There are new sickle-cell disease treatments approved by the FDA, but they come with a cost.
The Food and Drug Administration (FDA) recently approved two new treatments for sickle-cell disease for the first time in two decades. But, there’s a catch. They’re extremely expensive.
Patients inflicted with the disease have two copies of a mutated, hemoglobin-producing gene. The mutation makes the molecule warp into a rigid sickle shape. Adakveo, made by Novartis, can prevent episodes of severe pain induced when malformed blood cells get stuck in blood vessels. Patients sixteen years of age and over can get an Adakveo infusion once monthly. The second drug, Oxbryta, made by Global Blood Therapeutics, can prevent severe anemia, which, if left untreated, can cause permanent organ damage. It is taken in pill form daily and has been approved for patients twelve years of age and older. Thirty more sickle-cell drugs are now in late-stage clinical trials.
While these treatments can be life-changing, they each cost nearly $100,000 a year and must be taken for the rest of a patient’s life. There are currently approximately 100,000 people with sickle-cell disease in the United States and the number totals in the millions around the world.
“Those prices are about double the median family income in the United States, highlighting a growing dysfunction in the pharmaceutical market,” said Ameet Sarpatwari, assistant director of the Program on Regulation, Therapeutics and Law at Brigham and Women’s Hospital in Boston.
“This is an extraordinary time,” said Dr. Alexis Thompson, former president of the American Society of Hematology and a sickle-cell expert at Northwestern University, referring to the drugs that have recently and will soon hit the market. And, Novartis and Global Blood Therapeutics have reported they are optimistic the drugs will be covered by most medical insurers.
A spokesperson for Novartis said, “We’ve taken a thoughtful approach to the price of Adakveo, balancing the innovation it brings to the treatment of sickle-cell disease, the benefits it can provide to patients, and the importance of ensuring that appropriate patients have access to it.”
However, David Mitchell, founder of Patients for Affordable Drugs, an advocacy group, said, “Drug companies want us to ask this question: What are we willing to pay to ease the pain and challenge of living with sickle-cell? When it’s your child facing the disease, or your friend in unbearable pain, the answer is ‘anything.’” Yet, he said, a better question is, “What amount should drug companies make on these drugs?” And the cost extends even beyond that.
“Many adults with sickle-cell disease have subtle or overt brain damage, which can make it difficult for them to fully understand, plan or adhere to treatment,” ‘said Dr. Sujit Sheth, a sickle-cell expert at Weill Cornell Medicine in New York.
“What is killing patients is limited oxygen delivery,” said Dr. Love, of Global Therapeutics.
Dr. Thompson, of Northwestern University, advised against using both of the new drugs together to treat different aspects of the disease because of the limited data on the safety of doing so. Yet, this may change as the medications become more widely available.
Sources:
Two New Drugs Help Relieve Sickle-Cell Disease. But Who Will Pay?
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