- Novartis needs to sharply cut the price of its new sickle cell drug Adakveo and Global Blood Therapeutics needs to steeply discount its therapy Oxbryta in order to meet cost-effectiveness thresholds, the Institute for Clinical and Economic Review said in a review of new drugs for the blood disorder.
- Adakveo significantly reduced episodes of pain stemming from the blockage of blood vessels, leading to a conclusion that it delivers a net health benefit, ICER said. Oxbryta was only able to show that it improved levels of oxygen-carrying hemoglobin, the effects of which are less clear, according to the report.
- The sickle cell report was ICER's first using real world evidence from its partnership with Aetion. The analysis incorporated data from the IBM Truven MarketScan database of commercial claims to develop models of health outcomes and costs related to the condition.
Sickle cell disease shortens lifespans, causes periodic crises of pain stemming from blocked blood vessels and leads to many other health complications. Until the introduction of Emmaus Life Sciences' Endari in early 2018, patients had only one treatment, hydroxyurea, which inhibits the characteristic "sickling" of red blood cells and prevents them from clumping together.
Last year, Novartis and Global Blood launched Adakveo and Oxbryta, respectively. The Novartis drug prevents deformed red blood cells from sticking to each other, while the Global Blood agent increases the oxygen carrying capacity of red blood cells.
Because of health complications and hospitalizations, sickle cell disease can be an expensive condition, which helped explain the high annual cost of both agents at their list prices. Adakveo costs between $7,000 and $9,500 a month, while Oxbryta was priced at just under $10,500 per month.
After looking at the clinical evidence, ICER's report concluded that those were far too high.
Adakveo had a significant effect on pain crises, with patients taking it experiencing them at an average rate of 1.63 a year compared with 2.98 a year for patients taking placebo. The drug did not lead to a statistically significant reduction of days spent in the hospital, nor in quality of life, ICER said.
Oxbryta's main advantage was that patients taking it saw an increase in hemoglobin, but they did not see a decrease in pain crises nor an improvement in quality of life. The short- and long-term benefits of an increase in hemoglobin are unclear, but specialists cited in ICER's report said it "can reduce patient fatigue and potentially reduce the risks for specific longer-term harms such as high-output congestive heart failure."
The report also assessed Endari, which costs just one-third of the two newer drugs. It is believed to work by reducing oxidative damage to red blood cells, and won approval on the basis of reducing pain crises.
However, even at its lower price, ICER said Emmaus needs to discount Endari by up to 76% to meet cost-effectiveness thresholds.
The report will likely set a baseline for evaluations of clinical-stage gene therapies for sickle cell disease, should those be successful in the clinic. Bluebird Bio has two, one of which is similar to its treatment Zynteglo, now approved in Europe for beta thalassemia.