[The application of negative pressure wound therapy (NPWT) in potentially lethal conditions]. / A negatív nyomással végzett sebkezelés (NPWT) alkalmazása potenciálisan letális állapotokban.

Bemutatásra kerülo esetünkben egy 47 éves, generalizált septicus állapotú férfi beteg komplex terápiás megoldást igénylo kezelését ismertetjük, negatív nyomásterápia segítségével (NPWT). A páciens kezeletlen diabéteszes láb szindróma talaján kialakult szepszis, fasciitis necrotisans klinikai-radiomorfológiai képével került osztályunkra, akinél sürgosséggel feltárást, az alsó végtag valamennyi kompartmentjét érinto fasciotomiát végeztünk, NPWT-kezelést indítottunk. Kezelése során a beteg állapotát súlyosbító szövodmények léptek fel: Curling-fekély, toxicus epidermalis necrolysis (TEN). A fascitis kapcsán kialakult kb. 6% TBSA (total body surface area) kiterjedésu hámhiányt a TEN-szindróma további epidermális állományvesztéssel tovább súlyosbította. Állapotstabilizálást, kezdeti lokalis kontroll biztosítását követoen a hámhiányos felület csökkentése érdekében a sebeket szukítettük, a feltisztult sebalapok fedése 1:3 arányban hálósított félvastag bor transzplantációjával történt. Az NPWT-kezelést a transzplantációt követoen is folytattuk. A beteg három hónapos intenzív osztályos és sebészeti kezelést követoen sebészi szempontból meggyógyult. A negatív nyomásterápia korai - a kórlefolyásnak megfelelo - adekvát üzemmódban és fedési technikával történo alkalmazása a végtagvesztéssel és életveszéllyel járó nagy fokú hámhiány esetében hatékony eszköznek bizonyult. A multidiszciplináris terápiának köszönhetoen betegünk sebészeti alapbetegségét sikeresen gyógyítottuk, azonban az évtizedes tartamú kezeletlen cukorbetegsége, SARS-Covid peumoniája, a relabáló septicus állapota során fellépo szövodmények következtében felépülni már nem tudott.

Mapping horizon scanning systems for medical devices: similarities, differences, and lessons learned.

OBJECTIVES: This article presents the mapping of horizons scanning systems (HSS) for medical devices, conducted by the Medical Devices Working Group of the International Horizon Scanning Initiative (IHSI MDWG). It provides an overview of the identified HSS, highlights similarities and differences between the systems, and lessons learned. METHODS: Potentially relevant HSS were identified through literature searches, scan of an overview of EuroScan members, and input from the IHSI MDWG members. Structured information was collected from organizations that confirmed having an HSS for medical devices. RESULTS: Sixteen initiatives could be identified, of which 11 are currently ongoing. The purposes of the HSS range from raising awareness of trends and new developments to managing informed decisions on innovative health services in hospitals. The time-horizon is most often 3 years up to a few months before market entry. Three models of identification of new technologies crystallized: a reactive (stakeholders outside HSS inform), a pro-active (actively searching multifold sources), and a hybrid model. Prioritization is often conducted by separate committees via scoring or debate. The outputs focus either on in-depth information of single technologies or on a class of technologies or on technologies in specific disease areas. CONCLUSIONS: The identified HSS share the common experience that horizon scanning (HS) for medical devices is a resource-intensive exercise that requires a dedicated and skilled team. Insights into the identified HSS and their experiences will be used in the continued work of the IHSI MDWG on its proposal for an IHSI HSS for medical devices.

Possible long COVID healthcare pathways: a scoping review.

BACKGROUND: Individuals of all ages and with all degrees of severity of the coronavirus disease (COVID) can suffer from persisting or reappearing symptoms called long COVID. Long COVID involves various symptoms, such as shortness of breath, fatigue, or organ damage. The growing number of long COVID cases places a burden on the patients and the broader economy and, hence, has gained more weight in political decisions. This scoping review aimed to give an overview of recommendations about possible long COVID healthcare pathways and requirements regarding decision-making and communication for healthcare professionals. METHODS: A systematic search in four databases and biweekly update-hand searches were conducted. In addition to guidelines and reviews, expert opinions in consensus statements or clinical perspectives were also considered. Data were systematically extracted and subsequently narratively and graphically summarised. RESULTS: Fourteen references, five guidelines, four reviews, one consensus paper, and four clinical perspectives were included. The evidence recommended that most long COVID-related healthcare should be in primary care. Patients with complex symptoms should be referred to specialized long COVID outpatient assessment clinics. In contrast, patients with one dominant symptom should be directed to the respective specialist for a second assessment. Depending on the patients' needs, further referral options include, e.g. rehabilitation or non-medical health services. Self-management and good communication between healthcare professionals and patients are crucial aspects of the long COVID management recommendations. CONCLUSIONS: The quality of the included guidelines and reviews is limited in the methods applied due to the novelty of this topic and the associated urgency for research. Hence, an update review with more rigorous data is recommended. Furthermore, the systematic collection of real-world data on long COVID surveillance needs to be set up soon to gather further information on the duration and severity of long COVID and thereby facilitate long COVID care planning.

EUnetHTA relative effectiveness assessments: efforts to increase usability, transparency and inclusiveness.

OBJECTIVES: The objective of the European Network for Health Technology Assessment (EUnetHTA) Joint Action 3 (JA3) was to develop a sustainable European model for future collaboration on HTA, by reducing duplication in HTA production and increasing patient access to health technologies. Compared to the previous JA2, several procedural changes were made aiming to increase usability, transparency, and inclusiveness of relative effectiveness assessments (REAs). This article presents and highlights these changes, explains their rationale as well as their influence on HTA production. METHODS: Feedback from REA teams and project managers was collected. At the end of JA3, all lessons learned were mapped, resulting in a set of recommendations for a future REA production process. RESULTS: In JA3, forty-three EUnetHTA REAs have been produced. Efforts to increase the usability of the REAs were made by focussing on the needs of REA producers and users (HTA agencies) and by increasing stakeholder involvement. Huge steps were taken with regard to transparency, which was achieved through publication of guidances, templates, and up-to-date information on the EUnetHTA website. In an attempt to improve inclusiveness, (stakeholder) interaction and involvement as well as feedback procedures were enhanced and streamlined. The fine-tuned project management brought all aspects together and facilitated a consistent and reliable workflow. CONCLUSIONS: Despite that HTA agencies have different national requirements, the procedural changes made in JA3 proved to counteract some of these challenges. Nevertheless, it is of utmost importance that further perceived methodological differences are being resolved to ensure a strong base for future European collaboration on REA production.

Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data.

OBJECTIVES: This systematic review aimed to assess mid- and long-term (at least 12 months) real-world study data from all types of spinal muscular atrophy (SMA) patients treated with any of the approved drugs or combination therapies. METHODS: A systematic literature search was carried out in five databases. Two authors selected the studies based on pre-defined selection criteria and independently graded the risk of bias at study level. RESULTS: Five hundred forty-six records were identified in the literature search and 22 studies (in 26 publications) were included in the analysis. Nusinersen, onasemnogene abeparvovec and combination therapies improved motor endpoints in SMA type 1 patients. SMA type 2 to type 4 patients treated with nusinersen showed stabilisation or small improvements in motor endpoints with some deterioration observed. Quality of life endpoints, such as respiratory and nutritional support were poorly reported on. Drug-related adverse events occurred rarely in all types of SMA patients with all assessed drugs. Mid- and long-term studies on risdiplam could not be identified. CONCLUSIONS: The large quantity of missing data and heterogeneity of studies hinder comparability. Although stability and further improvement on the long-term is still uncertain, the results from the included evidence, as well as from pivotal trials show a striking contrast to the natural progression of the disease.

Synthesizing quantitative and qualitative information on multiple comparisons of health interventions to facilitate knowledge transfer: an example from an EUnetHTA multi-HTA.

OBJECTIVE: Presenting an approach to synthesize quantitative and qualitative information from systematic reviews of multiple health interventions. METHODS: Within the context of an EUnetHTA multi-health technology assessment of twenty-three surgical techniques, we developed synthetic single tables, using color gradients and abbreviations, with information on which technologies had been compared, estimates of the size of differences for available comparisons, their clinical relevance, and certainty of the related evidence. RESULTS: The proposed methodology provided, through a single depiction, information normally included in multiple figures/tables such as network plots, league tables, and summary of findings tables. CONCLUSION: Transferring information on benefits, risks, and certainty of the available evidence on health interventions may be challenging, especially when assessing multiple treatments: more pieces of information need to be integrated in order to show an overall picture for each of the chosen outcomes, and usual reporting tools may be targeted to researchers more than to different kinds of decision makers. While more in-depth layers of information can always be added to satisfy needs of different audiences, the proposed tools could favor a quick interpretation of articulated scientific data by both decision makers and researchers.

Developing a quality management system for the European Network for Health Technology Assessment (EUnetHTA): toward European HTA collaboration.

OBJECTIVES: The European Network for Health Technology Assessment (EUnetHTA) was established in 2006 and comprises over eighty organizations from thirty European countries. In its fifth project phase (Joint Action 3), EUnetHTA set up a quality management system (QMS) to improve the efficiency and standardization of joint work. This article presents EUnetHTA's new QMS and outlines experiences and challenges during its implementation. METHODS: Several working groups defined processes and methods to support assessment teams in creating high-quality assessment reports. Existing guidelines, templates, and tools were refined and missing parts were newly created and integrated into the new QMS framework. EUnetHTA has contributed to Health Technology Assessment (HTA) capacity building through training and knowledge sharing. Continuous evaluation helped to identify gaps and shortcomings in processes and structures. RESULTS: Based on a common quality management concept and defined development and revision procedures, twenty-seven partner organizations jointly developed and maintained around forty standard operating procedures and other components of the QMS. All outputs were incorporated into a web-based platform, the EUnetHTA Companion Guide, which was launched in May 2018. Concerted efforts of working groups were required to ensure consistency and avoid duplication. CONCLUSIONS: With the establishment of a QMS for jointly produced assessment reports, EUnetHTA has taken a significant step toward a sustainable model for scientific and technical collaboration within European HTA. However, the definition of processes and methods meeting the numerous requirements of healthcare systems across Europe remains an ongoing and challenging task.

European Collaboration in Health Technology Assessment (HTA): goals, methods and outcomes with specific focus on medical devices.

The European Network for Health Technology Assessment (EUnetHTA) was founded to support efficient production and use of health technology assessments (HTAs) across Europe by reducing redundancies through collaboration. To facilitate collaboration, a range of practical tools, methods and process definitions were developed. The article describes when and how these tools and methods are used along the HTA process with specific focus on "other technologies", that is medical devices and non-pharmaceutical procedures. EUnetHTA was able to deliver tangible achievements complying with its goals. The practical tools and the developed methods formed a basis for close collaboration among over 70 agencies at a European level. The activities of EUnetHTA laid a strong foundation for sustainable cooperation. In the long run, jointly produced assessments could realise economies of scale with improved quality, consistency and transparency for the health systems in Europe.

Novel Surgical Treatments for Gastroesophageal Reflux Disease: Systematic Review of Magnetic Sphincter Augmentation and Electric Stimulation Therapy.

Electric stimulation therapy (EST) and magnetic sphincter augmentation (MSA) represent novel methods for the surgical treatment of gastroesophageal reflux disease (GERD). The aim of this review was to assess the effectiveness and safety of EST and magnetic sphincter augmentation device (MSAD) comapred to laparoscopic fundoplication (LF) and proton pump inhibitor therapy (in case of EST). We performed a systematic literature search without restrictions on publication dates in five electronic databases (MEDLINE, EMBASE, the Cochrane library, PubMed, and Centre for Reviews and Dissemination), complemented by hand search, search in trial registries, and documentation provided by the manufacturers. No study passed inclusion criteria for analyzing EST effectiveness. Concerning safety, lead erosion through the esophagus and trocar perforation of the small bowel occurred in 2.4% of patients (in one study). Only the registry study fulfilled inclusion criteria for effectiveness analysis of MSAD. The crucial outcome of GERD-health-related quality of life (HRQL) score improved from 20 to 3 points in MSAD patients, and from 23 to 3.5 points in LF patients. However, the LF patients were in a more severe stage of the disease. The results yield indefinite conclusions about the use of both MSAD and EST. Clinical effectiveness and safety of both MSAD and EST are not sufficiently proven and are yet to be supported by high quality evidence from randomized controlled trials.

Factors influencing coverage decisions on medical devices: A retrospective analysis of 78 medical device appraisals for the Austrian hospital benefit catalogue 2008-2015.

BACKGROUND: Health technology assessments of medical devices (MD) present a well-recognized challenge to evaluators: the evidence on safety and clinical effectiveness is often of lower quality than for pharmaceuticals making a reliable assessment of the risk-benefit ratio difficult. Thus other factors might gain importance in decision making. OBJECTIVE: To analyse which factors impact MD reimbursement decisions within the Austrian appraisal programme on "extra medical services" (procedures reimbursed in addition to case flat rates) for inpatient care over the past eight years. METHODS: We collected variables on evidence base and device characteristics from all MD appraisals and assessed their impact on the reimbursement decision by means of odds ratios. Separate analyses were carried out for subgroups based on the risk class of the medical device subject of the assessment or the number of randomised controlled trials (RCTs) available for the assessment. RESULTS: Of 59 devices, 23 (39%) were accepted for reimbursement (18 with restrictions) and 36 (61%) were rejected. Variables addressing the quality of the evidence base were positive predictors for risk class II devices only, whereas no significant association could be determined in devices of risk class III. Inversely, high risk device characteristics were positive predictors in the subgroup not supported by RCTs only. CONCLUSION: Our data indicate that the combination of high risk characteristics and a low evidence base are factors favouring a positive reimbursement decision of MD, albeit with restrictions. Further research should analyse if these restrictions are appropriate to generate evidence development and to contain risks associated with early access to these MD.

[HTA goes Europe: European collaboration on joint assessment and methodological issues becomes reality]. / HTA goes Europe: Europäische Zusammenarbeit in gemeinsamen Nutzenbewertungen und methodischen Fragen wird real.

INTRODUCTION: The standardisation of European HTA and thus the reduction of redundancies require clearly defined processes and methods. The HTA Core Model®, a tool developed by the European Network EUnetHTA, is intended to ensure the transparent production of standardised and high-quality assessments in international collaboration. METHODS: The present paper describes the experience with already published EUnetHTA assessments as well as possibilities for national/local adaptations of these assessments. The integration of jointly developed methods in routine processes of individual HTA agencies will be explained on the basis of a selected example. Further methodological initiatives in Europe will be presented. RESULTS: So far, EUnetHTA has published four rapid assessments conducted through European cooperation between 6-9 HTA institutes during Joint Action 2 (2012-2015). Two assessments dealt with pharmaceuticals and two with non-pharmaceutical interventions. The overall duration of these assessments ranged from 7 to 9 months. There is initial information about the frequency and manner in which these assessments have been used for national/local HTA reports. According to a survey, a total of 28 HTA institutes have indicated that they want to make use of these assessments in their own context. In Austria, the Ludwig Boltzmann Institute for Health Technology Assessment (LBI-HTA) has produced two reports based on EUnetHTA assessments. A further step towards cross-border collaboration and harmonisation is the implementation of these tools in a national and regional context. Beginning in 2015 the LBI-HTA will adjust two programme lines to the format of the HTA Core Model® in order to increase the transferability of HTAs and to reduce redundancies. DISCUSSION: Barriers to European collaboration include the relevance of topics for individual HTA institutes and the timing of joint assessments. Implementing commonly developed methods as standard practice in local/national HTA institutes is mainly impeded by legislative requirements. CONCLUSION: Despite the initial positive experiences with international collaboration on specific topics and methods, the coming years will have to prove whether existing barriers can be overcome effectively.

Contrasting clinical evidence for market authorisation of cardio-vascular devices in Europe and the USA: a systematic analysis of 10 devices based on Austrian pre-reimbursement assessments.

BACKGROUND: European medical device regulation is under scrutiny and will be re-regulated with stricter rules concerning requirements for clinical evidence for high-risk medical devices. It is the aim of this study to analyse the differences between Europe and USA in dealing with risks and benefits of new cardio-vascular devices. METHODS: Since no information is available on clinical data used by the Notified Body for CE-marking, data from Austrian pre-reimbursement assessments close to European market approval were used as proxy and compared with clinical data available at time of market approval by FDA in the USA. RESULTS: 10 cardio-vascular interventions with 27 newly CE approved medical devices were analysed. The time lag between market authorisation in Europe and in the USA is 3 to 7 years. Only 7 CE-marked devices also hold a FDA market approval, 7 further devices are in FDA approved ongoing efficacy trials. For 4 of the CE-marked devices the FDA market application or the approval-trial was either suspended due to efficacy or safety concerns or the approval was denied. Evidence available at time of CE-marking are most often case-series or small feasibility RCTs, while large RCTs and only in rare cases prospective cohort studies are the basis of FDA approvals. Additionally, the FDA often requires post-approval studies for high-risk devices. CONCLUSIONS: Market authorisation based on mature clinical data deriving from larger RCTs and longer follow-ups do not only change the perspective on the risk-benefit ratio, but also secures real patient benefit and safety and assures payers of investing only in truly innovative devices.

Planned and ongoing projects (pop) database: development and results.

OBJECTIVES: The aim of this study was to present the development, structure and results of a database on planned and ongoing health technology assessment (HTA) projects (POP Database) in Europe. METHODS: The POP Database (POP DB) was set up in an iterative process from a basic Excel sheet to a multifunctional electronic online database. The functionalities, such as the search terminology, the procedures to fill and update the database, the access rules to enter the database, as well as the maintenance roles, were defined in a multistep participatory feedback loop with EUnetHTA Partners. RESULTS: The POP Database has become an online database that hosts not only the titles and MeSH categorizations, but also some basic information on status and contact details about the listed projects of EUnetHTA Partners. Currently, it stores more than 1,200 planned, ongoing or recently published projects of forty-three EUnetHTA Partners from twenty-four countries. Because the POP Database aims to facilitate collaboration, it also provides a matching system to assist in identifying similar projects. Overall, more than 10 percent of the projects in the database are identical both in terms of pathology (indication or disease) and technology (drug, medical device, intervention). In addition, approximately 30 percent of the projects are similar, meaning that they have at least some overlap in content. CONCLUSIONS: Although the POP DB is successful concerning regular updates of most national HTA agencies within EUnetHTA, little is known about its actual effects on collaborations in Europe. Moreover, many non-nationally nominated HTA producing agencies neither have access to the POP DB nor can share their projects.