The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products

Palomo, Gloria M.; Pose-Boirazian, Tomas; Naumann-Winter, Frauke; Costa, Enrico; Duarte, Dinah M.; Kalland, Maria E.; Malikova, Eva; Matusevicius, Darius; Vitezic, Dinko; Larsson, Kristina; Magrelli, Armando; Stoyanova-Beninska, Violeta; Mariz, Segundo

doi:10.1016/j.ymthe.2023.09.020

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2024

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Cited by 4 publications

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Gene and cell therapy in 2023: Rich pipeline, slimming resources?

High

2024

Molecular Therapy

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Gene and cell therapy in 2023: Rich pipeline, slimming resources?

High

2024

Molecular Therapy

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Towards ethical drug pricing: the European Orphan Genomic Therapies Fund

Risse,

Krzemien,

Schnalke

et al. 2024

Gene Ther

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An increasing number of novel genomic therapies are expected to become available for patients with rare or ultra-rare diseases. However, the primary obstacle to equal patient access to these orphan genomic therapies are currently very high prices charged by manufacturers in the context of limited healthcare budgets. Taking into account ethical pricing theories, the paper proposes the implementation of a pricing infrastructure covering all European member states, which has the potential to promote distributive justice while maintaining the attractiveness of genomic therapy development.

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Characterizing Gene Therapy Medicinal Products Post-Marketing Safety Reports based on WHO Pharmacovigilance Database, VigiBase

Lim,

Paek,

Shin

2024

Preprint

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Background Safety assessments of gene therapy medicinal products (GTMPs) require comprehensive data collection from a variety of sources to ensure thorough evaluation. This study aimed to analyze the characteristics of adverse event (AE) reports for GTMPs, dividing them into Solicited Reports (SRs) and Unsolicited Reports (USRs), and to provide insights for developing effective pharmacovigilance strategies customized for GTMPs.Methods Individual case safety reports (ICSRs) collected from GTMPs were retrieved using VigiBase. We identified GTMPs approved by the United States Food and Drug Administration (US FDA) and/or European Medicines Agency (EMA) by December 31, 2022, and maintained their approval status as of July 28, 2023. Descriptive statistics were used to summarize the ICSRs’ characteristics. The reported AEs were classified into System Organ Classes (SOCs) and Preferred Terms (PTs).Results A total of 12,180 ICSRs for 13 GTMPs were retrieved from VigiBase. Of these, 27.6% were SR and 72.4% USR. As the number of ICSRs increased, the proportion of USR tended to increase. Compared to the SR, consumer reports were much less common in the USR. The majority of the reported events were serious in both groups. In the drug-AE pairs, cytokine release syndrome and pyrexia were the most commonly reported in both the SR and USR. Meanwhile the most frequently reported SOCs were “Investigations” in SR and “Nervous system disorders” in USR, respectively.Conclusions Our study highlights the need for robust pharmacovigilance measures customized for GTMPs. The continued refinement of pharmacovigilance strategies customized for GTMPs is essential to ensure their safe and effective use.

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The European landscape for gene therapies in orphan diseases: 6-year experience with the EMA Committee for Orphan Medicinal Products

Cited by 4 publications

References 7 publications

Gene and cell therapy in 2023: Rich pipeline, slimming resources?

Gene and cell therapy in 2023: Rich pipeline, slimming resources?

Towards ethical drug pricing: the European Orphan Genomic Therapies Fund

Characterizing Gene Therapy Medicinal Products Post-Marketing Safety Reports based on WHO Pharmacovigilance Database, VigiBase

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