The regulatory approval by the Food and Drugs Administration (FDA) of a groundbreaking new Sickle cell disease drug in the United States earlier this month sparked much hope and optimism, as well as the news that it is already moving for clinical trials in certain African countries was also appreciated.
Sickle cell disease, sometimes known as sickle cell anemia, is an inherited red blood cell disorder that affects more than 12 million people, mostly from sub-Saharan Africa, Asia, Saudi Arabia, and the Mediterranean. The condition is known to be a silent baby killer because an estimated 50% to 80% of babies born with the illness die before age 5. About 300,000 children were born with severe sickle cell disorders each year, including more than 200,000 cases in Africa, according to the WHO.
Though, a healthcare worker would also have to administer up to four vials every month to each patient as an infusion to achieve these best-case outcomes from the new drug, Novartis says. It contrasts sharply with the hydroxyurea available in tablets and does not include hospital visits that would result in additional costs for patients.
But, while sub-Saharan Africa accounts for the greatest burden of disease and, according to the WHO, sickle cell disease affects most countries in the African region, it is unlikely that underfunded health systems in most countries in the region will be able to subsidize the cost of the drug to patients.
Out-of-pocket hegemony as the main payment method for healthcare services in African countries means that the medicines will be out of reach for the majority of people who most need it.
Despite African markets ‘ numerous challenges, Novartis informed Quartz that if approved, Adakveo can play an important role in minimizing the burden of Africa’s sickle cell pain crisis and is still in talks with pricing authorities.