Gene therapy of today, payment models of last century

New gene therapies will be here soon—but payment models are stuck in the 20th century, said experts at the BIO International Convention. Plus, the onus is on innovators to educate regulators about their science—a few companies explain why. (584 words, 2 minutes, 55 seconds)

The gene therapy of today, the payment models of last century

The Food and Drug Administration (FDA) recently approved the first two cell-based gene therapies for sickle cell disease, developed by Vertex Pharmaceuticals and bluebird bio—and more one-time curative therapies could be here soon. But payment models are stuck in the 20th century, said experts at the 2024 BIO International Convention.

A new breakthrough: After announcing in January that gene therapy restored hearing in one ear in children with hereditary deafness, researchers reported last week that it can restore hearing in both ears. Altering one gene reversed DFNB9 congenital deafness with a new treatment that could be available in 3-5 years.

The challenge now: “We’re trying to administer 21st-century medicines within a 20th-century payment model,” said Jay Newman of Spark Therapeutics, Inc., who spoke during one of several panels on gene therapy at the 2024 BIO International Convention.

“One-time curative therapies are different than what has been historically approved,”said Tom Klima, Chief Operating and Commercial Officer at bluebird bio, in an interview with Good Day BIO Live. “So, when you think about a patient who goes in every 2-5 weeks for red blood cell transfusions, and suddenly, they don’t have to go back to the hospital—completely transformative.”

“As you think about value with a one-time curative therapy like gene therapy, you have to think about the holistic costs,” said Klima at the event. “And when you think about price, you really have to tie that to thinking about an outcomes-based agreement.” 

What’s next: “There’s a lot of appetite at the state for some sort of alternative payment arrangement,” perhaps based on value or outcomes, said Stuart Portman, Executive Director of Georgia’s Division of Medical Assistance Plans. “But how you handle all those different situations requires changes to the rules of the road, if you will.” 

Read more at Bio.News.

Watch our interview with bluebird bio’s Tom Klima:

Why innovators need to educate regulators

Innovators in agriculture, energy, and climate biotech must help regulators keep up with rapid scientific development, said a panel of companies at the 2024 BIO International Convention.

The challenge: “For biotechnology products like those that are represented up here on the dais, more than one agency has jurisdiction over a single product at different points in the value chain,” said Tad Sonstegard, Ph.D., BIO Board Member and CEO of Acceligen, which is developing animals resistant to heat and disease. This can lead to delays that impact the return on investment.

A solution: “You need to interact with the regulators, and you need to interact with the legislators,” said Cameron Bishop of J.R. Simplot Company, a pioneer in genetically engineered potatoes. “Even if you don’t think you have anything to say, go to D.C. and educate them on what you do.”

But it’s not just regulators who need convincing—you also have the public and detractors, who are often influenced by “science fiction and not science,” added Bishop.

The good news: The general public is pro-innovation regarding sustainable aviation fuel (SAF), said Emily Carlton, SAF Policy Specialist at LanzaJet.

The bottom line: “The onus may actually be on us,” said Sonstegard, “to communicate to our policymakers the need for updated regulatory and policy paradigms.” 

Read more at Bio.News.

Watch our interview with LanzaJet’s Emily Carlton:

Catch up on BIO 2024 with Good Day BIO Live 

• Keep an eye on Bio.News for panel recaps, exclusive Q&As, and much more.
• Join the conversation on X and LinkedIn, with hashtag #BIO2024. Watch for more exclusive video content and repost-worthy quotes.
• Watch your inbox for more right here each weekday at 10 AM ET/7 AM PT.

President Biden’s Wednesday: Heading to the G7 Summit in Italy, where he’s expected to discuss sanctions on Russia, per Reuters.

What’s Happening on Capitol Hill: The House Judiciary Committee has scheduled a hearing, “The U.S. Intellectual Property System and the Impact of Litigation Financed by Third-Party Investors and Foreign Entities.”