The FDA recently approved the first new treatments for sickle cell disease in nearly 20 years, but each costs about $100,000 per year and raises questions around how patients can access the treatments, according to The New York Times.
Novartis' Adakveo was approved Nov. 15 to treat patients 16 and older and is given through an infusion once a month. It can prevent episodes of severe pain that are common with the disease.
Global Blood Therapeutics' Oxbryta was approved Nov. 26 for patients 12 and older and is taken as a daily pill. It can prevent severe anemia associated with sickle cell disease, which can lead to permanent brain damage.
Both drugs cost about $100,000 per year and have to be taken for the rest of a patient's life. The cost is about twice the median family income in the U.S., according to the Times.
Ameet Sarpatwari, PhD, assistant director of the Program on Regulation, Therapeutics and Law at Brigham and Women’s Hospital in Boston, told the Times that he is suspicious of the drugmakers' cost-benefit analyses of the drugs. He said they are based on limited evidence and are designed to allow the drugmakers maximize profit regardless of actual development costs or taxpayer support that may have been involved.
Novartis and Global Blood Therapeutics both told the Times they believe most insurers will pay for the drugs. Medicaid covers about 50 percent of patients with sickle cell disease, and Medicare covers another 15 percent, but it is not clear how the programs could pay for all patients who need the drugs.
About 100,000 people in the U.S. are diagnosed with sickle cell disease, along with millions more across the world.
Although patients may be able to afford Adakveo and stick to monthly infusions, many primary care physicians may not be able to administer them, according to the Times.
Despite hurdles to using the new drugs, many experts say they represent significant advances in treating sickle cell disease.
Read the full article here.
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