Expert: Cell, Gene Therapies Provide the Important Opportunity to ‘Move Beyond Chronic Disease Management to Curative Areas’

Pharmacy Times interviewed Aklilu Tedla, vice president of strategy and business development at Cardinal Health, to discuss the future of pharmacies as more cell and gene therapy products become available to patients.

Question: What are some recent advancements in the cell and gene therapy space, and what has been the impact in the field?

Aklilu Tedla: Yeah, so today there are about 10 products in the marketplace, they span from medications that treat oncology, various neuromuscular types of issues to eye.

Just to give you a sense of the impact, for example, Spark Therapeutics’ [voretigene neparvovec-rzyl (Luxturna)] has had cases where people's visions have been restored. So just think about that from a patient, from a caregiver, or from a provider perspective—that's unprecedented.

In some ways, that's the promise of cell and gene therapies—moving us beyond chronic disease management into curative areas. Now, we're still early—a lot of work to be done. But these 10 are laying the groundwork for a rich pipeline of products that's going to lead to a very robust market down the road.

Question: How has the use of cell and gene therapies evolved over time?

Aklilu Tedla: Yeah, that's a good question; I can answer that in 3 ways. So if you look at it from a therapeutic area.

So normally, or historically, targeted towards a narrow set of population, or an orphan kind of designation, to therapeutic areas where you have thousands of patients, so going from just a few patients, or a narrow population of patients that can get it to patients that have like stroke—stroke effects thousands of people. There are products in the pipeline that are going to target a broader population. So that's one from a therapeutic area, you see expansion.

Second, when you say cell therapy, for example, you can think of it in 2 modalities. There are the targets—autologous is out of my body and back into my body, that's naturally just one in one. There's allogeneic, which is out of my body into many. Now, this innovation is actually making this therapy or therapies available to a broader set. If you go back to the hope of more curative outcomes, you're now making this outcome available to more.

Finally, while gene therapy has had a number of rocky—I wouldn't say rocky—but a slow start, from 2022 onward, we see a massive acceleration of these therapies, which again, will bring a lot of improved outcomes for patients.

Question: How has the evolution of the cell and gene therapy market increased patient access to these treatments?

Aklilu Tedla: So today, I would say it's fair to say that patients who need these gene therapies have access to them. They’re a narrow set of population of patients, and so they're getting access to them from a just a payer perspective, for example.

But as this market grows, so we think this market could be close to $100 billion in the next 7 years, you're going to have to manage 2 things—outcomes, that's the hope of selling gene therapies, but then cost. So how do you manage costs? Well, you want to make sure that you optimize safety, these types of new therapies will require more safety monitoring, more long-term monitoring. So if you look at it from a patient perspective, what's going to get you comfortable to try these novel therapies? If you think about it from a payer perspective, what's going to get you to pay for this therapy—it's the promise of an outcome that you'll pay for.

So I think there are a lot of things that need to happen in order to increase access, but it starts with the efficacy and then the ability to kind of monitor and ensure safety over the course of 10 to 15 years is what we think that the monitoring aspect of cell and gene therapies is going to be needed.