AveXis, a Novartis company, announced innovative access programs for Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene. AveXis is working closely with payers to offer pay-over-time options up to 5 years and outcomes-based agreements up to 5 years, as well as providing a patient program to support affordability and access.
“Zolgensma is a historic advance for the treatment of SMA and a landmark one-time gene therapy. Our goal is to ensure broad patient access to this transformational medicine and to share value with the healthcare system,” said Vas Narasimhan, CEO of Novartis. “We have used value based pricing frameworks to price Zolgensma at around 50% less than multiple established benchmarks including the 10-year current cost of chronic SMA therapy. In addition, the price of Zolgensma is expected to be within the range of traditional cost-effectiveness thresholds used by ICER when updated for its full labeled indications. We believe by taking this responsible approach, we will help patients benefit from this transformative medical innovation and generate significant cost savings for the system over time.”
“Innovative science like Zolgensma required us to be equally innovative in offering customized access solutions to meet the many needs of payers and patients,” said Dave Lennon, president of AveXis. “We are partnering to accelerate coverage decisions with both government and commercial payers. We are offering a pay-over-time model for this one-time treatment to accommodate the current structure of the US healthcare system and we have also established outcomes-based agreements with payers because we believe in the long-term value of Zolgensma and are willing to stand behind the therapy.”
The current 10-year cost of chronic therapy, which is given over the patient’s lifetime, can often exceed USD 4 million in just the first 10 years of a young child’s life. In addition, that therapy stops working if treatment is stopped. Zolgensma is expected to save costs in the healthcare system compared to chronic treatment for the treatment and care of SMA. The wholesale acquisition cost of Zolgensma of USD 2.125 million is:
- 50% of the 10-year cost of current chronic SMA treatment (estimated at USD 4.1 million)
- 50% below 10-year treatment costs for genetic pediatric ultra-rare diseases (estimated at USD 4.4 million to USD 5.7 million)
- 50% below the ICER ultra-rare disease cost-effectiveness threshold; Zolgensma pricing places it at approximately USD 250,000 per quality-adjusted life-year (QALY)
“We are at the forefront of an exciting time in healthcare when we’ll be able to see major advancements in medical care with potentially curative gene therapies. While there are many questions that we as a healthcare system need to consider, what does not change is our work to ensure that these life-saving medications are affordable and available to the patients that need them,” said Steve Miller, M.D., chief clinical officer, Cigna Corporation. “We look forward to continuing the work we have started with AveXis to find unique solutions like installment payments and outcomes-based agreements for these life changing gene therapies.”
AveXis has partnered with Accredo to offer a pay-over-time option of up to 5 years to help ease possible short-term budget constraints, especially for states, small payers and self-insured employers. In addition, CuraScript SD has been selected as the sole specialty distributor given its rare disease experience, including gene and cell therapies.
Reflecting the pioneering nature of these programs, more than 15 payers are in advanced discussions of terms with AveXis, with some having already agreed, in principle, to terms.
“We are thrilled to be able to offer our members access to this groundbreaking gene therapy, particularly in light of AveXis agreeing to place a portion of the cost at risk, contingent upon demonstrating continued performance over a five-year period,” said Michael Sherman, M.D., M.B.A., chief medical officer of Harvard Pilgrim Health Care. “The clinical benefits of gene therapy for infants with life-threatening genetic diseases, such as SMA, are undeniable, and our innovative, outcomes-based agreement helps ensure that we balance access and affordability for our members. While we anticipate that Harvard Pilgrim would see a small number of newly-diagnosed patients with the very rare SMA Type 1 each year, we believe it is our responsibility to provide access to this lifesaving treatment.”