Cost of Zolgensma, with details on aid programs, money-saving strategies, and additional info
Zolgensma, a groundbreaking gene therapy approved in 2019 for spinal muscular atrophy (SMA), comes with a hefty price tag. The cost ranges between $2.1 to $2.9 million per treatment, a figure significantly higher than that of biosimilar drugs. Let's delve into why Zolgensma is so expensive.
Unique, Complex Technology
Zolgensma is a gene therapy that delivers a functioning copy of the faulty gene causing SMA. This cutting-edge biotechnology, complex manufacturing, and specialized delivery systems are not needed for biosimilars, which are copies of existing biologic drugs.
One-Time Curative Intent vs. Ongoing Treatment
Zolgensma is designed as a "one-and-done" treatment, offering a potential lifelong benefit from a single dose, in contrast to chronic treatments that require ongoing dosing. While the upfront costs are high, this may offset long-term cumulative costs of alternative therapies.
Rare Disease Market and Small Patient Population
The target population for Zolgensma is very small, with around 450-500 children diagnosed annually in the US. This limited patient base increases per-patient development and manufacturing costs. Additionally, the exclusivity of the treatment disincentivizes competition and the development of biosimilars.
Innovative Drug Pricing Models
Novartis justifies the high price of Zolgensma using value-based arguments, comparing lifetime treatment costs of alternative drugs to the one-time cost of Zolgensma. However, the price remains extraordinarily high, reflecting the novelty of gene therapy pricing models and challenges in healthcare financing to absorb a massive upfront cost.
Regulatory and Reimbursement Challenges
Insurance companies carefully scrutinize Zolgensma due to its cost, causing delays and further complicating access. No biosimilars exist yet because of the complexity of gene therapies, unlike biologics for which biosimilars can enter the market after patents expire.
In comparison, biosimilar drugs are follow-on biologics that replicate existing biologic drugs with established production processes and larger patient populations, allowing for more competitive pricing and lower costs.
Accessing Zolgensma and Its Cost
To find out the cost of Zolgensma for your child, contact your doctor, pharmacist, insurance company, or the Novartis Patient Support program for Zolgensma. It's essential to note that Zolgensma may be billed through your primary health insurance instead of the prescription drug portion, depending on your child's insurance coverage and where they receive the treatment. Prior authorization may be required from your insurance company before they cover Zolgensma.
Lastly, always consult your doctor or another healthcare professional before taking any medication, including Zolgensma. Medical News Today's drug information is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects.
[1] Novartis Patient Support program: https://www.zolgensma.com/patient-support/ [2] Medical News Today: https://www.medicalnewstoday.com/articles/327043 [3] Genetic and Rare Diseases Information Center: https://rarediseases.info.nih.gov/diseases/10603/spinal-muscular-atrophy [4] Investopedia: https://www.investopedia.com/terms/b/biosimilar.asp
- The unique, complex technology used in gene therapy like Zolgensma, which includes specialized delivery systems and complex manufacturing, contributes to its high price tag, unlike biosimilar drugs that replicate existing biologic drugs.
- Unlike ongoing treatment options for spinal muscular atrophy, Zolgensma, a one-time curative treatment, may offset long-term cumulative costs of alternative therapies despite its high upfront costs.
- The small patient population for Zolgensma, which is around 450-500 children diagnosed annually in the US, increases per-patient development and manufacturing costs, making it less viable for biosimilar drug development.
