The FDA’s accelerated approval for gene therapies: A Q&A with Josh McGee

This year, the Food and Drug Administration announced it was taking steps to help gene therapies gain accelerated approval. Throughout 2022, the FDA approved three new gene therapy treatments for adult patients with various health issues including bladder cancer, Hemophilia B and more.

In April, scientists at Trinity College Dublin announced a significant development towards a new therapeutic treatment of glaucoma.

As scientists continue to make strides in gene therapy to provide new treatments and companies evaluate these health care options, consumers are fearing exorbitantly high costs, and insurers are left with questions on how this impacts plans and policies.

We talked to Josh McGee, Vice President of Program Development at Stealth Partner Group, an Amwins Group Company, to discuss the FDA’s recent moves and their impact on group benefits insurance, advice for insurers as they review these new treatments and insights on the future of gene therapy treatments, as well as the advancement in glaucoma treatment with gene therapy.

What is the significance of the FDA’s goal and other moves recently taken by the agency/?

To answer this question, it’s important to take into consideration the historical context of gene therapy. The first gene therapy was approved back in 2017, and it was a therapy named Luxturna that is used to treat Leber Congenital Amaurosis, a degenerative disease in the eyes that leads to blindness. It was a very critical and forward-thinking therapy that was followed sometime later in 2019 with the approval of Zolgensma, which is used to prevent any further deterioration associated with Spinal Muscular Atrophy. However, with the COVID-19 pandemic, the FDA had to shift their focus over to a solution for COVID and we saw this significant gap between 2017 and 2019, where there were no therapies approved. Once the pandemic subsided, there was a spike in gene therapy approvals, with 4 therapies released in the latter half of 2022: Zynteglo, Skysona, Hemgenix, and Adstiladrin. This increase in activity is sending a clear message that the FDA is reshifting their focus to progressive therapies that are either life saving, life changing, or curative in nature and really going to have an impact on the medical field.

What are the benefits of accelerating gene therapy approval?

One of the biggest benefits of accelerating gene therapy approval is that these therapies are intended to be a one-and-done kind of treatment. While we do not necessarily know the durability of these therapies due to the fact that they are so new to the market, when we take a look at the therapies that are approved today or currently being tested by the FDA, the intent of these therapies is that patients only need one treatment. Zynteglo is a great example of that. Zynteglo is used to treat Beta Thalassemia, a blood disorder that reduces the production of hemoglobin, and individuals with Beta Thalassemia require ongoing, regular blood transfusions. Not only is this time-consuming, it’s also incredibly painful. The fact that these therapies are one-and-done is life changing. They allow individuals to do things in their day-to-day life that they may not have been able to do in the past and, ultimately, while they may be costly, these therapies hopefully eliminate or reduce the amount of money spent on health care in the future.

What are the notable roadblocks and challenges these actions pose from a benefits and insurance perspective?

Although we’re seeing more and more therapies approved, we’re also seeing the price point increase quite dramatically. Luxturna, which was approved in December of 2017, costs $850,000, Zolgensma came next in 2019 and that costs $2.2 million, Zynteglo is $2.8 million, Hemgenix is $3.5 million, so you definitely see this trend of increasing cost and this market, which is $6-7 billion today is forecasted to be somewhere closer to $30 billion in the next two to three years. With this drastic change, no one is going to be immune to the impacts of the difference in market position. It’s going to hit fully-insured, self-funded groups, potentially even Medicare and Medicaid. Furthermore, insurance providers will begin to question, “Do I cover these or not?”, and these can be life and death conversations. We have to approach with another layer of caution and take into consideration the people it would affect the most.

What advice would you give to insurers as they review these new treatments and prepare for coverage impacts?

When preparing coverage, the biggest thing is for insurers to know what’s out there. They need to have experience and credible resources to keep up to date of what’s happening in the gene therapy space. “What is the FDA doing? What does the pipeline for gene therapies look like? What is the market opportunity for these types of treatments?” These are all questions insurers should be asking themselves. Insurers should also stay informed of what the administrators are doing and how the different administrators are managing these conditions when they’re identified to make sure that the treatments are appropriate, timely, and that the outcomes of these therapies are as expected. Another key role insurers can take is verbalizing and vocalizing support of continued innovation in this space. I celebrate anybody who is coming to this space and trying to bring a solution to the table. The market here is still young and there’s still a lot to be learned, but we need to be investing and having conversations about this to make sure we get the best outcome possible, while also creating accountability and responsibility for the outcomes.

What trends are you expecting to see in the future for gene therapy treatments?

The therapies that are in the market today represent diseases with very low prevalence rates, meaning that there aren’t that many people that are affected by these particular diseases. However, as we look to the future, we’re seeing therapies that are currently being reviewed by the FDA that treat diseases with much higher prevalence rates. For instance, when considering gene therapies for sickle cell disease, we’re looking at over 50,000 potential candidates in the U.S. alone. We’re going to continue to see an acceleration in the space, and an increase in potential candidates for these treatments. Another thing we’re going to see, whether it’s in gene therapy, cell therapy, or other, is the customization to each individual, using the unique genome and unique makeup of someone to create a treatment that is specifically tailored to that person. It’s exciting what the future holds in this space.

Related: Sarepta’s gene therapy gets FDA accelerated approval: Are fast-tracked drugs safe?                                                              

How is gene therapy administered to glaucoma patients?

Gene therapy for glaucoma is administered through a vector, which is an engineered virus, because physicians do not have mechanisms small enough to manage your DNA by hand. A virus enters your cells and manipulates your DNA creating copies of the virus. Technology allows us to alter the virus to deliver a positive outcome. Vectors can be delivered In vivo, an injection of the new genes or Ex vivo, where they extract unhealthy cells and add the vector and then return the altered cell to the patient.

If the FDA approves gene therapy for glaucoma, will the costs be affordable and/or will insurers cover costs?

The estimated cost of the glaucoma gene therapy is $1.85 million per treatment. This places significant pressure on payers as this disease state impacts nearly 80 million people globally, around 3 million in the U.S. Because this disease is generally identified in individuals over the age of 65, Medicare is likely to get hit the hardest.

Do you expect gene therapies to escalate due to the Trinity College announcement?

There are high hopes that this gene therapy discovery will lead to the creation of other treatments preventing blindness. The function of this gene therapy is to deliver cells to the body that increase the flow of aqueous fluid which alleviates pressure in the globe of the eye. In glaucoma patients, this fluid build-up is a result of a drainage blockage which overtime creates too much pressure on the optic nerve and ultimately causes blindness.