An updated systematic review on spinal muscular atrophy patients treated with nusinersen, onasemnogene abeparvovec (at least 24 months), risdiplam (at least 12 months) or combination therapies.

Objective: This systematic review provides an update on outcomes for patients with spinal muscular atrophy (SMA) type 1 to 4 treated with approved therapeutics, including the most recent, risdiplam, for an observation period of up to 48 months.

Methods: A systematic literature search was conducted in July 2023 in four databases. Selected publications were assessed for internal validity and risk of bias by two authors and relevant data were extracted into standardised tables.

[The application of negative pressure wound therapy (NPWT) in potentially lethal conditions].

Bemutatásra kerülő esetünkben egy 47 éves, generalizált septicus állapotú férfi beteg komplex terápiás megoldást igénylő kezelését ismertetjük, negatív nyomásterápia segítségével (NPWT). A páciens kezeletlen diabéteszes láb szindróma talaján kialakult szepszis, fasciitis necrotisans klinikai-radiomorfológiai képével került osztályunkra, akinél sürgősséggel feltárást, az alsó végtag valamennyi kompartmentjét érintő fasciotomiát végeztünk, NPWT-kezelést indítottunk. Kezelése során a beteg állapotát súlyosbító szövődmények léptek fel: Curling-fekély, toxicus epidermalis necrolysis (TEN).

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.

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.

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.

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.

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.

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.

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.

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.

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.

[HTA goes Europe: European collaboration on joint assessment and methodological issues becomes reality].

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.

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.

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.