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Introduction: This paper summarizes the results from a forum of healthcare experts, academia representatives, and public agency officials from emerging and established market countries on Value-Based Healthcare (VBHC) and Health Technology Assessment (HTA). Presentations from experts provided insights into current developments and challenges, followed by interactive roundtable discussions. Emerging markets have unique healthcare systems, patient populations, resource constraints and needs., Areas Covered: Each roundtable explored specific topics including the role of HTA and Real-world evidence (RWE) in healthcare decision-making, challenges in biosimilar value assessment and incorporating non-price criteria reflecting context-related specifications of emerging markets such as the multifaceted nature of value in healthcare decision-making, emphasizing stakeholder perspectives and system complexities., Expert Opinion: RWE emerged as important in understanding biosimilar value recognition and decision-making processes, with insights into its applications and challenges. Recommendations were provided for utilizing Multi-Criteria Decision Analysis (MCDA) in pharmaceutical procurement, particularly for off-patent medicines, underscoring the importance of comprehensive evaluation frameworks and adherence to value-based principles. Overall findings suggest avenues for collaboration between industry, academia, and public agencies to address implementation barriers and promote equitable, efficient, and high-quality healthcare systems in emerging markets through public-private partnerships, joint capacity building and training initiatives, and knowledge transfers.

Although platform trials have many benefits, the complexity of these designs may result not only in increased methodological but also regulatory and ethical challenges. These aspects were addressed as part of the IMI project EU Patient-Centric Clinical Trial Platforms (EU-PEARL). We reviewed the available guidelines on platform trials in the European Union and the United States. This is supported and complemented by feedback received from regulatory interactions with the European Medicines Agency and the US Food and Drug Administration. Throughout the project we collected the needs of all relevant stakeholders including ethics committees, regulators, and health technology assessment bodies through active dialog and dedicated stakeholder workshops. Furthermore, we focused on methodological aspects and where applicable identified the corresponding guidance. Learnings from the guideline review, regulatory interactions, and workshops are provided. Based on these, a master protocol template was developed. Issues that still need harmonization or clarification in guidelines or where further methodological research is needed are also presented. These include questions around clinical trial submissions in Europe, the need for multiplicity control across the whole master protocol, the use of non-concurrent controls, and the impact of different randomization schemes. Master protocols are an efficient and patient-centered clinical trial design that can expedite drug development. However, they can also introduce additional operational and regulatory complexities. It is important to understand the different requirements of stakeholders upfront and address them in the trial. While relevant guidance is increasing, early dialog with relevant stakeholders can help to further support such designs., (© 2024 The Authors, Johnson & Johnson Innovative Medicines and Sanofi. Clinical Pharmacology & Therapeutics published by Wiley Periodicals LLC on behalf of American Society for Clinical Pharmacology and Therapeutics.)

Significant progress has been achieved in human health in the European Union in recent years. New medicines, vaccines, and treatments have been developed to tackle some of the leading causes of disease and life-threatening illnesses. It is clear that investment in research and development (R&D) for innovative medicines and treatments is essential for making progress in preventing and treating diseases. Ahead of the legislative process, which should begin by the end of 2022, discussions focus on how Europe can best promote the huge potential benefits of new science and technology within the regulatory framework. The challenges in European healthcare were spelled out by the panellists at the roundtable organised by European Alliance for Personalised Medicine (EAPM). Outcomes from panellists' discussions have been summarized and re-arranged in this paper under five headings: innovation, unmet medical need, access, security of supply, adapting to progress, and efficiency. Some of the conclusions that emerged from the panel are a call for a better overall holistic vision of the future of pharmaceuticals and health in Europe and a collaborative effort among all stakeholders, seeing the delivery of medicines as part of a broader picture of healthcare., Competing Interests: Chorostowska-Wynimko reports grants, personal fees, and non-financial support from Grifols, grants, personal fees and non-financial support from AstraZ˛en˛eca, grants, personal fees and non-financial support from Pfizer, personal fees and non-financial support from IVSD, personal fees from BMS, personal fees from Novartis, personal fees from Chiesi, personal fees from Roche, grants and personal fees from Boehringer-lngelheim, grants, personal fees and non-financial support from CSL Behring, grants, personal fees and non-financial support from CelonPharma, personal fees from Amgen, personal fees from Adamed, grants and personal fees from Mereo Biopharma, personal fees from Takeda, outside the submitted work; Rafal Dziadziuszko received honoraria for lectures/ advisory boards: Astra Zeneca, Roche, Takeda, Pfizer, Novartis, MSD, Karyopharm, Bristol-Myers Squibb; D.H. and M.K. are employees of the European Alliance for Personalised Medicine, which receives funding from both the public and private sectors. Marie-Helene Fandal is an employee of SANOFI.

Managed entry agreements (MEA) represent one of the main topics of discussion between the European National Payers Authorities. Several initiatives on the subject have been organized over the past few years and the scientific literature is full of publications on the subject. There is currently little international sharing of information between payers, mainly as a result of the confidentiality issues. There are potential benefits from the mutual sharing of information, both about the existence of MEAs and on the outcomes and results. The importance of involving all the players in the decision-making process on market access for a medicinal product (MP) is that it may help to make new therapies available to patients in a shorter time. The aim of this project is to propose a new pathway of value-based MEA (VBMEA), based on the analysis of the current Italian pricing and reimbursement framework. This requires elaboration of a transparent appraisal and MEA details with at least a 24-month contract. The price of the MP is therefore valued based on the analysis of the VBMEA registries of the Italian Medicines Agency. Although the proposal focuses on the Italian context, a similar approach could also be adapted in other nations, considering the particularities of the single health technology assessment (HTA)/payer system., Competing Interests: OW was employed by Medi-Qualité Omega. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest., (Copyright © 2022 Xoxi, Rumi, Kanavos, Dauben, Gutierrez-Ibarluzea, Wong, Rasi and Cicchetti.)

Health Technology Assessment (HTA) is a systematic evaluation of a health technology, designed to appraise the direct or intended effects and indirect or unintended consequences of the technology with an overall goal of supporting informed decision making regarding the use of these health technologies in the healthcare system. In this paper, we present fundamental HTA concepts and provide a conceptual framework that embraces the processes and outcomes required for integrated healthcare decision-making. The "HTA Metro Map" was designed to guide the user through the different areas on: where to use, what and whom to involve within the decision process. The map reflects the complexity and inter-connectedness of the different kind of healthcare services that need to work together to be able to efficiently deliver coordinated decisions at local, regional, national, and international levels. This tool may also serve as base for facilitating developments and improvements of the HTA structure worldwide. The paper discusses the main features of the "HTA Metro Map" while reinforcing the key concepts underlying HTA's integrated approach. The first view of the map provides the several layers of complexity seen in HTA and the various lines within the map represent the main actors involved in the assessment processes. The map connections and crossings symbolize the interprofessional and interpersonal collaborations while the stations denote the knowledge, skills, experiences, and attitudes of each professionals as they interact within this framework. Every line represents a HTA stakeholder and the circular line in the centre represents the patient at the centre of the system. The zones, from social to community and hospital level, represent the need for integration from the perspective of health systems. The HTA Metro Map also has different dimensions depicted by the level of profoundness. Finally, the concepts of different healthcare stakeholder perspectives are introduced both in visual and temporal terms. The "HTA Metro Map" is designed as a flexible model for easy adaptability and in accurately capturing the complexity inherent in any healthcare system. It is hoped that the map will assist different stakeholders to build network capacity, pool existing resources, and develop a more holistic vision that will result in a sustainable, efficient and collaborative decision-making process., (Copyright © 2019 Chiumente et al.)

Medical classifications systematize medical concepts (e. g. diagnoses, procedures). They are essential for statistics and reimbursement systems in health care systems. Diagnoses are classified worldwide with the International Classification of Diseases (ICD) of the World Health Organization (WHO). The situation for procedure classifications is quite different. Many countries developed their own procedure classifications in different ways and for different purposes.Since 2007, the International Classification of Health Interventions (ICHI) is been developing as a common tool for reporting and analyzing health interventions for statistical purposes as well as for the use in reimbursement systems.ICHI covers not only medical and surgical procedures but also interventions carried out by a broad range of providers across the full scope of health systems, including rehabilitation, assistance with functioning, prevention and public health.The multiaxial classification is built around three axes: target (the entity on which the action is carried out), action (a deed done by an actor to a target) and means (the processes and methods by which the action is carried out). Extension codes are provided to allow users to describe additional detail about the intervention in addition to the relevant ICHI stem-code. ICHI was designed with a low level of complexity for countries seeking a classification, while also serving as a basis for international comparisons. ICHI can also be used in reimbursement systems, by adding cost-relevant information through extension codes.The recent 2018 ICHI beta version is available on the platform https://mitel.dimi.uniud.it/ichi . This version and further ICHI tools will be tested during later reviews and field testing in 2018 and 2019. Once finalized, probably in 2020, ICHI will be freely available for adoption by member states of the WHO.

We see a backlog in the effective and efficient integration of personalized medicine applications such as genome-based information and technologies into healthcare systems. This article aims to expand on the steps of a published innovative model, which addresses the bottleneck of real-time integration into healthcare. We present a deconstruction of the Learning-Adapting-Leveling model to simplify the steps. We found out that throughout the technology transfer pipeline, contacts, assessments and adaptations/feedback loops are made with health needs assessment, health technology assessment and health impact assessment professionals in the same order by the academic-industrial complex, resulting in early-on involvement of all stakeholders. We conclude that the model steps can be used to resolve the bottleneck of implementation of personalized medicine application into healthcare systems.

Objectives: The aim of this study was to develop an health technology assessment (HTA) decision tool to support the decision-making process on health technologies for hospital decision makers in cross-border regions., Methods: Several methods were used to collect information necessary to develop the cross-border mini-HTA decision tool. The literature was inventoried on HTA in border regions and local settings and the use of HTA by local decision makers. Semi-structured interviews with hospital decision makers in cross-border regions were also performed. Based on group discussion of the resulting information, it was decided to use the Danish mini-HTA guideline as a starting point for development of the decision tool. After finishing the first version of the decision tool it was tested in two pilot studies., Results: Some questions in the Danish mini-HTA guideline were not relevant. Other questions needed rephrasing and questions about cross-border situations were added. The pilots showed several missing topics, including legal questions and reimbursement issues. The final decision tool consists of three sections: a general section, a section for hospitals not cooperating cross-border and a section for hospitals that are cooperating with hospitals across a national or regional border., Conclusions: Based on our literature search, this may be the first cross-border mini-HTA decision tool. The decision tool will be of help for healthcare professionals and decision makers in border settings who would like to use HTA evidence to support their decision-making process.

Background: Under the mandate of the European Association for Endoscopic Surgery (EAES) a guideline on methodology of innovation management in endoscopic surgery has been developed. The primary focus of this guideline is patient safety, efficacy, and effectiveness., Methods: An international expert panel was invited to develop recommendations for the assessment and introduction of surgical innovations. A consensus development conference (CDC) took place in May 2009 using the method of a nominal group process (NGP). The recommendations were presented at the annual EAES congress in Prague, Czech Republic, on June 18th, 2009 for discussion and further input. After further Delphi processes between the experts, the final recommendations were agreed upon., Results: The development and implementation of innovations in surgery are addressed in five sections: (1) definition of an innovation, (2) preclinical and (3) clinical scientific development, (4) scientific approval, and (5) implementation along with monitoring. Within the present guideline each of the sections and several steps are defined, and several recommendations based on available evidence have been agreed within each category. A comprehensive workflow of the different steps is given in an algorithm. In addition, issues of health technology assessment (HTA) serving to estimate efficiency followed by ethical directives are given., Conclusions: Innovations into clinical practice should be introduced with the highest possible grade of safety for the patient (nil nocere: do no harm). The recommendations can contribute to the attainment of this objective without preventing future promising diagnostic and therapeutic innovations in the field of surgery and allied techniques.

Quality-oriented medicine, which has the goal of healing or improving the patients quality of life as highest imperative, also has the obligation to fulfil the required standards itself. Standards, defined for the implementation of this goal, are supported thereby by standard operating procedures (SOPs), the structured and systematic descriptions of the necessary working processes. Both (Standards and SOPs) are part of the integrated quality management of the Total Quality Management (TQM).The quality demands of the German agency for Health Technology Assessment (DAHTA@DIMDI) apply to the processes of product creation as well as to the product itself. The quality management used by DAHTA@DIMDI orients itself on internationally recognized quality assurance standards (e.g. DIN EN ISO 9000:2000FF). The DAHTA@DIMDI pursues the goal, that the development processes and the contents of the evaluation of medical procedures (Health Technology Assessment - HTA) attains the highest acceptance possible in all target groups.

With the Health Reform Act 2000 the assessment of medical procedures (Health Technology Assessment, HTA) was first applied to quality assurance in German public health. Since taking this step Germany belongs to the group of countries (Great Britain, Sweden, Australia, etc.) that employ HTA as an objective instrument to determine scientific grounds for political health decisions, for instance for decisions concerning absorption of costs or the development of a benefit catalogue. The German Agency for Health Technology Assessment was founded in September 2000 at the German Institute for Medical Documentation and Information (DAHTA@DIMDI) to fulfil the legal requirements. DAHTA set up and operates a databank-supported information system to supply HTA- relevant information. Additionally research assignments are commissioned for this area and quality standards determined. The goal is to create an up to date and objective information base for health politics and also for medical qualified personnel and consumers that considers the social, ethical, economical and legal results besides the efficacy and effectiveness of medical procedures.

Recently, we have demonstrated that human platelet antigen 1b (HPA-1b or P1A2) is a hereditary risk factor for platelet thrombogenicity leading to premature myocardial infarction in preexisting coronary artery disease. However, HPA-lb does not represent a risk factor for coronary artery disease itself. The aim of our present study was to evaluate the role of HPA-lb on the outcome in patients after coronary-artery bypass surgery. We prospectively determined the HPA-1 genotype in 261 consecutive patients prior to saphenous-vein coronary-artery bypass grafting. The patients were followed for one year. Among patients with bypass occlusion, myocardial infarction, or death more than 30 days after surgery, the prevalence of HPA-lb was significantly higher than among patients without postoperative complications (60 percent, 6/10, vs. 24 percent, 58/241, p <0.05, odds ratio 4.7). Using a stepwise logistic regression analysis with the variables HPA-1b, age, sex, body mass index, smoking (pack-years), hypertension, diabetes, cholesterol and triglyceride concentration, only HPA-lb had a significant association with bypass occlusion, myocardial infarction, or death after bypass surgery (p = 0.019, odds ratio 4.7). This study shows that HPA-1b is a hereditary risk factor for bypass occlusion, myocardial infarction, or death in patients after coronary-artery bypass surgery.

Background: This study aims to evaluate the early and late outcome of patients treated by surgery for myasthenia gravis and the diagnostic value of the Besinger Score, which is based on a correlation of severity of symptoms with specific antibodies to acetylcholine receptors, in the follow-up investigation after surgical therapy., Methods: Between June 1984 and April 1992 thoracotomy was performed in 51 myasthenia gravis cases at our department. The retrospective analysis considered patients with (n = 13) or without thymoma (n = 38). The Besinger score was used to describe the severity of disease preoperatively and up to 5 years postoperatively., Results: The Besinger score fell continually post surgery. Changes in relative serum concentrations of antibodies were similar to the Besinger score. Five years after thymectomy complete remission was diagnosed in 40% of the patients. The required dosage of pyridostigmine had fallen by two thirds after 5 years. Patients with follicular hyperplasia had significantly higher remission rates than those with thymoma., Conclusions: Surgery for myasthenia gravis is successful. The Besinger score well quantifies the severity of the disease.

Objective: As Aspirin (ASA) has proven efficacy in preventing patients with CAD from complications related to cardiovascular diseases, most patients scheduled for CABG are treated with ASA therapy. Consequently, impaired hemostasis is a problem in the management of CABG patients. Clinical studies have shown that Aprotinin can reduce bleeding and the use of blood products by 50% in patients both with and without pre-operative ASA therapy. Concerning the combined effect of peri-operative low-dose ASA therapy and intra-operative high-dose Aprotinin therapy, the gathering of additional and prospective data seemed to be necessary., Methods: We conducted a double-blind two-centre randomised three-arm study in patients with elective primary CABG surgery. Three groups have been tested, comprising 119 patients in total (group A: ASA + Aprotinin, group B: placebo + Aprotinin, group C: placebo + placebo) to investigate a possible reduction of bleeding in Aprotinin treated patients. For all patients, thromboxane levels were used to identify ASA or placebo treatment., Results: The post-operative blood loss is significantly reduced by 21% after Trasylol administration (B vs. C; P = 0.009). The unexpected result of this study has been that the pre-treatment with ASA led to a further reduction of 18% (A vs. C; P < 0.0001). The difference between the two Aprotinin groups (A and B) is significant (P = 0. 01) in favour of ASA pre-treatment. Myocardial infarction (MI) had been diagnosed at levels of 1.8% in total (2/113), 2.6% (1/38) in group B and 3.2% (1/31 ) in group C. An additional blinded evaluation of ECG, enzyme levels and clinical status revealed 'definite, probable and possible' MIs of 5% in group A, compared to 16% in group B and 13% in group C, thus providing no evidence for a higher risk of infarction by Aprotinin treatment. When comparing the ASA group to non-ASA pre-treatment, a strong trend towards a reduction in MI rate becomes obvious, from 15% to 5% in favour of the ASA pre-treatment (P = 0.08). Concerning other peri-operative complications, no statistical difference between the groups could be detected., Conclusions: A reduction in post-operative blood loss in primary elective CABG surgery with intra-operative Aprotinin treatment could be confirmed. A low-dose ASA treatment combined with a high-dose aprotinin administration during surgery not only neutralized a potentially higher risk of bleeding, but did in fact reduce the post-operative blood loss. The protective effect of ASA on peri-operative MI has been evident through a reduction of MI rate in ASA treated patients.

Carrying out a 1,000 patient prospective, randomized study comparing a roller pump and the BioMedicus centrifugal pump (CP), hematological parameters, blood loss, renal function, postoperative complications, and lethality data were evaluated. Using a validated preoperative risk stratification method (Cardiac RiskMaster), patients were divided into different risk categories for statistical analysis. This study verified an improved outcome with the use of a CP in routine cardiac surgery, demonstrated by blood handling, blood loss, renal function, and nephrological complication data. There was also a significant reduction in neurological complications. There was no significant difference in postoperative lethality, but high risk patients demonstrated outcomes comparable to those being defined for medium risk patients. Routine cardiac surgical patients as well as multimorbid patients benefit from the use of a CP. Preoperative risk stratification is a valid tool to demonstrate how the employment of new technologies can provide for an improved outcome without increasing overall costs at the same time.