Programs Covering the Cost of Gene Therapies Increase

By 2025, the FDA expects to approve 10 to 20 new cell and gene therapies each year. By leveraging a patient’s own biology, cell and gene therapies are already reversing congenital blindness, curing aggressive forms of pediatric leukemia and may soon grant infants with neurological genetic conditions a second chance at life.

Cell and gene therapies are revolutionizing the field of medicine by offering life-changing benefits to people with rare diseases and cancers. These therapies have been gaining considerable attention recently, both for addressing long-standing unmet needs and for being exceptionally pricey.

There are a limited number of currently FDA-approved gene therapies available, including:

  • Imlygic (talimogene laherparepvec) is a genetically modified herpes virus used to treat melanoma. Imlygic was approved by the FDA in 2015.
  • Luxturna (voretigene neparvovec-rzyl) was the first medication approved for an inherited genetic disease ever. Luxturna was approved by the FDA in 2017.
  • Kymriah is a treatment for B-cell acute lymphoblastic leukemia (ALL) which uses the body’s own T cells to fight cancer (adoptive cell transfer).  In 2017, it became the first FDA-approved treatment that included a gene therapy step in the United States. In 2018, the FDA further approved Kymriah to treat adults with relapsed or refractory diffuse large B-cell lymphoma.
  • Yescarta (axicabtagene ciloleucel) is a treatment for large B-cell lymphoma that has failed conventional treatment. The FDA granted approval in 2017 for the second-line treatment of diffuse large B-cell lymphoma.
  • The FDA approved Zolgensma (onasemnogene abeparvovec-xioi) in 2019, the first gene therapy approved to treat children less than two years of age with spinal muscular atrophy (SMA), the most severe form of SMA and a leading genetic cause of infant mortality.
  • The FDA approved nusinersen (Spinraza) for the treatment of SMA in 2019.

As gene therapies continue to enter the market, the leading PBMs have developed initiatives to make it easier for plan sponsors to afford the costs.

CVS Health announced plans to provide self-insured employers with a stop-loss program with Aetna health plans and cover Zolgensma, Luxturna, and potentially other therapies. CVS Health is also developing a broader reinsurance program for high-cost treatments.

Anthem also is exploring special insurance setups, including reinsurance or stop-loss options, to provide employers with protection against the negative financial effects of costly gene therapies.

Express Scripts has an arrangement where it ships Luxterna  through Accredo Specialty Pharmacy or CuraScript Specialty Distribution to the provider after the patient receives health plan authorization. Novartis has partnered with Accredo to offer a five-year payment plan for Zolgensma.

Prime Therapeutics launched the new PreserveRx program that will pay a per-member per-month fee and in exchange will be shielded from the extremely high one-time cost of new therapies such as Zolgensma. PreserveRx is offered in conjunction with BCS Insurance Company, a reinsurance company backed by Blues plans. 


Takeaway: Determining the financing of life-changing gene therapies is essential to achieve the full benefit for patients.