Addressing the Barriers to Equitable Access in Cell and Gene Therapies
Joe DePinto, MBA, discussed the findings of the McKesson 2024 Cell and Gene Therapy Report, providing insight into the challenges and opportunities in equitable access to these treatments.
Joe DePinto, MBA
Cell and gene therapies offer a new and novel advancement in medical science, delivering clinical outcomes that are continuously exceeding benchmarks. However, equitable access to these therapies remains a significant challenge, particularly for underserved populations.
A major barrier is the concentration of treatment facilities in academic centers, as they are often located in large metropolitan areas. According to Joe DePinto, MBA, many patients must travel over 50 miles to receive care, which can be both a logistical and financial burden for socioeconomically disadvantaged groups.
“Cell and gene therapy is such an exciting space. The science has outperformed the benchmarks in every area that cell and gene therapy has explored. The commercial success has not caught up at this point, because it is still very much a nascent space, and much of the delivery of cell and gene therapy is done at academic centers and hospitals,” DePinto, head of cell, gene, and advanced therapies at McKesson Pharmaceutical Solutions and Services, told Targeted OncologyTM, in an interview.
In the interview, DePinto discussed the findings of the McKesson 2024 Cell and Gene Therapy Report, providing insight into the challenges and opportunities in equitable access to these treatments.
DNA Molecule: © Design Cells - stock.adobe.com
Targeted Oncology: What are some of the key challenges to equitable access to cell and gene therapies, especially in underserved areas?
DePinto: Cell and gene therapy is such an exciting space. The science has outperformed the benchmarks in every area that cell and gene therapy has explored. The commercial success has not caught up at this point, because it is still very much a nascent space, and much of the delivery of cell and gene therapy is done at academic centers and hospitals. By the fact that this is given in these academic centers and it is given in hospital settings, there tends to be some treatment area gaps.
In October, we published the McKesson 2024 Cell and Gene Therapy Report, and we mapped out in the US all of the sites that have given cell and gene therapies. Then we broke it out by cell and by gene therapies, and you can see there's delivery of care deserts that exist, and most of the deliveries in the large metropolitan areas. There are a lot of total available patients that may have access to these products that have to travel greater than 50 miles, and that is a challenge for some socioeconomic areas to do that travel, and to be able to travel and stay for an extended period of time with a caregiver, and that reduces, obviously, the ability for those therapies to be given to that patient population.
Why do oncologists view cell and gene therapies as such a major innovation? How do you see them changing cancer treatment in the next 5 to 10 years?
In the same report, we surveyed 124 hematologists/oncologists in the US, and 99% of them felt like cell and gene therapy is one of the most innovative interventions of their time. Ninety-four percent of them felt that these therapies had a significant impact on the quality of life of these patients. They focused initially on later-stage patients, like 4 plus lines, which is typical in the drug development of oncology type products. We are seeing that same playbook evolve for cell therapies, drugs that have started in the later stage starting to move up into earlier lines of therapy, line 3, line 2, and that total available patient pool grows when you go from a later stage line 4 plus after multiple failures to an earlier stage, so that has been powerful. When the immune system is more intact and you are giving these treated cells, it makes sense to me that clinically, that those outcomes would be strong, and that is where we are seeing the clinical research in the space in oncology.
What are some of the most promising cell and gene therapies in oncology? What upcoming clinical data should oncologists watch out for?
We have had multiple approvals this year, and in the report, which we have gathered from the FDA website, you will see an increasing number of products moving into earlier phases. We are also starting to see some TILs approved for solid tumors, which is exciting. Initially, early movers in this space focused on hematologic malignancies and non-malignant hematology, but now TILs are being approved for solid tumors. This is particularly exciting because [patients with] solid tumors represent a larger pool, often using chemotherapy, targeted therapies, and other treatments. If we can use cell therapies to treat these tumors, it would be a significant advancement. This is an area of focus in research as we analyze the pipelines.
What are some of the main barriers preventing oncologists from offering cell and gene therapies to patients, and how can that be addressed?
We have addressed the site of care previously, where these therapies are typically given in academic settings. Another aspect to consider is the delivery of cell and gene therapies in oncology, specifically the value chain. The delivery of these products is not the same across the board; it is a heterogeneous population of products that are delivered, and navigating the value chain can often be circuitous rather than linear, as with most products. [For example], in autologous cell therapy, the patient is part of the product. The process starts with manufacturing, then moves to a CDMO or manufacturing site where it is manipulated using viral or non-viral vectors. It is stored, then sent back under temperature control. Navigating this value chain is more complex. We seek solutions to help our manufacturing clients and health systems navigate this complexity with services that make the process more transparent.
How do insurance coverage or out of pocket costs impact patient access to cell and gene therapies?
In the report, that was clearly identified as one of the challenges by those surveyed. It is interesting because the US healthcare system is not quite set up for how cell and gene therapies are delivered from a cost. They tend to have this bit of a cost density issue. What I mean by that is most cell and gene therapies are given at 1 time, and the benefit is seen over an extended period of time. The payer system in the US really is not set up to absorb that, because they look at 1 year at a time, and these costs tend to be a bit more costly than the available products on the market. The outcomes are quite strong. So, what was identified was the fact that the systems do not align, and the copay components could be a challenge for patients.
However, they also felt like the solve for that could be innovative payment models, whether that be a value-based or outcomes-based, an annuity type payment warranties, a variety of innovative payment models, those respondents that were surveyed felt could be a nice solve, That would entail having the manufacturers, the payers and the providers, all working together to deliver this value, because the reality is, like with any product, we want to make sure the right product gets to the right patient so they can get the full value of that product, and innovative payment models may help facilitate that moving forward.
For those who might be unaware of cell and gene therapies, how can awareness be increased?
The ability to have a high degree of awareness was not coming through when oncologists were surveyed.Actually, 47% of the oncologists say patients rarely or never are aware that cell and gene therapies are available. So, patient awareness is not there. This is a nascent space, still. It is still early days for cell and gene therapy. We only have 30 plus approvals in the space. Not all are in oncology, not all are cells, not all our genes.
We also had some challenges early on around manufacturing. A lot of the manufacturing was a bit of a challenge of whether slots would be available. There was not a full out blitz of information around this to patients. But as the space continues to evolve, as manufacturing processes and operational processes improve, we are starting to see more companies enter the space for the first time and do a lot of defining of the market and setting the market up, as well as their products. That will all improve, especially as the space grows and moves through the process. We are seeing a tremendous amount of excitement around the space because of the clinical outcomes, and we are seeing excitement about the patient outcomes clinically. I think that will lead to more awareness of the space of the clinical data we got [and are going to get from meetings like] the American Society of Hematology Annual Meeting and Exposition, which typically leads to approvals.
What major milestones do you expect in this landscape to come out in sometime soon? How should oncologists prepare for broader adoption?
There are several milestones in this field that I find particularly exciting, both in oncology and beyond. One key development is the migration of cell and gene therapy from academic centers to community hospitals and clinics across the United States. This shift is driven by the growing demand for treatment. Currently, academic centers don’t have the capacity to handle both clinical trials and the increasing number of commercial patients. Expanding into community settings will require the establishment of more authorized and qualified treatment centers, which is a critical step forward.
Another milestone is the progression of research from late-stage oncology into earlier stages of the disease. Beyond oncology, we are also seeing promising data in areas like autoimmune diseases and neurological disorders, which represent much larger patient populations. For example, lupus is a significant patient population, and we are starting to see more mature data in this area.
The growing focus on non-oncology applications highlights the need to industrialize and standardize the delivery of these therapies. While rare diseases and late-stage precision treatments can operate with smaller, customized processes, treating large populations like those with lupus and autoimmune diseases requires a more streamlined and scalable approach. This includes standardizing everything from ordering to revenue recognition across the value chain. We are passionate about these developments because they allow us to help more patients access these transformative therapies. This expansion and standardization are critical to ensuring that these treatments reach all who need them.
Overall, what are the key findings from the report?
The key themes of the report highlight the strong potential for improved outcomes with cell and gene therapy. However, realizing this potential will require addressing the growing pipeline's need for more capacity and an institutionalized or industrialized approach to delivering these therapies. To achieve scale, care must also move into community settings. The report emphasizes optimism, access, and awareness as critical factors for advancing these groundbreaking treatments.
What stands out about the report is its dual focus: identifying barriers with real data from primary market research, supported by global and FDA data, and presenting potential solutions. Subject matter experts have contributed insights on overcoming these challenges, making the report solutions-oriented. We aim to address barriers in the value chain, including patient access, distribution, and logistics, so that more patients can benefit from these transformative therapies. This is the outcome we envisioned and the goal we set out to achieve with our contributions to the report.
