Dataset Information

Compassionate use of interventions: results of a European Clinical Research Infrastructures Network (ECRIN) survey of ten European countries.

ABSTRACT:

Background

'Compassionate use' programmes allow medicinal products that are not authorised, but are in the development process, to be made available to patients with a severe disease who have no other satisfactory treatment available to them. We sought to understand how such programmes are regulated in ten European Union countries.

Methods

The European Clinical Research Infrastructures Network (ECRIN) conducted a comprehensive survey on clinical research regulatory requirements, including questions on regulations of 'compassionate use' programmes. Ten European countries, covering approximately 70% of the EU population, were included in the survey (Austria, Denmark, France, Germany, Hungary, Ireland, Italy, Spain, Sweden, and the UK).

Results

European Regulation 726/2004/EC is clear on the intentions of 'compassionate use' programmes and aimed to harmonise them in the European Union. The survey reveals that different countries have adopted different requirements and that 'compassionate use' is not interpreted in the same way across Europe. Four of the ten countries surveyed have no formal regulatory system for the programmes. We discuss the need for 'compassionate use' programmes and their regulation where protection of patients is paramount.

Conclusions

'Compassionate use' is a misleading term and should be replaced with 'expanded access'. There is a need for expanded access programmes in order to serve the interests of seriously ill patients who have no other treatment options. To protect these patients, European legislation needs to be more explicit and informative with regard to the regulatory requirements, restrictions, and responsibilities in expanded access programmes.

SUBMITTER: Whitfield K

PROVIDER: S-EPMC2997627 | biostudies-literature | 2010 Nov

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Compassionate use of interventions: results of a European Clinical Research Infrastructures Network (ECRIN) survey of ten European countries.

Whitfield Kate K Huemer Karl-Heinz KH Winter Diana D Thirstrup Steffen S Libersa Christian C Barraud Béatrice B Kubiak Christine C Stankovski Lea L Grählert Xina X Dreier Gabriele G Geismann Sebastian S Kuchinke Wolfgang W Strenge-Hesse Anke A Temesvari Zsuza Z Blasko Gyorgy G Kardos Gabriella G O'Brien Timothy T Cooney Margaret M Gaynor Siobhan S Schieppati Arrigo A Serrano Mariantonia M de Andres Fernando F Sanz Nuria N Hernández Raquel R Kreis Germán G Asker-Hagelberg Charlotte C Johansson Hanna H Asghar Adeeba A Husson Jean-Marc JM Demotes Jacques J Gluud Christian C

Trials 20101112

<h4>Background</h4>'Compassionate use' programmes allow medicinal products that are not authorised, but are in the development process, to be made available to patients with a severe disease who have no other satisfactory treatment available to them. We sought to understand how such programmes are regulated in ten European Union countries.<h4>Methods</h4>The European Clinical Research Infrastructures Network (ECRIN) conducted a comprehensive survey on clinical research regulatory requirements, i ...[more]

PMID: 21073691

Similar Datasets

Project description:The use of empirical research methods in bioethics has been increasing in the last decades. It has resulted in discussions about the 'empirical turn of bioethics' and raised questions related to the value of empirical work for this field, methodological questions about its quality and rigor, and how this integration of the normative and the empirical can be achieved. The aim of this paper is to describe the attitudes of bioethics researchers in this field towards the use of empirical research, and examine their actual conduct: whether they use empirical research methods (and if so, what methods), and whether (and how) they have made attempts at integrating the empirical and the normative.An anonymous online survey was conducted to reach scholars working in bioethics/biomedical ethics/ethics institutes or centers in 12 European countries. A total of 225 bioethics researchers participated in the study. Of those, 200 questionnaires were fully completed, representing a response rate of 42.6%. The results were analysed using descriptive statistics.Most respondents (n?=?175; 87.5%) indicated that they use or have used empirical methods in their work. A similar proportion of respondents (61.0% and 59.0%) reported having had at least some training in qualitative or quantitative methods, respectively. Among the 'empirical researchers', more than a fifth (22.9%) had not received any methodological training. It appears that only 6% or less of the 'empirical researchers' considered themselves experts in the methods (qualitative or quantitative) that they have used. Only 35% of the scholars who have used empirical methods reported having integrated empirical data with normative analysis, whereas for their current projects, 59.8% plan to do so.There is a need to evaluate the current educational programs in bioethics and to implement rigorous training in empirical research methods to ensure that 'empirical researchers' have the necessary skills to conduct their empirical research in bioethics. Also imperative is clear guidance on the integration of the normative and the empirical so that researchers who plan to do so have necessary tools and competences to fulfil their goals.

Project description:AimTo analyze access (availability, affordability and acceptability) to biologicals for Crohn's disease (CD) in ten European countries and to explore the associations between these dimensions, the uptake of biologicals and economic development.MethodsA questionnaire-based survey combined with desk research was carried out in May 2016. Gastroenterologists from the Czech Republic, France, Germany, Hungary, Latvia, Poland, Romania, Slovakia, Spain and Sweden were invited to participate and provide data on the availability of biologicals/biosimilars, reimbursement criteria, clinical practice and prices, and use of biologicals. An availability score was developed to evaluate the restrictiveness of eligibility and administrative criteria applied in the countries. Affordability was defined as the annual cost of treatment as a share of gross domestic product (GDP) per capita. Correlations with the uptake of biologicals, dimensions of access and GDP per capita were calculated.ResultsAt the time of the survey, infliximab and adalimumab were reimbursed in all ten countries, and vedolizumab was reimbursed in five countries (France, Germany, Latvia, Slovakia, Sweden). Reimbursement criteria were the least strict in Sweden and Germany, and the strictest in Hungary, Poland and Slovakia. Between countries, the annual cost of different biological treatments differed 1.6-3.3-fold. Treatments were the most affordable in Sweden (13%-37% of the GDP per capita) and the least affordable in the Central and Eastern European countries, especially in Hungary (87%-124%) and Romania (141%-277%). Biosimilars made treatments more affordable by driving down the annual costs. The number of patients with CD on biologicals per 100000 population was strongly correlated with GDP per capita (0.91), although substantial differences were found in the uptake among countries with similar economic development. Correlation between the number of patients with CD on biologicals per 100000 population and the availability and affordability was also strong (-0.75, -0.69 respectively).ConclusionSubstantial inequalities in access to biologicals were largely associated with GDP. To explain differences in access among countries with similar development needs further research on acceptance.

Project description:BackgroundThe aim of this paper was to map consumers' food hygiene practices from 10 European countries and evaluate which demographic groups are more likely to be exposed to foodborne pathogens and establish a ranking of adherence to food hygiene practices in 10 European countries.MethodsThe research design consisted of a cross-national quantitative consumer survey regarding food safety and hygiene practices during meal preparation (SafeConsume project) and was conducted in ten European countries (France, Denmark, Germany, Greece, Hungary, Norway, Portugal, Romania, Spain and UK). The survey questions were based on recommended hand hygiene practices and on observed practices from a field study performed in 90 European households from six of the countries covered by the survey (France, Hungary, Norway, Portugal, Romania, and UK). SPSS Statistics 26 (IBM Software Group, Chicago, IL) was used for the descriptive and regression analyses of the data. Regression analyses were used to check the relation between demographic characteristics, country of origin and self-reported hand hygiene practices.ResultsAccording to the regression models, families with elderly members aged over 65 showed a higher tendency to follow proper hand washing practices compared to families without elderly members. Meanwhile, families with children under the age of 6 reported being up to twice as likely to wash their hands at critical moments compared to families without children. Overall, taking into consideration the likelihood of washing hands after touching raw chicken and the percentages scores for proper hand cleaning methods and key moments for hand washing, the rank of the countries regarding proper hand hygiene practices was the following: Denmark, Greece, Norway, Romania, Hungary, Germany, UK, Portugal, France, and Spain.ConclusionsInformation and education should point both at the key moments as suggested by the Royal Society for Public Health (RSPH) and the International Scientific Forum on Home Hygiene (IFH) and safe practices. Public health burden generated by improper hand washing may be significantly reduced if education is targeted on consumers' behaviour and practices.

Dataset Information

Compassionate use of interventions: results of a European Clinical Research Infrastructures Network (ECRIN) survey of ten European countries.

Background

Methods

Results

Conclusions

Publications

Compassionate use of interventions: results of a European Clinical Research Infrastructures Network (ECRIN) survey of ten European countries.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets