Cell & Gene Therapy

Sep 28, 2021 - Sep 29, 2021, Digital Conference, Exhibition & Networking

Join cell and gene leaders to define commercialization approaches and get solutions to patients faster.

Accelerating commercial success in CAGT

To build on the successes thus far in cell and gene therapy, pharma must foster further payer, physician and patient buy-in



Cell and gene therapies (CAGTs) are beginning to provide significant value across multiple therapy areas driven by a combination of their clinical benefit, durability and the overall response rate.
 
Despite this, CAGTs have yet to achieve the anticipated market penetration, with fewer than 20% of patients eligible for Chimeric Antigen Receptor T-cell (CART) therapy in the US receiving it, meaning there is still a significant way to go before all patients who could benefit from these therapies, do benefit.
 
The audience of the Reuters Events’ Cell & Gene Therapy Commercialization Conference, in November 2020 heard that there are a number of issues for pharma to address.
 
For one, healthcare infrastructure is not ready. The processes and expertise are not in place to provide patients with access to CAGT treatments in a timely manner, said Dean Griffiths, Principal, Consulting Services, IQVIA. The ongoing revolution in personalised medicine, of which CAGT is one part, requires a different approach to that of classical pharma.
 
CAGT developers will need to be able to clearly communicate these complex therapeutic modalities effectively to a cross-section of different stakeholders, added Oscar Segurado, Chief Medical Officer at ASC Therapeutics. “We should consider establishing a solid education framework for health care professionals (HCPs), payers and patients, beyond our usual marketing and sales channels, maybe even considering an industry-wide strategy, through partnerships.”
 
Manufacturer-led education is also critical to overcoming the challenge of ensuring that the right patients are identified in the community setting, helping to enable efficient referral to specialist treatment centres, said Chia Chen-Rogers, Senior Director for Oncology Cell Therapy New Products at GSK. Patients and referring providers should have a clear understanding of what the treatment can achieve and what is involved.
 
Despite substantial investment to overcome the well-known bottlenecks of CAGT manufacturing, reimbursement remains a key challenge and innovative approaches are needed, including outcomes-based payment models, which have shown the potential to assess the value of CAGT products differently, said Kelly Page, Head of Global Cell Therapy Commercialisation at Takeda. She also noted that there is some way to go and emphasised that payer concerns about the totality of costs remained an issue. 
 
2nd generation cancer cell therapies
The current challenges in cell and gene therapies are, in part, being overcome through scientific innovations. We are now seeing advances which include the use of CAGT therapies on solid tumours, said Chen-Rogers.
 
For example, some logic-gated cancer cell therapies can only be engaged in response to target cells expressing two antigens concurrently, thus increasing tumour recognition and limiting off-target effects. Innovations such as this can both increase efficacy and reduce adverse events and their associated costs. 
 
Currently marketed CAR-T therapies are indicated for patients in later lines of disease. Many patients are unable to wait long enough given the current turnaround time required for autologous therapies. Therefore, there is a significant opportunity for allogeneic treatments, given their off-the-shelf nature, to significantly expand the number of patients who could access cell therapies.  
 
Moreover, ongoing studies will validate whether it will be feasible to deliver allogeneic therapies in an outpatient setting. These therapies could dramatically reduce the cost of care, while increasing patients' quality of life. 
 
The overall consensus from an audience of more than 400 people who participated in the Reuters Events Cell and Gene Therapy USA 2020 event was that allogeneic cell therapies would become much more prominent in the next five years. CAR-T therapies are expected to remain the mainstream, with high anticipation for other cell types, such as allogenic Natural Killer (NK) cells. 
 
Partnerships
To develop safe and effective cell therapies, partnering is required across all stakeholder groups, not just research organizations. Collaboration with advocacy groups, community clinics and hospitals can provide valuable insight as we look to address the needs of patients and the overarching healthcare industry.
 
When partnering with HCPs and patient associations, it is important to look holistically at the entire patient journey and ensure that every person involved has the right training and that each point in the care pathway is effectively enabled, said Page.
 
“When developing new therapies, we need to think about those micro-moments that so greatly impact the patient’s experience, like getting to go home and put your kids to bed after a round of treatment versus an inpatient stay – from there, consider what kind of partnerships you need to make sure there is ultimately success in that area of care.” 
 
Engagement must also occur much earlier than normal to enable time to sufficiently educate patients and build the longstanding relationships required for monitoring the long-term impact of cell therapies, added Chen-Rogers.
 
This does not yet seem to be happening since only 30% of participants in the Reuters Events Cell and Gene Therapy USA 2020 audience were in exploratory discussions with provider and patient associations, with only 9% having more than 10 agreements in place, suggesting that a shift in current efforts to establish success is still required.
 
Partnering with regulators and payers is also required to ensure that assessment of value can be achieved. Companies should consider partnering with each other to develop a unified curriculum and clearly articulate the benefit of these therapies to patients, providers and payers alike. 
 
The future
To achieve future success in CAGT, companies need to be agile, moving away for the one-size-fits-all approach and instead developing segmented, or even individualised, approaches to meet the needs of different stakeholders, said Page. This will be particularly important with the introduction of allogeneic therapies, which will require a different depth of expertise across treatment centres. 
 
Realising the value of CAGTs will also require new approaches to clinical evidence generation. Well-designed clinical studies to demonstrate initial benefit will need to be coupled with long-term follow-up studies showing sustained benefit in the initial patients, but also incorporating broader cohorts once the medicines have been marketed.
 
Given that manufacturers will rely on HCPs to collect data, potentially on multiple different therapies, Chen-Rogers highlighted the importance of standardization to make this easier and more consistent. 
 
Despite these challenges, there is reason to remain enthusiastic about the future of cell and gene therapies. The potentially transformative clinical benefit for diseases which have potentially devastating consequences to patients and their families can be addressed in fundamentally more impactful ways with cell and gene therapies. 
 
“We have made great strides in using cells as medicine and harnessing what is inside us to help fight cancer," said Chen-Rogers. "Given the activity in this space, more breakthroughs are just a matter of time. The sky’s the limit.” 
 

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Cell & Gene Therapy

Sep 28, 2021 - Sep 29, 2021, Digital Conference, Exhibition & Networking

Join cell and gene leaders to define commercialization approaches and get solutions to patients faster.