In this podcast, we spoke with Fran Gregory, Vice President of Emerging Therapies at Cardinal Health about the cell and gene therapy landscape, innovative solutions to reduce cost, the regulatory environment, and reimbursement.

Fran Gregory brings extensive experience in the biologic drug sector. As a pharmacist, she has worked across various areas, including payer PBM, pharmaceutical manufacturing, and now at Cardinal Health. Gregory oversees the advanced therapy solutions and biosimilars business units, which focus on cell and gene therapies and cost-saving biologics, respectively.

Cell and Gene Therapy Landscape

In the cell and gene therapy landscape, there are about 25 FDA-approved products in the U.S., split into 35% cell therapies and 65% gene therapies (at the time of the interview, now 38). This field has rapidly evolved since the approval of the first CAR-T cell therapies in 2017, and the FDA continues to support innovation, with a pipeline of around 1,500 products under development. The agency aims to approve over 100 products by 2030, potentially benefiting more than 100,000 patients. Therapeutic areas include oncology, hematology, neurology, diabetes, and even conditions affecting vision and hearing.

Gregory notes the unique challenges in this field, such as conducting clinical trials with small patient populations, complex manufacturing processes, and stringent cold chain logistics for distribution. The high cost of these therapies also poses a challenge, as some treatments can cost millions. However, opportunities abound as the healthcare system innovates to improve regulatory processes, distribution methods, and patient experiences.

Reducing Cost

She explains that the high cost of cell and gene therapies is due to the intensive research, development, and manufacturing requirements, particularly for treatments targeting rare diseases. Although the upfront cost is high, these therapies can offer long-term savings by reducing ongoing medical expenses for patients. New payment models, such as outcomes-based agreements, annuities, and warranties, are being developed to increase patient access and manage costs.

One innovative approach is the Cell and Gene Therapy Access Model, inspired by President Biden’s 2022 executive order on lowering prescription costs. This model enables CMS to negotiate with manufacturers on behalf of states, enhancing access for patients and encouraging manufacturer participation. The first products under this model, aimed at sickle cell disease, are expected to launch in early 2025. Gregory expresses optimism about the future of these therapies and their potential to drive further healthcare innovation.

Regulatory Environment

The regulatory environment for cell and gene therapies is evolving quickly as the FDA is committed to expediting the market availability of these products. The agency offers pathways like accelerated approval, where manufacturers can bring products to market based on indicative clinical outcomes and continue gathering real-world evidence post-approval. The FDA’s regenerative medicine advanced therapy (RMAT) designation also addresses the small patient populations in cell and gene therapy trials, focusing less on traditional statistical significance. Looking ahead, the FDA will increasingly emphasize outcome measures and closely monitor manufacturing processes to ensure safety and efficiency as technologies evolve.

Reimbursement

Organizations like the Institute for Clinical and Economic Review (ICER) will also play a significant role in evaluating both clinical and economic outcomes, influencing pricing and reimbursement discussions between manufacturers, governments, and insurers. As the landscape grows, these evaluations will guide not only regulatory decisions but also payment models, ensuring that gene therapies offer value and affordability.

Cardinal Health is deeply involved in the commercialization of cell and gene therapies, providing end-to-end support from regulatory consulting and market access preparation to logistics and patient services. The company assists manufacturers with FDA processes and market readiness, including payer and provider engagement well before a product’s launch. Cardinal Health’s expertise in distribution, particularly with cold storage and just-in-time delivery, ensures that products reach the right place at the right time. They also offer financial risk management and patient support services, including benefits verification and travel logistics, ensuring that patients can access and adhere to their treatments seamlessly.

Biosimilars

In the biosimilars space, there are currently over 50 FDA-approved biosimilars (at the time of the interview, now 60), with a pipeline promising even more competition in coming years. Biosimilars are expected to deliver significant healthcare savings, estimated to reach over $180 billion by 2027. Cardinal Health emphasizes that optimizing the adoption of biosimilars is essential for reallocating resources to fund new, innovative therapies. Uptake has been strong in oncology but slower in other areas like immunology, mainly due to provider education gaps and pharmacy benefit manager (PBM) formularies.

Cardinal Health works to influence biosimilar adoption through provider and patient education, streamlining processes, and addressing regulatory complexities unique to the U.S. market, such as the interchangeability designation. The organization emphasizes the importance of PBM placement of biosimilars on formularies and the removal of reference products to accelerate adoption. By simplifying processes and ensuring healthcare providers understand biosimilars, Cardinal Health aims to expand access, reduce costs, and create opportunities for more innovative treatments.

The biosimilar adoption is crucial for the healthcare ecosystem as it offers a rare opportunity to reduce costs, especially in biologics. By leveraging biosimilars, the healthcare system can reinvest savings into groundbreaking treatments like cell and gene therapies, ultimately improving patient access and outcomes while managing the economic burden.

In summary, optimizing biosimilar use and integrating innovative gene therapies are pivotal to the future of healthcare. Cardinal Health is positioned to facilitate both, ensuring the healthcare system remains sustainable and capable of accommodating new treatments as they emerge.