The conditioning required for some cell and gene therapy treatments can lead to fertility issues in both men and women. Vertex’s Casgevy, for example, is approved as a potential cure for sickle cell disease and transfusion-dependent beta-thalassemia, but requires patients to receive chemotherapy to prepare for the gene therapy.
Thus there is the dilemma for patients: would you rather be cured or would you rather have biological children? It’s also important to note that, although the long-term outcomes of Casgevy are very good, a cure is not guaranteed.
Not everyone wants to be a biological parent, but many people do. Infertility has also been linked to a range of mental health issues. Even in the context of receiving a gene therapy, fertility support is often not covered by insurance, including Medicaid.
Understanding the dilemma, Vertex has developed a fertility preservation program to help patients that receive its gene therapy. Patients choose their own fertility providers and treatments as part of the program, but receive financial support from Vertex of up to $70,000.
However, it is not allowed to offer the program to patients on federal healthcare programs, such as Medicaid, without violating anti-kickback and beneficiary inducement statute laws. The laws, in theory, prevent pharma companies from offering renumeration or anything of value that may influence patients in government healthcare programs to use a certain drug.
The laws make sense – until you add the context of gene therapies damaging infertility. Vertex previously sought an advisory opinion from the HHS Office of Inspector General (OIG) on whether its preservation program would be allowed, but received a negative opinion in January 2024. According to Vertex, OIG stated that the program “poses more than a low risk of fraud and abuse, and does not promote access to gene therapy care.”
Vertex is now taking the issue to the courts. “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,” states the filing.
Vertex wants the court to allow its fertility program to be offered to Medicare patients – without risking enforcement action – and is also demanding the OIG issue a written advisory opinion. The OIG opinion in January 2024 was delivered orally and despite repeated requests Vertex has not received a written opinion – something the company describes as “delaying tactics” in its lawsuit. A written opinion is supposed to be provided within 60 days of receipt of the request.
Vertex is not the only gene therapy company running into issues when it comes to helping patients with their fertility. Bluebird bio offers a handful of gene therapies that also require chemotherapy. It sought advice from OIG on whether its own fertility support program (offering $22,500 to patients, in addition to travel expenses) would run afoul of anti-kickback laws. OIG’s response was that it could – saying that it did not have the necessary data to determine the risk of fraud and abuse. Essentially, the program could be viewed as remuneration to patients that would encourage them to buy bluebird’s therapy.
Vertex argues that its fertility program should not trigger anti-kickback and beneficiary inducement statute laws, claiming that the laws should criminalize “corrupt quid-pro-quo transactions, like a bribe or kickback, in which remuneration is sought or offered to corruptly skew medical decision making.”
The lawsuit goes on to emphasize that the laws should not “prohibit assistance like the Fertility Preservation Program because such assistance merely removes a financial or medical barrier to care and thereby allows patients to receive appropriately prescribed medical treatment … the Fertility Preservation Program would not improperly skew medical decision-making or provide an improper inducement to prescribe the Product. Nor would patients choose to undergo treatment with CASGEVY in exchange for the Fertility Preservation Program. Rather, doctors will prescribe CASGEVY, and patients will choose to undergo treatment with CASGEVY, because it offers a potential cure for a debilitating ultimately fatal disease.”
Here's hoping that common sense will prevail. Yes, we need to be cautious that patients aren’t bribed into taking certain medicines, but for the conditions treated with these gene therapies – there aren’t many other options. Without fertility programs, patients may choose to delay or forgo treatment entirely. It’s great to see companies thinking about patient needs and creating fertility support programs. Companies should not be disincentivized to go the extra mile.
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