The Biggest Challenges Facing Broader Adoption of Novel Cell and Gene Therapies

The report highlights a rise in cell and gene therapy research, with a significant number of first-in-class launches in 2023. What are the biggest challenges facing the broader adoption of these novel therapies, and what strategies can be implemented to overcome them?

So, the cell and gene therapy area suddenly get a lot of attention, a lot of investment and a lot of interest, and definitely shows tremendous potential for transforming the way in which many diseases can be treated. And we note in our report, over 600 trials were initiated last year in in the cell and gene therapy in space, we also note, there were eight launches of cell and gene therapies globally, including first six first in class launches in the US. So, there is an enormous amount of activity, and it is coming through the pipeline through registration, and potentially available to patients.

Now the issue is, in a sense, the delivery and of healthcare systems hasn't quite caught up with the pace of the clinical innovation, that that is coming through. So, we do have multiple bottlenecks in these treatments reaching the patients who would benefit from them. manufacturing and delivery are a big one, establishing the infrastructure to be able to deliver these therapies. You know, it's not a retail pharmacy, kind of a product. And the, you know, ensuring there are sufficient treatment centers that they have the resources that are required, the training and skills required, remains a bottleneck as well. reimbursement and payment for these therapies, also is slowing things down. You know, particularly if you look broadly across the major global markets, even the high-income countries, we still see a very uneven level of reimbursement and access in those markets. And then I think, finally, it's also important to recognize that the patient journey, if you will, for these sorts of treatments is typically long and complex. It's difficult for patients, and in many cases, these are patients with very severe disease, and where their ability to actually be able to receive the Car-T therapy or the gene therapy may be constrained simply because they're not well enough to receive those treatments. And I think that's something that we're all learning more about, certainly clinicians, you know, who have not been involved in this area for a long time, are still finding their way, I would say in terms of, you know, how to treat these patients.

I will also add newborn screening, which is a critical part of finding patients who will benefit from these treatments, especially babies, and you know, very, very, very young people. Newborn Screening is something that is not always widespread, or fully adopted. So, these all add up to bottlenecks that are preventing the value of the science from reaching the patients who will benefit from them. There is a lot of effort to raise awareness, to prioritize investments in these areas and to find ways to de bottleneck and as more of these therapies come into the marketplace and more companies are investing in these areas. You know, well, we definitely expect to see progress. But I think there's still a long way to go in this area. And, you know, again, the science is advancing more rapidly than the ability of the health system to actually deliver these therapeutics.