Development and Regulation of Gene and Cell-Based Therapies in Europe: A Quantification and Reflection

Ham, Renske M.T. ten; Hövels, Anke M.; Klungel, Olaf H.; Leufkens, Hubert G. M.; Hoekman, Jarno

doi:10.1016/j.tips.2019.11.007

Cited by 7 publications

(12 citation statements)

References 13 publications

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“…GTMPs are a rapidly growing therapeutic modality with the potential for curative treatment of many genetic diseases unresponsive to conventional therapies 9 . In addition to genetic defects, several other conditions that currently have limited or no effective therapeutic options can be cured with gene therapy.…”

Section: Discussionmentioning

confidence: 99%

“…GTMPs are a rapidly growing therapeutic modality with the potential for curative treatment of many genetic diseases unresponsive to conventional therapies. 9 In addition to genetic defects, several other conditions that currently have limited or no effective therapeutic options can be cured with gene therapy. However, due to their complexity, GTMP developers have faced major regulatory challenges during assessment of clinical trial applications and marketing authorisation applications 1,2 and these include aspects related to environmental risk assessment.…”

Section: Discussionmentioning

confidence: 99%

“…The current approach is not ideal for the conduct of multinational clinical trials and has been considered by many an impediment to the effective translation of research findings into clinical applications. [7][8][9] In 2019, Iglesias-Lopez et al 10 compared the procedures for ERA assessment in the European Union and the United States. Since then, there have been significant updates that impact ERA to the advice and procedures from the European Commission for clinical trial applications and EMA for marketing authorisation applications.…”

Section: Introductionmentioning

confidence: 99%

“…There are significant differences between Member States in the documentation and procedures required for authorisation of GMO aspects of clinical trials for medicines containing a GMO and a public consultation is also required in some countries. The current approach is not ideal for the conduct of multinational clinical trials and has been considered by many an impediment to the effective translation of research findings into clinical applications 7–9 …”

Section: Introductionmentioning

confidence: 99%

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Environmental risk assessment of advanced therapies containing genetically modified organisms in the EU

Whomsley

Reig

Hidalgo‐Simon

2021

Brit J Clinical Pharma

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Gene therapy medicinal products have the potential to provide curative treatment for many diseases with current limited therapeutic options. As advanced therapy medicinal products (ATMPs), these therapies undergo a centralised, single European Union authorisation by the European Medicines Agency (EMA), but the risks and potential harm to the environment and population at large are weighted in each application, and different interpretations at national level exist. A streamlined procedure is now in place to facilitate a consistent approach for the assessment of the environmental risks of medicines containing genetically modified organisms for both clinical trial applications and marketing authorisation applications. This article provides an overview of basic requirements across the EU, an overview of the new streamlined process and discusses available guidance for developers with particular emphasis on marketing authorisation applications. All these initiatives are aimed to remove hurdles for ATMP developers and facilitate faster access to patients.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Environmental risk assessment of advanced therapies containing genetically modified organisms in the EU

Whomsley

Reig

Hidalgo‐Simon

2021

Brit J Clinical Pharma

View full text Add to dashboard Cite

show abstract

“…GTMPs are a rapidly growing therapeutic modality with the potential for curative treatment of many genetic diseases unresponsive to conventional therapies [ 9 ]. In addition to genetic defects, several other conditions that that currently have limited, or no effective therapeutic options can be cured with gene therapy.…”

Section: Discussionmentioning

confidence: 99%

Environmental risk assessment of advanced therapies containing genetically modified organisms in the EU

Whomsley

Reig

Simon

2020

Preprint

View full text Add to dashboard Cite

Gene therapy medicinal products have the potential to provide curative treatment to many diseases with current limited therapeutic options. As advanced therapy medicinal products (ATMPs), these therapies undergo a centralised, single European Union authorisation by the European Medicines Agency, but the risks, and potential harm to the environment and population at large are weighted in each application, and different interpretations at national level exist. A streamlined procedure is now in place to facilitate a consistent approach for the assessment of the environmental risks of medicines containing genetically modified organisms. This article provides an overview of basic requirements across the EU, an overview of the new streamlined process and discusses available guidance for developers. All these initiatives are aimed to remove hurdles for ATMP developers and facilitate faster access to patients.

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The Evolution of Drug Regulatory Sciences in the Netherlands: More than a Country Report

Pasmooij,

Mol,

Bot

et al. 2024

Clin Pharma and Therapeutics

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In the Netherlands, drug regulatory science is a vibrant national and internationally oriented community. In this review, we present the factors that have contributed to this successful collaboration between relevant stakeholders and that led to a surge of activities around how regulatory science became embedded in the ecosystem of medicines research, clinical pharmacology, policymaking and regulation. We distinguished three pivotal episodes: (i) TI Pharma Escher‐project, (ii) Dutch Medicines Evaluation Board as catalyst of the big jump, and (iii) Regulatory Science Network Netherlands and multistakeholder engagement. The research agenda has been influenced by the dynamic evolution of legal frameworks in Europe, such as the EU orphan medicines legislation of 2001 and the EU pharmacovigilance legislation of 2012. All these developments have inspired and have raised pertinent regulatory sciences questions. Furthermore, clinical pharmacology as a discipline has been very influential in shaping regulatory science, contributing to discussions on the level of clinical evidence that is necessary to justify marketing approval of a new medicine. With a growing interest of multiple parties such as academics, European Medicines Agency, national agencies, patient organizations and EFPIA, connecting regulatory science activities is key.

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Development and Regulation of Gene and Cell-Based Therapies in Europe: A Quantification and Reflection

Cited by 7 publications

References 13 publications

Environmental risk assessment of advanced therapies containing genetically modified organisms in the EU

Environmental risk assessment of advanced therapies containing genetically modified organisms in the EU

Environmental risk assessment of advanced therapies containing genetically modified organisms in the EU

The Evolution of Drug Regulatory Sciences in the Netherlands: More than a Country Report

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