Challenges associated with access to recently developed hemophilia treatments in routine care: perspectives of healthcare professionals
Bibliographic 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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How this classification was reachedexpand
Full frame distilled prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.
Codex and Gemma teacher scores by category
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.000 | 0.000 |
| Meta-epidemiology (narrow) | 0.000 | 0.000 |
| Meta-epidemiology (broad) | 0.001 | 0.000 |
| Bibliometrics | 0.000 | 0.001 |
| Science and technology studies | 0.000 | 0.000 |
| Scholarly communication | 0.000 | 0.000 |
| Open science | 0.000 | 0.000 |
| Research integrity | 0.000 | 0.000 |
| Insufficient payload (model declined to judge) | 0.000 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one teacher head, not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".