FDA braces for increase in cell and gene therapies

By 2020, the FDA expects to receive over 200 IND applications per year, with approvals reaching 20 per year in 2025

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Natasha Taylor

The US Federal Drug Administration Commissioner Scott Gottlieb has said the agency is “witnessing a surge of cell and gene therapy products entering early developing” predicting that this will mean the number of product approvals will grow in the coming years.

By 2020, the FDA expects to receive more than 200 Investigational New Drug (IND) applications per year, building upon the total of more than 800 active cell-based or directly administered gene therapy INDs currently on file with the FDA. Based on the success of the current pipeline, the FDA expects that by 2025, approvals for cell and gene therapy products will rise to 10-20 per year.

To manage this rise in applications, the FDA plan to expand their review group, with the eventual goal of adding 50 additional clinical reviewers.

By 2025, the FDA expects to be approving 10-20 new cell and gene therapy products every year

Dr Gottlieb noted that “gene therapy products now have the potential to cure intractable diseases, and fundamentally alter the trajectory of many other vexing illnesses”. To advance these opportunities, the FDA plans to introduce new policy guidelines.

This guidance will focus on helping sponsors to make maximum use of their expedited programs, including regenerative medicine advanced therapy (RMAT) designation and accelerated approval. The accelerated approval pathway could offer a faster route to approval for new treatments and provide the FDA with additional tools for post market follow-up studies. This is crucial for therapies where it may not be feasible to conduct pre-market trials to address all theoretical risks in any reasonably sized study.

Focusing on post market follow up studies would allow for faster routes to approval and larger data sets to be reviewed

The agency also plans to release a series of clinical guidance documents related to different areas of active product development. These will cover; development of certain products, such as those for inherited blood disorders and neurodegenerative diseases, the circumstances where accelerated approval pathways will be applicable and the treatments where a more traditional clinical study would be necessary and how to achieve this.

Dr Gottlieb expanded on the clinical study guidance expected, commenting that this year the agency intend to outline a proposed innovative trial design through which individual researchers can pool their clinical data after following a common manufacturing protocol. This would allow for more robust data sets to be developed so that a Biologics License Application can be obtained.

Dr Gottlieb recognized that “cell-based and gene therapy technologies hold tremendous promise for addressing some of the most intractable disease”. However, he continued that “with their novelty, also comes new uncertainties and some unique, theoretical risks”. He asserts that their guidance is intended to help innovators proactively address these risks.

"With their novelty, also comes new uncertainties and some unique, theoretical risks"

However, he remains concerned of the individuals working in the space who are operating outside of regulatory compliance when developing products and in some cases are creating potentially significant safety concerns for patients.  The FDA confirm they will pursue enforcement actions in the coming year to address such product posing this level of risk and potential harm to patients.


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