Facilitating the development of gene therapies

News 20/05/2015

EMA invites feedback on its new draft guideline

The European Medicines Agency (EMA) has released a PDF icon draft guideline on quality, non-clinical and clinical aspects of gene therapies for a three-month public consultation. The document aims to support and facilitate the development of these innovative medicines by giving guidance to developers on the types of evidence they should generate to support a marketing-authorisation application with a regulatory authority in the European Union.

Gene therapy holds great potential for the cure of many diseases, in particular diseases caused by a defective gene. 80% of rare diseases are caused by faulty genes.

These therapies are very different to conventional medicines as they consist of genetic material that needs to be delivered into the patient's cells.

Most developers of gene therapies are very small companies or come from academia, and are not familiar with the regulatory environment.

The draft guideline follows a new approach which aims to address the needs of these stakeholders. It follows the structure of the common technical document (CTD), the document that needs to be completed at the time of submission of a marketing-authorisation application.

It also provides detailed guidance on both the scientific and development aspects of this type of medicines, and on the regulatory requirements that companies need to fulfil, including good manufacturing practice requirements.

This approach is expected to help companies conduct robust development and understand and follow the different regulatory steps.

The draft guideline takes stock of the experience gained with gene therapies in recent years through scientific advice, the classification of advanced therapies and marketing-authorisation applications, and addresses issues observed during these procedures. This includes in particular critical quality issues in a number of applications involving gene therapies which have an impact on the validity of the non-clinical and clinical studies.

The draft guideline puts particular emphasis on these issues, stressing the fact that high quality standards need to be reached before non-clinical and clinical studies can be started.

Once finalised, this new guideline will replace the current PDF icon 2001 note for guidance on gene therapies .

Stakeholders can send their comments on this draft guideline to advancedtherapies@ema.europa.eu by 31 August 2015 using the form provided.

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