Vertex Sues for Right to Offer Fertility Assistance to Gene Therapy Patients

Vertex Pharmaceuticals is taking legal action against the U.S. government to allow its fertility assistance program to extend to Medicaid patients with sickle cell disease (SCD) and beta thalassemia (TDT). Currently, patients must undergo chemotherapy to prepare for Vertex and CRISPR Therapeutics' $2.2 million gene therapy, Casgevy, which involves replacing blood marrow stem cells with treated cells. This chemotherapy can impair reproductive abilities, necessitating additional fertility preservation procedures.

Vertex provides financial support for fertility treatments for patients with commercial insurance, covering costs up to $70,000 for those whose income does not exceed 670% of the federal poverty level. However, offering the same support to government program beneficiaries is prohibited by U.S. law, which considers it an illegal inducement to use Casgevy. This has led Vertex to file a lawsuit in federal court in Washington, D.C., against the U.S. Department of Health and Human Services (HHS). The lawsuit challenges the "erroneous legal positions" of the HHS Office of the Inspector General (OIG).

Vertex argues that without judicial relief, thousands of SCD and TDT patients will face an unfair dilemma: choosing between potential cure and biological parenthood. According to Vertex, approximately 100,000 Americans suffer from SCD, with 90% being Black and 50% covered by Medicaid. Additionally, 45% of the 2,000 Americans with TDT are under government healthcare programs.

In June of the previous year, Vertex sought an advisory opinion from the OIG regarding whether its fertility program violated federal kickback or inducement statutes. The OIG responded in November, indicating a likely unfavorable opinion, prompting Vertex to restrict the program to commercially insured patients. Thirteen months later, Vertex still has not received a final advisory opinion, which OIG is supposed to provide within 60 days. The lawsuit seeks to force the OIG to expedite its ruling.

Bluebird Bio, another company marketing gene therapies for SCD and TDT that also require chemotherapy, has faced similar challenges. For over two years, Bluebird Bio has attempted, unsuccessfully, to obtain government approval for a comparable fertility assistance program. In a statement, Bluebird emphasized the importance of equitable access to fertility preservation services regardless of a patient's insurance type or state of residence. The company remains committed to collaborative efforts with patients, advocates, and policymakers to achieve legislative or policy changes permitting manufacturers to offer compliant fertility assistance for those on federal healthcare programs.

The lawsuit underscores the critical issue of equitable access to fertility preservation for patients undergoing life-changing and potentially life-saving gene therapies. Both Vertex and Bluebird Bio highlight the need for policy adaptations that do not force patients to make impossible choices between health and family aspirations. Through this legal battle, Vertex aims to bring attention to the regulatory barriers that prevent equitable health support and push for reforms that accommodate the needs of all patients, irrespective of their insurance coverage.