NEW YORK – Vertex Pharmaceuticals is asking a federal district court to step in and allow it to provide fertility preservation treatment to patients with sickle cell disease and transfusion-dependent thalassemia receiving its CRISPR-based gene therapy Casgevy (exagamglogene autotemcel).
In a complaint filed on Monday, Vertex, which sells Casgevy with CRISPR Therapeutics, said it is seeking declaratory judgment from the US District Court for the District of Columbia that a program it has created to provide fertility preservation services to patients receiving Casgevy does not violate the Anti-Kickback Statute (AKS) or the Beneficiary Inducement Statute (BIS), which restrict drugmakers from paying remuneration to induce federal healthcare programs to purchase their treatments. Vertex is suing the US Department of Health and Human Services, HHS Secretary Xavier Becerra, the Office of Inspector General, and Inspector General Christi Grimm.
Casgevy, which carries a list price of $2.2 million, is designed to promote production of functional hemoglobin through a one-time infusion of patients' own cells that are modified outside of the body to increase production of fetal hemoglobin through editing of the BCL11A gene. The treatment has been hailed as a potentially curative gene-editing treatment for SCD and TDT, both diseases involving dysfunctional hemoglobin. However, Casgevy treatment carries the risk of infertility. To prepare the body to receive genetically modified stem cells, patients go through an intensive conditioning process involving chemotherapy, which reduces both female and male fertility.
However, fertility preservation services can cost tens of thousands of dollars and are rarely covered by insurance. Following the approval of Casgevy in the US, there's been a spotlight on the fact that anti-kickback and inducement laws have restricted drugmakers from being able to support patients in this regard.
Vertex launched a program to offer fertility preservation services to patients who lack insurance coverage and are eligible for financial assistance. However, as the firm states in its complaint, before extending this program to federal healthcare programs, the firm sought the opinion of the OIG as to whether this would run afoul of the anti-kickback and inducement laws. There's a particular need for fertility preservation services among Medicaid patients, Vertex noted, since half of those with SCD, a condition most commonly affecting Black Americans, are covered under that federal program, and Medicaid doesn't cover these services.
Last November, the OIG told Vertex that it would not issue a favorable advisory opinion on its fertility preservation program, because "the program implicates the AKS and BIS, poses more than a low risk of fraud and abuse, and does not promote access to gene therapy care." However, the agency hasn't issued a written opinion, which it is required to provide within 60 days of receiving a request for an advisory opinion.
"Even though Medicaid and most other insurers already deny Americans with SCD or TDT fertility coverage, the federal government, through OIG's refusal to issue a favorable advisory opinion, has effectively prohibited those patients from receiving free fertility services from others — leaving them with the Hobson's choice between undergoing a potentially curative treatment or becoming biological parents," Vertex states in its complaint, in which the company also asks the court to compel the OIG to issue a written advisory opinion.
Vertex further argues in its complaint that its fertility preservation program doesn't violate the AKS, since it wouldn't influence doctors' decisions to prescribe Casgevy in the first place or induce patients to undergo treatment in exchange for free fertility treatment. "It strains credulity to suggest that someone suffering from SCD or TDT would choose Casgevy to obtain fertility services rather than to be potentially cured of a debilitating illness," Vertex states in its complaint. "Patients eligible for the fertility preservation program have been diagnosed with SCD or TDT and prescribed Casgevy by a qualified physician, all before the possibility of fertility services is even raised."
Moreover, Vertex points out that under the BIS there is a safe harbor for programs that promote access to care and have a low risk of abuse, and in the firm's view, its fertility preservation program qualifies for such a safe harbor under the law. The US Centers for Medicare & Medicaid Services recently launched the Innovation Center's Cell and Gene Therapy Access Model to increase Medicaid patients' access to innovative products like Casgevy and drugmakers participating in the pilot will be required to partially cover fertility preservation services, specifically the collection and storage of oocytes or sperm. In its complaint, Vertex points out that in launching this access program, CMS has recognized that limited access to fertility preservation services is a significant barrier restricting patients' access to these treatments.
As a result, Vertex is asking the court to determine that the OIG's opinion that its fertility preservation program violates the AKS and BIS is "contrary to law, arbitrary and capricious, and an abuse of discretion." The company also accuses the OIG of trying to discourage Medicaid patients from getting a costly treatment like Casgevy.
"While HHS may wish to avoid the financial cost of providing medically necessary, potentially curative treatment to predominantly Black Americans with SCD or TDT, the AKS is a criminal statute, not a policy to discourage Americans who are eligible for federal healthcare programs from obtaining the benefit of that insurance," Vertex said. "Americans with SCD or TDT who are covered by federal healthcare programs should be entitled to the same fertility assistance as SCD or TDT patients with commercial insurance if third parties are willing to provide it, particularly when the barrier being removed relates to a serious side effect of the regimen delivering the therapy. Any desire by HHS to save money by keeping eligible patients from obtaining critical healthcare does not render that fertility support an illegal 'kickback.'"