BACKGROUND: Advanced Therapy Medicinal Products (ATMPs) represent an innovative therapeutic approach with the potential to impact the treatment of rare diseases significantly. Although authorised centrally in the European Union, their market launch differs across Member States (MS). This study aimed to describe the ATMP market availability in MS and explore potential influencing factors, providing insights into specific barriers beyond pricing and reimbursement policies. METHODS: ATMP availability was defined as the product launch in each MS. Data was collected through open governmental sources, databases, and communication with national competent authorities. Spearman's correlation coefficients were calculated to examine the relationship between ATMP availability and their characteristics (time since granting marketing authorisation, target patient population size, and cost). RESULTS: We collected the availability data on 18 ATMPs from 23 EU MS. Market uptake varied significantly, with Germany (89%), France and Italy (61%) leading. Estonia and Latvia confirmed that no ATMP has been launched on their markets yet. Six ATMPs were available in more than one-third of the analysed MS. No significant correlation was observed between ATMP availability and analysed product characteristics except for time dependency for CAR T-cell therapies. CONCLUSION: Beyond pricing and reimbursement processes, the ATMP commercialisation in particular MS is influenced by the marketing authorisation holder's decision and capacity. ATMPs face product-specific challenges in achieving EU-wide availability, including complex manufacturing, distribution, and administration processes. To increase the accessibility of innovative ATMP-based treatments, implementing the cross-border access framework or individual ATMP production under the hospital exemption is essential, especially in underserved MS.

• MeSH
• dávkové mechanismy * MeSH
• Evropská unie MeSH
• vzácné nemoci terapie   MeSH
• Publikační typ
• časopisecké články MeSH

• Publikační typ
• abstrakt z konference MeSH

Typizace lidských leukocytárních antigenů HLA-C na DNA úrovni pomocí skupinově specifické polymerázové řetězové reakce a hybridizace se sondami,určení HLA-C alelických frekvencí v naší populaci, zpřesnění výběru dárce při transplantaci kostní dřeně.

• MeSH
• HLA-C antigeny MeSH
• testování histokompatibility MeSH

• Konspekt
• Patologie. Klinická medicína
• NLK Obory
• alergologie a imunologie
• NLK Publikační typ
• závěrečné zprávy o řešení grantu IGA MZ ČR

We pooled immunogenetic data obtained in independent studies in two European populations (Italian and Czech) of patients affected by sarcoidosis. Correspondence analysis was used to investigate the associations between clinical and immunogenetic data. Two hundred and thirty-three patients were enrolled in the study, of which 126 were from the Czech Republic and 107 from Italy. Using a common protocol, we examined each patient for sex, age of disease onset, roentgenologic stage, extrapulmonary spread, and clinical course. One thousand and ten healthy individuals, HLA typed for class I and II serologic polymorphisms, served as controls. Findings that were essentially in agreement in both populations were: (1) a positive association of sarcoidosis with HLA-A1, B8, and DR3 markers, and a negative association with HLA-B12 and DR4; (2) a prevalence of HLA-DR3 and DR4 among females and of DR5 among males; (3) a relationship of B13 and B35 with early onset and of A30, B8, DR3, and DR4 with late onset of disease; (4) an association of B27 with sarcoidosis restricted to the lungs; (5) a relationship of A1, B8, B27, and DR3 to roentgenologic stage I and of B12 and DR4 to stage III; and (6) an association of HLA-DR3 with a good outcome. Population-restricted findings essentially concerned the alleles HLA-B13 and B22, the former being associated with the disease, male sex, early onset, extrapulmonary localization and relapse only in Czechs, and the latter to disease spread only in Italians. Our results seem to support the concept that immunogenetic background may at least partly account for the clinical heterogeneity of sarcoidosis.

• MeSH
• alely MeSH
• dospělí MeSH
• HLA antigeny * genetika   MeSH
• HLA-A antigeny genetika   MeSH
• HLA-A1 antigen genetika   MeSH
• HLA-B antigeny genetika   MeSH
• HLA-B27 antigen genetika   MeSH
• HLA-B35 antigen genetika   MeSH
• HLA-B8 antigen genetika   MeSH
• HLA-DR3 antigen genetika   MeSH
• HLA-DR4 antigen genetika   MeSH
• HLA-DR5 antigen genetika   MeSH
• imunogenetika MeSH
• lidé MeSH
• plicní nemoci genetika   imunologie   MeSH
• polymorfismus genetický genetika   MeSH
• sarkoidóza genetika   imunologie   patologie   radiografie   MeSH
• sexuální faktory MeSH
• studie případů a kontrol MeSH
• věk při počátku nemoci MeSH
• Check Tag
• dospělí MeSH
• lidé MeSH
• mužské pohlaví MeSH
• ženské pohlaví MeSH
• Publikační typ
• práce podpořená grantem MeSH
• Geografické názvy
• Česká republika MeSH
• Itálie MeSH

• MeSH
• HLA antigeny klasifikace   MeSH
• HLA-A antigeny MeSH
• HLA-B antigeny MeSH
• paternita MeSH
• sérotypizace MeSH

• MeSH
• ABO systém krevních skupin MeSH
• HLA antigeny MeSH
• paternita zákonodárství a právo   MeSH

• MeSH
• geny MHC třídy I MeSH
• geny MHC třídy II MeSH
• HLA antigeny MeSH
• transplantace ledvin imunologie   MeSH
• tvorba protilátek MeSH

• Publikační typ
• abstrakty MeSH

• MeSH
• biomedicínský výzkum MeSH
• imunogenetika MeSH
• isoantigeny MeSH
• lidé MeSH
• sarkoidóza etiologie   imunologie   MeSH
• Check Tag
• lidé MeSH

• MeSH
• dítě MeSH
• dospělí MeSH
• genetické nemoci vrozené MeSH
• isoantigeny MeSH
• lidé MeSH
• plicní fibróza MeSH
• Check Tag
• dítě MeSH
• dospělí MeSH
• lidé MeSH
• mužské pohlaví MeSH
• ženské pohlaví MeSH
• Publikační typ
• kazuistiky MeSH