FDA eyes collaborative review pilot for gene therapies

The US Food and Drug Administration (FDA) is working on a pilot program that will explore the possibility of concurrent collaborative review of new gene therapy applications with other global regulators. The pilot was detailed by Peter Marks, director of FDA’s Center for Biologics Evaluation and Research (CBER) at a meeting this week held by the Alliance for Regenerative Medicines (ARM) in San Francisco.

Marks said that the idea of a gene therapy collaboration “finally has some traction,” which he said was “probably helped by the pandemic.” The Collaboration on Gene Therapies Global Pilot (CoGenT) will include initially include regulatory members of the International Council for Harmonisation (ICH), including the EU, the US, Japan, Canada, and Switzerland. An FDA spokesperson told Focus the pilot is “still in the early stages of development.”

The goal of the pilot will be for partners to participate in internal regulatory meetings with sponsors of new gene therapy applications. The reviews would be shared with regulatory partners. The meetings will be conducted, and information will be shared, under strict confidentiality agreements, Marks said. The goal is to increase the efficiency of the regulatory reviews, reducing the time and the cost for agencies and sponsors to bring these therapies to the market.

Marks said one of the lessons learned from the pandemic is that “global regulators realize that what we do is more common than divergent. What we do at FDA has a lot in common” with the European Medicines Agency.” He added that “there are some subtle differences, we do some things that they don’t do and vice versa, but if we can harmonize our requirements and pull forces to review these products, we can make it much more attractive for people to go into this rare disease area.”

Marks added that “this unfortunate divergence in this field of rare diseases where it affects so few individuals this is something that we have to do something about because we owe it to our patients globally.”

Marks further noted that “certain rare diseases may only have a population of 50 patients in the US and the EMA has 75 patients and Japan has a number of patients. The 50 patients in the US do not tip the net balance calculations into the positive. If you aggregate everyone, you have a much higher chance of tipping that calculation to where it actually makes sense” to incentivize sponsors to invest in these treatments.

Marks has been promoting the idea of a collaborative review program for gene therapies since last year, similar to the reviews conducted under Project Orbis in the oncology space. (RELATED: Top FDA official interested in ‘Project Orbis’ for cell and gene therapies, Regulatory Focus 13 February 2023)

FDA eyes collaborative review pilot for gene therapies

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