Intervention Services for Autistic Adults: An ASDEU Study of Autistic Adults, Carers, and Professionals' Experiences

The Autism Spectrum Disorders in the European Union (ASDEU) survey investigated local services' use experiences of autistic adults, carers and professionals with interventions for autistic adults. The majority of the 697 participants experienced recommended considerations prior to deciding on intervention and during the intervention plan and implementation. Psychosocial interventions were the most commonly experienced interventions, while pharmacological interventions NOT recommended for core autistic symptoms were reported by fairly large proportions of participants. Family interventions were experienced slightly more commonly by carers than adults or professionals. Less than the 26% of autistic adult responders who had experienced challenging behaviors reported receiving an intervention to change them. These results provide insights for improving gaps in service provision of interventions among autistic adults.Peer reviewe

RARE-Bestpractices: a platform for sharing best practices for the management of rare diseases

No abstract available

RARE-Bestpractices: a platform for sharing best practices for the management of rare diseases

From 7th European Conference on Rare Diseases and Orphan Products (ECRD 2014).Rare diseases; clinical practice guidelines; recommendations. RARE-Bestpractices (http://www.rarebestpractices.eu) is a 4-year project (2013-2016) funded by the EC FP7. The project aims at improving clinical management of patients with rare diseases (RD) and at narrowing the existing gap in quality of healthcare among countries. Methods: RARE-Bestpractices (http://www.rarebestpractices.eu) involves 9 EU countries, including 15 partners from academic institutions, governmental bodies, patient organizations and networks, which will exploit the added value of integrating different contributions and viewpoints. The platform is developed involving both experts in RD research as well as experts in clinical practice guidelines (CPG) and systematic reviews. Results: Project expected outputs include: 1) identification of challenges to be considered in deriving high quality standards for CPG on RD; 2) transparent procedures and criteria for the evaluation of CPG and their collection in a publicly searchable database; 3) identification of notation criteria to improve user understandability and implementation of CPG; 4) production of mechanisms to assess RD clinical research needs; 5) development of training activities targeted to key stakeholders to disseminate process and tools for developing and evaluating CPG; 6) the publication of a new scientific journal (http://rarejournal.org). Discussion: RARE-Bestpractices addresses the demands from both patients and health care providers for updated and high quality CPG on RD. The project will meet the requirements laid down by to the Directive 2011/24/EU, which endorses EU MS to develop European Reference Networks (ERNs) for RD; in fact, one main criterion for ERNs should be the competence to produce CPG and actively disseminate them among Centers of Expertise.N

RARE-Bestpractices: a platform for sharing best practices for the management of rare diseases

No abstract available

EQ-5D in Central and Eastern Europe : 2000-2015

Objective: Cost per quality-adjusted life year data are required for reimbursement decisions in many Central and Eastern European (CEE) countries. EQ-5D is by far the most commonly used instrument to generate utility values in CEE. This study aims to systematically review the literature on EQ-5D from eight CEE countries. Methods: An electronic database search was performed up to July 1, 2015 to identify original EQ-5D studies from the countries of interest. We analysed the use of EQ-5D with respect to clinical areas, methodological rigor, population norms and value sets. Results: We identified 143 studies providing 152 country-specific results with a total sample size of 81,619: Austria (n=11), Bulgaria (n=6), Czech Republic (n=18), Hungary (n=47), Poland (n=51), Romania (n=2), Slovakia (n=3) and Slovenia (n=14). Cardiovascular (20%), neurologic (16%), musculoskeletal (15%) and endocrine/nutritional/metabolic diseases (14%) were the most frequently studied clinical areas. Overall 112 (78%) of the studies reported EQ VAS results and 86 (60%) EQ-5D index scores, of which 27 (31%) did not specify the applied tariff. Hungary, Poland and Slovenia have population norms. Poland and Slovenia also have a national value set. Conclusions: Increasing use of EQ-5D is observed throughout CEE. The spread of health technology assessment activities in countries seems to be reflected in the number of EQ-5D studies. However, improvement in informed use and methodological quality of reporting is needed. In jurisdictions where no national value set is available, in order to ensure comparability we recommend to apply the most frequently used UK tariff. Regional collaboration between CEE countries should be strengthened

Autistic Adult Services Availability, Preferences, and User Experiences: Results From the Autism Spectrum Disorder in the European Union Survey

There is very little knowledge regarding autistic adult services, practices, and delivery. The study objective was to improve understanding of current services and practices for autistic adults and opportunities for improvement as part of the Autism Spectrum Disorder in the European Union (ASDEU) project. Separate survey versions were created for autistic adults, carers of autistic adults, and professionals in adult services. 2,009 persons responded to the survey and 1,085 (54%) of them completed at least one of the services sections: 469 autistic adults (65% female; 55% 50% responded "don't know"). Five of seven residential services features recommended for autistic adults were experienced by <50% of adults. The knowledge of good local services models that work well for autistic adults was generally low across all services areas. The variation in services experiences and perceptions reported by autistic adults, carers, or professionals underscore the need to query all groups for a complete picture of community services availability and needs. The results showed areas for potential improvement in autistic adult services delivery in the EU to achieve recommended standards

Изменение уровня металлотионеинов, цинка и меди в плазме крови пациентов с хроническим лимфоцитарным лейкозом

   Изучена взаимосвязь между содержанием металлотионеинов, ионов меди и цинка в плазме периферической крови при хронических лимфоцитарных лейкозах (ХЛЛ) в зависимости от прогрессирования заболевания. Показано, что у пациентов с ХЛЛ при прогрессирующем заболевании, частичной и полной ремиссии значения отношений цинка к меди в плазме крови достоверно снижались по сравнению с аналогичными показателями, характерными для практически здоровых доноров. Установлено, что в плазме крови у пациентов с ХЛЛ при прогрессировании заболевания уменьшается уровень металлотионеинов по сравнению с другими исследуемыми группами, что говорит об обнаруженной нами сниженной функции цистеинсодержащих белков-металлотионинов. На основании полученных данных можно заключить, что отношение концентрации цинка к меди и уровень металлотионенинов в плазме периферической крови могут быть потенциальными диагностическими и прогностическими маркерами при терапии ХЛЛ.   The relationship between the content of metallothioneins, copper and zinc ions in peripheral blood plasma in chronic lymphocytic leukemia (CLL) depending on the disease progression was studied. It was shown that in patients with CLL in blood plasma with a progressive disease, partial and complete remission, the values of the zinc-to-copper ratios significantly decreased in comparison to similar indicators of practically healthy donors. It was established that in patients with CLL, the level of metallothioneins in blood plasma decreases with the disease progression compared to other studied groups, which indicates a reduced function of cysteine-containing metallothionein proteins. Based on the obtained data, it can be concluded that the zinc-to-copper concentration ratio and the level of metallothionenins in peripheral blood plasma are potential diagnostic and prognostic markers in chronic leukemia.   Изучена взаимосвязь между содержанием металлотионеинов, ионов меди и цинка в плазме периферической крови при хронических лимфоцитарных лейкозах (ХЛЛ) в зависимости от прогрессирования заболевания. Показано, что у пациентов с ХЛЛ при прогрессирующем заболевании, частичной и полной ремиссии значения отношений цинка к меди в плазме крови достоверно снижались по сравнению с аналогичными показателями, характерными для практически здоровых доноров. Установлено, что в плазме крови у пациентов с ХЛЛ при прогрессировании заболевания уменьшается уровень металлотионеинов по сравнению с другими исследуемыми группами, что говорит об обнаруженной нами сниженной функции цистеинсодержащих белков-металлотионинов. На основании полученных данных можно заключить, что отношение концентрации цинка к меди и уровень металлотионенинов в плазме периферической крови могут быть потенциальными диагностическими и прогностическими маркерами при терапии ХЛЛ

Systematic review on the evaluation criteria of orphan medicines in Central and Eastern European countries.

BACKGROUND: In case of orphan drugs applicability of the standard health technology assessment (HTA) process is limited due to scarcity of good clinical and health economic evidence. Financing these premium priced drugs is more controversial in the Central and Eastern European (CEE) region where the public funding resources are more restricted, and health economic justification should be an even more important aspect of policy decisions than in higher income European countries. OBJECTIVES: To explore and summarize the recent scientific evidence on value drivers related to the health technology assessment of ODs with a special focus on the perspective of third party payers in CEE countries. The review aims to list all potentially relevant value drivers in the reimbursement process of orphan drugs. METHODS: A systematic literature review was performed; PubMed and Scopus databases were systematically searched for relevant publications until April 2015. Extracted data were summarized along key HTA elements. RESULTS: From the 2664 identified publications, 87 contained relevant information on the evaluation criteria of orphan drugs, but only 5 had direct information from the CEE region. The presentation of good clinical evidence seems to play a key role especially since this should be the basis of cost-effectiveness analyses, which have more importance in resource-constrained economies. Due to external price referencing of pharmaceuticals, the relative budget impact of orphan drugs is expected to be higher in CEE than in Western European (WE) countries unless accessibility of patients remains more limited in poorer European regions. Equity principles based on disease prevalence and non-availability of alternative treatment options may increase the price premium, however, societies must have some control on prices and a rationale based on multiple criteria in reimbursement decisions. CONCLUSIONS: The evaluation of orphan medicines should include multiple criteria to appropriately measure the clinical added value of orphan drugs. The search found only a small number of studies coming from CEE, therefore European policies on orphan drugs may be based largely on experiences in WE countries. More research should be done in the future in CEE because financing high-priced orphan drugs involves a greater burden for these countries