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AEI Panel Discussion: Navigating Emerging Technologies in Cell and Gene Therapy

On February 14, IGT Chairman Erik Paulsen joined a panel of experts at the American Enterprise Institute’s (AEI) event, “Navigating Emerging Technologies in Cell and Gene Therapy,” which focused on the regulatory and payment challenges that exist as more gene therapies are approved and expect to be approved in the coming years.


Moderator, Brian J. Miller of AEI, and panelists highlighted the unique value of gene therapies and the need for flexibility in our payment system.


“Think about if you can cure sickle cell, what it does for a patient’s life, their family, and the budget,” said Congressman Brett Guthrie (R-KY).


“Gene therapies are giving patients with rare disorders hope - it’s an exciting time. We need to make sure there’s a path forward for access so it’s critical to get the value equation right and do so with the flexibility that these rare and ultra-rare conditions require,” added IGT Chairman Paulsen.


“When we talk about cost of these therapies, we’re really changing lives of children, their families, and their communities. There are economic benefits because they will be productive citizens, there will be less (long-term) costs, parents continue to be employed - there are huge benefits,” explained Sheila Mikhail, Cofounder of Asklepios Bio Pharmaceutical.


The panel also discussed the challenges related to the manufacturing of gene therapies – which requires significant time and resources – and the criticisms and misconceptions surrounding the accelerated approval pathway.


“I view accelerated approval as critical for helping patients in a timely manner. To ignore the ability to get something to a child one or two years sooner will make the difference of whether they can function in society or if society needs to care for them. Accelerated approval allows us to do what we do best at FDA to put together an approval which by and large are confirmed subsequently. It’s an extremely good pathway,” said Dr. Peter Marks, Director of FDA’s Center for Biologics Evaluation and Research (CBER).


Watch the full discussion here:


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