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Objectives: The aim of the study was to assess the reimbursement policy for orphan drugs (ODs) in selected European countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedures and reimbursement decision-making. Materials and Methods: A list of authorized ODs was extracted from a web-based registry of the European Medicines Agency, including information on active substance, Anatomical Therapeutic Chemical (ATC) classification code, and therapeutic area. A country-based questionnaire survey was conducted between September 2022 and September 2023 among selected experts from 12 European countries. A descriptive and statistical analysis was performed to identify correlations between country characteristic, HTA procedures, drug indication and positive recommendations or reimbursement decisions for ODs. Results: Safety assessment for ODs was mandatory in 10 countries, while it was optional in one country (Italy) and not required in one country (Iceland). Efficacy assessment for ODs was mandatory in 11 countries and not required in one country (Iceland). The impact of safety and efficacy assessment on reimbursement decisions was rated as high in 10 countries and as low in one country (Germany). Dedicated OD legislation and policies were reported in seven countries. In two countries (Belgium, Iceland), the HTA was not mandatory, and in one country (Germany), it only had an informative function. A positive recommendation (from an HTA agency or advisory body) guaranteed reimbursement in four countries, while a negative recommendation excluded reimbursement only in one country (Iceland). The proportion of reimbursed ODs ranged from 23.5% in Iceland to 86% in Germany (p < 0.001). ODs with ATC code L represented the largest group of medicines (n = 49). They were also very frequently reimbursed ODs in the countries studied, with a mean of 61.8% (p < 0.001). Conclusion: European countries differ in terms of the impact of clinical issues and additional clinical aspects on the reimbursement policy for ODs. Reimbursement decisions were affected by OD-specific legislation, policies, and EMA authorization status. HTA dossiers and procedures significantly influenced reimbursement decisions, although some ODs were reimbursed regardless of the positive or negative recommendations. ATC codes were significantly correlated with reimbursement status and positive recommendation. [ABSTRACT FROM AUTHOR]

Introduction: Access to medicines is a shared goal across healthcare stakeholders. Since health technology assessment (HTA) informs funding decisions, it shapes access to medicines. Despite its wide implementation, significant access variations due to HTA are observed across Europe. This paper elicited the opinions of European stakeholders on how HTA can be improved to facilitate access. Methods: A scoping review identified HTA features that influence access to medicines within markets and areas for improvement, while three access dimensions were identified (availability, affordability, timeliness). Using the Delphi method, we elicited the opinions of European stakeholders to validate the literature findings. Results: Nineteen participants from 14 countries participated in the Delphi panel. Thirteen HTA features that could be improved to optimise access to medicines in Europe were identified. Of these, 11 recorded a positive impact on at least one of the three access dimensions. HTA features had mostly a positive impact on timeliness and a less clear impact on affordability. 'Early scientific advice' and 'clarity in evidentiary requirements' showed a positive impact on all access dimensions. 'Established ways to deal with uncertainty during HTA' could improve medicines' availability and timeliness, while more 'reliance on real-world evidence' could expedite time to market access. Conclusions: Our results reiterate that increased transparency during HTA and the decision-making processes is essential; the use of and reliance on new evidence generation such as real-world evidence can optimise the availability of medicines; and better collaborations between regulatory institutions within and between countries are paramount for better access to medicines. [ABSTRACT FROM AUTHOR]

A health technology assessment (HTA) does not systematically account for the circumstances and needs of children and youth. To supplement HTA processes, we aimed to develop a child-tailored value assessment framework using a multicriteria decision analysis approach. We constructed a multicriteria-decision-analysis-based model in multiple phases to create the Comprehensive Assessment of Technologies for Child Health (CATCH) framework. Using a modified Delphi process with stakeholders having broad disciplinary and geographic variation (N = 23), we refined previously generated criteria and developed rank-based weights. We established a criterion-pertinent scoring rubric for assessing incremental benefits of new drugs. Three clinicians independently assessed comprehension by pilotscoring 9 drugs. We then validated CATCH for 2 childhood cancer therapies through structured deliberation with an expert panel (N = 10), obtaining individual scores, consensus scores, and verbal feedback. Analyses included descriptive statistics, thematic analysis, exploratory disagreement indices, and sensitivity analysis. The modified Delphi process yielded 10 criteria, based on absolute importance/relevance and agreed importance (median disagreement indices = 0.34): Effectiveness, Child-specific Health-related Quality of Life, Disease Severity, Unmet Need, Therapeutic Safety, Equity, Family Impacts, Life-course Development, Rarity, and Fair Share of Life. Pilot scoring resulted in adjusted criteria definitions and more precise score-scaling guidelines. Validation panelists endorsed the framework's key modifiers of value. Modes of their individual prescores aligned closely with deliberative consensus scores. We iteratively developed a value assessment framework that captures dimensions of child-specific health and nonhealth gains. CATCH could improve the richness and relevance of HTA decision making for children in Canada and comparable health systems. • Health technology assessment (HTA) methods for child health technologies are challenged by evidentiary limitations and incomplete consideration of the unique biological and social circumstances and needs of children and youth. Multicriteria decision analysis has emerged as an HTA tool for explicitly considering nonconventional values. • This article presents novel child-specific value assessment criteria based in societal preferences, which reflect the importance of family impacts and long-term flourishing of children beyond immediate health gains obtained through treatment. • A child-tailored multicriteria decision analysis approach facilitating HTA deliberations may broaden the normative lens through which child health technologies are evaluated, contributing to more comprehensive and relevant decision making. [ABSTRACT FROM AUTHOR]

To comprehensively identify and map an exhaustive list of value criteria for the assessment of next-generation sequencing/comprehensive genomic profiling (NGS/CGP), to be used as an aid in decision making. We conducted a systematic review to identify existing value frameworks (VFs) applicable to any type of healthcare technology. VFs and criteria were mapped to a previously published Latin American (LA) VF to harmonize definitions and identify additional criteria and or subcriteria. Based on this analysis, we extracted a comprehensive, evidence-based list of criteria and subcriteria to be considered in the design of a NGS/CGP VF. A total of 42 additional VFs were compared with the LA VF, 88% were developed in high-income countries, 30% targeted genomic testing, and 16% specifically targeted oncology. A total of 242 criteria and subcriteria were extracted; 227 (94%) were fully/partially included in the LA VF; and 15 (6%) were new. Clinical benefit and economic aspects were the most common criteria. VFs oriented to genomic testing showed significant overlap with other VFs. Considering all criteria and subcriteria, a total of 18 criteria and 36 individual subcriteria were identified. Our study provides an evidence-based set of criteria and subcriteria for healthcare decision making useful for NGS/CGP as well as other health technologies. The resulting list can be beneficial to inform decision making and will serve as a foundation to co-create a multistakeholder NGS/CGP VF that is aligned with the needs and values of health systems and could help to improve patient access to high-value technologies. • Value frameworks play a crucial role in enhancing transparency and facilitating informed decision making within the healthcare system. They serve as valuable guides, highlighting key dimensions that need evaluation when assessing various healthcare technologies. • Institute for Clinical Effectiveness and Health Policy recently developed a value framework for diagnostic technologies, based on a targeted systematic review and a participatory process with health technology assessment stakeholders in Latin America. • In this article, we report a systematic review to update the previous framework and develop a comprehensive evidence-based list of criteria and subcriteria that could be considered for creation of a next-generation sequencing/comprehensive genomic profiling-targeted value framework, as well as a starting point for value frameworks targeted to other health technologies. [ABSTRACT FROM AUTHOR]

The new EU Regulation on health technology assessment (HTAR) provides for joint clinical assessments (JCA) of health technologies at EU level. When Member States carry out health technology assessments (HTA) at the national level, they shall give due consideration to the results of a JCA and comply with other obligations of the Regulation. This article aims to clarify what these obligations mean for the Member States and whether JCA results have to be considered outside a national health technology assessment as well. In this context, the question of which processes qualify as 'national HTA' and which requirements need to be fulfilled to trigger the obligations under Article 13 HTAR are discussed in more detail in this paper. [ABSTRACT FROM AUTHOR]

Chronic RhinoSinusitis with Nasal Polyps (CRSwNP) represents a condition mainly caused by the type 2 inflammation presence and marked by the existence of polyps within the nasal and paranasal sinuses. The standard of care includes intranasal steroids, additional burst of systemic steroids, if needed, and surgery. However, recurrence is common, especially among patients with comorbid type 2 inflammatory diseases. Recently, biological drugs, addressing the underlying cause of the disease, have been approved in Italy (dupilumab, omalizumab, and mepolizumab). A Health Technology Assessment was conducted to define multidimensional impact, assuming Italian NHS perspective and a 12-month time horizon. The EUnetHTA Core Model was deployed, using the following methods to analyze the domains: (i) literature evidence; (ii) administration of semi-structured questionnaires to 17 healthcare professionals; (iii) health economics tools to define the economic sustainability for the system. Evidence from NMA and ITC showed a more favorable safety profile and better efficacy for dupilumab compared with alternative biologics. All the analyses, synthesizing cost and efficacy measures, showed that dupilumab is the preferable alternative. Specifically, the cost per responder analysis for dupilumab, exhibiting a 67.0% response rate at Week 52, is notably economical at 14,209EUR per responder. This presents a more economical profile compared with the cost per responder for omalizumab (36.2% response rate) at 24,999EUR and mepolizumab (28.5% response rate) at 31,863EUR. These results underscore dupilumab's potential, not merely in terms of clinical outcomes, but also in terms of economic rationality, thereby solidifying its status as a valid and preferrable alternative in the management of CRSwNP, in the context of the Italian NHS. [ABSTRACT FROM AUTHOR]

Background: There is limited evidence-informed guidance on TISP processes for countries where health technology assessment (HTA) is in a nascent phase. We aimed to explore the range of topic identification, selection and prioritization (TISP) processes and practices for HTA in selected countries and identify aspects relevant to emerging HTA systems. Methods: This mixed design study included a systematic literature review, an electronic survey, and individual interviews. We conducted a systematic literature review with criteria that were developed a priori to identify countries deemed to have a recently formalized HTA system. Based on the literature review, a twenty-three item online survey was shared with the identified countries, we completed follow-up interviews with ten participants who have experience with HTA. We analyzed documents, survey responses and interview transcripts thematically to identify lessons related to TISP processes and practices. Results: The literature review identified 29 nine candidate countries as having a "potential" recently formalized HTA system. Twenty-one survey responses were analyzed and supplemented with ten individual interviews. We found variation in countries' approaches to TISP — particularly between pharmaceutical and non-pharmaceutical interventions. Results indicate that TISP is heavily driven by policy makers, expert involvement, and to a lesser extent, relevant stakeholders. The use of horizon-scanning and early warning systems is uncommon. Interviewee participants provided further insight to the survey data, reporting that political awareness and an institutional framework were important to support TISP. TISP can be optimized by stronger national regulations and legislative structures, in addition to education and advocacy about HTA among politicians and decision-makers. In some settings regional networks have been useful, particularly in the development of TISP guidelines and methodologies. Additionally, the technical capacity to conduct TISP, and access to relevant local data were factors limiting TISP in national settings. Increased network collaboration and capacity building were reported as future needs. Conclusions: This study provides current insights into a topic where there is limited published peer reviewed literature. TISP is an important first step of HTA, and topics should be selected and prioritized based on local need and relevance. The limited capacity for TISP in settings where HTA is emerging may be supported by local and international collaboration to increase capacity and knowledge. To succeed, both TISP and HTA need to be embedded within national health care priority setting and decision-making. More in-depth understanding of where countries are situtated in formalizing the TISP process may help others to overcome factors that facilitate or hinder progress. [ABSTRACT FROM AUTHOR]

In response to rapidly rising pharmaceutical costs, many countries have introduced health technology assessment (HTA) as a 'fourth hurdle'. We evaluated the causal effect of HTA based regulation on access to pharmaceuticals by using the introduction of Germany's HTA system (AMNOG) in 2011. We obtained launch data on pharmaceuticals for 30 European countries from the IQVIA (formerly IMS) database. Using difference-in-difference models, we estimated the effect of AMNOG on launch delay, the ranking order of launch delays and the availability of pharmaceuticals. We then compared the results for Germany to Austria, Czechia, Italy, Portugal and the UK. Across all six countries, launch delay decreased from the pre-AMNOG period (25.01 months) to the post-AMNOG period (14.34 months). However, the introduction of AMNOG consistently reduced the magnitude of the decrease in launch delay in Germany compared to the comparator countries (staggered DiD: + 4.31 months, p = 0.05). Our logit results indicate that the availability of pharmaceuticals in Germany increased as a result of AMNOG (staggered logit: + 5.78%, p = 0.009). We provide evidence on the trade-off between regulation and access. This can help policymakers make better informed decisions to strike the right balance between cost savings achieved through HTA based regulation and access to pharmaceuticals. [ABSTRACT FROM AUTHOR]

Introduction: Patient and public involvement (PPI) is an expectation when conducting research, including Health Technology Assessment (HTA), but practical guidance for those wishing to embed PPI into the grant application process is not always easily accessible. We wanted to ensure that PPI was central when preparing a proposal for an investigator‐led evidence synthesis HTA investigating nonsurgical interventions for pelvic organ prolapse (POP) in women. Here, we describe our methods. Methods: We recruited two patient co‐applicants separately through an open process to help ensure that patient voice was present within our proposal's management and direction. We invited co‐applicants to attend research team meetings and comment on the full proposal. We designed, recruited to and facilitated a scoping workshop, as well as undertook its subsequent evaluation. The insight shared within the workshop for patients with a lived experience of POP, including our patient co‐applicants, helped us develop the scope and rationale behind our HTA proposal. We particularly considered the interventions to include within the evidence synthesis. We also considered the outcome measures for both the evidence synthesis and economic evaluation. We elicited ideas about where and how results could be disseminated. Feedback suggested the workshop was as valuable for the attendees as it was for the researchers, making them feel valued and listened to. The time spent by researchers working on the activity was substantial and not directly funded but a necessary and valuable activity in developing our potential HTA. Our work was informed using the UK Standards for Public Involvement and the Authors and Consumers Together Impacting on eVidencE (ACTIVE) framework. Conclusions: PPI can be enormously valuable in both developing and strengthening research proposals. However, further guidance is needed to help researchers recognise the level and type of involvement to use at this early stage, particularly given the large time investment needed to embed meaningful PPI. Patient and Public Contribution: Women with a lived experience of POP were involved at every stage of the grant application process; their involvement is documented in full throughout this work. [ABSTRACT FROM AUTHOR]

Background: The present paper aims at evaluating the potential benefits of high-energy devices (HEDs) in the Italian surgical practice, defining the comparative efficacy and safety profiles, as well as the potential economic and organizational advantages for hospitals and patients, with respect to standard monopolar or bipolar devices. Methods: A Health Technology Assessment was conducted in 2021 assuming the hospital perspective, comparing HEDs and standard monopolar/bipolar devices, within eleven surgical settings: appendectomy, hepatic resections, colorectal resections, cholecystectomy, splenectomy, hemorrhoidectomy, thyroidectomy, esophago-gastrectomy, breast surgery, adrenalectomy, and pancreatectomy. The nine EUnetHTA Core Model dimensions were deployed considering a multi-methods approach. Both qualitative and quantitative methods were used: (1) a systematic literature review for the definition of the comparative efficacy and safety data; (2) administration of qualitative questionnaires, completed by 23 healthcare professionals (according to 7-item Likert scale, ranging from − 3 to + 3); and (3) health-economics tools, useful for the economic evaluation of the clinical pathway and budget impact analysis, and for the definition of the organizational and accessibility advantages, in terms of time or procedures' savings. Results: The literature declared a decrease in operating time and length of stay in using HEDs in most surgical settings. While HEDs would lead to a marginal investment for the conduction of 178,619 surgeries on annual basis, their routinely implementation would generate significant organizational savings. A decrease equal to − 5.25/−9.02% of operating room time and to − 5.03/−30.73% of length of stay emerged. An advantage in accessibility to surgery could be hypothesized in a 9% of increase, due to the gaining in operatory slots. Professionals' perceptions crystallized and confirmed literature evidence, declaring a better safety and effectiveness profile. An improvement in both patients and caregivers' quality-of-life emerged. Conclusions: The results have demonstrated the strategic relevance related to HEDs introduction, their economic sustainability, and feasibility, as well as the potentialities in process improvement. [ABSTRACT FROM AUTHOR]

New therapies in a publicly funded healthcare system are first appraised by health technology assessment agencies that provide funding recommendations to the payers. Treatment with Chimeric Antigen Receptor-T cell (CAR-T) therapy is revolutionizing the management of patients with relapsed/refractory aggressive B-cell lymphoma by providing an effective alternative to the standard of care. Yet, the implementation of CAR-T treatment has a substantial impact on the healthcare system due to its high cost, complex manufacturing process, and requirement for highly specialized services and expertise. CAR-T Cells, as a "living drug", are fundamentally different from usual medications, and their approvals and funding recommendations pose unique challenges to the health technology agency. In this paper, we explore the specific challenges that face the health technology agencies in reviewing reimbursement recommendations for CAR-T therapy. We take a Canadian perspective and use CAR-T treatment of relapse/refractory aggressive B-cell lymphoma as an example. [ABSTRACT FROM AUTHOR]

Background: Few antibiotics have entered the market in recent years despite the need for new treatment options. Some of the challenges of bringing new antibiotics to market are linked to the marketing authorization and health technology assessment (HTA) processes. Research shows great variation in geographic availability of new antibiotics, suggesting that market introduction of new antibiotics is unpredictable. We aimed to investigate regulatory authorities' and HTA agencies' role in developing non-financial incentives to stimulate antibiotic research and development (R&D). Methods: We conducted individual, semi-structured, stakeholder interviews. Participants were recruited from regulatory authorities (EMA and FDA) and HTA agencies in Europe. Participants had to be experienced with assessment of antibiotics. The data were analyzed using a deductive and inductive approach to develop codes and identify key themes. Data were analyzed using thematic analysis including the constant comparison method to define concepts, and rival thinking to identify alternative explanations. Results: We found that (1) interpretation of key concepts guiding the understanding of what type of antibiotics are needed vary (2) lack of a shared approach on how to deal with limited clinical data in the marketing authorization and HTA processes is causing barriers to getting new antibiotics to market (3) necessary adaptations to the marketing authorization process causes uncertainties that transmit to other key stakeholders involved in delivering antibiotics to patients. Conclusions: A shared understanding of limited clinical data and how to deal with this issue is needed amongst stakeholders involved in antibiotic R&D, marketing authorization, and market introduction to ensure antibiotics reach the market before resistance levels are out of control. Regulatory authorities and HTA agencies could play an active role in aligning the view of what constitutes an unmet medical need, and direct new economic models towards stimulating greater diversity in the antibiotic armamentarium. [ABSTRACT FROM AUTHOR]

As innovative oncology medicines are rapidly developed, there is increasing pressure on payers to offer patients timely access to life-saving therapies. The uncertainty surrounding these therapies when phase III clinical trials are pending has necessitated new, adapted pathways to market access, with timelines that greatly vary by country. Understanding differences between pathways may identify opportunities to expedite patient access universally. To describe early access pathways for new oncology medicines among selected countries with established health technology assessment (HTA) frameworks and publicly funded health systems, with a special focus on real-world evidence (RWE). We reviewed the HTA agency websites of the selected OECD countries: National Institute for Health and Care Excellence (NICE) for England and Wales; Haute Autorité de Santé (HAS) for France; IQWiG and G-BA for Germany; Agenzia Italiana del Farmaco (AIFA) for Italy; Pharmaceutical Benefits Advisory Committee (PBAC) for Australia; and CADTH and Institut National d'Excellence en Santé et Services Sociaux (INESSS) for Canada as the primary source of evidence. Processes for early patient access to innovative oncology therapies varied across selected countries; however, most countries have an established pathway for publicly funded early access (England and Wales, France, Germany, Italy, and Australia). The utilization of RWE to support earlier access (coverage with evidence) also varied by country, with some HTA organizations being actively engaged in these agreements (NICE, AIFA, and HAS) and others having no established processes in place (G-BA and CADTH/INESSS). This review of early access pathways for novel oncology medicines found substantial variability between countries of interest. Coverage with evidence frameworks may provide a unique opportunity for industry and payers to collaborate on earlier access to innovative cancer therapies with life-saving potential. [ABSTRACT FROM AUTHOR]

Objectives: Dealing with uncertainty is one of the critical topics in health technology assessment. The greater decision uncertainty in appraisals, the less clear the clinical- and cost-effectiveness of the health technology. Although the development of targeted cancer therapies (TCTs) has improved patient health care, additional complexity has been introduced in drug appraisals due to targeting more specific populations. Real-world data (RWD) are expected to provide helpful information to fill the evidence gaps in appraisals. This study compared appraisals of TCTs with those of non-targeted cancer therapies (non-TCTs) regarding sources of uncertainty and reviewed how RWD have been used to supplement the information in these appraisals.Methods: This study reviews single technology appraisals (STAs) of oncology medicines performed by the National Institute for Health and Care Excellence (NICE) over 11 years up to December 2021. Three key sources of uncertainty were identified for comparison (generalisability of clinical trials, availability of direct treatment comparison, maturity of survival data in clinical trials). To measure the intensity of use of RWD in appraisals, three components were identified (overall survival, volume of treatment, and choice of comparators).Results: TCTs received more recommendations for provision through the Cancer Drugs Fund (27.7, 23.6% for non-TCT), whereas similar proportions were recommended for routine commissioning. With respect to sources of uncertainty, the external validity of clinical trials was greater in TCT appraisals (p = 0.026), whereas mature survival data were available in fewer TCT appraisals (p = 0.027). Both groups showed similar patterns of use of RWD. There was no clear evidence that RWD have been used more intensively in appraisals of TCT.Conclusions: Some differences in uncertainty were found between TCT and non-TCT appraisals. The appraisal of TCT is generally challenging, but these challenges are neither new nor distinctive. The same sources of uncertainty were often found in the non-TCT appraisals. The uncertainty when appraising TCT stems from insufficient data rather than the characteristics of the drugs. Although RWD might be expected to play a more active role in appraisals of TCT, the use of RWD has generally been limited. [ABSTRACT FROM AUTHOR]

How will market access and value demonstration evolve? In this episode of the Value Insider podcast, host Mike Chambers speaks with Ass. Prof. Panos Kanavos about the future of value assessments in healthcare. Dr Kanavos is Associate Professor of the International Health Policy in the Department of Health Policy at London School of Economics and Political Science as well as Deputy Director at LSE Health and Program Director of the Medical Technology Research Group (MTRG), and advisor to prominent organizations including the European Commission, the European Parliament, the World Bank, the WHO and OECD. Discussing key changes, Dr Kanavos outlines which opportunities and challenges will alter the way we value healthcare interventions. [ABSTRACT FROM AUTHOR]

How is a cost-effectiveness analysis constructed and used? In this episode of the Value Insider podcast, host Mike Chambers speaks with Prof. Maureen Rutten-van Mölken about economic evaluations in healthcare and how value is considered in these evaluations. Prof. Rutten-van Mölken is professor of Economic Evaluation of Innovations for Health at the Erasmus University Rotterdam in the Netherlands. She leads the Erasmus HTA department and is Scientific Director of the Institute for Medical Technology Assessment (iMTA). Prof Rutten-van Mölken explains how comparative assessments can help us understand which treatments offer the best value-for-money as reimbursement can only be allocated to a selection of all possible treatments. [ABSTRACT FROM AUTHOR]

Pharmaceutical industry investments in Italy must be supported with a reform process that promotes simplifications to generate an attractive ecosystem that can enhance innovation. Key actions include facilitating the start-up of clinical trials, promoting public-private partnerships to support technology transfer, integrating data infrastructures to overcome the logic of silos, expanding programs for prevention and early identification of diseases, simultaneous reimbursement for therapy and diagnostic testing, and launching a structural program for early access to therapies. The total contribution of the sector in the last 10 years to the Italian economy was € 315 bn. Looking at employment (67 thousand people employed in the sector in 2020), it is possible to estimate about 280 thousand jobs activated in Italy just in 2020. An Altems study quantified a leverage effect of 2.77 in terms of benefits to the Italian NHS from direct investment in clinical research, which means that for every € 1 invested by companies in clinical trials, € 1.77 of additional savings were generated for the NHS. Applying the multiplier on R&D investments of the entire pharmaceutical sector in 2020 (€ 1.6 bn), we could estimate approximately € 4.4 bn in benefits for the NHS. In addition to these benefits there are the noneconomic benefits of clinical research, including early access for patients to innovative therapies, resulting in improved clinical outcomes and quality of life for patients and caregivers in general.

The importance of payers and HTA: How did we end up here? In this episode of the Value Insider podcast, host Mike Chambers speaks with Prof. Mike Drummond about Health Technology Assessment (HTA) and how value is defined. Prof. Drummond is professor of Health Economics of York University, former president of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR), and author of two major textbooks in the field, as well as over 700 publications, and has acted as consultant to the WHO as well as European Union with regards to value assessment. Starting from the very beginning, Prof. Drummond explains in a simple yet engaging way why demonstrating value of new interventions has become so important. [ABSTRACT FROM AUTHOR]

There is an alarming delay in Europe for anticancer medicines becoming accessible for children. Following a paediatric European Union marketing authorisation, national Health Technology Assessment (HTA) agencies evaluate effectiveness, and safety of medicines to support decision on their cost and reimbursement. This study (a SIOPE Access to Medicines project) aimed to evaluate how these HTA evaluations take place for anticancer medicines indicated for paediatric use in Europe and to explore where the delays for market access originate. We obtained HTA reports from the public domain for nine European countries for blinatumomab, dinutuximab beta and tisagenlecleucel. We evaluated the time elapsed between marketing authorisation for a paediatric indication and a national HTA decision and the nature of the decision. Out of 23 HTA decisions (four countries without blinatumomab report), 18 were positive, two with restrictions, three negative. For blinatumomab, tisagenlecleucel and dinutuximab beta, the median time to an HTA decision after regulatory approval for paediatric use was 353 days (range 193–751), 141 days (range 77–517) and 515 days (range 0–780), respectively, with variability between countries. Dinutuximab beta and tisagenlecleucel were first introduced in children, but did not result in shorter time to HTA decision. For blinatumomab, marketing authorisation followed 1008 days after the indication in adults, with HTA applications submitted a median of 167 days later, and a recommendation after 145 days. This study reveals ample variability in HTA decision making in nine European Union countries. Collaboration and alignment of required evidence is needed to facilitate robust scientific HTA assessments, also considering methodological challenges in paediatric oncology. • Health technology assessments of paediatric oncology drugs vary greatly within Europe. • The timing of HTA evaluation adds to the delay of patient access. • Differences between adult and paediatric trial methodology should be considered. • Multistakeholder collaboration between EMA, academia, pharmaceutical companies, patients advocates and HTAs is needed. [ABSTRACT FROM AUTHOR]

Background: The Health Technology Assessment (HTA) has encountered different issues and challenges over the last two decades. The main purpose of this research is to review the issues and challenges in high- and middle-income countries through reviewing studies related to the HTA.Methods: The HTA area literature of different countries was collected from 2009 to 2020 and analyzed using scoping review, based on Scopus and WoS databases.Results: Given the fact that the HTA is practically done in high- and middle-income countries, the results of reviewing the studies and articles of countries reveal that high-income countries seek to increase the participation of stakeholders and enhance the transparency of processes, policy-making, and regulation of the HTA, as well as the systematization of various participant institutions in this area. Middle-income countries, on the other hand, are mostly involved in raising awareness, training and skill development of HTA-related staff, institutionalizing the concept of HTA, and allocating appropriate resources for effective and safe decision-making in their health system.Conclusion: The problem of incoordination between stakeholders (participant institutions) in the HTA, and thereby, problems in decision-making were found in many of the studied reports and articles. Thus, one of the useful efforts to be made by different countries to maintain the integrity of this system would be the process of involving all members of this system and the formation of a healthy ecosystem in the HTA. [ABSTRACT FROM AUTHOR]

Objective This analysis aimed to assess patient advocacy groups' (PAGs) perspectives on patients' and PAGs' potential role in Health Technology Assessment (HTA) decision-making in Greece. Methods In total, 22 representatives of PAGs participated in a consensus panel meeting. Participants' views were elicited via a consensus panel meeting, through a televoting procedure, based on a structured questionnaire with close-ended questions. Voting was anonymous, to avoid the influence of the dominant personality. After voting, the results were presented to the participants and televoting was repeated to achieve consensus. Key findings The majority of participants (94.8%) believe that valid information on the developments regarding HTA in Greece is not conveyed to them by the official national regulatory sources. Key sources of valid information on developments regarding HTA were: conferences (95.2%), international PAGs (85.7%) and HTA experts/scientists (76.2%). About 76.2% of participants evaluated PAGs' competence to participate in policy-making concerning HTA scheme formation as moderate or higher. Regarding patients' effective participation in HTA decision-making, greater importance (points 4 and 5 on a 5-point scale) was given to their education on treatment effects in terms of quality of life (100.0%), basic principles of health economics and pharmaceutical policy (95.5%) and ethical aspects of HTA (95.0%). About 55.0% strongly agree that patients should formally express their opinion on HTA issues, while 72.7% believe that patients' opinions on HTA issues should be expressed through participation in the process and right to vote. Conclusions The participation of patients in HTA decision-making is an issue not only of justice but also of essence. For a productive participation, PAGs training is essential. [ABSTRACT FROM AUTHOR]

This paper is aimed at addressing all the critical aspects linked to the implementation of intensive care ventilators in a pediatric setting, highlighting the most relevant technical features and describing the methodology to conduct health technology assessment (HTA) for supporting the decision-making process. Four ventilator models were included in the assessment process. A decision-making support tool (DoHTA method) was applied. Twenty-eight Key Performance Indicators (KPIs) were identified, defining the safety, clinical effectiveness, organizational, technical, and economic aspects. The Performance scores of each ventilator have been measured with respect to KPIs integrated with the total cost of ownership analysis, leading to a final rank of the four possible technological solutions. The final technologies’ performance scores reflected a deliver valued, contextualized, and shared outputs, detecting the most performant technological solution for the specific hospital context. HTA results had informed and supported the pediatric hospital decision-making process. This study, critically identifying the pros and cons of innovative features of ventilators and the evaluation criteria and aspects to be taken into account during HTA, can be considered as a valuable proof of evidence as well as a reliable and transferable method for conducting decision-making processes in a hospital context. [ABSTRACT FROM AUTHOR]

The definition of criteria and processes for the submission of price and reimbursement requests (P&R) of a drug in Italy cannot be separated from the definition of an overall “Pharmaceutical Policy” that includes, in an organic vision: (i) the governance related to the research, marketing and monitoring of drugs in the Italian market; (ii) the availability of drugs on the national territory as an element included in the essential levels of care (LEA) and the related conditions in terms of timing and equity of access between different regions, as well as towards other European countries; (iii) the assessment criteria used. The decree published in the Italian Official Journal in the summer of 2020, which defines the new criteria for the regulation of P&R of medicines in Italy, focuses on the final part of the process, i.e. the price negotiation. It would be necessary to frame this last step within a broader and more organic structure of drug policies aimed at: 1. optimising healthcare funding by encouraging competition between healthcare technologies; 2. reducing assessment time by simplifying processes; 3. improving early access to drugs for unmet need; 4. increasing the quality of P&R dossiers by improving interaction with the companies; 5. encouraging innovative agreements and complementary elements to the price; 6. encouraging the reproducibility of assessment methodologies in a value-based pricing system; 7. considering cost-benefit analyses as tools for the definition of price and conditions of reimbursability; 8. creating a place for discussion on drug policies.

Objectives: A recent debate in health economics and outcomes research community identified option value as one of the elements warranting consideration in the assessment of medical technologies. To conduct a scoping review of contributions on option value in the healthcare sector and identify relevant conceptual aspects and methods used to incorporate it in standard economic evaluations.Methods: A systematic search was conducted up to July 2020 to identify contributions from electronic bibliographic database and gray literature. Data on the proposed definitions of option value, theoretical implications of its use in economic evaluations, and methods used to estimate it were extracted and analyzed.Results: We found 57 eligible studies. Three different definitions emerged: insurance value, real option value, and option value of survival. Focusing on the latter (24 studies), we analyzed in depth 8 empirical applications across 7 therapeutic areas. The most relevant methodological challenges were on the perspective used in economic evaluations and how to robustly manage forecasting uncertainty, update cost-effectiveness thresholds, and avoid double-counting issues. For empirical studies assessing the total value of the technology, including option value, estimates ranged from +7% to +469% of its conventional value.Conclusions: This review synthesizes theoretical and empirical aspects on option value of healthcare technologies and proposes a terminology to distinguish 3 different concepts identified. Future work should focus primarily on agreeing on whether option value should be included in economic evaluations and, if so, on developing and validating reliable methods for its ex-ante estimation. [ABSTRACT FROM AUTHOR]

Standard outcome sets developed by the International Consortium for Health Outcomes Measurement (ICHOM) facilitate value-based health care in healthcare practice and have gained traction from regulators and Health Technology Assessment (HTA) agencies that regularly assess the value of new medicines. We aimed to assess the extent to which the outcomes used by regulators and HTA agencies are patientrelevant, by comparing these to ICHOM standard sets. We conducted a cross-sectional comparative analysis of ICHOM standard sets, and publicly available regulatory and HTA assessment guidelines. We focused on oncology due to many new medicines being developed, which are accompanied by substantial uncertainty regarding the relevance of these treatments for patients. A comparison of regulatory and HTA assessment guidelines, and ICHOM standard sets showed that both ICHOM and regulators stress the importance of disease-specific outcomes. On the other hand, HTA agencies have a stronger focus on generic outcomes in order to allow comparisons across disease areas. Overall, similar outcomes are relevant for market access, reimbursement, and in ICHOM standard sets. However, some differences are apparent, such as the acceptability of intermediate outcomes. These are recommended in ICHOM standard sets, but regulators are more likely to accept intermediate outcomes than HTA agencies. A greater level of alignment in outcomes accepted may enhance the efficiency of regulatory and HTA processes, and increase timely access to new medicines. ICHOM standard sets may help align these outcomes. However, some differences in outcomes used may remain due to the different purposes of regulatory and HTA decision-making. [ABSTRACT FROM AUTHOR]

Background: Health technology assessment (HTA) agencies have an important role in the evaluation and approval of new technologies. They determine their value within a health system so to promote equitable, quality care with available healthcare resources. Many HTA agencies have some mechanism for involving patients in their processes, but there is great variability and an absence of comprehensive, robust practices for involvement. The accelerating pace of medical innovation creates a need to improve the depth and breadth of patient involvement in the HTA process. Main body: In this 'Call to action', we present ideas from three HTA expert commentaries calling for collaborative learning and to share innovative ideas for changes in HTA. We also draw on examples of HTA agencies creatively pursuing this goal. We propose a 'Call to action' for HTA stakeholders to undertake serious dialogue with patient advocates aimed at creating shared goals. HTA agencies can use these goals to ensure meaningful patient involvement at every step of the HTA process. Five elements are explored. In 'Recognizing the value of shared purpose', we highlight examples of HTA agencies that have patients working in partnership with medical practitioners and HTA staff. Results include improved processes that instil confidence. 'Committing to patient involvement as part of HTA culture' highlights several initiatives aimed at changes in HTA organisational culture to be more inclusive of patients. In 'Aligning patient and HTA goals' we cite work in Belgium and New Zealand which places a greater emphasis on quality of life rather than life expectancy and cost-effectiveness. By 'Integrating patient involvement at every step of the HTA process' patients can make vital contributions at every stage of the HTA process. We provide two examples of where HTA agencies have successfully involved patients early in the process in order to broaden the scope of evaluations. 'Developing a common language and working together' can support transformative dialogue through 'unified language'. Conclusion: The authors of this commentary ask that agencies and stakeholders involved in HTA take up this call to work together for visionary and transformative elevation of the voice of patients in HTA worldwide. [ABSTRACT FROM AUTHOR]

In this viewpoint, we discuss the approach promoted by the Italian Drug Agency (AIFA) to the assessment of the value of new pharmaceuticals in the Italian Healthcare Service. On top of traditionally acknowledged components, such as quality adjusted life years gained and net costs, the overall value framework might include other elements such as productivity and adherence, equity, severity of disease, reduction in uncertainty, spillover effects. There is a residual dimension in the value framework that may capture the option value or reduction in fear of contagion for infectious disease treatments. We debate measurement issues on these elements of value and discuss open issues from a methodological and policy standpoint.

The article presents the concept of the HB-HTA (Hospital-Based Health Technology Assessment) project implementation as one of the options for implementing HB-HTA in Poland. The model involves the creation of structures in selected hospitals dealing with the assessment of technologies that would be implemented in the units. The model is characterized by a small number of stakeholders and their interests do not conflict with one another. The basic condition for the success of the project is the reimbursement of the mentioned technologies, which would allow financing the described activities. The implementation of the project requires, above all, specialized staff (already present on the market), supported by appropriate technical and organizational solutions. The implementation of the project in the proposed version should bring benefits in the entire healthcare system - improved access to services, as well as improved quality of hospital management and improved financial results of the hospitals and the system itself. [ABSTRACT FROM AUTHOR]

The new European Union (EU) Regulations on medical devices and on in vitro diagnostics provide manufacturers and Notified Bodies with new tools to improve pre-market and post-market clinical evidence generation especially for high-risk products but fail to indicate what type of clinical evidence is appropriate at each stage of the whole lifecycle of medical devices. In this paper we address: i) the appropriate level and timing of clinical evidence throughout the lifecycle of high-risk implantable medical devices; and ii) how the clinical evidence generation ecosystem could be adapted to optimize patient access. The European regulatory and health technology assessment (HTA) contexts are reviewed, in relation to the lifecycle of high-risk medical devices and clinical evidence generation recommended by international network or endorsed by regulatory and HTA agencies in different jurisdictions. Four stages are relevant for clinical evidence generation: i) pre-clinical, pre-market; ii) clinical, pre-market; iii) diffusion, post-market; and iv) obsolescence & replacement, post-market. Each stage has its own evaluation needs and specific studies are recommended to generate the appropriate evidence. Effective lifecycle planning requires anticipation of what evidence will be needed at each stage. [ABSTRACT FROM AUTHOR]

In this paper we have compiled and summarized the steps which manufacturers and clinical investigators need to undertake to perform a target population definition during the Health technology assessment process of medical device, namely external cardio stimulator (pacemaker). Based on available data using top-down approach we have defined target population for external cardiostimulator and estimated, that the size of the target population for external pacing will not exceed 16000 patients per year in the Czech Republic and is of comparable size with other states in the region. [ABSTRACT FROM AUTHOR]

Background: Decision-making in public health and health policy is complex and requires careful deliberation of many and sometimes conflicting normative and technical criteria. Several approaches and tools, such as multi-criteria decision analysis, health technology assessments and evidence-to-decision (EtD) frameworks, have been proposed to guide decision-makers in selecting the criteria most relevant and appropriate for a transparent decision-making process. This study forms part of the development of the WHO-INTEGRATE EtD framework, a framework rooted in global health norms and values as reflected in key documents of the World Health Organization and the United Nations system. The objective of this study was to provide a comprehensive overview of criteria used in or proposed for real-world decision-making processes, including guideline development, health technology assessment, resource allocation and others. Methods: We conducted an overview of systematic reviews through a combination of systematic literature searches and extensive reference searches. Systematic reviews reporting criteria used for real-world health decision-making by governmental or non-governmental organization on a supranational, national, or programme level were included and their quality assessed through a bespoke critical appraisal tool. The criteria reported in the reviews were extracted, de-duplicated and sorted into first-level (i.e. criteria), second-level (i.e. sub-criteria) and third-level (i.e. decision aspects) categories. First-level categories were developed a priori using a normative approach; second- and third-level categories were developed inductively. Results: We included 36 systematic reviews providing criteria, of which one met all and another eleven met at least five of the items of our critical appraisal tool. The criteria were subsumed into 8 criteria, 45 sub-criteria and 200 decision aspects. The first-level of the category system comprised the following seven substantive criteria: "Health-related balance of benefits and harms"; "Human and individual rights"; "Acceptability considerations"; "Societal considerations"; "Considerations of equity, equality and fairness"; "Cost and financial considerations"; and "Feasibility and health system considerations". In addition, we identified an eight criterion "Evidence". Conclusion: This overview of systematic reviews provides a comprehensive overview of criteria used or suggested for real-world health decision-making. It also discusses key challenges in the selection of the most appropriate criteria and in seeking to implement a fair decision-making process. [ABSTRACT FROM AUTHOR]

Context: The National Institute of Excellence in Health and Social Services (INESSS), which functions as the Québec health technology assessment (HTA) agency, tested a new way to engage patients along with health‐care professionals in the co‐construction of recommendations regarding implantable cardioverter‐defibrillator replacement. Objective: The objective of this article was to describe the process of co‐construction of recommendations and to propose methods of building best practices for patient involvement (PI) in HTA. Design: Throughout the process, documents were collected and participant observations were made. Individual interviews were conducted with patients, health‐care professionals and the INESSS scientific team, from January to March 2018. Results: Three committees were established: an expert patient committee to reflect on patient experience literature; an expert health professional committee to reflect on medical literature; and a co‐construction committee through which both patients and health‐care professionals contributed to develop the recommendations. The expert patients validated and contextualized a literature review produced by the scientific team. This allowed the scientists to consider aspects related to the patient experience and to integrate the feedback from patients into HTA recommendations. The most important factor contributing to a positive PI experience was the structured methodology for selecting patient participants, and a key factor that inhibited the process was a lack of training in PI on the part of the scientific team. Conclusions: This experience demonstrates that it is possible to co‐construct recommendations, even for technically complex HTA subjects, through a more democratic process than usual which led to more patient‐focused guidance. [ABSTRACT FROM AUTHOR]

India is a diverse land with different cultures, social norms, castes, religions, faiths, languages, politics, and a complex healthcare system. As a step to enhance healthcare, the government of India announced a move toward universal health coverage to increase accessibility and affordability of health-related services. Recently, there has been an introduction of health technology assessment (HTA) in India to help inform evidence-based decision making in cases of limited resources and budgets. Nevertheless, there are challenges related to biased decision making, an unregulated healthcare framework, and the lack of data and capacity that will (directly or indirectly) affect the use of HTA in India. For HTA to be successful in India and in similar low- and middle-income countries, it is important that the decision makers acknowledge these challenges and embrace differences in ideologies, cultures, and politics instead of ignoring them. Drawing lessons from countries with well-developed HTA bodies may help, but these need to be modified for the country-specific context. Ensuring quality and transparency is key to building trust in medical decision making. Improved coordination at all levels of healthcare is vital to ensure the long-term success of HTA in India. This is challenging but achievable by spreading awareness among stakeholders and achieving moderate health-sector regulation that can combat corruption. HTA will prosper in India if it incorporates cultural and institutional diversity, alongside tackling socioeconomic inequalities. [ABSTRACT FROM AUTHOR]

Oral communications

Reimbursement decisions on new oncology drugs are now often made while uncertainty remains about a drug's risk–benefit profile. One consequence of this is a delay in patient access to valuable new medicines. We share our perspectives on strategies to mitigate sources of uncertainty in the health technology assessment process. These include flexible approaches for evaluating the additional benefit, such as better use of surrogate endpoints and health-related quality of life data, and renewed research efforts to define the optimal target population and generate real-world evidence post-authorisation. [ABSTRACT FROM AUTHOR]

The Agency for Health Technology Assessment and Tariff System is national institution supervised by Minister of Health and funded by government. Agency is an advisory body in the reimbursement process. Agency has an impact on content of benefit basket, not only by evaluating new technologies but also re-evaluating benefit basket. Main goal of Agency is to provide patients access to the most effective and safe technologies. The most important Agency statements concern medical technologies and its modifications, qualification, removal in reference to guaranteed medical services. The Minister of Health, taking decisions about which health technology should be financed by public found, takes into account: recommendations and opinions of President of Agency, opinions and statements of the Transparency Council and the Council for Tariffs Affairs. The Agency's task is also: setting tariffs for health care services; evaluating diagnostic and therapeutic guidelines prepared by medical societies; preparing opinions about national and local government health care programs. [ABSTRACT FROM AUTHOR]

The Analytic Hierarchy Process (AHP) is a methodology, based on both mathematical and psychological approaches, exploited to analyze and solve complex problems, in order to make the best decision. AHP is also widely employed for the evaluation of healthcare systems. It works by splitting the decision problem into a hierarchy of more easily-comprehended sub-problems, each of which can be independently analyzed. Starting from these assumptions, our work aims to implement a dynamic framework for the AHP methodology, able to overcome the static nature of the technique, taking into account the evaluations associated with criteria and sub-criteria as a function of time. In the context of this paper, the Dynamic AHP is implemented as a tool for Health Technology Assessment (HTA). Indeed, the proposed case study concerns the evaluation of a new health technology for the thyroglobulin assay in patients with differentiated thyroid cancer. To this aim, we have defined the decision problem and formalized criteria and sub-criteria hierarchically; then, in order to deal with the problem from a dynamical point of view, i.e., to take into account the time dependence of criteria and sub-criteria, we developed several System Dynamics models, one for each criterion. In this context, it is shown that the Dynamic AHP approach provides a dynamic evaluation of the system, which allows to identify the best decision by exploiting the information on the possible evolution of the problem due to its time behavior, rather than taking the decision at a fixed time point, as the Static AHP does. [ABSTRACT FROM AUTHOR]

Purpose: Centrally authorised medicinal products (CAMPs) in the European Union may offer added therapeutic value (ATV) but may be linked to high prices and limited efficiency. Health technology assessment (HTA) and managed entry schemes (MES) may facilitate the reimbursement decision by providing reliable estimates of the medicinal product's value and costs and by controlling the remaining uncertainty, respectively. We investigated the impact of HTA criteria and the initiation of a MES on the reimbursement decision of CAMPs in Belgium. Methods: We selected all reimbursement submissions for new centrally authorised medicinal products in the 2010–2015 period. We retrieved data relating to the reimbursement decision, the HTA outcome and the use of a managed entry scheme. Results: The decision of the Minister was available for 115 dossiers, covering 36 (31.3%) orphan medicinal products (OMPs) and 79 ATV products. A MES was used in 41 submissions. A positive reimbursement decision was obtained in 65% of cases. The significant factors affecting the reimbursement decision were the approval of ATV, the medical need if it was considered 'important or major' and the use of a managed entry scheme. Price, budget impact and efficiency had no significant impact. Conclusions: Added therapeutic value and high medical need increase the odds for a positive reimbursement decision. No impact could be demonstrated of the cost-related HTA criteria. Cost elements may be biased by the use of a confidential MES. Without a MES, only 53% of the centrally authorised medicinal products, including OMPs, are reimbursed in Belgium. [ABSTRACT FROM AUTHOR]

Several Multi-Criteria Decision Analysis (MCDA) models for use in health technology assessment (HTA) have been developed over the years, including some for orphan drugs. However, there is no general consensus yet on MCDA structure and which criteria should be included and implementation of MCDA into HTA practice has been slow so far. Our study tested the criteria preferences and possibilities for implementation of the EVIDEM MCDA framework for orphan drugs with a diverse group of 140 stakeholders in Kazakhstan, Netherlands, Poland, Romania, Russia, Turkey and Ukraine (KZ, NL, PL, RO, RU, TR, UA). The research elicited stakeholder preferences (weighting) for EVIDEM domains, criteria and sub-criteria to measure their relative importance. Correlations of the workplace and HTA/rare diseases experience with weighting results were investigated. Results showed that the 'Need for intervention' domain was assessed as the most important in RO/NL/RU/TR, in KZ/PL the 'Type of benefit of intervention' and in UA 'Economic consequences of intervention'. 'Population Priorities' was uniformly given a low priority. Further research, sharing of experiences and multi-stakeholder discussion is necessary to define a path forward for a robust and sustainable improvement of MCDA models and its application in orphan drug HTA. Model simplification and clarification of outcomes would be beneficial. [ABSTRACT FROM AUTHOR]

Background: Proportional hazards (PH) is an assumption often made by researchers, despite evidence of nonproportionality in a significant proportion of clinical trials. In the presence of non-PH, the interpretation of hazard ratios, medians, and landmark survival as summary measures of treatment effect can become problematic. Several recent studies have recommended restricted mean survival time (RMST) as an alternative metric for survival analysis, particularly where non-PH may apply.Objectives: To determine the current approaches of health technology assessment (HTA) agencies to value assessment in the presence of non-PH, and the extent to which RMST is accepted as an alternative measure of treatment benefit.Methods: Methodological guidelines published by 10 HTA agencies were reviewed to establish recommended approaches for presenting survival benefit from clinical trials. Published HTA reports for 23 oncology agents approved by the US Food and Drug Administration and the European Medicines Agency since 2014 were reviewed to determine how guidelines are implemented in practice and identify instances where the PH assumption was tested and RMST analyses reported.Results: Testing for non-PH is not widely incorporated into HTA except by the UK National Institute for Health and Care Excellence. RMST is used infrequently but has been used in a number of countries, particularly by agencies that focus on cost effectiveness.Conclusions: HTA agencies vary in their approaches to non-PH. Most do not routinely check the PH assumption. RMST has played a role in assessing clinical benefit within HTA, although not consistently within countries (across drugs) or across countries (for the same drug). [ABSTRACT FROM AUTHOR]

Health technology assessment is a well-established scientific methodology. It serves healthcare managers and policymakers to make informed decisions about new medicines and medical devices reimbursement. HTA is a relatively new process for Bulgaria, which is constantly evolving and changing. Objective: Study of the of awareness level of the HTA process, as well as to collect and analyze data from the experts' opinions, representatives of pharmaceutical companies, media, patients and the society about the organization of HTA process by 31.03.2019, incl. evaluation of the regulatory changes, introduced after this date. Materials and methods: A survey was conducted among 150 respondents: academic lecturer, physicians, experts in the field of HTA, representatives of the pharmaceutical industry and the media, patients. An online-based anonymous questionnaire, containing 19 closed and open-ended questions was used. Results: The relative share of respondents who said they were familiar with the HTA process was 88% and 12% have answered negatively. 13% of respondents said that they did not know the place and the role of HTA in the health care system, medicines policy and accordingly making decisions about access to new technologies. A large part (67%) of the respondents believe that the HTA process has started successfully in our country, which gives a predominantly positive assessment of the activity carried out so far. Approximately one quarter (22%) of the respondents said „I don't know" and only 11% said that the HTA process had not started successfully. The proportion of respondents, who believe that the HTA works in the public interest is high - 89%. Conclusions: The HTA process, after the three years gaining experience, is already sufficiently well known in Bulgaria, with respondents predominantly taking into account the positive aspects of the HTA introduction and evaluating the organization of the HTA process positively. [ABSTRACT FROM AUTHOR]

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Introduction: Health Technology Assessment (HTA) is a systematic multidisciplinary activity which assesses application of health technology. Surgical robots provides an improved 3D view of surgical area and a better manipulation of surgical arms as compared to conventional laparoscopic surgery. Objective of the study is to perform Cost benefit analysis of robotic surgery (RS). Originality: Original research work. Materials and Method: A retrospective cross sectional study was done for a period of 2 months. 50 robotic and 40 open surgical cases were selected. Direct & indirect costs of care were estimated along with assessment of operational costs for RS. Statistical analysis was performed. Correlation of length of stay (LOS) was done with direct and indirect costs incurred. Results: Robotic surgery is a high end CapEx model but financially viable. A positive NPV was seen with ROI in the 5th year. Operational cost and material cost were found to be statistically higher for RS. Pharmacy cost and nursing cost showed a high degree of correlation with LOS in gynaecological oncology department. Reduced LOS, less pain and faster recovery are the major positive impact that harbinger the socioeconomic benefit. Surgeons too have greater visualization and dexterity. RS provides better patient care by shortening recovery period and post-operative care. Conclusion: HTA is 'a bridge' between evidence and policy-making. It guides decision-makers about appropriate use of technology and efficient allocation of resources. RS is an advanced approach which significantly improves patient care and provides more flexibility and precision to surgeons. [ABSTRACT FROM AUTHOR]

Health Technology Assessment is one of the standard instruments in support of the decision-making to define the public health services both internationally and in the German health care system. Besides system- atic reviews, benefit-harm-analyses, health economic evaluations, and, decision-analytic modelling, especially epidemiological and biometrical questions and methods play a key role. From this perspective discus- sions on increased European cooperation including calls for wider harmonization are attracting greater interests. The overall aim is, to present at the GMDS workshop relevant information on this emerging field of harmonization in Europe across similarities and differences in the HTA process. Current developments around the composition of the EU-HTA Network are provided. This network accepts the challenge to define and establish a "Joint Work" across Europe. Special emphasis was placed on the discussion on "Harmonization of HTA: is it a threat or does it mean support?". Furthermore, methodolo- gical discussions and questions are being addressed: "Are Real World Data and Surrogates possible parameters for decision-making or HTA?" [ABSTRACT FROM AUTHOR]