Cell and gene therapies (CGTs) are reshaping the health care landscape, offering hope for patients with rare, complex and previously untreatable conditions.  

In the latest “Pharmacy Friends” episode, “Reimagining the pipeline: Cell & gene therapy decisions that shape care,” Prime Therapeutics (Prime) experts Nicole Kjesbo, PharmD, BCPS, senior clinical program development director, and Landon Marshall, PharmD, Ph.D., principal health outcomes researcher, alongside host Maryam Tabatabai, PharmD, associate vice president of clinical information, explore the potential clinical, regulatory, financial and human impact of these next-generation treatments.  

Here are some of the key takeaways from their discussion.  

The science behind CGT innovation 

So what exactly are CGTs?  

CGTs represent a revolutionary class of treatments that go beyond managing symptoms to target some of the root causes of diseases, including a person’s unique cellular structures and DNA. Specifically, gene therapy is a treatment that fixes or replaces a faulty gene, whereas cell therapy involves transplanting healthy cells to repair damaged tissue. These can be one-time treatments with potentially long-lasting effects.  

Because they’re such new areas of treatment, long-term outcomes are still a major topic of research. 

“These therapies are designed to address the root cause of a condition, but they come with layers of complexity, from specialized administration to unknown long-term durability,” Marshall said.  

Navigating new treatments 

CGTs are not simple medications. Costs can reach millions of dollars per treatment, administration often occurs at specialized centers and treatment may require major life considerations, including fertility impact or extended monitoring.  

That’s where Prime’s comprehensive approach can help.  

Prime combines predictive analytics, rigorous clinical policies, proactive pipeline monitoring and outcomes tracking to help ensure members receive appropriate, high-value treatments. It also helps payers manage costs through personalized strategies.  

“Reimagining pharmacy solutions means keeping patients and their families at the center, making sure we deliver the kind of care we’d want for our loved ones,” Tabatabai said.  

As more therapies launch, Prime expects richer insights from real-world evidence, helping payers better understand durability, long-term outcomes and total cost of care. The team continues to update forecasting models and pipeline insights to support clients navigating this transformative space. 

A rapidly expanding frontier 

With more than 3,200 global trials underway, and new regulatory pathways accelerating review times, CGT innovation is moving faster than ever.  

Prime’s Cell & Gene Therapy Pipeline Outlook helps payers plan ahead by tracking when new drugs may hit the market. 

“We want conversations to start well before these products hit the (U.S. Food and Drug Administration), because these aren’t traditional treatments,” Kjesbo said. “Patients and providers need time to weigh questions that are clinical, financial and deeply personal.”  

What to watch in the year ahead 

Several therapies may reach the market sooner than expected, including candidates for autoimmune diseases, genetic hearing loss and other conditions.  

One promising treatment is a chimeric antigen receptor (CAR) T-cell therapy for stiff person syndrome, an autoimmune neurological disorder — potentially the first CAR T-cell therapy outside oncology — which has shown promising early results.  

Looking further out, advancements in clustered regularly interspaced short palindromic repeats (CRISPR) and new applications in chronic conditions, such as type 1 diabetes and heart failure, also signal a broadening pipeline beyond rare diseases and cancer. 

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Listen to the latest episode of the “Pharmacy Friends” podcast  

Read the latest Cell & Gene Pipeline Outlook   

Learn more about how Prime manages cell and gene therapies 

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