Understanding gene therapy coverage for sickle cell disease

Comparing the Drugs

To begin with, Cyr discussed the similarities and differences between the two treatments, explaining that while the FDA categorizes both treatments as cell-based gene therapy, their mechanisms are slightly different.

“Casgevy is a gene editing product that prevents the cells from taking on distinctive crescent shape apparent in sickle cell and usually eliminates pain episodes in almost all patients,” said Cyr.

Casgevy, manufactured by Vertex Pharmaceuticals, uses CRISPR/Cas9 gene-editing technology to edit the erythroid-specific enhancer region of the BCL11A gene in CD34+ hematopoietic stem cells (HSCs).

“The other, Lyfgenia, is a lentivirus. It [targets the] underlying cause of sickle cell by adding a functional gene. It also shows a reduction in vaso-occlusive events and pain,” he continued. The treatment inserts a functional beta-globin gene into HSCs to address SCD.

Cyr told LifeSciencesIntelligence that the efficacy of both drugs is similar; however, Casgevy may have a slightly better safety profile. He cautions that this edge on safety does not necessarily indicate that it is the better gene therapy as Lyfgenia had a somewhat more extended follow-up period, which could show a better understanding of the duration of effect.

“The big thing is that one is priced lower than the other,” Cyr added. According to an article in the American Family Physician, Casgevy costs $2.2 million per one-time treatment, while Sickle Cell Disease News notes that Lyfgenia costs $3.1 million per treatment.

Despite the price discrepancy, Cyr commented, "The one that's priced higher is also starting to say that they will enter into contracts.”

He noted that each treatment had its own benefits or advantages depending on the context.

Insurance Coverage

Beyond discussing the two therapies, Lopata and Cyr discussed the complexities surrounding insurance coverage of SCD gene therapies.

“It was a unique experience having two similar products approved literally on the same day to allow for such a comparison,” Lopata emphasized.

Lopata told LifeSciencesIntelligence that payers are currently using their traditional drug review process to assess gene therapies. These processes include looking at the cost relative to the standard of care, economic modeling, assessing the patient population, evaluating unmet needs, considering treatment guidelines, and understanding provider perception, among other factors.

“What's made the cell and gene therapy space so challenging for payers and really a lot of different stakeholders — providers, and employers as well — has been the large price tag associated with these treatments,” she explained.

“That's added some additional challenges and layers to the review process from the payer side. In some cases, the decision to allow coverage of cell and gene therapies doesn't even necessarily fall with the payer like it would for a traditional specialty product. Many payers are carving out cell and gene therapies due to the cost and the complexity of managing the overall process.”

Instead, outcomes-based agreements arise from the employer’s side. She continued, “As we start to talk about the outcomes-based agreements and even from the employer side — employers are ultimately making decisions around benefits for commercial populations — employers have also started to make decisions around carving out or excluding coverage for cell and gene therapy. So the process has become a lot more complicated.”

“One of the unique things that we are seeing and historically gene therapies, especially the high priced ones, payers have kind of looked at cost-effectiveness, so like cost per quality-adjusted life here, which is a way of thinking of value and especially with sickle cell, people are starting to think of affordability a little bit more in the budget impact not necessarily within commercial plans but within Medicaid plans,” Cyr added.

Lopata added that having both drugs for SCD approved simultaneously offered a unique opportunity to assess whether payers or employers would select a preferred product based on the clinical outcomes, safety, cost, and complexity of follow-up care.

So far, Lopata notes that payers provide access to both products without indicating a preference for one, as the nuances of treatments have caused payers to acknowledge the need to cover both.

Another factor that may impact coverage is access to treatment centers and how that varies based on geography. Payers' decisions are likely also considering the reasonable distance to a treatment option when making coverage decisions.

Accessing Care

“In terms of equitable access with this population, it comes down to patient identification and allowing patients to be identified,” highlighted Lopata.

For example, some patients in this population may have insurance but still find it challenging to see a provider regularly.

“Are patients merely receiving care in the ER without subsequent follow-up or access to long-term management?” posed Lopata. “That presents the opportunity [to say], ‘let’s treat this patient with gene therapy.’”

Lopata told LifeSciencesIntelligence that there are a lot of opportunities to use technology to identify patients for these therapies. Analyzing data from electronic health records and identifying patterns of emergency room use or unplanned care can be a strategy for identifying patients who may benefit from a one-time treatment.

Additionally, she noted, “Telehealth is great when there are geographical barriers or work schedule barriers to going to the doctor's office, especially if it's in an inconvenient location,” she added.

Ongoing Challenges

Even with the significant advancements made for SCD in the cell and gene therapy space, there are ongoing challenges that need to be acknowledged and addressed.

“This has the potential to revolutionize sickle cell disease care and the lives of individuals suffering from it. At the same time, we must be mindful not to [do] what I've seen with other gene therapies, Cyr concluded.

He went on to explain that people have the perception that gene therapies fix or change the gene and act as a cure-all, fixing all the patient's problems. He cautions that that is not the case. While these gene therapies will be critical tools for treating these conditions, they are not perfect solutions that make other issues obsolete.

Lopata added that fertility preservation and whether payers will include that in their coverage are other challenges in gene therapies.

“The chemotherapy that is associated with [some gene therapy processes] can lead to infertility,” noted Lopata; however, the generations of patients who may be good candidates for these treatments may also be at a stage in their lives where fertility considerations pose a significant barrier.

As the landscape evolves, payers and other stakeholders must change, addressing these challenges — and others — as they arise.

Editor's Note: This article has been edited to reflect Precision Value & Health's rebranding to Precision AQ.