The next chapter in cell and gene therapy

March 22, 2023
ATW 2023 celebrated a record-breaking year of approvals, with an eye on new realities

‘Gottlieb mathematics’ were recently trending in Miami.

Back in 2019, when Scott Gottlieb was heading up the Food and Drug Administration, he predicted that the agency would be approving 10-20 cell and gene therapies (CGT) per year by 2025. A lot of people had doubts.

When Kim Barnes, executive vice president for Phacilitate kicked off Advanced Therapies Week back in January, she commemorated 2022 as the year the commercial reality of CGT became apparent. In 2022, the U.S. saw a record-setting five CGT commercial approvals, including the first gene therapy authorized for hemophilia B.

The 2023 Advanced Therapies Week took place at the Miami Beach Convention Center from January 17-22 — and the vibe was both celebratory and future-focused.

It would seem that Gottlieb’s somewhat aspirational math is on track to add up — which is good news for both patients and those dedicated to the advanced therapy space. But the excitement of new approvals is tempered with the reality of costs.

The problem of cost

Chairing the plenary, Anthony Davies, founder and chief executive officer of Dark Horse Consulting cut right to the chase, pointing out that many CGTs are “outrageously expensive” and “it’s a problem.” “For the first time in history, we can make lifesaving therapies that we can’t afford,” said Davies.

When CSL Behring’s Hemgenix became the first FDA-approved gene therapy for hemophilia B last November, the drug also set another record — price. The one-time treatment comes with a $3.5 million price tag — making it the most expensive drug in the world. While the price can be justified by the therapy’s ability to decrease or even eliminate the need for regular injections of factor IX (patients with hemophilia B are typically given the protein once or twice a week to help blood to clot), the high upfront cost for payers is problematic.

With more gene therapies intended for conditions impacting larger patient groups such as diabetes and heart failure cascading down drug pipelines, something must be done to address costs because current payment models in the U.S. can’t support them.

“A great prescription if the patient can’t fill it is meaningless,” said Gwen Nichols, chief medical officer, Leukemia & Lymphoma Society.

Multipronged solutions

In order to realize “the most important next chapter in health care” it’s going to take a “multipronged effort,” from all stakeholders, said Steven Miller, chief clinical officer, Cigna Corporation.

First, the U.S. needs to put policies in place to stop wasting money, according to Miller. Perhaps one of the more shocking numbers shared during the event, Miller estimated that one-third of all health care spending in the U.S. is waste.

“There is enough money in the health care system to pay for cell and gene therapies, there’s no doubt about it,” said Miller. “If we can find that waste, get that waste out, that money can be reallocated for other things, like cell and gene therapies.”

Insurance companies, for their part, are establishing risk pools to attempt to combine medical costs to calculate premiums for specific high-value medicines.

But perhaps most vital is the need for drug manufacturers to get production costs down as low as possible.

Several panelists throughout the ATW event pointed out that this cost-reduction starts in development. In order to engineer product to optimize manufacturing costs, CGT developers need to worry about robustness and reproducibility from the beginning, said Peter Zandstra, professor, University of British Columbia. This includes integrating novel technologies into the process to further enhance control of cell product identity, reproducibility and efficacy.

In a separate session focused on commercialization, Lung-I Cheng, vice president, Cell & Gene Therapy, AmerisourceBergen emphasized that CGT manufacturers should address commercialization functions in R&D discussions, designing and building for scalability.

“To ensure success many commercialization decisions need to be addressed early in the journey during the clinical stage,” said Cheng.

Decisions should also be made with patients’ best interests mind, stressed Cheng, advocating for a patient-centric approach to the treatment journey that is already very complex for patients.

By the end of Advanced Therapies Week 2023, it was clear that the industry has a lot of celebrate — despite all past and current obstacles, the age of advanced therapies has arrived.

About the Author

Karen P. Langhauser | Chief Content Director, Pharma Manufacturing

Karen currently serves as Pharma Manufacturing's chief content director.

Now having dedicated her entire career to b2b journalism, Karen got her start writing for Food Manufacturing magazine. She made the decision to trade food for drugs in 2013, when she joined Putman Media as the digital content manager for Pharma Manufacturing, later taking the helm on the brand in 2016.

As an award-winning journalist with 20+ years experience writing in the manufacturing space, Karen passionately believes that b2b content does not have to suck. As the content director, her ongoing mission has been to keep Pharma Manufacturing's editorial look, tone and content fresh and accessible.

Karen graduated with honors from Bucknell University, where she majored in English and played Division 1 softball for the Bison. Happily living in NJ's famed Asbury Park, Karen is a retired Garden State Rollergirl, known to the roller derby community as the 'Predator-in-Chief.'