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.
Axicabtagene ciloleucel (tradename YESCARTA) is an autologous chimeric antigen receptor T-cell (CAR-T) therapy that targets CD19 and is approved for the treatment of aggressive, relapsed or refractory forms of B-cell non-Hodgkins lymphoma (NHL). The active substance is composed of a patient’s cells that have undergone ex vivo modification, which causes ...
This continuing professional development (CPD) supplement focuses on the regulatory framework available to drug developers for expediting their products’ development and review processes in the EU and US. These mechanisms are relevant for products which address an unmet medical need in the treatment of a serious and/or life-threatening condition.
As highlighted within this CPD article, a conditional marketing authorisation (CMA) can be revoked in Europe. However only one occurrence has occurred since 2006 (in April 2019). Lartruvo (olaratumab), a humanised antibody, was granted a CMA via accelerated assessment on 9 November 2016 for adult patients with a locally advanced, ...
2020-05-01T13:31:00+01:00By Paul Chamberlain
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.
2020-05-01T13:43:00+01:00By Paul Chamberlain
The purpose of this case study is to illustrate the creation of an integrated summary of immunogenicity report (ISI) that fulfils the recommendations described in current regulatory guidance for the scope of information needed to facilitate assessment of immunogenicity-related risks to support marketing authorisation of novel and biosimilar versions of ...
2020-02-01T14:11:00+00:00By Joe Cheal
Within this continuing professional development (CPD) article we introduce some practical models and tools that can be actively utilised to the benefit of any manager who is responsible for implementing change.
2020-02-01T14:19:00+00:00By Joe Cheal
The following exercise will give some insights into people’s immediate reactions to change. This could apply to you or your teams’ reactions:
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