On the Gene Therapy Pipeline

In a session at PBMI’s 2019 Annual Conference, Megan Pasicznyk, PharmD, MBA, senior director, specialty market development, Express Scripts, presented on recent breakthroughs on gene therapies, and discussed the implications payers must understand surrounding the rapidly growing and changing market.  

Dr Pasicznyk explained that gene therapies are different from biologics. “Biologics are designed to manage a condition, whereas gene therapies are intended to solve underlying issues, essentially curing a patient.”

To demonstrate the scale of significance, Dr Pasicznyk pointed out that it was only 1953 when Watson and Crick discovered the components of DNA. The first successful patient gene therapy perfo
rmed was in 1990. In 2018, the first, FDA -approved, commercial in vivo gene therapy, Luxturna, designed to treat inherited retinal disease. 

“Luxturna contains viral particles and has a lot of stability and storage requirements to ensure that when it leaves the pharmacy, it arrives at the treatment facility with the same integrity throughout the process,” said Dr Pasicznyk. This product in particular has to be stored at -80ºF, requiring specialized freezers, power sources, and equipment. 

As gene therapies become more commercial, we can expect them to require intensive service and clinical models, flexibility and customization, unique and drug-specific support, complex conditions in small patient populations, and advancements in patient management and monitoring strategies.  

“Drug pricing is at the forefront of the conversation right now. Some of these new gene therapies are coming up the pipeline right now are front and center in Washington as we have these conversations.” It is important to note that gene therapies are not introduced as a cost-saving treatment and do introduce additional spend to market place, however, these drugs are intended to be revolutionary and innovative. Gene therapy treatments could make the difference between a child making it to college, explained Dr Pasicsnyk.

“While we are very early on in commercialized gene therapy, we’re starting to see competitive price matching in products,” she said, noting that most prices are upward of $300,000 but as studies and trials continue, these could go down. 

“Going forward, how do we make this work for everyone? What does the regulatory environment look like? This is something that we are determining over time so that none of these therapies disappear. There are a lot of developments being made and we need regulations to support the innovation,” said Dr. Pasicznyk. 

“While a lot of this information can seem daunting, these therapies are really not far off or outrageous to think about. It is an exciting place. We’ve been able to leverage a lot of experience from similar products like biologics to the study of gene therapies,” explained Dr Pasiczynk. “The pool of patients that become a candidate for gene therapies is continuing to grow. Patients with compromised immune systems, like children with specialized cancers, could benefit from gene therapies to improve their immune system. There are a lot of challenges associated with them, but if we work together we could find a way that these revolutionary treatments are available to those who need them in a way that is responsible for all of us.—Edan Stanley