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OVERVIEW OF REGULATORY INITIATIVES IN THE EUROPEAN UNION TO STIMULATE RESEARCH AND ACCELERATE ACCESS TO ORPHAN DRUGS AND OTHER HIGH MEDICAL NEED PRODUCTS

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Baran-Kooiker A., Czech M., Kooiker C.
Acta Poloniae Pharmaceutica - Drug Research
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2019
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vol. 76
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issue 1
3-17
EN
The European Union (EU) is continuously working towards further promoting and facilitating registration and development of OMPs. The goal of this article is to present a broad overview of existing legislation, programmes and incentives in the EU that allow for accelerated access of orphan drugs and high medical need products, as well as give some historical context and how these programs are interconnected. The authors conducted an explorative review of publicly available sources of regulatory intelligence policy and information on orphan drug and rare disease policies as well as information on accelerated assessment programs in the EU. The authors identified and looked into the following programs and initiatives: orphan drug legislation, accelerated assessment, conditional approval, Priority Medicine (PRIME), compassionate use programs, off-label use and RTU, Medicines Adaptive Pathways to Patients (MAPPs), ADAPT-SMART, HTA initiatives (EUnetHTA, Parallel Consultation), STAMP, IMI and IMI2 and the European Framework Programmes (incl. FP7 IDEAL, Asterix and Inspire). It can be concluded that promoting innovation and early access to medicines has been firmly put on the agenda in the EU, with various programs and initiatives, either national or EU-wide. Hurdles in regulatory science remain present, especially for rare diseases, but fundamental research as done in FP7 could lead to more efficient orphan drug development. Barriers in the field of HTA, pricing and reimbursement still need to be addressed to enhance patient access. The true effectiveness of all these programs will only become clear over time.
2
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Bridging East with West of Europe – a comparison of orphan drugs policies in Poland, Russia and the Netherlands

88%
Baran A., Czech M., Kooiker C., Hołownia M., Sykut- Cegielska J.
Acta Poloniae Pharmaceutica - Drug Research
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2018
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vol. 75
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issue 6
1409-1422
EN
The goal of this article is to provide an in-depth review of rare disease policies and the reimbursement of ODs in 3 European countries, two EU members (Poland, the Netherlands) and a non-EU one (Russia). A review of publicly available information on rare disorder policies and HTA processes was performed. Experts were consulted for unclear or scarce information. Russia has a five times higher frequency threshold for its rare disease definition than Poland and the Netherlands (both using the EU definition). The Netherlands has vastly increased its disease registries by instituting 300 expert centres via its National Plan, in Poland there are only 6 registries while in Russia one central registry exists. All 3 countries have an HTA process in place, however, the Russian one is relatively undeveloped. The access to ODs in the Netherlands is the broadest with 80 out of 83 EMA approved ODs reimbursed in 2015; Poland reimbursed 49, whereas Russia reimbursed 4 on the federal level and 43 in Moscow region. In all countries new rare disease policies are under development. The availability of healthcare systems solutions and the reimbursement of ODs differs greatly in all 3 countries, mainly in Russia. Even though both states are EU member with common regulations and access to EMA approved drugs, marked differences between Poland and the Netherlands in the range of policies, access to treatments and screening programs exist.
3
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Analysis of diabetes-related complications and the cost of diabetes in Poland. The methodology and the research sample used in Economedica DM-PL Survey

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Bogusławski S., Wojciechowska A., Smaga A., Strojek K., Wróbel K., Wojtyniak B., Juszczyk G., Czech M., Zdrojewski T.
European Journal of Translational and Clinical Medicine
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2023
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vol. 6
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issue 1
31-40
EN
Background: Knowledge about diabetes mellitus (DM)-related complications, the direct and indirect costs incur red by patients with DM is still insufficient in Poland. Economedica DM-PL (Economedica) survey was a cross-sectional observational study that aimed to assess these issues. Material and methods: The survey was carried out in 2016 on a representative sample of 2032 diabetic patients recruited by 195 general practioners (GP) and 48 diabetologists (D) randomly selected from the national registry of physicians. Each GP and D recruited 8 to 10 patients with DM according to the specified protocol. The study included 1938 medical questionnaires (comple ted by the GPs and Ds), 1938 questionnaires about costs (completed by the patients) and 1532 treatment adherence questionnaires. Results: The Economedica survey provided new insights into DM epidemiological and cost data. 70% of patients with type-1 DM were diagnosed with the disease before the age of 40. For 76% patients with type-2 DM, it was diagnosed aeftr the age of 50. The average monthly out-of-pocket medical costs for DM patients in 2016 reached 66.2 EUR, while their non-medical costs were 36.8 EUR. Conclusions: The methodology applied in our study proved useful for providing new dimensions of DM epidemiological and cost data in Poland.
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