FDA provides updated guidance for industry regarding the long term follow-up of gene therapy products

Fri, 2018 / 08 / 17

About the author

Ihr Ansprechpartner Prof. Matthias P. Schönermark, M.D., Ph.D.
Prof. Matthias P. Schönermark, M.D., Ph.D.
Founder and Managing Director
Fon: +49 511 64 68 14 – 0
Fax: +49 511 64 68 14 18
Last month, the Center for Biologics Evaluation and Research of the U.S. FDA published several new guidances for industry providing information and recommendations regarding cellular and gene therapies. Among these there is a new draft guidance focusing on the long term follow-up after administration of human gene therapy products. The finalized draft will supersede the guidance “Gene Therapy Trials – Observing Subjects for Delayed Adverse Events” from 2006. This draft guidance especially applies to gene therapies that potentially produce life-long lasting genetic effects with a potential risk of delayed adverse events following the gene therapeutic application. Already in the former guidance from 2006, the FDA advised to observe subjects for as long as 15 years after treatment with an investigational gene therapy product. The new draft was updated based on the clinical experience gained since 2006 and takes into consideration innovative gene therapy applications such as genome editing technologies, which may be associated with an elevated risk of adverse events.

In recent years, gene therapies have increasingly been in the public spotlight and five gene therapies have already been approved by the FDA and the EMA for commercial use. The release of the new draft guidance highlights once more the high actuality and the increasing interest in innovative therapies as well as the importance of such extended observation periods to monitor the safety of certain gene therapy products, even years after administration.

In our latest white paper "Successful Market Access for Gene Therapies", we describe and discuss the key strategic aspects that will become relevant with the approaching market launch of gene therapy products. In addition, possible solutions to unresolved problems, such as cost-bearers and pricing, are presented. We would be happy to provide you with our exclusive white paper on request. Simply send us an email with your contact details to contact@skc-beratung.de. We look forward to hearing from you.

Moreover, we like to invite you to our seminar "successful market access for gene therapies – strategic solutions" taking place in the Harvard Faculty Club in Cambridge, MA (USA) on September, 24th 2018. Register now and get informed about key learnings for the approval and reimbursement process of gene therapies.

BY Prof. Matthias P. Schönermark, M.D., Ph.D., managing director and Dr. Alexandra Kuhn

Sources:
FDA: Long Term Follow-Up After Administration of Human Gene Therapy Products - Draft Guidance for Industry
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