The U.S. Department of Health and Human Services’ Office of Inspector General (OIG) has issued an advisory opinion stating that gene therapy developers who cover the costs of fertility preservation for their patients will not receive protection from federal fraud and abuse laws.
The opinion, made public on Monday, followed a request from an unnamed gene therapy company seeking guidance on whether financing fertility preservation services for patients undergoing gene therapy would violate federal laws. Bluebird Bio, a prominent gene therapy developer, had previously sought similar guidance for its fertility preservation program.
Last year, the FDA approved Bluebird Bio’s Lyfgenia (lovotibeglogene autotemcel) and Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel), the first gene therapies available for treating sickle cell disease. Casgevy is also notable for being the first commercialized therapy utilizing CRISPR gene-editing technology. Both therapies necessitate an intensive chemotherapy regimen that can lead to infertility, prompting the need for fertility preservation.
While these companies covered fertility preservation costs during clinical trials, they now face uncertainties about continuing such coverage for patients with public insurance, such as Medicaid. Vertex Pharmaceuticals has also sought an advisory opinion from the OIG regarding its own fertility preservation program but refrained from commenting on the opinion. Additionally, Vertex has sued the OIG, requesting that the U.S. District Court for the District of Columbia mandate a written opinion from the agency, which had previously only provided verbal feedback.
In its advisory opinion issued on Monday, the OIG indicated that fertility preservation programs could violate the federal Anti-Kickback Statute (AKS) and the Beneficiary Inducement Statute (BIS). These statutes prohibit offering compensation for business funded through government programs and penalize inducements that influence patients’ choice of providers or suppliers.
The OIG’s opinion was partly favorable; it confirmed that covering certain travel expenses, such as for lodging and meals, would not face sanctions as it supports patient access to care. However, the OIG expressed concern that providing up to $22,500 for fertility support could be viewed as remuneration under the AKS and BIS, potentially incentivizing treatment centers to favor Bluebird’s therapy over competitors and influencing patient choice.
The OIG emphasized its decision was based on the limited data available regarding the impact of fertility support on healthcare access, program costs, patient outcomes, and potential for inappropriate steering. The agency acknowledged that cell and gene therapies are still emerging, and comprehensive data on associated fraud and abuse risks are lacking.
Looking forward, the OIG anticipates gathering more evidence through the Center for Medicare and Medicaid Innovation’s Cell and Gene Therapy Access Model, which involves CMS working with state Medicaid programs and drug manufacturers on outcomes-based agreements. This model may include requirements for covering certain services, including fertility preservation.
Bluebird Bio expressed its disappointment in the decision and emphasized its commitment to advocating for Medicaid patients’ access to fertility preservation services. The company highlighted concerns about the disparities between states participating in the CMMI model and those that do not.
The OIG clarified that its opinion does not prevent Bluebird Bio or other companies from implementing such arrangements but indicates that the agency will not offer prospective immunity for these programs. The advisory opinion reflects the agency’s stance but does not constitute a final determination of legality under the AKS.
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