FDA Takes First Step Toward International Regulation of Gene Therapies to Treat Rare Diseases

Rare Daily Staff

In January, the FDA announced the launch of its Collaboration on Gene Therapies Global Pilot, an effort to harmonize regulation for cell and gene therapies across authorities in various countries to shorten review time, cut costs, and improve the commercial viability of these therapies.

The effort, known as CoGenT Global draws on the success of the Oncology Center for Excellence’s Project Orbis, which was established in 2019 and provides for the concurrent submission and review of oncology products among international partners, including Australia, Brazil, Canada, Israel, Singapore, Switzerland, and the United Kingdom.

Writing in The National Law Review, Chia-Feng Lu and Caroline Abbott of Greenberg Traurig, note that there are more than 900 ongoing clinical trials of cell and gene therapies in North America and that by 2025, the FDA expects to approve 10 to 20 cell and gene therapy products each year. With 34 FDA-approved cell and gene therapies and many more on the way, they say it has raised several policy questions that FDA expects to address.

Among those are the challenge of how to address the large number of ultra-rare diseases that afflict small patient populations and pose economic challenges for therapeutic developers to pursue treatments and potential cures. “These scale issues are exacerbated by disjointed global regulatory schemes that lack uniformity or harmonization,” the authors write. “This regulatory misalignment, in turn, increases review timetables and costs, which further deters investment in and development of gene therapies for rare diseases.”

They said the CoGenT Global initiative aims to increase investment in and development of such therapies and promote the development and accessibility of gene therapies globally by leveraging the global patient population and streamlining the review process by coordinating review efforts and reducing redundant submissions and regulatory reviews.

Though the agency hasn’t announced any specifics about how companies can participate in the pilot, the authors expect the agency to provide greater detail about the initiative in the next few months.

You can read the full article here.