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APPLICABILITY OF THE EVIDEM MULTI-CRITERIA DECISION ANALYSIS FRAMEWORK FOR ORPHAN DRUGS – RESULTS FROM A STUDY IN 7 EURASIAN COUNTRIES.

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Baran-Kooiker A., ATIKELER K., GAITOVA K., HOLOWNIA-VOLOSKOVA M., TURCU-STIOLICA A., Kooiker C., PINIAZHKO O., CZECH M.

Acta Poloniae Pharmaceutica - Drug Research

2019

vol. 76

issue 3

581-598

Several Multi-Criteria Decision Analysis (MCDA) models for use in health technology assessment (HTA) have been developed over the years, including some for orphan drugs (OD). However, there is no general consensus yet on MCDA structure and which criteria should be included and implementation of MCDA into HTA practice has been slow so far. Our study tested the criteria preferences and possibilities for implementation of the EVIDEM MCDA framework for OD with a diverse group of 140 stakeholders in Kazakhstan, Netherlands, Poland, Romania, Russia, Turkey and Ukraine (KZ,NL,PL,RO,RU,TR,UA). The research elicited stakeholder preferences (weighting) for EVIDEM domains, criteria and sub-criteria to measure their relative importance. Correlations of work place and HTA/rare diseases experience with weighting results were investigated. Results showed that the ‘Need for intervention’ domain was assessed as the most important in: RO/NL/RU/TR, in KZ/PL the ‘Type of benefit of intervention’ and in UA ‘Economic consequences of intervention’. ‘Population Priorities’ was uniformly given a low priority. Further research, sharing of experiences and multi-stakeholder discussion is necessary to define a path forward for a robust and sustainable improvement of MCDA models and its application in orphan drug HTA. Model simplification and clarification of outcomes would be beneficial.

Bridging East with West of Europe – a comparison of orphan drugs policies in Poland, Russia and the Netherlands

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Baran A., Czech M., Kooiker C., Hołownia M., Sykut- Cegielska J.

Acta Poloniae Pharmaceutica - Drug Research

2018

vol. 75

issue 6

1409-1422

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.

Wybrane rzadkie przyczyny udaru mózgu u dzieci i młodzieży

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Pilarska E., Kopyta I.

Neurologia Dziecięca

2020

vol. 29

issue 58

71-75

Arterial ischemic stroke (AIS) in the population of children and adolescents occurs with the frequency of about 3 new cases per 100,000 children per year; it appears more often in the neonatal period, and then - in adolescence. The factors that predispose to childhood stroke are different from that of the adult population, yet very complex. Currently, the most important risk factors for AIS, due to their frequency, include arteriopathies of cerebral vessels, congenital and acquired heart diseases and thrombophilia. The clinical presentation of stroke in children depends on the location and size of ischemic focus as well as on the age of patient. Most cases of pediatric stroke come from the anterior part of brain arterial vasculature so clinical symptoms at stroke onset are paresis or paralysis of extremities and cranial nerves and aphasia. In newborns and infants the stroke symptoms are non-specific. Most children who underwent ischemic stroke present persistent outcomes, most commonly- hemiparesis, aphasia and post-stroke epilepsy. In the differential diagnosis of acute cerebral ischemia in children, however, rare causes should also be considered, especially if their diagnosis enables treatment and avoiding recurrence of stroke. The authors present selected rare diseases in the course of which ischemic strokes occur in the pediatric population.

Udar mózgu (ang. AIS, arterial ischemic stroke) w populacji dzieci i młodzieży występuje z częstością około 3 nowych zachorowań na 100 000 dzieci w ciągu roku; częściej pojawia się w okresie noworodkowym, a kolejno- w wieku adolescencji. Czynniki predysponujące do wystąpienia udaru dziecięcego są inne niż w populacji pacjentów dorosłych, a jednocześnie bardzo złożone. Aktualnie wśród najistotniejszych czynników ryzyka AIS u dzieci, z uwagi na częstość występowania, wyróżnia się arteriopatie naczyń mózgowych, wrodzone i nabyte choroby serca oraz stany sprzyjające zakrzepicy. Obraz kliniczny zależy od lokalizacji ogniska udarowego, a także od wieku pacjenta. Większość udarów dziecięcych lokalizuje się w zakresie przedniego kręgu unaczynienia mózgowia, wobec czego w obrazie klinicznym ostrej fazy choroby dominują porażenia i niedowłady kończyn oraz nerwów czaszkowych, a także afazja. U noworodków i niemowląt objawy często są niespecyficzne. Większość dzieci po przebyciu udaru mózgu prezentuje trwałe następstwa, zwłaszcza niedowłady, zaburzenia mowy o typie afazji oraz padaczkę pourazową. W diagnostyce różnicowej ostrego niedokrwienia mózgu u dzieci należy jednak uwzględnić także przyczyny rzadko występujące, zwłaszcza, jeśli ich rozpoznanie daje możliwość leczenia i uniknięcia nawrotów udaru. Autorki przedstawiają opis wybranych rzadkich schorzeń, w przebiegu których występują udary niedokrwienne mózgu w populacji pediatrycznej.

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2 Acta Poloniae Pharmaceutica - Drug Research

1 Neurologia Dziecięca

1 2020

1 2019

1 2018

2 Kooiker C.

1 ATIKELER K.

1 Baran A.

1 Baran-Kooiker A.

1 CZECH M.

1 Czech M.

1 GAITOVA K.

1 HOLOWNIA-VOLOSKOVA M.

1 Hołownia M.

1 Kopyta I.

1 PINIAZHKO O.

1 Pilarska E.

1 Sykut- Cegielska J.

1 TURCU-STIOLICA A.

Search results

APPLICABILITY OF THE EVIDEM MULTI-CRITERIA DECISION ANALYSIS FRAMEWORK FOR ORPHAN DRUGS – RESULTS FROM A STUDY IN 7 EURASIAN COUNTRIES.

Bridging East with West of Europe – a comparison of orphan drugs policies in Poland, Russia and the Netherlands

Wybrane rzadkie przyczyny udaru mózgu u dzieci i młodzieży