ArticleHealthcare (Basel, Switzerland)2024
Navigating Gene Therapy Access: The Case of Bulgaria in the Context of the EU Regulatory Landscape.
Article in Healthcare (Basel, Switzerland), 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 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
6 citing papers in PubMed.
- Pricing of innovative medicines and advanced therapies worldwide: an scoping review.The European journal of health economics : HEPAC : health economics in prevention and care · 2026Article
- Two Decades of Progress and Remaining Challenges: Comparative Analysis of Rare Disease Policy Implementation in Bulgaria, Romania, and Greece.Therapeutic innovation & regulatory science · 2026Article
- When Do Annuity-Based Payments Help to Address the Affordability Challenge of Funding Advanced Therapies? Insights from a Budget Impact Simulation.Journal of market access & health policy · 2026Article
- The impacts of pricing and reimbursement policies on access to cell and gene therapies across Europe.Journal of community genetics · 2026Review
- Beyond Reimbursement Status: Availability of Advanced Therapy Medicinal Products Across the European Union.Therapeutic innovation & regulatory science · 2025Article
- The Socio-Economic Burden of Spinal Muscular Atrophy: A Cost-of-Illness Study in Bulgaria.Healthcare (Basel, Switzerland) · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapies (GTs) have recently emerged as revolutionary personalized therapeutic options. Despite their promising potential, challenges such as uncertainty regarding long-term health benefits and safety, along with extreme price tags, pose significant obstacles to patient access. Within the EU, the European Medicines Agency plays a pivotal role with regards to GT market authorization. However, national authorities are responsible for pricing and reimbursement, which results in fragment patient access within the EU. This study aimed to provide an overview of the complex landscape of post-market authorization accessibility for GT products in Bulgaria, comparing it with neighboring EU countries. We applied a mixed-methods approach, including desk research, public data requests, and list price comparisons. As of 1 April 2023, 14 GTs had a valid market authorization at the EU level. In Bulgaria, Kymriah
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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.