Real-World Data Crucial to Prove the Value of Expensive Cell and Gene Therapies: Fran Gregory, PharmD

Although the majority of cell and gene therapies have been in oncology, there are exciting developments in the pipeline for areas that haven’t had these life-changing therapies in the past, said Fran Gregory, PharmD, MBA, vice president of emerging therapies at Cardinal Health. With more therapies coming to market, it’s important to have long-term patient data assessment to understand the value of these therapies in order to get coverage.

Transcript has been lightly edited; captions were auto-generated.

Transcript

What therapeutic areas have been impacted the most by developments in the cell and gene therapy space, and what areas do you think are most likely to see near-term FDA approvals?

Right now, we're seeing over 50% of cell and gene therapy approvals in the pipeline and on the market being in the oncology space. We truly do see that oncology/hematology space not only being strong today but continuing to be strong as we look into the pipeline.

However, there are a couple of areas that are really exciting that are developing in the pipeline that maybe have 6% to 10% of that pipeline. Those include rheumatology, immunology, neurology, and even blindness and deafness conditions are being researched in some gene and cell therapies. [Those are] some really exciting areas that we haven't had really novel, potentially life-changing treatments for in the past [and] are definitely alive and well in the pipeline. We're really excited to see what those bring.

How are real-world evidence and longer-term follow-up data shaping our understanding of patient outcomes in cell and gene therapies, and how might that affect payer coverage decisions?

This is one of the most critical topics that we are talking about now and really trying to highlight with our manufacturer and our payer partners is the importance of really, really good solid health economic studies. Not only the health economic studies that we have done for years—the budget impact models, cost-effectiveness analyses, things like that—but really looking at patient data over a longer term period and assessing truly what are these products worth from a patient quality of life and patient extension of life perspective, which is really a new way to think about things that is not as much seen in chronic condition treatments.

As more cell and gene therapies enter the market, how can managed care providers use real-world data to assess treatment durability and justify long-term investment in these high-cost therapies?

[It’s] really, really important to work with payers, work with manufacturers to ensure that that long-term data is being collected. If we think about proving the value of these products, not having the data from the patients or the providers collecting that data really limits the payer's ability to see that long-term viability of the product and limits their willingness to reimburse for it.

It's more important than ever right now that we really, really follow those patients closely and that we really are collecting outcomes data. Those patients may not want to be followed after they've been treated, and it's really important for us to find unique ways to engage with those patients, post their treatment and ensure that we're following those outcomes appropriately.