How to Bring World’s Most Innovative Treatments to Least Wealthy Places

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Cell and gene therapy (CGT) is considered to be an innovative treatment for a number of diseases; however, production costs are high, meaning that most low- and middle-income countries will struggle to afford them.

At the Financial Times’ Global Pharma and Biotech Summit 2024, a panel consisting of Nabiha Saklayen, CEO, co-founder, Cellino; Helen Tayton-Martin, chief business and strategy officer, Adaptimmune; Savita Rangarajan, professor, clinical hematology, human development and health, University of Southampton; and Joshi Venugopal, general manager, head of Europe, Novartis gene therapies and rare diseases, discussed ways in which biopharma companies are already engaging to support access to innovative treatments. They also addressed how manufacturing capabilities and commercial models for cell and gene therapies could evolve by the end of the decade; how health care systems in developing countries can evolve to support access; and what it will take to bring innovative products, such as cell and gene therapies, to those who can’t afford them.

Kicking things off, panel moderator Andrew Dunn, senior biopharma correspondent, Endpoints, asked Tayton-Martin to speak about therapies that Adaptimmune is currently working on and her thoughts on navigation.

“It’s been 16 years in the making to get our first product approval in the summer,” said Tayton-Marton. “So, we have a T-cell therapy, and we engineered the T-cell receptors from science out of Oxford University. We put we put them back into a patient’s own T-cells. Now, we’re a NASDAQ-listed, fully transatlantic company. Five-hundred people are in manufacturing facilities. We have to use a viral vector to put the T-cells into a patient. Lastly, we need a companion diagnostic for the target that the receptor sees. It’s complicated, but it works.”

Dunn then directed the conversation toward Rangarajan, asking her discuss her work and the biggest challenges that she sees today.

“I’m a simple clinician, and I’m a hematologist by background,” Rangarajan said. “I got into the cell and gene therapy space with hemophilia gene therapies, and I was involved with the gene therapy trials in the UK. Clearly, it’s fascinating to see that you can actually change people's lives completely or normalize them with a gene therapy.

“The current cost is not going to be easily affordable, even in the NHS for current products. Going to the other countries, particularly [low- and middle-income countries], I’ll talk specifically about India, as I work there. I have a large clinical trials unit there and I'm working with an indigenous company to deliver gene therapy because one of the goals of our current prime minister is making India a bigger space for technology.”

Moving forward, Saklayen discussed high science approaches, lowering the costs of breakthrough treatments, and what her team is currently working on.

“I invented new optical technologies to engineer cells with high precision,” said Saklayen. “The last few years of my PhD, I transitioned very quickly from working in a clean room semiconductor fab to working with cells collaborating with biologists, and I had some terrific early collaborators who all happened to be very entrepreneurial, believing that my technology could solve a number of problems. After launching my startup, the first thing I focused on was what problem we were going to solve. The way I saw it, I felt that I needed to solve a problem that will have commercial impact.”

The problem that Saklayen identified was how the cells could be manufactured while still maintaining quality, which she said came back to current manufacturing practices.

“Depending on the tissue type, the process can be several months in duration, where highly skilled experts are going into clean room, looking at and picking cells. So, we came in and started small with automating,” she said. “We then said ‘Ok, let’s automate imagery, because that’s something that scientists are doing.’ So, we had this proprietary way to remove unwanted cells in the culture. It's a laser bubble generation technique that's inspired by my PhD work but it's very precise and removing cells. So we applied it to remove passaging, to remove picking of colonies, to remove spontaneously differentiating cells, and that became the closed loop of using imaging training algorithms and then doing this laser process.”

Among a number of topics, Dunn turned to Venugopal to discuss his experience with launching CGTs and dealing with conversations around prices.

“When we introduced them as one-time therapies, it was a completely different model. Historically, payers are used to reimburse chronic treatments with deferred treatment costs and now we are talking about one-time therapy,” Dunn said. “The data was limited because it's a rare disease and the costs have to be given up front. So, the question was, ‘How can we de-risk the value proposition for both the company and for the payer?’

“I would say that in the majority of the markets, we have some innovative pay for performance agreements. Payments are not taken care of at the beginning, but they’re split over time as installments, and then in some cases, rewards for patient hitting a milestone and sometimes penalties if the patients are not hitting the milestone so that way the payers are spreading their cost over time.”