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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]

Purpose: Health technologies are advancing rapidly and becoming more expensive, posing a challenge for financing healthcare systems. Health technology assessment (HTA) improves the efficiency of resource allocation by facilitating evidence-informed decisions on the value of health technologies. Our study aims to create a customized HTA roadmap for Oman based on a gap analysis between the current and future status of HTA implementation. Design/methodology/approach: We surveyed participants of an advanced HTA training program to assess the current state of HTA implementation in Oman and explore long-term goals. A list of draft recommendations was developed in areas with room for improvement. The list was then validated for its feasibility in a round table discussion with senior health policy experts to conclude on specific actions for HTA implementation. Findings: Survey results aligned well with expert discussions. The round table discussion concluded with a phasic action plan for HTA implementation. In the short term (1–2 years), efforts will focus on building capacity through training programs. For medium-term actions (3–5 years), plans include expanding the HTA unit and introducing multiple cost-effectiveness thresholds while from 6–10 years, publishing of HTA recommendations, critical appraisal reports, and timelines is recommended. Originality/value: Although the HTA system in Oman is still in its early stages, strong initiatives are being taken for its advancement. This structured approach ensures a comprehensive integration of HTA into the healthcare system, enhancing decision-making and promoting a sustainable, evidence-based system addressing the population's needs. [ABSTRACT FROM AUTHOR]

Background: According to scientific literature, some 99% of patients affected by Alzheimer's disease (AD) suffer from behavioral and psychological symptoms of dementia (BPSD), also known as neuropsychiatric symptoms (NPSs). In particular, agitation is one of the most difficult disorders to treat. States of agitation represent a very serious problem as they make these subjects dangerous for themselves and others and worsen as the disease advances. To date, there are no specific solutions for treating agitation. The only authorized drug is risperidone (as well as brexpiprazole, approved by the FDA on 11 May 2023), which can be used for no longer than 6–12 weeks because it increases the risk of death—owing to cardiocerebrovascular accidents—by 1.6–1.7 times. Methods: In order to address the latter noteworthy unmet medical need, NanoBEO was produced. The aim of the present work is to generate the health technology assessment (HTA) of this nanotechnological device. The latter consists of a controlled release system, based on solid lipid nanoparticles loaded with bergamot essential oil (BEO). Results: The results of the present research assessed the current evidence in the field of non-pharmacological treatments for this condition, including relevant primary preclinical and clinical data studies supporting the use of this device and the production of the operative plan for its launch on the market. The findings offer recommendations for decision-making on its implementation in dementia. Conclusions: NanoBEO represents a public-worth innovation in this neglected area, marking a significant advancement in the history of dementia, moving from academic research to product development. [ABSTRACT FROM AUTHOR]

Background/Objectives. Many vaccines have been developed in recent decades, and many more will be available in the future. When new safe and effective vaccines are available, decision-makers must extensively assess them before including them in the national immunization plan and issuing recommendations. The Health Technology Assessment (HTA) could be an objective, transparent, and comprehensive approach to guiding the decision-making process for the use of vaccines. Objectives and Methods. The aim of this study was to review the indications for HTA use contained in Italian institutional documents on vaccination, namely the National Immunization Plans (NIPs) and available full Italian HTA reports on vaccines, assessing their availability at the time of national recommendations' introductions. Results. HTA has been recognised as an eligible approach to deciding upon the introduction of vaccines through the NIPs of 2012–2014 and 2017–2019, and the last NIP, of 2023–2025, highlights the lack of funding dedicated to the production of independent HTA reports that can be used for issuing recommendations. In 2007–2023, twenty full HTA reports on vaccines were published in Italy: eight reports on influenza vaccines, five on Human Papilloma Virus (HPV), three each on meningococcal and pneumococcal vaccines, and one on rotavirus vaccine. HTA was applied with different purposes, namely the evaluation of new vaccines or their re-assessment, but it was not always timely with respect to both the marketing authorisation and the issuing of national recommendations for use. Conclusions. As HTA can be considered the best tool to disentangle the overall value of vaccines, it would be desirable for it to be used more and more to provide the evidence for efficient resource use. This calls for action to improve the transfer of HTA results to decision-makers, to try to fill the gap between research and decision and foster evidence-based recommendations. [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]

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: National cancer control plans (NCCPs) are complex public health programs that incorporate evidence‐based cancer control strategies to improve health outcomes for all individuals in a country. Given the scope of NCCPs, small and vulnerable populations, such as patients with childhood cancer, are often missed. To support planning efforts, a rapid, modifiable tool was developed that estimates a context‐specific national budget to fund pediatric cancer programs, provides 5‐year scale‐up scenarios, and calculates annual cost‐effectiveness. Methods: The tool was codeveloped by teams of policymakers, clinicians, and public health advocates in Zimbabwe, Zambia, and Uganda. The 11 costing categories included real‐world data, modeled data, and data from the literature. A base‐case and three 5‐year scale‐up scenarios were created using modifiable inputs. The cost‐effectiveness of the disability‐adjusted life years averted was calculated. Results were compared with each country's projected gross domestic product per capita for 2022 through 2026. Results: The number of patients/total budget for year 1 was 250/$1,109,366 for Zimbabwe, 280/$1,207,555 for Zambia, and 1000/$2,277,397 for Uganda. In year 5, these values were assumed to increase to 398/$5,545,445, 446/$4,926,150, and 1594/$9,059,331, respectively. Base‐case cost per disability‐adjusted life year averted/ratio to gross domestic product per capita for year 1, assuming 20% survival, was: $807/0.5 for Zimbabwe, $785/0.7 for Zambia, and $420/0.5 for Uganda. Conclusions: This costing tool provided a framework to forecast a budget for childhood‐specific cancer services. By leveraging minimal primary data collection with existing secondary data, local teams obtained rapid results, ensuring that childhood cancer budgeting is not neglected once in every 5 to 6 years of planning processes. The Childhood Cancers Budgeting Rapidly to Incorporate Disadvantaged Groups for Equity (CC‐BRIDGE) tool fills a current gap by providing a feasible, simplified toolkit for policymakers and childhood cancer stakeholders to calculate the costs required to develop a multiyear childhood cancer–specific budget, within existing National Cancer Control Plan (NCCP) activities. As governments continue to plan NCCPs, the CC‐BRIDGE tool can ensure that budgeting for vulnerable populations, such as childhood cancer patients, is not neglected once in every 5 to 6 years of planning processes. [ABSTRACT FROM AUTHOR]

Objectives: The aim of this study was to characterize the reimbursement policy for orphan drugs (ODs) in Central and Eastern European (CEE) countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedure, selected economic indicators, and the drug type according to indications. Materials and methods: A list of authorized medicines with orphan designation and information about active substance, Anatomical Therapeutic Chemical (ATC) classification, and therapeutic area was extracted from the web-based register of the European Medicines Agency (EMA). A country-based questionnaire survey was performed between September 2021 and January 2022 in a group of selected experts from nine CEE countries (an invitation was sent to 11 countries). A descriptive and statistical analysis was conducted to determine statistical significance, correlations, between the drug or country characteristic and the positive recommendation or reimbursement of ODs. Results: The proportion of reimbursed orphan drugs differed between countries, ranging from 17.7% in Estonia to 49.6% in Hungary (p < 0.001). The odds that ODs were reimbursed were reduced in countries with a "strong" level of impact of drug safety and efficacy on reimbursement decisions (p=0.018), the presence of other additional specific clinical aspects (e.g., genomic data) considered in the reimbursement decision (p < 0.001) and mandatory (without exception) safety assessments (p=0.004). The probability that ODs were reimbursed was increased in countries with a "moderate" level of impact of drug safety and efficacy on reimbursement decisions (p=0.018), when reimbursement decisions are dependent on the EMA registration status and orphan drug designation (p < 0.001), the presence of the "positive HTA recommendation guarantees reimbursement" policy (p < 0.001), higher GDP per inhabitant (p=0.003), and higher healthcare expenditure (p < 0.001). Conclusion: We found that there are differences among CEE countries in the reimbursement of orphan drugs, and we identified aspects that may influence these differences. Safety, efficacy, and specific clinical aspect issues significantly influenced reimbursement decisions. Antineoplastic and immunomodulating agents drugs were the largest group of ODs and increased the chance of getting a positive recommendation. The higher GDP per inhabitant and healthcare expenditures per inhabitant were positively linked to the chance that an OD receives reimbursement. [ABSTRACT FROM AUTHOR]

To ensure that limited domestic resources are invested in the most effective interventions, immunization programs in low- and middle-income countries (LMICs) must prioritize a growing number of new vaccines while considering opportunities to optimize the vaccine portfolio, as well as other components of the health system. There is a strong impetus for immunization decision-making to engage and coordinate various stakeholders across the health system in prioritization. To address this, national immunization program decision-makers in LMICs collaborated with WHO to structure deliberation among stakeholders and document an evidence-based, context-specific, and transparent process for prioritization or selection among multiple vaccination products, services, or strategies. The output of this effort is the Country-led Assessment for Prioritization on Immunization (CAPACITI) decision-support tool, which supports using multiple criteria and stakeholder perspectives to evaluate trade-offs affecting health interventions, taking into account variable data quality. Here, we describe the user feedback from Indonesia and Ethiopia, two initial countries that piloted the CAPACITI decision-support tool, highlighting enabling and constraining factors. Potential immunization program benefits and lessons learned are also summarized for consideration in other settings. [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]

The Half-Transform Ansatz (HTA) is a proposed method to solve hyper-geometric equations in Quantum Phase Space by transforming a differential operator to an algebraic variable and including a specific exponential factor in the wave function, but the mechanism which provides this solution scheme is not known. Analysis of the HTA’s application to the Hydrogen atom suggests an underlying mechanism which the HTA is a part of. Observations of exponential factors that act on the wave function naturally suggest modeling quantum operator definitions as a point on a Riemannian manifold in the 4D Operator Space, a novel idea we call the Operator Space Manifold Theory. On this manifold, we explore the concepts of superposition, regions of unique energy eigenvalues, and translation operators. We also find the theoretical backing to derive the HTA and how Operator Space Manifold Theory can be used to describe and solve quantum systems by manipulating how a quantum state perceives position and momentum. [ABSTRACT FROM AUTHOR]

AlN epilayers were grown on magnetron-sputtered (MS) (11–22) AlN buffers on m-plane sapphire substrates at 1450 °C via hydride vapour phase epitaxy (HVPE). The MS buffers were annealed at high temperatures of 1400–1600 °C. All the samples were characterised using X-ray diffraction, atomic force microscopy, scanning electron microscope and Raman spectrometry. The crystal quality of epilayers regrown by HVPE was improved significantly compared to that of the MS counterpart. With an increasing annealing temperature, the crystal quality of both MS buffers and AlN epilayers measured along [11–23] and [1–100] improved first and then decreased, maybe due to the decomposition of MS buffers, while the corresponding anisotropy along the two directions decreased first and then increased. The optimum quality of the AlN epilayer was obtained at the annealing temperature of around 1500 °C. In addition, it was found that the anisotropy for the epilayers decreased significantly compared to that of annealed MS buffers when the annealing temperature was below 1500 °C. [ABSTRACT FROM AUTHOR]

Copyright of Mali Médical is the property of Mali Medical, Faculte de Medecine, de Pharmacie et d'Odonto-stomatologie and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)

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]

The aim of this study is to explore the current practice in Cyprus regarding the introduction and reimbursement of innovative pharmaceuticals through Managed Entry Agreements (MEA), assess its operational context, and suggest approaches toward spanning the knowledge gap consequential to these efforts, especially the barriers of a small country context. The recent introduction of a National Health System (NHS), brought about fundamental reforms in Cyprus' Healthcare sector. Among such reforms, of particular interest, has been the introduction of a Managed Entry Agreements (MEA) mechanism. The first preliminary results indicate that despite being a small and unattractive market, Cyprus can apply a substantial MEA program. Concomitantly, it annotates the need to design an operational framework which should include, the definition of important technical parameters, clear demarcation of the scope, cooperation principles ensuring the effective operation of scientific committees, and clear delineation of what 'value' is. Moreover, in the context of the unified healthcare market, budget transfers should be considered, which could alleviate the inordinate budget impact of new products, which nevertheless will cut down on hospital expenditures. Narrative synthesis and health policy analysis-related resources were used. The implementation of MEA in Cyprus provides an ideal testing ground for innovative reimbursement approaches. This will streamline the country's efforts toward reimbursement of innovation, while concomitantly add to the collective MEA experience. [ABSTRACT FROM AUTHOR]

BACKGROUND: High-speed health trains are important tools and guarantees in major accidents, epidemic pandemics, disasters, and warfare health care, and the health trains currently developed for common train platforms have more functional defects. OBJECTIVE: The purpose of this study is to analyze the relationship between the medical transfer system and the medical system, and to obtain a better medical transfer train formation through the established model. METHODS: Based on the case study of medical transport tools, this paper analyzes the components and interrelationships of the medical transport system and the medical system, and then analyzes the medical transport task process of the health train by using the hierarchical task analysis (HTA) method. Combined with the Chinese standard EMU, a medical transport task model of the high-speed health train is established. Through this model, the functional compartment unit of the high-speed health train and the marshaling scheme of the high-speed health train are obtained. RESULTS: The expert system is used to evaluate the scheme. The results show that the train formation scheme formulated by the model in this paper is superior to other train formation schemes in three indicators, meeting the requirements of large medical transfer tasks. CONCLUSION: The results of this study can improve the ability of on-site treatment of patients, and can provide a basis for the research and development of a high-speed health train, which has a certain practical application value. [ABSTRACT FROM AUTHOR]

Since the groundwork published by Torres-Vega and Frederick, the Quantum Phase Space Representation (QPSR) has been explored as a method for solving a multitude of physical systems and describing phenomena. Most recently, Valentino A. Simpao has developed a method, the Heaviside Operational Ansatz, to solve the Time Dependent Schrodinger Equation (TDSE) in the QPSR, but there are still no general, direct methods to solve the Time Independent Schrodinger Equation in the QPSR. There is also no current formulation of quarkonium in phase space. In this paper, we describe the strong interactions of non-relativistic heavy quarks using the Cornell potential, and present a method, the Half-Transform Ansatz, to cast the Schrodinger Equation into a hyper-geometric form which can be solved for the phase space wave function and its energy eigenvalues using the Nikiforov-Uvarov method. This solution can be generalized for any two particle system with a scleronomic potential made up of polynomial and reciprocal terms. These results are compared to experimental results and other theoretical models. We also analyze the behavior of these wave functions, which suggest a correlation between radial momentum and the upper limit of existence in charm-anticharm mesons. [ABSTRACT FROM AUTHOR]

Copyright of Revista Científica Arbitrada Multidisciplinaria PENTACIENCIAS is the property of Revista Científica Arbitrada Multidisciplinaria PENTACIENCIAS and its content may not be copied or emailed to multiple sites or posted to a listserv without the copyright holder's express written permission. However, users may print, download, or email articles for individual use. This abstract may be abridged. No warranty is given about the accuracy of the copy. Users should refer to the original published version of the material for the full abstract. (Copyright applies to all Abstracts.)

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]

Purpose: 2021 marks the tenth anniversary of the AMNOG process and brought with it a new German administration—two good reasons to take stock of where we stand today, what has been achieved so far, and how the path of early benefit assessments in Germany should continue. Results: From the perspective of manufacturers of cancer drugs, the AMNOG process, as a constantly evolving system, has for the most part proved itself—which does not mean that there is no longer room for improvement. Significant improvements have been achieved in the area of early consultation of medical societies regarding the selection of the appropriate comparator therapy as well as in the reimbursement of biomarker diagnostic tests in the outpatient sector. However, there is still a need for improvement, especially in the areas of patient-relevant outcomes accepted by the G-BA, the inclusion of real-world data in evidence assessments, or the transfer of evidence from certain patient groups to others. Conclusion: The current AMNOG structures were developed for the most part at a time when no one saw immuno-oncology or gene and cell therapies coming, when there were no multi-tumor drug approvals, and when few imagined that within a few years, the established tumor entities would be broken down into dozens of sub-entities on the basis of molecular genetic markers. Society wants these and other advances, and the HTA process must, therefore, take this into account in a healthcare system based on solidarity. [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 delays 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]

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: 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]

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]

Highly mechanized forestry operations are essential for efficient timber harvesting. Therefore, the skills of harvester operators appear to be key to productive and sustainable use of the machines. Recent research has revealed a knowledge deficit regarding the work practices of forest machine operators. This urges systematic research into forestry machine handling and a corresponding refinement of analytical methods. Current analyses of operator tasks in forestry are less formalized and focus predominantly on machine efficiency and overall performance, but not so much on the human-related conditions of work performance and workload. Therefore, the objective of this paper is to introduce hierarchical task analysis (HTA) into forestry science. HTA is a versatile, formalized human-factors method that can be used to describe the work objectives of forest machine operators. HTA is suitable, for example, for describing (in)efficient work practices and thus as a basis for designing machine operator training and for systematically evaluating assistive technologies. The task analyses in this paper draw on a recently published empirical approach to analyzing work practices, workflows, and machine operator behavior for optimal human–machine collaboration in forestry application research. Specifically, the main work methods of clearcutting and thinning stand in European forestry were considered, with examples from Scandinavian and German method application. The process of HTA is described and a prototypical approach to HTA for both working methods provided. As a result, this work could show that a single work practice affects operator goals within different work elements and sets out how inefficient work practices can be described in terms of operator goals. With the introduction and exemplary application of HTA, a structured task definition in human-centered approaches is encouraged to analyze work practices, workflows, and machine operator behavior for optimal human–machine collaboration in forestry application research. [ABSTRACT FROM AUTHOR]

Evaluating rare disease interventions poses challenges for HTA agencies, including uncertainties and ethical issues and tensions. INESSS has recently adopted a Statement of Principles and Ethical Foundations which proposes a multidimensional approach to value appraisal as well as five principles to frame the evaluation process. Our aim was to identify and analyze HTA challenges for appraising interventions for rare diseases, using the Statement's approach to value appraisal as an analytical framework, and outline how the Statement's principles can help address these challenges. Challenges, covering a diversity of aspects, were identified by leveraging institutional experience in diverse domains of expertise and consolidated through narrative literature review. Challenges were categorized by value dimension (clinical, populational, economic, organizational, and sociocultural), which allowed to pinpoint how each challenge affects the ability to appraise the value of an intervention. Key ethical tensions across dimensions were also identified. Specific approaches to addressing these challenges – related to knowledge mobilization and integration, deliberation, and recommendation-making – were outlined on the basis of the principles promulgated in the Statement. A multidimensional approach can be fruitful for analyzing challenges for appraising the value of rare disease interventions and help guide approaches to tackle them. [ABSTRACT FROM AUTHOR]

Background: Health technology assessment (HTA) is growing in low- and middle-income countries (LMICs) to ensure optimal use of limited resources. However, the impact of HTAs on decision making in LMICs has been limited. The study aimed to provide an overview of Turkiye’s progress since establishing the first HTA agency in 2012. Methods: The web sites of three national HTA agencies in Turkiye were searched for HTA guidelines and national HTA reports. The HTA guidelines were assessed by two researchers independently against the key principles of HTA developed by Drummond et al., and the HTA reports against the national guidelines. Results: The study included one HTA guideline and eight national HTA reports. The guideline included very limited technical guidance. Compliance with the principles was poor to moderate, and significant methodological limitations were identified. The reports were inconsistent regarding the scope and the HTA assessment criteria. The link between HTA findings, HTA decision making, and health policies were not clear. Discussion: The inconsistencies between the reports and the methodological limitations demonstrate the need for national HTA guidelines. Improving the characteristics of the HTA might impact implementation. Among the key issues is transparency regarding priority setting, the HTA process, and decision making. Conclusion: Establishing and adopting national HTA guidelines at international standards is needed. Involving external scientific committees and health economists in the HTA processes might help ensure that the key principles of HTA are followed. The study findings might be helpful for countries that are developing their HTA systems. [ABSTRACT FROM AUTHOR]

Oortwijn et al continue their guide to good practice in the use of deliberative processes in health technology assessment (HTA) based on a survey of international practice. This is useful, and I applaud their care in maintaining objectivity, especially regarding the treatment of moral and politically controversial issues, in reporting how jurisdictions have handled such matters in designing HTA procedures and in their execution. To their suggestions for future research, I add: the historical development of deliberation in healthcare decision-making and in other fields of public choice, with comparisons of methods, successes and failures; development of guidance on the design and use of deliberative processes that enhance decision-making when there is no consensus amongst the decision-makers; ways of identifying and managing context-free and context-sensitive evidence; and a review of high-level capacity building to raise awareness of HTA and the use of knowledge translation and exchange (KTE) and deliberation amongst policy makers, especially in low and middle-income countries. [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]

Engine damage as a consequence of foreign object debris (FOD) during flight is frequently caused by birds. One approach to minimising disruption caused by this damage is to provide flight crew with accurate information relating to the continuing operational status of the aircraft's engines. Before designing such avionic systems however, understanding of current procedures is needed. Hierarchical Task Analysis (HTA) and Systematic Human Error Reduction and Prediction Approach (SHERPA) were used to identify potential failures that flight crew may make when managing an engine bird strike. Workshops with commercial pilots generated insights into current practice and a commercial pilot SME reviewed outputs for accuracy. Over 200 potential failures were identified, most commonly related to communication. Remedial measures, considering future avionic systems, are proposed to mitigate identified failures. This analysis provides a starting point for future design concepts for assisting flight crew in dealing with engine malfunction due to FOD strikes. Practitioner summary: Hierarchical Task Analysis was conducted to show all tasks involved in dealing with an in-flight aircraft engine bird strike. Systematic Human Error Reduction and Prediction Approach analysis was performed and over 200 possible failures were identified when managing this event. Remedial measures are proposed to help mitigate possible failures. [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]

What other aspects of value may be relevant? In this episode of the Value Insider podcast, host Mike Chambers speaks with Prof. Lou Garrison about the societal perspective on value of healthcare interventions. Prof. Garrison is Professor Emeritus in The Comparative Health Outcomes, Policy, and Economics institute (CHOICE) in the School of Pharmacy at the University of Washington. Lou is past president of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and he is currently co-chair of its Policy Outlook Committee. Going beyond the "conventional" elements of value assessment, Prof. Garrison highlights which additional impacts may be at play. He shares his concept of the "Value Flower", explaining elements such as insurance value and scientific spill-over. [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]

How does budget impact and affordability in healthcare work? In this episode of the Value Insider podcast, host Mike Chambers speaks with Prof. Sean Sullivan about affordability and budget impact for the "payers" of healthcare interventions. Prof. Sullivan is Dean of the University of Washington School of Pharmacy. He is past president of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and served as chair of the health technology assessment (HTA) committee of US Health Insurer Premera Blue Cross, was part of the US Governmental Medicare coverage evidence committee and led the ISPOR Task Force on Methods for Conducting and Reporting Budget Impact Assessments. Prof. Sullivan explains how budget impact and affordability are intertwined and how this plays a role in decisions in the US, but also the rest of the world. [ABSTRACT FROM AUTHOR]

How do we measure quality of life and utility of interventions? In this episode of the Value Insider podcast, host Mike Chambers speaks with Prof. Nancy Devlin about incorporating quality of life in value assessment. Prof. Devlin is professor of health economics at the University of Melbourne. Her past roles include Director of Research at the Office of Health Economics (OHE) London, Professor of Health Economics at City University of London, and she has been director of the International Society of Pharmacoeconomics and Outcomes Research (ISPOR). She is the Chair of the Board of the EuroQol Research Foundation, the international research organization which has developed and maintains the EQ-5D patient-reported outcome (PRO) instrument. Prof Devlin explains the value of the patient voice and how it can be measured and taken into account when considering the value of healthcare interventions. [ABSTRACT FROM AUTHOR]

Digital health and mobile medical apps (MMAs) have shown great promise in transforming health care, but their adoption in clinical care has been unsatisfactory, and regulatory guidance and coverage decisions have been lacking or incomplete. A multidimensional assessment framework for regulatory, policymaking, health technology assessment, and coverage purposes based on the MMA lifecycle is needed. A targeted review of relevant policy documents from international sources was conducted to map current MMA assessment frameworks, to formulate 10 recommendations, subsequently shared amongst an expert panel of key stakeholders. Recommendations go beyond economic dimensions such as cost and economic evaluation and also include MMA development and update, classification and evidentiary requirements, performance and maintenance monitoring, usability testing, clinical evidence requirements, safety and security, equity considerations, organizational assessment, and additional outcome domains (patient empowerment and environmental impact). The COVID‐19 pandemic greatly expanded the use of MMAs, but temporary policies governing their use and oversight need consolidation through well‐developed frameworks to support decision‐makers, producers and introduction into clinical care processes, especially in light of the strong international, cross‐border character of MMAs, the new EU medical device and health technology assessment regulations, and the Next Generation EU funding earmarked for health digitalization. [ABSTRACT FROM AUTHOR]

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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]

Introduction: Drug reimbursement decisions are often made based on a price set by the manufacturer. In some cases, this price leads to public and scientific debates about whether its level can be justified in relation to its costs, including those related to research and development (R&D) and manufacturing. Such considerations could enter the decision process in collectively financed health care systems. This paper investigates whether manufacturers' costs in relation to drug prices, or profit margins, are explicitly mentioned and considered by health technology assessment (HTA) organisations. Method: An analysis of reimbursement reports for cancer drugs was performed. All relevant Dutch HTA-reports, published between 2017 and 2019, were selected and matched with HTA-reports from three other jurisdictions (England, Canada, Australia). Information was extracted. Additionally, reimbursement reports for three cases of expensive non-oncolytic orphan drugs prominent in pricing debates in the Netherlands were investigated in depth to examine consideration of profit margins. Results: A total of 66 HTA-reports concerning 15 cancer drugs were included. None of these reports contained information on manufacturer's costs or profit margins. Some reports contained general considerations of the HTA organisation which related prices to manufacturers' costs: six contained a statement on the lack of price setting transparency, one mentioned recouping R&D costs as a potential argument to justify a high price. For the case studies, 21 HTA-reports were selected. One contained a cost-based price justification provided by the manufacturer. None of the other reports contained information on manufacturer's costs or profit margins. Six reports contained a discussion about lack of transparency. Reports from two jurisdictions contained invitations to justify high prices by demonstrating high costs. Conclusion: Despite the attention given to manufacturers' costs in relation to price in public debates and in the literature, this issue does not seem to get explicit systematic consideration in the reimbursement reports of expensive drugs. [ABSTRACT FROM AUTHOR]

Objectives: Italy was the first European country to introduce universal vaccination of adolescents, for both males and females, against Human Papilloma Virus (HPV) starting in 2017 with the NIP 2017–2019′s release. However, vaccine coverage rates (VCRs) among adolescents have shown a precarious take-off since the NIP's release, and this situation worsened due to the impact of the COVID-19 pandemic in 2020. The aim of this work is to estimate the epidemiological and economic impact of drops in VCRs due to the pandemic on those generations that missed the vaccination appointment and to discuss alternative scenarios in light of the national data. Methods: Through an analysis of the official ministerial HPV vaccination reports, a model was developed to estimate the number of 12-year-old males and females who were not vaccinated against HPV during the period 2017–2021. Based on previously published models that estimate the incidence and the economic impact of HPV-related diseases in Italy, a new model was developed to estimate the impact of the aggregated HPV VCRs achieved in Italy between 2017 and 2021. Results: Overall, in 2021, 723,375 girls and 1,011,906 boys born between 2005 and 2009 were not vaccinated against HPV in Italy (42% and 52% of these cohorts, respectively). As compared with the 95% target provided by the Italian NIP, between 505,000 and 634,000 girls will not be protected against a large number of HPV-related diseases. For boys, the number of the unvaccinated population compared to the applicable target is over 615,000 in the 'best case scenario' and over 749,000 in the 'worst case scenario'. Overall, between 1.1 and 1.3 million young adolescents born between 2005 and 2009 will not be protected against HPV-related diseases over their lifetime with expected lifetime costs of non-vaccination that will be over EUR 905 million. If the 95% optimal VCRs were achieved, the model estimates a cost reduction equal to EUR 529 million, the net of the costs incurred to implement the vaccination program. Conclusion: Suboptimal vaccination coverage represents a missed opportunity, not only because of the increased burden of HPV-related diseases, but also in terms of economic loss. Thus, reaching national HPV immunization goals is a public health priority. [ABSTRACT FROM AUTHOR]

Introduction: New requirements in Canada's pricing processes for patented drugs may exacerbate delays in regulatory and reimbursement reviews. This study seeks to better understand the impact of any additional delays on non-small cell lung cancer (NSCLC) patients by measuring the following: (a) durations and outcomes of regulatory and reimbursement reviews of NSCLC drugs in Canada and reference countries; (b) delays in Canada's reviews of three NSCLC drugs (nivolumab, afatinib, and pemetrexed [NAP]); and (c) estimating clinical, patient, and economic impacts of delays in Canada's reviews on access to NAP. Methods: Information from the Context Matters database and the literature (2005–2020) was used to evaluate the durations and outcomes of reimbursement reviews of NSCLC drugs in Canada and comparator countries. Public information was used to assess delays in Canada's reviews of NAP. Empirical modeling with data from the literature and the Southern Alberta Lung Cancer database was used to estimate the impact of delays in Canada's NAP reviews on patients (i.e., as losses in person-years of life and quality-adjusted life-years [QALYs]). Results: Regulatory and reimbursement reviews in countries of interest take 12–18 months. In Canada, reviews of NSCLC drugs took 216 days (median), with a 24% rejection rate (mean = 19%). Delays in NAP reviews ranged from 5 to 94 days at Health Canada, 0–80 days at CADTH/pCODR, and 12–797 days in Canadian provinces. These delays may have affected 6400 patients, who lost up to 1740 person-years of life and 1122 QALYs (valued at CA$112 million). Conclusion: Changes to Canada's prescription drug pricing processes may prolong reviews. [ABSTRACT FROM AUTHOR]

Introduction. New medicines for the management of type 2 diabetes became available internationally in 2005, yet only in 2018 did the first of these become available in New Zealand. Access to these new medicines in New Zealand is largely dependent on decisions made by the Pharmaceutical Management Agency (PHARMAC). Aim. This study sought to describe the decision-making processes to better understand access to new medicines in New Zealand. Methods. We conducted an analysis of publicly accessible information on therapeutic committee deliberations, prices of medicines and registration and formulary listing dates. Results. Prices for the new diabetes medicines in New Zealand are lower than comparator countries, but access to them takes longer. Discussion. Given that knowledge on efficacy, safety and quality is widely available to support decision-making on new medicines, differences in access to them between nations appears to depend on the fourth hurdle of cost. However, we suggest that a rush to market is the norm, that activities of the pharmaceutical industry and regulatory agencies are less transparent than desirable, and that greater focus on availability of safety data is required. Deliberations of PHARMAC therapeutic committees are robust yet protracted. Opportunities to expedite decision-making, as well as resolving inequities, may be worthy of examination. [ABSTRACT FROM AUTHOR]

Objectives: Artificial intelligence (AI) is seen as a major disrupting force in the future healthcare system. However, the assessment of the value of AI technologies is still unclear. Therefore, a multidisciplinary group of experts and patients developed a Model for ASsessing the value of AI (MAS-AI) in medical imaging. Medical imaging is chosen due to the maturity of AI in this area, ensuring a robust evidence-based model.Methods: MAS-AI was developed in three phases. First, a literature review of existing guides, evaluations, and assessments of the value of AI in the field of medical imaging. Next, we interviewed leading researchers in AI in Denmark. The third phase consisted of two workshops where decision makers, patient organizations, and researchers discussed crucial topics for evaluating AI. The multidisciplinary team revised the model between workshops according to comments.Results: The MAS-AI guideline consists of two steps covering nine domains and five process factors supporting the assessment. Step 1 contains a description of patients, how the AI model was developed, and initial ethical and legal considerations. In step 2, a multidisciplinary assessment of outcomes of the AI application is done for the five remaining domains: safety, clinical aspects, economics, organizational aspects, and patient aspects.Conclusions: We have developed an health technology assessment-based framework to support the introduction of AI technologies into healthcare in medical imaging. It is essential to ensure informed and valid decisions regarding the adoption of AI with a structured process and tool. MAS-AI can help support decision making and provide greater transparency for all parties. [ABSTRACT FROM AUTHOR]

Objectives: Clinical practice is shifting toward an era of precision medicine. The use of comprehensive genomic profiling (CGP) in oncology has broad potential as a universal companion diagnostic for targeted therapies which may significantly improve health outcomes while using healthcare resources more efficiently. Given the nature of this technology, assessing the value of CGP presents unique challenges. Methods: This paper draws on evidence from the academic and policy literature in oncology, as well as stakeholder interviews (health economists, payers, clinicians, and public policy officials) in countries using incremental cost-effectiveness ratios (ICER) as part of health technology assessment (HTA). Results: The degree to which CGP is subject to a value assessment varies significantly across healthcare systems. Current HTA processes focus on evaluating diagnostic testing through co-dependent assessment of diagnostic testing and associated therapeutic interventions. Diagnostic tests with multiple associated therapeutic interventions are rapidly evolving and poorly unsuited to current HTA approaches. Moreover, HTA approaches are limited in their ability to consider broader systemic benefits of the expanded diagnostic capabilities and enhanced opportunities for clinical trial participation offered by CGP. Conclusions: The assessment of the overall value of CGP is limited by the current models of HTA. This paper suggests policy proposals for value assessment and funding reforms to help broaden patient access to CGP. These include investing in genomic testing infrastructure; decoupling the assessment of the value of CGP testing to identifying predetermined therapeutic interventions; tailoring evaluation methodology; and developing approaches to collecting evidence of clinical, healthcare system and societal benefit. [ABSTRACT FROM AUTHOR]

In 2020, European Network for Health Technology Assessment (EUnetHTA) published a relative effectiveness analysis (REA) of Pretomanid in combination with Bedaquiline and Linezolid for the treatment of extensively drug-resistant (XDR) or treatment-intolerant or nonresponsive multidrug-resistant (MDR) tuberculosis (TB) (REA PTJA14). This REA may have a significant value for low- and middle-income countries (LMICs) outside Europe, particularly those with a high burden of drug-resistant TB. This commentary focuses on whether the REA PTJA14 can be transferred and to what extent a REA can be translated to LMICs context outside Europe. We found that the assessments on the clinical effectiveness and risks of bias reported in REA PTJA14 are useful for LMICs outside Europe. The highly standardized management of TB will support the applicability of the REA to LMICs outside of Europe. Transferring this REA can reduce workload and efficiently use limited resources to conduct health technology assessment (HTA). However, the transfer should consider several critical issues, including variations in health system delivery and clinical practice and setting-specific constraints. In the TB context, the differences in the current standard treatment for XDR or nonresponsive MDR TB, resources availability for drug-resistant TB management, and how healthcare is delivered in the countries can complicate the applicability of the REA PTJA14. Given that LMICs have limitations in doing HTA, it is now critical to develop standard guidelines for transferring REA or other HTA results from high-income countries or other LMICs to maximize the benefits of the REA for LMICs outside Europe. [ABSTRACT FROM AUTHOR]

Adequate methods are urgently needed to guarantee the good practice of health technology assessment (HTA) for technologies with novel properties. The aim of the study was to construct a conceptual framework to help understand the innovation of HTA methods (IHTAM). The construction of the IHTAM framework was based on two scoping reviews, one on the current practice of innovating methods, that is existing HTA frameworks, and one on theoretical foundations for innovating methods outside the HTA discipline. Both aimed to identify and synthesize concepts of innovation (i.e., innovation processes and roles of stakeholders in innovation). Using these concepts, the framework was developed in iterative brainstorming sessions and subsequent discussions with representatives from various stakeholder groups. The framework was constructed based on twenty documents on innovating HTA frameworks and fourteen guidelines from three scientific disciplines. It includes a generic innovation process consisting of three phases ("Identification," "Development," and "Implementation") and nine subphases. In the framework, three roles that HTA stakeholders can play in innovation ("Developers," "Practitioners," and "Beneficiaries") are defined, and a process on how the stakeholders innovate HTA methods is included. The IHTAM framework visualizes systematically which elements and stakeholders are important to the development and implementation of novel HTA methods. The framework could be used by all stakeholders involved in HTA innovation to learn how to engage dynamically and collaborate effectively throughout the innovation process. HTA stakeholders in practice have welcomed the framework, though additional testing of its applicability and acceptance is essential. [ABSTRACT FROM AUTHOR]

Pharmaceutical industry investments in Italy must be supported with a reform process that promotes simpli- fications 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.e [ABSTRACT FROM AUTHOR]

To control pharmaceutical spending and improve access, the United States could adopt strategies similar to those introduced in Germany by the 2011 German Pharmaceutical Market Reorganization Act. In Germany, manufacturers sell new drugs immediately upon receiving marketing approval. During the first year, the German Federal Joint Committee assesses new drugs to determine their added medical benefit. It assigns them a score indicating their added benefit. New drugs comparable to drugs in a reference price group are assigned to that group and receive the same reimbursement, unless they are therapeutically superior. The National Association of Statutory Health Insurance Funds then negotiates with manufacturers the maximum reimbursement starting the 13th month, consistent with the drug's added benefit assessment and price caps in other European countries. In the absence of agreement, an arbitration board sets the price. Manufacturers accept the price resolution or exit the market. Thereafter, prices generally are not increased, even for inflation. US public and private insurers control prices in diverse ways, but typically obtain discounts by designating certain drugs as preferred and by restricting patient access or charging high copayment for nonpreferred drugs. This article draws 10 lessons for drug pricing reform in US federal programs and private insurance. [ABSTRACT FROM AUTHOR]