PDA Letter Article

Global Regulatory Convergence Required to Expand Access to ATMPs

by Rebecca Stauffer, PDA

Regulatory convergence will be key to ensuring access to life saving therapies across the globe, asserted Peter Marks, MD/PhD, Director, U.S. FDA CBER, during his presentation, “Moving Toward Global Regulatory Convergence for Advanced Therapy Medicinal Products (ATMPs).” Marks spoke in the final session of the PDA’s Advanced Therapy Medicinal Products webinar series, which wrapped up on June 30.

“We would like to deliver safe and effective cell and gene therapies to those in need globally,” he said. So many people are suffering from serious diseases that could be helped by cell and gene therapies, particularly gene therapies, that it would be nice to have those developed and distributed not just in the high-income counties but also in the low- and middle-income countries. We believe that global regulatory convergence in high income countries could help facilitate commercial availability and pave the way for the use of such therapies in low- and middle-income countries.”

Regulatory challenges for ATMPs include the need facilitating the manufacturing of safe ATMPs without being overly burdensome, achieving a balance between innovation and ensuring documentation of safety and efficacy and keeping pace with rapid advances in the science and technology of ATMPs. These challenges are common across the board globally and regulators recognize that the existing regulatory framework is not ideal for these products.

While these challenges affect high income countries, they are intensified within low- and middle-income countries, especially for gene therapies. Within these markets, manufacturing of gene therapies faces further challenges—production of viral vectors can be inefficient and costly, purification procedures for gene therapies are complicated and not well understood and concerted effort is required to produce and deliver gene therapies.

For more perspectives on the manufacturing challenges of individualized therapies, read a summary of the opening talks at the FDA March workshop, Facilitating End-to-End Development of Individualized Therapeutics.

Marks said that FDA recognizes the need for global collaboration for gene therapies as the science is rapidly evolving while manufacturing innovation lags. Due to the intricate worldwide supply chain, improperly manufactured product could have disastrous consequences not matter where it is manufactured or administered.

He outlined some of CBER’s regulatory convergence efforts. These include a proposed WHO white paper, regular meetings (virtual and otherwise) among high income countries’ regulatory bodies and encouraging sponsors to consider global development programs that allow FDA to invite other regulators to early stage meetings.

“We really think that by taking a global approach, we can hopefully have gene and cell therapies come to bare improvement for serious disease in a variety of different settings in high income counties but low and middle income countries as well,” Marks concluded.

About the Expert

Peter MarksPeter Marks, MD, PhD, joined the FDA in 2012 as Deputy Center Director for CBER and became Center Director in January 2016.