The approval from the Food and Drug Administration on Friday for a groundbreaking gene-editing treatment brings potential relief to individuals suffering from sickle cell disease. This genetic disorder, which primarily affects Black individuals, is rare but causes significant impairment.

Around 100,000 individuals in the United States, and millions globally, experience the effects of a condition where a flawed gene changes blood cells and leads to intense discomfort. The FDA has authorized this therapy, which necessitates a lengthy hospital stay and can impair fertility, for patients starting at age 12 with no maximum age restriction. The agency anticipates that physicians will recommend it for those with severe versions of the illness.

The cost for the single treatment will surpass $2 million and it is unclear if those who require it will receive it. There has been no indication from either the government or private insurance companies on whether they will cover it.

Private insurance companies may hesitate to cover the costs of therapy because Americans tend to switch insurance providers frequently. Even if an insurer covers the high cost of the therapy, they may not see long-term savings when patients no longer require other treatments such as pain medication, blood transfusions, and bone marrow transplants.

The safety of Casgevy, a gene-editing treatment, over a long period of time is currently uncertain.

Patients in the U.S. with the condition and their insurers typically pay an average of $1.6 to $1.7 million for disease management throughout their lifetime, if the cost is between $1.35 to $2.05 million.

Two FDA-approved treatments for blood disorders, which do not involve editing genes, have a price tag of $2.8 million and $3.5 million. These therapies involve the transplantation of stem cells from healthy donors to patients without modifying their DNA.

Is it covered by insurance?

According to Beth Devine, a health economics professor at the University of Washington, both Medicaid and private insurance companies may oppose the idea of paying for the treatment in advance. Studies conducted by government and private researchers suggest that approximately 50,000 individuals with sickle cell disease are covered by Medicaid.

According to the speaker, the United States faces challenges in this area due to the existence of a private insurance market and frequent changes in coverage. The insurance companies who cover the cost of therapy may not see the long-term benefits of improved patient healthcare.

Are there any safety concerns?

Vertex and CRISPR Therapeutics stated in their submission to the FDA that there were no significant safety issues for patients who underwent the therapy. However, it is not completely risk-free.

In October, a group of unbiased specialists who advise the FDA gathered to deliberate on whether the companies had provided adequate evidence to prove that the treatment would only alter the specific genetic material in stem cells that it is intended to target.

The majority of panel members concurred that the safety record of the medication was satisfactory, despite potential risks of off-target editing leading to cancer in patients.

The FDA suggests that companies developing gene therapy should monitor patients for potential long-term consequences for a minimum of 15 years. Those receiving Casgevy or Lyfgenia will participate in a study to assess the safety and efficacy of the treatments.

According to Julie Gerberding, current President and CEO of the Foundation for the National Institutes of Health and former director of the Centers for Disease Control and Prevention, the FDA’s approval of pioneering gene therapies is a positive indication for the market, despite the uncertainties.

She stated that it communicates a distinct signal to individuals within this area that there exists a regulatory route for obtaining authorization and approval. This is true even if the treatment is only applicable to a specific portion of the population and the number of participants in the approval-based trials is limited.