All CPD Supplements articles
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JournalFDA meetings: How to hold a successful meeting across the drug development lifecycle
This continuing professional development supplement discusses the available meeting options for products regulated by CDER and CBER, which should help the reader select the appropriate avenue to receive the Agency’s advice and develop a viable regulatory interactions strategy.
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CPD SupplementQualification of stride velocity endpoint in Duchenne Muscular Dystrophy
In April 2019, the Committee for Medicinal Products for Human Use (CHMP) issued a qualification opinion on stride velocity as an endpoint in clinical studies of medicines for the treatment of Duchenne Muscular Dystrophy (DMD).[1] Mutations in the dystrophin gene cause this inherited, x-linked disorder which causes progressive muscle weakness ...
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CPD SupplementDigital health technologies – an overview of EU and US frameworks
Digital health technologies have revolutionised the global healthcare system. This continuing professional development supplement provides an overview of the qualification frameworks for digital health technologies, which are used as drug development tools in the EU and the US.
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JournalFundamentals of regulatory writing: the document development process
Regulatory writers lead the development of high-quality regulatory documents by working in collaboration with colleagues across multiple functions. Document development is a multistep process with the regulatory writer acting as project leader. This article describes each of the stages of the process and suggests best practices for achieving timely project completion and document delivery
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CPD SupplementCTIS national pilot – Belgium
Although the CTIS is still under development, several EU countries (eg, Belgium, France, Germany, Finland and the UK prior to Brexit) are running national pilots to test the new way of working introduced by the Clinical Trial Regulation 536/2014. Pilot conditions in the various countries are different. The Heads of ...
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CPD SupplementThe EU Clinical Trial Regulation (EU) No 536/2014-The Clinical Trials Information System
This continuing professional development article addresses changes introduced by Regulation (EU) No 536/2014 (the Clinical Trial Regulation/CTR) and focuses on the revised submission process for clinical trial applications in the EU/EEA via the new EU portal
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CPD SupplementCase Study- Luxturna
The ATMP Luxturna (voretigene neparvovec) is a GTMP which utilises a recombinant adeno-associated viral vector serotype 2 (rAAV2) capsid as a delivery vehicle for the human retinal pigment epithelium 65 kDa protein (hRPE65) cDNA to the retina. Luxturna is approved for patients with retinal dystrophy associated with biallelic RPE65 mutations ...
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JournalGlobal regulatory frameworks for the development of gene therapy products
Gene therapies have the potential to revolutionise the global healthcare system. Some of these products may offer curative benefits to patients with devastating conditions. This continuing professional development (CPD) supplement provides an overview of the regulatory framework for the development of gene therapy products in key markets.
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CPD SupplementPresentation of immunogenicity-related information in regulatory dossiers
The purpose of this continuing professional development supplement is to explain how an integrated summary report can be created to consolidate the information required for assessment of immunogenicity-related risks of different types of biopharmaceuticals.
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JournalFDA regulatory pathways for medical devices
The regulations, developed as a result of the 1976 Medical Device Amendments to the Food, Drug, and Cosmetic Act of 1938, share a common goal with the pharmaceutical regulations: they both strive to ensure that new medical treatments reach the public as quickly as possible while protecting patients and ensuring that the new treatments have a positive benefit–risk balance. However, they approach this goal in different ways. This continuing professional development supplement explains the fundamentals of the FDA regulatory pathways for medical device manufacturers that wish to bring their products to the US market.