ArticleHaematologica2025
Challenges associated with access to recently developed hemophilia treatments in routine care: perspectives of healthcare professionals.
Article in Haematologica, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
3 citing papers in PubMed.
- Disease burden and unmet medical need in severe hemophilia in Greece: insights from clinicians and patients.Frontiers in public health · 2026Article
- Global estimation of the bleeding episodes treatable with desmopressin in von Willebrand disease and hemophilia A.Haematologica · 2025Review
- Therapeutic advances in hemophilia: from molecular innovation to patient-centered global care.Frontiers in medicine · 2025Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The treatment landscape for hemophilia continues to rapidly develop, and expectations for future treatment success are high. There is limited information on the challenges of accessing new and innovative therapies. The aim of this study was to explore challenges of accessing hemophilia treatment from the perspective of healthcare professionals (HCP). A cross-sectional study design was used. A pilot-tested, online survey was distributed to hemophilia treatment centers in Australia, Canada, France, Italy, New Zealand, Republic of Ireland, Turkey, USA and UK. The questionnaire covered questions on product access, economic considerations, health technology assessment requirements, and patient organization involvement. The results were analyzed descriptively using SPSS. A total of 154 HCP completed the questionnaire. There was heterogeneity across countries, regions, and centers regarding HCP knowledge of access to novel recently developed treatments. Notable limitations to access were reported such as differences in access based on age of patient and type of product, economic considerations, and the growing influence of health technology assessment bodies. Many countries have a hemophilia patient organization that does not have a vote at the decision-making table. There is a need to empower HCP to better understand national healthcare structures and decisions that lead to access limitations. Requirements from health technology assessment bodies must be understood to optimally design clinical studies and value generation of treatment options. This may strengthen the hemophilia treatment center's voice to collectively mandate for exchange with key involved individuals, such as the payers and politicians for the provision of optimal therapy.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.