The path from novel science to a functional business model is rarely a straight one. Besides regulatory hurdles, there remain barriers to manufacturing cell and gene therapies on a commercial scale, as well as issues with costs and safety.
“What has changed the most for me is that we’ve gone from being this niche science to an industry — and we are having the growing pains of industry,” says Dr. Anthony Ting, who has more than two decades of industry experience in cell therapy and regulatory filing.
Nevertheless, growing numbers of cell and gene therapies are entering the market. As of April 2024, the U.S. Food and Drug Administration (FDA) has approved 37 cell and gene therapy products1 — with the majority for oncology treatments. And 2023 was a record year for cell and gene therapy FDA approvals, with seven treatments approved.2
However, there is a sense that more progress is required to fulfill the immense potential of such therapies and ensure that more treatments get to the market and patients.
Market considerations for cell and gene therapy development
Historically, cell and gene therapies have been driven by research rather than market considerations. For such therapies to benefit the highest number of patients, Dr. Ting suggests that researchers must think beyond the lab earlier in development and toward eventual business models.
“If I was talking to a young developer, I would say: ‘Think about the endgame first’,” he says. “What’s your patient population? How are you going to make enough products to treat that patient population? Is it going to be cost-effective?’”
“We’ve got great science, now we have to figure out the next step,” adds Dr. Ting. “How do we change the manufacturing process? How do we get the cost of goods down? How do we get the product to the patient? The majority of products are given in highly specialized academic centers. Not every patient who qualifies for these products is getting them.”
“It is an exciting time to be in the space,” says Dr. Ting. “But we’re a growing, maturing industry. We need to figure out how to do it better.”
Navigating regulatory issues with cell and gene therapy
Regulators have a vital role to play in cell and gene therapies entering the market. According to Dr. Ting, regulators are willing to engage with developers of such therapies on a level that would have been unlikely in previous generations.
“What I’ve enjoyed about being in the cell and gene therapy space is there has been really good communication and collaboration with regulators,” he says. “They are aware that this is a young, growing space. And they are very participatory. From the very beginning, when I had my first meetings with the FDA, it was it was not antagonistic — it was very collaborative.”
“They’ve got a lot more remit,” adds Dr. Ting. “They’re adding more personnel because there is a recognition that this is a very fast-growing space.”
Crucially, once a treatment is understood, regulatory approval can become faster for similar types of cell and gene therapies. New treatment options for other conditions are building on the foundation of oncology. The FDA approved the first gene therapies for sickle cell disease in December 2023.3 In addition, much hope is attached to the treatments in development for autoimmune diseases.
“The experience using CAR-Ts will be able to easily translate for the autoimmune space,” adds Dr. Ting. “But again, it’s a different patient population. And there are many different autoimmune diseases. Each patient population will have their unique challenges.”
Addressing cost issues with cell and gene therapies
A major barrier to building the cell and gene therapy market is the costs involved. Cell therapies can cost hundreds of thousands of dollars for patients, with some gene therapies running into the millions. The higher short-term costs have left national healthcare systems not always able to afford certain treatments. Consequently, reimbursement is a significant challenge.
To reach more patients in need, the mindset surrounding pricing must change. For example, the higher upfront costs of a one-time cell therapy could end up being a cheaper option over the longer term for patients than being on a course of regular treatments over potentially decades.
What could make a difference in costs is the success of CAR-T therapies, which is attracting investment. Additionally, the expansion of treatments available for different conditions could help to drive down market prices.
“Seeing the CAR-T space have success, in terms of getting to the market and patients, has spurred additional investment in cell and gene therapies,” explains Dr. Ting.
“Big Pharma also is getting more into the picture, which is nice,” he adds. “Because they have the resources to really drive the commercialization of a product.”
Why standardization is needed for the cell and gene therapy market
Within the next decade, there is expected to be a substantial rise in the number of patients treated with blood cancer from CD19-directed chimeric antigen receptor T cells (CD19 CAR-T). GlobalData analysis suggests there will be an increase from the 3,700 patients treated in 2021 to almost 13,500 patients by 2031.4
To meet market demand, upgrades are required for cell and gene therapy manufacturing. While standardization will be crucial to unlock the full potential of cell and gene therapies, recent examples of success in this area of biopharma suggest it is possible. Dr. Ting draws parallels with monoclonal antibodies, which share some similarities with cell and gene therapies in terms of production processes.
“Akin to the monoclonal antibody space, in the beginning, there’s lots of modalities and different ways that developers are generating monoclonal antibodies,” says Dr. Ting. “But they finally figured out standardization. Now there are two cell lines that pretty much everybody uses.”
How automation and data can accelerate cell and gene therapy manufacturing
With many production processes still using slower manual methods, greater uses of automation could provide further support for cell and gene therapy production. Not only can automation enable faster manufacturing with lower contamination risks, but digital technology can also process greater volumes of data far more quickly than manual input. Additional data can then be used to support regulatory approval.
“With automation, and then the incorporation of digitalization, you can accelerate the manufacturing processes,” says Dr. Ting. “When you’re looking at an electronic batch record versus a handwritten batch record — you can save considerable time and reduce errors.”
“The development of tools to collect data has been fantastic. That’s really the game-changer,” adds Dr. Ting. “We now have to figure out how we collect all this data. How do we put it in a format that we can develop machine learning algorithms and incorporate AI? But it’s coming.”
Technological innovations have already changed the cell and gene therapy market. This trend shows no sign of slowing down.
Overcoming challenges has been fundamental to the development of cell and gene therapies. Provided progress continues at the current rate, the cell and gene therapy market will continue to grow and ultimately enable more patients to benefit.
References:
1. Approved Cellular and Gene Therapy Products. FDA.com. 26 April 2024. https://www.fda.gov/vaccines-bloodbiologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products
2. Number of FDA Approvals Increased in 2023, With Notable Agents Including Cell and Gene Therapies.mmitnetwork.com. 1 February 2023.
https://www.mmitnetwork.com/aishealth/spotlight-on-market-access/number-of-fda-approvals-increased-in-2023-with-notable-agents-including-cell-and-gene-therapies/
3. FDA approves first gene therapies to treat patients with sickle cell disease. FDA.com. 8 December 2023.
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
4. GlobalData. Approved CD19 CAR-T agents to treat over 13,000 cancer patients annually by 2031, driving sales of Breyanzi and Yescarta, says GlobalData. GlobalData.com. 8 February 2023.
https://www.globaldata.com/media/pharma/approved-cd19-car-t-agents-treat-13000-cancer-patients-annually-2031-driving-sales-breyanzi-yescarta-says-globaldata/
