BACKGROUND: Out of 185 orphan medicinal products (OMPs) registered in 2015-2021, a mere 110 (59 percent) were available to Czech patients, and only 54 (29 percent) were officially reimbursed. Moreover, this proportion has steadily decreased over time. After years of public debate induced by this unsatisfactory OMP patient access, the national viewpoint shifted toward creating a special pathway for the reimbursement of OMP. Thus, a rigorous pricing and reimbursement procedure with strict timelines and elaborated methodology has been recently adopted in Czechia. METHODOLOGY: The innovative legislation follows the recommendations for value assessment and funding processes for rare diseases and incorporates additional elements of value, such as the societal perspective. First, the application with clinical evidence, cost-effectiveness, and budget impact analyses is submitted to the governmental health technology assessment (HTA) agency by the Marketing Authorization Holder or a Health Insurance Fund. Moreover, professional associations and patients' organizations are rightful participants in the proceeding, providing evidence and comments. Then, the HTA agency performs the assessment/appraisal of the evidence. It subsequently publishes the assessment report summarizing available information. The report is then forwarded to the Ministry of Health and its advisory body consisting of patients, clinical experts, health insurance funds, and the State. They critically evaluate the documents and issue a binding opinion following prespecified decision-making criteria. Based on this binding opinion, the decision is issued by the HTA agency. Thus, the role of the advisory body in this process is crucial. CONCLUSION: We believe that this novel approach may offer satisfactory patient access to orphan drugs. Moreover, it serves as a real-world example of "value-based" decision making.
- MeSH
- hodnocení biomedicínských technologií MeSH
- lidé MeSH
- rozhodování MeSH
- úhrada zdravotního pojištění MeSH
- výroba orphan drugs * MeSH
- vzácné nemoci * farmakoterapie MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- Geografické názvy
- Česká republika MeSH
- MeSH
- hematologie * ekonomika statistika a číselné údaje trendy zákonodárství a právo MeSH
- hodnocení biomedicínských technologií ekonomika organizace a řízení trendy MeSH
- imunoterapie MeSH
- lékařská onkologie * ekonomika statistika a číselné údaje trendy zákonodárství a právo MeSH
- lidé MeSH
- nádory farmakoterapie terapie MeSH
- onkologická péče - zařízení ekonomika statistika a číselné údaje MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- novinové články MeSH
- MeSH
- akademie a ústavy ekonomika organizace a řízení MeSH
- biografie jako téma MeSH
- hodnocení biomedicínských technologií * ekonomika organizace a řízení MeSH
- technologie lékařská ekonomika organizace a řízení MeSH
- Publikační typ
- novinové články MeSH
- rozhovory MeSH
- Geografické názvy
- Slovenská republika MeSH
In this paper we have compiled and summarized the steps which manufacturers and clinical investigators need to undertake to perform a target population definition during the Health technology assessment process of medical device, namely external cardio stimulator (pacemaker). Based on available data using top-down approach we have defined target population for external cardiostimulator and estimated, that the size of the target population for external pacing will not exceed 16000 patients per year in the Czech Republic and is of comparable size with other states in the region.
BACKGROUND: The reimbursement of orphan drugs (OD) is an increasingly important for country policymakers, and still insufficiently understood, especially in Central and Eastern Europe. The aim of this research was to provide a comprehensive description of country-specific health technology assessment (HTA) policies as well as evaluate the percentage of HTA recommendations and reimbursement decisions for oncology OD. In addition, the study was designed to elucidate the impact of reimbursement of these drugs on the public budget and the agreement between HTA recommendations and reimbursement decisions in the analysed countries. A questionnaire survey was used to collect data on the reimbursement status, HTA recommendation, marketing authorisation, and public expenses on reimbursement in 2014, 2015, and 2016 for all oncology drugs with an orphan designation by the European Medicine Agency in 2017 in Bulgaria, Croatia, Czechia, Estonia, Hungary, Latvia, Lithuania, Poland, Romania, and Slovakia. The agreement between the HTA recommendation and reimbursement status was assessed using the kappa coefficient. The Pearson's correlation was used to analyse the relationship between gross domestic product (GDP) and GDP per capita and reimbursement expenses. RESULTS: A total of 36 drugs were analysed (25% conditionally approved; 5.56% approved under exceptional circumstances). The share of reimbursed drugs ranged from 11.11% in Latvia to 41.67% in Poland. The highest share of positive recommendations was observed for Bulgaria and Estonia (36.11%), and the lowest, for Latvia (11.11%). The agreement varied from 0.4 for Poland to 1 for Latvia, Hungary, and Slovakia. Expenses were correlated with GDP (0.95 [0.81-0.99]), and not with GDP per capita (0.54 [- 0.136 to 0.873]). Expenses per capita were not correlated with GDP per capita (0.52 [- 0.15 to 0.87]). CONCLUSIONS: In Hungary, Latvia, and Slovakia, a positive recommendation was associated with a reimbursement, and a negative one, with the lack of reimbursement. The reimbursement of oncology OD is associated with a growing burden for public budget, and the expenses are correlated with the total GDP. The highest share of drugs with any recommendation was observed in Poland, and the lowest, in Latvia and Romania. The share of reimbursed drugs was the lowest in Latvia and the highest in Poland.
- MeSH
- hodnocení biomedicínských technologií * MeSH
- léčivé přípravky * MeSH
- lidé MeSH
- postup MeSH
- výroba orphan drugs MeSH
- zdravotní politika MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- Geografické názvy
- Česká republika MeSH
- Evropa MeSH
- Lotyšsko MeSH
- Polsko MeSH
- MeSH
- biologické markery analýza MeSH
- hodnocení biomedicínských technologií * MeSH
- imunoglobuliny - kappa-řetězce analýza MeSH
- imunoglobuliny - lambda-řetězce analýza MeSH
- imunoturbidimetrie trendy MeSH
- lidé MeSH
- paraproteinemie * diagnóza MeSH
- proteiny analýza MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- validační studie MeSH
The purpose of the note below is to familiarize readers with the current status and future initiatives of cooperation in the field of health technology assessment (HTA) in the European Union (EU). The European Commission is trying to create a framework for efficient collaboration between member states of EU in the field of HTA and is gradually working on strengthening it. The following initiatives, projects and programs have already been implemented: - Project on Coordination and Development of Health Care Technology Assessment in Europe - The European Collaboration for Assessment of Health Interventions and Technology - European Network for Health Technology Assessment - Beneluxa Initiative, The Valletta Declaration, FINOSE and Fair and Affordable Pricing - European Network for Health Technology Assessment Currently there is an ongoing proposal for an HTA Regulation of the European Parliament and the Council, which is the measurable result of this cooperation. A key element of the proposed regulation is a centralized joint clinical assessment (for HTA purposes) of the efficacy of the medicinal product/medical device.
- MeSH
- Evropská unie * MeSH
- hodnocení biomedicínských technologií * MeSH
- lidé MeSH
- vytváření politiky * MeSH
- zdravotní politika * MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- Geografické názvy
- Evropa MeSH
1 online zdroj
- MeSH
- biomedicínské technologie * MeSH
- hodnocení biomedicínských technologií MeSH
- Publikační typ
- periodika MeSH
- Konspekt
- Veřejné zdraví a hygiena
- NLK Obory
- management, organizace a řízení zdravotnictví
