Criteria to define innovation in the field of medical devices: a Delphi approach.

Defining innovation in the field of medical devices can be extremely challenging due to the peculiarity of the products within this class. Short life-cycle, incrementality, learning curve effect, impact of the organizational setting, uncertainty of effect and level of evidence are only some of these aspects. A clear set of criteria to define innovation would be of paramount relevance in this field. Twelve criteria to define innovation were proposed to a multistakeholder panel within a consensus process. A Delphi method on two rounds was used to reach consensus. In total, 53 of the 93 (47%) invited panelists responded to the first round of the survey. Among them, 51 (96%) completed also the second round. At the first round, consensus was reached for four of the 12 proposed criteria. Three of the remaining eight criteria reached consensus at the second round. It was not possible to reach consensus for the remaining five criteria. The criteria that collected the highest scores (close to 100%) were from the clinical impact domain, namely the ability of the technology to offer significant advantages over existing alternatives in terms of improving relevant clinical outcomes, and the ability to address an unmet need defined in terms of unavailability of diagnosis/treatment alternatives. High levels of consensus (about 80%) were registered on criteria belonging to non-clinical domains of analysis and, in particular, the ability of the technology to introduce organizational benefits, and the ability of the technology to bring cost reduction providing the same clinical benefit of current alternatives.

The value and impact of health technology assessment: discussions and recommendations from the 2023 Health Technology Assessment International Global Policy Forum.

Health technology assessment (HTA) programs inform decision making about the value and reimbursement of new and existing health technologies; however, they are under increasing pressure to demonstrate that they are a cost-effective use of finite healthcare resources themselves. The 2023 HTAi Global Policy Forum (GPF) discussed the value and impact of HTA, including how it is assessed and communicated, and how it could be enhanced in the future. This article summarizes the discussions held at the 2023 HTAi GPF, where the challenges and opportunities related to the value and impact of HTA were debated. Core themes and recommendations identified that defining the purpose of value and impact assessment is an essential first step prior to undertaking it, and that it can be done through the use and expansion of existing tools. Further work around aligning HTA programs with underlying societal values is needed to ensure the long-term value and impact of HTA. HTA could also have a role in assessing the efficiency of the wider health system by applying HTA methods or concepts to broader budgetary allocations and organizational aspects of health care. Stakeholders (particularly patients, industry, and clinicians but also payers, wider society, and the media) should ideally be actively engaged when undertaking the value and impact assessment of HTA. More concerted efforts in communicating the role and remit of HTA bodies would also help stakeholders to better understand the value and impact of HTA, which in turn could improve the implementation of HTA recommendations and application to future actions in the lifecycle of technologies.

Duchenne muscular dystrophy in Italy: A systematic review of epidemiology, quality of life, treatment adherence, and economic impact.

OBJECTIVE: This systematic review aims to update the evidence on Duchenne muscular dystrophy (DMD) in Italy, describing the epidemiology, quality of life (QoL) of patients and caregivers, treatment adherence, and economic impact of DMD. METHODS: Systematic searches were conducted in PubMed, Embase and Web of Science up to January 2023. Literature selection process, data extraction and quality assessment were performed by two independent reviewers. Study protocol was registered in PROSPERO (CRD42021245196). RESULTS: Thirteen studies were included. The prevalence of DMD in the general population is 1.7-3.4 cases per 100,000, while the birth prevalence is 21.7-28.2 per 100,000 live male births. The QoL of DMD patients and caregivers is lower than that of healthy subjects, and the burden for caregivers of DMD children is higher than that of caregivers of children with other neuromuscular disorders. The compliance of real-world DMD care to clinical guidelines recommendations in Italy is lower than in other European countries. The annual cost of illness for DMD in Italy is € 35,000-46,000 per capita while, adding intangible costs, the total cost amounts to € 70,000. CONCLUSION: Although it is a rare disease, DMD represents a significant burden in terms of quality of life of patients and their caregivers, and economic impact.

Health technology assessment 2025 and beyond: lifecycle approaches to promote engagement and efficiency in health technology assessment.

Lifecycle considerations have always been part of health technology assessment (HTA). However, the concept of taking a fuller, more holistic "lifecycle approach" is gaining interest in the HTA community. The 2022 HTAi Global Policy Forum (GPF) discussed how adopting a lifecycle approach could promote stakeholder engagement and robust evidence generation, and whether it could enhance information sharing and transparency across stakeholder groups. This article summarizes the discussions held at the 2022 HTAi GPF and subsequent HTAi Annual Meeting panel session that debated some of the key challenges and opportunities, with particular focus on the pre- and postmarket and disinvestment phase activities. Core themes and recommendations identified that collaboration and patient involvement are happening but still needs to be strengthened, and moving to disease-based approaches may help, although individual contexts still need to be considered. Appropriately developed and mandated core outcome sets may help with information sharing and efficiency in all lifecycle activities. Further, methods for the appropriate use of big data and digital data collection should be developed and driven by the HTA community. The value of lifecycle activities should be reviewed; in particular, scientific advice appears valuable, but the magnitude of effect is somewhat unknown due to the challenges around the confidential nature of these activities. Not all lifecycle activities can be conducted for every technology, and while there is a move away from disinvestment phase activities, more structured prioritization criteria are required. This article ends with suggested next steps to bring forward some of the priority recommendations.

A collaborative pilot on current awareness alerts for disinvestment and horizon scanning.

OBJECTIVE: In 2019, members of the Health Technology Assessment international (HTAi) Interest Group for Disinvestment and Early Awareness (DEA-IG) and the HTAi Interest Group for Information Retrieval (IR-IG) agreed to produce quarterly current awareness alerts for members of the DEA-IG. The purpose was to pilot a predefined strategy for sharing new publications on methods and topical issues in this area. METHODS: Literature search strategies for PubMed and Google were developed. Retrieved citations were posted on the DEA-IG Web site. Members of the DEA-IG received an email notification when new alerts were available. An informal survey of the DEA-IG members was used to provide feedback after the pilot. RESULTS: Six alerts were issued during the pilot (June 2019-September 2020) with a total of 170 citations. The bulk of the information were 124 PubMed indexed citations, and of these, 96 were retrieved by the PubMed search strategies. Google searches were not found to be useful, but ongoing horizon scanning work at the Canadian Agency for Drugs and Technologies in Health (CADTH) provided additional information. Based on retrospective sorting, we considered thirty-five PubMed citations to be highly relevant for health technology assessment (HTA). The response rate to the survey was limited (seventeen respondents), but most respondents found the alerts useful for their work. CONCLUSIONS: The results of this pilot project can be used to revise search strategies and information sources, improve the relevance of the alerts, and plan for expanded dissemination strategies.

Defining priority medical devices for cancer management: a WHO initiative.

Medical devices are indispensable for cancer management across the entire cancer care continuum, yet many existing medical interventions are not equally accessible to the global population, contributing to disparate mortality rates between countries with different income levels. Improved access to priority medical technologies is required to implement universal health coverage and deliver high-quality cancer care. However, the selection of appropriate medical devices at all income and hospital levels has been difficult because of the extremely large number of devices needed for the full spectrum of cancer care; the wide variety of options within the medical device sector, ranging from small inexpensive disposable devices to sophisticated diagnostic imaging and treatment units; and insufficient in-country expertise, in many countries, to prioritise cancer interventions and to determine associated technologies. In this Policy Review, we describe the methods, process, and outcome of a WHO initiative to define a list of priority medical devices for cancer management. The methods, approved by the WHO Guidelines Review Committee, can be used as a model approach for future endeavours to define and select medical devices for disease management. The resulting list provides ready-to-use guidance for the selection of devices to establish, maintain, and operate necessary clinical units within the continuum of care for six cancer types, with the goal of promoting efficient resource allocation and increasing access to priority medical devices, particularly in low-income and middle-income countries.

Implantable devices for the closure of patent foramen ovale in adults: an Italian rapid health technology assessment.

Percutaneous closure of patent foramen ovale (PFO) is offered to patients suffering from cryptogenic stroke, transient ischemic attack or persistent migraine. Several PFO closure devices are CE marked but none have yet been approved by the FDA. On the Italian market 11 different PFO closure devices are available and more than 2500 PFO procedures were reported in 2012. We report findings of a systematic review and meta-analysis of studies in which patients with PFO and transient ischemic attacks, cryptogenic stroke or persistent migraine who underwent PFO closure were compared to patients treated by usual care. We included five controlled clinical trials and one randomized controlled trial. Included studies had poor methodological quality and heterogeneity. In the included randomized controlled trial, 4.7% of procedure-related serious adverse events were observed. Large multicenter, sufficiently powered, and properly randomized trials need to be conducted in Europe with particular attention to patient selection.

Implementing a national early awareness and alert system for new and emerging health technologies in Italy: the COTE Project.

OBJECTIVES: The aim of this study was to establish a national Early Awareness and Alert (EAA) system for the identification and assessment of new and emerging health technologies in Italy. METHODS: In 2008, Agenas, a public body supporting Regions and the Ministry of Health (MoH) in health services research, started a project named COTE (Observatory of New and Emerging Health Technologies) with the ultimate aim of implementing a national EAA system. The COTE project involved all stakeholders (MoH, Regions, Industry, Universities, technical government bodies, and Scientific Societies), in defining the key characteristics and methods of the EAA system. Agreement with stakeholders was reached using three separate workshops. RESULTS: During the workshops, participants shared and agreed methods for identification of new and emerging health technologies, prioritization, and assessment. The structure of the Horizon Scanning (HS) reports was discussed and defined. The main channels for dissemination of outputs were identified as the EuroScan database, and the stakeholders' Web portals. During the final workshop, Agenas presented the first three HS reports produced at national level and proposed the establishment of a permanent national EAA system. CONCLUSIONS: The COTE Project created the basis for a permanent national EAA system in Italy. An infrastructure to enable the stakeholders network to grow was created, methods to submit new and emerging health technologies for possible evaluation were established, methods for assessment of the technologies selected were defined, and the stakeholders involvement was delineated (in the identification, assessment, and dissemination stages).

Health Technology Assessment: managing the introduction and use of medical devices in clinical practice in Italy.

Technology assumes a key role in current clinical practice. A number of innovative or improved products are constantly being launched on the market and offered directly to the users (i.e., clinicians) or even to the patients. However, in most cases, the regulation for admission to commerce is slower than the innovation process and may be inadequate for assessing the real clinical effectiveness and safety of medical devices in the premarket phase. Health Technology Assessment (HTA) can be used as a tool for the evaluation of clinical effectiveness, cost-effectiveness and risk to patients of medical devices. HTA products (e.g., periodic reports) may aid healthcare payers to make informed choices regarding the appropriate use, coverage and reimbursement of medical devices. We present the strengths and limitations of the first three Italian HTA reports we coauthored and critically explore some of the aspects related to the introduction, adoption and use of medical technologies in clinical practice.

Bicompartmental device for dynamic cell coculture: design, realisation and preliminary results.

Low-shear perfusion and compartmental systems are very important conceptual designs for the realization of high-density cell cultures and cocultures, thus allowing very realistic models and therefore reliable results. In this work, we combines low-shear perfusion and multi-compartments in one device. We also describe the design, fabrication and validation of a bicompartmental, low-perfusion culture chamber specifically designed for applications in various fields, such as tissue engineering, pharmacology, molecular biology, pharmacokinetics, nanomedicine, and drug delivery testing. The device properties in terms of flow features and metabolite exchange capabilities are fully characterized. In vitro tests performed on cell lines confirm the effectiveness of the device as an experimental tool for measurements in dynamic and multiple-cell cultures and cocultures.

Controlled in vitro growth of cell microtubes: towards the realisation of artificial microvessels.

The aim of this study is to realise in vitro microtubular structures for Tissue Engineering applications. The strategy adopted consists in the use of cylindrical polymeric scaffolds, on which connective tissue cells (murine fibroblasts) can adhere and proliferate. We supply stereotropic and chemical stimuli which induce the cells to cover the whole cylindrical structure and to produce an extra-cellular matrix (ECM) which acts as an adhesive for cell aggregation and assembly. Stereotropic stimuli were provided using purpose designed seeding and proliferation chambers whilst the addition of ascorbic acid to the culture medium furnished chemical stimuli. To obtain cylindrical cellular structures two approaches are considered: the first one consists in the use of biodegradable scaffolds, the second one consists in the use of non-biodegradable scaffolds which can subsequently be withdrawn. The results show that a 14-microm thick cell and ECM coating grows over nylon strands, and can also be coerced to form an effective connection system with external tubing.