NEW YORK – The US Department of Health and Human Services' Office of Inspector General on Monday issued its opinion that gene therapy developers offering to pay for patients' fertility preservation procedures won't be granted a safe harbor from federal fraud and abuse laws.
The statement is in response to a request for an advisory opinion from a company whose name is redacted in the document, and which the OIG issued last week but posted publicly on Monday, on whether offering fertility preservation services to certain patients receiving a gene therapy treatment would run afoul of such laws. Bluebird Bio previously said it had sought an advisory opinion from the OIG on its fertility preservation program.
The US Food and Drug Administration last year approved Bluebird Bio's Lyfgenia (lovotibeglogene autotemcel) and Vertex Pharmaceuticals and CRISPR Therapeutics' Casgevy (exagamglogene autotemcel), making them the first marketed gene therapies for sickle cell disease, and making Casgevy the first commercialized therapeutic to incorporate CRISPR gene-editing.
Both gene therapies require patients to undergo an intensive conditioning process that involves chemotherapy, which can reduce infertility for female and male patients. While the companies manufacturing the gene therapies paid for fertility preservation during clinical trials, they're uncertain they'll be able to continue to do so for patients insured by public payors such as Medicaid.
Vertex, which had also asked the OIG for an advisory opinion on the question of whether its fertility preservation program would break anti-kickback and inducement laws, said it did not have a comment to share on the opinion. Vertex last week sued the HHS and the OIG and asked the US District Court for the District of Columbia to step in and allow it to provide fertility preservation treatment. In its complaint, the company said that while the OIG had verbally communicated that it would not issue a favorable opinion on the matter last November, the agency hadn't issued a written opinion, which it is required to do within 60 days of receiving a request for an advisory opinion. Vertex asked the court to compel the OIG to issue a written opinion.
In its written opinion issued on Monday, the OIG confirmed fertility support programs likely would not comply with the federal Anti-Kickback Statute (AKS), which prohibits companies from offering compensation in exchange for business paid for through government programs, or the federal Beneficiary Inducement Statute (BIS), under which companies can be fined civil monetary penalties (CMP) for compensating patients insured by Medicare or state programs in a way that influences their selection of a provider or supplier.
The watchdog agency characterized its decision as "favorable in part and unfavorable in part."
In Bluebird's request for an advisory opinion, it had also asked whether paying for certain travel expenses for patients would run afoul of the law. The OIG in its advisory opinion said that it would not impose sanctions on the proposed program to provide support for certain expenses related to travel, lodging, and meals under the AKS and that such a program isn't prohibited under the beneficiary inducements CMP, since it satisfies an exception related to promoting access to care.
However, paying for fertility preservations could constitute grounds to impose sanctions under both the AKS and BIS. Specifically, Bluebird's proposed arrangement would involve providing up to $22,500 in fertility support to patients, including for fertility preservation procedures and storage.
The OIG said that providing $22,500 worth of fertility support could qualify as remuneration under the AKS, as it could spur treatment centers to recommend Bluebird's gene therapy over a competitor's or alternative treatment that didn't offer a similar program. In the OIG's view, the program could also qualify as remuneration under the BIS, since it could encourage patients to choose treatment centers participating in Bluebird's program. The exception for promoting access to care that the travel support program falls under wouldn't protect the fertility support program, since the agency hasn't seen data showing the program would improve patients' access to gene therapies.
The OIG said it made its decision on Bluebird Bio's request "based on the relevant facts certified in [the] request for an advisory opinion and supplemental submissions and the limited data available regarding, for example, the impact of fertility support on access to healthcare services; costs to federal healthcare programs; patient outcomes; competition; and the risk of improper steering."
The agency stressed that it currently lacks data on the fraud and abuse risk associated with fertility preservation programs for cell and gene therapies. "These [cell and gene therapy] treatments are novel, and much is yet unknown about them and optimal arrangements for ensuring appropriate access to them," the agency wrote. "This uncertainty makes it difficult to assess the risk of the fertility support and offer prospective immunity under our fraud and abuse authorities."
The OIG said it expects to gain additional evidence related to the provision of fertility services through the Center for Medicare and Medicaid Innovation's Cell and Gene Therapy Access Model, a voluntary program under which the Centers for Medicare and Medicaid Services will partner with state Medicaid programs and drug manufacturers on outcomes-based agreements for novel sickle cell treatments. Drugmakers partaking in this pilot will be required to pay for certain services, including fertility preservation.
CMS has the authority to establish safe harbors within CMMI's model arrangements, but not permanently. "As more data become available, we may consider them in future risk assessments regarding arrangements similar to the fertility support," the OIG wrote.
Bluebird Bio issued a statement expressing disappointment in the decision and said it would continue to advocate for Medicaid patients' access to fertility preservation services.
"No patient should have to choose between a potentially life-changing or lifesaving therapy and the chance to have a child," a company spokesperson said. "While we recognize that the CMMI model could play a role in overcoming fertility preservation as a barrier to access, we remain concerned about the impact of a two-tiered system between states who participate and those who do not. Now, without clear guidance or safe harbor for manufacturers, HHS risks exacerbating an already gaping inequity between patients insured through Medicaid and those who are covered by commercial insurance."
The OIG said that its opinion doesn't prohibit Bluebird and others from carrying out this type of arrangement and that the unfavorable opinion doesn't mean that such programs violate the AKS, since that would require an assessment of intent. The advisory opinion only reflects that the agency will not offer prospective immunity for such programs, the OIG said.