Regulatory
Rapporteur
Write to the Editor at publications@topra.org
May 2024 | Vol. 21 | No.5
EDITORIAL
Unlocking a new paradigm of healthcare through advanced therapies
New categories of advanced therapy medicines are emerging, most notably gene editing technologies, which offer the potential to cure disease and provide real hope to patients suffering from life-threatening or life-altering diseases.
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By Harriet Edwards
FOCUS
Reflecting innovation through FDA regulation
Harriet Edwards and Dr Katherine Bowen spoke to Dr Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA), about his career and the continued evolvement of regulation to support innovative medicines development
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By Harriet Edwards and Dr Katherine Bowen
FOCUS
Cell and gene therapy: clinical application procedure of CAR-T therapy in Europe
Due to its complexity, chimeric antigen receptor T cell (CAR-T) therapy falls under the framework for genetically modified organisms which can make the clinical trial application process challenging. This article provides an overview of the regulatory requirements for clinical trial applications involving CAR-T products in the EU.
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By Ivana Turek
FOCUS
Near-term challenges of advanced therapy medicinal products: a maturing product class
Advanced therapy medicinal products have played a transformational role in the lives of patients with rare diseases. As this class of therapies continues to advance, challenges inevitibly arise including longevity of product effect and manufacturing scalability. This article discusses these challenges and puts forward solutions to break down the barriers to accessing these innovative treatments.
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By Dr Jörg Schneider, Dr Christian K. Schneider, Carolin Denzinger and Dr Herbert Altmann
FOCUS
The first approval of a CRISPR-Cas9 gene editing medicinal product
Casgevy is the first gene-editing technology using CRISPR-Cas9 to receive marketing authorisation in the UK, EU and US. This article discusses the process through which marketing authorisation was granted for Casgevy in the EU and the relevant scientific, clinical and regulatory considerations.
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By Tom Oakley
STANDALONE
European Association of Authorised Representatives
The main point of contact between the manufacturer and the European National Competent Authorities is the European Association of Authorised Representatives. This article discusses the role of authorised representatives, the clients they serve and the challenges faced by manufacturers.
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By Ludger Moeller
STANDALONE
Navigating the process complexities and challenges associated with the EU-CTR
Following the implementation of the EU Clinical Trial Regulation on 31January 2022, there is a three-year transition period during which time sponsors are required to submit a transition application for trials running beyond 31 January 2025. This article provides insight into the challenges associated with transition applications.
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By Shivam Vashisth and Marion Schoeffel
STANDALONE
Medical device standards update
This edition of our regular column updates the progress of applicable standards for medical devices and in vitro diagnostic devices up to March 2024.
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By Mehryar Behizad
PODCAST
RegRapPod – In Conversation with Helena Corte-Real
In this episode of RegRapPod – In Conversation, host Alan Booth hears from Helena Corte-Real who is Vice President, Regulatory Portfolio Data and Content Lead with Roche. Helena has previously been a speaker at TOPRA’s Regulatory Careers Live events.
PODCAST
RegRapPod
In the May 2024 episode of RegRapPod, Alan Booth hears from Issue Editor Harriet Edwards about advanced therapy medicinal products
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