First Gene Therapies for Sickle Cell Disease Approved
New gene and cell therapies are reaching the market at a rapid clip. Last month the FDA simultaneously approved two therapies for the treatment of sickle cell disease, a genetic blood disorder: Casgevy, priced at $2.2M, and Lyfgenia priced at $3.1M. While they join other multi-million gene therapies already in the market, the pivotal difference is that sickle cell disease is more prevalent than the conditions targeted by the earlier therapies. For plan sponsors, the greater likelihood of claims for these new therapies underscores the importance of developing a comprehensive strategy for managing costs while ensuring proper access to these life savings treatments.
A New Hope for Sickle Cell Disease Patients
Sickle cell disease affects approximately 100,000 patients in the US. The genetic mutation present in individuals with sickle cell disease causes red blood cells, which deliver oxygen throughout the body, to become rigid and sickle shaped. This affects blood flow and may cause serious health problems, including attacks of pain (called sickle cell crisis), anemia, swelling of hands and feet, frequent infections, and damage to multiple tissues and organs. The average life expectancy of adults affected by sickle cell disease is only 52 years.
Current treatment consists of medications that act to reduce disease severity, with cost ranging from about $2,000 to $120,000 annually. Patients may also incur significant medical costs, as well as increased absenteeism. Until now, the only curative treatment has been hematopoietic stem cell transplant; however, the opportunity to receive this treatment is limited by donor availability.
Gene therapies are novel new treatments that modify or replace missing or faulty genes, potentially curing or significantly improving a chronic genetic disease. Both Casgevy and Lyfgenia are one-time infusions that require chemotherapy beforehand and a potential lengthy hospital stay afterwards; the entire process takes about 3-6 months. While the therapies are similar from a clinical perspective, Casgevy's lower price point compared to Lyfgenia may provide a competitive market advantage. Given the complex administration, cost, and availability of other treatment options, it is possible that market uptake of these therapies may be initially slow.
Health Equity Considerations
African Americans are disproportionately affected by sickle cell disease, with an incidence rate in the US of about 1 in 365 births. Sickle cell disease patients have been a historically marginalized population, with experts indicating these patients often face systemic inequity in medical care. About 45% of sickle cell disease patients are Medicaid beneficiaries, while 35% of patients have commercial coverage and 20% have Medicare. Commercial and government plan sponsors should consider health equity when developing coverage criteria for the new gene therapies, and how health disparities may impact access and pursuit of treatment in the patient population.
Strategies for Plan Sponsors to Consider
Gene therapies are administered by healthcare professionals in specially designated treatment facilities; they require high-touch clinical support for optimal health outcomes and are typically billed through the medical benefit. In addition to the high cost of the drug itself, substantial administration and procedural fees may also come into play. Given that long-term data showing outcomes is still limited, it’s important to recognize the potential for an incomplete clinical response or even treatment failure.
As sickle cell and other high-cost gene therapies enter the market, employers should consider a long-term comprehensive approach to managing these therapies from a clinical and cost perspective. A key first step is assessing the likelihood of claims occurring in your plan’s population; ideally, such assessments should be conducted on a regular basis as the member population changes. The next step is to inventory and evaluate vendor strategies, which may include reviewing your medical carrier’s utilization management programs, network strategy, site of service and claims payment strategy, and care management programs. All plan sponsors should also explore the availability of outcomes-based reimbursement and other payment models, while keeping in mind that these options may currently be limited and their value to plan sponsors remains to be determined. Lastly, as the market continues to evolve, regularly engage with your medical and pharmacy vendor on availability of new strategies.