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Background Tuberous sclerosis complex (TSC) is a rare autosomal dominant genetic disorder. Many gaps remain in the understanding of TSC because of the complexity in clinical presentation. The T uber O us SC lerosis registry to increase disease A wareness (TOSCA) is an international disease registry designed to address knowledge gaps in the natural history and management of TSC. Here, we present the baseline data of TOSCA cohort. Methods Patients of any age diagnosed with TSC, having a documented visit for TSC within the preceding 12 months, or newly diagnosed individuals were included. The registry includes a “core” section designed to record detailed background information on each patient including disease manifestations, interventions, and outcomes collected at baseline and updated annually. “Subsections” of the registry recorded additional data related to specific features of TSC. Results Baseline “core” data from 2093 patients enrolled from 170 sites across 31 countries were available at the cut-off date September 30, 2014. Median age of patients at enrollment was 13 years (range, 0–71) and at diagnosis of TSC was 1 year (range, 0–69). The occurrence rates of major manifestations of TSC included – cortical tubers (82.2%), subependymal nodules (78.2%), subependymal giant cell astrocytomas (24.4%), renal angiomyolipomas (47.2%), lymphangioleiomyomatosis (6.9%), cardiac rhabdomyomas (34.3%), facial angiofibromas (57.3%), forehead plaque (14.1%), ≥ 3 hypomelanotic macules (66.8%), and shagreen patches (27.4%). Epilepsy was reported in 1748 (83.5%) patients, of which 1372 were diagnosed at ≤ 2 years (78%). Intellectual disability was identified in 451 (54.9%) patients of those assessed. TSC-associated neuropsychiatric disorders (TAND) were diagnosed late, and not evaluated in 30–50% of patients. Conclusion TOSCA is the largest clinical case series of TSC to date. It provided a detailed description of the disease trajectory with increased awareness of various TSC manifestations. The rates of different features of TSC reported here reflect the age range and referral patterns of clinics contributing patients to the cohort. Documentation of TAND and LAM was poor. A widespread adoption of the international TSC assessment and treatment guidelines, including use of the TAND Checklist, could improve surveillance. The registry provides valuable insights into the necessity for monitoring, timing, and indications for the treatment of TSC.

Background Drug shortages are a growing global problem, posing clinical and economic challenges. To understand them better, we conducted an inventory of national public drug shortage registers and their comparability in Europe and the USA. Methods The study was based on openly accessible drug shortage notifications published by national drug authorities. These data were obtained from all national data sources mentioned on the European Medicines Agency’s (EMA’s) web page and FDA in the USA. After selection of the countries with comparable data, descriptive statistics were used to present characteristics of the shortages both across countries and within countries for 9 months (January–September) in 2020. We studied whether the shortages that occurred in these countries were the same, and how shortages were distributed by therapeutic uses and formulations. We also investigated price variation between the United States and Finland among drugs in shortage in one formulation category (creams and gels). Results Finland, Sweden, Norway, Spain, and the United States had suitable registers and were included. Altogether 5132 shortage reports from Finland ( n  = 1522), Sweden ( n  = 890), Norway ( n  = 800), Spain ( n  = 814), and the United States ( n  = 1106) were published during the study period. Of active ingredient level shortages 54% occurred in only one country, and 1% occurred in all five. However, at the country level, where there was one or more shortage notifications in an ATC active ingredient category, 19–41% were in a single country. The distributions by ATC therapeutic class and drug formulation differed substantially between countries, particularly between the USA and European countries. Injectables had a high shortage risk in the USA (57% of all shortages versus 17–31% of all shortages in the European countries). By contrast, shortages in gels and creams occurred only in European data (4–6% of all shortages). In the price comparison, creams and gels in shortage in Finland were 160% more expensive in the USA where these shortages were not detected. Conclusions Public drug shortage registers are vital data sources for proactively maintaining and managing a reliable drug supply. However, our study demonstrates that much work remains to standardize the contents and quality of public register data. Shortages may not be solely a consequence of manufacturing disruptions but may reflect other contributing factors in the international drug distribution and supply mechanisms, including price differences and profit margins between national pharmaceutical markets. Data to perform practical and useful international comparisons to understand these shortages are required.

PurposeThe purpose of this paper is to present a concept of the protocol for public registries based on blockchain. New database protocol aims to use the benefits of blockchain technologies and ensure their interoperability.Design/methodology/approachThis paper is framed with design science research (DSR). The primary method is exaptation, i.e. adoption of solutions from other fields. The research is looking into existing technologies which are applied here as elements of the protocol: Name-Value Storage (NVS), Berkley DB, RAID protocol, among others. The choice of NVS as a reference technology for creating a database over blockchain is based on the analysis and comparison with two other similar technologies Bigchain and Amazon QLDB.FindingsThe proposed mechanism allows creating a standard database over a bundle of distributed ledgers. It ensures a blockchain agnostic approach and uses the benefits of various blockchain technologies in one ecosystem. In this scheme, blockchains play the role of journal storages (immutable log), whereas the overlaid database is the indexed storage. The distinctive feature of such a system is that in blockchain, users can perform peer-to-peer transactions directly in the ledger using blockchain native mechanism of user access management with public-key cryptography (blockchain does not require to administrate its database).Originality/valueThis paper presents a new method of creating a public peer-to-peer database across a bundle of distributed ledgers.

This paper examines the alignment of blockchain architecture with the specific requirements of public business registers in Europe. Through a comprehensive literature review, categorization of blockchain models, and analysis of European public business registry cases, this study develops a framework to guide blockchain adoption in public registries. A distributed ledger taxonomy tailored for business registers is presented. This article also introduces a registry mapping procedure and addresses security concerns essential for the transition to blockchain-based architectures. Blockchain technology, popularized in 2009, has evolved into a versatile tool with applications across various domains, including the public sector, where its potential for decentralized solutions is especially promising. In particular, strategic systems that constitute the backbone of the legal and economic order require special considerations providing superior quality and the appropriate level of security. This study proposes a categorization method, outlines a registry mapping procedure, and discusses security concerns integral to blockchain implementation in public registries. The potential of blockchain technology to change the architecture of public business registries is also discussed.

The requirement that public officials declare their income and assets can help deter the use of public office for private gain. Income and asset disclosure (IAD) systems can provide a means to detect and manage potential conflicts of interest, and can assist in the prevention, detection, and prosecution of illicit enrichment by public officials. Growing attention to anticorruption policies, institutions, and practices has led to increased interest in financial disclosure systems and the role they can play in supporting national anticorruption strategies and in helping to instill an expectation of ethical conduct for individuals in public office. IAD systems are also a key element in the implementation and enforcement of provisions of the United Nations Convention against Corruption and other international anticorruption agreements. This attention has sparked interest among policy makers and practitioners in the design features and implementation practices that make for effective financial disclosure administration. The case studies collected in this volume are intended to profile a range of systems and practices to help respond to this growing interest.

In the text, I discuss the abilities and challenges of information systems interoperability. The anticipated and expected result of interoperability is to improve the provision of public utility services to citizens and companies by means of facilitating the provision of public utility services on the basis of a “single window” principle and reducing the costs incurred by public administrations, companies, and citizens, resulting from the efficiency of the provision of public utility services. In the article, the conceptual framework of interoperability is elaborated upon. Moreover, information systems and public registers for entrepreneurs in Poland exemplify whether the interoperability may be applied and, if so, whether interoperability fulfils its targets to the extent of e-Government services for entrepreneurs.

Micro, small and medium enterprises (MSMEs) represent more than 99% of enterprises in Europe. Therefore, knowledge about this sector, also in the spatial context is important to understand the patterns of economic and social development. The main goal of this article is an analysis of spatial conditions and the situation of MSMEs on a local level using combined sources of information. This includes data collected in the Social Insurance Institution and Tax registers in Poland, which provides information on the employment, wages, revenues and taxes paid by the MSMEs on a local level as well as contextual statistical information. The data is used for a diagnosis of spatial circumstances and discussion of conditions influencing the status of the MSMEs sector in a selected region (voivodeship) in Poland. Taxonomy methods including factor analysis and clustering methods based on k-means and SOM Kohonen were used for selecting significant information and grouping of the local units according to the situation of the MSMEs. There are eight factors revealed in principal component analysis and five clusters of local units distinguished using these factors. These include two clusters with a high share of rural local units and two clusters with a high share of rural-urban and urban local units. Additionally, there was an outstanding cluster with only two dominant urban local units. Factors show differences between clusters in the situation of MSMEs sector and infrastructure. Different spatial conditions in different regions influence the situation of MSMEs.

El Parlamento Europeo aprobó, el 19 de febrero de 2009, el Informe sobre Economía Social elaborado por la parlamentaria europea Patrizia Toia (Informe Toia). En él se reconoce la Economía Social como actor empresarial en la Unión Europea, se reclama su reconocimiento jurídico, su consideración de interlocutor social, y se advierte de la necesidad de que la economía social tenga mayor visibilidad. La Propuesta de Ley Marco de Economía Social, a cuya aproximación se dedica el presente trabajo, intenta materializar todos los aspectos enunciados del Informe Toia, partiendo del reconocimiento jurídico de la Economía Social en nuestro Estado vía elaboración de una ley ad hoc. / On February 19th, 2009, the European Parliament passed a resolution on Social Economy, drafted by European Parliament Member Patrizia Toia (the so-called Toia Report). Within the Report, Social Economy is recognised as a rightful entrepreneurial actor, claiming for both its Legal and rightful consideration and its role as a social agent, demands which serve to highlight the Social Economy needs for a higher profile. The Draft Paper for a framework Law on Social Economy, which this paper will also deal with, aims to mirror all of the above-referred Toia Report statements, starting with the very Legal recognition of Social Economy under the protection of a national ad hoc Law.

Basic registers of public administration have been fundamental part of the Czech eGovernment already for more than 4 years. Public authorities work with reference data recorded in the basic registers in rightful cases and in the scope pursuant to the legal regulations, whereas a list of these cases and relevant authorizations are filed in the Basic Register of Rights and Obligations in the form of agendas and agenda activity roles. The Register of Rights and Obligations should become a map for public authorities, their competencies and activities used to fulfil these lawful competencies, and it should be used for procedural optimization of public administration and as a background for performance allowance for state administration performance. This contribution deals with quality analysis of registration agenda in the Register of Rights and Obligations, high level of which is necessary for achieving set goals. The research is based on three different approaches: a statistical analysis of registered agenda code list, a case study of agenda changes concerning keeping a permanent list of voters and a survey of current use of agenda activity roles for two selected agendas. Synthesis of obtained results gives us a picture of quality of agenda registration and activities in the Register of Rights and Obligations.