A U.S. nonprofit organization found Biogen Inc's Spinraza treatment for spinal muscular atrophy and Novartis AG's experimental gene therapy are both expensive but have the potential to be more cost effective with additional research, according to Reuters.
Here are seven details:
1. The nonprofit suggested learning more about its U.S. price and long-term success rates. The Institute for Clinical and Economic Review in Boston used the "quality-adjusted life year" assessment to evaluate the drugs.
2. ICER noted both drugs provide substantial clinical benefit while still having questions about the long-term effectiveness.
3. Spinraza is U.S.-approved and one of the most expensive drugs, costing $750,000 for the first year of use and $357,000 annually in the years following, according to the report. Patients who use the drug have experienced slow progression of the diseases. However, the drug is not a cure.
4. The ICER assessment estimated that Spinraza represents a cost of $728,000 per quality-adjusted life year, or year of healthy or near healthy life, for patients diagnosed with spinal muscular atrophy.
5. Novartis aims to find a cure for spinal muscular atrophy with its gene therapy. The drug Zolgensma is expected to earn U.S. approval in the first half of 2019. The company estimates the drug would be cost-effective at $4 million to $5 million as a one-time treatment. ICER estimated it at $2 million and said it represents a cost of $240,000 per quality-adjusted life year.
6. Treatments ranging between $100,000 and $150,000 per quality-adjusted life year are considered a good value. However, this can be a stretch for rare conditions and diseases.
7. It is unclear how payers will respond to the drugs hitting the market. Express Scripts told Reuters the Novartis estimated value would be "unsustainable."