Pathing the way from regulatory approval to market access for gene therapy products (GTPs): An integrative review in the US, EU5, Japan and China. [PDF]
Regen TherShi J, Hu H, Ung COL.
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Navigating market access after conditional reimbursement: a communication roadmap for disinvesting orphan drugs. [PDF]
Int J Technol Assess Health CareAbdallah K +6 more
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The EMA as a potential driver of competitiveness of orphan medicines in Europe: a focus on regulatory incentives and funding opportunities. [PDF]
Orphanet J Rare DisGomez-Ferreria MA +2 more
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FDA approvals of specialty drugs, 2000-2024. [PDF]
Health Aff SchKnox SE +4 more
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Comparative TSS-seq analysis among natural variations of <i>Arabidopsis</i> reveals the genetic stability of genic and nongenic promoters. [PDF]
NAR Genom BioinformSugekawa K, Ezeh OS, Yamamoto YY.
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Trends in oncology marketing applications in the European Union: a five-year systematic review. [PDF]
Front PharmacolBasto R, Karaya S, Zwiers A.
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Horizon scanning and drug expenditure for rare diseases: three-year predictive model in Italy 2025-2027. [PDF]
Health Econ RevMarcellusi A +10 more
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Assessing rare disease understanding: a novel disease readiness level framework. [PDF]
Orphanet J Rare DisKitahara K, Kano S.
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Priced Out of Treatment: The Exorbitant Cost of Antiparasitic Drugs in the United States. [PDF]
Open Forum Infect DisAlperin C, Nguyen N, Duffey M, Clark EH.
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GeneExt: a gene model extension tool for enhanced single-cell RNA-seq analysis. [PDF]
BioinformaticsZolotarov G +2 more
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