In this Pharmaceutical Executive video interview, Murray Aitken, Executive Director of the IQVIA Institute for Human Data Science, discusses the biggest challenges facing the broader adoption of novel cell and gene therapies from IQVIA's Global Trends in R&D 2024 report.
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.
Key Findings of the NIAGARA and HIMALAYA Trials
November 8th 2024In this episode of the Pharmaceutical Executive podcast, Shubh Goel, head of immuno-oncology, gastrointestinal tumors, US oncology business unit, AstraZeneca, discusses the findings of the NIAGARA trial in bladder cancer and the significance of the five-year overall survival data from the HIMALAYA trial, particularly the long-term efficacy of the STRIDE regimen for unresectable liver cancer.
Cell and Gene Therapy Check-in 2024
January 18th 2024Fran Gregory, VP of Emerging Therapies, Cardinal Health discusses her career, how both CAR-T therapies and personalization have been gaining momentum and what kind of progress we expect to see from them, some of the biggest hurdles facing their section of the industry, the importance of patient advocacy and so much more.
ROI and Rare Disease: Retooling the ‘Gene’ Value Machine
November 14th 2024Framework proposes three strategies designed to address the unique challenges of personalized and genetic therapies for rare diseases—and increase the probability of economic success for a new wave of potential curative treatments for these conditions.
Tirzepatide Demonstrates Significant Benefits for Patients with Pre-Diabetes, Obesity Over 176 Weeks
November 14th 2024Results from the Phase III SURMOUNT-1 study show that tirzepatide, a dual GIP and GLP-1 receptor agonist, achieved substantial average weight loss of 22.9% in patients with pre-diabetes and obesity.