Health Technology Assessment Network Research Articles

Conducting a health technology assessment in the West Bank, occupied Palestinian territory: lessons from a feasibility project.

To achieve universal health coverage (UHC), countries must make difficult choices to optimize the use of scarce resources. There is a growing interest in using evidence-based priority setting processes, such as Health Technology Assessment (HTA), to inform these decisions. In 2020, the Palestinian Institute of Public Health (PNIPH) and the Norwegian Institute of Public Health (NIPH) initiated a pilot to test the feasibility of coproducing an HTA on breast cancer screening in the West Bank, occupied Palestinian Territory. Additionally, a secondary aim was to test whether using an adaptive HTA (aHTA) approach that searched and transferred published evidence syntheses could increase the speed of HTA production. The applied stepwise approach to the HTA is described in detail and can be summarized as defining a core team, topic selection, and prioritization; undertaking the HTA including adaptation using tools from the European Network for HTA (EUnetHTA) and stakeholder engagement; and concluding with dissemination. The aHTA approach was faster but not as quick as anticipated, which is attributed to (i) the lack of availability of local evidence for contextualizing findings and (ii) the necessity to build trust between the team and stakeholders. Some delays followed from the COVID-19 pandemic, which showed the importance of good risk anticipation and mitigation. Lastly, other important lessons included the ability of virtual collaborations, the value of capacity strengthening initiatives within low- and middle-income countries (LMICs), and the need for early stakeholder engagement. Overall, the pilot was successfully completed. This was the first HTA of its kind produced in Palestine, and despite the challenges, it shows that HTA analysis is feasible in this setting.

PP52 Will Joint European Health Technology Assessment Provide Additional Benefits Over Individual Country-wise Assessments?

IntroductionIn December 2021 the European Union (EU) Health Technology Assessment (HTA) Regulation, a key pillar of the EU Pharmaceutical Strategy, was adopted by the Council and the European Parliament. The focus areas of Joint HTA Cooperation include Joint Clinical Assessments (JCA), Joint Scientific Consultations (JSC), and joint early-stage horizon scanning. The European HTA regulation will be adopted in a stepwise approach and from 2030 onwards, all products (drugs, high-risk medical devices, and in vitro diagnostics) approved in all indications will be subjected to JCA in EU.MethodsA targeted literature research was performed for policies and the European Network for HTA (EUnetHTA) methodological guidelines describing the HTA methods including scoping process, comparators, endpoints, the applicability of evidence, and validity of clinical studies. Additionally, the anticipated opportunities and challenges were also summarized with respect to these methods.ResultsEUnetHTA put forward a timeline for different activities over the next three years as part of the new EU HTA Regulation, including key deadlines for ongoing EUnetHTA consultations on the processes and methods. EUnetHTA will set up a new ecosystem across the EU as it aims to reduce duplication and time to access by supplementing multiple national clinical assessments with a joint central assessment. In any case, assessment of added value and pricing and reimbursement decisions will still occur at the national level.Additionally, EU HTA may promote harmonization of processes, standards, and evidence requirements, which will increase predictability and simplify evidence requirements. However, differences in clinical practice, standard of care, and national priorities may lead to assessments that are not generalizable to all Member States.ConclusionsThe joint EU HTA cooperation will benefit countries which have less developed or do not have established HTA expertise or infrastructure. However, the JCA process could result in increased requirements for clinical evidence generation as relative effectiveness and relevance of outcomes to patients gain further importance for products to successfully gain access across countries.

Reporting reimbursement price decisions for onco-hematology drugs in Spain.

Even using well-established technology assessment processes, the basis of the decisions on drug price and reimbursement are sometimes perceived as poorly informed and sometimes may be seen as disconnected from value. The literature remains inconclusive about how Health Technology Assessment Bodies (HTAb) should report the determinants of their decisions. This study evaluates the relationship between oncology and hematology drug list prices and structured value parameters at the time of reimbursement decision in Spain. The study includes all new onco-hematological products (22), with a first indication authorized between January 2017 and December 2019 in Spain and pricing decisions published up until October 2022. For each product, 56 contextual and non-contextual indicators reflecting the structured multiple criteria decision analysis (MCDA) - Evidence-based Decision-Making (EVIDEM) framework were measured. The relationship between prices and the MCDA-EVIDEM framework was explored using univariate statistical analyses. Higher prices were observed when the standard of care included for combinations, if there were references to long-lasting responses, for fixed-duration treatment compared to treatment until progression and treatment with lower frequencies of administration; lower prices were observed for oral administration compared to other routes of administration. Statistically significant associations were observed between prices and the median duration of treatment, the impact on patient autonomy, the ease of use of the drug, and the recommendations of experts. The study suggests that indicators related to the type of standard of care, references to long-lasting responders, the convenience of the use of the drug, and the impact of treatment on patient autonomy, as well as contextual indicators such as the existence of previous clinical consensus, are factors in setting oncology drug prices in Spain. The implementation of MCDA-EVIDEM methodologies may be useful to capture the influence on pricing decisions of additional factors not included in legislation or consolidated assessment frameworks such as the European Network for Health Technology Assessment (EunetHTA) core model. It may be opportune to consider this in the upcoming revision of the Spanish regulation for health technology assessments and pricing and reimbursement procedures.

P539 Patient Preferences for Inflammatory Bowel Disease Treatments: Development of a European Preference Survey using a Discrete Choice Experiment

Abstract Background There is a growing consensus across stakeholders on the value of patient preference (PP) studies, as recognized by the European Medicines Agency and the European Network for Health Technology Assessment. This study aimed to develop a PP survey to quantitatively evaluate which characteristics are most important to inflammatory bowel disease (IBD) patients, so that the results can inform the medicinal product life cycle (MPLC). Methods Following the PREFER recommendations, a four-step approach was followed to develop the PP survey: 1) stated preference method selection, 2) attribute and level development, 3) survey implementation, and 4) survey piloting. Throughout the different steps of the survey development, an advisory board (AB) consisting out of patients, patient organizations, gastroenterologists, IBD nurses, and statisticians were consulted. Results First, a discrete choice experiment (DCE) was chosen as the preferred elicitation method because it allows to reach the objectives of the survey, namely to: 1) quantify the relative importance of different attributes, 2) elicit the trade-offs patients make, and 3) measure preference heterogeneity. Second, a scoping literature review of IBD clinical trials, available IBD treatments, and previously published preference studies was performed. Discussions on these characteristics with patients (n=11) during focus group discussions (FGDs) using the nominal group technique revealed the following patient prioritized characteristics: prevent surgery, long-term clinical remission, improved quality of life, occurrence of urgency, and improved labor rate. Based upon the results of the FGDs, a preliminary list of possible attributes for inclusion in the DCE was refined during AB meetings. This list was iteratively adapted until consensus was found on 14 attributes, their levels, and corresponding explanations (Figure 1). Third, the DCE survey was designed to include these 14 attributes using partial profiles next to measures about sociodemographic variables, health literacy, and treatment experience. Fourth, piloting the study with IBD patients (n=4) revealed that the survey was comprehensible, had a good flow, and contained graphics that supported the content. Only minor text edits were needed. Conclusion It is key to develop a PP survey in partnership with patients and other members of the AB. This survey will be rolled out across Europe in 11 different languages and will increase understanding of which treatment aspects are most important to IBD patients. These aspects should be included in drug development and evaluation so that the MPLC can be more patient-centered and future treatments will target aspects that are most important to IBD patients themselves.

Stool DNA testing for early detection of colorectal cancer: systematic review using the HTA Core Model® for Rapid Relative Effectiveness Assessment.

Stool DNA testing for early detection of colorectal cancer (CRC) is a non-invasive technology with the potential to supplement established CRC screening tests. The aim of this health technology assessment was to evaluate effectiveness and safety of currently CE-marked stool DNA tests, compared to other CRC tests in CRC screening strategies in an asymptomatic screening population. The assessment was carried out following the guidelines of the European Network for Health Technology Assessment (EUnetHTA). This included a systematic literature search in MED-LINE, Cochrane and EMBASE in 2018. Manufacturers were asked to provide additional data. Five patient interviews helped assessing potential ethical or social aspects and patients' experiences and preferences. We assessed the risk of bias using QUADAS-2, and the quality of the body of evidence using GRADE. We identified three test accuracy studies, two of which investigated a multitarget stool DNA test (Cologuard®, compared fecal immunochemical test (FIT)) and one a combined DNA stool assay (ColoAlert®, compared to guaiac-based fecal occult blood test (gFOBT), Pyruvate Kinase Isoenzyme Type M2 (M2-PK) and combined gFOBT/M2-PK). We found five published surveys on patient satisfaction. No primary study investigating screening effects on CRC incidence or on overall mortality was found. Both stool DNA tests showed in direct comparison higher sensitivity for the detection of CRC and (advanced) adenoma compared to FIT, or gFOBT, respectively, but had lower specificity. However, these comparative results may depend on the exact type of FIT used. The reported test failure rates were higher for stool DNA testing than for FIT. The certainty of evidence was moderate to high for Cologuard® studies, and low to very low for the ColoAlert® study which refers to a former version of the product and yielded no direct evidence on the test accuracy for ad-vanced versus non-advanced adenoma. ColoAlert® is the only stool DNA test currently sold in Europe and is available at a lower price than Cologuard®, but reliable evidence is lacking. A screening study including the current product version of ColoAlert® and suitable comparators would, therefore, help evaluate the effectiveness of this screening option in a European context.

Stakeholder perspectives on cooperation in the clinical and nonclinical health technology assessment domains.

The aim of this study was to deliver insights from multiple stakeholders into actual and future collaboration for health technology assessment (HTA) in general and in oncology in particular. Eighteen semi-structured interviews were conducted with experts from European HTA bodies (HTAbs), former board members of the European Network for Health Technology Assessment (EUnetHTA), and representatives from the pharmaceutical industry, a regulatory agency, academia, and patient organizations. The stakeholders were asked about their support of the EUnetHTA's intent, about the general strengths and challenges of the EUnetHTA and its Joint Action 3 (JA 3), the strengths and challenges of the clinically oriented HTA collaboration in oncology during JA 3 across the technology life cycle, about future challenges to HTA in oncology with consequences for collaboration, and about collaboration in the economic domains of HTA. The transcribed interviews were analyzed qualitatively. The participants perceived the intention and work quality of the EUnetHTA as positive. The experts described methodological, procedural, and capacity challenges in early dialogues (EDs) and rapid relative effectiveness assessments (REAs) meant to analyze clinical effectiveness in oncology. The majority attached increasing importance to collaboration in the future to cope with the uncertainty of HTA. Several stakeholders also proposed the incorporation of joint postlaunch evidence generation (PLEG) activities. Some gave sporadic suggestions for voluntary nonclinical collaboration as well. Stakeholders' continued readiness to discuss the remaining challenges to and sufficient resources for implementing HTA regulation, as well as further cooperative expansion along the technology life cycle, are necessary for improved HTA collaboration in Europe.

How is the Societal Perspective Defined in Health Technology Assessment? Guidelines from Around the Globe.

Some researchers have argued that the aim of an economic evaluation should be to offer guidance on resource allocation based on public interest from a societal perspective. The application of a societal perspective in health technology assessment (HTA), while common in many published studies, is not mandated in most countries, and there is limited discussion on what the societal perspective should encompass. This study aimed to systematically compare and contrast the HTA guidelines in different countries. HTA methods guidelines were identified through international HTA networks, such as the Professional Society for Health Economics and Outcomes Research (ISPOR) and Guide to Economic Analysis Research (GEAR). The respective HTA agencies were grouped into two categories: well-established and newly developed, based on the establishment date. Data extracted from the guidelines summarised the methodological details in the reference cases, including specifics on the societal perspective. The database search yielded 46 guidelines, and 65% explicitly considered the societal perspective. The maturity of these agencies is reflected in their attitudes towards the societal perspective; the societal perspective is defined in 73% of the guidelines of well-established agencies and only 56% of those of newly developed agencies. The guidelines from multipayer healthcare systems are more likely to consider the societal perspective. Although most guidelines from the well-established agencies recommend the inclusion of a societal perspective, the types of costs and consequences that should be included and the recommended approaches to valuing them are variable. The direct costs to family and carers were included in 73% of the societal perspective definitions, while non-health outcomes were considered in only 40%. Most HTA guidelines lack clear guidance on what to include under specific perspectives. Considering the recent advancements in economic evaluation methods, it is timely to rethink the role of the societal perspective in HTA guidelines and adopt a more comprehensive perspective to include all costs and consequences of healthcare services.

PP70 Enhancing HTA Processes In The Maltese System For Introducing New Medicines

IntroductionMost European countries use Health Technology Assessment (HTA) as input for decisions on reimbursement of (new) medicines. In 2018, following the approval of European Social Funding, the Directorate for Pharmaceutical Affairs (DPA) within the Malta Ministry for Health led a specific Work Package aimed at enhancing knowledge, skills and HTA processes to inform reimbursement decisions.MethodsSince the start of the project, the Institute for Medical Technology Assessment (iMTA) and the National Health Care Institute of the Netherlands (ZIN) collaborate in providing guidance and training to DPA on relative effectiveness assessments (REAs) and pharmacoeconomic assessments (PEAs) of pharmaceuticals. Several activities were organized: site visits, face-to face interviews with stakeholders, a qualitative assessment of the core process of the Maltese system, health economics training and tutorials, meetings introducing the European Network for Health Technology Assessment (EUnetHTA) and Dutch HTA processes, development of a new framework for assessment, and a shadow assessment.ResultsOur assessment identified important methodological challenges and crucial processes interdependencies to optimize within the Maltese system. Based on the learnings, DPA created a template based on the EUnetHTA REA assessment format. IMTA created a template to perform PEAs. Currently, a shadow assessment is ongoing in which DPA performs the REA and PEA of a pharmaceutical using the new templates. ZIN and iMTA will provide feedback to DPA on several aspects, including, but not limited to, the PICO, assessment of therapeutic efficacy and safety, identifying uncertainties, input costs and effects, and formulating a clear discussion of the assessment. If necessary, the templates will be adapted during the process. The project will be evaluated and finalized in 2022.ConclusionsWith the guidance and training provided by iMTA and ZIN, DPA structured and aligned their REA and PEA to enhance their assessment process and to improve the presentation of their HTA report to the two appraisal committees. This project emphasizes the importance of international collaboration to enhance HTA processes within the

OP13 EUnetHTA Relative Effectiveness Assessments Of Pharmaceutical And Other Technologies: Procedural Changes Implemented During Joint Action 3

IntroductionThe European Network for Health Technology Assessment (EUnetHTA) Joint Action 3 (JA3) aimed to develop a sustainable European model for scientific and technical collaboration on HTA. It succeeded EUnetHTA JA2, which focused on strengthening practical applications of approaches and tools in the European HTA collaboration. Compared to JA2, several changes in procedures and processes were undertaken throughout JA3 in order to improve the different steps in joint HTA production.MethodsFindings and identified challenges regarding the assessment production processes from JA2 were considered as a basis. In JA3, vast majority of structured and informal feedback was gathered from the assessment teams and project managers via feedback surveys and meetings. Only limited informal feedback from stakeholders (such as patients, health care professionals, and health technology developers) that were involved in EUnetHTA assessments was collected. To this end, experiences were documented and recommendations for a future production process were developed.ResultsDuring the course of JA3, the joint production resulted in 16 pharmaceutical assessments and 27 assessments of other technologies. The latter included medical devices, diagnostics, interventions, and screening. Due to the different context of pharmaceuticals and other technologies, some technology-specific changes needed to be made in their production process. However, the majority of implemented changes were made for both types of technologies to ensure maximum possible alignment in processes. The implemented changes affected several steps in the production process as well as the involvement of stakeholders in EUnetHTA assessments. The production and related project management of assessments was fine-tuned and resulted in clearer, standardized, and comprehensible processes that facilitated transparency and inclusiveness.ConclusionsThe procedural changes led to further standardization and elaboration of assessment production processes in preparation for a future European HTA system under the EU HTA Regulation. However, some methodological challenges remained to be tackled further in the currently ongoing EUnetHTA 21 service contract.

PP96 Joint Clinical Assessments – Implementation And Lessons For The Next Stage Of EU HTA

IntroductionThe European Network for Health Technology Assessment (EUnetHTA) was a voluntary cross-border initiative on HTA harmonization established by European Commission in 2005. Between 2016-2021, EUnetHTA completed 56 Joint Assessments (28 drugs; 28 other technologies) including 14 reviews of COVID treatments.MethodsWe conducted a review of the 14 EUnetHTA joint assessment reports of drugs in non-COVID 19 indications. We cross-referenced recommendations with national guidance in 30 member countries (including UK) and conducted an analysis of time to national assessment, choice of comparator, direct reference to EUnetHTA assessment, and time to reimbursement decision.ResultsSix products in oncology, 2 in endocrine and metabolic diseases, 2 in infectious and parasitic diseases, and cardiovascular, digestive system, eye disorders and central nervous system (one each) were identified. On average, EUnetHTA published its recommendation 52 days after market authorization for oncology products and 33 days for non- oncology products. EUnetHTA recommendations considered on average 4 comparators (range 1-8) as part of the assessment. All of the 6 oncology products have been assessed by national HTA bodies, however uptake was low with an average of 5 reports referencing the EUnetHTA report. Similarly for the non-oncology products assessed only 3 of 30 HTA bodies cite the EUnetHTA report. Citing HTA bodies were: AETSA (Spain), HAS (France), INFARMED (Portugal), NoMA (Norway), and TLV (Sweden). There was no clear reduction in the time to reimbursement for these products in these markets.ConclusionsAccording to EUnetHTA, there has been an increased use and dissemination of joint assessment reports since 2016. Our analysis shows that the level of implementation across countries is heterogeneous despite publication of the EUnetHTA reports shortly after market authorization. The future the EU HTA will depend on the timeliness, rigor and transparency of joint clinical assessment reports and improved uptake of these reports at a national level.

PP82 First Educational Trainings According To New Health Technology Assessment Guideline For Medicines In Ukraine

IntroductionThe success of the health technology assessment (HTA) implementation depends on the level of communication efficiency between all stakeholders. Taking this into consideration, the leading HTA experts of HTA Department of State Expert Center of the Ministry of Health of Ukraine launched the new educational initiative which includes training programs for pharmaceutical companies.MethodsСomprehensive review of the first developed HTA training programs based on the HTA Guideline (Number 593) “The state health technology assessment for medicines” approved on 29 March 2021 was conducted.ResultsLeading HTA experts of the HTA Department developed a training program, which was conducted for industry representatives according to the provisions of the HTA Guideline. In 2021 over twelve training sessions and two webinars were conducted. These learning events reflect harmonized international recommendations and approaches to HTA training programs, The Professional Society for Health Economics and Outcomes Research, ISPOR short courses in particular, European Network for Health Technology Assessment (EUnetHTA), The National Institute for Health and Care Excellence (NICE), Institute for Clinical and Economic Review (ICER), Agency for Health Technology Assessment and Tariff System (AOTMiT) guidelines. Two-hour webinars were aimed at giving theoretical and practical bases for building a Markov model with the help of Excel and TreeAge Software. Training sessions titled “HTA as a tool for assessing the value of health technology” differ by duration (7-hour, 4-hour, 3-hour) and cover core topics adapted to the needs of the audience. The main program components include HTA Roadmap in Ukraine, clinical section, economic section and practical case studies of building a Markov model. All training options included questionnaires at the beginning and i end to assess the quality of each program. Questionnaires are the tool that gives presenters the possibility to trace progress and transform the training material accordingly.ConclusionsDevelopment of extended capacity building programs in HTA for users and doers is highly prospective for further steps in HTA institutionalization in Ukraine.

PD37 High-Dose Vitamin D For The Treatment Of COVID-19

IntroductionThe aim of this EUnetHTA (European Network for Health Technology Assessment) Rolling Collaborative Review on high dose vitamin D for the treatment of COVID-19 was to inform health policy at an early stage in the life cycle of therapies and to monitor ongoing studies in the format of a Living Document.MethodsThe systematic literature search was conducted in Medline, Pubmed, medRxiv, bioRxiv, arXivso, Cochrane COVID-19 Study Register, ClinicalTrials.gov, ISRCTN Registry, EU Clinical Trials Register. The first search was done in January 2021, and the last in November 2021. English and German randomized controlled studies (RCTs) investigating treatment of severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infected individuals with high dose vitamin D2, D3 or their metabolites were included if examining mortality, length of hospital stay, viral burden, clinical progression, hospitalization rates, intensive care unit (ICU) admission, mechanical ventilation, quality of life or adverse events. Two reviewers independently screened search results and assessed risk of bias and certainty of evidence. One reviewer extracted study data, checked by another.ResultsOf the nine RCTs published to date, two investigate calcifediol, one calcitriol and six vitamin D3. All used different dosing regimens. Disease severity and proportion of vitamin D deficiency varied between studies. Calcifediol treated patients in one study required significantly less ICU admissions than untreated patients. Vitamin D3 in another study led to significantly more SARS-CoV-2 PCR-negative patients before day 21 than placebo. There were no other significant differences between groups. Twenty-five RCTs are ongoing, five of them with over 1,000 patients.ConclusionsThe current evidence is heterogenous regarding form and dosage of vitamin D, baseline disease severity and baseline vitamin D deficiency. There is currently no standardized/recommended level of what constitutes a (beneficial) “high dose”. Most results did not show significant differences between vitamin D treated groups and no vitamin D / placebo groups. Many of the studies are very small and certainty of evidence is predominantly low or very low.

PP115 Analysis Of Previous Joint Clinical Assessment And Potential Transferability To Four European Countries: Case Study And Conceptual Approach

IntroductionThe European Network for Health Technology Assessment (EUnetHTA) has led together, with member states, several joint actions, including Joint Clinical Assessments (JCA), under the form of project-based voluntary cooperation, which outputs and transferability of those projects in other European countries remains somehow limited. In June 2021, the European Council has reached an agreement on the European Health Technology Assessment (HTA) regulation, which is entering into force gradually. Initially limited to oncology products, then extended to orphan/advanced therapy, and after a five to eight-year additional period, it will apply to all centrally approved products. The JCA will consist of a focused scientific analysis on relative effectiveness assessment, including the health condition, technology description, clinical effectiveness, and safety. These analyses will also include information relating to the degree of certainty. We consider that the evidence appraisal might have limitations, and transferability would not be generalizable. We aim to determine the potential drivers and barriers for HTA transferability in EU4, employing the analysis of a case example where JCA was conducted.MethodsEmploying an oncology JCA, we will compare an HTA analysis conducted in EU4 countries (Germany, France, Italy, and Spain). Overview and background information on countries involved in the JCA, and EU4 HTA system will be provided, followed by HTA outcome and main evidence requirements, reimbursement outcome, and pricing agreements.ResultsStudy results supporting HTA outcomes may focus on the population assessed, the comparator considered, and uncertainty management. A conceptual adaptation about the scope of the EU JCA regulation will be discussed, to understand its potential advantages to individual HTAs in Europe and remaining gaps to effectively inform HTA or decision-making process.ConclusionsThe analysis of pricing and reimbursement outcomes can further help understand potential barriers and drivers for JCA transferability and potential areas of evidence generation requirements.

OP49 A Systematic Review Of The Activities Of Early Advice, Early Dialogue, Scientific Advice By HTA Doers

IntroductionThere is a range of activities that health technology assessment (HTA) doers have started to improve the process of generation of required evidence for new technologies, and the alignment of regulatory and reimbursement processes that retard the access to patients to them. Different organizations call those processes early advice, early dialogue, or scientific advice to those activities.MethodsWe performed a systematic review of the activities named scientific advice (SA), early advice (EA) and early dialogue (ED). Major databases and HTA organizations were explored. The protocol and search strategy were published in PROSPERO. The selection of final articles and documents was done in pairs, and when discrepancies were found a third person resolved with the consensus of the others. A matrix was used to define the commonalities and differences of the described processes.ResultsWe initially retrieved 949 documents, after the analysis of duplications and the full text reading of the selected ones, we finally selected 39 documents and described: the type of technologies, the process, the stakeholders, the duration, the costs, and the impact. Big HTA agencies such as the Canadian Agency for Drugs and Technologies in Health (CADTH) or the National Institute for Health and Care Excellence (NICE) included EA or SA among their portfolio of activities as well as networks (European Network for HTA (EUnetHTA) or smaller agencies such as HTA Wales or Basque Office for HTA (Osteba) among others. The type of activity, the process, duration, purpose and costs differ among HTA doers.ConclusionsThere is a need to define what we meant when we are talking about SA, ED, and EA. In fact, regulators used the same processes with different purposes. Our systematic review and the lessons learnt from the European-funded SAFENMEDTECH project will propose a detailed framework that can be useful to better understanding the needs of each of the involved parties and how to make the processes involved more efficient.

HTA87 Evaluating the Framework and Evolution of the Italian Ministry of Health HTA Reports

Technology and innovation assume a key role in the current clinical practice, greatly influencing Health Technology Assessment (HTA) development and the role of the International HTA network (EUnetHTA) in standardizing HTA process among Member States. Our objective is to assess the main domains and technologies investigated in HTA reports developed by the National Agency for Regional Health Services (Agenas) in collaboration with the Italian Ministry of Health (MoH).

5.M. Workshop: The need to account for vaccine-specificities in the implementation of the EU Regulation on HTA

Abstract In January 2022, the EU Regulation on Health Technology Assessment (HTAR) entered into force. The HTAR provides for the centralized conduct of EU-wide joint clinical assessment (JCA) for oncology treatments (2025 onwards), and for other medicinal products incl. vaccines (2030 onwards). HTA is a multidisciplinary process aiming to disentangle the value of a health technology in comparison to other. It has been used, to different extent across EU countries, to inform vaccine price and reimbursement decisions. Nevertheless, existing assessment frameworks utilized by HTA bodies, incl. National Immunization Technical Advisory Groups (NITAGs), as well as the relative effectiveness assessment (REA) framework developed by the European Network for Health Technology Assessment (EUnetHTA), do not fully account for the specificities of vaccines. In fact, to account for the broad value of vaccines several aspects ought to be considered, including societal benefits. Attention has been paid to these topics for years by the public health community, and in particular EUPHA-HTA, and also Vaccines Europe, a specialized vaccines group within the European Federation of Pharmaceutical Industries and Associations (EFPIA), has recently released a position statement on the need to tackle vaccine specificities in future JCA. Furthermore, as the COVID-19 pandemic has highlighted, such aspects are critical for joint procurement efforts to combat the current and future pandemics. The objective of this workshop is to address the current and future role of HTA in vaccine regulation across EU countries, inform on key specificities of vaccines in respect to JCA and HTA as a whole and discuss opportunities to develop vaccine-specific methodological guidelines within the HTAR. Following an introduction, the following topics will be addressed: • Competent bodies and HTA impact on vaccine decision-making process, incl. a discussion of implementation challenges and potential impacts of the HTAR. • JCA specificities in respect to vaccines and the importance of addressing the whole value of vaccines and vaccinations. • The proposal of Vaccines Europe for guiding principles to account for vaccines specificities in JCA built on the outcomes of an ‘across-the-industry’ project incl. also non-industry experts. The presentations will be followed by a panel discussion to further explore the themes, consider next steps and address questions from the audience. Key messages • The new EU HTAR is paving the way for JCAs of several medicinal products, incl. vaccines necessitating expert guidance in terms of determining their whole value. • Vaccines specificities should be duly considered in JCAs to inform national and EU-wide decision-making for vaccination programmes, reimbursement, and pricing.

2.G. Workshop: The development of HTA collaboration in Europe: where we are and where we are going

Abstract Health decision-making should be always well-informed. This draws the attention on the need to make the best use of available evidence in respect to different clinical and non-clinical issues to make a value-based allocation of resources. Health Technology Assessment (HTA) is a method for supporting evidence-informed health policymaking as it is defined as a multidisciplinary process that uses explicit methods to determine the value of a health technology at different points in its lifecycle. The role of HTA is of utmost importance at European Union level considering that citizens have the right to access healthcare in any EU country with reimbursement by their home country. Therefore, Member States (MS) cooperation in the evaluation of health technologies is envisaged to ensure safe, high-quality, and efficient healthcare. Several efforts have been put in place at EU level to make this collaboration possible. The cross-border healthcare directive 2011/24/EU have led to the establishment of a voluntary network of MS to facilitate their cooperation and the exchange of scientific information. This network has been supported by another network, namely the European Health Technology Assessment Network (EUnetHTA) that was launched in 2006 with the aim to increase the contribution of HTA to decision-making in EU MS, strengthen the link between HTA and health policymaking, and reduce the overlap and duplication of HTA efforts by promoting a more effective use of resources. EUnetHTA has created a long-lasting Joint Action that has developed principles, methodological guidelines, as well as functional online tools for the sharing production of HTA. In the meantime, starting from 2018, a proposal for an EU regulation on HTA has been discussed within the European Commission first and the European Council and Parliament later. The discussion led to the approval of the Regulation (EU) 2021/2282 on health technology assessment (HTAR) that entered into force on 11 January 2022 and will apply from 12 January 2025 onwards. The HTAR calls for mandatory participation in and use of joint clinical assessments at MS level opening a new era for HTA at EU level. In order to make public health professionals ready for the challenge, this skill-building seminar will provide the audience with basic knowledge in respect to the objective, the application, the methods, and the history of HTA at European level. After two formal presentations on these issues, a case study will lead the following interactive discussion with the audience. Key messages • Health decisions should be informed by the determination of the value of health technologies through shared methods, such as Health Technology Assessment. • Collaboration on Health Technology Assessment at European level has had a long-lasting story and is now looking out into a new challenging era that will be the EU regulation being implemented.

Implementation of Health Technology Assessment and Management Course in Undergraduate Program in Biomedical Engineering ITB.

The Undergraduate Program in Biomedical Engineering ITB, Indonesia, introduce the Health Technology Assessment and Management as an elective course in 2021. This course is implemented to support the World Health Assembly that urges the member states to establish national strategies in health technology assessment and management, particularly medical devices. Furthermore, it is designed to give biomedical engineering students a broader insight into their career opportunities. Therefore, this course is delivered by the practitioner and guided by the main lecturer. The course syllabus is developed from the WHO Medical Devices Technical Series and European Network for Health Technology Assessment. It tries to implement HTA Core Model for Rapid Relative Effectiveness Assessments. A questionnaire is used to measure the students' perception of the course implementation. Moreover, it is used to obtain the students' comments and feedback. The course that is delivered by the practitioner not only gives the course content but also the context. After attending the course, students have a broader insight into the career opportunities as biomedical engineers in Indonesia.

Strengthening the Interface of Evidence-Based Decision Making Across European Regulators and Health Technology Assessment Bodies

ObjectivesAccess to medicines in Europe depends on a benefit-risk decision taken by regulators and a relative effectiveness assessment performed by health technology assessment bodies (HTABs) to inform, as one element, a reimbursement decision. Although various similarities in evidence needs exist, understanding of their needs is currently suboptimal and therefore the evidence generated does not always meet their needs. Subsequently, delays in decision making can be expected, negatively affecting access. To overcome this, this study reviewed the evidentiary needs of European regulators and HTABs at European level and analyzed how their collaboration can further facilitate optimal evidence generation plans, evidence use, and evidence presentation. MethodsThrough systematic literature review, expert interviews, and pairwise comparison of assessment reports by the European Medicines Agency and European network for health technology assessment, respective clinical evidence requirements and impact of product-specific collaboration between European Medicines Agency and HTABs were established. ResultsClinical evidence needs are quite similar but differences exist in comparator choice, preferred efficacy endpoints, and target population. Results of the impact of collaboration to date were mixed: preapproval joint advice procedures were successful and highly valued by all stakeholders; information exchange at the time of regulatory decision is coming together, yet the European Public Assessment Report can be further optimized; and collaboration on postlicensing evidence generation requirements shows potential but needs solidifying. ConclusionsThese findings demonstrate the potential to further improve the evidence utilization across stakeholders to avoid duplication and streamline decision making, to ultimately improve access to medicines for European patients.

European Network for Health Technology Assessment's Response to COVID-19: Rapid Collaborative Reviews on Diagnostic Tests and Rolling and Rapid Collaborative Reviews on Therapeutics.

In spring 2020, The European network for Health Technology Assessment (EUnetHTA) decided to join forces to produce best evidence to inform health policy in the COVID-19 pandemic. The objective of this paper is to describe the process and output of the coordinated and collaborative activities of EUnetHTA. Relevant published and internal documents were retrieved for a descriptive analysis of EUnetHTA processes, methods, and outputs related to EUnetHTA's response to the pandemic. Process: In April 2020, a COVID-19 task force was set up and a survey collected pressing health policy questions across Europe. Two coordinating agencies for diagnostic tests and therapeutics were assigned. A process for prioritization and selection was set up for therapeutics, as well as explicit starting and stopping rules. Methodology: To increase a timely response, it was agreed that the rapid collaborative reviews (rapid CRs) would not require the consultation of manufacturers and the involvement of external experts, but would not differ in the methods and conduct of the systematic search, review, and synthesis of all available evidence, nor in the requirement for reviewing by EUnetHTA partners. Final reports: The joint effort resulted in the production of two rapid CRs on diagnostic tests, nineteen collaborative rolling reviews on therapeutics, three of which later moved to rapid CRs. During COVID-19 pandemic, the EUnetHTA partners proved capable of prompt collaboration, which allowed speeding up the production and release of high-quality EUnetHTA outputs, while the relationships with the other European institutions facilitated their quick dissemination.