comparison to HTA guidance for pharmaceuticals, there is a dearth of medical device guidance. Therapeutic and implantable devices appear to be disproportionately overrepresented in the MTEP process. This may be because their appraisal is most akin to pharmaceuticals, for which HTA processes are well established. To encourage more HTAs of medical devices, HTA guidance should elaborate on issues specifically related to medical devices.

PP452 Impact Of Patient Access Schemes On Health Technology Assessment Agency Guidance For Rare Diseases In England and Scotland

IntroductionPatient access schemes (PAS) are agreements that may enable patients to access drugs or other treatments that may not be cost effective under normal circumstances. The aim of this study was to determine whether the use of PAS by the National Institute for Health and Care Excellence (NICE) and Scottish Medicines Consortium (SMC) for recommended drugs can lead to greater access to medications for rare diseases.MethodsReimbursement data for rare diseases between 2004 and 2021 from health technology assessment (HTA) agencies, namely the SMC (Scotland) and NICE (England), were included. The reviews with positive HTA decisions were considered, while those with negative decisions were excluded. Several observations were made from these data and reported.ResultsAmong the total positive reviews (n = 81), 43 included PAS. The inclusion of PAS in manufacturer submissions was more frequent for NICE than for the SMC (79% and 40% percent, respectively). Most of the drugs with PAS were included in the HTA guidance from both agencies. The positive NICE reviews contingent on PAS consisted of 20 drugs. For the same set of drugs, the SMC recommended 14 with PAS and one without PAS; five drugs were not assessed. Adalimumab was recommended by NICE with a PAS (base-case incremental cost-effectiveness ratio of GBP12,336 [EUR14,256]; GBP13,676 [EUR15,804]) and by the SMC without a PAS (base-case incremental cost-effectiveness ratio of GBP22,519 [EUR26,023]). Hence, without a PAS, the drug was costlier per quality-adjusted life-year for the National Health Service (NHS) Scotland.ConclusionsPAS submissions for rare diseases are more frequent for NICE than for the SMC. With the PAS discounts, the overall cost of the drugs is reduced, resulting in cost effectiveness. The SMC approved some drugs for which NICE required a PAS to improve the economic argument. Hence, the use of PAS for these drugs could lead to potential cost-savings to the NHS Scotland.

PP612 Use of Rapid Review Methods In Health Technology Assessment For Central Procurement Decisions: Experience In Ukraine

IntroductionThe Health Technology Assessment (HTA) Department of the “State Expert Center of the Ministry of Health of Ukraine” was established in 2019 for conducting assessments and development of recommendations for informing decisions of the Ukrainian Ministry of Health regarding financing of the health technologies. Since 2015, procurement of medicines by the state budget in Ukraine was carried out through international specialized organizations. The central procurement program covers forty-one programs for different disease areas. The list of medicines and medical devices purchased were based of procurement agreements with specialized organizations approved by Cabinet of Ministers Decree of Ukraine № 255 of 13 March 2019.MethodsDescriptive analysis of methodological processes around rapid review methods in HTA used for the procedure of central procurements of medicines. In the process of conducting rapid assessment we use a modern tool based on the HTA Core Model® for Rapid Relative Effectiveness Assessments version (V3.0). HTA Guidance in Ukraine was developed in 2017.ResultsThe list of government programs, for which rapid review methods in HTAhave been conducted, included following diseases: adult and pediatric oncology, juvenile rheumatoid arthritis, hemophilia, and orphan diseases. Reports include an overview of comparative efficacy, effectiveness and safety, as well as analysis of reports from HTA agencies and thresholds, budget impact for informing decisions for development of the central procurement programs in 2019. The HTA Department conducted more than forty reviews in less than 6 months.ConclusionsUse of rapid review methods in HTA in Ukraine for informing decisions for the central procurement programs of medicines is the perspective for rational resource allocation and spending. This increases and improves patients’ access to effective, safe and cost-effective medicines.

Aligning text mining and machine learning algorithms with best practices for study selection in systematic literature reviews

BackgroundDespite existing research on text mining and machine learning for title and abstract screening, the role of machine learning within systematic literature reviews (SLRs) for health technology assessment (HTA) remains unclear given lack of extensive testing and of guidance from HTA agencies. We sought to address two knowledge gaps: to extend ML algorithms to provide a reason for exclusion—to align with current practices—and to determine optimal parameter settings for feature-set generation and ML algorithms.MethodsWe used abstract and full-text selection data from five large SLRs (n = 3089 to 12,769 abstracts) across a variety of disease areas. Each SLR was split into training and test sets. We developed a multi-step algorithm to categorize each citation into the following categories: included; excluded for each PICOS criterion; or unclassified. We used a bag-of-words approach for feature-set generation and compared machine learning algorithms using support vector machines (SVMs), naïve Bayes (NB), and bagged classification and regression trees (CART) for classification. We also compared alternative training set strategies: using full data versus downsampling (i.e., reducing excludes to balance includes/excludes because machine learning algorithms perform better with balanced data), and using inclusion/exclusion decisions from abstract versus full-text screening. Performance comparisons were in terms of specificity, sensitivity, accuracy, and matching the reason for exclusion.ResultsThe best-fitting model (optimized sensitivity and specificity) was based on the SVM algorithm using training data based on full-text decisions, downsampling, and excluding words occurring fewer than five times. The sensitivity and specificity of this model ranged from 94 to 100%, and 54 to 89%, respectively, across the five SLRs. On average, 75% of excluded citations were excluded with a reason and 83% of these citations matched the reviewers’ original reason for exclusion. Sensitivity significantly improved when both downsampling and abstract decisions were used.ConclusionsML algorithms can improve the efficiency of the SLR process and the proposed algorithms could reduce the workload of a second reviewer by identifying exclusions with a relevant PICOS reason, thus aligning with HTA guidance. Downsampling can be used to improve study selection, and improvements using full-text exclusions have implications for a learn-as-you-go approach.

PNS145 Access to Medicines in the United Kingdom: A Review of HTA Decisions for New Medicines

To examine Health Technology Assessment (HTA) decisions for new innovative medicines in the UK (England, Scotland, Wales, Northern Ireland). The study examined HTA decisions made between 2017 and 2020 across UK countries for innovative medicines licensed by European Medicines Agency (EMA) in 2017. The analysis was done using published documents. The decisions were classified as “recommend without restrictions,” “recommend with restrictions,” or “not recommended”. EMA approved licenses for 56 new innovative medicines in 2017. From the date of licensing the medicines in 2017 until January 2020, the National Institute for Health and Care Excellence (NICE) in England had completed the appraisal of 28 out of the 56 medicines. Out of these 28, 12 were “recommended without restrictions” 14 were “recommended with restrictions” and 2 were “not recommended”. Majority of the NICE recommendations (i.e. 21 of 26 or 81%) were done through managed access agreements. The Scottish Medicines Consortium (SMC) has assessed 31 out of the 56 medicines licensed in 2017. Out of these 31, 8 were, “recommended without any restriction”, 22 were “recommended with restriction” and 1 was “not recommended”. AWMSG in Wales follows NICE guidance and does not review a medicine if NICE is considering assessing it in the next 12 months. Northern Ireland follows HTA guidance issued by NICE or SMC and adopts a decision shortly after either has made its decision. In Northern Ireland, 37 recommendations were adopted from either NICE or SMC. These 37 include: 10 without restrictions, 21 with restrictions and 6 not recommended decisions. Adoption of HTA recommendations is low and it varies across UK countries. The number of HTA assessments resulting in “not recommended” decision was found consistently low across all UK countries. However, the number of “recommendation with restriction” is relatively high compared to the “recommendation without restrictions”.

Critical Review of European Health-Economic Guidelines for the Health Technology Assessment of Medical Devices.

Background: Health-technology assessment (HTA) is a recognized mechanism to determine the relative benefits of innovative medical technologies. One aspect is their health-economic impact. While the process and methodology for pharmaceuticals is well-established, guidance for medical devices is sparse.Aim: To provide an overview of the health-economic aspect in current European HTA guidelines concerning medical devices and identifying issues raised and potential improvements proposed in recent literature.Methodology: Available guidelines by European agencies were each reviewed and summarized. To complement this, a full systematic review of current literature concerning potential improvements to existing HTA practices for medical devices, from PubMed and EMBASE, was conducted; the focus was on health economics. Authors could only review documents in English, French, or German. The systematic review yielded 518 unique articles concerning HTA for medical devices, 32 of which were considered for full-text review after screening of all abstracts.Results: There is very limited consensus in—and mostly a complete lack of—guidance specific to medical devices in official HTA guidelines, for both clinical and economic analyses. Twenty two of 41 European countries had published official HTA guidance in English, French, or German. Among these only 4 (England, France, the Netherlands, and Sweden) dedicated a chapter or separate document to medical devices. In the literature, there is sufficient evidence to suggest medical devices need to be addressed separately from pharmaceuticals. However, mostly challenges are discussed rather than implementable solutions offered. We present the following set of frequently discussed issues and summarize any solutions that pertain to them: a weak evidence base, learning-curve effects, organizational impact, incremental innovation, diversity of devices, dynamic pricing, and transferability. We further combine reviewed information to suggest a set of possible best practices for health-economic assessment of medical devices.Conclusion: For greater efficiency in medical-device innovation, European agencies should look to (re-)address the specific requirements of medical devices in their HTA guidelines. When both the health-economic and data requirements for the HTA of medical devices are defined, the development of practical solutions will likely follow.

Beyond cost-effectiveness: A five-step framework for appraising the value of health technologies in Asia-Pacific.

Given resource constraints and the potential for increasingly high-cost, cost-effective medicines to become available, policymakers require strategies that go beyond cost-effectiveness when making resource allocation decisions. This manuscript presents a five-step framework that complements traditional health technology assessment (HTA) guidance documents that policymakers in Asia-Pacific and elsewhere may consider when setting up HTA guidelines and/or evaluating whether or not to subsidize a medicine or other health innovations. The framework recommends that subsidy decisions be based on five criteria: the relative burden of the condition as compared with other conditions (step 1), comparative and cost-effectiveness of the medicine (steps 2 and 3), the short-term impact on the budget (step 4), and other considerations including patient and societal preferences (step 5). Our approach, which is a complement to traditional HTA guidance documents, is not prescriptive but provides an evidence-based framework that HTA agencies in Asia-Pacific can follow as they aim to deliver value-based medicines to their constituents.

PP148 A Stakeholder-Informed Strategy For Effective Communication

IntroductionEffective communication is vital for engaging stakeholders in health technology assessment (HTA), as well as the successful dissemination and adoption of HTA research and guidance. As a relatively new organization, Health Technology Wales (HTW) has an ideal opportunity to take an effective, strategic approach to communication and stakeholder engagement from the outset.MethodsHTW commissioned Pagoda Public Relations to develop an informed communications strategy and delivery framework. The strategy used OASIS methodology for public relations planning: Objectives, Audience insight, Strategy, Implementation, and Scoring (evaluation). Initial objectives were developed with input from the HTW team and members of the HTW Assessment Group and Appraisal Panel. Stakeholder insights were collected through an online survey and telephone interviews. These insights were used to inform the communications strategy and framework, outlining key audiences, key messages, communication objectives, methods, tactics, and evaluations.ResultsSeven key objectives were identified, each of which were supported by recommended actions. These were underpinned by the key aims and messages reflecting how we will achieve these objectives. National Health Service boards, government, clinicians, the technology and research sector, patients, and the general public were identified as priority audiences. Various different communication channels and activities were identified, aimed at various audiences. These included the website, social media, traditional media, and exhibitions or workshops, as well as targeted e-mail dissemination of guidance. Evaluation of HTW communications will be aligned with the wider HTW evaluation strategy, and evidence will be recorded through OutNav software (Matter of Focus Ltd).ConclusionsHTW is committed to a strategic, effective approach to communication and engagement. We now have an audience-informed communications strategy and plan that outlines our key objectives, and how to achieve and evaluate these objectives. Successful implementation will raise awareness of and value in profile and outputs of HTW, both in Wales and internationally.

PP135 Setting The Scope For Assessing e-Health Technologies In Hungary

IntroductionE-health and m-health are emerging health technology fields that could possibly give a new scope to health technology assessment (HTA). The Division for Health Technology Assessment (DfHTA) is currently assessing medicines and non-drug technologies (medical devices intended for patient use or for use in hospitals). The experience assessing medical devices for use in hospitals yielded difficulties which could also arise from the critical appraisal of e-health or m-health technologies. The objective of this study was to explore the foundations for HTA guidance on e-health or m-health technologies.MethodsA targeted literature review was conducted to map the current status of technology assessment practices for e-health and m-health technologies and to assess its concordance with current reimbursement processes in countries belonging to the Organisation for Economic Co-operation and Development. Experiences from past evaluations of other medical devices that could not be evaluated under the current guidance guided the literature search. The findings of this research were used to create a recommendation to amend the current Hungarian Guideline for Health Economic Analyses.ResultsThe resulting articles of the targeted literature review provided an insight into current practices on of assessing e-health and m-health products, particularly with respect to the domains of safety, quality, and impact. Recommendations suggested including a list of requirements for companies to submit for critical evaluations of e-health and m-health technologies, in support of a self-assessment approach.ConclusionsAs for other HTA bodies, there is an urgent need for the DfHTA to increase its capacity to assess digital health technologies for entry into the healthcare system, with a focus on the relevant clinical domains. The reimbursement process for these technologies remains a challenge for public funding bodies.

Comparison of Recommendations and Use of Cardiovascular Risk Equations by Health Technology Assessment Agencies and Clinical Guidelines

ObjectivesTo identify risk equations for cardiovascular diseases (CVDs) in primary and secondary prevention settings that are used or recommended by health technology assessment (HTA) organizations and in clinical guidelines (CGs). MethodsA targeted literature review was conducted using a two-stage search strategy. First, HTA reviews of manufacturers’ drug submissions, reports from established HTA organizations (Europe, Canada, and Australia), and CGs from countries with and without HTA organizations, including the United States, were identified. Documents published between September 30, 2006 and September 30, 2016, were examined for cardiovascular risk equations, recommendations, and commentaries. Next, publications associated with risk equations and cited by HTA and CG documents were retrieved. This literature was examined to extract commentaries and risk equation study characteristics. ResultsThe review identified 47 risk equations, 25 in the primary CVD prevention setting (i.e., patients with no CVD history), including 5 for CVD prevention in diabetes and 22 solely in secondary prevention settings; 11 were identified for heart failure, 3 for stroke or transient ischemic attack, 2 for stable angina, and 11 for acute coronary syndrome or related conditions. A small set of primary prevention equations was found to be commonly used by HTAs, whereas secondary prevention equations were less common in HTA documents. CGs provided more risk equations as options than HTA documents. ConclusionsAlthough there is an abundance of risk equations developed for primary and secondary prevention, there remains a need for additional research to provide sufficient clinical and HTA guidance for risk estimation, particularly in high-risk or secondary prevention settings.

EUPATI Guidance for Patient Involvement in Medicines Research and Development: Health Technology Assessment.

The main aim of health technology assessment (HTA) is to inform decision making by health care policy makers. It is a systematic process that evaluates the use of health technologies and generally involves a critical review of international evidence related to clinical effectiveness of the health technology vs. the best standard of care. It can also include an evaluation of cost effectiveness, and social and ethical impacts in the local health care system. The HTA process advises whether or not a health technology should be used, and if so, how it is best used and which patients are most likely to benefit from it. The importance of patient involvement in HTA is becoming widely recognized, for scientific and democratic reasons. The extent of patient involvement in HTA varies considerably across Europe. Commonly HTA is still focused on quantitative evidence to determine clinical and/or cost effectiveness, but the interest in understanding patients' experiences and preferences is increasing. Some HTA bodies provide support for participation in their processes, but again this varies widely across Europe. The involvement of patients in HTA is determined at the national and regional level, and is not subject to any European-wide legislation. The guidance text presented in this article was developed as part of the work of the European Patients' Academy on Therapeutic Innovation (EUPATI) and covers the interaction between HTA bodies and patients and their representatives when medicines are being assessed. Other EUPATI guidance documents relate to patient involvement in pharmaceutical industry-led research and development, ethics committees, and regulatory authorities. The guidance provides recommendations for activities to support patient involvement in HTA bodies and specific guidance for individual HTA processes. It seeks to improve patient involvement, using the outcomes of published research and consensus-building exercises. It also draws on good practice examples from individual HTA bodies. The guidance is not intended to be prescriptive and should be used according to specific circumstances, national legislation, or the unique needs of each interaction. This article represents the formal publication of the HTA guidance text with discussion about recent progress in, and continuing barriers to, patient involvement in HTA.

Impact of country-specific health economic guidelines on cost-effectiveness (CE) estimates: An illustrative study of the CE of cetuximab in the first-line (1L) treatment of RAS wild-type (wt) metastatic colorectal cancer (mCRC).

702 Background: Adding cetuximab to conventional 1L chemotherapy (FOLFIRI or FOLFOX) improves survival outcomes in patients with RASwt mCRC. Conflicting evidence exists regarding the CE of cetuximab in mCRC, due in part to differences in country-specific health technology assessment (HTA) guidelines. Methods: CE estimates for the 1L treatment of RASwt mCRC with FOLFIRI or FOLFOX ± cetuximab were calculated for the UK (NICE), Brazil, Netherlands, and Belgium using a Markov cohort model populated with country-specific HTA requirement inputs and assumptions. Model outcomes were treatment costs, life-years (LY) or quality-adjusted LY (QALY) gained, and the incremental CE ratio (ICER). ICERs were compared against CE thresholds. Results: Country-specific HTA guidance resulted in differing CE outcomes for patients with RASwt mCRC receiving FOLFIRI or FOLFOX ± cetuximab (Table). Conclusions: We applied our Markov model to four markets, incorporating country-specific HTA requirements into our calculations. Although our analyses with similar comparators led to a varying range of CE estimates, they met the HTA threshold guidelines in the four markets. [Table: see text]

A review of the economic tools for assessing new medical devices.

Whereas the economic evaluation of pharmaceuticals is an established practice within international health technology assessment (HTA) and is often produced with the support of comprehensive methodological guidance, the equivalent procedure for medical devices is less developed. Medical devices, including diagnostic products, are a rapidly growing market in healthcare, with over 10,000 medical technology patent applications filed in Europe in 2012-nearly double the number filed for pharmaceuticals. This increase in the market place, in combination with the limited, or constricting, budgets that healthcare decision makers face, has led to a greater level of examination with respect to the economic evaluation of medical devices. However, methodological questions that arise due to the unique characteristics of medical devices have yet to be addressed fully. This review of journal publications and HTA guidance identified these characteristics and the challenges they may subsequently pose from an economic evaluation perspective. These unique features of devices can be grouped into four categories: (1) data quality issues; (2) learning curve; (3) measuring long-term outcomes from diagnostic devices; and (4) wider impact from organisational change. We review the current evaluation toolbox available to researchers and explore potential future approaches to improve the economic evaluation of medical devices.

Knowledge mobilization in the context of health technology assessment: an exploratory case study

BackgroundFinding measures to enhance the dissemination and implementation of their recommendations has become part of most health technology assessment (HTA) bodies' preoccupations. The Quebec government HTA organization in Canada observed that some of its projects relied on innovative practices in knowledge production and dissemination. A research was commissioned in order to identify what characterized these practices and to establish whether they could be systematized.MethodsAn exploratory case study was conducted during summer and fall 2010 in the HTA agency in order to determine what made the specificity of its context, and to conceptualize an approach to knowledge production and dissemination that was adapted to the mandate and nature of this form of HTA organization. Six projects were selected. For each, the HTA report and complementary documents were analyzed, and semi-structured interviews were carried out. A narrative literature review of the most recent literature reviews of the principal knowledge into practice frameworks (2005-2010) and of articles describing such frameworks (2000-2010) was undertaken.Results and discussionOur observations highlighted an inherent difficulty as regards applying the dominant knowledge translation models to HTA and clinical guidance practices. For the latter, the whole process starts with an evaluation question asked in a problematic situation for which an actionable answer is expected. The objective is to produce the evidence necessary to respond to the decision-maker's request. The practices we have analyzed revealed an approach to knowledge production and dissemination, which was multidimensional, organic, multidirectional, dynamic, and dependent on interactions with stakeholders. Thus, HTA could be considered as a knowledge mobilization process per se.ConclusionsHTA's purpose is to solve a problem by mobilizing the types of evidence required and the concerned actors, in order to support political, organizational or clinical decision-making. HTA relies on the mediation between contextual, colloquial and scientific evidence, as well as on interactions with stakeholders for recommendation making. Defining HTA as a knowledge mobilization process might contribute to consider the different orders of knowledge, the social, political and ethical dimensions, and the interactions with stakeholders, among the essential components required to respond to the preoccupations, needs and contexts of all actors concerned with the evaluation question's issues.

HEALTH TECHNOLOGY ASSESSMENT IN POLAND AND SCOTLAND: COMPARISON OF PROCESS AND DECISIONS

We compared Polish and Scottish Health Technology Assessment (HTA) process in order to elicit recommendations for future development of HTA methodological guidelines in Poland. We studied the differences between Polish and Scottish HTA methodological guidelines. HTA recommendations issued by Polish HTA agency (AHTAPol) in the period January 1 through December 31, 2008, were benchmarked to HTA guidance published by Scottish Medical Consortium (SMC) for the same drug technology. The Scottish HTA methodological guidelines were more instructive in terms of clinical and economic evaluations than Polish guidelines. SMC evaluated forty-eight of sixty-eight drug technologies appraised by AHTAPoL. There were thirty drug technologies that received similar guidance in both countries and eighteen with contradictory HTA recommendations. In Scotland, there were more positive HTA recommendations than there were in Poland. While comments about efficacy or safety were commonplace among reasons for negative recommendations in Poland, insufficient justification of treatment's cost in relation to benefits was the most often cited reason for rejection in Scotland. SMC tended to recommend restricted use to specific sub-populations for several drug technologies negatively appraised by AHTAPoL. The comparison between SMC and AHTAPoL suggests that there is potential room of improvement of the Polish HTA methodological guidelines. Comparative effectiveness and safety, subgroup analysis, and adaptation of models to local settings were identified as key areas for further development of Polish HTA methodological guidelines.