Leadership Series: The future of gene therapy


Emily Boerger


Bhash Parasuraman, vice president of value and access for rare disease and internal medicine at Pfizer, joined State of Reform on Thursday for a conversation on the future of gene therapy. 

As part of our virtual “Leadership Series,” Parasuraman discussed the outlook for gene therapy, the policies she’d like to see to support these treatments, and conversations around access to care.


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Genetic diseases occur when there is damage to an individual’s DNA code. This damage can impact the normal functioning of various body systems, cause severe disability, and reduce life expectancies. Most cases of genetic diseases are inherited, and about 80% of rare diseases are genetic. Gene therapies are either treatments that replace a “bad gene” with a functioning gene, says Parasuraman, or treatments that can edit genes. 

“Those different approaches to gene therapy hold the promise of taking a non-functioning or a ‘bad gene’ and replacing that with a ‘good gene’ and therefore giving years of life and quality of life to patients and their families.”

Parasuraman says clinical trials for gene therapy started taking off about 5 to 7 years ago and there has been substantial progress in the last 3 to 5 years.  

“It’s still early days for these therapies to finally make it into the market and make it into humans. But if I look at some of the reports … I think this decade we’re going to see quite a few of them potentially coming into the marketplace.”

When asked about some of the policymaking that needs to take place to better support gene therapies, Parasuraman said it’s important to look at innovative payment models. She says that unlike most medicines today, gene therapies are one-time treatments. 

“These medicines are not going to be priced the same way as a biologic or even a pill. These are going to be more expensive and that’s going to create some budget challenges, particularly for Medicaid. And therefore, there needs to be mechanisms or policy changes in place to allow for different payment models.”

Rather than paying for these treatments up front, says Parasuraman, perhaps they can be paid for overtime and evaluated based on how the product performs in the real world. She says there should be “pay-for-performance” where the payment is tied to real world performance of the treatment. 

She’d like to see Congress enable states to “allow them to get into arrangements to pay for performance or pay over time.”

Parasuraman also discussed access challenges. One barrier to care, she says, is that patients often have to cross state borders to receive treatment. This can create complications, in particular, for Medicaid enrollees where their home state would have to agree to pay hospital rates in another state.  

“These policies that exist more for just regular therapies could become a barrier for patients and families because any delay results in the disease progressing. And some of the promise that gene therapies have is that they may not completely reverse the disease, but they may keep the patient in a steady state.” 

The full conversation is available in the video above.