One of the makers of Casgevy — the world’s first CRISPR-based medicine — filed a lawsuit Monday intended to clear the way for the company to pay for fertility preservation services and treatment for people receiving the therapy, which can interfere with reproduction.
In the lawsuit, Vertex Pharmaceuticals challenged an oral opinion issued by the Health and Human Services Office of the Inspector General that its fertility support program would violate federal anti-kickback laws and accused the agency of failing to meet a statutory deadline to issue a written determination. The suit names, in addition to the OIG, HHS Secretary Xavier Becerra, and agency Inspector General Christi Grimm as defendants.
When the Food and Drug Administration approved Casgevy in December, the decision brought newfound hope to patients living with sickle cell disease, which despite being one of the most common genetic conditions, has been underfunded and under-researched for decades.
The groundbreaking treatment, made by Vertex and CRISPR Therapeutics, delivers a potential cure for people born with the painful, life-shortening blood disorder. But it’s not an easy treatment to receive; patients must first undergo chemotherapy to remove bone marrow cells carrying an inherited genetic glitch, a process which can cause serious side effects, including severe infections, mouth sores, and infertility.
There are methods for preserving fertility prior to treatment — freezing sperm or eggs — but many insurers, including Medicaid, do not provide coverage for such procedures in the context of sickle cell disease treatment. The majority of the estimated 100,000 Americans with the inherited blood disorder rely on Medicaid for health care coverage, leaving many to face the impossible choice of a cure or a chance of having biologically related children.
In January, the Centers for Medicare and Medicaid Services announced plans to increase access to Casgevy and Lyfgenia, a gene therapy for sickle cell disease developed by Bluebird Bio, which cost $2.2 million and $3.1 million, respectively. Those plans include requiring manufacturers of such treatments to cover certain fertility preservation services.
According to the lawsuit, Vertex has developed a fertility preservation program for patients who meet certain income requirements and don’t have fertility care coverage through their insurer. This program provides financial support for a range of services including counseling, fertility drugs, collection and storage of sperm and eggs, genetic testing, and in vitro fertilization, up to $70,000. Under the program, payments for treatments provided to Casgevy patients are made directly to fertility providers.
However, programs aimed at making it easier for patients to access certain kinds of medicines can risk running afoul of federal laws known as the Anti-Kickback Statute and the Beneficiary Inducement Statute, which together prohibit pharmaceutical companies from offering or paying, directly, or indirectly, any remuneration — which includes money or anything else of value — to induce Medicare or other federal programs to purchase their drugs.
In recent years, federal prosecutors have more aggressively been pursuing violations of these laws as a way to rein in programs that provide patients with free medications, copay assistance, or help with navigating insurance coverage, as well as donations made to charitable foundations that provide financial assistance to patients.
It was in that context that in June of last year, according to the lawsuit, Vertex asked the OIG to weigh in on whether or not the company’s fertility preservation program would violate either statute. In November, the company was told that the agency would not be issuing a favorable opinion. As a result, Vertex opted to offer its fertility preservation program only to privately insured individuals, which STAT previously reported.
However, the company has still not received a written statement to this effect, which by law the OIG has to provide within 60 days of receiving a request for an advisory opinion. In the lawsuit, which was filed in the District of Columbia, Vertex asked for OIG to be compelled to issue a written ruling without further delay.
Separately, Vertex argued that its program does not violate federal kickback laws because it does not impact doctors’ decision-making — the program will only be offered to patients after their health care providers have prescribed Casgevy. The company described its fertility preservation program as increasing access to medically necessary care, and the OIG’s determination to be “arbitrary and capricious.”
The HHS Office of the Inspector General declined to comment on the suit.
“The government’s refusal to allow comprehensive fertility support for government insured patients will only further exacerbate healthcare inequalities in the sickle cell disease and beta thalassemia patient populations,” a Vertex spokesperson said in an emailed statement to STAT.
“Thousands of Americans with sickle cell disease or beta thalassemia should not be forced to choose between the potential of a functional cure for their debilitating disease and the possibility of having children.”
The Vertex spokesperson declined to say how many patients have received Casgevy to date, and whether any of them are beneficiaries of a government insurer. On an earnings call in May, the company said that as of mid-April, it had collected cells from five patients who were slated to receive infusions of the cells, once they had been modified with CRISPR gene-editing technology to boost the amount of hemoglobin they’re able to produce.