La Food and Drug Administration a récemment approuvé deux nouveaux traitements transformateurs contre la drépanocytose, le premier en 20 ans. Mais les médicaments sont extrêmement chers, renouvelant des questions troublantes sur l’accès aux médicaments de pointe.
Adakveo, fabriqué par Novartis, peut prévenir des épisodes de douleur presque insupportable qui se produisent lorsque des cellules sanguines malformées se coincent dans les vaisseaux sanguins. Approuvé uniquement pour les patients âgés de 16 ans et plus, il est administré en perfusion une fois par mois.
Oxbryta, fabriqué par Global Blood Therapeutics, peut prévenir une anémie sévère de la maladie qui peut entraîner des dommages permanents au cerveau et à d’autres organes. Une pilule quotidienne, le médicament est approuvé pour les patients âgés de 12 ans et plus.
Chaque traitement coûte environ 100 000 $ par an et doit être pris à vie. Bien qu’il ne soit pas rare qu’un médicament traitant une maladie rare ait un prix aussi élevé, 100 000 personnes sont atteintes de drépanocytose aux États-Unis et des millions d’autres dans le monde.
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.
Questions of access worry sickle-cell specialists even as they welcome powerful new treatments expected in the next few years. About 30 more sickle-cell drugs are now in late-stage clinical trials.
The companies argue that without drugs, management of sickle-cell disease itself is expensive. It costs an average of about $10,000 a year to treat children, and about $30,000 a year to treat adults, for complications like pain crises, organ damage and strokes.
But Dr. Sarpatwari is leery of companies’ cost-benefit analyses, which he said are based on limited evidence and assume that the drug makers ought to be able to extract a maximum price for the treatments, without regard to actual development costs or any taxpayer support that may have been involved.
David Mitchell, founder of Patients for Affordable Drugs, an advocacy group, said patients and insurers should not agree to just any price for these medications.
“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?” he said. “When it’s your child facing the disease, or your friend in unbearable pain, the answer is ‘anything.’”
But that’s the wrong way to approach pricing, he added, and the more appropriate question is: What amount should drug companies make on these drugs?
#Medicaid covers about 50 percent of patients with sickle-cell disease, and #Medicare covers another 15 percent. It’s not clear how these programs can afford to pay for all who might need the new drugs.
An older drug approved in 1998, hydroxyurea, is now generic and costs about $1,000 a year, and it is approved for children.
Hydroxyurea can reduce the incidence of pain crises and strokes by half. Some patients on public insurance programs have no co-pays for it, noted Dr. J. Eric Russell of the University of Pennsylvania.
Yet only about 30 percent of sickle-cell patients take it. So should sickle-cell patients be required to try hydroxyurea before moving on to one of the newer, pricier treatments?
Insurers, said Dr. Enrico Novelli of the University of Pittsburgh, “will want at least an attempt to treat with hydroxyurea. Why jump to a very expensive drug as front-line therapy?”
Adakveo can make patients’ blood cells less sticky. In clinical trials, Novartis found that the drug reduced episodes of pain by 45 percent, compared to placebo, whether patients also were taking hydroxyurea or not.
But the study did not show an effect on the severe anemia that is a grave consequence of sickle-cell disease. [...]
“What is killing patients is limited oxygen delivery,” said Dr. Love, of Global Therapeutics. Oxbryta was developed to help red cells retain oxygen and prevent them from becoming misshapen.
In trials sponsored by the company, patients who took the daily pill saw an increase in their hemoglobin levels within two weeks; some returned to levels near normal.
Should the two new drugs be used together, one to prevent pain and the other to prevent organ damage? [...]
“It will come down to cost and what providers will pay for,” Dr. Novelli said.