Biotechs Seek Federal Coverage of Fertility Preservation Before Gene Therapy
Both bluebird bio and Vertex Pharmaceuticals have received unfavorable opinions from the Office of the Inspector General on the matter.
Haydar Frangoul, MD
Credit: Tristar Health
The Office of the Inspector General (OIG) of the Department of Health and Human Services (HHS) has provided an unfavorable opinion to bluebird bio’s attempt to secure Medicaid coverage for its fertility support program for patients receiving its gene therapies.1
The OIG explained its unfavorable opinion, stating that it did not have the necessary data to determine the program’s risk of fraud and abuse and therefore grant it immunity to the federal anti-kickback statute. The OIG stated that the fertility support program could be viewed as “remuneration” to patients encouraging them to buy the therapy.1
bluebird's program is currently offered to patients with commercial insurance receiving its gene therapies. These therapies include lovotibeglogeneautotemcel, approved as Lyfgenia for treating sickle cell disease (SCD); elivaldogene autotemcel, approved as Skysona for cerebral adrenoleukodystrophy; and betibeglogene autotemcel, approved as Zynteglo for beta thalassemia. For all these ex-vivo edited gene therapies, patients must undergo lymphodepleting chemotherapy beforehand, which impacts fertility.
“Individuals undergoing bone marrow transplantation or any type of transplant that can increase the risk of infertility have very limited access to fertility preservation. And some third party payers do not cover fertility preservation prior to chemotherapy. So patients with sickle cell disease are going to requireto undergo high dose chemotherapy that can result in infertility. So, I personally think it is important to continue to advocate that fertility preservation should be offered to every individual undergoing chemotherapy,” Haydar Frangoul, MD, Medical Director of Pediatric Hematology/Oncology at Sarah Cannon Research Institute and Pediatric Transplant and Cellular Therapy Program at TriStar Centennial, told CGTLive®.
WATCH NOW: Haydar Frangoul, MD, on Insurance and Fertility Concerns After Sickle Cell Gene Therapy
Frangoul served as primary investigator on the phase 3 CLIMB SCD-121 trial (NCT03745287) which evaluated Vertex Pharmaceuticals’ exagamglogene autotemcel, approved as Casgevy, for treating SCD. Patients receiving Casgevy, also an ex vivo-edited gene therapy, face the same fertility concerns as those receiving bluebird’s therapies. Similarly to bluebird, Vertex filed a complaint against the Department of Health and Human Services earlier this month to make its fertility preservation program available to federally insured patients. The complaint challenges an unfavorable oral opinion Vertex received from the OIG in January 2024 for which a written opinion has not been provided despite the company’s request.2
Vertex outlined the issue in its complaint, stating that many patients “make the difficult decision to delay or forego treatment,” due to the inaccessibility of fertility preservation services, which could lead to their condition worsening “to the point that they are no longer eligible for treatment.”2
“Because we're giving myeloablative conditioning, there is an impact on fertility. And so part of this whole process, and a really important part of this process, is offering fertility preservation procedures to even very young children. Because once they have yourself and based conditioning, there's no doubt that that will affect their long-term fertility and so that that is a part of our process as well,” Madeleine Powys, MBBS, formerly a locum consultant of pediatric bone marrow transplantation and leukemia at Royal Manchester Children’s Hospital, previously told CGTLive about her experience with administering Orchard Therapeutics’ atidarsagene autotemcel, approved as LIbmeldy in Europe and Lenmeldy in the United States, for the treatment of metachromatic leukodystrophy.
