FDA's Marks Urges International Collaboration at ASGCT Policy Summit

In order to move toward regulatory convergence between countries developing gene therapies, international collaboration among high income countries is critical to help pave the way for increased patient access around the world, said Peter Marks, M.D., Ph.D., Director of the Center for Biologics Evaluation and Research (FDA CBER), during his keynote address at the ASGCT Policy Summit in Washington, D.C.

“Safe and effective gene therapies should be a global priority,” Marks said. The Summit, held Nov. 4-6 at the Westin City Center, brings together regulatory and industry staff, scientists, patient advocates and more to discuss issues related to the development and responsible use of gene therapy.

Now is the time, Marks said, for regulators to work towards a globally harmonized framework that will facilitate the safe and ethical development of transformative gene therapy products. By 2025, the FDA expects to approve 10 to 20 gene and cell therapy products per year. By 2030, more than 500,000 people could be treated with gene therapy products.  

“For three decades, we’ve been on the cusp on gene therapies, and we’re here now,” he said.

While this is an exciting time in the field, Marks noted that the development and delivery of gene therapies could have a “chilling effect” if not properly executed. These treatments must be efficient and cost effective, Marks said, because many conditions treated with gene therapies have small populations. Additionally, when requirements are not aligned, barriers exist that make patients access more difficult. 

To remedy this, Marks called for more active harmonization of regulatory approaches in high income countries. He said marketing in high income countries could help pave the way for accessibility in low and middle income countries, where patients may benefit most because many lack access to care.

In the U.S. and Europe, many countries have already been collaborating to make new drug development more efficient. The FDA has been encouraging sponsors to invite regulatory partners like the European Medicines Agency (EMA) and Health Canada to preliminary consultation meetings before the more formal discussions that are necessary to bring a new drug to market. During these meetings, sponsors can get regulatory advice as they prepare preclinical studies and identify issues or deficiencies they need to address as they develop their products. 
 
Marks said putting regulations in place in African and Asian countries in the coming years is essential. Those countries will need educational processes in place so people can become knowledgeable about drug regulation. The longer we wait, Marks said, the harder it will be to get treatments to patients. The FDA is currently working on a white paper on the regulatory framework for gene and cell therapies in low and middle income countries. 

Other priorities should include collaborating on gene and cell therapy production and manufacturing methods, and developing public-private partnerships to facilitate gene therapies for ultra rare disorders Marks said.

Marks said areas for convergence include preclinical requirements, clinical outcomes, manufacturing information, and environmental assessments. However, these areas will require a dialogue between international stakeholders before an agreement is reached. 

ASGCT continues to advance these conversations throughout the remainder of the Policy Summit during payment policy discussions on Nov. 5 and a frank discussion on the ethical, societal, and policy issues surrounding germline gene editing on Nov. 6.

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