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
- Reimbursement Mechanisms * MeSH
- European Union MeSH
- Rare Diseases therapy MeSH
- Publication type
- Journal Article MeSH
V posledních letech začínají na neonatologických odděleních pracovat jako členové multidisciplinárního týmu také kliničtí logopedi. Vzhledem k absenci doporučeného postupu pro práci klinických logopedů na tomto typu oddělení se množí nejasnosti ohledně toho, jaké terapie je klinický logoped oprávněn provádět. Zajímal nás názor pracovníků neonatologických oddělení na to, zda považují klinického logopeda za kompetentního v provádění následujících typů terapií: vývojová péče, bazální stimulace, Bobath handling, respirační fyzioterapie a handling, viscerální manipulace, kraniosakrální terapie a metody CBF (cue-based feeding), tedy techniky responsivního vedení příjmu potravy dle klíčových znaků chování dítěte. Využita byla metoda dotazníku, jenž byl rozeslán do všech perinatologických center II. a III. stupně. Osloveni byli pracovníci následujících profesí: lékař, sestra, fyzioterapeut, ergoterapeut a klinický logoped. Na otázku, zda může logoped provádět vývojovou péči, bazální stimulaci, Bobath koncept a viscerální manipulaci, byl respondenty z řad lékařů, sester a fyzioterapeutů vyjádřen spíše souhlas, počty kladných odpovědí se pohybovaly v rozmezí 50–65 %. U otázky na kompetenci logopedů v provádění respirační fyzioterapie a handlingu a kraniosakrální terapie převažoval nesouhlas, ten vyjádřilo více než 90 % respondentů. Oproti tomu většina respondentů (97,5 %) souhlasila s názorem, že logoped může provádět techniky vedení příjmu potravy dle klíčových znaků chování kojence. Cílem tohoto článku je informovat pracovníky z řad lékařských i nelékařských profesí působících na neonatologickém oddělení o možnostech využití dalších podpůrných doplňkových terapií, které je klinický logoped po proškolení oprávněn vykonávat.
In recent years, clinical speech therapists have begun to assume roles within the multidisciplinary teams that care for neonates in neonatal wards. In the absence of a recommended procedure for the work of clinical speech and language therapists in this type of ward, there is confusion as to which therapies clinical speech and language therapists are authorised to perform. The objective of this study was to ascertain the opinion of neonatal ward staff as to the competence of clinical speech therapists in performing the following types of therapies: developmental care, basal stimulation, Bobath handling, respiratory physiotherapy and handling, visceral manipulation, craniosacral therapy and cue-based feeding (CBF) methods, namely guiding feeding according to the child’s key behavioural features. A questionnaire was distributed to all perinatology centres at levels II and III. The following professionals were consulted: physicians, nurses, physiotherapists, occupational therapists and clinical speech therapists. When asked whether the speech therapist could perform developmental care, basal stimulation, the Bobath concept and visceral manipulation, the respondents from the medical and nursing professions, as well as physiotherapists, indicated that they believed a therapist to be competent in these areas, with the numbers of positive responses ranging from 50% to 65%. With regard to the question of competence in performing respiratory physiotherapy and craniosacral therapy, a significant proportion of respondents (more than 90%) expressed disagreement with the view that speech therapists are capable of performing these therapies. Conversely, the majority of respondents (97.5%) indicated that they believed speech therapists can effectively utilise feeding management tech- niques in accordance with the infant’s key behavioural features.
- MeSH
- Intensive Care Units, Neonatal MeSH
- Speech-Language Pathology * MeSH
- Humans MeSH
- Musculoskeletal Manipulations MeSH
- Infant, Premature * MeSH
- Infant, Newborn MeSH
- Professional Competence MeSH
- Surveys and Questionnaires MeSH
- Check Tag
- Humans MeSH
- Infant, Newborn MeSH
- Publication type
- Research Support, Non-U.S. Gov't MeSH
