Search Results (16)

Patient involvement in health technology assessment (HTA) processes is increasingly recognized as pivotal for informed, equitable, and patient-relevant health care decision-making. With the implementation of Joint Scientific Consultations (JSCs) and Joint Clinical Assessments (JCAs) under Regulation (EU) 2021/2282, the European Union has a unique opportunity to design harmonized mechanisms that reflect best practices from established HTA systems. This article, drawing on the Acute Leukemia Advocates Network (ALAN)’s comparative analysis of HTA practices across seven countries (Canada, England, Scotland, France, Germany, Spain, and Italy), examines how current patient involvement processes can inform the JCA framework. It identifies opportunities to replicate effective practices and proposes strategies to embed patient voices meaningfully into the JCA process. By prioritizing robust and inclusive patient involvement, the EU can establish a global benchmark for impactful and consistent HTA processes. By leveraging lessons from international HTA systems and prioritizing clear frameworks, early involvement, and capacity building, the EU can set a global standard for meaningful patient participation in HTA processes. ALAN is an independent global network of patient organizations dedicated to improving outcomes for patients with acute leukemia. Full article

The European Health Technology Assessment (HTA) regulation (HTAR) came into effect in January 2025 and impacts the HTA process in all European Member States. Member States must give due consideration to the joint clinical assessment (JCA) report. This may require adaptations at the national level. This paper describes the anticipated changes to the Dutch national HTA process and how the Dutch National Health Care Institute (Zorginstituut Nederland, ZIN) prepared for this, because sharing experience between Member States can be of general interest for future expansion of the EU HTAR. ZIN’s implementation activities were facilitated by a project-governance structure and by a continuous gap analysis of the current national assessment and appraisal process of medicinal products, resulting in a concrete action plan. The implementation of the HTAR has two major implications for ZIN’s HTA process, namely that the scoping phase starts much earlier and that the JCA report is the starting point for the national assessment. Gaps, challenges and issues were identified in the categories: information and knowledge, IT and template, communication and stakeholder engagement, capacity and resources, and financial aspects. Based on a thorough and well-defined implementation plan, ZIN is ready to implement the HTAR in national HTA processes and to take on (co-)assessor roles for JCA of medicinal products in 2025. Full article

Background and Objective: Innovation is a key enabler of patient-centered care in cardiology, with new medical devices and digital health technologies offering the potential to improve outcomes and efficiency. However, the evaluation of these innovations poses challenges for clinicians, regulators, and procurement stakeholders, particularly within the complex European healthcare landscape. This review aims to explore the current state of health technology assessment (HTA) for cardiology-related medical devices in Europe, offering a clinical perspective. Material and Methods: Three independent scoping reviews were conducted following the PRISMA-ScR guidelines. Keywords included “innovation”, “health technology assessment”, and “cardiology”. The search was supplemented by the relevant literature on European HTA policies, regulatory directives, and emerging technologies. Results: The review identified three central themes: (1) the evolving role of clinicians in HTA processes, (2) the integration of innovative technologies such as digital tools and artificial intelligence within HTA frameworks, and (3) the considerable variation in HTA practices and policies across EU member states. Conclusions: HTA in Europe is undergoing a transformation, with increasing emphasis on interdisciplinary collaboration and frameworks that support innovation. While the goal of harmonization across the EU remains a work in progress, new regulatory efforts, such as the HTA Regulation (HTAR), offer promising avenues for aligning clinical practice with evidence-based assessment and reimbursement decisions. Full article

The evolving landscape of Health Technology Assessment (HTA) in Europe, shaped by the implementation of the new EU HTA Regulation (HTAR), places an emphasis on engaging all stakeholders, including patients, in collaborative evidence generation. Yet integrating patients’ perspectives into critical processes like PICO scoping remains a challenge, with concerns around subjectivity, representativeness, and methodological robustness. This opinion paper examines the complexities of patient engagement in HTA, highlighting both the opportunities for patients to make meaningful contributions and the barriers that stand in the way. We propose a framework that employes the Delphi panel methodology to (1) foster scientific validity and increase transparency in patient contributions, (2) establish a structured and consistent patient engagement framework, and (3) and understand European patients’ perspectives while promoting collaboration among EU countries. By facilitating iterative feedback and fostering agreement among diverse groups of patients and caregivers contributing with their expertise, consensus methods like Delphi panels can help refine PICO criteria, align diverse stakeholders’ expectations, and increase the relevance of HTA outcomes. A study is now underway to evaluate the feasibility and value of using the modified Delphi panel methodology for patient engagement in PICO scoping. The authors propose that embracing patient engagement through carefully designed consensus frameworks could enhance the legitimacy and completeness of HTA processes, driving more patient-centered decision making across Europe. Full article

The harmonization of pharmaceutical regulations within the European Union has been a crucial step towards ensuring high safety standards and efficient access to innovative medicines. The evolution from fragmented national regulations to a unified legal framework has streamlined the marketing authorization process, fostered scientific collaboration, and reduced administrative burdens. The establishment of the centralized marketing authorization procedure and the European Medicines Agency (EMA) has played a pivotal role in coordinating regulatory efforts across member states. This article examines the historical developments, regulatory milestones, and the impact of harmonization on pharmaceutical assessments. Furthermore, it explores key lessons learned—including the value of centralized coordination, standardization, capacity and knowledge-sharing, transparency and trust—from the regulatory landscape that could inform the evolving Health Technology Assessment (HTA) framework in the EU. Full article

Background: Given their crucial role in vaccine assessment, National Immunization Technical Advisory Groups (NITAGs) should be considered in the Regulation on Health Technology Assessment (EU HTAR) to maximize the benefits of the EU HTAR for vaccines. This review and perspective piece identifies the gaps arising from NITAGs potential lack of involvement and proposes strategies for involving them. Methods: A targeted literature and guideline review was conducted to evaluate NITAGs’ current and future role in relation to the EU HTAR. The impact of the EU HTAR on diverse national HTA frameworks was explored in a three-country case study. Recommendations were developed to leverage strengths and address weaknesses to ensure consistent and cohesive vaccine assessments. Results: The case study revealed potential overlaps between NITAGs and the EU HTAR, particularly regarding horizon scanning and joint scientific consultations. The involvement of NITAGs in national assessments varies, influencing how well joint clinical assessment reports will ultimately align with and be applicable to individual Member States. Conclusions: Stronger consideration of vaccines within the EU HTAR and NITAG involvement can streamline assessments, reduce duplication, and improve alignment between European and national processes. Strategic actions, including capacity building and collaborations between NITAGs, are key in facilitating this process. Full article

Advanced Therapy Medicinal Products (ATMPs) are revolutionising modern medicine. By addressing the root cause rather than the symptoms of disease, ATMPs hold the promise of long-lasting benefits or even cures for severe, genetic, and rare diseases—including rare cancers—for patients with few or no viable treatment options. At the same time, the inherent complexities of ATMPs pose challenges to traditional HTA frameworks. Unlike conventional treatments, ATMPs are often one-time therapies with a high magnitude of effect. However, their long-term durability remains uncertain at launch. The Joint Clinical Assessment (JCA), under the EU’s Health Technology Assessment (HTA) Regulation, represents a once-in-a-generation opportunity to consolidate the strengths of national HTA processes into a unified framework that accounts for the specificities of ATMPs and streamlines decision-making, cementing Europe’s position as a pioneer in innovative HTA approaches. While concerns remain regarding the suitability of current JCA methodologies for ATMPs, the HTA Regulation continues to bring the HTA ecosystem closer together, with numerous benefits already emerging from EU-wide collaboration on JCAs. This article outlines the HTA challenges posed for and by ATMPs, and ARM’s perspective on the JCA’s implementation. A ‘fit for purpose’ JCA holds the promise to unlocking these therapies’ benefits for individuals across Europe. Full article

Background: This study explored the readiness and strategic considerations of companies and key stakeholders for the implementation of the Joint Clinical Assessment (JCA) under the European Health Technology Assessment Regulation (HTAR). It examined the implications of the JCA process for jurisdictional submission strategies, and decision-making across Europe. The study aimed at identifying key measures for an efficient and effective JCA process to enable national rollout. Methods: A survey was conducted with international pharmaceutical companies, followed by a multi-stakeholder workshop that expanded on the findings. The survey and workshop focused on key areas such as time to market, submission strategies, and the role of JCA in national decision-making processes. Descriptive and qualitative analyses were performed to identify recommendations for measuring and improving the JCA process. Results: 13 companies responded to the survey, respondents were generally prepared for the JCA process (readiness rated 6–7/10), but concerns persist about timeline uncertainties and timely JCA report delivery. In the short term, success for the HTAR from the company perspective is measured by positive recommendations across EU jurisdictions. Long term, the focus shifts to aligning HTA methodologies and evidence requirements across the EU. Establishing metrics to assess the efficiency and effectiveness of the JCA is a key step in the HTAR’s ongoing learning journey. To enhance the efficiency of the JCA process, a list of metrics is recommended for continuous improvement, as well as establishing training programs to strengthen member states’ capabilities, fostering open dialog for sharing technology-specific insights, and creating open-source tools to support companies. Additionally, research should be conducted to understand agencies’ expectations of the JCA and how they will use its reports, grouping agencies by archetype to identify trends. A key recommendation is the development of a product-based scorecard to evaluate JCA submissions and reviews from various perspectives, ensuring the process meets stakeholders’ needs and can be effectively utilized in national decision-making. Conclusions: The JCA process offers a significant opportunity to streamline HTA decision-making across Europe. This study highlights several key measures and consideration for its successful rollout, including the need for clearer communication about the role of JCA in national decisions, measurement of rollout time components, and the development of quality evaluation frameworks. A collaborative, iterative approach, where stakeholders continually refine the system, will be essential for its effectiveness. Addressing these challenges will enable the JCA to enhance efficiency, consistency, and ultimately improve access to treatments for patients. Full article

The focus of manufacturers preparing for implementation of the EU HTA Regulation (HTAR) in 2025 has understandably been on their market access teams, and how they can be best equipped to adapt to this significant change. Considering the critical nature of market access in ensuring innovation reaches patients, it should be no surprise that the EU HTAR will have impacts far beyond this function. Here, we utilize published EU HTAR guidance, a pragmatic literature review, internal analysis, and insights from engagements with manufacturers, to outline some of the key cross-functional considerations arising from JSC and JCA, and how manufacturers should account for these in their EU HTAR readiness plans. Full article

Many countries around the world use health technology assessment (HTA) to inform reimbursement and pricing decisions. HTA is often split into two steps, called assessment and appraisal. While the term HTA itself has been defined by international consortia, there is heterogeneity in the way different stakeholders use the terms assessment and appraisal. This creates ambiguity regarding which activities are included in technology assessment. With the new EU HTA Regulation, the HTA community should urgently seek to clarify the distinction between assessment and appraisal, as the regulation aims to centralize the clinical part of technology assessment at the European level. Failure to clarify this terminology will put the ambition of the regulation such as increased efficiency and reduction in duplication at risk. In this article, we argue that the distinction between assessment and appraisal should be seen as a science/value dichotomy. We discuss the transition from centralized assessment activities to country-level appraisal, which should culminate in a categorization of the overall added benefit in a local context. Finally, we touch on the important dimension of uncertainty always present in medical decision making. Full article

Background: This work aimed to determine the role and action points for the involvement of medical societies in the European Health Technology Assessment (EU HTA) Methods: An online pre-convention survey was developed addressing four areas related to the EU HTA: (i) medical societies’ role; (ii) role of clinical guidelines; (iii) interface with the European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS); and (iv) approaching ‘best-available evidence’ (BAE). A descriptive analysis of questionnaire outcomes was conducted to inform the European Access Academy (EAA) Fall Convention 2023. Within the working groups (WGs), action points were identified and prioritised. Results: A total of 57 experts from 15 countries responded to the survey. The WGs were attended by (i) 11, (ii) 10, (iii) 12, and (iv) 12 experts, respectively, representing a variety of national backgrounds and stakeholder profiles. The most relevant action points identified were as follows: (i) incorporation of clinical context into population, intervention, comparator, outcomes (PICO) schemes, (ii) timely provision of up-to-date therapeutic guidelines, (iii) ensuring the inclusion of MCBS insights into the EU HTA process, and (iv) considering randomized controlled trials (RCTs) as the gold standard and leveraging regulatory insights if development programs only include single-arm trials. Conclusions: The involvement of medical societies is a critical success factor for the EU HTA. The identified key action points foster the involvement of patient associations and medical societies. Full article

The vision of a unified European HTA is by no means a new endeavor. At its core are the publicly declared ambitions to harmonize assessments of clinical data within the EU and avoid the duplication of efforts. Not surprisingly, these ambitions are publicly announced to be motivating the new 2022 EU HTA regulation. However, industry experts typically see more of a risk for additional bureaucracy resulting in delays, further scrutiny, and one additional EU (clinical) dossier to submit on top of all national HTA dossiers, which could be considered a duplication of effort and therefore counterproductive. Regardless of how the details of the process will be defined and how the entire process will work in practice, we can be sure that EU officials will refer to the EU HTA and Joint Clinical Assessment (JCA) in particular as a learning system. The purpose of this article is to take a closer look at the new EU HTA regulation and analyze threats and opportunities for manufacturers and what the resulting opportunities and threats will be at the affiliate level throughout the EU. Full article

Objectives: Stakeholder involvement has long been considered a success factor for a joint European health technology assessment (HTA) process, and its relevance is now anchored in the EU HTA Regulation’s (EU HTAR) legislative wording. Therefore, we aimed to explore the roles, challenges, and most important activities to increase the level of involvement per stakeholder group. Methods: At the 2022 Fall Convention of the European Access Academy (EAA), working groups addressed the involvement of patients, clinicians, regulators, health technology developers (HTD), and national HTA bodies and payers within the EU HTA process. Each working group revisited the pre-convention survey results, determined key role characteristics for each stakeholder, and agreed on the most important activities to fulfill the role profile. Finally, the activities suggested per group were prioritized by plenary group. Results: The prioritized actions for patients included training and capacity building, the establishment of a patient involvement committee, and the establishment of a patient unit at the EC secretariat. For clinicians, it included alignment on evidence assessment from a clinical vs. HTA point of view, capacity building, and standardization of processes. The most important actions for regulators are to develop joint regulatory-HTA guidance documents, align processes and interfaces under the regulation, and share discussions on post-licensing evidence generation. HTDs prioritized scientific advice capacity and the review of the scoping process, and further development of the scope of the assessment report fact checks. The top three actions for national HTA bodies and payers included clarification on the early HTD dialogue process, political support and commitment, and clarification on financial support. Conclusions: Addressing the activities identified as the most important for stakeholders/collaborators in the EU HTA process (e.g., in the implementation of the EU HTA Stakeholder Network and of the guidance documents developed by the EUnetHTA 21 consortium) will be key to starting an “inclusive civil society dialogue”, as suggested by the European Commission’s Pharmaceutical Strategy. Full article

Involvement of all relevant stakeholders will be of utmost importance for the success of the developing EU HTA harmonization process. A multi-step procedure was applied to develop a survey across stakeholders/collaborators within the EU HTA framework to assess their current level of involvement, determine their suggested future role, identify challenges to contribution, and highlight efficient ways to fulfilling their role. The ‘key’ stakeholder groups identified and covered by this research included: patients’, clinicians’, regulatory, and Health Technology Developer representatives. The survey was circulated to a wide expert audience including all relevant stakeholder groups in order to determine self-perception by the ‘key’ stakeholders regarding involvement in the HTA process (self-rating), and in a second, slightly modified version of the questionnaire, to determine the perception of ‘key’ stakeholder involvement by HTA bodies, payers, and policymakers (external rating). Predefined analyses were conducted on the submitted responses. Fifty-four responses were received (patients 9; clinicians: 8; regulators: 4; HTDs 14; HTA bodies: 7; Payers: 5; policymakers 3; others 4). The mean self-perceived involvement score was consistently lower for each of the ‘key’ stakeholder groups than the respective external ratings. Based on the qualitative insights generated in the survey, a RACI Chart (Responsible/Accountable/Consulted/Informed) was developed for each of the stakeholder groups to determine their roles and involvement in the current EU HTA process. Our findings suggest extensive effort and a distinct research agenda are required to ensure adequate involvement of the key stakeholder groups in the evolving EU HTA process. Full article