Asembia AXS25 Summit: Can cell and gene therapies survive the tipping point?

An important question raised at Asembia 2025 was “Cell and gene therapies improve outcomes and even cure diseases – so why is commercial uptake slow?” Presenters Rocky Billups (HCA Healthcare), Joe DePinto (McKesson), and Dan Kirby (Iovance) provided valuable insights. They concluded that cell and gene therapies can offer significant hope to patients who have very limited treatment options, but these therapies are “at a tipping point”. Payers can adopt various strategies to help realize the full potential of these therapies.

Introduction to cell and gene therapies

Cell and gene therapies target conditions that are complex and difficult to treat.[i] The therapies are specifically tailored to the individual patient and their disease or condition. For the manufacturers – mainly Big Pharma and smaller privately-held companies – the development of such therapies is a lengthy and costly process. Cell and gene therapies are innovative medical approaches that use cells or genetic material to treat, prevent, or potentially cure diseases and genetic conditions such as sickle cell disease, hemophilia B, and certain types of cancer. While first wave cell therapies did not modify cells but relied on bone marrow transplants, the latest advancements are based on engineering immune cells. In the case of certain cancers, such as leukemia or lymphoma, for example, chimeric antigen receptor T-cell (CAR T-cell) therapies modify the genome of the patient’s own T-cells. The chimeric antigen receptors (CARs) on the modified T-cells recognize and bind to the specific antigens on the surface of cancer cells. This binding prompts the CAR T-cells to recognize and attack the cancer cells – thus eliminating them and putting the patient into remission.

Gene therapies introduce DNA or RNA into the patient’s cells to correct or reengineer processes that drive disease. Some treatments extract the patient’s stem cells and engineer them to overcome a specific condition before infusing back into the patient for an auto-reset.

Multiple approvals but also challenges

It’s fair to say that cell and gene therapies are perceived to be the ultimate in personalized medicine. However, despite their potential to cure or dramatically improve serious diseases, they face significant challenges around access, affordability, and implementation. At Asembia 2025, stakeholders across the specialty pharmacy, payer, and manufacturer landscape considered a central tension – how to unlock the promise of cell and gene therapies while navigating the complexities that come with them.

One of these complexities is that although there has been an uptick in approvals of cell and gene therapies, in real-world clinical practice uptake has been slow. The Food and Drug Administration approved nine cell and gene therapy products in 2024 and projects that from this year forward it will approve 10 to 20 cell and gene therapies a year.[i] Despite this healthy pipeline of existing approvals and more on the way, the slow uptake is affecting more than patient outcomes. It is directly affecting biopharma revenues and dampening investor confidence across the industry. Infrastructure limitations, payer hesitancy, and logistical challenges are creating bottlenecks, making it clear that approvals alone do not translate to impact.

Steps toward engaging payers

The transition of cell and gene therapies into community settings is essential for scalability, driven by expanding indications and maturing treatment protocols. But without payer confidence and streamlined delivery models, patient access will remain limited.

Innovative payment models

Innovative payment models such as value-based contracts mark a step away from the traditional fee-for-service model and focus more squarely on the quality of outcomes of care provided to patients. These contracts focus on the health outcomes of specific populations of patients. While value-based contracts are necessary, practical barriers – such as member turnover – patients leaving or transferring to other health plans –  and difficulty in long-term tracking – are slowing adoption. Discussions at Asembia explored outcomes-based milestones, payer risk-pooling, and new contracting approaches to close this gap.

Specialty pharmacy’s expanding role

At Asembia, the conclusion was that specialty pharmacy has a central role to play in payer strategies to deliver potentially life-changing cell and gene therapies. Specialty pharmacies can help to manage the high costs and complexities of specialty medications and can improve patient outcomes through comprehensive support and care coordination. Specialty pharmacists can also provide critical data and compliance support, aiding outcomes-based reimbursement. This can help health care payers to achieve their goals of cost containment, quality care, and improved patient health outcomes.

Specialty drugs are expensive. Managing the cost of these medications is crucial for health insurance companies, government programs, and other healthcare payers. Specialty pharmacies help to control these costs through various strategies and services. These include medication therapy management, patient education and support, adherence programs, prior authorization assistance, negotiating discounts with manufacturers, and implementing cost-effective dispensing practices. By providing personalized care and ensuring that patients use their medications correctly, specialty pharmacies can reduce waste, prevent complications, and improve health outcomes, ultimately leading to more efficient healthcare spending.

Moreover, specialty medications such as cell and gene therapies typically require special handling, storage, and administration. Specialty pharmacies are equipped to manage these complexities and can ensure they are stored and delivered properly.

Pressure from self-funded employers

While there is little doubt about the expertise of specialty pharmacy, the perceived high costs of cell and gene therapies has meant that self-funded employers who provide their employees with access to healthcare insurance are increasingly skeptical that they can afford to cover such costs. Consequently, biopharma manufacturers will likely need to offer:

• Stop-loss and subscription-based models to manage budget impact
• Competitive pricing structures
• Discounts as the market matures

Conclusion

Cell and gene therapies used to be a futuristic concept – that is no longer the case given the stream of approvals in the US and Europe. The structural and financial frictions that are causing slow uptake must be addressed otherwise the full potential of these therapies will remain out of reach for patients. The discussion at Asembia was a step toward building payer confidence, reimagining reimbursement, and aligning stakeholders around scalable, patient-centered solutions.

References

[i] European Consortium for Communicating Gene and Cell Therapy Information (EuroGCT). 2025. What conditions can currently be treated using gene and cell therapy? (Full table) | EuroGCT

[ii] Food and Drug Administration. 2025. Approved Cellular and Gene Therapy Products | FDA