WWhen Eli Lilly announced it was phasing out quinidine in 2017, it was the only drug approved for the treatment of severe malaria in the United States.
As supplies of quinidine declined, the Centers for Disease Control and Prevention provided artesunate, the top drug that is the international gold standard for treating severe malaria, free of charge upon request. But this process created problems.
Because artesunate had not yet been approved by the FDA, patients seeking treatment for severe malaria had to sign long and convoluted consent forms to “research use” the drug. It also had to be shipped directly from a limited number of CDC quarantine stations, resulting in fatal delays in relief.
In May 2020, the FDA approved a form of artesunate manufactured by Amivas LLC. World health and travel medicine experts have been thrilled that this excellent severe malaria drug, which has been available in other countries for years, is finally available in the United States. However, this approval was accompanied by a 150-fold jump in price: Artesunat costs about $ 30,000. for the average course of treatment for an adult compared to less than $ 200 for quinidine in 2018.
In most African countries, treatment with artesunate costs $ 5 or less, highlighting the pitfalls of the American drug market.
Artesunate made outside the US cannot be imported here because it does not currently meet FDA requirements. Amivas attributed the set price to the FDA’s licensing and manufacturing costs, coupled with the small US artesunate market. Only about 300 people develop severe malaria in the United States each year, but without prompt, effective treatment, the disease can be fatal.
This is a perfect example of why Congress and the FDA need to address market failures for so-called rare diseases, including severe malaria and many other neglected tropical infections such as intestinal worms, leprosy, or parasitic brain infections such as neurocysticercosis. , toxoplasmosis and meningitis from rat lungworm. Together, these infections affect thousands of Americans.
It is time to prioritize legislative and policy approaches that ensure affordable, timely, and reliable access to medicines for rare diseases in the United States. Medicines deemed essential by the World Health Organization, such as artesunate, should be at the top of the list. FDA approval of imported drugs from qualified suppliers may be one way to address this problem.
Meanwhile, given the high risk of critical illness and death from severe malaria, the price and availability of artesunate are urgent.
The financial crisis that health systems faced during the pandemic and the associated tight budget for hospitals mean that medicines for unusual diseases like malaria may not be stocked in most hospitals. When purchased after a case has been diagnosed, due to limited availability, it takes distributors from 12 hours – at best – up to five days to deliver the drug, a dangerous delay in care.
The US government should consider mechanisms to cover the cost of artesunate drugs for hospitals, for example, through support for the current CDC allocation program, direct reimbursement of health care systems, or a partnership with Amivas, the only US manufacturer of artesunate. Another way a manufacturer could provide artesunate at a more reasonable cost is by providing the FDA with a priority validation voucher for orphan drugs. This program aims to encourage the launch of drugs for underserved individuals, such as artesunate, on the market by offsetting entry costs. Amivas’ sale of a voucher valued in the millions could enable him to sell artesunate at a lower price.
It is shocking to think that US citizens can get better treatment for severe malaria at a lower cost in a low-income country than at home. Even more shocking is the idea that some people with severe malaria in the United States may not be able to get the treatment they need, given the cost and barriers that prevent doctors from getting the only FDA-approved therapy.
We urge legislators and the FDA to consider ways to ensure that artesunate and other rare disease drugs are available in a timely and affordable manner to everyone in the United States who needs it.
Ann E.P. Frosch is an infectious disease physician at Hennepin Healthcare and an assistant professor of medicine at the University of Minnesota. Eileen Ahiskali is an infectious disease pharmacist at Hennepin Healthcare. Chandi S. John is a pediatric infectious disease physician and director of the Center for Pediatric Infectious Diseases and Global Health. Ryan White and Professor of Pediatrics and Medicine at Indiana University School of Medicine.