Government Innovation is Key to Creating Regulatory Convergence

Regulators worldwide have yet to truly feel the impact of emerging gene and cell therapies, speakers agreed during the kickoff session at last week’s ASGCT Policy Summit in Washington, D.C., and the field’s rapid growth creates the need for innovative ways to oversee drugs that don’t fit into traditional regulatory frameworks.

During the Policy Summit’s Nov. 4 session, Developments in Gene Therapy Policy Landscape, speakers emphasized the importance of balancing predictability with flexibility as they discussed ways to align regulatory frameworks for gene and cell therapies. Before 2018, no gene therapies were approved in multiple regions of the world. Now, the limited experience has led to challenges in meeting current regulatory requirements, resulting in different timelines, pathways, and documentations for regulation.

“When you’re doing something different, it’s hard to get lots of regulators to feel the same about it,” said Keith Wonnacott, Ph.D., a member of the ASGCT Regulatory Affairs Committee. “We want to get products to patients as soon as possible, but we need to know what regulators will say. We need to know that they’ll accept the approach we take.”

In Canada, a new regulatory pathway for advanced therapeutic products (ATPs) ensures that “patients are the center of the ecosystem,” said Liz Anne Gillham-Eisen, a director in the Health Products and Food Branch, Health Canada.

ATPs are drugs or devices that are so new and complex that current regulations aren’t equipped to handle them, a definition that could include gene and cell therapies. Because these products are being developed so quickly and they are continuously changing, there’s a lack of appropriate regulatory oversight that needs to be addressed, Gillham-Eisen said.

The new system that Health Canada is developing will help guide product regulation for innovative technological, scientific, or medical developments. Under this system, new products that Health Canada determines can’t be regulated in the existing framework under the Food and Drugs Act will move into a “regulatory sandbox,” Gillham-Eisen said. In that sandbox, regulatory partners will design rules for the product’s market access and address uncertainties before the product moves to the market with an individual license or an order of permission. The product would then be added to a specific product category (Schedule G) based on factors like risks, benefits, and the extent to which it differs from previously authorized products.

The requirements can always be adjusted and products can be removed from the market based on evidence from market access and ongoing consultations, Gillham-Eisen said. She said the flow of the sandbox will help regulators enter the process early and build trust among stakeholders by facilitating information sharing. Ultimately, this process will help patients get access to therapies sooner.

“We need to make sure the government is an enabler, not a barrier, to innovation,” Gillham-Eisen said.

Health Canada is currently examining options to pilot this new pathway and plans to develop a draft guidance document in 2020. They hope to get the pathway up and running in 2022 or 2023.

Creating these new flexible pathways may mean more work because developers need to show regulators that they have systems in place, Wonnacott said.

However, “we need to find ways to innovate, not just around the science, but around the process,” he added.  “Get ready for a wild ride.”

Watch ASGCT.org in the coming days for slide presentations from all Policy Summit sessions.

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