Bluebird bio's gene therapy fertility support program gets thumbs down from HHS inspector general

The door is closing on bluebird bio’s attempt to secure Medicaid coverage for its fertility support program, which is already offered to patients with commercial insurance who receive its gene therapies.

On Monday, the Office of the Inspector General (OIG) of the Department of Health and Human Services (HHS) published an unfavorable opinion in response to bluebird’s request for an advisory review of the program.

The OIG stated that it didn’t have the necessary data to determine that the program would pose a low enough risk of fraud and abuse to grant it immunity to the federal anti-kickback statute. The program would provide “remuneration” to patients that would induce them to buy the drugs, the OIG wrote.

Bluebird said in a statement that it was “extremely disappointed,” in the ruling “after engaging on this issue for more than two-and-a-half years.”

“We are also surprised that OIG does not view infertility as a barrier to treatment—particularly since HHS’s own Center for Medicare and Medicaid Innovation (CMMI) clearly recognizes the essential role of fertility preservation through its focus on these services within the Cell and Gene Therapy Access Model and its mandate that participating manufacturers provide them at no cost,” bluebird added.

For patients to receive treatment with any of bluebird’s gene therapies—Lyfgenia, for sickle cell disease (SCD); Zynteglo, for transfusion dependent beta thalassemia (TDT); and Skysona, for cerebral adrenoleukodystrophy (CALD)—they first must first undergo chemotherapy to eliminate stem cells in the blood marrow that will be replaced by treated cells. But the chemo treatments can rob patients of their reproductive ability.

List prices for the one-and-done therapies run between $2.8 million for Zynteglo and $3.1 million for Lyfgenia.

“No patient should have to choose between a potentially lifechanging or lifesaving therapy and the chance to have a child,” bluebird added. “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.”

Last week in federal court, Vertex Pharmaceuticals filed a lawsuit against the HHS over the same issue involving its $2.2 million gene therapy Casgevy, which treats patients with SCD and TDT.